ALNY - Alnylam posts detailed Phase 3 data for ATTR amyloidosis therapy

RNAi therapeutics company Alnylam Pharmaceuticals ( NASDAQ: ALNY ) announced full results from its APOLLO-B Phase 3 study of patisiran, an investigational medication for transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.

The 12-month trial involving 360 adult patients reached its primary endpoint with a statistically significant and clinically meaningful benefit on functional capacity.

That metric favored patisiran indicating a median difference of 14.7 meters as measured by the 6-Minute Walk Test (6-MWT) compared to placebo.

The study also met its secondary endpoint, with a statistically significant and clinically meaningful benefit on health status and quality of life compared to the placebo.

Measured by the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) score, that indication favored patisiran with least squares (LS) mean difference of 3.7 points compared to placebo.

However, the company said there was a non-significant result for the composite endpoint of all-cause mortality, frequency of cardiovascular events, and change from baseline in 6-MWT over 12 months compared to placebo.

Regarding safety, 5% or more patients in the patisiran arm were found to develop treatment-emergent AEs. There were five (2.8%) and eight deaths (4.5%) in the patisiran and placebo groups, respectively.

Patisiran, known as Onpattro, is already indicated in the U.S. for adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis.

In the U.S., ALNY expects to seek a label expansion for patisiran as a treatment for ATTR amyloidosis with cardiomyopathy in late 2022.

In early August, the company shared topline data from the trial.

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