PepGen Stock More Than Doubles as FREEDOM2-DM1 Data Show Record Splicing Correction

PepGen Inc. (NASDAQ: PEPG) shares more than doubled in premarket trading Tuesday, jumping 136.4% to $5.65 from Monday’s close of $2.39. The surge came after the company reported positive topline results from the 10 mg/kg cohort of its Phase 2 FREEDOM2-DM1 trial. The data showed that PGN-EDODM1, its treatment candidate for myotonic dystrophy type 1 (DM1), delivered what PepGen calls the highest splicing correction reported in a multiple-dose DM1 study. The drug also improved several functional measures.

Key Points

• PEPG shares rose 136.4% to $5.65 in premarket trading Tuesday, up from Monday’s close of $2.39.
• Patients treated at 10 mg/kg showed mean splicing correction of 46.5% at Week 13, compared with 6.8% for the pooled placebo group.
• Hand-opening time, a key measure of myotonia, improved by 3.9 seconds, or about 52.7%, at Week 16. Placebo patients improved by 0.2 seconds.
• PepGen reported no serious or severe adverse events, no dose-limiting toxicities and no treatment discontinuations.
• The 12.5 mg/kg cohort is fully enrolled, and results are expected in the first half of 2027.
• PepGen ended the quarter with $104.8 million in cash and expects that to fund operations into 2028. The FDA’s partial clinical hold remains in place.

PepGen (NASDAQ: PEPG) Shows a Sharp Step Up From Its Starting Dose

The 10 mg/kg cohort enrolled eight patients, randomized 3:1 to PGN-EDODM1 or placebo. Each patient received four intravenous doses, one every four weeks, and all eight completed the Week 16 assessment. The response was also broad across patients: five of six treated patients exceeded 20% splicing correction, and four of six topped 50%.

That marks a clear improvement over the starting dose. In March, the 5 mg/kg cohort showed mean splicing correction of just 7.3% versus 6.8% for placebo, largely because one outlier patient dragged down the group average.

Functional Gains Support the Splicing Data

The functional results followed the same direction. Hand-grip strength improved by about 16.2%, a gain of 6.3 points in maximum predicted percent of normal, compared with 0.9 points for placebo. In addition, the 10-Meter Walk/Run Test improved by 6.8%, or 0.4 seconds, versus 0.2 seconds for placebo. Timed Up and Go improved by 8.5%, or 0.6 seconds, while placebo patients got 0.5 seconds slower.

Patients’ own ratings were less clear. Patient Global Impression of Change showed a favorable trend versus placebo. However, scores on the Myotonic Dystrophy Health Index improved in every group, including placebo, and PepGen described the patient-reported outcomes as inconclusive.

Safety Holds Up at the Higher Dose

PGN-EDODM1 was generally well tolerated at 10 mg/kg. All treatment-emergent adverse events were mild or moderate, and nausea was the most common. PepGen also reported four mild kidney-related events. These included temporary protein in the urine after the first dose, which decreased with later doses, and the company saw no buildup of kidney toxicity over 16 weeks.

The FDA Hold Still Shapes PepGen’s Timeline

Even so, regulatory questions remain. The FDA placed a partial clinical hold on FREEDOM2 in March 2026 over findings in mouse studies, which PMV covered when the stock fell 26%. PepGen is running additional mouse studies and remains in talks with the agency. The company has tied removal of the hold to an end-of-Phase 2 meeting, which it plans for the second half of 2027.

At the same time, there are signs of FDA engagement. In October, the agency selected PGN-EDODM1 for its CMC Development and Readiness Pilot program, which aims to reduce manufacturing risk ahead of Phase 3 and an eventual approval filing. The drug also holds FDA Orphan Drug and Fast Track designations, as well as Orphan Designation from the European Medicines Agency.

What Comes Next for PepGen (NASDAQ: PEPG)

PepGen has a busy calendar ahead. It expects interim data from its open-label extension study in January 2027, with safety data at 10 mg/kg and clinical data at 5 mg/kg. After that, results from the 12.5 mg/kg cohort should arrive in the first half of 2027. In addition, PepGen plans a 10 mg/kg expansion cohort, run alongside the 12.5 mg/kg cohort, to collect up to nine months of data.

Financially, PepGen reported a third-quarter net loss of $19.4 million, or $0.28 per share, compared with $18.0 million, or $0.52 per share, a year earlier. Research and development spending rose to $14.5 million from $13.4 million. The lower per-share loss mostly reflects a larger share count, which roughly doubled to 69.3 million weighted-average shares after the company’s $100 million public offering.

Investor Takeaway

For PepGen stock, the 10 mg/kg data change the story. Last year, the FREEDOM single-dose results sent shares up 180%. Now, multiple-dose data show strong, consistent splicing correction and improvements across several functional tests. On the other hand, the cohort was small, the patient-reported results were mixed, and the U.S. partial clinical hold remains unresolved. As a result, the 12.5 mg/kg readout and the path to an end-of-Phase 2 meeting are the next catalysts to watch for investors following the PRISM Biotech Index.