Verastem Inc (VSTM)

VS-7375 Demonstrates Clinical Activity with a Favorable Safety and Tolerability Profile in TARGET-D 101 Phase 1/2 Clinical Trial in Patients with Advanced KRAS G12D-Mutated Solid TumorsJune 23, 2026 4:01 PM
Business Wire Broad anti-tumor activity observed with VS-7375 across dose levels in multiple solid tumors, including pancreatic, colorectal, and lung cancers Early evidence of anti-tumor activity with either anti-EGFR therapy or standard-of-care chemotherapy supports broad development strategy including combinations Favorable safety and tolerability profile characterized by predominately low-grade gastrointestinal adverse events that attenuate after cycle 1; favorable tolerability in combination regimens Verastem and Erasca announce their intent to evaluate VS-7375 in combination with ERAS-0015, Erasca’s investigational pan-RAS molecular glue, in advanced KRAS G12D-mutant solid tumors Company to host conference call and webcast today at 4:30 p.m. ET Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced positive preliminary data from the ongoing TARGET-D 101 Phase 1/2 clinical trial evaluating VS-7375, an investigational oral KRAS G12D (ON/OFF) inhibitor, with best-in-class potential, in patients with advanced KRAS G12D-mutated solid tumors. The data demonstrate encouraging clinical activity along with a favorable safety and tolerability profile across multiple dose levels and tumor types, including metastatic pancreatic ductal carcinoma (mPDAC), metastatic colorectal cancer (mCRC), and advanced non-small cell lung cancer (NSCLC). “VS-7375 has demonstrated anti-tumor activity across multiple dose levels and tumor types, encouraging signals from rational combination strategies, and a favorable safety profile that improves meaningfully beyond the first treatment cycle, underscoring its potential to be not only the best-in-class oral KRAS G12D inhibitor, but also the preferred treatment option for patients with KRAS-G12D-mutated cancers,” said Michael Kauffman, M.D., Ph.D., president of development at Verastem Oncology. “Importantly, VS-7375 has demonstrated compatibility with both anti-EGFR therapy and standard-of-care chemotherapy, supporting the broad development strategy we are pursuing across pancreatic, non-small cell lung, and colorectal cancers. As patient follow-up matures in the TARGET-D 101 study, we are enrolling patients in our three Phase 2 registration-directed studies. We look forward to sharing additional data on VS-7375 in patients with KRAS G12D-mutated cancers later this year." Highlights of TARGET-D 101 Phase 1/2 Dose Escalation & Dose Expansion Trial
In the TARGET-D 101 trial, dose-escalation is ongoing at 1200 mg once daily (QD). In updated pharmacokinetic (PK) data, the 900 mg QD dose continues to achieve target plasma levels of VS-7375 and provides clear separation from the 600 mg QD dose. VS-7375 demonstrated anti-tumor activity at multiple dose levels, including 400 mg QD, 600 mg QD and 900 mg QD both as monotherapy and in combination with anti-EGFR therapy, across multiple KRAS G12D-driven tumors, including mPDAC, mCRC and advanced NSCLC. In addition, patient follow-up continues to mature across both monotherapy and combination cohorts. Metastatic PDAC Promising clinical activity observed at 900 mg QD monotherapy in previously treated mPDAC, with evidence of dose-dependent anti-tumor activity between 600 mg QD and 900 mg QD 93% (13/14) of heavily pretreated (2L-4L) patients with mPDAC receiving 900 mg QD monotherapy achieved greater than 50% reduction in the tumor marker CA19-9. All 14 evaluable patients had elevated baseline CA19-9 levels (>37 U/mL) and at least one scheduled on-treatment CA19-9 assessment. All patients remain on treatment. Preliminary data suggest the combination with the anti-EGFR antibody cetuximab is associated with deeper and more rapid tumor reductions, even at a subtherapeutic VS-7375 dose of 400 mg QD. Combination cohorts in previously treated mPDAC demonstrate good combinability with standard-of-care chemotherapy, gemcitabine plus Nab-paclitaxel (Gem/NabP). VS-7375 600 mg QD in combination with full-dose Gem/NabP has been DLT-cleared, and enrollment is ongoing with 900 mg QD plus full-dose Gem/NabP. Among patients with mPDAC who had received at least one prior therapy (2L+), 7 of 20+ patients enrolled at the 600 mg QD dose level and 1 of 20+ patients enrolled at the 900 mg QD dose level had completed at least six months of follow-up. Metastatic CRC In the mCRC cohort, promising preliminary efficacy was observed with full dose cetuximab at both the 600 mg QD and 900 mg QD dose levels of VS-7375. VS-7375 900 mg QD in combination with full dose cetuximab was DLT-cleared in May 2026, with no overlapping toxicities observed to date. Additional patients will be enrolled at this dose level in the TARGET-D 203 Phase 2 registration-directed mCRC trial. Follow-up remains early in the mCRC cohort, with no patients out of 20+ at 600 mg QD in combination with full dose cetuximab having more than six months of follow-up. Advanced NSCLC In the advanced NSCLC cohort, promising preliminary efficacy was observed at 600 mg QD monotherapy. Follow-up remains early in the NSCLC cohort, with only one out of 20+ patients at 600 mg QD having more than six months of follow-up. The 900 mg QD dose level in advanced NSCLC will be studied in the registration-directed TARGET-D 202 Phase 2 study. Updated Safety & Tolerability from Phase 1/2 TARGET-D 101
Across monotherapy and combination cohorts in TARGET-D 101, VS-7375 continued to demonstrate a favorable and manageable safety profile, consistent with prior observations and supported by increasing patient exposure and longer follow-up. As of the June 12, 2026 data cutoff, VS-7375 monotherapy has demonstrated a favorable and manageable safety profile at both the 600 mg QD (n=57) and 900 mg QD (n=25) dose levels. Treatment-related adverse events (TRAEs) were primarily low-grade nausea, vomiting and diarrhea, which generally diminished over time, with substantially reduced incidence after cycle 1 dosing. The vast majority of the gastrointestinal (GI) side effects were effectively managed with standard supportive care measures, with only 1 reported Grade 3 case of nausea at the 900 mg QD dose that resolved in 4 days after optimization of anti-emetic agents. A very low frequency of rash was observed in either the 600 mg QD or 900 mg QD dose level and no rash above Grade 1. TRAEs occurring in more than one patient were largely confined to the first treatment cycle and attenuated substantially thereafter among patients with at least 29 days of follow-up receiving VS-7375 at both the 600 mg QD (n=51) and 900 mg QD (n=22) dose levels. No unexpected adverse events (AEs) were observed, and rates of Grade 3 AEs remained low. Importantly, no clinically meaningful cytopenias or liver function abnormalities were reported at either the 600 mg QD or 900 mg QD dose level. The limited dose-response relationship observed for gastrointestinal AEs is consistent with a localized irritant effect rather than systemic toxicity. Emerging longer-term follow-up data are encouraging, with no clinically significant cumulative toxicities observed to date. VS-7375 Development Collaboration
Verastem and Erasca, Inc., announced today their intent to enter into an agreement to evaluate VS-7375, Verastem’s potential best-in-class oral KRAS G12D (ON/OFF) inhibitor, in combination with ERAS-0015, Erasca’s potential best-in-class oral pan-RAS molecular glue, across KRAS G12D mutant solid tumor models. Subject to the execution of a definitive agreement and the outcome of the preclinical evaluation, the Companies intend to explore a future clinical trial collaboration to evaluate the combination in patients with advanced solid tumors. Additional details regarding the potential collaboration will be announced at a later date. “In the first half of this year we have made tremendous progress in advancing the development of VS-7375 in order to bring this truly differentiated KRAS G12D inhibitor with promising emerging clinical efficacy and a favorable safety and tolerability profile both as monotherapy and in combination regimens as quickly as possible to patients,” said Dan Paterson, president and chief executive officer of Verastem Oncology. “The momentum behind the VS-7375 program continues to accelerate with the initiation of three Phase 2 registration-directed trials in only three months. We are now expanding the development strategy through a collaboration designed to explore a complementary mechanism and address areas of significant unmet need within KRAS G12D-mutated cancers. Overall, the development strategy for VS-7375 is aimed at maximizing the therapeutic potential of this program across multiple tumor types and treatment settings and supporting multiple potential registration pathways.” Expected Key Milestones: Report an update on the TARGET-D 101 trial in 2H 2026. Complete target enrollment in TARGET-D 101 PDAC and NSCLC monotherapy cohorts and mCRC cetuximab combination cohorts by the end of June 2026. Announce first patient dosed in the TARGET-D 202 and TARGET-D 203 clinical trials in mid-2026. Complete enrollment across all three TARGET-D Phase 2 trials by the end of 2026. Meet with the U.S. Food and Drug Administration (FDA) before the end of the year to review Phase 3 pivotal trial designs in 1L mPDAC, 1L mCRC and 1L advanced NSCLC. Enroll the first patient in each of the Phase 3 pivotal trials in the first half of 2027. Webcast Information
On June 23rd at 4:30 p.m. ET, a live audio webcast of the call, along with accompanying slides, will be available under "Events & Presentations" in the Investor section of the Company's website, https://investor.verastem.com/events. A replay of the webcast will be archived and available following the event. About KRAS G12D
KRAS G12D represents 26% of all KRAS mutations, making it the most prevalent KRAS mutation in human cancers. When the KRAS gene is mutated, it can promote cancer development and growth. Patients with KRAS G12D-mutant tumors often have poorer outcomes, underscoring the need for therapies designed specifically to inhibit this mutation potently and for a long duration. The KRAS G12D mutation occurs most commonly in pancreatic (40%), colorectal (15%), endometrial (8%), biliary tract (7-15%), and non-small cell lung (5%) cancers. Currently, no therapies are approved by the U.S. Food and Drug Administration (FDA) specifically targeting KRAS G12D mutations in cancer. About VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor & TARGET-D Clinical Program
VS-7375 is a potential best-in-class, potent, and selective investigational oral KRAS G12D dual ON/OFF inhibitor. It is designed to uniquely bind to both the active (ON) and inactive (OFF) states of KRAS G12D, with the potential to inhibit KRAS G12D signaling and tumor growth more completely than compounds that block KRAS G12D only in the OFF state or only in the ON state. In June 2025, Verastem initiated TARGET-D 101, a Phase 1/2 dose escalation, dose expansion, and combination clinical trial evaluating the safety and efficacy of VS-7375 in patients with advanced KRAS G12D mutant solid tumors. Verastem has further expanded the VS-7375 clinical program with the initiation of three Phase 2 registration-directed, open-label clinical trials: TARGET-D 201 (NCT07644559) in second-line advanced or metastatic pancreatic ductal carcinoma, TARGET-D 202 (NCT07659782) in second/third-line advanced or metastatic non-small cell lung cancer, and TARGET-D 203 (NCT07659795) in metastatic colorectal cancer. On June 16, 2026, the first patient was dosed in the TARGET-D 201 trial. In July 2025, U.S. Food and Drug Administration (FDA) granted Fast Track Designation (FTD) to VS-7375 for the first-line treatment of patients with KRAS G12D-mutated locally advanced or metastatic adenocarcinoma of the pancreas and for the treatment of patients with KRAS G12D-mutated locally advanced or metastatic pancreatic ductal carcinoma who have received at least one prior line of standard systemic therapy. In June 2026, the FDA also granted FTD to VS-7375 for the treatment of adult patients with KRAS G12D-mutated unresectable locally advanced or metastatic non-small cell lung cancer (NSCLC) who have received platinum-based chemotherapy and an anti-PD-(L)1 antibody either concurrently or sequentially. In December 2023, Verastem selected VS-7375 as its lead program from its collaboration with GenFleet Therapeutics, which aims to advance three oncology discovery programs related to RAS/MAPK pathway-driven cancers. The collaboration provides Verastem with an exclusive option to obtain a license for each of the three compounds in the collaboration after the successful completion of pre-determined milestones in a Phase 1 trial. In January 2025, Verastem exercised its license for VS-7375. The licenses would give Verastem development and commercialization rights outside the GenFleet markets of mainland China, Hong Kong, Macau, and Taiwan. GenFleet is developing VS-7375 as GFH375 in China. About Verastem Oncology
Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI® FAKZYNJA® CO-PACK in the U.S. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn. Forward-Looking Statements
This press release includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as “anticipate,” “expect,” “plan,” “could,” “may,” “believe,” “estimate,” “forecast,” “goal,” “potential,” “project,” and other words of similar meaning. Such forward-looking statements address various matters about, among other things, Verastem Oncology’s programs and product candidates, strategy, future plans and prospects, including statements related to the potential for and timing of commercialization of product candidates, the anticipated timing for any amendments to the IND application for VS-7375/GFH375, the expected outcome and benefits of the Company’s collaboration with GenFleet Therapeutics (Shanghai), Inc., the timing of commencing and completing trials and compiling data, the expected timing of the presentation of data by the Company and the potential clinical value of various of the Company’s clinical trials. Each forward-looking statement contained in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others: the uncertainties inherent in research and development, such as the possibility of negative or unexpected results of clinical trials; that we may not see a return on investment on the payments we have and may continue to make pursuant to the collaboration and option agreement with GenFleet, or that GenFleet may fail to fully perform under the agreement; that we may not be successful in our continued commercialization of AVMAPKI FAKZYNJA CO-PACK; that the development and commercialization of our product candidates may take longer or cost more than planned, including as a result of conducting additional studies or our decisions regarding execution of such commercialization; that data may not be available when expected; that preclinical studies and any positive preliminary, initial “top-line,” and interim data, from our clinical trials of our product candidates may not necessarily be predictive of the results of ongoing or later clinical trials; risks associated with the regulatory and policy actions proposed and enacted by the current U.S. presidential administration that may adversely affect our business; risks associated with the current administration’s reductions to the FDA’s workforce and any subsequent reductions that may lead to disruptions and delays in the FDA’s review and oversight of our product candidates and impact the FDA’s ability to provide timely feedback on our development programs; that our product candidates may not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients; and the risks identified under the heading "Risk Factors" as detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, as filed with the Securities and Exchange Commission (SEC) on March 4, 2026, as well as the other information we file with the SEC, are possibly realized. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at www.sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of these statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties. View source version on businesswire.com: https://www.businesswire.com/news/home/20260623312556/en/ For Investor and Media Inquiries:
Julissa Viana
Senior Vice President, Corporate Communications,
Investor Relations & Patient Advocacy
investors@verastem.com or
media@verastem.com Original: VS-7375 Demonstrates Clinical Activity with a Favorable Safety and Tolerability Profile in TARGET-D 101 Phase 1/2 Clinical Trial in Patients with Advanced KRAS G12D-Mutated Solid Tumors

