PepGen’s experimental oligonucleotide drug PGN-EDODM1 repaired, on average, 46.5 percent of the splicing errors caused by myotonic dystrophy type 1 after four monthly doses in six treated patients, with four of the six above 50 percent, and the company said the results were the highest splicing correction yet seen for a multi-dose DM1 drug. Side effects were judged mild to moderate, with nausea most common and no sign of cumulative kidney damage over 16 weeks, though dosing in the United States remains under a partial FDA hold dating to last year and results from a higher dose cohort are expected in the first half of next year. The same day, PepGen filed a preliminary prospectus supplement to sell $125 million of common stock and pre-funded warrants, raising money into a share price that rose more than 90 percent on the data.
PepGen shares surge on myotonic dystrophy drug data
PepGen shares rose more than 90 percent on Tuesday after its experimental drug PGN-EDODM1 corrected an average of 46.5 percent of splicing errors in six patients with myotonic dystrophy type 1, and the company filed to raise $125 million in stock and pre-funded warrants.
Why it matters: No curative therapy exists for myotonic dystrophy type 1, and PepGen's splicing correction topped figures reported for rival candidates from Novartis and Dyne, though US dosing remains under a partial FDA hold and the company is now selling shares into the rally.
Data as of BioPharma Dive report of November 10, 2026, read in full, reporting PepGen's own statement of the same day on the Phase 2 FREEDOM trial of PGN-EDODM1; the offering figures are from PepGen's preliminary prospectus supplement on Form 424B5 filed with the SEC on November 10, 2026 (accession 0001193125-26-455711), read from the filings lane copy. The trial data are the company's own, in six treated patients, and have not been independently verified. Developing item.
Sources
- PepGen soars on promising early data for muscular dystrophy drug - BioPharma Dive - 2026-11-10
- PepGen Inc., preliminary prospectus supplement on Form 424B5, $125,000,000 common stock and pre-funded warrants (accession 0001193125-26-455711) - PepGen Inc. (SEC EDGAR) - 2026-11-10