Dynacure Announces FDA Acceptance of Investigational New Drug Application for DYN101 for Myotubular and Centronuclear Myopathies

Dynacure, a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, announced today that the US Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for DYN101, an investigational antisense product candidate designed to reduce the expression of dynamin 2 protein (DNM2), for the treatment of Myotubular and Centronuclear Myopathies (CNM). DYN101 is currently being evaluated in a Phase 1/2 clinical trial, UNITE-CNM (DYN101-C101), at multiple clinical sites in Europe. DYN101 has been granted orphan drug designation by the FDA and European Medicines Agency (EMA) and rare pediatric disease designation by the FDA. There is currently no FDA- or EMA-approved therapeutic treatment for CNM.

“The acceptance of our IND for DYN101 builds upon the clinical development path we are following in the EU and will implement in the US with our ongoing UNITE-CNM trial,” said Leen Thielemans, Chief Development Officer of Dynacure. “There remains significant unmet medical need for the treatment of CNM and we look forward to expanding our Phase 1/2 clinical trial of DYN101 into the United States.”

“FDA acceptance of the IND for DYN101 significantly advances our clinical development of DYN101 by expanding our trial into the United States and underscores our commitment to deliver a novel medicine for CNM patients around the world,” added Stephane van Rooijen, Chief Executive Officer of Dynacure.

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