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Alnylam Completes Enrollment of HELIOS-A Phase 3 Study of Vutrisiran for the Treatment of hATTR Amyloidosis with Polyneuropathy
– HELIOS-A Topline Results Expected in Early 2021 – CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n– HELIOS-A Topline Results Expected in Early 2021 –\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that it has achieved full enrollment in its HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis. The study was designed to enroll 160 patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy across 68 sites in 22 countries. Alnylam is on track to report topline results from HELIOS-A in early 2021.\n\n\n“We are pleased to have reached another important milestone for our TTR program by completing enrollment of the HELIOS-A study,” said Rena Denoncourt, Program Leader, Vutrisiran Program at Alnylam. “We are committed to developing multiple therapeutic options for this progressive, life-threatening and multisystem disease, and believe that vutrisiran, as a low-dose, once-quarterly, subcutaneously administered investigational therapeutic, can be an attractive option for patients. We look forward to sharing topline results in early 2021.”\n\n\nHELIOS-A is a Phase 3 global, randomized, open-label study to evaluate the efficacy and safety of vutrisiran in patients with hATTR amyloidosis with polyneuropathy. The trial randomized patients 3:1 to receive either 25mg of vutrisiran subcutaneously once every 12 weeks or 0.3 mg/kg of patisiran intravenously once every three weeks. For most endpoints, results from the vutrisiran arm will be compared to results from the placebo arm of the landmark APOLLO Phase 3 study, which evaluated the efficacy and safety of patisiran in people with hATTR amyloidosis with polyneuropathy. The co-primary endpoints of HELIOS-A are the change from baseline in the modified Neurologic Impairment Score +7 (mNIS+7) and in the Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) score, at 9 months. Secondary endpoints include the change from baseline in key clinical evaluations including the timed 10-meter walk test (10-MWT), modified body mass index (mBMI), and Rasch-built Overall Disability Scale (R-ODS). The percent reduction in serum transthyretin (TTR) levels in the vutrisiran arm will be compared to the within-study patisiran arm. Additional exploratory endpoints will be assessed to determine the effect of vutrisiran on other...