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Alnylam : Phase 3 Study Of Patisiran In ATTR Amyloidosis With Cardiomyopathy Meets Main Goal

Alnylam Pharmaceuticals Inc. (ALNY) said that the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated or ATTR amyloidosis with cardiomyopathy, met the primary endpoint of change from baseline in the 6-Minute Walk Test (6-MWT) at 12 months compared to placebo.

The study also met the first secondary endpoint of change from baseline in quality of life compared to placebo, as measured by the Kansas City Cardiomyopathy Questionnaire.

Patisiran also demonstrated an encouraging safety and tolerability profile, with deaths numerically favoring the patisiran arm.

The company looks forward to sharing full results at an upcoming conference in September, and it plans to submit a supplemental NDA for patisiran with the U.S. Food and Drug Administration in late 2022.

Transthyretin-mediated amyloidosis is a rare, rapidly progressive, debilitating disease caused by misfolded transthyretin (TTR) proteins which accumulate as amyloid fibrils in multiple tissues including the nerves, heart, and gastrointestinal (GI) tract.

There are two different types of ATTR amyloidosis - Hereditary ATTR (hATTR) amyloidosis, caused by a TTR gene variant, and Wild-type ATTR amyloidosis, which occurs without a TTR gene variant.

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