PepGen reports early Phase 2 results for myotonic dystrophy drug

PepGen said its experimental oligonucleotide drug PGN-EDODM1 showed early signs of activity in a Phase 2 trial for myotonic dystrophy type 1, a muscle-wasting condition.

After 13 weeks, six treated patients had an average 46.5% correction of splicing errors following 4 monthly doses, the company reported. Four patients had correction above 50%. PepGen said the results coincided with improvements on measures of mobility and muscle strength. Investigators rated treatment-related side effects mild to moderate, with nausea the most common.

PepGen is testing a higher dose and expects to report results in the first half of next year. Dosing has not occurred in the U.S. because of a partial hold related to animal-study findings flagged by the FDA, according to BioPharma Dive.