Dynacure, a clinical stage biotechnology company, announced that the US Food and Drug Administration (FDA) has granted Fast Track Designation for its lead product candidate, DYN101. The Fast Track process is designed to facilitate the development and expedite the review of drugs to treat serious conditions and address significant unmet medical need.

DYN101 is currently being studied in UNITE-CNM, a multicenter phase 1/2 clinical trial for the two most common forms of CNM, X-linked myotubular myopathy (XLMTM) and autosomal dominant CNM (ADCNM). Initial data from this ongoing ascending dose study is expected within the second half of 2022.

“CNM is a progressively debilitating life-threatening disease with no approved therapies to help. Receiving Fast Track Designation will provide us with greater access to FDA and guidance on regulatory pathways,” said Leen Thielemans, chief development officer of Dynacure. “We appreciate this acknowledgement by FDA of the severity of CNM, and the urgency to support this patient community with safe and effective therapies.”

Myotubular and centronuclear myopathies (CNM) are serious, rare, life-threatening disorders that affect skeletal muscles from birth. People with CNM begin experiencing muscle weakness at any time from birth to early adulthood and many patients die within the first 18 months of life. Patients who survive longer require intense medical management and nearly uninterrupted support, including permanent ventilation, brace with head support and feeding tubes. CNM derives its name based on the central location of the muscle fiber nucleus, which is an abnormal finding observed in muscle biopsies. The disease is driven by mutations in multiple genes including MTM1, DNM2 and BIN1 and Dynacure scientists have discovered a link between an increase in DNM2 protein and the direct cause of the disease. The three classical forms of CNM are X-linked myotubular myopathy (XLCNM), autosomal dominant CNM (ADCNM), and autosomal recessive CNM (ARCNM), which are all associated with poor prognosis. Myotubular and centronuclear myopathies affect an estimated 4,000 to 5,000 patients in the European Union, United States, Japan and Australia.

Dynacure is 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.