PepGen Inc (PEPG)

Looking forward to good news in July or August!

PepGen Reports First Quarter 2026 Financial Results and Recent Corporate HighlightsMay 12, 2026 4:05 PM
Business Wire – FREEDOM2-DM1 5 mg/kg cohort demonstrated favorable safety, splicing and vHOT data, with the totality of results supporting the potential of the ongoing 10 mg/kg dose cohort – – The FREEDOM2 10 mg/kg cohort is fully enrolled, with data on track for 2H 2026 – – Well-funded with $132.3M of cash as of March 31, 2026, sufficient to fund operations through FREEDOM2 12.5 mg/kg MAD readout and into 2H 2027 – PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today reported financial results and recent corporate highlights for the quarter ended March 31, 2026, and recent corporate highlights. "We made encouraging progress during the first quarter of 2026 in our FREEDOM2-DM1 trial. The 5 mg/kg cohort — the initial dose cohort — demonstrated a favorable tolerability profile and promising efficacy trends, reinforcing our confidence in PGN-EDODM1's potential at higher doses," said James McArthur, PhD, President and Chief Executive Officer of PepGen. "Enrollment in the 10 mg/kg cohort is now complete and we remain on track to report data in the second half of this year. With funding in place through the 10 and 12.5 mg/kg cohorts, we are well positioned to continue dose escalating and deliver on the promise of this program for the DM1 community." Recent Program Updates PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1) FREEDOM2 Phase 2 Multiple Ascending Dose (MAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: PepGen reported promising topline results from the 5 mg/kg MAD cohort, demonstrating favorable safety, splicing and vHOT data. The Company believes the totality of safety and efficacy results support the potential of PGN-EDODM1 in the ongoing 10 mg/kg dose cohort. Read the full release here. The Company has fully enrolled the 10 mg/kg MAD cohort of FREEDOM2. PepGen expects to report data from this cohort in the second half of 2026, with data from the 12.5 mg/kg cohort anticipated in 2027. PepGen has currently enrolled 13 patients in the open label extension (OLE) at 5 mg/kg, including 6 patients from FREEDOM2, with no patient discontinuations. The Company has received regulatory clearance to initiate the FREEDOM2 trial in South Korea, Australia, and New Zealand. Sites are currently open and active in Canada, the UK and South Korea, with plans to open sites in New Zealand and Australia. The U.S. Food and Drug Administration (FDA) placed a partial clinical hold on the FREEDOM2 study. The partial clinical hold questions raised by the FDA relate to previously submitted preclinical pharmacology and toxicology studies. The timing of the ongoing FREEDOM2 clinical study has not been impacted. The Company continues to work with the FDA to address the Agency’s questions as quickly as possible. Financial Results for the Three Months Ended March 31, 2026 Cash, Cash Equivalents and Marketable Securities were $132.3 million as of March 31, 2026. Based on currently planned operations, the Company believes that its existing cash, cash equivalents, and marketable securities will be sufficient to fund its operations into the second half of 2027. Research and Development Expenses were $13.0 million for the three months ended March 31, 2026, compared to $25.4 million for the same period in 2025. General and Administrative Expenses were $5.9 million for the three months ended March 31, 2026, compared to $5.9 million for the same period in 2025. Net Loss was $17.8 million, or $(0.26) basic and diluted net loss per share, for the three months ended March 31, 2026, compared to $30.2 million, or $(0.92) basic and diluted net loss per share, for the same period in 2025. PepGen had approximately 69.2 million shares outstanding on March 31, 2026. About PGN-EDODM1 PGN-EDODM1, PepGen's investigational candidate in development for the treatment of DM1, utilizes the Company's proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to restore the normal splicing function of MBNL1, a key RNA splicing protein. PGN-EDODM1 addresses the deleterious effects of cytosine-uracil-guanine (CUG) repeat expansion in the dystrophia myotonica protein kinase (DMPK) transcripts which sequester MBNL1, by binding to the pathogenic CUG trinucleotide repeat expansion present in the DMPK transcripts, and disrupting the binding between the CUG repeat expansion and MBNL1. PepGen believes this innovative therapeutic approach may have considerable advantages over oligonucleotide modalities that rely on knockdown or degradation of the DMPK transcripts as it will allow the DMPK transcripts to continue to perform their normal function within the cell, while also liberating MBNL1 to correct downstream mis-splicing events. The U.S. Food and Drug Administration has granted PGN-EDODM1 both Orphan Drug and Fast Track Designations for the treatment of patients with DM1. About Myotonic Dystrophy Type 1 (DM1) Myotonic dystrophy type 1 (DM1) is a rare, progressive, and highly variable genetic