Image courtesy of 123rf.com

PepGen Inc. (Nasdaq: PEPG) experienced a dramatic surge on Thursday, September 25, 2025, with shares soaring over 114% following the release of groundbreaking clinical trial results. The clinical-stage biotechnology company announced unprecedented splicing correction results from its FREEDOM-DM1 Phase 1 study, treating patients with myotonic dystrophy type 1 (DM1), a rare genetic disorder. The remarkable data showed a mean splicing correction of 53.7% in patients receiving a 15 mg/kg dose of PGN-EDODM1, substantially higher than any previously reported splicing correction in DM1 patients. This breakthrough represents a significant milestone for the company’s pivot strategy after discontinuing its Duchenne muscular dystrophy programs earlier this year.

PEPG: Breakthrough DM1 Data Exceeds Expectations

The FREEDOM-DM1 Phase 1 study results demonstrated dose-proportional efficacy improvements across cohorts, with splicing correction rates of 12.3% at 5 mg/kg, 29.1% at 10 mg/kg, and the remarkable 53.7% at 15 mg/kg. All six patients (100%) in the highest dose cohort showed improved splicing correction, indicating consistent therapeutic response. The treatment was generally well-tolerated with no serious treatment-related adverse events, and all side effects were classified as mild to moderate and transient.

Paul Streck, Executive Vice President of Research and Development at PepGen, expressed satisfaction that the study achieved all key objectives with unprecedented splicing correction following a single dose. The company’s focus on DM1 represents a strategic shift after discontinuing its DMD programs when PGN-EDO51 failed to meet targets. PepGen anticipates reporting results from its FREEDOM2-DM1 multiple ascending dose study in Q1 2026, with plans to begin dosing the 10 mg/kg cohort in the same timeframe.

PepGen Shares Double on Historic Trading Volume

PEPG shares closed at $5.72, representing a 114.99% gain from the previous close of $2.66, with extraordinary trading volume of 89.4 million shares compared to the typical 786,746 average volume. The stock opened at $5.88 and traded in a range of $4.90 to $6.15 during the session. Despite the dramatic single-day gain, the stock remains down 36.59% over the past year, reflecting the broader challenges faced by clinical-stage biotechnology companies.

The company simultaneously announced the pricing of an underwritten public offering of 31.25 million shares at $3.20 per share, expected to raise $100 million in gross proceeds with closing anticipated around September 26, 2025. This financing provides crucial runway for advancing the DM1 program through upcoming clinical milestones. With a current market capitalization of approximately $179 million and $74.65 million in cash reserves, PepGen maintains a relatively strong balance sheet position for a clinical-stage company, though it continues to burn cash with levered free cash flow of -$56.16 million.

More By This Author: