Today, the Medicines and Healthcare products Regulatory Agency (MHRA) has approved clinical trials of the drug DYN101.
This drug regulates the production of the enzyme dynamin 2 in the body. This could lead to an improvement in the symptoms of people with various centronuclear myopathies (CNM) (those with mutations in the genes MTM1 and DNM2). How exactly this drug works is explained here.
The company that has registered this study for approval is Dynacure. They estimate that in the second half of 2019, the first clinical trial will be conducted on approximately 18 people over the age of 16 with mutations in the MTM1 or DNM2 genes.
In a 12-week study, scientists will measure the muscle function and respiratory activity in the study participants, evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of DYN101.
Chris Freitag (MD), Chief Medical Officer of Dynacure, added, “The results in several mouse models have shown that this drug has a very positive effect and give us great confidence in our ability to alleviate the symptoms in people with CNM”.
About DYN101 for Centronuclear Myopathies
DYN101, an antisense oligonucleotide under investigation, is designed to regulate the expression of dynamin 2 (DNM2) for the treatment of centronuclear myopathies.