On 5th March 2020, Dynacure announced that the first myotubular myopathy patient has been dosed with DYN101 in ‘UNITE-CNM’ – their clinical trial for centronuclear myopathies. DYN101 is an antisense oligonucleotide (ASO), which is expected to decrease the production of a protein, named dynamin-2, which is elevated in patients with X-linked MTM, and thought to be overly active in patients with the DNM2 mutation as well. This is the first time a patient with myotubular myopathy has ever been dosed with antisense treatment.
Unite CNM is a European, multicenter, ascending dose study to evaluate DYN101 for safety, tolerability, pharmacokinetics (how the drug works around the body) and preliminary efficacy (the effect on MTM and CNM symptoms). It will include approximately 18 patients, who are over 16 years of age, and have XLMTM or DNM2 mutations. Centres for this trial are now open in 3 locations in the UK, Belgium and the Netherlands, with a further 5 sites due to open.
Read more about the trial, including inclusion and exclusion criteria, site location, recruitment status and contact details.
“We are committed to providing CNM patients and their families with a therapeutic solution to treat this devastating, progressive muscle disease. Today, we are pleased to have made progress toward this goal with the first dose of DYN101 in a patient in our Phase 1 / 2 ‘Unite-CNM’ study,” said Chris Freitag (M.D.), Chief Medical Officer of Dynacure. “We look forward to gaining valuable clinical insights from this study and we expect to report interim results from ‘Unite-CNM’ in 2021.”
