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Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-101
- Initial Data from Global, Multiple Ascending Dose Phase 1/2 ACHIEVE Clinical Trial in Myotonic Dystrophy Type 1 Anticipated in the Second Half of 2023 -
About this update from Dyne Therapeutics, Inc.
[{"type":"text","content":"- Initial Data from Global, Multiple Ascending Dose Phase 1/2 ACHIEVE Clinical Trial in Myotonic Dystrophy Type 1 Anticipated in the Second Half of 2023 - WALTHAM, Mass., May 25, 2023 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that the European Medicines Agency (EMA) has granted orphan drug designation for DYNE-101. DYNE-101 is being evaluated in the Phase 1/2 ACHIEVE global clinical trial in adults with myotonic dystrophy type 1 (DM1). “We are pleased to receive orphan drug designation from the EMA for DYNE-101, further supporting our efforts to develop a potentially transformative therapy for DM1,” said Wildon Farwell, M.D., MPH, chief medical officer of Dyne. “The DM1 community has waited far too long for a therapy that addresses the underlying cause of this devastating rare muscle disease. We are committed to advancing DYNE-101 as quickly as possible and anticipate our first clinical data readout from our ACHIEVE trial later this year.” The EMA grants orphan drug designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare, life-threatening or chronically debilitating diseases or conditions that affect fewer than five in 10,000 people in the European Union. Orphan designation allows companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and up to 10 years of market exclusivity in the European Union if approved. About DYNE-101 and the ACHIEVE Trial DYNE-101 is an investigational therapeutic being evaluated in the Phase 1/2 global ACHIEVE trial, for people living with myotonic dystrophy type 1 (DM1). The ACHIEVE clinical trial consists of a 24-week multiple ascending dose (MAD) randomized placebo-controlled period, a 24-week open-label extension and a 96-week long-term extension. The trial, which is designed to be registrational, is expected to enroll approximately 72 adult patients with DM1 who are 18 to 49 years of age. The primary endpoints are safety and tolerability with secondary endpoints of pharmacokinetics and pharmacodynamics, including change from baseline in splicing, as well as measures of muscle strength and function. Dyne anticipates reportin...