STRASBOURG, France and PHILADELPHIA, Sept. 28, 2020 /PRNewswire/ -- Dynacure, a clinical stage drug development company focused on improving the lives of patients with rare and orphan disorders, announced today that the company will present two posters containing data on the natural history of Myotubular and Centronuclear Myopathies (CNM) at the 25th Annual Congress of the World Muscle Society (WMS25). The WMS25 meeting is being held in a virtual format.
Dynacure is developing DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2), for the treatment of CNM. DYN101 is currently being evaluated in a Phase 1/2 clinical study, 'UNITE-CNM' (DYN101-C101), at multiple clinical sites in Europe.
Details of the poster presentations are listed below:
Poster Abstract: # LSP (late submitted poster) 6 Title: Hierarchical Bayesian model of disease progression in centronuclear myopathy allows to demonstrate treatment efficacy with a small sample size Date: Thursday, October 1, 2020, 17:30-19:30
Poster Abstract: # LSP 7 Title: Clinical and genetic aspects of DNM2-related centronuclear myopathy; a retrospective, medical chart review to establish natural history Date: Thursday, October 1, 2020, 17:30-19:30
"We are pleased to continue to present our data at the WMS25 meeting, which is globally recognized as one of the most important scientific meetings to advance research for serious muscle diseases," said Leen Thielemans, Dynacure's Chief Development Officer. "We would like to take this opportunity to thank the experts and recognized leaders in the CNM field for their collaboration to advance our understanding of these rare, life-threatening disorders."
"The data presented provide additional insight into the progression of myotubular and centronuclear myopathies in patients, which supports and informs our ongoing Phase 1 / 2 UNITE-CNM study evaluating DYN101 for CNM," said Chris Freitag, Dynacure's Chief Medical Officer. "In particular, one poster examines the options for analyzing such data and using them in an innovative way to aid clinical research in other rare diseases."
The 2020 WMS25 posters will be available on Dynacure's website and further details on the WMS25 meeting can be accessed here: https://www.wms2020.com/.
About Myotubular and Centronuclear Myopathies
Myotubular and Centronuclear Myopathies (CNM) are serious, rare, life-threatening disorders that affect skeletal muscles from birth. CNMs derive their 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 the link between an increase in DNM2 and the direct cause of the disease4. There are many genetic forms of CNM including X-linked recessive (XLCNM/ Myotubular Myopathy), autosomal dominant (ADCNM), and autosomal recessive (ARCNM), which are all associated with poor prognosis. Myotubular and Centronuclear Myopathies affect between 4,000 and 5,000 patients in the EU, US, Japan and Australia1.
DYN101, an investigational antisense oligonucleotide using Ionis Pharmaceuticals' proprietary antisense technology, is designed to modulate the expression of dynamin 2 (DNM2) for the treatment of Myotubular and Centronuclear Myopathies (CNM). Preclinical studies have demonstrated that DYN101 has the potential to be disease modifying in CNM, with compelling preclinical efficacy in treating animal models of XLCNM and ADCNM2,3. Prevention and reversion of the disease was observed with a clear dose-dependent improvement in whole body strength and mice survival. The development plan for DYN101 was designed to be very broad and it is the only known program being investigated for most CNM populations, XLCNM and ADCNM. DYN101 has been granted Orphan Drug designations by the US FDA and EMA.
About the Phase 1 / 2 Study 'Unite-CNM' (DYN101-C101)
'Unite-CNM' (DYN101-C101) study is a multicenter, ascending dose study to evaluate the safety, tolerability, pharmacokinetics and preliminary efficacy of DYN101 in approximately 18 patients greater than 16 years of age with X-linked (XLCNM) or autosomal dominant CNM (ADCNM). Enrolled patients will have a run-in period or be rolled over from an ongoing natural history study, sponsored by the Institute of Myology in France, which includes 60 subjects that have XLCNM or ADCNM. While the Phase 1 / 2 study will primarily focus on finding an optimal dose of the drug via safety, tolerability and after 12 weeks of treatment, multiple domains of efficacy will also be assessed in an exploratory analysis, which include muscular function, respiratory function and muscle strength. After completing the Unite-CNM study, Dynacure expects to investigate a potentially registration-directed Phase 2 / 3 study (all age groups) that would include US and European sites.
Dynacure is a clinical-stage drug development company focused on improving the lives of patients with rare and orphan diseases. The Dynacure team leverages its strong track record in rare disease drug development to build a pipeline of novel drugs. Dynacure is developing DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 for the treatment of Myotubular and Centronuclear Myopathies, with Ionis Pharmaceuticals. The company maintains its headquarters in Strasbourg, France and a corporate office in Philadelphia, PA, USA. Dynacure's investors are Andera Partners, Bpifrance Large Venture, Bpifrance through its FABS and Fonds Biothérapies Innovantes et Maladies Rares funds, Idinvest, Ionis Pharmaceuticals, Kurma Partners, Perceptive Advisors, Pontifax and funds managed by Tekla Capital Management LLC.
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)