Benzinga | Jun 1, 2021 07:06AM EDT

Alnylam Completes Enrollment In APOLLO-B Phase 3 Study Of Patisiran In Transthyretin-Mediated Amyloidosis Patients With Cardiomyopathy

? APOLLO-B is the Industry's Furthest Progressed Randomized, Double-Blind, Placebo-Controlled Pivotal Study of an Investigational TTR Silencer in Hereditary and Wild-type ATTR Amyloidosis with Cardiomyopathy ?

? Topline Results Expected in Mid-2022 ?

Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that it has achieved full patient enrollment in its APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment of cardiomyopathy in patients with transthyretin-mediated (ATTR) amyloidosis. Enrollment was completed with over 300 ATTR amyloidosis patients across 90 sites in more than 20 countries. ATTR amyloidosis is a rare, progressively debilitating, and fatal disease that is caused by misfolded transthyretin (TTR) proteins that accumulate as amyloid deposits in multiple tissues, including the nerves, heart and gastrointestinal (GI) tract and encompasses hereditary ATTR (hATTR) amyloidosis and wild-type ATTR (wtATTR) amyloidosis. hATTR amyloidosis is estimated to impact approximately 50,000 people worldwide and is a multisystem disease that can include sensory and motor, autonomic, and cardiac symptoms. wtATTR amyloidosis is estimated to impact between 200,000 and 300,000 people worldwide and primarily manifests as cardiomyopathy, which can lead to heart failure and mortality. APOLLO-B is the industry's furthest progressed randomized, double-blind, placebo-controlled study of a TTR silencer investigational medicine for the treatment of cardiomyopathy in hATTR and wtATTR amyloidosis patients.

Patisiran is the established name for ONPATTRO(r), which is approved in the United States, Canada and Japan for the treatment of the polyneuropathy of hATTR amyloidosis in adults and in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy.

"The APOLLO-B trial was initiated based on encouraging data from previous studies that support further investigation of patisiran in ATTR amyloidosis patients with cardiomyopathy. Specifically, encouraging results were obtained from the landmark Phase 3 APOLLO study in hATTR amyloidosis patients with polyneuropathy on a number of exploratory cardiac endpoints. Today's milestone marks an important step forward as we continue to study the safety and efficacy of patisiran in the treatment of cardiovascular-related manifestations of ATTR amyloidosis, which can often be devastating for these patients," said Rena Denoncourt, Vice President, TTR Franchise Lead. "We look forward to announcing topline APOLLO-B results in mid-2022 and are excited about the potential of patisiran to treat multiple manifestations of this rapidly progressive disease and further advance our aspiration to build the industry leading franchise of RNAi therapeutics for the treatment of ATTR amyloidosis."