What Went Wrong With Alnylam's Revusiran Program?

Alnylam Pharmaceuticals, Inc. ALNY announced the discontinuation of the development of its late stage pipeline drug, Revusiran due to safety concerns.

Goldman Sachs’ Terence Flynn maintained a Neutral rating on the company, while lowering the price target from $65 to $34.

Safety Concerns

“Safety concerns were raised by reports of new or worsening peripheral neuropathy (PN, nerve damage) in a Ph2 extension trial,” Flynn mentioned.

Related Link: Alnylam Has Had Its Share Of Recent Pipeline Setbacks; Barclays Cuts PT To $50

These concerns led Alnylam to request a safety review of the Phase 3 trial by its DMC. While the DMC didn't observe and conclusive evidence of drug-related peripheral neuropathy, it recommended the suspension of dosing due to the skewed risk/benefit.

“ALNY subsequently reviewed the un-blinded Ph3 data and chose to discontinue the Revusiran program given an imbalance in deaths. At this point, ALNY does not have more details on potential drivers of PN,” the analyst stated.

Other Drugs Fine

Flynn pointed out that the company’s drugs are based on a unique platform that utilizes RNA interference (RNAi) to target diseases at the genetic level, and that Revusiran utilizes Alnylam’s first generation SQ formulation.

Although the company reported that it has not observed any safety concerns for its nine other programs in more than 800 treated patients, the analyst believes that the discontinuation of Revusiran could be “an incremental negative for ALNY’s platform” and could increase the overhand on the RNA therapy space.

Do you have ideas for articles/interviews you'd like to see more of on Benzinga? Please email feedback@benzinga.com with your best article ideas. One person will be randomly selected to win a $20 Amazon gift card!