Strasbourg (France), April 30, 2019 

Company expects to initiate Phase 1 / 2 study ‘Unite-CNM’ in 2H2019 

Dynacure, a clinical stage drug development company focused on improving the lives of patients with rare and orphan disorders, today announced that The Medicines and Healthcare products Regulatory Agency (MHRA) has approved the Clinical Trial Application (CTA) for DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2) for the treatment of Centronuclear Myopathies (CNM).

Dynacure expects to initiate its first in human study, a Phase 1 / 2 study ‘Unite-CNM’, in the second half of 2019.

DYN101 is being developed in collaboration with Ionis Pharmaceuticals [NASDAQ: IONS], the leader in RNA-targeted drug discovery.

• “This is the first regulatory approval for a clinical trial of DYN101, representing a promising advancement for patients suffering from most forms of centronuclear and myotubular myopathies,” said Stephane van Rooijen (M.D. MBA), Chief Executive Officer of Dynacure. “With antisense, we believe there is an opportunity to reimagine the treatment of rare muscle-related disorders where no viable therapy exists.”
   
• Chris Freitag (M.D.), Chief Medical Officer of Dynacure, added, “Evaluating DYN101 in patients is an important next step in our efforts to understand the potential of this molecule to treat most forms of CNM and potentially other diseases driven by the overexpression of DNM2. The target engagement and efficacy observed in multiple mouse models of the disease give us great confidence in our ability to demonstrate potential disease-modifying results in humans.”
   
• Nicol Voermans (M.D. PhD), Neurologist Radboud University Medical Centre (the Netherlands) commented, “The start of clinical evaluation of the effect of DYN101 in patients greater than 16 years of age with X-linked and ADCNM is a promising advancement in the treatment of CNM. Dynacure’s approach has the potential to provide therapeutic benefit to a wide range of CNM patients by modulating DNM2.”