#Myotonic-Dystrophy

2 stories

Clinical Trials May 13, 2026 · 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 May 9, 2026 · View digest

Vertex Q1: VX-670 Cyclic-Peptide-Oligonucleotide DM1 Conjugate On Track for GALILEO Phase 1/2 Readout in H2 2026

Vertex Pharmaceuticals' Q1 business update confirmed continued enrollment and dosing in the multiple-ascending-dose portion of GALILEO, the global Phase 1/2 study of VX-670 in adults with myotonic dystrophy type 1, with results guided for H2 2026. VX-670 is an oligonucleotide linked to a cyclic peptide endosomal-escape vehicle from Entrada Therapeutics' EEV platform; the oligonucleotide engages CUG-repeat RNA to liberate bound MBNL1 splicing factor and correct the upstream missplicing that drives DM1 pathology. The trial is the first clinical readout for the Vertex–Entrada DM1 collaboration, originally signed February 2023 with $250M upfront. DM1 has no disease-modifying therapy.