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August 10, 2021

Alnylam closes enrolment in vutrisiran’s Phase III cardiomyopathy trial

The company expects to report top-line full data from the HELIOS-B trial, which includes an optional interim analysis, in 2024.

Alnylam Pharmaceuticals has completed participant enrolment for the Phase III HELIOS-B clinical trial of vutrisiran to treat cardiomyopathy in transthyretin-mediated (ATTR) amyloidosis patients.

A total of more than 600 patients were enrolled at 123 activated sites across 32 countries. The enrolment was closed before the scheduled timeframe, Alnylam noted.

Meant for subcutaneous administration, vutrisiran is an experimental, RNAi therapeutic that acts on and silences specific messenger RNA in order to hinder the generation of wild-type and variant transthyretin protein even before it is made.

The randomised, double-blind, placebo-controlled, global Phase III trial is designed to assess the efficacy and safety of 25mg vutrisiran compared with placebo when given once every three months for up to 36 months.

Efficacy of the drug on the composite endpoint of all-cause mortality and recurrent cardiovascular events at 30 months is the primary goal of the study.

Alnylam Pharmaceuticals TTR Franchise Lead vice-president Rena Denoncourt said: “We reached full study enrolment for HELIOS-B, our largest clinical trial to date, approximately 20 months from study initiation, reflecting the high interest from patients and physicians in a potential treatment option with subcutaneous administration, quarterly dosing, and potent and reversible serum TTR reduction.

“If positive, the HELIOS-B study would support our efforts to advance the development of an industry-leading franchise of RNAi therapeutics for the treatment of ATTR amyloidosis.”

Top-line full data from the HELIOS-B trial are set to be available in 2024.

The protocol of the trial covers an optional interim analysis. If pursued, the analysis will be performed after the data readout from the company’s Phase III APOLLO-B trial of patisiran for ATTR amyloidosis with cardiomyopathy.

With enrolment concluding in June 2020, APOLLO-B is anticipated to report top-line data mid-next year.

The future path for an interim analysis of HELIOS-B will be refined, including a potential earlier top-line data readout, based on the APOLLO-B results and following regulatory interactions.

In February 2020, Alnylam completed enrolment in the Phase III HELIOS-A trial of vutrisiran for the treatment of hereditary transthyretin (hATTR) amyloidosis.

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