Following the news on April 8, 2019 that Alnylam and Regeneron have entered into an $800m pact to develop RNA interference (RNAi) therapies for eye and CNS diseases,

Paul Jeng, Senior Pharma Analyst at GlobalData, a leading data and analytics company, offers his view on the growing opportunities for RNAi:

“RNAi therapies are still relatively new to the commercial landscape, having only recently overcome years of clinical barriers including off-target effects, delivery challenges, and undesired immune activation.

“The partnership between Alnylam and Regeneron signals that momentum for RNAi will continue in force on the heels of the landmark FDA approval of Alnylam’s Onpattro in 2018 for treatment of hATTR amyloidosis. In the coming months, Alnylam is also expected to license its experimental product givosirin for treatment of acute hepatic porphyria (AHP), following positive results from the Phase III ENVISION study.

“However, the promise of RNAi has always extended beyond the scope of rare genetic disorders to include other indications like cardio-metabolic and nervous system diseases. Alnylam’s new partnership is an opportunity to leverage Regeneron’s extensive experience in genetic research to explore innovative applications of RNAi therapies in eye and CNS programs. This pact will be the start of a particularly significant new phase for Alnylam, given the recent conclusion if its R&D partnership with Sanofi.

“One of the other intriguing prospects for RNAi is its applications in infectious diseases. In its early stage pipeline, Alnylam is developing a gene silencing therapy – ALN-HBV02 – targeting the hepatitis B virus (HBV) genome. Chronic disease arising from acute HBV infection is a lifelong affliction for most patients, and complete eradication of HBV would be an achievement for RNAi that is unlikely to be matched by antivirals. However, Alnylam currently trails Arrowhead Pharmaceuticals in this indication, which reported positive interim Phase I/II data for its product ARO-HBV in 2018.”