TLDR
- PepGen stock jumped 145% after achieving record 53.7% splicing correction in myotonic dystrophy patients
- Single dose treatment outperformed all previous DM1 clinical results with manageable side effects
- Company secured $100 million funding at $3.20 per share to advance clinical development
- FREEDOM2 study results expected Q1 2026 with multiple dose evaluation planned
- All six patients in highest dose group showed therapeutic improvement
PepGen shares rocketed 145% following groundbreaking clinical trial results for its myotonic dystrophy type 1 treatment. The biotechnology company reported unprecedented splicing correction rates that far exceed previous industry benchmarks.
The FREEDOM-DM1 Phase 1 study delivered a mean splicing correction of 53.7% after a single 15 mg/kg dose of PGN-EDODM1. This result represents the highest splicing correction ever reported in DM1 patients.
All six patients in the 15 mg/kg treatment group experienced improved splicing correction. The outcomes validate PepGen’s Enhanced Delivery Oligonucleotide platform technology.
Previous dose cohorts showed steady improvement with treatment escalation. The 5 mg/kg group achieved 12.3% correction while the 10 mg/kg group reached 29.1%.
The dose-response relationship exceeded proportional increases. This suggests higher doses could deliver even greater therapeutic benefits for patients.
Paul Streck, Executive Vice President of Research and Development, praised the study outcomes. He confirmed the trial met all primary objectives with the single-dose treatment approach.
Treatment Safety Profile Maintained
PGN-EDODM1 demonstrated good tolerability at the highest tested dose. No serious treatment-related adverse events occurred during the study period.
All recorded side effects were classified as mild to moderate in severity. Most adverse events resolved without requiring medical intervention or treatment discontinuation.
The positive safety data supports potential dose escalation in future studies. This could lead to improved therapeutic outcomes for myotonic dystrophy patients.
$100 Million Capital Raise Completed
PepGen simultaneously completed a $100 million public stock offering. The company issued 31.25 million shares priced at $3.20 each through underwritten placement.
Leerink Partners and Stifel managed the transaction as joint bookrunners. The offering includes a 30-day option for underwriters to purchase 4.69 million additional shares.
Proceeds will fund ongoing FREEDOM-DM1 and FREEDOM2-DM1 clinical trials. The capital also supports working capital requirements and general corporate activities.
The funding provides sufficient runway to advance the treatment through key development milestones. This includes multiple dose studies and regulatory preparation activities.
Clinical Development Timeline
PepGen expects to report FREEDOM2 multiple ascending dose study results in Q1 2026. The first cohort data will evaluate repeated dosing effects over time.
The company plans to initiate 10 mg/kg cohort dosing in the same quarter. This represents continued dose optimization within the multiple dose framework.
FREEDOM2 will assess whether repeated treatments provide sustained or enhanced therapeutic benefits. Multiple doses could improve long-term patient outcomes compared to single treatments.
The Enhanced Delivery Oligonucleotide platform uses cell-penetrating peptides to improve drug uptake. This technology enables better therapeutic delivery to target muscle tissues.
PepGen completed the offering through an existing Form S-3 registration statement with the SEC.