neuromuscular disease caused by an abnormal expansion of cytosine-thymine-guanine (CTG) repeats in the dystrophia myotonica protein kinase (DMPK) gene. DM1 affects over 115,000 individuals in the U.S. and EU and is characterized by widespread, multisystem symptoms that may include myotonia, progressive muscle weakness, fatigue, cardiac abnormalities, respiratory impairment, and cognitive dysfunction. The disease is driven by toxic RNA transcripts containing expanded cytosine-uracil-guanine (CUG) repeats that sequester muscleblind-like 1 (MBNL1), a key RNA splicing protein, leading to widespread mis-splicing across multiple tissues. There are currently no approved disease-modifying therapies for DM1, underscoring the significant unmet medical need for patients living with the disease. About PepGen PepGen Inc. is a clinical-stage biotechnology company developing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, the Company is generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases. For more information, please visit PepGen.com. Follow PepGen on LinkedIn and X. Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will,” and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding the therapeutic potential and safety profile of PGN-EDODM1 based on data from the 5, 10 and 15 mg/kg cohorts of the single dose FREEDOM-DM1 study and data from 5 mg/kg cohort of the multi-dose FREEDOM2-DM1 study, expected timelines for data reports from our FREEDOM2-DM1 trial, forecasts relating to PepGen’s cash runway, and ongoing and planned regulatory interactions, including the potential timing and successful resolution of questions from the FDA relating to the partial clinical hold. Any forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to risks related to: delays or failure to successfully initiate or complete our ongoing and planned development activities for our product candidates, including PGN-EDODM1; our ability to enroll patients in our clinical trials, including FREEDOM2; that our interpretation of clinical and preclinical study results may be incorrect, or that we may not observe the levels of therapeutic activity in clinical testing that we anticipate based on prior clinical or preclinical results, including for PGN-EDODM1; our product candidates, including PGN-EDODM1, may not be safe and effective or otherwise demonstrate safety and efficacy in our clinical trials; adverse outcomes from our regulatory interactions, including delays in regulatory review, clearance to proceed or approval by regulatory authorities with respect to our programs, including release of the partial clinical hold placed by the FDA and/or clearance to commence planned clinical studies of our product candidates, or other regulatory feedback requiring modifications to our development programs, including in each case with respect to our FREEDOM2 program; changes in regulatory framework that are out of our control; unexpected increases in the expenses associated with our development activities or other events that adversely impact our financial resources and cash runway; and our dependence on third parties for some or all aspects of our product manufacturing, research and preclinical and clinical testing. Additional risks concerning PepGen’s programs and operations are described in our most recent reports filed with the SEC. PepGen explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law. This release discusses PGN-EDODM1, an investigational therapy that has not been approved for use in any country, and is not intended to convey conclusions about its efficacy or safety. There is no guarantee that PGN-EDODM1 or any other investigational therapy will successfully complete clinical development or gain regulatory authority approval. Condensed Consolidated Statements of Operations (unaudited, in thousands) Three Months Ended
March 31,     2026       2025   Operating expenses: Research and development $ 13,004   $ 25,378   General and administrative   5,938     5,943   Total operating expenses $ 18,942   $ 31,321   Operating loss $ (18,942 ) $ (31,321 ) Other income (expense) Interest income   1,250     1,122   Other (expense) income, net   (56 )   (3 ) Total other income, net   1,194     1,119   Net loss before income tax $ (17,748 ) $ (30,202 ) Income tax expense   (15 )   —   Net loss $ (17,763 ) $ (30,202 ) Net loss per share, basic and diluted $ (0.26 ) $ (0.92 ) Weighted-average common stock outstanding, basic and diluted   69,091,100     32,674,720   Condensed Consolidated Balance Sheets (unaudited, in thousands)
March 31,
2026 December 31,
2025 Assets Cash, cash equivalents and marketable securities $ 132,303 $ 148,456 Other assets   23,992   25,451 Total assets $ 156,295 $ 173,907 Liabilities and stockholders’ equity Liabilities $ 22,315 $ 26,463 Stockholders’ equity   133,980   147,444 Total liabilities and stockholders’ equity $ 156,295 $ 173,907   View source version on businesswire.com: https://www.businesswire.com/news/home/20260512245445/en/ Investor Contact
Laurence Watts
New Street Investor Relations
laurence@newstreetir.com