Verastem Oncology Announces Positive Updated Results from RAMP 205 Evaluating Avutometinib Plus Defactinib in Combination with Standard-of-Care Chemotherapy in First-Line Metastatic Pancreatic CancerJune 17, 2026 7:30 AM
Business Wire 90% of patients in the study presented with metastatic (Stage IV) disease at diagnosis 6-month overall survival rate was 86%; follow-up continues and survival data continue to mature Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced positive updated safety and efficacy results from the RAMP 205 Phase 1b/2a Recommended Phase 2 Dose (RP2D) cohort of 29 patients evaluating avutometinib plus defactinib in combination with gemcitabine and nab-paclitaxel in first-line metastatic pancreatic ductal adenocarcinoma (PDAC). KRAS is mutated in more than 90% of pancreatic cancers, making it a key driver of tumor growth. The RAMP 205 trial was designed to evaluate whether simultaneous inhibition of KRAS-driven signaling and FAK-mediated resistance pathways, in combination with standard-of-care chemotherapy, could improve outcomes for patients living with metastatic pancreatic cancer. “The updated data from the RAMP 205 trial provide important clinical insights into the potential impact of combined RAF/MEK and FAK inhibition as a therapeutic option for pancreatic cancer. While pancreatic cancer remains one of the most challenging cancers to treat, the early survival trends and deep responses observed in this study indicate that avutometinib and defactinib are combinable with standard-of-care chemotherapy and may help overcome resistance mechanisms inherent in pancreatic cancer and support further exploration of strategies designed to address oncogenic signaling and mechanisms of treatment resistance,” said John Hayslip, M.D., chief medical officer at Verastem Oncology. “We are grateful to PanCAN, the RAMP 205 investigators, and especially the patients and families who participated in the trial.” In the Phase 1b/2a study, 29 patients were enrolled and treated at the RP2D with avutometinib 2.4 mg twice weekly (BIW), defactinib 200 mg twice daily (BID) for 3 weeks on and one week off, and gemcitabine (800 mg/m2) plus nab-paclitaxel (125 mg/m2) administered on Days 1, 8, and 15 of each 28-day cycle. At diagnosis, 90% of patients presented with metastatic disease. As of the June 5, 2026 data cutoff, with a median follow up of 9.8 months, the combination demonstrated encouraging clinical activity, including an 86% overall survival (OS) rate at 6 months, with the OS data continuing to mature. The progression-free survival (PFS) rate at 6 months was 68%, and the confirmed objective response rate (ORR) was 52% (15/29). At the RP2D dose, the majority (83%) of patients experienced tumor shrinkage. Nine patients remain on treatment at this dose level. Adverse events remained generally consistent with the previously reported safety and tolerability profile, with no new safety signals observed. “For patients and their families facing a pancreatic cancer diagnosis, every advance in research and understanding of the underlying biology driving this cancer matters,” said Anna Berkenblit, M.D., chief scientific and medical officer of the Pancreatic Cancer Action Network (PanCAN). “We awarded Verastem the PanCAN Therapeutic Accelerator Award in 2022 to invest in research and development of novel treatment approaches. The results of the RAMP 205 trial underscore the importance of continued research in the RAS/MAPK-pathway to help improve outcomes for patients living with pancreatic cancer.” “We will continue to evaluate the potential role of avutometinib plus defactinib in metastatic pancreatic cancer, including future development opportunities and potential strategic collaborations, informed by the final overall survival results from the study as well as emerging data from VS-7375, our investigational potential best-in-class oral KRAS G12D (ON/OFF) inhibitor, currently being evaluated in metastatic pancreatic cancer as both a monotherapy and in combination regimens,” said Dan Paterson, president and chief executive officer of Verastem Oncology. “We will also assess opportunities to share these data in the future, including at a medical meeting." In May 2022, Verastem Oncology was selected by PanCAN to receive the inaugural PanCAN Therapeutic Accelerator Award, supporting evaluation of avutometinib in combination with defactinib in front-line metastatic pancreatic cancer. Designed to accelerate the development of new pancreatic cancer treatments, the award provided Verastem with $3.8M following a rigorous, competitive process involving scientific, business, and programmatic review from leading experts in the field. In parallel, a working group led by PanCAN was formed as a partnership between Verastem and the academic community to further understand the science behind and the potential of this investigational treatment combination to improve outcomes for patients. About Metastatic Pancreatic Cancer Pancreatic cancer is the third leading cause of cancer-related death in the U.S. and seventh leading cause of cancer-associated mortality worldwide. Metastatic pancreatic cancer, or stage IV disease, occurs when the cancer spreads beyond the pancreas to distant organs. More than 90% of pancreatic cancers harbor KRAS mutations, underscoring the central role of KRAS in the development and the progression of the disease. Approximately 40% of pancreatic tumors harbor a KRAS G12D mutation, the most prevalent subtype in pancreatic cancer. Patients with KRAS G12D-mutant tumors often have poorer outcomes, underscoring the need for therapies designed specifically to inhibit this mutation potently and for a long duration. Each year, more than 30,000 people in the U.S. and over 240,000 people globally are diagnosed with metastatic pancreatic cancer. There has been minimal progress with treatment, and the five-year survival rate remains approximately 3%. Current treatment approaches may include surgery, chemotherapy, radiation therapy, targeted therapies, or a combination of these modalities. About RAMP 205 Phase 1b/2a Study RAMP 205 is a multicenter, open-label, single arm Phase 1b/2a study to evaluate the safety, tolerability, and efficacy of avutometinib and defactinib in combination with standard of care chemotherapy (gemcitabine and nab-paclitaxel) in patients with previously untreated metastatic pancreatic ductal adenocarcinoma. Part A of the study evaluated varied dose and schedule combinations to determine the recommended Phase 2 dose for expansion into Part B. RAMP 205 is supported by a PanCAN Therapeutic Accelerator Award. About AVMAPKI and FAKZYNJA Combination Therapy AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors. The U.S. Food and Drug Administration (FDA) approved AVMAPKI® FAKZYNJA® CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Verastem is conducting RAMP 301 (GOG-3097/ENGOT-ov81/GTG-UK) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC) with and without a KRAS mutation. Verastem is also evaluating avutometinib plus defactinib with standard-of-care chemotherapy as a potential treatment in the first-line for patients with advanced pancreatic cancer (RAMP 205; NCT05669482). Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on a stand-alone basis for any use. AVMAPKI FAKZYNJA CO-PACK U.S. Indication Indication AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Important Safety Information Warnings and Precautions Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity. Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration. Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality. Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction. Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception. Adverse Reactions The most common (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count. Drug Interactions Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK. Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK. Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin. Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid. Use in Specific Populations Lactation: Advise not to breastfeed. Fertility: May impair fertility in males and females. Click here for full Prescribing Information. About Verastem Oncology Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI® FAKZYNJA® CO-PACK in the U.S. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn. Forward-Looking Statements This press release includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as “anticipate,” “expect,” “plan,” “could,” “may,” “believe,” “estimate,” “forecast,” “goal,” “project,” and other words of similar meaning. Such forward-looking statements address various matters about, among other things, Verastem Oncology’s programs and product candidates, strategy, future plans and prospects, including statements related to the potential for and timing of commercialization of product candidates, the expected outcome and benefits of the Company’s collaboration with GenFleet Therapeutics (Shanghai), Inc., the timing of commencing and completing trials and compiling data, the expected timing of the presentation of data by the Company and the potential clinical value of various of the Company’s clinical trials. Each forward-looking statement contained in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others: the uncertainties inherent in research and development, such as the possibility of negative or unexpected results of clinical trials; that we may not see a return on investment on the payments we have and may continue to make pursuant to the collaboration and option agreement with GenFleet, or that GenFleet may fail to fully perform under the agreement; that we may not be successful in our continued commercialization of AVMAPKI FAKZYNJA CO-PACK; that the development and commercialization of our product candidates may take longer or cost more than planned, including as a result of conducting additional studies or our decisions regarding execution of such commercialization; that data may not be available when expected; risks associated with preliminary and interim data, which may not be representative of more mature data; risks associated with the recent changes in administration policy or actions that may create regulatory uncertainty that may adversely affect our business; risks associated with the current administration’s reductions to the FDA’s workforce and any subsequent reductions that may lead to disruptions and delays in the FDA’s review and oversight of our product candidates and impact the FDA’s ability to provide timely feedback on our development programs; that our product candidates may not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients; and the risks identified under the heading "Risk Factors" as detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, as filed with the Securities and Exchange Commission (SEC) on March 4, 2026, as well as the other information we file with the SEC, are possibly realized. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at www.sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of these statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties. View source version on businesswire.com: https://www.businesswire.com/news/home/20260617680929/en/ For Investor and Media Inquiries:
Julissa Viana
Senior Vice President, Corporate Communications
Investor Relations and Patient Advocacy
investors@verastem.com or
media@verastem.com Original: Verastem Oncology Announces Positive Updated Results from RAMP 205 Evaluating Avutometinib Plus Defactinib in Combination with Standard-of-Care Chemotherapy in First-Line Metastatic Pancreatic Cancer

Verastem Oncology Doses First Patient in TARGET-D 201 Phase 2 Registration-Directed Trial of VS-7375 Oral KRAS G12D (ON/OFF) Inhibitor for KRAS G12D-Mutated Metastatic Pancreatic CancerJune 16, 2026 4:01 PM
Business Wire TARGET-D 201 trial to evaluate VS-7375 both as monotherapy and in combination with cetuximab for second-line treatment of metastatic pancreatic cancer A cohort in TARGET-D 201 will also evaluate the combination of VS-7375 and cetuximab for frontline treatment of metastatic pancreatic cancer VS-7375 previously granted FDA Fast Track designation for KRAS G12D-mutated advanced or metastatic pancreatic cancer KRAS G12D is the most common KRAS mutation in pancreatic cancer, occurring in approximately 40% of patients, and is often associated with poorer prognosis Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced that the first patient has been dosed in the TARGET-D 201 Phase 2 registration-directed trial (NCT07644559) evaluating VS-7375, an investigational oral KRAS G12D (ON/OFF) inhibitor with best-in-class potential, to treat patients with KRAS G12D-mutated metastatic pancreatic ductal carcinoma (mPDAC). “Dosing the first patient in the TARGET-D 201 trial marks an important milestone in the development of VS-7375 for patients with KRAS G12D-mutated metastatic pancreatic cancer, where unmet need remains high,” said Michael Kauffman, M.D., Ph.D., president of development at Verastem Oncology. “We believe VS-7375 has a differentiated profile, including potent KRAS G12D target inhibition and sustained target coverage, supporting its potential to play a significant role in treating multiple KRAS G12D-mutated tumor types. With three registration-directed Phase 2 clinical trials now underway across pancreatic, non-small cell lung, and colorectal cancers, and FDA Fast Track designations in pancreatic and non-small cell lung cancers, we are focused on accelerating development of VS-7375 as quickly as possible across multiple tumor types where no approved treatments exist.” TARGET-D 201 is a Phase 2, open-label, multi-center study to evaluate VS-7375 at 900 mg once daily (QD) both as monotherapy and in combination with full-dose cetuximab in patients with second-line metastatic pancreatic ductal carcinoma. The study is also evaluating VS-7375 900 mg and cetuximab in the first-line mPDAC setting, representing a potential chemotherapy-free frontline regimen. FDA Fast Track Designation was previously granted for VS-7375 for the treatment of KRAS G12D-mutated advanced or metastatic pancreatic cancer in both the first- and second-line treatment settings. In June 2025, Verastem initiated TARGET-D 101, its Phase 1/2 dose escalation, dose expansion, and combination clinical trial evaluating VS-7375 in patients with advanced KRAS G12D-mutated pancreatic, non-small cell lung, colorectal, and other solid tumor cancers. Enrollment is ongoing with monotherapy dose escalation progressing from 400 mg through 900 mg QD doses without dose-limiting toxicities or major safety concerns observed to date. The study is currently evaluating a 1200 mg QD monotherapy dose and a 900 mg QD dose of VS-7375 in combination with full-dose cetuximab, as well as combinations with either gemcitabine plus nab-paclitaxel or carboplatin/pemetrexed/pembrolizumab. About Pancreatic Cancer
Pancreatic cancer is the third leading cause of cancer-related death in the U.S. and seventh leading cause of cancer-associated mortality worldwide. Metastatic pancreatic cancer, or stage IV disease, occurs when the cancer spreads beyond the pancreas to distant organs. More than 90% of pancreatic cancers harbor KRAS mutations, underscoring the central role of KRAS in the development and the progression of the disease. Approximately 40% of pancreatic tumors harbor a KRAS G12D mutation, the most prevalent subtype in pancreatic cancer. Each year, more than 30,000 people in the U.S. and over 240,000 people globally are diagnosed with metastatic pancreatic cancer. There has been minimal progress with treatment, and the five-year survival rate remains approximately 3%. Current treatment approaches may include surgery, chemotherapy, radiation therapy, targeted therapies, or a combination of these modalities. About KRAS G12D
KRAS G12D represents 26% of all KRAS mutations, making it the most prevalent KRAS mutation in human cancers. When the KRAS gene is mutated, it can promote cancer development and growth. Patients with KRAS G12D-mutant tumors often have poorer outcomes, underscoring the need for therapies designed specifically to inhibit this mutation potently and for a long duration. The KRAS G12D mutation occurs most commonly in pancreatic (40%), colorectal (15%), endometrial (8%), biliary tract (7-15%), and non-small cell lung (5%) cancers. Currently, no therapies are approved by the U.S. Food and Drug Administration specifically targeting KRAS G12D mutations in cancer. About VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor & the TARGET-D Clinical Program
VS-7375 is a potential best-in-class, potent, and selective investigational oral KRAS G12D dual ON/OFF inhibitor. It is designed to uniquely bind to both the active (ON) and inactive (OFF) states of KRAS G12D, with the potential to inhibit KRAS G12D signaling and tumor growth more completely than compounds that block KRAS G12D only in the OFF state or only in the ON state. In June 2025, Verastem initiated TARGET-D 101, a Phase 1/2 dose escalation, dose expansion, and combination clinical trial evaluating the safety and efficacy of VS-7375 in patients with advanced KRAS G12D mutant solid tumors. Verastem has further expanded the VS-7375 clinical program with the initiation of three Phase 2 registration-directed, open-label clinical trials: TARGET-D 201 (NCT07644559) in metastatic pancreatic ductal carcinoma, TARGET-D 202 in advanced non-small cell lung cancer, and TARGET-D 203 in metastatic colorectal cancer. In July 2025, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation (FTD) to VS-7375 for the first-line treatment of patients with KRAS G12D-mutated locally advanced or metastatic adenocarcinoma of the pancreas and for the treatment of patients with KRAS G12D-mutated locally advanced or metastatic pancreatic ductal carcinoma who have received at least one prior line of standard systemic therapy. In June 2026, the FDA also granted FTD to VS-7375 for the treatment of adult patients with KRAS G12D-mutated unresectable locally advanced or metastatic non-small cell lung cancer (NSCLC) who have received platinum-based chemotherapy and an anti-PD-(L)1 antibody either concurrently or sequentially. In December 2023, Verastem selected VS-7375 as its lead program from its collaboration with GenFleet Therapeutics, which aims to advance three oncology discovery programs related to RAS/MAPK pathway-driven cancers. The collaboration provides Verastem with an exclusive option to obtain a license for each of the three compounds in the collaboration after the successful completion of pre-determined milestones in a Phase 1 trial. In January 2025, Verastem exercised its license for VS-7375. The licenses would give Verastem development and commercialization rights outside the GenFleet markets of mainland China, Hong Kong, Macau, and Taiwan. GenFleet is developing VS-7375 as GFH375 in China. About Verastem Oncology
Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI® FAKZYNJA® CO-PACK in the U.S. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn. Forward-Looking Statements
This press release includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as “anticipate,” “expect,” “plan,” “could,” “may,” “believe,” “estimate,” “forecast,” “goal,” “project,” and other words of similar meaning. Such forward-looking statements address various matters about, among other things, Verastem Oncology’s programs and product candidates, strategy, future plans and prospects, including statements related to the potential for and timing of commercialization of product candidates, the anticipated timing for the IND application for VS-7375/GFH375, the expected outcome and benefits of the Company’s collaboration with GenFleet Therapeutics (Shanghai), Inc., the timing of commencing and completing trials and compiling data, the expected timing of the presentation of data by the Company and the potential clinical value of various of the Company’s clinical trials. Each forward-looking statement contained in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others: the uncertainties inherent in research and development, such as the possibility of negative or unexpected results of clinical trials; that we may not see a return on investment on the payments we have and may continue to make pursuant to the collaboration and option agreement with GenFleet, or that GenFleet may fail to fully perform under the agreement; that we may not be successful in our continued commercialization of AVMAPKI FAKZYNJA CO-PACK; that the development and commercialization of our product candidates may take longer or cost more than planned, including as a result of conducting additional studies or our decisions regarding execution of such commercialization; that data may not be available when expected; risks associated with preliminary and interim data, which may not be representative of more mature data; risks associated with the regulatory and policy actions proposed and enacted by the current U.S. presidential administration that may adversely affect our business; risks associated with the current administration’s reductions to the FDA’s workforce and any subsequent reductions that may lead to disruptions and delays in the FDA’s review and oversight of our product candidates and impact the FDA’s ability to provide timely feedback on our development programs; that our product candidates may not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients; and the risks identified under the heading "Risk Factors" as detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, as filed with the Securities and Exchange Commission (SEC) on March 4, 2026, as well as the other information we file with the SEC, are possibly realized. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at www.sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of these statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties. View source version on businesswire.com: https://www.businesswire.com/news/home/20260616690184/en/ For Investor and Media Inquiries:
Julissa Viana
Senior Vice President, Corporate Communications,
Investor Relations & Patient Advocacy
investors@verastem.com or
media@verastem.com Original: Verastem Oncology Doses First Patient in TARGET-D 201 Phase 2 Registration-Directed Trial of VS-7375 Oral KRAS G12D (ON/OFF) Inhibitor for KRAS G12D-Mutated Metastatic Pancreatic Cancer

Verastem Oncology to Host Investor Conference Call and Report Updated Data and Progress Across VS-7375 Oral KRAS G12D (ON/OFF) Inhibitor TARGET-D Clinical ProgramJune 15, 2026 4:01 PM
Business Wire Featuring updated preliminary clinical data across tumor types from the Phase 1/2 TARGET-D 101 trial Company to host investor conference call and webcast on Tuesday, June 23, 2026, at 4:30 pm ET Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced it will host an investor conference call and webcast on Tuesday, June 23 at 4:30 pm ET to report progress across the VS-7375 oral KRAS G12D (ON/OFF) TARGET-D clinical program and updated preliminary data from the Phase 1/2 TARGET-D 101 dose escalation and dose expansion trial evaluating VS-7375 alone and in combination in patients with KRAS G12D-mutated advanced solid tumors. During the call, Verastem’s management team will present preliminary data from the TARGET-D 101 dose-escalation and dose expansion cohorts across pancreatic, colorectal, and non-small lung cancers, including updated safety and tolerability, pharmacokinetic findings, as well as patient case studies evaluating VS-7375 both as a monotherapy and in combination across multiple tumor types. Webcast Information
On June 23rd at 4:30 p.m. ET, a live audio webcast of the call, along with accompanying slides, will be available under "Events & Presentations" in the Investor section of the Company's website, https://investor.verastem.com/events. A replay of the webcast will be archived and available following the event. About VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor & TARGET-D Clinical Program
VS-7375 is a potential best-in-class, potent, and selective investigational oral KRAS G12D dual ON/OFF inhibitor. It is designed to uniquely bind to both the active (ON) and inactive (OFF) states of KRAS G12D, with the potential to inhibit KRAS G12D signaling and tumor growth more completely than compounds that block KRAS G12D only in the OFF state or only in the ON state. In June 2025, Verastem initiated TARGET-D 101, a Phase 1/2 dose escalation, dose expansion, and combination clinical trial evaluating the safety and efficacy of VS-7375 in patients with advanced KRAS G12D mutant solid tumors. Verastem has further expanded the VS-7375 clinical program with the initiation of three Phase 2 registration-directed, open-label clinical trials: TARGET-D 201 (NCT07644559) in second-line advanced or metastatic pancreatic ductal carcinoma, TARGET-D 202 in second/third-line advanced or metastatic non-small cell lung cancer, and TARGET-D 203 in metastatic colorectal cancer. In July 2025, U.S. Food and Drug Administration (FDA) granted Fast Track Designation (FTD) to VS-7375 for the first-line treatment of patients with KRAS G12D-mutated locally advanced or metastatic adenocarcinoma of the pancreas and for the treatment of patients with KRAS G12D-mutated locally advanced or metastatic pancreatic ductal carcinoma who have received at least one prior line of standard systemic therapy. In June 2026, the FDA also granted FTD to VS-7375 for the treatment of adult patients with KRAS G12D-mutated unresectable locally advanced or metastatic non-small cell lung cancer (NSCLC) who have received platinum-based chemotherapy and an anti-PD-(L)1 antibody either concurrently or sequentially. In December 2023, Verastem selected VS-7375 as its lead program from its collaboration with GenFleet Therapeutics, which aims to advance three oncology discovery programs related to RAS/MAPK pathway-driven cancers. The collaboration provides Verastem with an exclusive option to obtain a license for each of the three compounds in the collaboration after the successful completion of pre-determined milestones in a Phase 1 trial. In January 2025, Verastem exercised its license for VS-7375. The licenses would give Verastem development and commercialization rights outside the GenFleet markets of mainland China, Hong Kong, Macau, and Taiwan. GenFleet is developing VS-7375 as GFH375 in China. About Verastem Oncology
Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI® FAKZYNJA® CO-PACK in the U.S. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn. View source version on businesswire.com: https://www.businesswire.com/news/home/20260615846661/en/ For Investor and Media Inquiries:
Julissa Viana
Senior Vice President, Corporate Communications,
Investor Relations & Patient Advocacy
investors@verastem.com or
media@verastem.com Original: Verastem Oncology to Host Investor Conference Call and Report Updated Data and Progress Across VS-7375 Oral KRAS G12D (ON/OFF) Inhibitor TARGET-D Clinical Program

Verastem Oncology Announces U.S. FDA Fast Track Designation for VS-7375, an Oral and Potential Best-in-Class Investigational KRAS G12D (ON/OFF) Inhibitor for the Treatment of KRAS G12D-Mutated Locally Advanced or Metastatic Non-Small Cell Lung Cancer

