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StocksMedium•30 March 2026•

1 min read

PepGen Reports Positive Phase 2 Results for Myotonic Dystrophy Type 1 Treatment

Key Facts

1PepGen announced positive topline results from the 5 mg/kg dose cohort in the Phase 2 FREEDOM2-DM1 trial.

2The data demonstrated favorable safety, splicing, and vHOT data for patients with Myotonic Dystrophy Type 1 (DM1).

3These results are part of the development of oligonucleotide therapies for severe neuromuscular diseases.

7 days ago

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Version History

1 additional source confirmed this story

Version 17 days ago

What changed: Added analyst coverage from H.C. Wainwright with a 'Buy' rating and $20 price target, while noting the program is now considered 'de-risked' despite an anomalous patient skewing some data.

PepGen Inc. (Nasdaq: PEPG) has announced positive topline results from the 5 mg/kg dose cohort of its Phase 2 FREEDOM2-DM1 clinical trial for Myotonic Dystrophy Type 1 (DM1). The therapy demonstrated a favorable safety profile and promising results in genetic splicing, indicating biological activity even at the lowest dose level. Following these results, H.C. Wainwright maintained its 'Buy' rating on the stock with a $20.00 price target. Analysts characterized the DM1 program as 'de-risked' based on the recent biological data, despite the disclosure that an anomalous patient skewed the average splicing correction results. This milestone provides a positive signal for upcoming data from higher dose cohorts in the company's clinical pipeline. PepGen continues to focus on developing specialized therapies for severe neuromuscular diseases, drawing significant attention from the biotech sector.