STRASBOURG/PHILADELPHIA, June 9, 2021 –
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.
About Myotubular and Centronuclear Myopathies
Myotubular and Centronuclear Myopathies (CNM) are serious, rare, life-threatening disorders that affect skeletal muscles from birth. CNM derives its name based on the central location of the muscle fiber nucleus, which is an abnormal finding observed in muscle biopsies. 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. 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 4. 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 Australia1.
DYN101, an investigational antisense oligonucleotide product candidate using Ionis Pharmaceuticals’ proprietary antisense technology, is designed to reduce the expression of dynamin 2 protein (DNM2) for the treatment of Myotubular and Centronuclear Myopathies (CNM). Preclinical studies have shown that DYN101 has the potential to be disease modifying in CNM, with preclinical activity observed in animal models of XLCNM and ADCNM 2,3 . Prevention and reversion of the disease was observed with a dose-dependent improvement in whole body strength and mice survival. The development plan for DYN101 was designed to be broad and it is the only known program investigating a treatment for the majority of CNM patients, comprised of those who have XLCNM or ADCNM. DYN101 is currently under clinical investigation in several European countries. DYN101 has been granted Orphan Drug designations by the FDA and EMA and rare pediatric disease designation by the FDA.
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. The Dynacure team leverages its proven track record in rare disease drug development to build a pipeline of novel drug candidates. Dynacure is developing DYN101, an investigational antisense product candidate designed to reduce the expression of dynamin 2 protein for the treatment of Myotubular and Centronuclear Myopathies, in strategic collaboration with Ionis Pharmaceuticals. Dynacure is also building a complementary research portfolio targeting other rare disorders, including its DYN201 program for the treatment of Hereditary Spastic Paraplegias (caused by mutations in the SPG11 gene).
Dynacure is headquartered in Strasbourg, France with a corporate office in Philadelphia, PA, USA.
For more information, please visit www.dynacure.com.
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Lori McKenna Gorski
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1. Neuromuscul Disord. 2018 Sep;28(9):766-777. doi: 10.1016/j.nmd.2018.06.012. Epub 2018 Jul
2. Nat Commun. 2017 Jun 7;8:15661. doi: 10.1038/ncomms15661.
3. Proc Natl Acad Sci U S A. 2018 Oct 23;115(43):11066-11071. doi: 10.1073/pnas.1808170115. Epub 2018 Oct
4. (Cowling et al 2014 JCI)