TLDR
- PepGen stock traded at $5.76 on Tuesday, up about 146%.
- The 10 mg/kg cohort showed 46.5% average splicing correction at week 13.
- No serious or severe adverse events were reported.
- An FDA partial clinical hold on the FREEDOM2 study is still in place.
- Cash of $104.8M should fund operations into 2028.
PepGen (PEPG) stock traded at $5.76 on Tuesday, up $3.50 or about 146%. Earlier in pre-market trading, the stock was up more than 115%.
The move followed results from the 10 mg/kg cohort of the Phase 2 FREEDOM2-DM1 trial. The study tests PGN-EDODM1 in patients with type 1 myotonic dystrophy, or DM1.
PepGen also posted its third quarter results on Tuesday. The trial update was the main event.
What the 10 mg/kg Data Showed
Average splicing correction reached 46.5% at week 13. The company says that is the highest level recorded in a multiple-dose study.
Five of six participants had more than 20% splicing correction. Four of them topped 50%.
Video Hand Opening Time, a measure of myotonia, improved by 3.9 seconds at week 16. That is about 52.7%, compared with 0.2 seconds in the placebo group.
Handgrip strength rose about 16.2%. The Timed Up and Go test improved 8.5%, and the 10-Meter Walk/Run Test improved 6.8%.
On safety, there were no serious or severe adverse events and no dose-limiting toxicities. Every treatment-emergent event was mild or moderate, and nausea was the most common.
Four mild renal events were recorded. Urinary protein rose briefly after the first dose, then fell with later doses.
Stifel analyst Paul Matteis reiterated his Buy rating. He pointed to a consistent splicing change, efficacy signals across several measures, and no hypomagnesemia.
The FDA Hold and the Road Ahead
The FDA placed a partial clinical hold on FREEDOM2 in March 2026. It cited nonclinical findings in mice.
PepGen is running its own mouse studies. It is also talking with the FDA about additional ones.
An end-of-Phase 2 meeting is expected in the second half of 2027. That depends on the hold being lifted.
In October, the FDA picked PGN-EDODM1 for its CMC Development and Readiness Pilot program. The program offers closer FDA contact on manufacturing plans.
An independent safety board cleared the trial to move to the 12.5 mg/kg cohort. That group is fully enrolled, with results due in the first half of 2027.
The board also approved raising the open-label extension dose from 5 mg/kg to 10 mg/kg. Nineteen participants are enrolled, and interim data is due in January 2027.
PepGen also plans an expansion cohort at 10 mg/kg. It would run alongside the third cohort and collect up to nine months of data.
The quarterly net loss was $19.4 million, or $0.28 per share. That compares with $18.0 million, or $0.52 per share, a year earlier.
R&D spending was $14.5 million, up from $13.4 million. Cash stood at $104.8 million on September 30, which the company says lasts into 2028.
