PepGen's DM1 Oligonucleotide Posts Strong Splicing Correction in Small Phase 2 Cohort; Shares Double
- PGN-EDODM1 corrected on average about 46.5% of disease-linked splicing errors in six DM1 patients at 10 mg/kg after four monthly doses, with improvements on several mobility and strength tests.
- Side effects were mild to moderate; a transient rise in urine protein followed the first dose with no sign of cumulative kidney effects.
- A higher-dose cohort is due in the first half of next year; US dosing remains under a partial FDA clinical hold linked to animal findings.
💡 Points Based on public reports
Analysts put the splicing correction above Novartis's del-desiran and Dyne's z-basivarsen, but six patients and cross-trial comparison are weak footing, and splicing is a biomarker, not function. Novartis's recent pivotal miss shows that gap can bite. The unresolved clinical hold is the real overhang: until it lifts, PepGen can't run the US study that would test whether the lead holds up.
📰 Source · BioPharma Dive ↗ PepGen soars on promising early data for muscular dystrophy drug