PepGen Inc. (NASDAQ:PEPG) shares surged 145% following the biotech firm’s announcement of unprecedented splicing correction in patients with myotonic dystrophy type 1 (DM1) from its FREEDOM-DM1 Phase 1 study.
The company reported a mean splicing correction of 53.7% after a single 15 mg/kg dose of its PGN-EDODM1 therapy—substantially higher than any previously documented results in DM1 patients. Every patient in the 15 mg/kg group demonstrated improved splicing correction following treatment.
“We are delighted to report that the FREEDOM clinical study achieved all of its key objectives, including unprecedented splicing correction following a single dose of PGN-EDODM1 at 15 mg/kg,” said Paul Streck, Executive Vice President of Research and Development at PepGen.
These results build on earlier data from lower dose groups, which showed mean splicing correction of 12.3% at 5 mg/kg and 29.1% at 10 mg/kg, reflecting greater-than-dose-proportional improvements in efficacy.
The 15 mg/kg dose of PGN-EDODM1 was generally well tolerated, with no serious treatment-related adverse events reported. All related adverse events were mild or moderate, transient, and mostly did not require intervention.
In addition, PepGen announced the pricing of an underwritten offering of 31,250,000 shares at $3.20 each, expected to generate $100 million in gross proceeds, with the offering anticipated to close around September 26, 2025.
Looking ahead, PepGen expects to report results from the first cohort of its FREEDOM2 multiple ascending dose study in Q1 2026 and plans to begin dosing its 10 mg/kg cohort in the same quarter.
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