June 3, 2026

Link article

$VSTM 🗞️

Verastem Oncology Announces U.S. FDA Fast Track Designation for VS-7375, an Oral and Potential Best-in-Class Investigational KRAS G12D (ON/OFF) Inhibitor for the Treatment of KRAS G12D-Mutated Locally Advanced or Metastatic Non-Small Cell Lung CancerJune 3, 2026 4:01 PM
Business Wire Phase 1 TARGET-D 101 dose escalation, dose expansion, and combination clinical trial ongoing with early data updates expected in the first half of 2026 and more mature data expected in the second half of 2026 Phase 2 registration directed, open label TARGET-D 202 clinical trial initiated for 2L/3L advanced non-small cell lung cancer (NSCLC) FDA Fast Track Designation previously granted for VS-7375 for KRAS G12D-mutated advanced or metastatic pancreatic cancer Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to VS-7375, an oral and potential best-in-class investigational selective KRAS G12D (ON/OFF) inhibitor, for the treatment of adult patients with KRAS G12D-mutated unresectable locally advanced or metastatic non-small cell lung cancer (NSCLC) who have received platinum-based chemotherapy and an anti-PD-(L)1 antibody either concurrently or sequentially. FDA Fast Track Designation was previously granted for VS-7375 for the treatment of KRAS G12D-mutated advanced or metastatic pancreatic cancer. “Non-small cell lung cancer is the most prevalent lung disease with more than 8,000 patients in the U.S. diagnosed each year with KRAS G12D-mutations. Receiving Fast Track designation for VS-7375 reinforces both the significant unmet need and the potential of VS-7375 to improve outcomes for patients with KRAS G12D-mutated lung cancer,” said Michael Kauffman, M.D., Ph.D., president of development at Verastem Oncology. “We are continuing to accelerate the development of VS-7375 across multiple KRAS G12D-driven tumor types, including pancreatic, non-small cell lung, and colorectal cancers to bring a highly targeted, oral treatment option to patients as quickly as possible.” Non-small cell lung cancer (NSCLC) is the most prevalent type of lung cancer, accounting for approximately 80% to 85% of all cases. The majority of patients with NSCLC are diagnosed with advanced-stage disease, either locally advanced (30% to 35%) or metastatic (50% to 55%), which is inoperable. The KRAS G12D mutation occurs in five percent of NSCLC cases and patients with these mutations are known to have reduced responses to standard therapies and poor prognosis. Currently, no therapies are approved by the FDA specifically targeting KRAS G12D mutations in cancer. TARGET-D 202 is a Phase 2, open-label, multi-center study to evaluate VS-7375 900 mg oral dose once daily (QD) in patients with advanced NSCLC who have received one to two prior lines of therapy. Based on positive data in pre-clinical intracranial tumor models, the study is also evaluating VS-7375 in NSCLC patients with asymptomatic untreated brain metastases. In June 2025, Verastem initiated TARGET-D 101, its Phase 1/2 dose escalation, dose expansion and combination clinical trial evaluating VS-7375 in patients with advanced KRAS G12D-mutated solid tumors, including pancreatic, non-small cell lung, and colorectal cancers. Enrollment is ongoing with monotherapy dose escalation progressing from 400 mg through 900 mg once daily (QD) doses without dose-limiting toxicities or major safety concerns observed to date. The study is currently evaluating a 1200 mg QD monotherapy dose and a 900 mg QD cetuximab combination cohort. In the ongoing TARGET-D 101 study, data reported in March 2026 from 23 patients treated across the 400 mg, 600 mg and 900 mg QD dose levels, with a mean treatment duration of 1.6 months (range 0.7–5.6), showed VS-7375 was generally well tolerated, with no drug-related liver function test abnormalities or Grade >2 neutropenia observed as of the January 30, 2026 data cutoff, and lower reported rates of nausea, vomiting and diarrhea compared with data previously reported in China. Following feedback from the FDA, Verastem has further expanded the VS-7375 clinical program with the initiation of three Phase 2 registration-directed trials: TARGET-D 201 in metastatic pancreatic ductal adenocarcinoma, TARGET-D 202 in advanced non-small cell lung cancer, and TARGET-D 203 in metastatic colorectal cancer. About KRAS G12D KRAS G12D represents 26% of all KRAS mutations, making it the most prevalent KRAS mutation in human cancers. The KRAS G12D mutation occurs most commonly in pancreatic (37%), colorectal (12.5%), endometrial (8%), biliary tract (7-15%), and non-small cell lung (5%) cancers. Currently, no therapies are approved by the U.S. Food and Drug Administration (FDA) specifically targeting KRAS G12D mutations in cancer. About VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor VS-7375 is a potential best-in-class, potent, and selective oral KRAS G12D dual ON/OFF inhibitor. The mechanism of action of VS-7375 is unique in that it binds the active (ON) and inactive (OFF) states of KRAS G12D, with the potential to inhibit KRAS G12D signaling and tumor growth more completely than compounds that block KRAS G12D only in the OFF state or only in the ON state. In June 2025, Verastem initiated TARGET-D 101, a Phase 1/2 dose escalation, dose expansion, and combination clinical trial evaluating the safety and efficacy of VS-7375 in patients with advanced KRAS G12D mutant solid tumors. Verastem has further expanded the VS-7375 clinical program with the initiation of three Phase 2 registration-directed, open-label clinical trials: TARGET-D 201 in second-line advanced or metastatic pancreatic ductal carcinoma, TARGET-D 202 in second/third-line advanced or metastatic non-small cell lung cancer, and TARGET-D 203 in metastatic colorectal cancer. In July 2025, U.S. Food and Drug Administration (FDA) granted Fast Track Designation (FTD) to VS-7375 for the first-line treatment of patients with KRAS G12D-mutated locally advanced or metastatic adenocarcinoma of the pancreas and for the treatment of patients with KRAS G12D-mutated locally advanced or metastatic pancreatic ductal carcinoma who have received at least one prior line of standard systemic therapy. In December 2023, Verastem selected VS-7375 as its lead program from its collaboration with GenFleet Therapeutics, which aims to advance three oncology discovery programs related to RAS/MAPK pathway-driven cancers. The collaboration provides Verastem with an exclusive option to obtain a license for each of the three compounds in the collaboration after the successful completion of pre-determined milestones in a Phase 1 trial. In January 2025, Verastem exercised its license for VS-7375. The licenses would give Verastem development and commercialization rights outside the GenFleet markets of mainland China, Hong Kong, Macau, and Taiwan. GenFleet is developing VS-7375 as GFH375 in China. About Verastem Oncology Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI® FAKZYNJA® CO-PACK in the U.S. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn. Forward-Looking Statements This press release includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as “anticipate,” “expect,” “plan,” “could,” “may,” “believe,” “estimate,” “forecast,” “goal,” “project,” and other words of similar meaning. Such forward-looking statements address various matters about, among other things, Verastem Oncology’s programs and product candidates, strategy, future plans and prospects, including statements related to the potential for and timing of commercialization of product candidates, the expected outcome and benefits of the Company’s collaboration with GenFleet Therapeutics (Shanghai), Inc., the timing of commencing and completing trials and compiling data, the expected timing of the presentation of data by the Company and the potential clinical value of various of the Company’s clinical trials. Each forward-looking statement contained in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others: the uncertainties inherent in research and development, such as the possibility of negative or unexpected results of clinical trials; that we may not see a return on investment on the payments we have and may continue to make pursuant to the collaboration and option agreement with GenFleet, or that GenFleet may fail to fully perform under the agreement; that we may not be successful in our continued commercialization of AVMAPKI FAKZYNJA CO-PACK; that the development and commercialization of our product candidates may take longer or cost more than planned, including as a result of conducting additional studies or our decisions regarding execution of such commercialization; that data may not be available when expected; risks associated with preliminary and interim data, which may not be representative of more mature data; risks associated with the regulatory and policy actions proposed and enacted by the current U.S. presidential administration that may adversely affect our business; risks associated with the current administration’s reductions to the FDA’s workforce and any subsequent reductions that may lead to disruptions and delays in the FDA’s review and oversight of our product candidates and impact the FDA’s ability to provide timely feedback on our development programs; that our product candidates may not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients; and the risks identified under the heading "Risk Factors" as detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, as filed with the Securities and Exchange Commission (SEC) on March 4, 2026, as well as the other information we file with the SEC, are possibly realized. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at www.sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of these statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties. View source version on businesswire.com: https://www.businesswire.com/news/home/20260603521165/en/ For Investor and Media Inquiries:
Julissa Viana
Senior Vice President, Corporate Communications,
Investor Relations & Patient Advocacy
investors@verastem.com or
media@verastem.com Original: Verastem Oncology Announces U.S. FDA Fast Track Designation for VS-7375, an Oral and Potential Best-in-Class Investigational KRAS G12D (ON/OFF) Inhibitor for the Treatment of KRAS G12D-Mutated Locally Advanced or Metastatic Non-Small Cell Lung Cancer

Verastem Oncology to Present at Jefferies Healthcare ConferenceMay 27, 2026 7:30 AM
Business Wire Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced that its management team is scheduled to participate in a fireside chat at the Jefferies Global Healthcare Conference on Wednesday, June 3, 2026, at 8:10 am ET in New York. A live webcast of the fireside chat can be accessed under “Events & Presentations” on the Company’s website at www.verastem.com. A replay of the webcasts will be archived on the website for approximately 90 days following the presentation. About Verastem Oncology Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI® FAKZYNJA® CO-PACK in the U.S. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn. View source version on businesswire.com: https://www.businesswire.com/news/home/20260527590882/en/ For Investor and Media Inquiries:
Julissa Viana
Senior Vice President, Corporate Communications,
Investor Relations & Patient Advocacy
investors@verastem.com or
media@verastem.com Original: Verastem Oncology to Present at Jefferies Healthcare Conference

Verastem Oncology Appoints Michael P. Bailey to its Board of DirectorsMay 26, 2026 7:30 AM
Business Wire Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced the appointment of Michael P. Bailey to its Board of Directors. The Company also announced that Robert Gagnon stepped down from the Board following the end of his term on May 21, 2026. “We are excited to have Michael join our Board as he brings more than three decades of experience leading global pharmaceutical and biotechnology companies, with a proven track record of taking oncology medicines from clinical development through successful commercialization, including landmark therapies like ERBITUX®,” said Daniel Paterson, president and chief executive officer of Verastem Oncology. “We look forward to drawing on the insight and expertise Michael brings as we enter the next phase of growth for AVMAPKI® FAKZYNJA® CO-PACK and advance the development of VS-7375, our potential best-in-class oral KRAS G12D (ON/OFF) inhibitor.” “Michael is an accomplished biopharmaceutical executive with deep expertise across the full product lifecycle. Michael’s perspective will be an asset to our Board as Verastem advances its innovative pipeline and builds on its commercial success,” said John Johnson, chairman of Verastem’s Board of Directors. “We’d also like to thank Robert for his dedicated service and the contributions he made during his tenure.” Mr. Bailey added, “I am honored to join Verastem’s Board of Directors at such a pivotal time for the company. Verastem has built a strong business for an underserved rare cancer patient population, and with VS-7375, the company has the potential to make a significant difference in the lives of patients living with KRAS G12D-mutated cancers where there are currently no approved therapies. I look forward to supporting the team and contributing to the company’s continued growth.” Michael P. Bailey Mr. Bailey currently serves as president and chief executive officer (CEO) of AVEO Pharmaceuticals, Inc. (AVEO), an LG Chem, Ltd. Company, and has served on its Board since January 2015, having previously held the roles of chief business officer and chief commercial officer at AVEO. In 2023, Mr. Bailey led the acquisition of AVEO by LG Chem, Ltd. Before AVEO, he was senior vice president of business development and chief commercial officer at Synta Pharmaceuticals Corp. Earlier, Mr. Bailey spent nine years at ImClone Systems Inc., where he led commercial planning and the launch of ERBITUX® (cetuximab) across multiple oncology indications, drove new product planning for CYRAMZA® (ramucirumab) and PORTRAZZA® (necitumumab) and rose to senior vice president of commercial operations. Prior to ImClone, Mr. Bailey managed the cardiovascular new products development portfolio at Genentech. Mr. Bailey started his career as part of SmithKline Beecham’s executive marketing development program, where he held a variety of commercial roles, including sales, strategic planning and product management. Mr. Bailey received a B.S. in psychology from St. Lawrence University and an M.B.A. in international marketing from Mendoza College of Business at University of Notre Dame and continued his executive education in Finance at Harvard Business School and Board of Director Leadership at Wharton School of Business. About Verastem Oncology Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI® FAKZYNJA® CO-PACK in the U.S. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn. View source version on businesswire.com: https://www.businesswire.com/news/home/20260526488039/en/ For Investor and Media Inquiries:
Julissa Viana
Senior Vice President, Corporate Communications,
Investor Relations & Patient Advocacy
investors@verastem.com or
media@verastem.com Original: Verastem Oncology Appoints Michael P. Bailey to its Board of Directors

Verastem Oncology to Present at Upcoming Investor ConferencesMay 12, 2026 7:30 AM
Business Wire Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced that its management team is scheduled to participate and present at the following investor conferences in New York City: HCW 4th Annual BioConnect Investor Conference: Tuesday, May 19, 2026, 4:00-4:30 pm ET RBC Global Healthcare Conference: Wednesday, May 20, 2026, 9:30-9:55 am ET A live webcast of the fireside chat can be accessed under “Events & Presentations” on the Company’s website at www.verastem.com. A replay of the webcasts will be archived on the website for approximately 90 days following the presentation. About Verastem Oncology
Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI® FAKZYNJA® CO-PACK in the U.S. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn. View source version on businesswire.com: https://www.businesswire.com/news/home/20260512451690/en/ For Investor and Media Inquiries:
Julissa Viana
Senior Vice President, Corporate Communications,
Investor Relations & Patient Advocacy
investors@verastem.com or
media@verastem.com Original: Verastem Oncology to Present at Upcoming Investor Conferences

On World Ovarian Cancer Day, LGSOC Resource Guide Launches to Support People Living with Low-Grade Serous Ovarian CancerMay 8, 2026 7:30 AM
Business Wire Available at LGSOCAwarenessDay.Org, the LGSOC Resource Guide brings together resources from patient advocacy groups, covering topics from diagnosis to survivorship Today, STAAR Low-Grade Serous Ovarian Cancer Foundation, Not These Ovaries, and Verastem Oncology announced the launch of the LGSOC Resource Guide, a unique resource designed to support patients living with low-grade serous ovarian cancer (LGSOC), a rare and distinct form of ovarian cancer, along with their caregivers and advocates. Available at www.LGSOCAwarenessDay.org, the guide launches in conjunction with World Ovarian Cancer Day, a global effort to raise awareness of all types of ovarian cancer. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260508725775/en/Low-Grade Serous Ovarian Cancer Resource Guide LGSOC is a rare and distinct form of ovarian cancer that is often underrecognized and can be challenging to diagnose and manage. Patients frequently face delays in diagnosis, have limited access to disease-specific resources, and need to navigate complex treatment decisions. The LGSOC Resource Guide was created to help address these gaps by bringing together trusted educational materials and community resources into one accessible destination. “For many people living with LGSOC, finding clear, relevant information can be an ongoing challenge—especially given how different this disease is, including how it responds to treatment,” said Nicole Andrews, chair of the STAAR Low-Grade Serous Ovarian Cancer Foundation. “As both an advocate and a patient, the LGSOC Resource Guide represents another meaningful step forward for our community. We’re not only beginning to see new treatment advances that may give patients more confidence and hope for the future, we’re also building on resources from the broader ovarian cancer patient community to bring together trusted information specific for the LGSOC community.” In 2023, STAAR Low-Grade Serous Ovarian Cancer Foundation engaged directly with the U.S. Food and Drug Administration (FDA) through a first-ever Externally-led Patient-Focused Drug Development (EL-PFDD) meeting, bringing forward the lived experiences of patients with LGSOC and helping to establish broader recognition of LGSOC as a distinct disease requiring dedicated treatment approaches. This effort reinforced the importance of developing treatments tailored to the unique biology of LGSOC, which differs from high-grade ovarian cancer. This effort was foundational in supporting the first and only treatment currently approved by the U.S. Food and Drug Administration (FDA) for KRAS-mutated recurrent LGSOC following a prior systemic therapy, developed by Verastem Oncology. “A diagnosis of low-grade serous ovarian cancer can feel overwhelming, with patients often asked to make life-altering decisions in just days. While LGSOC is a rare disease, that rarity does not lessen the need for information, connection, and support—it makes it even more critical,” said Emily Campbell, executive director and co-founder of Not These Ovaries. “The LGSOC Resource Guide meets patients in those moments, helping them move from feeling powerless to becoming active participants in their care.” The LGSOC Resource Guide has compiled resources from dozens of advocacy organizations, including videos, articles, FAQs, and blog posts covering important topics such as diagnosis, treatment and care decisions, fertility and survivorship considerations, emotional wellbeing and caregiver support. It is designed as a flexible tool that individuals can return to over time, depending on their needs and stage of care. “The LGSOC Resource Guide represents the power of collaboration across the patient advocacy community. By bringing together organizations from across the ovarian cancer and LGSOC patient advocacy community, the Guide includes resources to help patients and their families access meaningful, relevant information throughout their experience,” said Julissa Viana, senior vice president, corporate communications, investor relations, and advocacy relations at Verastem Oncology. “We are deeply grateful to the patient advocacy groups who contributed to this effort. In rare diseases like LGSOC, progress depends on working together, and this guide is an important step toward making sure no patient feels overlooked or alone and can find the resources they need wherever they are looking.” This resource guide is intended for informational purposes only and reflects information available at the time of publication. As research and treatment options continue to evolve, content may become outdated. Verastem Oncology and its contributors assume no responsibility or obligation to update or modify any of the information contained in the resource guide. Further, Verastem Oncology and its contributors make no representations or warranties regarding the completeness or accuracy of the information and assume no responsibility for any actions taken based on its content. The LGSOC Resource Guide is available at www.LGSOCAwarenessDay.org. About Low-Grade Serous Ovarian Cancer (LGSOC) LGSOC is a rare ovarian cancer that is insidious and persistent. LGSOC is distinct and different from high-grade serous ovarian cancer (HGSOC) and requires different treatment. LGSOC is highly recurrent and less sensitive to chemotherapy compared to HGSOC. Approximately 6,000-8,000 women in the U.S. and 80,000 worldwide are living with this disease. LGSOC affects younger women with bimodal peaks of diagnosis at ages between 20-30 and 50-60 and has a median survival of approximately ten years. Approximately 70 percent of LGSOC shows RAS pathway-associated mutations, and 30 percent of people with LGSOC have a KRAS mutation. The majority of patients report a negative impact of LGSOC on their mental and physical health, fertility, and long-term quality of life. About STAAR Low-Grade Serous Ovarian Cancer STAAR was founded by three women—Alex, Bailey, and Jess—each diagnosed with low-grade serous ovarian cancer (LGSOC). United by their passion to extend lives through research, they launched the first U.S.-based nonprofit dedicated to finding effective treatments for LGSOC. STAAR is governed and run by a volunteer board, half of whom are women with LGSOC. Because STAAR is volunteer-led, nearly every donation dollar directly benefits life-saving research. Volunteers play a vital role in STAAR and serve on committees, the board of directors, lead or help with special projects, help at events, or join monthly volunteer meetings to lend ideas. About Not These Ovaries Not These Ovaries is a patient-driven nonprofit dedicated to improving outcomes for those affected by low-grade serous carcinoma (LGSOC) and borderline ovarian tumors (BOT). The organization empowers patients and caregivers by providing disease-specific education, resources on biomarkers and treatment options, and guidance on finding experienced healthcare providers. It also invests in research targeting these understudied areas. 100% of donations go directly to supporting patients and funding research. Learn more at www.nottheseovaries.org. About Verastem Oncology Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Our commercial and clinical pipelines are focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn. View source version on businesswire.com: https://www.businesswire.com/news/home/20260508725775/en/ For Investor and Media Inquiries:
Julissa Viana
Senior Vice President, Corporate Communications,
Investor Relations & Patient Advocacy
investors@verastem.com or
media@verastem.com Original: On World Ovarian Cancer Day, LGSOC Resource Guide Launches to Support People Living with Low-Grade Serous Ovarian Cancer

Verastem Oncology Launches HCP and Patient Reimagine Campaign to Increase Awareness of AVMAPKI® FAKZYNJA® CO-PACK for KRAS-Mutated Recurrent Low-Grade Serous Ovarian CancerApril 30, 2026 7:30 AM
Business Wire
Low-grade serous ovarian cancer (LGSOC) is a rare ovarian cancer with a high rate of recurrence affecting more than 80% of patients


Integrated healthcare professional (HCP) and patient campaign features new websites and expanded digital resources about the first and only treatment specifically FDA-approved for KRAS-mutated recurrent LGSOC who have received prior systemic therapy


Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced the launch of its Reimagine Recurrent Low-Grade Serous Ovarian Cancer (LGSOC) campaign for AVMAPKI® FAKZYNJA® CO-PACK (avutometinib capsules; defactinib tablets), the first and only treatment specifically approved by the U.S. Food and Drug Administration (FDA) for adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy. The integrated campaign encourages healthcare professionals (HCPs) and patients to rethink what may be possible at and beyond the earliest recurrence for people living with KRAS-mutated recurrent LGSOC.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260428101589/en/
LGSOC is a rare and distinct form of ovarian cancer, with unique biology and clinical behavior different from other ovarian cancers. Its tumor growth is primarily driven by the RAS/MAPK pathway with activating mutations, most commonly in the KRAS gene, found in many cancers that often correlate with poor prognosis. Approximately 30% of patients with LGSOC have a KRAS mutation, making it the most common type of gene mutation in the disease. Patients with LGSOC often experience delays in diagnosis and have historically had limited treatment options specifically for their disease.


