PepGen Inc. (NASDAQ: PEPG) stock is skyrocketing on Thursday, with a session volume of 53.04 million compared to the average volume of 943.2 thousand as per data from Benzinga Pro.
PepGen on Wednesday released clinical data today from the 15 mg/kg dose cohort of its ongoing FREEDOM-DM1 Phase 1 single ascending dose (SAD) study in patients with myotonic dystrophy type 1 (DM1).
Myotonic dystrophy is a rare, inherited genetic disorder characterized by progressive muscle weakness and myotonia, which is the inability of muscles to relax after a contraction.
It is a multisystemic condition affecting not only muscles but also the heart, brain, eyes, endocrine system, and other organs.
Also Read: PepGen Abandons Duchenne Program, Shifts Spotlight To Another Rare Disease Candidate
Data
FREEDOM Results for the 15 mg/kg (n=8) dose cohort, splicing and muscle concentration data:
Mean splicing correction of 53.7% in patients receiving 15 mg/kg of PGN-EDODM1 (n=6), as measured by the 22-gene panel at 28 days post-dosing. The splicing deregulation induces an aberrant protein expression that provokes the loss of cell function and viability.
The company previously reported mean splicing correction at 5 mg/kg (n=6) and at 10 mg/kg (n=4) of 12.3% and 29.1%, respectively, demonstrating greater than dose-proportional increases in splicing correction.
All six patients (100%) receiving a 15 mg/kg dose of PGN-EDODM1 responded to treatment by showing improved splicing correction.
In addition, greater than dose-proportional increases in muscle tissue concentrations of PGN-EDODM1 were observed across 5 mg/kg (n=6), 10 mg/kg (n=5) and 15 mg/kg (n=6) at Day 28.
PGN-EDODM1 was generally well-tolerated at 15 mg/kg, with no serious treatment-related adverse events (AEs).
All treatment-related AEs at 15 mg/kg were mild or moderate, transient, and with the exception of one patient (who received oral OTC antihistamines), did not require intervention.
There were no electrolyte-related AEs, including an absence of hypomagnesemia AEs.
All renal biomarker-related AEs were asymptomatic, transient (~48hrs) and resolved without intervention.
A transient and reversible kidney biomarker elevation in one patient qualified as a dose-limiting toxicity, as defined by the study protocol, and was classified as a mild AE, which resolved without intervention. The patient remained in the study.
What Next?
PepGen anticipates reporting results from the FREEDOM2-DM1 MAD study 5 mg/kg cohort in the first quarter of 2026 and also expects to begin dosing its 10 mg/kg cohort in the first quarter of 2026.
On Thursday, PepGen priced its offering of 31.25 million shares at $3.20 per share, gross proceeds expected to be $100 million.
PEPG Price Action: PepGen shares were up 122.18% at $5.91 at the time of publication on Thursday. The stock is trading within its 52-week range of $0.88 to $9.93, according to Benzinga Pro data.
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