FDA clears Dyne Therapeutics IND for DYNE-302 in rare muscle disease

Dyne Therapeutics, Inc. (Nasdaq: DYN) announced that the U.S. Food and Drug Administration has cleared its investigational new drug application for DYNE-302, an experimental treatment for facioscapulohumeral muscular dystrophy, known as FSHD.

FSHD is a rare, progressive genetic disease caused by a mutation in the DUX4 gene that leads to skeletal muscle loss, weakness, and wasting. An estimated 15,000 to 40,000 individuals in the United States and approximately 20,000 to 50,000 in Europe are affected by FSHD. No approved therapies currently exist for the condition.

DYNE-302 is designed to use a transferrin receptor 1-targeting antibody fragment to deliver a small interfering RNA payload to skeletal muscle with the aim of suppressing DUX4 expression. The candidate uses the same FORCE platform as the company's two other clinical programs targeting Duchenne muscular dystrophy and myotonic dystrophy type 1.

Dyne plans to conduct a Phase 1 randomized, placebo-controlled, double-blind, multiple ascending dose trial in ambulatory adults with FSHD. The primary endpoint will be safety and tolerability. The trial will also assess pharmacokinetics and pharmacodynamics, including changes in muscle DUX4 transcriptome and plasma KHDC1L levels.

The first cohort will enroll nine participants who will receive three intravenous doses every four weeks, randomized 2:1 to DYNE-302 at 1.5 mg/kg or placebo. Dyne said it intends to evaluate higher doses and less frequent administration in subsequent cohorts. Participants who complete the placebo-controlled period may enter an open-label extension to receive DYNE-302 for up to an additional 96 weeks.

In preclinical models, administration of DYNE-302 resulted in knockdown of the DUX4 transcriptome in skeletal muscle, reversal of muscle fiber damage, and functional improvement. Dyne said it intends to pursue a traditional approval pathway in the U.S. for DYNE-302.

You May Also Be Interested In

• FDA authorizes 11 ZYN Ultra nicotine pouches for Philip Morris
• Silexion Therapeutics faces Nasdaq delisting over equity shortfall
• Nu Skin EVP of global sales takes medical leave