Alnylam Pharmaceuticals Press Release | Nov 17, 2024

Alnylam's stock is down 30% in 2026 despite Amvuttra's success. Wainua's Phase 3 failure raised doubts about RNAi silencers like Amvuttra and nucresiran. Q2 earnings due July 30.

Alnylam Pharmaceuticals highlighted significant progress in its R&D pipeline at its R&D Day event. Key updates include:Phase 3 TRITON program for nucresiran in ATTR-CM and hATTR-PN, aiming for over 95% knockdown with twice-annual dosing.Phase 3 trial for zilebesiran to transform hypertension treatment, enrolling up to 11,000 patients globally.New programs for Huntington’s disease, bleeding disorders, and type 2 diabetes.Platform advances for improved delivery solutions and manufacturing innovations.Data from the HELIOS-B study showed vutrisiran reduced all-cause mortality by 36% in patients with ATTR amyloidosis with cardiomyopathy. Vutrisiran is under FDA review with a target action date of March 23, 2025.Alnylam is committed to expanding its RNAi therapeutics, targeting all major tissue types by 2030. The company will file IND applications for nine new programs by 2025, including two in new tissues, two in the CNS, and five in the liver.The event was webcast live and a replay will be available on Alnylam's website.