Alnylam: Patisiran Shows Improvement Of Cardiomyopathy In APOLLO Phase 3 Study

(RTTNews) - Alnylam Pharmaceuticals, Inc. (ALNY) reported new results from the APOLLO Phase 3 study of patisiran, an investigational, RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis. The company said the data 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.

Alnylam Pharma also reported new data from the Phase 1 study of ALN-TTRsc02, an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis. The company said single doses of ALN-TTRsc02 demonstrated robust TTR knockdown (mean maximum up to 97 percent) maintained over 320 days.