Media Contact
Julia Deutsch
Lyra Strategic Advisory, LLC
Jdeutsch@lyraadvisory.com Original: PepGen Reports First Quarter 2026 Financial Results and Recent Corporate Highlights

PepGen Announces Topline Results from Lowest Dose (5 mg/kg) MAD Cohort in the Ongoing Phase 2 FREEDOM2 Study Demonstrating Favorable Safety, Splicing and vHOT DataMarch 30, 2026 4:05 PM
Business Wire
– PGN-EDODM1 was generally well-tolerated with all adverse events mild or moderate and no serious adverse events reported –


– Mean splicing correction of 7.3% observed with PGN-EDODM1 (n=6) versus 6.8% with placebo (n=2); Excluding one outlier patient, treatment group demonstrated mean splicing correction of 22.9% (n=5) –


– Promising trends observed in vHOT in the PGN-EDODM1 treated group –


– Company on track to report clinical data from 10 mg/kg multiple dose cohort in 2H 2026 with sufficient cash expected to fund operations into 2H 2027–


– Conference call scheduled today at 4:30 p.m. ET –


PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced promising clinical data from the 5 mg/kg multiple ascending dose cohort (MAD) of the ongoing Phase 2 FREEDOM2-DM1 trial in patients with myotonic dystrophy type 1 (DM1). The Company believes the totality of safety and efficacy results support the potential of the ongoing 10 mg/kg dose cohort, with clinical results expected in the second half of 2026.


“We are excited to share data from the lowest dose cohort in our multiple ascending dose study of PGN-EDODM1 in patients with DM1,” said Paul Streck, MD, EVP Research and Development. “Overall, we are encouraged by the favorable safety and tolerability profile, and the positive trends exhibited with splicing improvement and vHOT. We look forward to reporting data from the ongoing 10 mg/kg multiple ascending dose cohort of FREEDOM2, which is more than halfway enrolled and remains on track to read out in the second half of this year.”


FREEDOM2 Results for the 5 mg/kg (n=8) Dose Cohort


FREEDOM2 is a Phase 2 MAD, randomized, placebo-controlled clinical trial evaluating PGN-EDODM1 in patients with DM1, with planned dose escalation up to 12.5 mg/kg. The 5 mg/kg cohort enrolled eight patients, who received PGN-EDODM1 or placebo (randomized 6:2) every four weeks over a 12-week period. Key endpoints include safety, splicing correction and functional outcome measures. The data cutoff was March 4, 2026.


Splicing, Muscle Tissue Concentration and Functional Data:



Patients (n=6) treated with PGN-EDODM1 at 5 mg/kg demonstrated a mean splicing correction of 7.3%, compared to 6.8% in placebo-treated patients (n=2).


Excluding an outlier, patients showed a mean splicing correction of 22.9%. The outlier patient exhibited a worsening in splicing correction (70.8%), reducing the overall group mean to 7.3%.






Middle finger vHOT in the treatment group showed a positive trend of improvement versus a worsening observed in the placebo group. Both returned to baseline at Week 16.



Mean muscle tissue concentrations of PGN-EDODM1 available in 5 of 6 treated patients were 158 ng/g, as measured approximately one week after the fourth dose in the MAD study; one concentration readout remains pending.



No meaningful improvements were observed in 10-meter walk/run test (10MWRT) or handgrip strength at the starting PGN-EDODM1 dose of 5 mg/kg.



Safety and Tolerability Data:



PGN-EDODM1 was generally well-tolerated, with no serious adverse events (SAEs), all related treatment emergent adverse events (TEAEs) reported as mild and non-related TEAEs reported as mild or moderate



Nausea was the most common TEAE



No treatment-related discontinuations



No TEAEs related to renal function and no signs of cumulative toxicity



The Company is actively dosing the 10 mg/kg MAD cohort of FREEDOM2, with 5 of 8 patients receiving up to three doses of PGN-EDODM1, and expects to report data in the second half of 2026. In addition, 12 patients have currently enrolled in the open label extension (OLE) at 5 mg/kg, including 5 patients from FREEDOM2.


Conference Call Details


To access the live audio webcast for today’s conference call beginning at 4:30 p.m. ET, please visit PepGen’s investor website at investors.pepgen.com. An archived replay of the webcast will be available on the Company’s website following the event.


About PGN-EDODM1


PGN-EDODM1, PepGen's investigational candidate in development for the treatment of DM1, utilizes the Company's proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to restore the normal splicing function of MBNL1, a key RNA splicing protein. PGN-EDODM1 addresses the deleterious effects of cytosine-uracil-guanine (CUG) repeat expansion in the dystrophia myotonica protein kinase (DMPK) transcripts which sequester MBNL1, by binding to the pathogenic CUG trinucleotide repeat expansion present in the DMPK transcripts, and disrupting the binding between the CUG repeat expansion and MBNL1. PepGen believes this innovative therapeutic approach may have considerable advantages over oligonucleotide modalities that rely on knockdown or degradation of the DMPK transcripts as it will allow the DMPK transcripts to continue to perform their normal function within the cell, while also liberating MBNL1 to correct downstream mis-splicing events. The U.S. Food and Drug Administration has granted PGN-EDODM1 both Orphan Drug and Fast Track Designations for the treatment of patients with DM1. The European Medicines Agency (EMA) has recently granted Orphan Designation for PGN-EDODM1.


About PepGen


PepGen Inc. is a clinical-stage biotechnology company developing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, the Company is generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases.


