Press release
Alnylam Reports Positive Topline 18-Month Results from HELIOS-A Phase 3 Study of Vutrisiran in Patients with hATTR Amyloidosis with Polyneuropathy
– Vutrisiran Met All 18-Month Secondary Endpoints, Including Statistically Significant Improvements in Progression of Neuropathy, Quality of Life (QOL), Gait
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n– Vutrisiran Met All 18-Month Secondary Endpoints, Including Statistically Significant Improvements in Progression of Neuropathy, Quality of Life (QOL), Gait Speed, Nutritional Status and Overall Disability, Relative to External Placebo –\n\n– At Month 18, Vutrisiran Also Showed Improvements in Exploratory Endpoints, Including Technetium Uptake Relative to Baseline in a Planned Cohort, Providing Potential Evidence for Reduced Cardiac Amyloid Burden –\n\n– In Addition, Vutrisiran Continued to Demonstrate an Encouraging Safety and Tolerability Profile –\n\n– Alnylam Intends to Present Full HELIOS-A 18-Month Results at a Medical Congress in Early 2022 –\n\n– Alnylam to Discuss Data as Part of Third Quarter 2021 Financial Results Conference Call to be Held Tomorrow, October 28 at 8:30 am ET –\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of the polyneuropathy associated with hereditary transthyretin-mediated (hATTR) amyloidosis, met all secondary endpoints measured at 18 months, including statistically significant improvements in neuropathy as measured by the modified Neuropathy Impairment Score (mNIS+7), quality of life (QOL), gait speed, nutritional status and overall disability, relative to external placebo data from the APOLLO Phase 3 study of patisiran. The final secondary endpoint, reduction in serum TTR levels with vutrisiran, demonstrated non-inferiority relative to the within-study patisiran arm, as expected. In addition, patients treated with vutrisiran showed improvements in exploratory endpoints, including the biomarker NT-proBNP and certain echocardiographic parameters, relative to placebo, and an improvement in technetium uptake relative to baseline in a majority of patients in a planned cohort, providing potential evidence for reduced cardiac amyloid burden. Vutrisiran continued to demonstrate an encouraging safety and tolerability profile. Alnylam previously announced that HELIOS-A met its primary and secondary endpoints at nine months and study results were presented at the 2021 American Academy of Neurology (AAN) Virtual Annual Meeting.\n\n“These results build on the positive vutrisiran data shared earlie...