Michigan survey ahead; Applied Digital surges; gold dips - what’s moving markets
BOSTON - PepGen Inc. (NASDAQ:PEPG), a clinical-stage biotechnology company with a market capitalization of $64 million, announced Wednesday that its experimental treatment for myotonic dystrophy type 1 (DM1) achieved a mean splicing correction of 53.7% following a single 15 mg/kg dose in its ongoing Phase 1 FREEDOM-DM1 study. According to InvestingPro data, the company’s stock has shown strong momentum, gaining over 14% in the past six months despite broader market volatility.
The clinical-stage biotechnology company reported that all six patients receiving the 15 mg/kg dose of PGN-EDODM1 showed improved splicing correction, a key measure of effectiveness in treating the underlying cause of DM1. While the company maintains a strong liquidity position with a current ratio of 4.74 and more cash than debt on its balance sheet, InvestingPro analysis indicates the company is rapidly burning through its cash reserves, typical for early-stage biotech firms.
The latest results demonstrated greater than dose-proportional increases compared to previously reported data from lower dose cohorts, which showed mean splicing correction of 12.3% at 5 mg/kg and 29.1% at 10 mg/kg.
PGN-EDODM1 was generally well-tolerated at the 15 mg/kg dose level, with no serious treatment-related adverse events reported. All treatment-related adverse events were mild or moderate and transient, according to the company.
"Since mis-splicing is the underlying cause of DM1, we believe high levels of splicing correction have the potential to reverse the underlying molecular defects," said Paul Streck, Executive Vice President of Research and Development at PepGen, in the press release.
The company also observed greater than dose-proportional increases in muscle tissue concentrations of PGN-EDODM1 across all dose levels at Day 28.
PepGen’s Enhanced Delivery Oligonucleotide (EDO) technology is designed to deliver therapeutic oligonucleotides that restore normal splicing function of MBNL1, a key RNA splicing protein affected in DM1.
The company expects to report results from the first cohort of its FREEDOM2-DM1 multiple ascending dose study in the first quarter of 2026.
DM1 is an inherited progressive disorder affecting approximately 40,000 patients in the United States. The condition primarily impacts skeletal, cardiac and smooth muscle, causing symptoms including muscle rigidity, weakness, cardiac abnormalities, and respiratory problems.
In other recent news, PepGen Inc. announced it has begun an underwritten public offering of its common stock and pre-funded warrants. The company has also planned to grant underwriters a 30-day option to purchase additional shares. Leerink Partners and Stifel are acting as joint book-running managers for this offering. The offering is subject to market conditions, and there is no guarantee it will be completed. These developments are part of PepGen’s recent activities in the market.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.