For more information, please visit PepGen.com. Follow PepGen on LinkedIn and X.


Forward-Looking Statements


This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will,” and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding various data demonstrating promising results that support the safety and clinical potential of PGN-EDODM1 as a treatment for DM1 and expected timelines for the data report from the 10 mg/kg cohort of our FREEDOM2-DM1 trial, our expected cash runway, the design, initiation and conduct of clinical trials, including expected timelines for our FREEDOM2-DM1 trial, and ongoing and planned regulatory interactions.


Any forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to risks related to: delays or failure to successfully initiate or complete our ongoing and planned development activities for PGN-EDODM1; our ability to enroll patients in our clinical trials, including FREEDOM2; that our interpretation of clinical and preclinical study results may be incorrect, or that we may not observe the levels of therapeutic activity in clinical testing that we anticipate based on prior clinical or preclinical results for PGN-EDODM1; that PGN-EDODM1 may not be safe and effective or otherwise demonstrate safety and efficacy in our clinical trials; adverse outcomes from our regulatory interactions, including delays in regulatory review, clearance to proceed or approval by regulatory authorities with respect to our programs, including release of the partial clinical hold or clearance to commence planned clinical studies of our product candidates, or other regulatory feedback requiring modifications to our development programs, including in each case with respect to our FREEDOM2 program; changes in regulatory framework that are out of our control; unexpected increases in the expenses associated with our development activities or other events that adversely impact our financial resources and cash runway; and our dependence on third parties for some or all aspects of our product manufacturing, research and preclinical and clinical testing. Additional risks concerning PepGen’s programs and operations are described in our most recent reports filed with the SEC. PepGen explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.


This release discusses PGN-EDODM1, an investigational therapy that has not been approved for use in any country, and is not intended to convey conclusions about its efficacy or safety. There is no guarantee that PGN-EDODM1 or any other investigational therapy will successfully complete clinical development or gain regulatory authority approval.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260330100190/en/
Investor Contact

Laurence Watts

New Street Investor Relations

Laurence@newstreetir.com
Media Contact

Julia Deutsch

Lyra Strategic Advisory

Jdeutsch@lyraadvisory.com


Original: PepGen Announces Topline Results from Lowest Dose (5 mg/kg) MAD Cohort in the Ongoing Phase 2 FREEDOM2 Study Demonstrating Favorable Safety, Splicing and vHOT Data

PepGen shares slide 20% as FDA clinical hold overshadows earnings beatMarch 5, 2026 10:38 AM
IH Market News
PepGen Inc. (NASDAQ:PEPG) reported a narrower-than-expected fourth-quarter loss on Thursday, but the stock plunged about 20% in after-hours trading as investors focused on regulatory concerns surrounding one of its key trials.The company posted a quarterly loss of $0.27 per share, beating analyst estimates of $0.37 per share. Despite the earnings surprise, shares fell sharply following news that the U.S. Food and Drug Administration has placed a partial clinical hold on the company’s FREEDOM2 study in the United States.PepGen reported a net loss of $18.3 million for the three months ended December 31, 2025, narrowing from a loss of $22.2 million in the same period a year earlier.Research and development expenses declined to $13.9 million from $19.0 million year over year, while general and administrative costs rose slightly to $5.9 million from $5.4 million.Investor concerns centered on the FDA’s partial hold on the FREEDOM2 trial. The agency’s review relates to previously submitted preclinical pharmacology and toxicology studies, though it did not raise issues regarding clinical data from the Phase 1 FREEDOM trial.Patient dosing is continuing outside the U.S., with ongoing treatment in Canada and the United Kingdom. Four of the eight patients in the 10 mg/kg cohort have received up to two doses so far.“2025 was a transformative year for PepGen as the Phase 1 FREEDOM trial delivered compelling data that set a new benchmark for splicing correction in DM1,” said James McArthur, President and Chief Executive Officer.The company expects to release results from the 5 mg/kg cohort of the FREEDOM2 trial during the first quarter of 2026, with data from the 10 mg/kg cohort anticipated in the second half of the year.Following a review by the Data Safety Monitoring Board, PepGen has been cleared to escalate dosing and has updated the study protocol to allow doses of up to 12.5 mg/kg.PepGen reported cash, cash equivalents and marketable securities totaling $148.5 million as of December 31, 2025, which management believes will be sufficient to fund operations into the second half of 2027.PepGen stock price

Original: PepGen shares slide 20% as FDA clinical hold overshadows earnings beat

PepGen Announces Regulatory Updates on FREEDOM2March 4, 2026 4:03 PM
Business Wire
- U.S. FDA places FREEDOM2 trial on partial clinical hold related to preclinical pharmacology and toxicology -


- Received regulatory clearance to initiate FREEDOM2 in South Korea, Australia, and New Zealand and dosing of the 10 mg/kg cohort continues in the UK and Canada -


- PepGen reiterates guidance for reporting data from the FREEDOM2 5 mg/kg cohort in Q1 2026 and from the 10 mg/kg cohort in 2H 2026 -


PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies to treat severe neuromuscular and neurological diseases, today announced regulatory updates related to the FREEDOM2 trial.


