Clinical Trial for Experimental Centronuclear Myopathy Drug Given the Green Light

According to a story from BioSpace, the drug development company Dynacure recently released an announcement in which the company stated that its application for a Phase 1/2 clinical trial had gained approval from the The Medicines and Healthcare Products Regulatory Agency. This clinical trial will test the company’s experimental drug called DYN101. An antisense drug that is designed to modulate dynamin 2 (DNM2) expression, DYN101 is intended to treat a group of diseases called centronuclear myopathies.

About Centronuclear Myopathy

Centronuclear myopathies are a form of congenital myopathies that are characterized by the abnormal location of the nucleus, a critical cellular organelle, in the cells of the skeletal muscles. Centronuclear myopathies can appear in a variety of different patterns of inheritance and symptoms can appear from the beginning of life or may be delayed until later. The different types of myopathy are distinguished by which genetic mutation causes them. The most common form is X-linked myotubular myopathy, which is ultimately lethal; some patients may die in infancy before being diagnosed. Symptoms include lack of muscle tone, muscle weakness, difficulty breathing and feeding, developmental delays, long digits, distinct facial features, and a bell shaped chest. Centronuclear myopathies cannot be cured and treatment is primarily supportive and symptomatic. To learn more about centronuclear myopathies, click here.

About DYN101 and The Clinical Trial

DYN101 is being developed in collaboration with Ionis Pharmaceuticals. The treatment has shown potential to be disease modifying in animal models of centronuclear myopathies. In these models, the investigational therapy was able to prevent symptoms and reverse them when they appeared, leading the survival of the mice in the study and improvements in body strength. 

The clinical trial is expected to include a total of 18 patients that are 16 years or older that have either the X-linked or the autosomal dominant form of centronuclear myopathy. The goal of this trial will be to test the safety and tolerability of DYN101 as well as determining the optimal dose for efficacy during a 12 week period of treatment.

There is an urgent need for an effective, disease modifying treatment centronuclear myopathy. Hopefully, this trial will produce encouraging results for DYN101.


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