Alnylam Presents Positive Results from HELIOS-A Phase 3 Study of Investigational Vutrisiran
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced full positive results from the HELIOS-A Phase 3 study of 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 results were presented today in an oral session at the 2021 American Academy of Neurology (AAN) Virtual Annual Meeting.
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 3 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 U.S. Food and Drug Administration (FDA) for the approval of vutrisiran for the treatment of the polyneuropathy of hATTR amyloidosis in adults.
“The HELIOS-A study results demonstrate that vutrisiran improves neuropathy, quality of life and gait speed as soon as 9 months – compared to the external placebo arm of APOLLO – in patients with hATTR amyloidosis with polyneuropathy, with an encouraging safety and tolerability profile. The reversal of polyneuropathy manifestations of disease demonstrated in patients treated with vutrisiran in the study, the encouraging safety profile, and the totality of data from the trial, reinforce our belief in the promise of our RNAi therapeutics’ mechanism of action, as first established with ONPATTRO (patisiran). Further, we are encouraged by the exploratory endpoint results showing the impact of vutrisiran on NT-proBNP, a marker of cardiac stress, and look forward to additional data on exploratory cardiac endpoints at the 18-month readout, expected in late 2021,” said Akshay Vaishnaw, M.D., Ph.D., President of R&D at Alnylam. “With our NDA filing now completed, we look forward to potentially bringing vutrisiran to patients as a new treatment option for the polyneuropathy of hATTR amyloidosis with subcutaneous administration and quarterly dosing. The positive HELIOS-A study and our NDA filing are key milestones as we continue our progress towards building an industry-leading franchise of medicines for the treatment of ATTR amyloidosis and towards our goal of expanding in the future the population of patients who may benefit from treatment with an RNAi therapeutic.”
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