Alnylam Pharmaceuticals, Inc. (ALNY) said Thursday that the HELIOS-A Phase 3 study of vutrisiran, met its primary and both secondary endpoints at nine months in patients with hATTR amyloidosis with polyneuropathy.
Vutrisiran is an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis.
Alnylam said that based on these positive results, it plans to submit a New Drug Application or NDA for vutrisiran with the U.S. Food and Drug Administration (FDA) in early 2021, and to follow with regulatory filings in additional countries, such as Brazil and Japan.
The primary endpoint of the HELIOS-A Phase 3 trial was the change from baseline in the modified Neuropathy Impairment Score (mNIS+7) at 9 months as compared to historical placebo data from the APOLLO Phase 3 study of patisiran.
The two secondary endpoints were changes in quality of life assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and gait speed assessed by the timed 10-meter walk test (10-MWT) compared to historical placebo.
Vutrisiran met the primary endpoint and achieved statistically significant results for each of the Norfolk QoL-DN and 10-MWT secondary endpoints, Alnylam said.
vutrisiran treatment also showed improvement compared to placebo on the exploratory cardiac biomarker endpoint, NT-proBNP as well as demonstrated an encouraging safety and tolerability profile.
Alnylam said it plans to submit a Marketing Authorisation Application or MAA in the EU upon obtaining the results of the 18-month analysis, which is expected in late 2021.
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