This story first appeared in the Information Point newsletter Our World in 2017 when the biotechnology company Dynacure announced its participation in the ‘Natural History Study and Functional Status of Patients with Myotubular Myopathy and other Centronuclear Myopathies’.
Dynacure is a biotechnology company developing new treatments for patients affected by serious orphan disorders. The organisation recently announced its participation in the ‘Natural History Study and Functional Status of Patients with Myotubular Myopathy and other Centronuclear Myopathies’ (NatHis-CNM). Founded in 2016 as a spin-off from the IGBMC (Institute of Genetic and Molecular and Cellular Biology – Unistra/INSERM/CNRS) of Strasbourg.
In its first drug discovery program, Dynacure is focusing on centronuclear myopathies (CNM). Dynacure’s Dyn101 development program is based on the modulation of the expression of the Dynamin 2 protein through the use of an antisense oligonucleotide developed in collaboration with Ionis Pharmaceuticals, the leading biopharmaceutical company in RNA-targeted drug discovery.
Ionis Pharmaceuticals recently announced the license, to Dynacure, of IONIS-DNM2-2.5Rx (Dyn101), a Generation 2.5 antisense drug targeting Dynamin 2 for the treatment of centronuclear myopathy. Dynacure will now assume all development and commercialisation responsibilities for the program.
About the study
This prospective non-interventional longitudinal study currently investigates the natural history and function of 60 patients with MTM and other CNM from Europe, United States and Canada. The study was started in 2014 by the Institute of Myology, Genethon and Valerion Therapeutics to collect data on patients with the MTM1 gene. The study objective is to follow-up the disease course, changes in disease severity and any progression of symptoms.
Dynacure will continue the study with the Institute of Myology (its sponsor) and extend it for 24 months, with Dr Laurent Servais from the Institute I-Motion (located at the Hôpital Trousseau, Paris France) as the coordinating investigator. Other subtypes of patients resulting from a mutation in BIN1 (autosomal recessive centronuclear myopathy) and DNM2 (autosomal dominant centronuclear myopathy) will also be enrolled.
Study specific functional assessments and patient questionnaires will be used and will be based on age and ambulatory status of the participant. Data from the study will be used to characterise the disease of and determine which outcome measures will be the best to assess the efficacy of potential therapies in future clinical trials.
Anne Lennox, from the Myotubular Trust said: “The Myotubular Trust thank Dynacure and the Institute of Myology for the extension of this NatHis-CNM study which will be crucial in understanding and quantifying the evolution of the disease. We are particularly pleased to see the inclusion in the study of more forms of centronuclear myopathy. Participants are children and young adults affected by this debilitating disease for which no treatment exists yet”.
Laurent Servais, coordinating investigator added: “I’m grateful to Genethon and Valerion for their support in the first 36 months of the study. The generated data are a source of valuable scientific medical knowledge. I’m very excited to expand this unique study with Dynacure to build a solid comparator platform for future clinical trials to evaluate new therapies”.
Stephane van Rooijen, of Dynacure concluded: “Dynacure is well positioned to drive forward its lead program in centronuclear myopathies in order to develop new therapeutic approaches and help patients who suffer from CNM. By joining the NatHis-CNM effort, Dynacure confirms its commitment to support patients and physicians to enhance medical knowledge on the natural course of this rare disease”.