“The Reimagine Recurrent LGSOC campaign shifts how we think about KRAS-mutated recurrent LGSOC treatment and supporting both patients and healthcare professionals in rethinking what may be possible at and beyond the earliest recurrence,” said Dan Paterson, president and chief executive officer at Verastem Oncology. “The FDA’s accelerated approval of AVMAPKI FAKZYNJA CO-PACK marked an important step forward for patients with KRAS-mutated recurrent LGSOC, underscoring the need for treatments specifically developed for this population. With this campaign, we’re building on that momentum to further inform treatment decisions and support patients throughout their care journey.”


The Reimagine campaign was informed by insights from patients and HCPs, highlighting the need for both emotional resonance and practical, accessible information. For HCPs, the campaign encourages re-evaluation of treatment approaches in the context of a treatment specifically approved by the FDA for KRAS-mutated recurrent LGSOC. For patients, it reflects a desire not only to manage their condition, but to envision a future shaped by progress and meaningful life milestones.


“Patients with KRAS-mutated recurrent LGSOC have historically had limited resources tailored to their specific experience,” said Linda Stamler, vice president of marketing at Verastem Oncology. “The Reimagine Recurrent LGSOC campaign was developed to help identify and support patients who may be appropriate for treatment with AVMAPKI FAKZYNJA CO-PACK. Through this campaign, we are offering resources such as patient stories, tools to help manage side effects, videos explaining mechanism of action and dosing, and guides to help set treatment expectations so patients and HCPs feel more informed and supported.”


For more information, HCPs can visit https://www.avmapkifakzynjaco-pack-hcp.com/ and patients can visit https://www.avmapkifakzynjaco-pack.com/.


About Low-Grade Serous Ovarian Cancer (LGSOC)


LGSOC is a rare ovarian cancer that is insidious and persistent. LGSOC is distinct and different from high-grade serous ovarian cancer (HGSOC) and requires different treatment. LGSOC is highly recurrent and less sensitive to chemotherapy compared to HGSOC. Approximately 6,000-8,000 women in the U.S. and 80,000 worldwide are living with this disease. LGSOC affects younger women with bimodal peaks of diagnosis at ages between 20-30 and 50-60 and has a median survival of approximately ten years. Approximately 70 percent of LGSOC shows RAS pathway-associated mutations, and 30 percent of people with LGSOC have a KRAS mutation. The majority of patients report a negative impact of LGSOC on their mental and physical health, fertility, and long-term quality of life.


About AVMAPKI and FAKZYNJA Combination Therapy


AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors.


The U.S. Food and Drug Administration (FDA) approved AVMAPKI® FAKZYNJA® CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Verastem is conducting RAMP 301 (GOG-3097/ENGOT-ov81/GTG-UK) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC) with and without a KRAS mutation. Verastem is also evaluating avutometinib plus defactinib with standard-of-care chemotherapy as a potential treatment in the first-line for patients with advanced pancreatic cancer (RAMP 205; NCT05669482). Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on a stand-alone basis for any use.


AVMAPKI FAKZYNJA CO-PACK U.S. Indication


Indication


AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.


This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.


Important Safety Information


Warnings and Precautions



Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity.



Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration.



Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality.



Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction.



Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception.



Adverse Reactions


The most common (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count.


Drug Interactions



Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.



Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.



Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin.



Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid.



Use in Specific Populations



Lactation: Advise not to breastfeed.



Fertility: May impair fertility in males and females.



Click here for full Prescribing Information.


About Verastem Oncology


Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI® FAKZYNJA® CO-PACK in the U.S. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn.


Forward-Looking Statements


This press release includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as “anticipate,” “expect,” “plan,” “could,” “may,” “believe,” “estimate,” “forecast,” “goal,” “project,” and other words of similar meaning. Such forward-looking statements address various matters about, among other things, Verastem Oncology’s programs and product candidates, strategy, future plans and prospects, including statements related to the potential for and timing of commercialization of product candidates, the expected outcome and benefits of the Company’s collaboration with GenFleet Therapeutics (Shanghai), Inc., the timing of commencing and completing trials and compiling data, the expected timing of the presentation of data by the Company and the potential clinical value of various of the Company’s clinical trials. Each forward-looking statement contained in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others: the uncertainties inherent in research and development, such as the possibility of negative or unexpected results of clinical trials; that we may not see a return on investment on the payments we have and may continue to make pursuant to the collaboration and option agreement with GenFleet, or that GenFleet may fail to fully perform under the agreement; that we may not be successful in our continued commercialization of AVMAPKI FAKZYNJA CO-PACK; that the development and commercialization of our product candidates may take longer or cost more than planned, including as a result of conducting additional studies or our decisions regarding execution of such commercialization; that data may not be available when expected; risks associated with preliminary and interim data, which may not be representative of more mature data; risks associated with the recent changes in administration policy or actions that may create regulatory uncertainty that may adversely affect our business; risks associated with the current administration’s reductions to the FDA’s workforce and any subsequent reductions that may lead to disruptions and delays in the FDA’s review and oversight of our product candidates and impact the FDA’s ability to provide timely feedback on our development programs; that our product candidates may not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients; and the risks identified under the heading "Risk Factors" as detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, as filed with the Securities and Exchange Commission (SEC) on March 4, 2026, as well as the other information we file with the SEC, are possibly realized. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at www.sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of these statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260428101589/en/
For Investor and Media Inquiries:

Julissa Viana

Senior Vice President, Corporate Communications,

Investor Relations & Patient Advocacy

investors@verastem.com or

media@verastem.com


Original: Verastem Oncology Launches HCP and Patient Reimagine Campaign to Increase Awareness of AVMAPKI® FAKZYNJA® CO-PACK for KRAS-Mutated Recurrent Low-Grade Serous Ovarian Cancer

Verastem Oncology Announces Two-Year Median Follow-Up Data on AVMAPKI® FAKZYNJA® Combination Therapy (avutometinib capsules; defactinib tablets) in Recurrent Low-Grade Serous Ovarian Cancer at the SGO 2026 Annual Meeting on Women’s CancersApril 10, 2026 7:00 AM
Business Wire
Patients in the Phase 2 RAMP 201 clinical trial demonstrated sustained clinical benefits across multiple efficacy measures highlighting durability of response with a median follow-up of two years


52% of patients with a KRAS mutation and 30% of patients with KRAS wild-type recurrent LGSOC remained on therapy for more than one year


After two years of follow-up, the combination therapy continued to demonstrate a well-tolerated safety profile, with no new safety signals and a low discontinuation rate due to adverse events


Poster presentation of new exposure response analysis demonstrates that the best therapeutic effect of avutometinib plus defactinib can be achieved when using the approved dose and schedule


Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced two-year median follow-up data from the Phase 2 RAMP 201 clinical trial that evaluated AVMAPKI® FAKZYNJA® combination therapy (avutometinib capsules; defactinib tablets) in patients with recurrent low-grade serous ovarian cancer (LGSOC) will be presented today during an oral plenary session at the Society of Gynecologic Oncology (SGO) 2026 Annual Meeting on Women’s Cancers taking place in San Juan, Puerto Rico, April 10-13, 2026.


“Patients who remained on treatment with avutometinib plus defactinib for two years were able to maintain the same level of response and duration of therapy as seen in the primary analysis, suggesting that patients can stay on the combination for a long period of time, derive benefit and have manageable toxicity during long-term administration of these medications,” said Rachel Grisham, M.D., RAMP 201 Presenting Investigator, Section Head, Ovarian Cancer at Memorial Sloan Kettering Cancer Center (MSK) in New York, NY, and Global Lead Principal Investigator of GOG-3097/ENGOT-ov81/GTG-UK/RAMP 301. “As we approach the one-year FDA approval anniversary of avutometinib in combination with defactinib, this analysis reinforces progress in bringing a durable and clinically meaningful option to patients.”


In the updated analysis, with ongoing patients presenting a median follow-up of 24.9 months, efficacy measures, including median duration of response (mDOR) and median progression free survival (mPFS), and safety, are consistent with the primary analysis, which was conducted more than 13 months prior:




 






Long-Term Median Follow-Up

(data cutoff of August 12, 2025)




(N=17)






Primary Analysis*

(data cutoff of June 30, 2024)




(N=109)








 






Total

(N=17)






KRAS mt

(N=12)






KRAS wt

(N=5)






Total

(N=109)






KRAS mt

(N=57)






KRAS wt

(N=52)








mDoR






31.1 months






31.1 months






12 months






31.1 months






31.1 months






9.2 months








mPFS






12.9 months






19.6 months






12.7 months






12.9 months






22 months






12.8 months







*The primary analysis was published in the Journal of Clinical Oncology (Banerjee et al, July 11, 2025).


Fifty percent of patients with KRAS-mutated and 30 percent with KRAS wild-type LGSOC remained on therapy for more than one year. Adverse events (AEs) were consistent with the primary analysis with no new safety signals observed, and a 12 percent discontinuation rate due to adverse events.


“With two years of follow-up demonstrating durable benefit, these analyses reinforce confidence in avutometinib plus defactinib in the real-world setting for patients living with recurrent LGSOC,” said Bradley Monk, M.D., FACOG, FACS, RAMP 201 Investigator and Gynecologic Oncologist with Florida Cancer Specialists & Research Institute and Director GOG-Partners. “Notably, seeing one-third of patients without KRAS mutations remain on therapy for over a year is clinically meaningful, especially in a patient population with limited treatment options. The data also underscore the combination’s manageable safety profile and how dose holds can be used effectively to maintain dose intensity, helping distinguish this treatment strategy from prior MEK-based approaches.”


Exposure-Response Analysis for Avutometinib in Combination with Defactinib in LGSOC


Efficacy analyses included patients with LGSOC (N=158) from the FRAME and RAMP 201 studies, while safety analyses included a broader population (N=303) from the FRAME, RAMP 201, and RAMP 202 studies.


All efficacy endpoints, including overall response rates, duration of response, and best target lesion response, suggest the best therapeutic effect is achieved with the FDA approved dose of avutometinib 3.2 mg twice weekly plus defactinib 200 mg twice daily. While a lower avutometinib dose may mitigate treatment emergent adverse events (TEAEs) (the most frequent being grade ≥2 skin disorders (39.9%; n=121), followed by grade ≥2 gastrointestinal toxicity (34.0%; n=103), grade ≥2 liver function tests (25.1%; n=76), and grade ≥3 creatine phosphokinase elevation (14.5%; n=44)), it may also compromise the efficacy. Importantly, these analyses demonstrated that TEAEs can be monitored and managed with dose interruptions, and subsequently resume treatment at the approved dose level, to allow patients to stay on treatment.


Verastem has an exhibition booth (#608) at the meeting to provide an overview of its approved therapy and ongoing cancer research. The full schedule and poster titles are available online at the SGO 2026 Annual Meeting on Women’s Cancer website.


About RAMP 201


RAMP 201 (ENGOTov60/GOG3052/NCRI) (NCT04625270) was an adaptive, two-part multicenter, parallel cohort, randomized, open-label Phase 2 registration-directed trial evaluating the efficacy and safety of avutometinib alone and in combination with defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC). The first part of the trial (Part A) determined the selection of the go-forward regimen, which was the combination of avutometinib and defactinib versus avutometinib alone, based on overall response rates. The expansion phases of the trial (Parts B and C) evaluated the safety and efficacy of the go-forward regimen of avutometinib 3.2 mg twice weekly and defactinib 200 mg twice daily. The Part D portion of the trial evaluated a low dose of the combination to inform individualized dose reduction.


About Low-Grade Serous Ovarian Cancer (LGSOC)


LGSOC is a rare ovarian cancer that is insidious and persistent. LGSOC is distinct and different from high-grade serous ovarian cancer (HGSOC) and requires different treatment. LGSOC is highly recurrent and less sensitive to chemotherapy compared to HGSOC. Approximately 6,000-8,000 women in the U.S. and 80,000 worldwide are living with this disease. LGSOC affects younger women with bimodal peaks of diagnosis at ages between 20-30 and 50-60 and has a median survival of approximately ten years. Approximately 70 percent of LGSOC shows RAS pathway-associated mutations, and 30 percent of people with LGSOC have a KRAS mutation. The majority of patients report a negative impact of LGSOC on their mental and physical health, fertility, and long-term quality of life.


About AVMAPKI and FAKZYNJA Combination Therapy


AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors.


The U.S. Food and Drug Administration (FDA) approved AVMAPKI® FAKZYNJA® CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Verastem is conducting RAMP 301 (GOG-3097/ENGOT-ov81/GTG-UK) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC) with and without a KRAS mutation. Verastem is also evaluating avutometinib plus defactinib with standard-of-care chemotherapy as a potential treatment in the first-line for patients with advanced pancreatic cancer (RAMP 205; NCT05669482). Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on a stand-alone basis for any use.


AVMAPKI FAKZYNJA CO-PACK U.S. Indication


Indication


AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.


This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.


Important Safety Information


Warnings and Precautions



Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity.



Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration.



Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality.



Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction.



Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception.



Adverse Reactions


The most common (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count.


Drug Interactions



Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.



Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.



Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin.



Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid.



Use in Specific Populations



Lactation: Advise not to breastfeed.



Fertility: May impair fertility in males and females.



Click here for full Prescribing Information.


About Verastem Oncology


Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI® FAKZYNJA® CO-PACK in the U.S. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn.


Disclosure: Dr. Grisham has financial interests related to Verastem Oncology.


Forward-Looking Statements


This press release includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as “anticipate,” “expect,” “plan,” “could,” “may,” “believe,” “estimate,” “forecast,” “goal,” “project,” and other words of similar meaning. Such forward-looking statements address various matters about, among other things, Verastem Oncology’s programs and product candidates, strategy, future plans and prospects, including statements related to the potential for and timing of commercialization of product candidates, the expected outcome and benefits of the Company’s collaboration with GenFleet Therapeutics (Shanghai), Inc., the timing of commencing and completing trials and compiling data, the expected timing of the presentation of data by the Company and the potential clinical value of various of the Company’s clinical trials. Each forward-looking statement contained in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others: the uncertainties inherent in research and development, such as the possibility of negative or unexpected results of clinical trials; that we may not see a return on investment on the payments we have and may continue to make pursuant to the collaboration and option agreement with GenFleet, or that GenFleet may fail to fully perform under the agreement; that we may not be successful in our continued commercialization of AVMAPKI FAKZYNJA CO-PACK; that the development and commercialization of our product candidates may take longer or cost more than planned, including as a result of conducting additional studies or our decisions regarding execution of such commercialization; that data may not be available when expected; risks associated with preliminary and interim data, which may not be representative of more mature data; risks associated with the recent changes in administration policy or actions that may create regulatory uncertainty that may adversely affect our business; risks associated with the current administration’s reductions to the FDA’s workforce and any subsequent reductions that may lead to disruptions and delays in the FDA’s review and oversight of our product candidates and impact the FDA’s ability to provide timely feedback on our development programs; that our product candidates may not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients; and the risks identified under the heading "Risk Factors" as detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, as filed with the Securities and Exchange Commission (SEC) on March 4, 2026, as well as the other information we file with the SEC, are possibly realized. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at www.sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of these statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260410817824/en/
For Investor and Media Inquiries:

Julissa Viana

Vice President, Corporate Communications,

Investor Relations & Patient Advocacy

investors@verastem.com or

media@verastem.com


Original: Verastem Oncology Announces Two-Year Median Follow-Up Data on AVMAPKI® FAKZYNJA® Combination Therapy (avutometinib capsules; defactinib tablets) in Recurrent Low-Grade Serous Ovarian Cancer at the SGO 2026 Annual Meeting on Women’s Cancers

Verastem Oncology Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)April 2, 2026 4:05 PM
Business Wire
Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced the grant of 141,000 restricted stock units to eleven new employees. The awards were granted pursuant to the Nasdaq inducement grant exception as an inducement material to the employee’s acceptance of employment with Verastem Oncology in accordance with Nasdaq Listing Rule 5635(c)(4). The 141,000 restricted stock units granted to the eleven new employees will vest at a rate of twenty-five percent (25%) on the one-year anniversary of the employee’s date of hire, with the remaining shares vesting quarterly over the next three (3) years in equal quarterly amounts, provided the employee is employed by or providing other services for Verastem Oncology on each such vesting date.


