Business
Alnylam Reports Positive Topline Results from HELIOS-A Phase 3 Study of Vutrisiran in Patients with hATTR Amyloidosis with Polyneuropathy
– Vutrisiran Met Primary and All Secondary Endpoints at 9 Months, with Statistically Significant Improvements in Progression of Neuropathy, Quality of Life
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n– Vutrisiran Met Primary and All Secondary Endpoints at 9 Months, with Statistically Significant Improvements in Progression of Neuropathy, Quality of Life (QOL), and Gait Speed, Relative to Placebo –\n\n– Majority of Patients Showed Reversal of Disease Manifestations with Improvements in Neuropathy Impairment and QOL, Relative to Baseline –\n\n– Vutrisiran Showed Improvements in the 9-Month Exploratory Cardiac Endpoint of NT-proBNP, Relative to Placebo –\n\n– In Addition, Vutrisiran Demonstrated Encouraging Safety and Tolerability Profile –\n\n– Alnylam Intends to Present Full 9-Month Results and File New Drug Application (NDA) in Early 2021 –\n\n– Alnylam to Host Conference Call Today at 8:00 am ET –\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, met its primary and both secondary endpoints at nine months in patients with hATTR amyloidosis with polyneuropathy. The primary endpoint was the change from baseline in the modified Neuropathy Impairment Score (mNIS+7) at 9 months as compared to historical placebo data from the APOLLO Phase 3 study of patisiran. The two secondary endpoints were changes in quality of life assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and gait speed assessed by the timed 10-meter walk test (10-MWT) compared to historical placebo. Vutrisiran met the primary endpoint (p less than 0.001) and achieved statistically significant results (p less than 0.001) for each of the Norfolk QoL-DN and 10-MWT secondary endpoints. In addition, vutrisiran treatment showed improvement compared to placebo on the exploratory cardiac biomarker endpoint, NT-proBNP (nominal p less than 0.05). Vutrisiran also demonstrated an encouraging safety and tolerability profile.\n\nBased on these positive results, the Company plans to submit a New Drug Application (NDA) for vutrisiran with the U.S. Food and Drug Administration (FDA) in early 2021, and to follow with regulatory filings in additional countries, such as Brazil and Japan. The Company plans to submit a Marketing Authorisation Application (MAA) in the EU upon obtaining the results...