Press release
Alnylam Presents Positive Results from the APOLLO-B Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy
– Study Results Validate the Hypothesis That TTR Silencing by an RNAi Therapeutic has the Potential to be an Effective Approach for Treating Cardiomyopathy
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n– Study Results Validate the Hypothesis That TTR Silencing by an RNAi Therapeutic has the Potential to be an Effective Approach for Treating Cardiomyopathy of ATTR Amyloidosis –\n\n– Patisiran met Primary Endpoint, Demonstrating Significant Clinical Benefit on Functional Capacity (6-MWT) Compared to Placebo at Month 12 –\n\n– Patisiran Also met the First Secondary Endpoint, Demonstrating Significant Clinical Benefit on Health Status and Quality of Life, as Measured by the Kansas City Cardiomyopathy Questionnaire, Compared to Placebo at Month 12 –\n\n– Patisiran Demonstrated Encouraging Safety and Tolerability Profile in Patients with ATTR Amyloidosis with Cardiomyopathy –\n\n– Alnylam to Host Conference Call Today at 8:00 a.m. ET –\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced positive results from the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. The results were presented today in a late-breaker session at the 18th International Symposium on Amyloidosis (ISA). The Company previously announced positive topline results from the APOLLO-B study in August 2022.\n\nThe 12-month study achieved its primary endpoint, with patisiran demonstrating a statistically significant and clinically meaningful benefit on functional capacity, as measured by the 6-Minute Walk Test (6-MWT), compared to placebo, with a median difference of 14.7 meters (p-value 0.0162) favoring patisiran. The study also met its first secondary endpoint, demonstrating a statistically significant and clinically meaningful benefit on health status and quality of life, as measured by the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) score, compared to placebo with least squares (LS) mean difference of 3.7 points (p-value 0.0397) favoring patisiran. The study also included additional secondary composite outcomes endpoints. A non-significant result (p-value 0.0574) was found on the composite endpoint of all-cause mortality, frequency of cardiovascular events, and change from baseline in 6-MWT over 12 months compared to placebo. As a result, formal statistical testing was not performed on the final two composite endpoints. Patisiran al...