About Verastem Oncology


Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI® FAKZYNJA® CO-PACK in the U.S. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260402851535/en/
For Investor and Media Inquiries:

Julissa Viana

Vice President, Corporate Communications, Investor Relations & Patient Advocacy

investors@verastem.com or

media@verastem.com


Original: Verastem Oncology Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Verastem Oncology Announces Late Breaking and Regular Abstracts Accepted for Presentation at the AACR Annual Meeting 2026March 17, 2026 6:03 PM
Business Wire
Preclinical research with VS-7375 in combination with PRMT5 inhibitors demonstrated strong durable tumor regressions in KRAS G12D pancreatic cancer models


In preclinical pancreatic, lung, and colorectal cancer models, VS-7375 (dual ON/OFF inhibitor) showed deeper and more sustained tumor regression compared to ON-only G12D or pan-RAS inhibitors


Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced multiple abstracts have been accepted for presentation at the American Association for Cancer Research (AACR) Annual Meeting to be held April 17-22, 2026, in San Diego, CA.


“Preclinical data accepted for presentation at the AACR Special Conference in Cancer Research: RAS Oncogenesis and Therapeutics and the AACR Annual Meeting highlight our highly differentiated investigational asset VS-7375, an oral, KRAS G12D (ON/OFF) inhibitor in clinical development for pancreatic, lung, colorectal and other cancers harboring a G12D mutation, the most prevalent KRAS mutation in human cancers,” said Jonathan Pachter, Ph.D., chief scientific officer at Verastem Oncology. “While most agents targeting KRAS G12D-driven cancers bind preferentially only to the “ON” (active) or “OFF” (inactive) state of KRAS, VS-7375 engages both active and inactive states with extremely high affinity and long residence time, which corresponds to superior efficacy across pre-clinical models of G12D mutated cancers.”


Late-Breaking Abstracts


Late-breaking abstracts will be available on the AACR Annual Meeting website on April 17, 2026, 12 p.m. PT.


Session: Late-Breaking Research: Experimental and Molecular Therapeutics 2

Abstract #: LB183 / 5

Title: Strong durable tumor regressions with the KRAS G12D ON/OFF inhibitor VS-7375 in combination with PRMT5 inhibition in MTAP-deleted/KRAS G12D-mutant pancreatic cancer

Date and Time: April 20, 2026, 2 p.m. - 5 p.m. PT


Session: Late-Breaking Research: Experimental and Molecular Therapeutics 2

Abstract #: LB197 / 19

Title: VS-7375, a non-covalent dual ON/OFF KRAS G12D inhibitor, displays superior activity to ON-only KRAS G12D inhibitors in preclinical models of pancreatic cancer

Date and Time: April 20, 2026, 2 p.m. - 5 p.m. PT


Regular Abstracts


The accepted abstracts are available on the AACR Annual Meeting website: AACR Annual Meeting 2026


Session: Novel Antitumor Agents 3

Abstract & Poster Details: Abstract/Poster #7100, Poster Section #13, Poster Board #20

Title: VS-7375: An oral, selective KRAS G12D dual ON/OFF inhibitor with potent anti-tumor activity as a single agent and in combination with other agents

Date and Time: April 22, 2026, 9 a.m. - 12 p.m. PT


In the KP4 KRAS G12D pancreatic cancer model, VS-7375 (50 mg/kg twice daily), zoldonrasib (100 mg/kg once daily) and daraxonrasib (25 mg/kg once daily), produced similar initial tumor regression through day nine. By approximately day 20, however, zoldonrasib and daraxonrasib lost anti-tumor activity with tumor outgrowth, (mean tumor volume >850 mm by day 30) whereas VS-7375 maintained sustained tumor regression (mean tumor volume ~80 mm by day 30), consistent with pharmacodynamic analyses showing durable pathway inhibition only with VS-7375. VS-7375 also showed deeper tumor regression compared to these RAS ON-only inhibitors in KRAS G12D-mutated lung and colorectal xenograft models.


Session: Therapies Targeting Metastasis

Abstract & Poster Details: Abstract/Poster #2232, Poster Section #32, Poster Board #7

Title: Investigating the combined inhibition of RAF, MEK, and FAK in melanoma molecular subtypes

Date and Time: April 20, 2026, 9 a.m. - 12 p.m. PT


In mutant BRAF-driven models, the combination of the FAK inhibitor VS-4718 and the RAF/MEK clamp avutometinib, with or without the mutant BRAF inhibitor encorafenib, significantly delayed tumor onset, induced regression of established tumors and brain metastases, and prolonged overall survival.


AACR Special Conference in Cancer Research: RAS Oncogenesis and Therapeutics


At the AACR Special Conference in Cancer Research: RAS Oncogenesis and Therapeutics held March 5-8, 2026, Dr. Jonathan Pachter, delivered a plenary oral presentation titled, “Anti-tumor efficacy of the selective oral KRAS G12D dual ON/OFF inhibitor VS-7375 as a single agent and in combination with targeted agents.” This presentation can be found on the Company’s website on the Resources page.


About VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor


VS-7375 is a potential best-in-class, potent, and selective oral KRAS G12D dual ON/OFF inhibitor. VS-7375 is the lead program from the Verastem Oncology discovery and development collaboration with GenFleet Therapeutics. Verastem initiated VS-7375-101, an international Phase 1/2 clinical trial, in June of 2025 in the U.S., that is evaluating the safety and efficacy of VS-7375 in patients with advanced KRAS G12D mutant solid tumors. In July 2025, U.S. Food and Drug Administration (FDA) granted Fast Track Designation (FTD) to VS-7375 for the first-line treatment of patients with KRAS G12D-mutated locally advanced or metastatic adenocarcinoma of the pancreas (PDAC) and for the treatment of patients with KRAS G12D-mutated locally advanced or metastatic PDAC who have received at least one prior line of standard systemic therapy.


About the GenFleet Therapeutics Collaboration


The collaboration with GenFleet Therapeutics aims to advance three oncology discovery programs related to RAS/MAPK pathway-driven cancers. The collaboration provides Verastem with an exclusive option to obtain a license for each of the three compounds in the collaboration after the successful completion of pre-determined milestones in a Phase 1 trial. Verastem selected VS-7375 (also known as GFH375), an oral KRAS G12D (ON/OFF) inhibitor, as its lead program in December 2023 and the license for VS-7375 that was exercised in January 2025 is the first one from this collaboration. The licenses would give Verastem development and commercialization rights outside the GenFleet markets of mainland China, Hong Kong, Macau, and Taiwan.


About Verastem Oncology


Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI™ FAKZYNJA™ CO-PACK in the U.S. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn.


Forward-Looking Statements


This press release includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as “anticipate,” “expect,” “plan,” “could,” “may,” “believe,” “estimate,” “forecast,” “goal,” “project,” and other words of similar meaning. Such forward-looking statements address various matters about, among other things, Verastem Oncology’s programs and product candidates, strategy, future plans and prospects, including statements related to the potential for and timing of commercialization of product candidates, the expected outcome and benefits of the Company’s collaboration with GenFleet Therapeutics (Shanghai), Inc., the timing of commencing and completing trials and compiling data, the expected timing of the presentation of data by the Company and the potential clinical value of various of the Company’s clinical trials. Each forward-looking statement contained in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others: the uncertainties inherent in research and development, such as the possibility of negative or unexpected results of clinical trials; that we may not see a return on investment on the payments we have and may continue to make pursuant to the collaboration and option agreement with GenFleet, or that GenFleet may fail to fully perform under the agreement; that we may not be successful in our continued commercialization of AVMAPKI FAKZYNJA CO-PACK; that the development and commercialization of our product candidates may take longer or cost more than planned, including as a result of conducting additional studies or our decisions regarding execution of such commercialization; that data may not be available when expected; risks associated with preliminary and interim data, which may not be representative of more mature data; risks associated with the recent changes in administration policy or actions that may create regulatory uncertainty that may adversely affect our business; risks associated with the current administration’s reductions to the FDA’s workforce and any subsequent reductions that may lead to disruptions and delays in the FDA’s review and oversight of our product candidates and impact the FDA’s ability to provide timely feedback on our development programs; that our product candidates may not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients; and the risks identified under the heading "Risk Factors" as detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, as filed with the Securities and Exchange Commission (SEC) on March 4, 2026, as well as the other information we file with the SEC, are possibly realized. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at www.sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of these statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260317972659/en/
For Investor and Media Inquiries:

Julissa Viana

Vice President, Corporate Communications,

Investor Relations & Patient Advocacy

investors@verastem.com or

media@verastem.com


Original: Verastem Oncology Announces Late Breaking and Regular Abstracts Accepted for Presentation at the AACR Annual Meeting 2026

Verastem Oncology Announces Abstracts Accepted for Presentation on AVMAPKI™ FAKZYNJA™ (avutometinib capsules; defactinib tablets) Combination Therapy at the Society of Gynecologic Oncology 2026 Annual Meeting on Women’s CancersMarch 17, 2026 4:44 PM
Business Wire
Plenary oral presentation features long-term follow-up data from the Phase 2 RAMP 201 recurrent low-grade serous ovarian cancer clinical trial


Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced it will present new long-term AVMAPKI™ FAKZYNJA™ combination therapy (avutometinib capsules; defactinib tablets) data from the Phase 2 RAMP 201 clinical trial at the Society of Gynecologic Oncology (SGO) 2026 Annual Meeting on Women’s Cancers. Additional abstracts accepted for presentation will also be shared at the meeting being held on April 10-13, 2026, in San Juan, Puerto Rico.


“We look forward to the upcoming data presentation at SGO, which includes an additional year of follow-up data beyond the initial presentation of this important study,” said John Hayslip, M.D., chief medical officer at Verastem Oncology. “These results reinforce the findings from the RAMP 201 primary analysis of avutometinib in combination with defactinib and continue to add to the clinical body of evidence demonstrating the long-term safety and efficacy of this combination for patients with recurrent LGSOC with and without KRAS mutations.”


Verastem will have an exhibition booth (#608) at the meeting to provide an overview of its approved therapy and ongoing cancer research. The full schedule and poster titles are available online at the SGO 2026 Annual Meeting on Women’s Cancer website.


Oral Plenary Presentation Details

Session: Scientific Plenary I: Advancing Science through Clinical Trials?

Title: Long-Term Efficacy and Safety of Avutometinib + Defactinib in Patients with Recurrent Low-Grade Serous Ovarian Cancer: Results from ENGOT-OV60/GOG-3052/RAMP 201?

Presenter: Rachel Grisham, M.D., Memorial Sloan Kettering Cancer Center

Date and Time: Friday, April 10, 2026, 5:05 pm - 5:13 pm?

Location: Exhibit Hall A


Poster Presentation Details

Session: Poster

Title: Exposure-Response Analysis for Avutometinib in Combination with Defactinib in Low-Grade Serous Ovarian Cancer

Presenter: Yaofeng Cheng, Ph.D., Verastem Oncology

Date: Sunday, April 12, 2026

Poster # and Location: #1112, Exhibit Hall B


Session: Poster? Tour

Title: Combination targeted and Hormonal treAtMEnt of Low-gradE serous Ovarian cancer in the upfroNt setting (CHAMELEON)

Presenter: Beryl Manning-Geist, M.D., Emory University School of Medicine

Poster # and Location: #272, Exhibit Hall B


About RAMP 201


RAMP 201 (ENGOTov60/GOG3052/NCRI) (NCT04625270) was an adaptive, two-part multicenter, parallel cohort, randomized, open-label Phase 2 registration-directed trial evaluating the efficacy and safety of avutometinib alone and in combination with defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC). The first part of the trial (Part A) determined the selection of the go-forward regimen, which was the combination of avutometinib and defactinib versus avutometinib alone, based on overall response rates. The expansion phases of the trial (Parts B and C) evaluated the safety and efficacy of the go-forward regimen of avutometinib 3.2 mg twice weekly and defactinib 200 mg twice daily. The Part D portion of the trial evaluated a low dose of the combination to inform individualized dose reduction.


About Low-Grade Serous Ovarian Cancer (LGSOC)


LGSOC is a rare ovarian cancer that is insidious and persistent. LGSOC is distinct and different from high-grade serous ovarian cancer (HGSOC) and requires different treatment. LGSOC is highly recurrent and less sensitive to chemotherapy compared to HGSOC. Approximately 6,000-8,000 women in the U.S. and 80,000 worldwide are living with this disease. LGSOC affects younger women with bimodal peaks of diagnosis at ages between 20-30 and 50-60 and has a median survival of approximately ten years. Approximately 70 percent of LGSOC shows RAS pathway-associated mutations, and 30 percent of people with LGSOC have a KRAS mutation. The majority of patients report a negative impact of LGSOC on their mental and physical health, fertility, and long-term quality of life.


About AVMAPKI and FAKZYNJA Combination Therapy


AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors.


The U.S. Food and Drug Administration (FDA) approved AVMAPKI™ FAKZYNJA™ CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Verastem is conducting RAMP 301 (GOG-3097/ENGOT-ov81/GTG-UK) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC) with and without a KRAS mutation. Verastem is also evaluating avutometinib plus defactinib with standard-of-care chemotherapy as a potential treatment in the first-line for patients with advanced pancreatic cancer (RAMP 205; NCT05669482). Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on a stand-alone basis for any use.


AVMAPKI FAKZYNJA CO-PACK U.S. Indication


Indication


AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.


This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.


Important Safety Information


Warnings and Precautions



Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity.



Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration.



Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality.



Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction.



Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception.



Adverse Reactions


The most common (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count.


Drug Interactions



Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.



Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.



Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin.



Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid.



Use in Specific Populations



Lactation: Advise not to breastfeed.



Fertility: May impair fertility in males and females.



Click here for full Prescribing Information.


About Verastem Oncology

Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI™ FAKZYNJA™ CO-PACK in the U.S. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn.


Forward-Looking Statements

This press release includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as “anticipate,” “expect,” “plan,” “could,” “may,” “believe,” “estimate,” “forecast,” “goal,” “project,” and other words of similar meaning. Such forward-looking statements address various matters about, among other things, Verastem Oncology’s programs and product candidates, strategy, future plans and prospects, including statements related to the potential for and timing of commercialization of product candidates, the expected outcome and benefits of the Company’s collaboration with GenFleet Therapeutics (Shanghai), Inc., the timing of commencing and completing trials and compiling data, the expected timing of the presentation of data by the Company and the potential clinical value of various of the Company’s clinical trials. Each forward-looking statement contained in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others: the uncertainties inherent in research and development, such as the possibility of negative or unexpected results of clinical trials; that we may not see a return on investment on the payments we have and may continue to make pursuant to the collaboration and option agreement with GenFleet, or that GenFleet may fail to fully perform under the agreement; that we may not be successful in our continued commercialization of AVMAPKI FAKZYNJA CO-PACK; that the development and commercialization of our product candidates may take longer or cost more than planned, including as a result of conducting additional studies or our decisions regarding execution of such commercialization; that data may not be available when expected; risks associated with preliminary and interim data, which may not be representative of more mature data; risks associated with the recent changes in administration policy or actions that may create regulatory uncertainty that may adversely affect our business; risks associated with the current administration’s reductions to the FDA’s workforce and any subsequent reductions that may lead to disruptions and delays in the FDA’s review and oversight of our product candidates and impact the FDA’s ability to provide timely feedback on our development programs; that our product candidates may not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients; and the risks identified under the heading "Risk Factors" as detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, as filed with the Securities and Exchange Commission (SEC) on March 4, 2026, as well as the other information we file with the SEC, are possibly realized. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at www.sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of these statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260317686920/en/
For Investor and Media Inquiries:

Julissa Viana

Vice President, Corporate Communications,

Investor Relations & Patient Advocacy

investors@verastem.com or

media@verastem.com


Original: Verastem Oncology Announces Abstracts Accepted for Presentation on AVMAPKI™ FAKZYNJA™ (avutometinib capsules; defactinib tablets) Combination Therapy at the Society of Gynecologic Oncology 2026 Annual Meeting on Women’s Cancers

Verastem Oncology Reports Fourth Quarter and Full Year 2025 Financial Results and Highlights Recent Business UpdatesMarch 4, 2026 4:01 PM
Business Wire
AVMAPKI™ FAKZYNJA™ CO-PACK net product revenues of $17.5 million for the fourth quarter of 2025 and $30.9 million for the full year 2025, following accelerated U.S. FDA approval in May 2025


Based on FDA guidance, Company to develop Phase 2 registration-directed protocols to evaluate VS-7375, a highly selective, oral KRAS G12D (ON/OFF) inhibitor with best-in-class potential, in 2L PDAC, 2L/3L NSCLC and 2L+ CRC in combination with cetuximab


Cleared multiple dose levels of VS-7375 with no DLTs, continuing dose escalation to 1200 mg QD; cleared 600 mg QD dose level of VS-7375 in combination with cetuximab with no DLTs; continuing higher dose evaluations


Company cash, cash equivalents, and investments of $205 million as of December 31, 2025; pro-forma year-end cash, cash equivalents and investments of $234 million inclusive of net proceeds from exercise of expiring cash warrants; expected cash runway into first half of 2027


Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today reported financial results for the three months and full year ended December 31, 2025, and highlighted recent progress.


“2025 was a transformative year for us, highlighted by the landmark FDA approval of AVMAPKI FAKZYNJA CO-PACK, the only medicine approved to specifically treat KRAS-mutated recurrent LGSOC. The launch is off to a strong start, and this novel-novel combination therapy is gaining positive response across the LGSOC community with gynecologic and medical oncologists in both academic and community settings increasingly turning to it when patients experience a first or subsequent recurrence. We also made considerable progress with VS-7375, our oral, KRAS G12D (ON/OFF) inhibitor with best-in-class potential for solid tumor cancers, clearing multiple dose levels across both monotherapy and cetuximab combination cohorts with no DLTs or major toxicities. Building on the insights from the China data, the tolerability profile that is emerging with VS-7375 in the U.S. has shown meaningful improvement and supports continued dose escalation,” said Dan Paterson, president and chief executive officer at Verastem Oncology. “In 2026, our priorities are to continue driving a strong launch of AVMAPKI FAKZYNJA CO-PACK to fuel sustainable growth, while we continue to accelerate the clinical development of VS-7375 and breakout Phase 2 registration-directed clinical trials in pancreatic, lung, and colorectal cancers.”


Fourth Quarter 2025 and Recent Highlights


AVMAPKI™ FAKZYNJA™ CO-PACK (avutometinib capsules; defactinib tablets) U.S. Launch



AVMAPKI FAKZYNJA CO-PACK generated net product revenues of $17.5 million for the fourth quarter of 2025 and $30.9 million for the full year 2025, following accelerated U.S. Food and Drug Administration (FDA) approval in May 2025, approximately two months ahead of its Prescription Drug User Fee Act (PDUFA) action date of June 30, 2025.



