PepGen announces excellent results from the low-dose (5 mg/kg) MAD cohort in the ongoing phase FREEDOM2 study, showing favorable safety, efficacy and vHOT data.
– PGN-EDODM1 was generally well tolerated with all adverse events being mild or moderate and no serious adverse events reported –
– Dissociation correction observed with PGN-EDODM1 (n=6) versus 6.8% with placebo (n=2); 7.3%; With the exception of one outpatient, the treatment group showed a mean improvement of 22.9% (n = 5).
– A significant trend in vHOT was observed in the PGN-EDODM1 treated group –
– The company expects sufficient cash to fund operations in 2H 2027 upon reporting clinical data from the 10 mg/kg multiple dose cohort in 2H 2026.
– The conference call is set for today at 4:30 pm –
Boston, March 30, 2026–( BUSINESS WIRE )–PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company developing the next generation of oligonucleotide therapies aimed at transforming the treatment of severe neurological and neurodegenerative diseases, today announced promising clinical data for the treatment of 5 mg/kgdse2c as a multidrug-resistant drug. FREEDOM2-DM1 trial in patients with myotonic dystrophy type 1 (DM1). The company believes the overall safety and efficacy results support the feasibility of the ongoing 10 mg/kg dose cohort, with clinical results expected in the second half of 2026.
“We are pleased to share data from the low-dose cohort in our multiple escalating dose study of PGN-EDODM1 in patients with DM1,” said Paul Strick, MD, EVP Research and Development. “Overall, we are encouraged by the favorable safety and tolerability profile, and the positive trends that have been shown with the progression and vHOT. We look forward to reporting data from FREEDOM2’s ongoing 10 mg/kg multi-escalation dose cohort, which is more than half enrolled and pending readmissions in the second half of this year.”
FREEDOM2 results for the 5 mg/kg (n=8) dose cohort
FREEDOM2 is a MAD phase 2, randomized, placebo-controlled clinical trial evaluating PGN-EDODM1 in DM1 patients, with a planned dose escalation of up to 12.5 mg/kg. The 5 mg/kg cohort enrolled eight patients to receive PGN-EDODM1 or placebo (randomized 6:2) every four weeks over a 12-week period. Key endpoints include safety, isolation adjustment and functional outcome measures. Data cut-off was March 4, 2026.
Isolation, muscle tissue concentration and performance data:
Patients (n=6) treated with PGN-EDODM1 at 5 mg/kg showed a mean separation correction of 7.3%, compared to 6.8% in placebo-treated patients (n=2).
Excluding one outlier, patients showed 22.9% separation correction. Outpatients showed worse separation correction (70.8%), reducing the overall group mean to 7.3%.
Middle finger vHOT in the treatment group showed a positive trend of improvement compared to the deterioration observed in the placebo group. Both returned to baseline at week 16.
The mean muscle tissue concentration of PGN-EDODM1 present in 5 of 6 treated patients was 158 ng/g, as measured approximately one week after the fourth dose in the MAD study; A concentration reading remains.
No significant improvement was observed in the 10-m walk/run test (10MWRT) or handgrip strength at the initial dose of 5 mg/kg of PGN-EDODM1.
Safety and tolerance data:
PGN-EDODM1 was generally well tolerated, with no serious adverse events (SAEs), all treatment-related emergent adverse events (TEAEs) reported as mild and unrelated TEAEs reported as mild or moderate.
Brain was the most common TEAE
There are no restrictions related to treatment
There were no TEAEs related to renal function and no signs of cumulative toxicity
The company is actively dosing a 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. Additionally, 12 patients are currently enrolled in the open-label extension (OgLE/5g/5g) of patients.
Conference call details
To access the live audio webcast for today’s conference call beginning at 4:30 p.m., please visit PepGen Investors’ 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, uses the company’s proprietary EDO technology to deliver a therapeutic oligonucleotide designed to restore the normal splicing function of MBNL1, a key RNA splicing protein. PGN-EDODM1 underlies the deleterious effects of a cytosine-uracil-guanine (CUG) repeat expansion in dystrophia myotonica protein kinase ((DMPK) The transcripts that isolate MBNL1, by binding to the CUG trinucleotide repeat expansion contained within. DMPK duplications, and disruption of binding between the CUG repeat expansion and MBNL1. PepGen believes that this innovative therapeutic approach may have significant advantages over oligonucleotide approaches that rely on blocking or degradation. DMPK Transcriptions such as these would allow DMPK transcripts to continue their normal function in the cell, while freeing MBNL1 to correct downstream mis-splicing events. The US 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) recently granted orphan designation for PGN-EDODM1.
About PepGen
PepGen Inc. is a clinical-stage biotechnology company developing the next generation of oligonucleotide therapeutics aimed at revolutionizing the treatment of severe neurological and neurodegenerative diseases. PepGen’s high-delivery oligonucleotide (EDO) platform is built on a decade of research and development and leverages cell-penetrating peptides to enhance the efficacy of conjugated oligonucleotide therapies. Using these EDO peptides, the company is developing a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases.
For more information, please visit PepGen.com. Follow PepGen LinkedIn and X.
Visible Statements
This press release contains significant statements within the meaning of the Private Securities Filing Reform Act of 1995 as amended. These statements may be identified by words such as “aim,” “expect,” “believe,” “may,” “estimates,” “anticipate,” “forecasts,” “goal,” “goal,” “may,” “plans,” “potential,” “likely,” “seek,” “hope,” and variations of those words or similar expressions intended to be statements. Any statements in this press release that are not statements of historical fact are considered forward-looking statements. These forward-looking statements include, without limitation, statements regarding various data showing promising results that support the safety and clinical potential of PGN-EDODM1 as a treatment for DM1 and the expected timelines for reporting data from the 10 mg/kg cohort of our FREEDOM2-DM1 trial, our expected cash flow path, including our anticipated timelines and design procedures, the FREEDOM2-DM1 trial, and ongoing and planned regulatory interactions.
Any forward-looking statements in this press release are based only on current expectations, estimates and projections 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 or adversely from those implied or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, risks related to: delays in or failure to successfully initiate or complete our ongoing and planned development activities for PGN-EDODM1; the ability to enroll patients in our clinical trials, including FREEDOM2; that our interpretation of clinical and preclinical study results may be inaccurate, or that we may not see the level of therapeutic activity in a clinical trial that we anticipate based on preclinical 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 or approval by regulatory authorities to proceed with our programs, including the release of a partial clinical hold to initiate planned clinical studies of our product candidates, or other regulatory feedback that requires adjustments to our development programs, including adjustments to our development programs with respect to FOMRE2; changes in the regulatory framework beyond our control; unexpected increases in costs associated with our development activities or other events that adversely affect our financial resources and cash flows; and our reliance on third parties for some or all aspects of our product manufacturing, research and clinical and clinical testing. Additional risks related to PepGen’s programs and operations are described in our most recent reports filed with the SEC. PepGen expressly disclaims any obligation to update any forward-looking statements unless required by law.
This publication discusses PGN-EDODM1, an investigational therapy that has not been approved for use in any country, and is not intended to draw conclusions about its efficacy or safety. There can be no assurance that PGN-EDODM1 or any other investigational therapy will successfully complete clinical development or receive regulatory approval.
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