Alnylam Pharmaceuticals, Inc. discontinues development of revusiran for the treatment of hereditary ATTR amyloidosis with cardiomyopathy.

Alnylam Pharmaceuticals, Inc. announced that upon the recommendation of the ENDEAVOUR Phase III study Data Monitoring Committee (DMC) to suspend dosing, the Company has decided to discontinue development of revusiran, an investigational RNA interference (RNAi) therapeutic that was being developed for the treatment of hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM). This decision was made yesterday evening and has been communicated to investigators, study sites, and regulatory authorities.

Following recent reports in the Phase II OLE study of new onset or worsening peripheral neuropathy, the ENDEAVOUR DMC assembled at the Company's request to review these reports and ENDEAVOUR data on an unblinded basis. The DMC did not find conclusive evidence for a drug-related neuropathy signal in the ENDEAVOUR trial, but informed the Company that the benefit-risk profile for revusiran no longer supported continued dosing. The Company subsequently reviewed unblinded ENDEAVOUR data which revealed an imbalance of mortality in the revusiran arm as compared to placebo.

The decision to discontinue development of revusiran does not affect patisiran, which is currently in Phase III development for the treatment of hATTR amyloidosis with polyneuropathy (hATTR-PN), or any other Alnylam investigational RNAi therapeutic program in development.