On February 4, 2026, the Company announced updated data for RAMP 201J in Japan with a data cutoff of January 30, 2026. Of the 16 patients enrolled with a median follow-up of 10 months, a confirmed overall response rate (ORR) of 38% (6/16) was achieved by investigator assessment. Among patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC), the confirmed ORR was 57% (4/7) and the disease control rate (DCR) was 100% (7/7). Among patients with KRAS wild-type recurrent LGSOC, the confirmed ORR was 22% (2/9) and the DCR was 89% (8/9). Of the 16 patients enrolled, 11 patients remain on treatment. No patients discontinued due to an adverse event. The safety profile was similar to previously reported data outside of Japan. Steady-state exposures of avutometinib and defactinib in the RAMP 201J study were comparable to those seen in RAMP 201.



On February 25, 2026, the annual update of the National Comprehensive Cancer Network® (NCCN®) Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Ovarian Cancer was released. The Guidelines did not expand the recommendation for avutometinib plus defactinib to include patients with recurrent LGSOC without a KRAS mutation. The Guidelines retained the category 2A recommendation for avutometinib plus defactinib for patients with KRAS-mutated recurrent LGSOC.
“We are disappointed for the patients with KRAS wild-type recurrent LGSOC, who currently have no targeted, FDA-approved treatment options specifically for their disease and face a particularly poor prognosis. Across three separate clinical trials (the FRAME study, RAMP 201, and RAMP 201J) we have observed what we believe are robust objective responses rates for patients with recurrent LGSOC with and without KRAS mutations. We remain committed to advancing the clinical evidence through longer term follow-up analyses from the RAMP 201 study planned for the SGO annual meeting, and completing our ongoing confirmatory RAMP 301 Phase 3 clinical trial, which includes patients with and without KRAS mutations, and look forward to sharing these data with the NCCN and the medical community to support future guideline consideration,” said John Hayslip, chief medical officer at Verastem Oncology.



Expected Key Milestones:



Maximize adoption of AVMAPKI FAKZYNJA CO-PACK in the U.S. as the treatment of choice at the earliest recurrence, leveraging its robust clinical data.



Report a topline readout of the primary endpoint in the RAMP 301 trial in mid-2027.



Continue to pursue regulatory paths for potential expansion into Europe and Japan.



VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor in Advanced Solid Tumors



The Company today announced an update on its progress with the VS-7375-101 Phase 1/2 study:


After clearing the 900 mg daily (QD) dose level with no dose-limiting toxicities (DLTs), the dose escalation phase will continue to 1200 mg QD to further interrogate the dose range and characterize the safety, tolerability, and efficacy profile of VS-7375.



The 600 mg QD dose level of VS-7375 in combination with cetuximab was cleared with no DLTs and higher doses are now being evaluated.



In a pharmacokinetics (PK) analysis, doses of VS-7375 at 600 mg QD and above, with feeding and anti-emetic prophylaxis, yielded similar exposures to fasted patients in China. The exposures achieved cover the exposures in preclinical models necessary for maximal anti-tumor efficacy.



As of the January 30, 2026 data cutoff, VS-7375 demonstrated an encouraging safety profile and was generally well-tolerated across all monotherapy dose levels evaluated to date. Patients (n=23) receiving VS-7375 at either 400 mg QD, 600 mg QD or 900 mg QD with a mean duration of therapy of 1.6 months (0.7-5.6), reported no drug related liver function test abnormalities. There was no drug-related neutropenia greater than Grade 2 and rates of nausea, vomiting and diarrhea remained lower than those reported by the Company’s partner in China. No DLTs have been reported to date, and the maximum tolerated dose has not been reached.



Following recent feedback from the FDA, the Company is amending the VS-7375-101 Phase 1/2 protocol to separate out disease-specific Phase 2 registration-directed trials for KRAS G12D mutated 2L pancreatic ductal adenocarcinoma (PDAC) and 2L/3L non-small cell lung cancer (NSCLC) (monotherapy) and 2L+ colorectal cancer (CRC) in combination with cetuximab.






In January 2026, the Company reported updates on its VS-7375-101 trial including that it had cleared the 400 mg QD, 600 mg QD and 900 mg QD dose levels with no DLTs and no major toxicities. The VS-7375 monotherapy expansion cohorts were initiated, and the cohort sizes were expanded in 2L PDAC, 2L/3L NSCLC, and 2L+ other KRAS G12D-mutated solid tumors. In the VS-7375 dose-escalation combination cohort, the 400 mg QD dose was cleared in combination with cetuximab with no DLTs. The combination dose escalation cohorts were initiated in 1L NSCLC and 2L PDAC at the end of 2025.



In October 2025, the Company announced a preliminary update on the Phase 1/2 monotherapy dose escalation trial of VS-7375 in patients with previously treated advanced KRAS G12D mutant solid tumors. In the study, VS-7375 cleared both the 400 mg QD and the 600 mg QD monotherapy doses with no DLTs observed. At the two dose levels evaluated in the U.S. cohort, no nausea, vomiting, or diarrhea greater than Grade 1 were reported. In addition, no new safety signals have been observed relative to earlier data presentations in both PDAC and NSCLC by GenFleet Therapeutics, the Company’s partner in China. Of the five efficacy evaluable patients in the VS-7375-101 study with at least one scan, four out of five patients have had a tumor reduction and were still on treatment.



The Company shared multiple updates from GenFleet and its ongoing evaluation of VS-7375, known as GFH375, in China:



On March 2, 2026, GenFleet announced that GFH375 was granted its first Breakthrough Therapy Designation in China for patients with KRAS G12D-mutated NSCLC who have received prior systemic therapy.



In December 2025, GenFleet announced the initiation of a registrational Phase 3 study for GFH375 in patients with pretreated KRAS G12D-mutated metastatic pancreatic cancer in China.



In October 2025, the Company announced updated data for GFH375 in PDAC featured in a late-breaking oral presentation at the European Society for Medical Oncology (ESMO) Congress. GenFleet shared additional analyses of this data set on October 27, 2025:


In a subgroup analysis, 12 patients with 2L PDAC at 600 mg QD achieved an ORR of 58.3% and a DCR of 100%. In the 3L+ setting, 47 PDAC patients receiving 600 mg QD achieved an ORR of 36.2% and a DCR of 95.7%. In the 2L subgroup, the median progression free survival (mPFS) and median overall survival (mOS) have not been reached. An additional analysis of gastrointestinal disorders, hematological toxicities, and liver enzyme abnormalities in 2L+ patients with PDAC (n=66) at 600 mg QD showed no adverse events Grade ≥3 occurred at rates above 8.0%.



In an analysis of pre-treated patients with NSCLC at 600 mg QD, the four-month PFS rate was greater than 75% and the mPFS has not been reached. The median follow-up time was 4.2 months.






In October 2025, GenFleet announced that the first patient has been dosed in a Phase 1b/2 study of GFH375 combined with cetuximab or chemotherapy for advanced solid tumors, including 1L PDAC, in China.



In August 2025, the Company announced data of GFH375 would be featured in a mini oral presentation at the IASLC 2025 World Conference on Lung Cancer (WCLC) on September 8, 2025. At the recommended Phase 2 dose (RP2D) of 600 mg QD, the ORR was 68.8% (11/16) (both confirmed and unconfirmed) and the DCR was 93.8% (15/16). Among the 26 evaluable patients with NSCLC treated across all dose levels, the ORR was 57.7% (15/26) (both confirmed and unconfirmed) and the DCR was 88.5% (23/26).



Expected Key Milestones:



Report early data from the VS-7375-101 trial in 1H 2026.



Select the RP2D with cetuximab and initiate the CRC combination expansion cohort in 1H 2026.



Complete enrollment in combination dose-escalation cohorts in mid-2026.



Complete enrollment in monotherapy expansion cohorts in 2H 2026.



Select the RP2D and plan to initiate the PDAC and NSCLC combination expansion cohorts in 2H 2026.



RAMP 205: Avutometinib Plus Defactinib in Combination with Chemotherapy in 1L Metastatic Pancreatic Cancer



In November 2025, the Company announced that enrollment was completed in the expansion cohort in Q3 2025.



Expected Key Milestone:



Report an update on the safety and efficacy of the RAMP 205 expansion cohort with at least six months of follow-up on all patients in Q2 2026.



Upcoming Presentations



Multiple abstracts were selected for oral and poster presentations at the Society of Gynecologic Oncology (SGO) 2026 Annual Meeting on Women’s Cancer on April 10-13 in Puerto Rico. These presentations will include a late-breaking oral presentation on the long-term analysis of the Phase 2 RAMP 201 trial of avutometinib and defactinib combination in recurrent LGSOC.



Corporate Updates



In December 2025, the Company announced John Johnson, current board member, was appointed to chairman of Verastem’s Board of Directors, and Michael Kauffman, M.D., Ph.D., lead director since 2016, was appointed to president of development of Verastem.



In November 2025, the Company announced it had completed a public offering of over $96.9 million of common stock and pre-funded warrants.



Fourth Quarter 2025 Financial Results


Verastem Oncology ended the fourth quarter of 2025 with cash, cash equivalents, and investments of $205 million. On a pro forma basis, taking into account the net proceeds from the exercise of warrants in January 2026 of $29.4 million, cash, cash equivalents, and investments were $234.4 million as of December 31, 2025. These additional sources of capital along with the existing cash, cash equivalents, and investments and ongoing product revenue provide an expected cash runway into first half of 2027.


Net product revenue for the three months ended December 31, 2025 (the “2025 Quarter”) was $17.5 million, compared to no revenue recognized for the three months ended December 31, 2024 (the “2024 Quarter”). The Company began commercial sales of the AVMAPKI FAKZYNJA CO-PACK within the U.S. following receipt of FDA approval in May 2025.


Total operating expenses for the 2025 Quarter were $59.0 million, compared to $31.6 million for the 2024 Quarter. Cost of sales associated with product revenue was $2.9 million for the 2025 Quarter, compared to no cost of sales recognized for the 2024 Quarter.


Research & development expenses for the 2025 Quarter were $31.7 million, compared to $20.8 million for the 2024 Quarter. The increase of $10.9 million, or 52.4%, was primarily due to higher costs incurred for drug substance and drug product manufacturing, contract research organizations, and investigator fees.


Selling, general & administrative expenses for the 2025 Quarter were $24.4 million, compared to $10.8 million for the 2024 Quarter. The increase of $13.6 million, or 125.9%, was primarily due to commercialization costs, including consulting, personnel costs, and professional fees, incurred in connection with the launch of AVMAPKI FAKZYNJA CO-PACK in KRAS-mutated recurrent LGSOC.


Net loss (GAAP basis) for the 2025 Quarter was $32.9 million, or $0.39 per share (basic), compared to $64.6 million, or $1.33 per share (basic and diluted) for the 2024 Quarter.


For the 2025 Quarter, non-GAAP adjusted net loss was $39.8 million, or $0.48 per share (basic) compared to non-GAAP adjusted net loss of $29.3 million, or $0.60 per share (basic), for the 2024 Quarter. Please refer to the GAAP to non-GAAP Reconciliation attached to this press release.


Full-Year 2025 Financial Results


Net product revenue for the year ended December 31, 2025 (the “2025 Period”) was $30.9 million, compared to no product revenue recognized for the year ended December 31, 2024 (the “2024 Period”). Sale of COPIKTRA license and related assets revenue was $0.0 million for the 2025 Period, compared to $10.0 million for the 2024 Period. Revenue for the 2024 Period was comprised of one sales milestone payment of $10.0 million due upon Secura Bio achieving cumulative worldwide net sales of COPIKTRA exceeding $100.0 million.


Total operating expenses for the 2025 Period were $201.0 million, compared to $125.0 million for the 2024 Period. Cost of sales associated with product revenue was $5.3 million for the 2025 period, compared to no cost of sales recognized for the 2024 Period.


Research & development expenses for the 2025 Period were $114.6 million, compared to $81.3 million for the 2024 Period. The increase of $33.3 million, or 41.0%, was primarily due to higher costs incurred for contract research organizations, investigator fees, and drug substance and drug product manufacturing.


Selling, general & administrative expenses for the 2025 Period were $81.1 million, compared to $43.6 million for the 2024 Period. The increase of $37.5 million, or 86.0%, was primarily due to commercialization costs, including consulting, personnel costs, and professional fees, incurred in connection with the launch of AVMAPKI FAKZYNJA CO-PACK in KRAS-mutated recurrent LGSOC.


Net loss for the 2025 Period was $209.5 million, or $3.02 per share (basic and diluted), compared to $130.6 million, or $3.66 per share (basic and diluted) for the 2024 period.


For the 2025 Period, non-GAAP adjusted net loss was $163.1 million, or $2.35 per share (basic) compared to non-GAAP adjusted net loss of $107.4 million, or $3.01 per share (basic), for the 2024 Period. Please refer to the GAAP to non-GAAP Reconciliation attached to this press release.


Conference Call and Webcast


Verastem will host a conference call and webcast today at 4:30 p.m. ET to review the fourth quarter and full year 2025 financial results and recent business updates. To access the conference call, please dial (888) 596-4144 (U.S.) or (646) 968-2525 (international) and enter the passcode 7321921 at least 10 minutes prior to the event start time. A live audio webcast of the call, along with accompanying slides, will be available under "Events & Presentations" in the Investor section of the Company's website, https://investor.verastem.com/events. A replay of the webcast will be archived and available following the event.


Use of Non-GAAP Financial Measures


To supplement Verastem Oncology’s condensed consolidated financial statements, which are prepared and presented in accordance with generally accepted accounting principles in the United States (GAAP), the Company uses the following non-GAAP financial measures in this press release: non-GAAP adjusted net loss and non-GAAP net loss per share. These non-GAAP financial measures exclude certain amounts or expenses from the corresponding financial measures determined in accordance with GAAP.


Management believes this non-GAAP information is useful for investors, taken in conjunction with the Company’s GAAP financial statements, because it provides greater transparency and period-over- period comparability with respect to the Company’s operating performance and can enhance investors’ ability to identify operating trends in the Company’s business. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of the Company’s operating results as reported under GAAP, not in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures for the three months and year ended December 31, 2025, and 2024 are included in the tables accompanying this press release after the unaudited condensed consolidated financial statements.


About AVMAPKI and FAKZYNJA Combination Therapy


AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors.


The U.S. Food and Drug Administration (FDA) approved AVMAPKI™ FAKZYNJA™ CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Verastem is conducting RAMP 301 (GOG-3097/ENGOT-ov81/GTG-UK) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC) with and without a KRAS mutation. Verastem is also evaluating avutometinib plus defactinib with standard-of-care chemotherapy as a potential treatment in the first-line for patients with advanced pancreatic cancer (RAMP 205; NCT05669482). Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on a stand-alone basis for any use.


AVMAPKI FAKZYNJA CO-PACK U.S. Indication


Indication


AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.


This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.


Important Safety Information


Warnings and Precautions



Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity.



Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration.



Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality.



Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction.



Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception.



Adverse Reactions


The most common (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count.


Drug Interactions



Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.



Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.



Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin.



Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid.



Use in Specific Populations



Lactation: Advise not to breastfeed.



Fertility: May impair fertility in males and females.



Click here for full Prescribing Information.


About VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor


VS-7375 is a potential best-in-class, potent, and selective oral KRAS G12D dual ON/OFF inhibitor. VS-7375 is the lead program from the Verastem Oncology discovery and development collaboration with GenFleet Therapeutics. Verastem initiated VS-7375-101, an international Phase 1/2 clinical trial, in June of 2025 in the U.S., that is evaluating the safety and efficacy of VS-7375 in patients with advanced KRAS G12D mutant solid tumors. In July 2025, U.S. Food and Drug Administration (FDA) granted Fast Track Designation (FTD) to VS-7375 for the first-line treatment of patients with KRAS G12D-mutated locally advanced or metastatic adenocarcinoma of the pancreas (PDAC) and for the treatment of patients with KRAS G12D-mutated locally advanced or metastatic PDAC who have received at least one prior line of standard systemic therapy.


About the GenFleet Therapeutics Collaboration


The collaboration with GenFleet Therapeutics aims to advance three oncology discovery programs related to RAS/MAPK pathway-driven cancers. The collaboration provides Verastem with an exclusive option to obtain a license for each of the three compounds in the collaboration after the successful completion of pre-determined milestones in a Phase 1 trial. Verastem selected VS-7375 (also known as GFH375), an oral KRAS G12D (ON/OFF) inhibitor, as its lead program in December 2023 and the license for VS-7375 that was exercised in January 2025 is the first one from this collaboration. The licenses would give Verastem development and commercialization rights outside the GenFleet markets of mainland China, Hong Kong, Macau, and Taiwan.


About Verastem Oncology


Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI™ FAKZYNJA™ CO-PACK in the U.S. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn.


Forward-Looking Statements


This press release includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as “anticipate,” “expect,” “plan,” “could,” “may,” “believe,” “estimate,” “forecast,” “goal,” “project,” and other words of similar meaning. Such forward-looking statements address various matters about, among other things, Verastem Oncology’s programs and product candidates, strategy, future plans and prospects, including statements related to the potential for and timing of commercialization of product candidates, the anticipated timing for the IND application for VS-7375/GFH375, the expected outcome and benefits of the Company’s collaboration with GenFleet Therapeutics (Shanghai), Inc., the timing of commencing and completing trials and compiling data, the expected timing of the presentation of data by the Company and the potential clinical value of various of the Company’s clinical trials. Each forward-looking statement contained in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others: the uncertainties inherent in research and development, such as the possibility of negative or unexpected results of clinical trials; that we may not see a return on investment on the payments we have and may continue to make pursuant to the collaboration and option agreement with GenFleet, or that GenFleet may fail to fully perform under the agreement; that we may not be successful in our continued commercialization of AVMAPKI FAKZYNJA CO-PACK; that the development and commercialization of our product candidates may take longer or cost more than planned, including as a result of conducting additional studies or our decisions regarding execution of such commercialization; that data may not be available when expected; risks associated with preliminary and interim data, which may not be representative of more mature data; risks associated with the recent changes in administration policy or actions that may create regulatory uncertainty that may adversely affect our business; risks associated with the current administration’s reductions to the FDA’s workforce and any subsequent reductions that may lead to disruptions and delays in the FDA’s review and oversight of our product candidates and impact the FDA’s ability to provide timely feedback on our development programs; that our product candidates may not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients; and the risks identified under the heading "Risk Factors" as detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, as filed with the Securities and Exchange Commission (SEC) on March 4, 2026, as well as the other information we file with the SEC, are possibly realized. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at www.sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of these statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.