The U.S. Food and Drug Administration (FDA) has placed a partial clinical hold on the FREEDOM2-DM1 Phase 2 multiple ascending dose (MAD), randomized, placebo-controlled clinical trial of PGN-EDODM1 in patients with myotonic dystrophy type 1 (DM1). The partial clinical hold questions raised by FDA relate to previously submitted preclinical pharmacology and toxicology studies. The partial clinical hold did not cite any questions regarding blinded clinical data from the Phase 1 FREEDOM study previously submitted to the FDA in order to initiate the FREEDOM2 study in the U.S. As part of its ongoing dialogue with the FDA, the Company is submitting additional analyses, including the recently unblinded FREEDOM data, and is committed to working with the FDA to address their questions as quickly as possible.


PepGen was recently cleared to open the FREEDOM2 study in New Zealand, Australia, and South Korea.


Following the Data Safety Monitoring Board’s recommendation to dose escalate, the FREEDOM2 trial is dosing patients at 10 mg/kg in Canada and the UK. No U.S. patients have been enrolled in FREEDOM2. Patients from the FREEDOM and FREEDOM2 studies in Canada are continuing into the Open Label Extension (OLE) study. The Company has received regulatory clearance for the OLE study in the UK and intends to open the OLE study in all geographies where FREEDOM2 is open.


The Company expects to report data from the 5 mg/kg cohort of FREEDOM2 in the first quarter of 2026, and from the 10 mg/kg cohort in the second half of 2026.


About PGN-EDODM1


PGN-EDODM1, PepGen's investigational candidate in development for the treatment of DM1, utilizes the Company's proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to restore the normal splicing function of MBNL1, a key RNA splicing protein. PGN-EDODM1 addresses the deleterious effects of cytosine-uracil-guanine (CUG) repeat expansion in the dystrophia myotonica protein kinase (DMPK) transcripts which sequester MBNL1, by binding to the pathogenic CUG trinucleotide repeat expansion present in the DMPK transcripts, and disrupting the binding between the CUG repeat expansion and MBNL1. PepGen believes this innovative therapeutic approach may have considerable advantages over oligonucleotide modalities that rely on knockdown or degradation of the DMPK transcripts as it will allow the DMPK transcripts to continue to perform their normal function within the cell, while also liberating MBNL1 to correct downstream mis-splicing events. The U.S. Food and Drug Administration has granted PGN-EDODM1 both Orphan Drug and Fast Track Designations for the treatment of patients with DM1. The European Medicines Agency (EMA) has recently granted Orphan Designation for PGN-EDODM1.


About PepGen


PepGen Inc. is a clinical-stage biotechnology company developing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, the Company is generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases.


For more information, please visit PepGen.com. Follow PepGen on LinkedIn and X.


Forward-Looking Statements


This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will,” and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding the potential of our EDO platform to deliver high levels of oligonucleotide to the nuclei, the design and conduct of clinical trials with our candidates, including expected timelines for the initial data report from our FREEDOM2-DM1 trial, and ongoing and planned regulatory interactions, including the potential timing and resolutions of questions from the FDA relating to the partial clinical hold.


Any forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to risks related to: delays or failure to successfully initiate or complete our ongoing and planned development activities for PGN-EDODM1; our ability to enroll patients in our clinical trials, including FREEDOM2; that our interpretation of clinical and preclinical study results may be incorrect, or that we may not observe the levels of therapeutic activity in clinical testing that we anticipate based on prior clinical or preclinical results for PGN-EDODM1; that PGN-EDODM1 may not be safe and effective or otherwise demonstrate safety and efficacy in our clinical trials; adverse outcomes from our regulatory interactions, including delays in regulatory review, clearance to proceed or approval by regulatory authorities with respect to our programs, including release of the partial clinical hold or clearance to commence planned clinical studies of our product candidates, or other regulatory feedback requiring modifications to our development programs, including in each case with respect to our FREEDOM2 program; changes in regulatory framework that are out of our control; unexpected increases in the expenses associated with our development activities or other events that adversely impact our financial resources and cash runway; and our dependence on third parties for some or all aspects of our product manufacturing, research and preclinical and clinical testing. Additional risks concerning PepGen’s programs and operations are described in our most recent reports filed with the SEC. PepGen explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.


