Drug development company Dynacure has dosed the first patient in the Phase I/II Unite-CNM study to evaluate investigational antisense medicine DYN101.
DYN101 is intended to modulate the expression of dynamin 2 (DNM2) for the treatment of Myotubular and Centronuclear Myopathies (CNM).
CNMs, which affect skeletal muscles from birth, are rare, life-threatening disorders. Around 5,000 patients are affected by the disease in the EU, US, Japan and Australia.
The multi-centre, ascending dose study, Unite-CNM (DYN101-C101), aims to examine the tolerability, safety, pharmacokinetics and preliminary efficacy of DYN101 in around 18 patients, above the age of 16 years, with X-linked (XLCNM) or autosomal dominant CNM (ADCNM).
The study will primarily focus on detecting an optimal dose of the drug. It will also access different domains of efficacy in an exploratory analysis involving muscular function, respiratory function and muscle strength.
Once Unite-CNM study is completed, the company will analyse a potentially registration-directed Phase II/III study on all age groups.
Dynacure is developing DYN101 in collaboration with Ionis Pharmaceuticals, a company known for its RNA-targeted drug discovery.
Preclinical efficacy of the medicine was observed in multiple mouse models of the disease.
Dynacure chief medical officer Chris Freitag said: “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 I/II ‘Unite-CNM’ study.”