PepGen Inc. (NASDAQ:PEPG) stock surged on Tuesday with a strong session Volume of 31.855 million, compared to an average volume of 983.011 thousand, according to Benzinga Pro data.
The clinical-stage biotechnology company reported positive topline results from the Phase 2 FREEDOM2-DM1 study, highlighting significant splicing correction and functional gains in patients with myotonic dystrophy type 1, a rare muscular disorder.
These developments arrive as Dyne Therapeutics Inc. (NASDAQ:DYN) nears completion of a pivotal trial, while Novartis AG (NYSE:NVS) recently reported a Phase 3 trial failure for its acquired DM1 candidate, creating sector uncertainty.
Record Splicing Correction In Muscle Tissue
Patients who received the 10 mg/kg dose achieved a 46.5% average muscle splicing correction at Week 13. In contrast, individuals in the pooled placebo arm averaged a 6.8% correction.
Five of the six treated patients achieved greater than 20% correction, while four surpassed 50%.
The company stated that these outcomes mark the highest level of splicing correction ever reported in a multiple-dose trial for the rare genetic condition.
Notable Functional And Mobility Improvements
The trial also demonstrated meaningful physical gains across several assessments through Week 16.
Video Hand Opening Time, an objective measure of myotonia, improved by 3.9 seconds, or approximately 52.7%, while the placebo arm improved by just 0.2 seconds.
Investigators also documented dose-dependent splicing correction of CLCN1, a primary driver of myotonia.
Additionally, patients registered a 16.2% improvement in hand grip strength.
Mobility tests showed further progress, with the Timed Up and Go assessment improving by 8.5% and the 10-Meter Walk/Run Test gaining 6.8%.
Patient-reported outcome measures yielded inconclusive results at Week 16, displaying favorable directional trends on one scale alongside mixed results on another.
Strong Safety Profile And Competitive Outlook
The 10 mg/kg regimen demonstrated favorable tolerability with no severe adverse events, treatment discontinuations, or cumulative kidney toxicity.
Researchers noted mild nausea as the most common side effect, alongside four mild instances of elevated urine protein that resolved with subsequent doses.
PepGen expects to release clinical results from its fully enrolled 12.5 mg/kg cohort in the first half of 2027. The drugmaker also plans to share open-label extension data in January 2027.
PEPG Price Action: PepGen shares were up 111.30% at $5.05 at the time of publication on Tuesday, according to Benzinga Pro data.
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