This release discusses PGN-EDODM1, an investigational therapy that has not been approved for use in any country, and is not intended to convey conclusions about its efficacy or safety. There is no guarantee that PGN-EDODM1 or any other investigational therapy will successfully complete clinical development or gain regulatory authority approval.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260304006219/en/
Investor Contact

Laurence Watts

New Street Investor Relations

laurence@newstreetir.com


Media Contact

Julia Deutsch

Lyra Strategic Advisory, LLC

Jdeutsch@lyraadvisory.com


Original: PepGen Announces Regulatory Updates on FREEDOM2

PepGen Reports Fourth Quarter and Year-End 2025 Financial Results and Recent Corporate HighlightsMarch 4, 2026 4:05 PM
Business Wire
– FREEDOM2-DM1 5 mg/kg cohort data expected in Q1 2026 –


– Patient dosing in the FREEDOM2 10 mg/kg cohort is proceeding in Canada and the UK, with data expected in 2H 2026 –


– FREEDOM2 trial open in South Korea, Australia, and New Zealand –


– Well-funded with $148.5M of cash as of December 31, 2025, sufficient to fund operations into the second half of 2027 –


PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today reported financial results for the quarter and year-ended December 31, 2025, and recent corporate highlights.


“2025 was a transformative year for PepGen as the Phase 1 FREEDOM trial delivered compelling data that set a new benchmark for splicing correction in DM1, reinforced our conviction in PGN-EDODM1 as a potential best-in-class treatment, and further demonstrated the differentiated potential of our EDO platform," said James McArthur, PhD, President and Chief Executive Officer of PepGen. “After a single dose, we observed robust target engagement beginning at the lowest dose of 5 mg/kg — a finding that exceeded our expectations and underscored the potency of our drug candidate. PGN-EDODM1 was generally well-tolerated across doses, and based on the totality of FREEDOM data, we believe that doses of 10 mg/kg could potentially offer a best-in-class treatment option for people living with this serious disease.”


Dr. McArthur continued: “As we prepare for the 5 mg/kg dose cohort readout from our ongoing randomized, placebo-controlled multiple ascending dose Phase 2 FREEDOM2 study later this quarter, we are focused on building upon the robust muscle oligonucleotide levels and splicing correction observed in FREEDOM, with the goal of translating sustained exposure into meaningful functional benefit. Following the Data Safety Monitoring Board’s recommendation to dose escalate, we are actively dosing the 10 mg/kg cohort in FREEDOM2 – four patients have been dosed, receiving up to two doses. PepGen is entering this new year with strong momentum and a clear path toward potentially delivering a meaningful new treatment for the DM1 community.”


Recent Program Updates


PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1)



FREEDOM2 Phase 2 Multiple Ascending Dose (MAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1:


The Company expects to report results from the 5 mg/kg cohort of the FREEDOM2 trial in the first quarter of 2026.



After reviewing safety data from the 5 mg/kg cohort, the Data Safety Monitoring Board (DSMB) recommended escalating to the 10 mg/kg dose. PepGen has since dosed 50% of patients (4/8) in the 10 mg/kg cohort with up to two doses. The Company remains on track to report results from the 10 mg/kg cohort in the second half of 2026.



Informed by the prior FREEDOM results, PepGen has amended the FREEDOM2 protocol to provide the option to dose up to 12.5 mg/kg.



Patients from the FREEDOM and FREEDOM2 studies in Canada are continuing into the Open Label Extension (OLE) study. The Company has received regulatory clearance for the OLE study in the UK and intends to open the OLE study in all geographies where FREEDOM2 is open.



The Company has recently received regulatory clearance to initiate the FREEDOM2 trial in South Korea, Australia, and New Zealand. Sites are currently enrolling in Canada, the UK and South Korea, with plans to open sites in New Zealand and Australia.



The U.S. Food and Drug Administration (FDA) has placed a partial clinical hold on the FREEDOM2 study. The partial clinical hold questions raised by FDA relate to previously submitted preclinical pharmacology and toxicology studies. The partial clinical hold did not cite any questions regarding blinded clinical data from the Phase 1 FREEDOM study previously submitted to the FDA in order to initiate the FREEDOM2 study in the U.S. The Company is committed to working with the FDA to address the Agency’s questions as quickly as possible.







Other FREEDOM Updates:


In October 2025, PepGen’s data were featured in two oral presentations at the 30th Annual International Congress of the World Muscle Society (WMS). One presentation included previously reported positive FREEDOM-DM1 Phase 1 clinical data.



In October 2025, the Company gave an oral presentation at the Oligonucleotide Therapeutics Society (OTS) Annual Meeting. The presentation reviewed the use of its Enhanced Delivery Oligonucleotide (EDO) Platform to develop a treatment for DM1 and included a review of the FREEDOM clinical data.






2026 MDA Conference Update


The Company will present unblinded final results from FREEDOM-DM1 — its Phase 1, placebo-controlled single ascending dose study of PGN-EDODM1 in DM1 adult patients — in oral and poster presentations at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, being held March 8–11, 2026 in Orlando, Florida.


