PepGen Inc. has achieved a significant milestone in the treatment of myotonic dystrophy type 1 (DM1) with the results from its FREEDOMDM1 Phase 1 study. The company announced these groundbreaking findings on September 25, 2025, and the report highlights positive developments indicating that these results have not only showcased the potential of their treatment but also led to a remarkable increase in their stock value.
Impressive Results from FREEDOMDM1 Study
In the FREEDOMDM1 study, patients administered a 15 mg/kg dose of PGNEDODM1 exhibited an impressive mean splicing correction of 537, surpassing all previously documented results in this area. This achievement has resulted in a substantial rise in PepGen's stock price, which closed at $5.72, marking an increase of over 114% from the previous trading session.
Consistent Therapeutic Responses and Safety
The study reported consistent therapeutic responses across all patient cohorts, with no serious treatment-related adverse events noted, underscoring the safety and efficacy of the treatment. Paul Streck, Executive Vice President of Research and Development at PepGen, expressed his satisfaction with these outcomes, indicating a strategic pivot for the company following the discontinuation of its Duchenne muscular dystrophy programs earlier this year.
Looking Ahead: FREEDOM2DM1 Study
Looking ahead, PepGen is set to unveil results from its FREEDOM2DM1 study in the first quarter of 2026, which could further solidify its position in the field of genetic disorders.
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