Verastem Oncology




Condensed Consolidated Balance Sheets




(in thousands)










 




December 31, 2025







December 31, 2024












 



Cash, cash equivalents, & investments






$






204,990







$






88,818








Accounts receivable, net






 






8,813






 






 






—








Inventory






 






1,833






 






 






—








Grants receivable







200








200








Prepaid expenses and other current assets







7,577








5,943








Property and equipment, net







—








32








Right-of-use asset, net







491








1,405








Intangible assets, net






 






16,426






 






 






—








Restricted cash and other assets







6,112








5,140








Total assets






$






246,442







$






101,538












 



Current Liabilities







72,268








30,973








Long term debt







76,330








40,724








Vendor financing arrangement, long-term






 






5,000






 






 






—








Lease liability, long-term






 






—






 






 






535








Warrant Liability







35,647








58,199








Stockholders’ equity







57,197








(28,893)








Total liabilities, convertible preferred stock and stockholders’ equity






$






246,442







$






101,538









Verastem Oncology




Condensed Consolidated Statements of Operations




(in thousands, except per share amounts)




(unaudited)


















 




Three months ended December 31,







Year ended December 31,









2025







2024







2025







2024








Revenue:



















Product Revenue, net






$






17,535







$






—







$






30,914







$






—








Sale of COPIKTRA license and related assets







—








—








—








10,000








Total revenue






 






17,535







 






—







 






30,914







 






10,000








Operating expenses:



















Cost of sales – product






 






2,611







 






—







 






4,600







 






—








Cost of sales – intangible amortization






 






280







 






—







 






698







 






—








Research and development







31,675








20,811








114,599








81,334








Selling, general and administrative






 






24,443







 






10,779







 






81,146







 






43,622








Total operating expenses






 






59,009







 






31,590







 






201,043







 






124,956








Loss from operations






 






(41,474)







 






(31,590)







 






(170,129)







 






(114,956)








Other income (expense)







(18)








9








(203)








(123)








Interest income






 






1,103







 






968







 






4,068







 






4,149








Interest expense






 






(415)







 






(1,146)







 






(1,138)







 






(4,562)








Loss on debt extinguishment






 






—







 






—







 






(1,826)







 






—








Change in fair value of preferred stock tranche liability







—








—







 






—








4,189








Change in fair value of warrant liability







10,485








(32,606)








(27,492)








(19,149)








Change in fair value of Notes






 






(2,597)







 






—







 






(12,751)







 






—








Net loss before taxes







(32,916)








(64,365)








(209,471)








(130,452)








Income tax expense







—








(185)








—








(185)








Net Loss






$






(32,916)







$






(64,550)







$






(209,471)







$






(130,637)








Net loss per share—basic






$






(0.39)







$






(1.33)







$






(3.02)







$






(3.66)








Net loss per share—diluted






$






(0.50)







$






(1.33)







$






(3.02)







$






(3.66)








Weighted average common shares outstanding used in computing:







 








 








 








 








Net loss per share – basic







83,400








48,709








69,309








35,713








Net loss per share – diluted






 






86,710







 






48,709







 






69,309







 






35,713









Verastem Oncology




Reconciliation of GAAP to Non-GAAP Financial Information




(in thousands, except per share amounts)




(unaudited)


















 




Three months ended December 31,







Year ended December 31,









2025







2024







2025







2024








Net loss reconciliation:



















Net loss (GAAP basis)






$






(32,916)







$






(64,550)







$






(209,471)







$






(130,637)








Adjust:






 


















Stock-based compensation expense






 






2,025







 






2,019







 






9,404







 






7,342








Amortization of acquired intangible assets






 






280








—








698








—








Non-cash interest, net






 






—







 






207







 






29







 






(5)








Change in fair value of preferred stock tranche liability






 






—







 






—







 






—







 






(4,189)








Change in fair value of warrant liability






 






(10,485)








32,606








27,492








19,149








Non-cash change in fair value of Notes






 






890







 






—







 






6,560







 






—








Loss on debt extinguishment






 






—







 






—







 






1,826







 






—








Severance and other






 






392







 






371







 






392







 






990








Adjusted net loss (non-GAAP basis)






$






(39,814)







$






(29,347)







$






(163,070)







$






(107,350)








 






 






 







 






 







 






 







 






 








Reconciliation of net loss per share






 






 







 






 







 






 







 






 








Net loss per share – basic (GAAP basis)






 






(0.39)







$






(1.33)







$






(3.02)







$






(3.66)








Adjust per basic share






 






 







 






 







 






 







 






 








Stock-based compensation expense






 






0.02







 






0.04







 






0.14







 






0.21








Amortization of acquired intangible assets






 






—







 






—







 






0.01







 






—








Non-cash interest, net






 






—







 






0.01







 






—







 






—








Change in fair value of preferred stock tranche liability






 






—







 






—







 






—







 






(0.12)








Change in fair value of warrant liability






 






(0.12)







 






0.67







 






0.39







 






0.53








Non-cash change in fair value of Notes






 






0.01







 






—







 






0.09







 






—








Loss on debt extinguishment






 






—







 






—







 






0.03







 






—








Severance and other






 






—







 






0.01







 






0.01







 






0.03








Adjusted net loss per share – basic (non-GAAP basis)






$






(0.48)







$






(0.60)







$






(2.35)







$






(3.01)








Weighted average common shares outstanding used in computing net loss per share—basic






 






83,400







 






48,709







 






69,309







 






35,713







 

View source version on businesswire.com: https://www.businesswire.com/news/home/20260304011595/en/
For Investor and Media Inquiries:

Julissa Viana

Vice President, Corporate Communications,

Investor Relations & Patient Advocacy

investors@verastem.com or

media@verastem.com


Original: Verastem Oncology Reports Fourth Quarter and Full Year 2025 Financial Results and Highlights Recent Business Updates

Verastem Oncology Provides Preliminary Fourth Quarter and 2025 Revenue and Business Updates and Outlines 2026 Strategic Priorities for Novel Portfolio Targeting RAS/MAPK Pathway-Driven CancersFebruary 4, 2026 7:30 AM
Business Wire
Based on preliminary, unaudited results, Verastem expects AVMAPKI™ FAKZYNJA™ CO-PACK net product revenues of approximately $17.5 million for the fourth quarter of 2025 and approximately $30.9 million for the full year 2025, following U.S. FDA approval in May 20251


Company plans to continue rapidly advancing clinical development program for VS-7375, a highly selective, oral KRAS G12D (ON/OFF) inhibitor with best-in-class potential, in several advanced KRAS G12D solid tumor indications


Company sees preliminary unaudited cash, cash equivalents, and investments of $205 million as of December 31, 2025; pro-forma year-end cash, cash equivalents and investments of $234 million inclusive of net proceeds from exercise of expiring cash warrants; expected cash runway into first half of 2027


Leadership to participate in a fireside chat at the Guggenheim Emerging Outlook Conference in New York City on February 11, 2026


Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced preliminary, unaudited fourth quarter and full year 2025 net product revenues for AVMAPKI™ FAKZYNJA™ CO-PACK, business updates, and 2026 priorities.


"2025 was a transformative year for Verastem Oncology and the patients we serve. We transitioned to a commercial-stage company with the launch of AVMAPKI FAKZYNJA CO-PACK, the first treatment specifically approved by the FDA for KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC). We also advanced multiple clinical programs across several RAS/MAPK pathway-driven solid tumor cancers,” said Dan Paterson, president and chief executive officer at Verastem Oncology. “In 2026, we are well-positioned to drive sustainable growth with our successful commercial launch and to accelerate our clinical path for VS-7375, our potential best-in-class oral KRAS G12D (ON/OFF) inhibitor, with several important data readouts and preparations for potential registration-directed clinical trials.”


Financial Update1



Based on preliminary, unaudited results, Verastem expects AVMAPKI FAKZYNJA CO-PACK net product revenues of approximately $17.5 million for the fourth quarter of 2025 and approximately $30.9 million for the full year 2025. Full year 2025 net product revenue reflects the launch period of May, following U.S. Food and Drug Administration (FDA) approval, through December 2025.



The Company also announced that as of January 25, 2026 outstanding cash warrants had been exercised, netting $29.4 million. No cash exercise warrants remain outstanding.



As of December 31, 2025, the Company had cash, cash equivalents, and investments (unaudited) of $205 million; on a pro forma basis, taking into consideration the net proceeds of the cash exercise warrants, cash, cash equivalents and investments were $234 million. With its ongoing product revenue, recent equity financing, and exercise of the remaining cash warrants, the Company expects its cash runway to extend into the first half of 2027.



Given the growth trajectory of the AVMAPKI FAKZYNJA CO-PACK, Verastem anticipates the LGSOC commercial launch and development program will be self-sustaining by the second half of 2026.



2026 Priorities


In 2026, Verastem will continue to focus on maximizing the commercial launch of AVMAPKI FAKZYNJA CO-PACK while advancing its differentiated pipeline targeting RAS/MAPK-pathway driven cancers to create sustainable long-term growth:



AVMAPKI FAKZYNJA CO-PACK (avutometinib; defactinib) – the first treatment specifically approved by the FDA for adults with KRAS-mutated recurrent LGSOC who have received prior systemic therapy. The combination is being evaluated in an ongoing international Phase 3 trial, RAMP 301, in recurrent LGSOC with or without a KRAS mutation. The trial is fully enrolled, as of December 2025, and will serve as a confirmatory study for the initial indication and has the potential to expand the indication regardless of KRAS mutation status. The results will also be leveraged for potential geographic expansion.
The Company announced today updated data from the ongoing RAMP201J Phase 2 clinical trial in Japan evaluating the combination in patients with LGSOC with or without a KRAS mutation. As of the data cutoff on January 30, 2026, 16 patients were efficacy evaluable by investigator assessment, having at least one post-baseline assessment with a median follow-up of 10 months. Of the 16 total patients, a confirmed overall response rate (ORR) of 38% (6/16) was achieved. Among patients with KRAS-mutated recurrent LGSOC, the confirmed ORR was 57% (4/7) and the disease control rate (DCR) was 100% (7/7). Among patients with KRAS wild-type recurrent LGSOC, the confirmed ORR was 22% (2/9) and the DCR was 89% (8/9). Of the 16 patients enrolled, 11 patients remain on treatment. No patients discontinued due to an adverse event. The safety profile was similar to previously reported data.
Verastem expects to:


Maximize adoption of AVMAPKI FAKZYNJA CO-PACK in the U.S. as the treatment of choice at the earliest recurrence, leveraging its robust clinical data.



Report a topline readout of the primary endpoint in the RAMP 301 trial in mid-2027.



Continue to pursue regulatory paths for potential expansion of the product launch into Europe and Japan.





VS-7375 – is an investigational, highly selective and oral KRAS G12D (ON/OFF) inhibitor being evaluated in an international Phase 1/2 trial in advanced KRAS G12D solid tumors. The trial continues to enroll in both the monotherapy dose-escalation and dose-expansion cohorts and multiple dose-escalation combination cohorts in various solid tumors including colorectal cancer (CRC), pancreatic ductal adenocarcinoma (PDAC), and non-small cell lung cancer (NSCLC).
As previously reported, the Company cleared the 400, 600, and 900 mg once daily (QD) dose levels with no dose-limiting toxicities (DLTs) and no major toxicities. The monotherapy expansion cohorts have been initiated, and the cohort sizes have been expanded in second-line (2L) PDAC, 2L/3L NSCLC, and 2L+ tumor agnostic solid tumors, including biliary tract cancer (BTC) and endometrial cancer. The Company cleared the 400 mg QD dose in combination with cetuximab with no DLTs and is currently evaluating the 600 mg QD dose level with cetuximab. The combination dose escalation cohorts were initiated in first-line (1L) NSCLC and 2L PDAC at the end of 2025.
Verastem expects to:


Report an interim update on the Phase 1/2 trial of VS-7375 in 1H 2026.



Select the recommended Phase 2 dose (RP2D) with cetuximab and initiate the CRC combination expansion cohort in 1H 2026.



Engage with the FDA in 1H 2026 to discuss its development path forward, including potential registration-directed clinical trials in PDAC, NSCLC, and CRC.



Complete enrollment in combination dose-escalation cohorts in mid-2026.



Complete enrollment in monotherapy expansion cohorts in 2H 2026.



Select the RP2D and plan to initiate the PDAC and NSCLC combination expansion cohorts in 2H 2026.





Avutometinib Plus Defactinib – is being evaluated in an ongoing RAMP 205 study in combination with standard-of-care chemotherapy in the front line for patients with metastatic PDAC. At the American Society of Clinical Oncology (ASCO) Annual Meeting in 2025, the Company reported that the first 12 patients enrolled to dose level 1 (DL1) in the RAMP 205 study reported an ORR of 83% (10/12). Seventeen additional patients were enrolled in the DL1 expansion between May and August 2025, and with a minimum follow-up of approximately 4 months, the majority of these patients remain on treatment as of January 2026.
Verastem expects to:


Report an update on the safety and efficacy of the RAMP 205 expansion cohort with at least six months of follow-up on all patients in Q2 2026.






1The information presented above is unaudited and reflects preliminary estimates subject to the completion of financial closing procedures and any adjustments that may result from the finalization of the quarter and annual review of the Company’s financial statements by external auditors. Verastem plans to report the final 4Q2025 and full year 2025 results during its fourth quarter 2025 earnings call in early March 2026.


Guggenheim Emerging Outlook: Biotech Summit


The Company’s leadership will participate in a fireside chat at the Guggenheim Emerging Outlook: Biotech Summit on Wednesday, February 11, 2026, at 2:00 pm ET in New York. A live webcast of the fireside chat can be accessed under “Events & Presentations” on the Company’s website at www.verastem.com. A replay of the webcast will be archived on the website for approximately 90 days following the presentation.


About AVMAPKI and FAKZYNJA Combination Therapy


AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors.


The U.S. Food and Drug Administration (FDA) approved AVMAPKI™ FAKZYNJA™ CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Verastem is conducting RAMP 301 (GOG-3097/ENGOT-ov81/GTG-UK) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC) with and without a KRAS mutation. Verastem is also evaluating avutometinib plus defactinib with standard-of-care chemotherapy as a potential treatment in the first-line for patients with advanced pancreatic cancer (RAMP 205; NCT05669482). Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on a stand-alone basis for any use.


AVMAPKI FAKZYNJA CO-PACK U.S. Indication


Indication


AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.


This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.


Important Safety Information


Warnings and Precautions



Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity.



Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration.



Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality.



Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction.



Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception.



Adverse Reactions


The most common (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count.


Drug Interactions



Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.



Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.



Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin.



Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid.



Use in Specific Populations



Lactation: Advise not to breastfeed.



Fertility: May impair fertility in males and females.



Click here for full Prescribing Information.


About VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor


VS-7375 is a potential best-in-class, potent, and selective oral KRAS G12D dual ON/OFF inhibitor. VS-7375 is the lead program from the Verastem Oncology discovery and development collaboration with GenFleet Therapeutics. Verastem initiated VS-7375-101, an international Phase 1/2 clinical trial, in June of 2025 in the U.S., that is evaluating the safety and efficacy of VS-7375 in patients with advanced KRAS G12D mutant solid tumors. Verastem announced in April 2025 that the U.S. Investigational New Drug (IND) application for VS-7375 was cleared.


About the GenFleet Therapeutics Collaboration


The collaboration with GenFleet Therapeutics aims to advance three oncology discovery programs related to RAS/MAPK pathway-driven cancers. The collaboration provides Verastem with an exclusive option to obtain a license for each of the three compounds in the collaboration after the successful completion of pre-determined milestones in a Phase 1 trial. Verastem selected VS-7375 (also known as GFH375), an oral KRAS G12D (ON/OFF) inhibitor, as its lead program in December 2023 and the license for VS-7375 that was exercised in January 2025 is the first one from this collaboration. The licenses would give Verastem development and commercialization rights outside the GenFleet markets of mainland China, Hong Kong, Macau, and Taiwan.


About Verastem Oncology


Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI™ FAKZYNJA™ CO-PACK in the U.S. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn.


Forward-Looking Statements Notice


This press release includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as “anticipate,” “expect,” “plan,” “could,” “may,” “believe,” “estimate,” “forecast,” “goal,” “project,” and other words of similar meaning. Such forward-looking statements address various matters about, among other things, Verastem Oncology’s programs and product candidates, strategy, future plans and prospects, including statements related to the potential for and timing of commercialization of product candidates, the conduct of the Phase 1/2a study for VS-7375/GFH375, the expected outcome and benefits of the Company’s collaboration with GenFleet Therapeutics (Shanghai), Inc., the timing of commencing and completing trials and compiling data, the expected timing of the presentation of data by the Company and the potential clinical value of various of the Company’s clinical trials. Each forward-looking statement contained in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Applicable risks and uncertainties include, among others: the uncertainties inherent in research and development, such as the possibility of negative or unexpected results of clinical trials; that we may not see a return on investment on the payments we have and may continue to make pursuant to the collaboration and option agreement with GenFleet, or that GenFleet may fail to fully perform under the agreement; that we may not be successful in our continued launch and commercialization of AVMAPKI FAKZYNJA CO-PACK; that the development and commercialization of our product candidates may take longer or cost more than planned, including as a result of conducting additional studies or our decisions regarding execution of such commercialization; that data may not be available when expected; risks associated with preliminary and interim data, which may not be representative of more mature data; risks associated with the recent changes in administration policy or actions that may create regulatory uncertainty that may adversely affect our business; risks associated with the current administration’s reductions to the FDA’s workforce and any subsequent reductions that may lead to disruptions and delays in the FDA’s review and oversight of our product candidates and impact the FDA’s ability to provide timely feedback on our development programs; that our product candidates may not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients; and the risks identified under the heading "Risk Factors" as detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024, as filed with the Securities and Exchange Commission (SEC) on March 20, 2025, as well as the other information we file with the SEC, are possibly realized. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at www.sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of these statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260204097979/en/
For Investor and Media Inquiries:

Julissa Viana

Vice President, Corporate Communications,

Investor Relations & Patient Advocacy

investors@verastem.com or

media@verastem.com


Original: Verastem Oncology Provides Preliminary Fourth Quarter and 2025 Revenue and Business Updates and Outlines 2026 Strategic Priorities for Novel Portfolio Targeting RAS/MAPK Pathway-Driven Cancers

$668B Oncology Surge: 5 Platforms Cracking the Code on GI TumorsJanuary 30, 2026 10:19 AM
PR Newswire (US)

