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Dyne Therapeutics 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."

Alnylam Pharmaceuticals announced new results from an interim analysis of exploratory data from the open-label extension period of the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated amyloidosis with cardiomyopathy. The results were presented at the Annual Congress of the Heart Failure Association of the European Society of Cardiology. The company previously announced that APOLLO-B achieved its primary endpoint and met its first secondary endpoint during the 12-month double-blind period and that it has submitted the 18-month data to the U.S. Food and Drug Administration as an amendment to the supplemental New Drug Application for patisiran for the treatment of the cardiomyopathy of ATTR amyloidosis. The 18-month findings indicate that the favorable effects on functional capacity and health status and quality of life, as measured by the 6-Minute Walk Test and the Kansas City Cardiomyopathy Questionnaire Overall Summary, respectively, observed during the DB period were sustained with continued patisiran treatment during the OLE period. Patients treated with patisiran through 18 months also appear to have maintained relative stability of NT-proBNP and Troponin I levels, measures of cardiac stress and injury, respectively. Patients who crossed over from placebo in the DB period to patisiran during the OLE period appear to show slowing of disease progression or relative stabilization across these same endpoints at Month 18. While the APOLLO-B study was not designed to show benefits in cardiac outcomes between patisiran and placebo, evidence of favorable, but non-statistically significant, trends were observed for composite all-cause death and hospitalization, and mortality analyses across the DB and OLE periods.