HELIOS-A phase III study of ALN TTRsc02 meets primary and secondary endpoints in ATTR (hATTR) amyloidosis with polyneuropathy.- Alnylam Pharma

Alnylam Pharmaceuticals announced full positive results from the HELIOS-A Phase III study of ALN TTRsc02 (vutrisiran), an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, which met its primary and both secondary endpoints at nine months in patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy. The 9-month results achieved the study’s primary endpoint, with vutrisiran showing improvement in the mean change from baseline in the modified Neuropathy Impairment Score (mNIS+7) as compared to external placebo data from the APOLLO Phase III study of patisiran. At 9 months vutrisiran also met all secondary endpoints, demonstrating improvement in quality of life as assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) instrument and improvement in gait speed as assessed by the timed 10-meter walk test (10-MWT), both compared to the external placebo group. The majority of patients experienced improvement in neuropathy and in quality of life, both relative to baseline, showing the potential for vutrisiran to reverse polyneuropathy manifestations of hATTR amyloidosis. Vutrisiran also demonstrated an encouraging safety profile with no drug-related discontinuations or deaths. Based on these positive data, Alnylam has submitted a New Drug Application (NDA) with the FDA for the approval of vutrisiran for the treatment of the polyneuropathy of hATTR amyloidosis in adults. The results were presented in an oral session at the 2021 American Academy of Neurology (AAN) Virtual Annual Meeting.