Issued on behalf of Oncolytics Biotech Inc.USANewsGroup.com News CommentaryVANCOUVER, BC, Jan. 30, 2026 /PRNewswire/ -- The global oncology sector is rocketing toward $668 billion in 2034[1], partly fueled by a massive structural pivot toward gastrointestinal cancers where clinical velocity is finally shattering historical benchmarks. With the FDA's 2026 framework explicitly prioritizing accelerated pathways for platforms showing superior objective response rates and durability[2], a select group of innovators is unlocking the immunologically "cold" GI tumors that have baffled researchers for decades. This regulatory tailwind places Oncolytics Biotech Inc. (NASDAQ: ONCY), Pfizer (NYSE: PFE), Verastem Oncology (NASDAQ: VSTM), Perspective Therapeutics (NYSE-A: CATX), and RenovoRx (NASDAQ: RNXT) directly at the intersection of pivotal execution and breakthrough immunotherapy.  As the market charges toward a staggering $668 billion valuation by 2034, capital is rotating aggressively toward execution-ready platforms over speculative assets[3]. Fresh FDA guidance issued in December 2025 and January 2026 signals a decisive new era emphasizing overall survival and robust clinical benefit[4]. This shift creates asymmetric upside for companies demonstrating disciplined execution in high-stakes battlegrounds like colorectal, pancreatic, and anal cancers, where targeted immunotherapies are establishing entirely new treatment paradigms.Oncolytics Biotech Inc. (NASDAQ: ONCY) is building out its leadership team as it moves its cancer treatment pelareorep through late-stage clinical trials targeting several gastrointestinal cancers.The company recently announced two key hires: John McAdory as Executive Vice President of Strategy and Operations, and Yujun Wu as Vice President, Head of Biostatistics. McAdory comes from CG Oncology, where he ran late-stage clinical trials for oncolytic virus therapies (cancer-fighting viruses, similar to pelareorep). Wu joins from Morphic Therapeutic, where he led the statistics department through the company's sale to Eli Lilly, and previously designed multiple Phase 3 oncology trials at Takeda. Both bring the specific expertise needed to execute the complex, multi-country registration trials that determine whether a drug gets FDA approval."John's background running complex, late-stage oncology trials makes him exceptionally well-suited to lead Oncolytics' next phase of execution," said Jared Kelly, CEO of Oncolytics Biotech. "As we progress toward pivotal and registration-enabling studies in anal, pancreatic, and colorectal cancers, his experience will be critical to ensuring disciplined execution, speed, and regulatory alignment."These hires complete a significant management upgrade. Kelly and Chief Business Officer Andrew Aromando both joined from Ambrx Biopharma, which sold to Johnson & Johnson for $2 billion in 2024. The company also added three leading cancer specialists from Memorial Sloan Kettering Cancer Center and MD Anderson Cancer Center to its gastrointestinal tumor scientific advisory board.The clinical data driving this expansion looks compelling. In colorectal cancer, pelareorep combined with chemotherapy and a vascular endothelial growth factor (VEGF) inhibitor achieved a 33% response rate in patients with KRAS mutations (a genetic marker found in about 40% of colorectal cancers). That's three times better than the 6-11% response rate chemotherapy and a VEGF inhibitor typically achieves in these patients. Response rate measures the percentage of patients whose tumors shrink significantly or disappear entirely.In anal cancer, where treatment options are extremely limited after first-line therapy fails, pelareorep is showing even stronger results. Third-line patients (those who've already failed two prior treatments) saw a 29% response rate with responses lasting around 17 months. That nearly triples the historical benchmarks in a setting where no FDA-approved treatments currently exist.In second-line patients, the 30% response rate more than doubled the 13.8% benchmark for the only FDA-approved immunotherapy available, with responses lasting 15.5 months versus 9.5 months for standard treatment.Oncolytics has also secured FDA agreement on its Phase 3 trial design for pancreatic cancer, clearing the way to launch what would be the only immunotherapy registration trial currently planned for this notoriously difficult-to-treat disease.CONTINUED… Read this and more news for Oncolytics Biotech at: https://usanewsgroup.com/2023/10/02/the-most-undervalued-oncolytics-company-on-the-nasdaq/ In other recent industry developments and happenings in the market include:Pfizer (NYSE: PFE) announced positive results from Cohort 3 of the pivotal BREAKWATER trial evaluating BRAFTOVI in combination with cetuximab and FOLFIRI in patients with previously untreated BRAF V600E-mutant metastatic colorectal cancer. The combination achieved a confirmed objective response rate of 64.4% compared to 39.2% with standard-of-care treatment, representing a statistically significant improvement."These data further strengthen the potential utility of BRAFTOVI for patients with BRAF V600E-mutant metastatic colorectal cancer. The significant improvement in response rates reflects the meaningful clinical benefit of this targeted combination therapy regimen for patients," said Jeff Legos, Chief Oncology Officer at Pfizer. "These results underscore the potential of BRAFTOVI as a standard of care for patients with this aggressive cancer."Among patients receiving the treatment regimen, 57.4% on BRAFTOVI plus cetuximab and FOLFIRI maintained response for six months or longer compared to 34.5% with standard therapy. Pfizer continues the BREAKWATER trial with an estimated completion in 2027.Verastem Oncology (NASDAQ: VSTM) announced it will discontinue its RAMP 203 Phase 1/2 trial in advanced KRAS G12C-mutated non-small cell lung cancer (NSCLC) to focus resources on clinical development of VS-7375, an oral KRAS G12D inhibitor. The decision reflects the evolving treatment landscape as next-generation G12C inhibitors establish higher response rate benchmarks."RAMP 203 has provided important insights into treatment strategies and demonstrated proof-of-concept. While avutometinib plus defactinib combined well with a G12C inhibitor to drive early and sustained anti-tumor responses, next generation G12C inhibitors are establishing a new benchmark with higher response rates," said John Hayslip, Chief Medical Officer at Verastem Oncology. "Accordingly, we are prioritizing our clinical development of VS-7375, a potentially best-in-class oral KRAS G12D (ON/OFF) inhibitor, that demonstrated a 69% response rate in advanced KRAS G12D NSCLC."In the doublet combination arm, 30 G12C-inhibitor treatment-naïve patients achieved an overall response rate of 40% with a median progression-free survival of 11.1 months. Verastem Oncology continues advancing RAMP 205, evaluating avutometinib plus defactinib with chemotherapy in first-line metastatic pancreatic cancer.Perspective Therapeutics (NYSE-A: CATX) presented updated interim data from its ongoing Phase 1/2a clinical trial of [212Pb]VMT-a-NET in patients with neuroendocrine tumors at the 2026 ASCO Gastrointestinal Cancers Symposium. The radiopharmaceutical demonstrated a 39% objective response rate in Cohort 2 patients regardless of SSTR2 expression profile, with 76% of patients remaining progression-free and alive."The updated interim results presented at ASCO-GI continue to support VMT-a-NET's compelling overall clinical profile at the 5 mCi dose," said Markus Puhlmann, Chief Medical Officer of Perspective Therapeutics. "With the robust clinical dataset being accrued from strong patient enrollment into our study, we believe we will be able to have meaningful engagement with regulatory agencies during 2026 on proceeding with VMT-a-NET into a registrational trial."The therapy continues to demonstrate favorable tolerability with no dose-limiting toxicities, treatment-related discontinuations, or clinically significant myelosuppression reported across 56 patients. Perspective Therapeutics has completed the Cohort 3 dose-limiting toxicity assessment and is cleared to enroll additional patients at the 6.0 mCi dose level.RenovoRx (NASDAQ: RNXT) announced continued commercial momentum with its RenovoCath FDA-cleared drug-delivery device now integrated at nine active U.S. cancer centers, up from five in September 2025. The company's TAMP therapy platform is designed to deliver chemotherapy near tumor sites, potentially enhancing effectiveness while minimizing systemic side effects associated with traditional intravenous administration."The growing adoption of RenovoCath by esteemed institutions like City of Hope Cancer Center and Moffitt Cancer Center, amongst others, is the driver of our commercial efforts and underscores the clinical need for more targeted oncology tools," said Shaun Bagai, CEO of RenovoRx. "We are encouraged by both the procedural feedback and the strong commercial interest we are seeing."The company generated approximately $900,000 in sales revenue through September 2025 and has strengthened its commercial team to drive growth in 2026. RenovoRx continues enrollment in its Phase III TIGeR-PaC trial evaluating intra-arterial gemcitabine delivery via RenovoCath for locally advanced pancreatic cancer.Source: https://usanewsgroup.com/2024/09/21/is-oncolytics-biotech-the-markets-most-undervalued-cancer-opportunity/ CONTACT:
USA NEWS GROUP
info @acblanke1DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. Please consult a licensed financial advisor before making any investment decision. This is a paid advertisement and is neither an offer nor recommendation to buy or sell any security. We hold no investment licenses and are thus neither licensed nor qualified to provide investment advice. The content in this report or email is not provided to any individual with a view toward their individual circumstances. USA News Group is a wholly-owned subsidiary of Market IQ Media Group, Inc. ("MIQ"). MIQ has been paid a fee for Oncolytics Biotech Inc. advertising and digital media from the company directly. There may be 3rd parties who may have shares of Oncolytics Biotech Inc., and may liquidate their shares which could have a negative effect on the price of the stock. This compensation constitutes a conflict of interest as to our ability to remain objective in our communication regarding the profiled company. Because of this conflict, individuals are strongly encouraged to not use this publication as the basis for any investment decision. The owner/operator of MIQ own shares of Oncolytics Biotech Inc. which were purchased in the open market, and reserve the right to buy and sell, and will buy and sell shares of Oncolytics Biotech Inc. at any time without any further notice commencing immediately and ongoing. We also expect further compensation as an ongoing digital media effort to increase visibility for the company, no further notice will be given, but let this disclaimer serve as notice that all material, including this article, which is disseminated by MIQ has been approved by Oncolytics Biotech Inc.; this is a paid advertisement, we currently own shares of Oncolytics Biotech Inc. and will buy and sell shares of the company in the open market, or through private placements, and/or other investment vehicles.While all information is believed to be reliable, it is not guaranteed by us to be accurate. Individuals should assume that all information contained in our newsletter is not trustworthy unless verified by their own independent research. Also, because events and circumstances frequently do not occur as expected, there will likely be differences between the any predictions and actual results. Always consult a licensed investment professional before making any investment decision. Be extremely careful, investing in securities carries a high degree of risk; you may likely lose some or all of the investment.SOURCES:https://www.precedenceresearch.com/oncology-markethttps://lumanity.com/perspectives/the-8-fda-regulatory-trends-shaping-2026-and-beyond/https://www.globenewswire.com/news-release/2025/12/02/3198196/0/en/Oncology-Market-Size-Worth-USD-668-26-Billion-by-2034.htmlhttps://www.fda.gov/vaccines-blood-biologics/biologics-guidances/recently-issued-guidance-documents Logo: https://mma.prnewswire.com/media/2838876/USA_News_Group_Logo.jpg





View original content to download multimedia:https://www.prnewswire.com/news-releases/668b-oncology-surge-5-platforms-cracking-the-code-on-gi-tumors-302675135.html

Original: $668B Oncology Surge: 5 Platforms Cracking the Code on GI Tumors

Verastem Oncology Announces Updated Data from Partner GenFleet Therapeutics’ Phase 1/2 Monotherapy Study in China of GFH375 (VS-7375) in Advanced KRAS G12D Mutant Pancreatic Ductal Adenocarcinoma

https://investor.verastem.com/news-releases/news-release-details/verastem-oncology-announces-updated-data-partner-genfleet-0

I have been following this pineline for years. Pancreatic is a Huge breakthru with any success towards unsolvable diseases. This reminds me of IMMU and IMGN. Only time before it gets bought. They have also had 2 approved drugs tgey know the protocol imo

Given the pipeline and the recent gains and volume, is VSTM a candidate for an early buy-out, like Verona's too early sale to Merck?

Does anyone have any thoughts? Mine are that Merck might be in the weeds again.

$ 8.31 Updated Data from RAMP 205 Phase 1/2 Clinical Trial in Frontline Metastatic PDAC


As of April 25, 2025, 60 patients (12 per cohort) had been treated in one of five dose regimens with the combination of avutometinib and defactinib with gemcitabine and Nab-paclitaxel in frontline metastatic PDAC. In the dose level 1 cohort, 12 patients received 2.4 mg of avutometinib twice a week (BIW), 200 mg of defactinib twice a day (BID) for 3 weeks out of every 4 and 800 mg/m2 of gemcitabine and 125 mg/m2 of Nab-paclitaxel on a schedule of day 1, day 8 and day 15. In dose level 1, 83% (10/12) of patients achieved partial responses (8 confirmed and 2 unconfirmed who remain on treatment). Given these strong and consistent results, the Company has selected dose level 1 as the RP2D, has met the pre-defined criteria to advance beyond the first stage of the expansion study, and is now enrolling up to 29 patients at this dose level.

In evaluating all the dose cohorts, dose level 1 demonstrated the highest response rate and across all five dose cohorts, 92% (48/52) of efficacy evaluable patients showed tumor reduction as best response. Adverse events across all dose cohorts remained generally consistent with the previously announced safety and tolerability profile, and no new safety signals have emerged. While anticipating results from the study expansion cohort, the Company is now developing plans for a registrational Phase 3 front-line metastatic PDAC trial to begin in 2026.

https://investor.verastem.com/news-releases/news-release-details/verastem-oncology-announces-positive-updated-results-ramp-205

$7.46 Updated safety and efficacy results from the RAMP 205 trial of avutometinib and defactinib in combination with current standard of care in first-line metastatic pancreatic cancer to be announced at the 2025 ASCO Annual Meeting

The ASCO 2025 Annual Meeting will be held from May 30 to June 3, 2025

Ended Q1 2025 with $117.6 million in cash and cash equivalents; pro-forma $192.6 million including the equity issuance in the private placement in April 2025

https://www.biospace.com/press-releases/verastem-oncology-reports-first-quarter-2025-financial-results-and-highlights-recent-business-updates

VSTM reports May 16 according to Seeking Alpha

well so far looks like they would have been better off producing videos selllng ED...

vstm........................https://stockcharts.com/h-sc/ui?s=Vstm&p=W&b=5&g=0&id=p86431144783

Verastem Oncology Announces FDA Acceptance and Priority Review of New Drug Application for Avutometinib in Combination with Defactinib for the Treatment of Recurrent KRAS Mutant Low-Grade Serous Ovarian CancerDecember 30 2024 - 4:30PM
Business Wire
PDUFA target action date is June 30, 2025If approved, avutometinib in combination with defactinib would be the first-ever FDA-approved treatment specifically for adults with recurrent KRAS mutant LGSOCVerastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) under the accelerated approval pathway for avutometinib, an oral RAF/MEK clamp, in combination with defactinib, an oral FAK inhibitor, for the treatment of adult patients with recurrent low-grade serous ovarian cancer (LGSOC), who received at least one prior systemic therapy and have a KRAS mutation. The NDA, which was completed in October 2024, has been granted Priority Review with a Prescription Drug User Fee Act (PDUFA) action date of June 30, 2025. In addition, the FDA has stated that it is not currently planning to hold an advisory committee meeting to discuss the application.“The FDA filing acceptance and Priority Review for the combination of avutometinib and defactinib underscores the critical unmet need among patients diagnosed with this rare and insidious disease. We are excited by today’s news and to potentially bring the first ever FDA-approved treatment specifically for recurrent KRAS mutant LGSOC to patients in the U.S.,” said Dan Paterson, president and chief executive officer of Verastem Oncology. “With the acceptance of this NDA, we’re taking an important step forward in addressing a condition that has long been overlooked, and we look forward to working with the FDA during its review process and preparing for a commercial launch in mid-2025.”There are currently no FDA-approved treatments specifically for LGSOC, a rare and distinct ovarian cancer that differs from high-grade serous ovarian cancer in both its biology and how it responds to treatment. Priority Review is granted by the FDA for treatments that offer, if approved, significant improvements over available options or that provide a treatment option where no adequate or approved therapy currently exists.The filing was based on a primary analysis of the Phase 2 RAMP 201 clinical trial that evaluated the combination of avutometinib and defactinib in patients with recurrent LGSOC. The results were presented in an oral presentation at the International Gynecologic Cancer Society (IGCS) Annual Global Meeting in October 2024 and demonstrated that the combination of avutometinib plus defactinib resulted in a substantial overall response rate confirmed by blinded independent central review, with responses that were typically durable, and that the combination was generally well-tolerated in patients with recurrent KRAS mutant LGSOC. The NDA also includes supportive data from the FRAME Phase 1 trial, the first study conducted with the combination therapy in recurrent LGSOC.The Company is currently enrolling patients with recurrent LGSOC regardless of KRAS mutation status for RAMP 301, an international Phase 3 trial, which will serve as a confirmatory study for the initial indication and has the potential to support an expanded indication regardless of KRAS mutation status.About RAMP 201RAMP 201 (ENGOTov60/GOG3052) (NCT04625270) is an adaptive, two-part multicenter, parallel cohort, randomized, open-label Phase 2 registration-directed trial evaluating the efficacy and safety of avutometinib alone and in combination with defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC). The first part of the study (Part A) determined the selection of the go-forward regimen, which was the combination of avutometinib and defactinib versus avutometinib alone, based on overall response rates. The expansion phases of the trial (Parts B and C) are evaluating the safety and efficacy of the go-forward regimen of avutometinib 3.2 mg twice weekly and defactinib 200 mg twice daily. The Part D portion of the trial is evaluating a low dose of avutometinib in combination with defactinib to inform individualized dose reduction.

4.75

VSTM..............................................a/h

stay tuned-----The oral presentation will include updated safety and efficacy data from the RAMP 201 trial evaluating the combination of avutometinib, an oral RAF/MEK clamp, and defactinib, an oral, selective FAK inhibitor, in patients with recurrent low-grade serous ovarian cancer (LGSOC), including overall response rate, progression free survival, and duration of response.

Company to host investor conference call and webcast on October 17, 2024, at 4:30 pm EDT

VSTM under $3

VSTM new 52 week low

https://www.verastem.com/research/pipeline/ Good pipeline!!!

For those who are curious why $VSTM is down 60% on premarket, being 50% yesterday afterhours, despite positive RAMP 205 data (High ORR)

$VSTM 12 patients experienced treatment emergent serious adverse events, 11 patients with grade ≥3 pic.twitter.com/pY34tS6maD— Swat (@SwatOptions) May 24, 2024
For those who are curious why $VSTM is down 60% on premarket, being 50% yesterday afterhours, despite positive RAMP 205 data (High ORR)❓

Today Company announce that they will submit BLA for combo treatment only for a mutant subgroup, which significantly reduces the TAM

👀 $XBI https://t.co/Au1lEsh0qU pic.twitter.com/LzSvNS1T7n— Dogtor🎰📈 (@Dogtor01416800) May 24, 2024

4.75 NOW VSTM over $18

Brutal morning sell off after what appeared to be good news.

VSTM over $18

VSTM over $10

Read about it at 3.00 it looks like.

Worth reading
https://www.onclive.com/view/avutometinib-defactinib-combo-generates-early-efficacy-signals-in-low-grade-serous-ovarian-cancer

It’s death after a RS. Wait for 4.00 or way under. Then Good risk reward

So, what is next for VSTM in terms of catalysts and results? Any upcoming data points? Is it now free from funding worries and free to focus on producing results? Tartia, your input would be appreciated.

Happy I hit and run. I would consider this at under 4.00

They definite are diluting. They need money. But in long run great for shareholder

highest volume in history -insert dd-

Yep

nice

oh my

Looks like dilution

Looks like real support at a dollar. Could be ready for next leg.