Key study findings include:


Efficacy: PGN-EDODM1 demonstrated greater than dose-proportional increases in muscle tissue concentration and splicing correction across all doses at Day 28. Mean splicing correction reached unprecedented levels after a single dose, achieving 12.3%, 29.1%, and 53.7% at 5 mg/kg (n=6), 10 mg/kg (n=5), and 15 mg/kg (n=6), respectively.


Safety: PGN-EDODM1 was generally well-tolerated across all doses; treatment-emergent adverse events (TEAEs) were mild to moderate, transient, and required no intervention (except one patient in the 15 mg/kg cohort, managed with oral antihistamines). No electrolyte-related TEAEs were observed, and magnesium levels remained within normal range across all doses. No renal biomarker-related TEAEs were observed at 5 or 10 mg/kg. Post-unblinding, transient moderate albuminuria increases at the 15 mg/kg dose and transient mild albuminuria increases at 10 mg/kg were apparent — both resolving to normal within two to seven days with no clinical symptoms or need for intervention.


Following the conference, the presentations presented at the 2026 MDA Clinical & Scientific Conference will be available on PepGen’s website under Scientific Publications.


Corporate Updates



In December 2025, PepGen appointed Joseph Vittiglio, Esq., as Chief Business and Legal Officer. Mr. Vittiglio brings more than two decades of executive leadership experience across legal, compliance, corporate development, and corporate governance within public biotechnology companies. He joins PepGen after providing consulting support to the Company in 2025.



In November 2025, the Company announced that the United States Patent and Trademark Office (USPTO) has issued the Company a new composition of matter patent covering PGN-EDODM1, which leverages PepGen’s proprietary EDO platform, including its unique peptide and linker chemistry. This newly issued composition of matter patent, which serves as a cornerstone of the Company’s expanding patient portfolio, is expected to provide exclusivity for PGN-EDODM1 in the United States into second half of 2042, with the possibility of patent term extension following a potential new drug application and approval by FDA of PGN-EDODM1.



Financial Results for the Three Months and Year Ended December 31, 2025



Cash, Cash Equivalents and Marketable Securities were $148.5 million as of December 31, 2025. Based on currently planned operations, the Company believes that its existing cash, cash, equivalents, and marketable securities will be sufficient to fund its operations into the second half of 2027.



Research and Development Expenses were $13.9 million for the three months ended December 31, 2025, compared to $19.0 million for the same period in 2024. Research and development expenses were $71.0 million for the year ended December 31, 2025, compared to $76.5 million for the same period in 2024.



General and Administrative Expenses were $5.9 million for the three months ended December 31, 2025, compared to $5.4 million for the same period in 2024. General and administrative expenses were $22.6 million for the year ended December 31, 2025, compared to $21.3 million for the same period in 2024.



Net Loss was $18.3 million, or $(0.27) basic and diluted net loss per share, for the three months ended December 31, 2025, compared to $22.2 million, or $(0.68) basic and diluted net loss per share, for the same period in 2024. Net loss was $89.7 million, or $(2.12) basic and diluted net loss per share, for the year ended December 31, 2025, compared to $90.0 million, or $(2.85) basic and diluted net loss per share, for the same period in 2024. PepGen had approximately 68,874,944 shares outstanding as of December 31, 2025.



About PGN-EDODM1


PGN-EDODM1, PepGen's investigational candidate in development for the treatment of DM1, utilizes the Company's proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to restore the normal splicing function of MBNL1, a key RNA splicing protein. PGN-EDODM1 addresses the deleterious effects of cytosine-uracil-guanine (CUG) repeat expansion in the dystrophia myotonica protein kinase (DMPK) transcripts which sequester MBNL1, by binding to the pathogenic CUG trinucleotide repeat expansion present in the DMPK transcripts, and disrupting the binding between the CUG repeat expansion and MBNL1. PepGen believes this innovative therapeutic approach may have considerable advantages over oligonucleotide modalities that rely on knockdown or degradation of the DMPK transcripts as it will allow the DMPK transcripts to continue to perform their normal function within the cell, while also liberating MBNL1 to correct downstream mis-splicing events. The U.S. Food and Drug Administration has granted PGN-EDODM1 both Orphan Drug and Fast Track Designations for the treatment of patients with DM1. The European Medicines Agency (EMA) has recently granted Orphan Designation for PGN-EDODM1.


About PepGen


PepGen Inc. is a clinical-stage biotechnology company developing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, the Company is generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases.


For more information, please visit PepGen.com. Follow PepGen on LinkedIn and X.


