26 April 2018 - - Cambridge, Massachusetts-based RNA interference therapeutics developer Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) has released 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.
A new post-hoc, exploratory recurrent event analysis revealed an approximately 50% decrease in the composite rate of all-cause hospitalisation and mortality over 18 months in patisiran-treated patients, relative to placebo, based upon hospitalizations and deaths designated as serious adverse events within 28 days after last dose of study drug.
A similar finding was observed with the composite rate of cardiac hospitalization and all-cause mortality, showing an approximately 45% decrease with patisiran, relative to placebo.
Patisiran also demonstrated halting or improvement in the modified Neurologic Impairment Score+7 (mNIS+7) primary endpoint in patients regardless of baseline neuropathy severity, in contrast to the progression in mNIS+7 seen in placebo-treated patients.
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 primary endpoint of the study was the change from baseline in mNIS+7 relative to placebo at 18 months. The trial enrolled 225 hATTR amyloidosis patients from 19 countries with 39 genotypes.
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.