US Market News
3日前
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
US Market News
1月前
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
US Market News
2月前
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
US Market News
3月前
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
US Market News
3月前
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
US Market News
3月前
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
US Market News
4月前
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
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