‘Unite-CNM’ (DYN101-C101) is a European multicenter, ascending dose study to evaluate the safety, tolerability, pharmacokinetics and preliminary efficacy of DYN101 in approximately 18 patients greater than 16 years of age with XLCNM or ADCNM. Enrolled patients will have a run-in period or be rolled over from an ongoing natural history study, sponsored by the Institute of Myology in France, which includes 60 subjects that have XLCNM or ADCNM.
While the Phase 1 / 2 study will primarily focus on finding an optimal dose of the drug via safety, tolerability and target attainment after 12 weeks of treatment, multiple domains of efficacy will also be assessed in an exploratory analysis, which include muscular function, respiratory function and muscle strength.
After completing the Unite-CNM study, Dynacure expects to investigate a potentially registration-directed Phase 2 / 3 study (all age groups) that would include European and US sites.
Dear CNM Community :
At the request of CNM patient organizations, we would like to give some information on our ongoing work to bring our investigational product DYN101 through clinical development and approval for use in the treatment of CNM patients with MTM-1 or DYN2 mutations.
Please be aware that we are still in the phase of developing DYN101 and as such are limited as to what we can present according to regulations around the world. The scientific background is complex, so that we have tried to simplify it as much as possible to be understandable and there is a glossary at the end that tries to explain some of the terms used.