Dyne's Investigational DMD Drug DYNE-251 Gets Japan Orphan Drug Status After Strong Trial Data

Dyne Therapeutics Inc. (NASDAQ:DYN) is one of the best stocks to buy under $20. On September 29, Dyne Therapeutics announced that Japan’s Ministry of Health, Labour and Welfare/MHLW granted Orphan Drug designation for its investigational therapeutic, DYNE-251. This designation is for individuals with Duchenne muscular dystrophy/DMD who have mutations in the DMD gene amenable to exon 51 skipping.

The company’s Chief Medical Officer, Doug Kerr, highlighted that data from the ongoing DELIVER trial showed unprecedented and sustained functional improvement through 18 months, driven by significant dystrophin expression.

DYNE-251 is being evaluated in the global, randomized, placebo-controlled, double-blind Phase 1/2 DELIVER clinical trial. The Orphan Drug designation in Japan is for drugs treating rare diseases affecting fewer than 50,000 patients and with a high medical need.

Dyne Therapeutics Inc. (NASDAQ:DYN) is a clinical-stage neuromuscular disease company that discovers and develops therapeutics for neuromuscular diseases in the US.

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Disclosure: None. This article is originally published at Insider Monkey.