TLDR
- PepGen stock surged 145% after reporting record 53.7% splicing correction in DM1 patients with single dose treatment
- All patients in the 15 mg/kg cohort showed improved outcomes with mild to moderate side effects
- Company raised $100 million through stock offering at $3.20 per share to fund clinical trials
- Results exceed any previous DM1 splicing correction data and showed dose-proportional increases
- FREEDOM2 multiple dose study results expected in first quarter 2026
PepGen stock exploded 145% after the biotechnology company reported record splicing correction results in myotonic dystrophy type 1 patients. The clinical trial data sent shares soaring as investors celebrated the unprecedented outcomes.
The company announced a mean splicing correction of 53.7% following a single 15 mg/kg dose of PGN-EDODM1. This result towers over any previously reported splicing correction in DM1 patients.
All six patients in the 15 mg/kg cohort showed improved splicing correction. The treatment targets the underlying genetic defect in myotonic dystrophy type 1.
Paul Streck, Executive Vice President of Research and Development, expressed delight with the clinical study results. The FREEDOM-DM1 Phase 1 study achieved all key objectives.
The latest results build on previous data from lower dose groups. The 5 mg/kg dose showed 12.3% mean splicing correction while 10 mg/kg reached 29.1%.
The dose increases demonstrated greater than proportional improvements in effectiveness. This trend suggests the higher dose provides enhanced therapeutic benefit.
Safety Profile Remains Strong
PGN-EDODM1 was well-tolerated at the 15 mg/kg dose level. No serious treatment-related adverse events occurred during the study period.
All treatment-related adverse events were mild or moderate. Most events were transient and did not require medical intervention.
The safety data supports the potential for higher doses. This could lead to even better therapeutic outcomes in future studies.
$100 Million Funding Round
PepGen simultaneously announced a $100 million stock offering. The company priced 31.25 million shares at $3.20 per share through an underwritten public offering.
Leerink Partners and Stifel served as joint bookrunning managers. The offering is expected to close around September 26, 2025.
The company granted underwriters a 30-day option for additional shares. They can purchase up to 4.69 million more shares at the offering price.
PepGen plans to use proceeds for ongoing research and clinical development. This includes funding the FREEDOM-DM1 and FREEDOM2-DM1 clinical trials.
The funds will also support working capital and general corporate purposes. This provides runway for advancing the treatment pipeline.
Future Trial Timeline
PepGen anticipates reporting FREEDOM2 multiple ascending dose study results in Q1 2026. The first cohort data will provide insights into repeated dosing effects.
The company expects to begin dosing its 10 mg/kg cohort in the same quarter. This represents the next step in the dose escalation strategy.
The FREEDOM2 study will evaluate multiple doses over time. This approach could provide better long-term therapeutic outcomes than single treatments.
The company’s Enhanced Delivery Oligonucleotide platform uses cell-penetrating peptides. This technology improves therapeutic uptake and activity in target tissues.
PepGen closed the offering through a previously filed registration statement on Form S-3.
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