Peptide News Digest

#Duchenne-Muscular-Dystrophy

2 stories

Clinical Trials · View digest

PepGen Pivots to DM1: Discontinues DMD Program After 10 mg/kg PGN-EDO51 Delivers Only 0.59% Dystrophin, Focuses on FREEDOM2-DM1 Phase 2

PepGen announced in May 2026 a strategic pivot away from Duchenne muscular dystrophy after the 10 mg/kg cohort of CONNECT1-EDO51 produced only 0.59% of normal dystrophin levels in four patients — well below the threshold of clinical meaningfulness. The company will discontinue DMD program development and focus on the FREEDOM2-DM1 Phase 2 program in myotonic dystrophy type 1, with 5 mg/kg cohort data anticipated. PepGen's Enhanced Delivery Oligonucleotide (EDO) platform conjugates peptide carriers to phosphorodiamidate morpholino oligomers (PMOs) for tissue-targeted delivery. The DM1 program competes with Vertex's VX-670 cyclic peptide-oligonucleotide conjugate (GALILEO Phase 1/2, H2 2026 readout) and Sarepta/Avidity Biosciences in the same indication.

Clinical Trials · View digest

Entrada Therapeutics ENTR-601-44 Phase 1/2 ELEVATE-44-201 Topline (May 7): 2.36% Mean Dystrophin Increase, Statistically Significant Time-to-Rise Velocity, Clean Safety at 6 mg/kg in Duchenne Muscular Dystrophy

Entrada Therapeutics announced May 7 positive topline results from Cohort 1 of the Phase 1/2 ELEVATE-44-201 study of ENTR-601-44, the first-in-class Endosomal Escape Vehicle (EEV) peptide-PMO conjugate for exon-44-amenable Duchenne muscular dystrophy. At 6 mg/kg, treated participants showed mean dystrophin increase of 2.36% from a 4.00% baseline, exon-skipping increase of 2.31% from a 2.66% baseline, and a statistically significant improvement in Time-to-Rise (TTR) velocity. No serious adverse events or treatment-driven discontinuations; the most common AE was headache. All eight Cohort 1 participants transitioned to the open-label portion. Cohort 2 (12 mg/kg) is now dosing, with year-end 2026 readouts planned for Cohort 1 OLE and Cohort 2 MAD; Cohort 3 (up to 18 mg/kg) follows.