The data presented at ISA highlighted that treatment with patisiran was associated with significant improvements in measures of cardiomyopathy, the leading cause of death in patients with hATTR amyloidosis, relative to placebo.
Patisiran was also associated with a favorable effect on gait speed in the cardiac subpopulation, relative to placebo. Furthermore, patisiran treatment led to a significant reduction in levels of a cardiac stress biomarker, NT-proBNP, relative to placebo at 9 and 18 months.
Twenty-five patients, originally in the Phase 2 study, have now received patisiran for up to four years. These patients show sustained mean improvements in neuropathy as assessed by the modified Neurologic Impairment Score (mNIS+7) at month 36, relative to baseline.
The APOLLO Phase 3 trial was a randomized, double-blind, placebo-controlled, global study designed to evaluate the efficacy and safety of patisiran in hATTR amyloidosis patients with polyneuropathy. The trial enrolled 225 hATTR amyloidosis patients from 19 countries with 39 genotypes who were randomized 2: 1, patisiran: placebo, with patisiran administered at 0.3 mg/kg once every three weeks for 18 months.
Patisiran is designed to silence specific messenger RNA, potentially blocking the production of TTR protein, which may help to enable the clearance of TTR amyloid deposits in peripheral tissues and potentially restore function to these tissues. The safety and efficacy of patisiran have not been evaluated by the US Food and Drug Administration or any other health authority.
Alnylam Pharmaceuticals is a biopharmaceutical company that discovers, develops, and commercializes novel therapeutics based on RNA interference.
Its pipeline of investigational RNAi therapeutics is focused on genetic medicines, cardio-metabolic diseases, and hepatic infectious diseases.
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