Global Study of Del-desiran for the Treatment of DM1

Summary

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1

Timeline

Start

2024-05-30

Primary completion

2026-08

Completion

2026-09

Outcome

Missed primary endpoint

release “HARBOR did not meet its primary endpoint of vHOT in DM1” novartis.com ↗

Drugs

Indications