Clinical trial recruitment
Unite-CNM, the Dynacure sponsored clinical trial of their antisense product, DYN101, began recruiting in early 2020 at sites in the UK, Belgium and Netherlands, with an additional five European sites to follow.
Pre-clinical studies have shown that ‘turning down’ Dynamin 2 can reduce the symptoms of centronuclear myopathy, so the clinical trial will evaluate DYN101 for safety, tolerability, pharmacokinetics (how the drug works around the body) and preliminary efficacy (the effect on symptoms). It will include approximately 18 patients, who are over 16 years of age and have XLMTM or DNM2 mutations.
Further information about the trial, including inclusion and exclusion criteria, site location, recruitment status and contact details can be found below.
- Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies (Unite-CNM)
Dynacure announces first patient dosed in phase 1 / 2 ‘UNITE-CNM’ Study of DYN101 for the treatment of centronuclear myopathies
In March, Dynacure announced that a patient had been dosed with DYN101. This milestone marks the first time any company has dosed a CNM patient with an antisense medicine. For further information view the press release below.
- Dynacure Announces First Patient Dosed in Phase 1 / 2 ‘UNITE-CNM’ Study of DYN101 for the Treatment of Myotubular and Centronuclear Myopathies (CNM)
Dynacure Announces €50M ($55M) Series C Financing
In March, Dynacure announced €50M ($55M) Series C Financing. The funding will support ongoing Phase 1 /2 ‘Unite-CNM’ study of DYN101 in patients with centronuclear and myotubular myopathy.
Dynacure announce that circulating myostatin levels are altered in centronuclear myopathy mice and patients and myostatin levels respond directly to DNM2-therapy in mice. Myostatin is now currently being explored as a biomarker in our Unite-CNM clinical trial.
- Myostatin: A Circulating Biomarker Correlating With Disease in Myotubular Myopathy Mice and Patients
In July the first two patients were dosed with investigational drug DYN101 in UNITE-CNM trial and Dynacure CSO Belinda Cowling was also named a finalist for the EU Prize for Women Innovators 2020.
Dynacure also received rare pediatric disease designation from the US Food and Drug Administation for DYN101 for myotubular and centronuclear myopathies.
- Dynacure receives rare pediatric disease designation from U.S. Food and Drug Administration for DYN101 for myotubular and centronuclear myopathies