Forward-Looking Statements


This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will,” and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding the therapeutic potential and safety profile of PGN-EDODM1 based on data from the 5, 10 and 15 mg/kg cohorts of the FREEDOM-DM1 study, our expectations regarding the potential for significant correction of mis-splicing with more doses of PGN-EDODM1 over a longer treatment period to potentially provide improved functional benefit for patients with DM1, the design and conduct of clinical trials with our candidates, including expected timelines for the initial data report from our FREEDOM2-DM1 trial and the potential to extend dosing to the 12.5 mg/kg dose level, the potential for any functional improvements that may result from robust splicing correction with PGN-EDODM1, dose-dependent increases in splicing suggesting that PGN-EDODM1 is getting into the muscle and effectively binding to the target, the potential for PGN-EDODM1 to offer a best-in-class treatment option, the potential for future patent term extension, forecasts relating to our cash runway, and ongoing and planned regulatory interactions and ongoing and planned regulatory interactions, including the potential timing and successful resolution of questions from the FDA relating to the partial clinical hold.


Any forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to risks related to: delays or failure to successfully initiate or complete our ongoing and planned development activities for our product candidates, including PGN-EDODM1; our ability to enroll patients in our clinical trials, including FREEDOM2; that our interpretation of clinical and preclinical study results may be incorrect, or that we may not observe the levels of therapeutic activity in clinical testing that we anticipate based on prior clinical or preclinical results, including for PGN-EDODM1; our product candidates, including PGN-EDODM1, may not be safe and effective or otherwise demonstrate safety and efficacy in our clinical trials; adverse outcomes from our regulatory interactions, including delays in regulatory review, clearance to proceed with planned clinical studies or approval by regulatory authorities with respect to our investigational drug candidates, including release of the partial clinical hold placed by FDA on the FREEDOM2 study, or other regulatory feedback requiring modifications to our development programs, including release of the partial clinical hold or clearance to commence planned clinical studies of our product candidates, or other regulatory feedback requiring modifications to our development programs, including in each case with respect to our FREEDOM2 program; changes in regulatory framework that are out of our control; unexpected increases in the expenses associated with our development activities or other events that adversely impact our financial resources and cash runway; and our dependence on third parties for some or all aspects of our product manufacturing, research and preclinical and clinical testing. Additional risks concerning PepGen’s programs and operations are described in our most recent reports filed with the SEC. PepGen explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.


This release discusses PGN-EDODM1, an investigational therapy that has not been approved for use in any country, and is not intended to convey conclusions about its efficacy or safety. There is no guarantee that PGN-EDODM1 or any other investigational therapy will successfully complete clinical development or gain regulatory authority approval.




Condensed Consolidated Statements of Operations




(unaudited, in thousands)










 




Twelve Months Ended

December 31,







Three Months Ended

December 31,









 






2025






 






 






 






2024






 






 






 






2025






 






 






 






2024






 








Operating expenses:















Research and development






$






71,043






 







$






76,478






 







$






13,851






 







$






18,961






 








General and administrative






 






22,569






 







 






21,261






 







 






5,854






 







 






5,384






 








Total operating expenses






$






93,612






 







$






97,739






 







$






19,705






 







$






24,345






 








Operating loss






$






(93,612






)







$






(97,739






)







$






(19,705






)







$






(24,345






)








Other income (expense)















Interest income






 






4,017






 







 






7,142






 







 






1,419






 







 






1,460






 








Other income (expense), net






 






(11






)







 






(1






)







 






(5






)







 






26






 








Total other income, net






 






4,006






 







 






7,141






 







 






1,414






 







 






1,486






 








Net loss before income tax






$






(89,606






)







$






(90,598






)







$






(18,291






)







$






(22,859






)








Income tax (expense) benefit






 






(49






)







 






617






 







 






(49






)







 






617






 








Net loss






$






(89,655






)







$






(89,981






)







$






(18,340






)







$






(22,242






)








Net loss per share, basic and diluted






$






(2.12






)






 






$






(2.85






)






 






$






(0.27






)






 






$






(0.68






)








Weighted-average common shares outstanding, basic and diluted






 






42,221,808






 






 






 






31,583,073






 






 






 






68,790,477






 






 






 






32,602,981






 









Condensed Consolidated Balance Sheets




(unaudited, in thousands)















December 31,

2025







December 31,

2024








Assets












Cash, cash equivalents and marketable securities







$






148,456







$






120,191








Other assets







 






25,451







 






30,692








Total assets







$






173,907







$






150,883








Liabilities and stockholders’ equity












Liabilities







$






26,463







$






32,263








Stockholders’ equity







 






147,444







 






118,620








Total liabilities and stockholders’ equity







$






173,907







$






150,883







 

View source version on businesswire.com: https://www.businesswire.com/news/home/20260304670027/en/
Investor Contact

Laurence Watts

New Street Investor Relations

laurence@newstreetir.com



Media Contact

Julia Deutsch

Lyra Strategic Advisory, LLC

Jdeutsch@lyraadvisory.com


Original: PepGen Reports Fourth Quarter and Year-End 2025 Financial Results and Recent Corporate Highlights

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