Alnylam Pharma Crashes on Discontinued Late-Stage Trial

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Alnylam Pharmaceuticals Inc. (NASDAQ: ALNY) saw its shares crash on Thursday after the company announced that it will be discontinuing one of its late-stage trials. Specifically, the company announced that upon the recommendation of the Endeavour Phase 3 study Data Monitoring Committee (DMC) to suspend dosing, it has decided to discontinue development of revusiran.

Revusiran is an investigational RNA interference (RNAi) therapeutic that was being developed for the treatment of hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM).

Following recent reports in the Phase 2 OLE study of new onset or worsening peripheral neuropathy, the Endeavor DMC assembled Wednesday at the company’s request to review these reports and Endeavour data on an unblinded basis. Unfortunately, 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.

As a result, Alnylam reviewed unblinded Endeavor data which revealed an imbalance of mortality in the revusiran arm as compared to placebo.

Although this is a devastating blow, it’s important to note that this decision to discontinue development of revusiran does not affect patisiran, which is currently in Phase 3 development for the treatment of hATTR amyloidosis with polyneuropathy (hATTR-PN), or any other Alnylam investigational RNAi therapeutic program in development.

John Maraganore, Ph.D., CEO at Alnylam, commented:

Patient safety comes first. We have stopped all dosing and are actively monitoring patients across revusiran studies to ensure their safety. We will also continue to evaluate ENDEAVOUR data to understand the potential cause of these findings. While this outcome is disappointing given the lack of available treatment options for patients suffering from this devastating disease, we remain committed to serving the needs of the ATTR amyloidosis community. We would like to thank patients, caregivers, investigators, and study staff who have been so supportive of the revusiran program.

Shares of Alnylam Pharma were trading down 45% at $38.21 on Thursday, with a consensus analyst price target of $111.79 and a 52-week trading range of $35.31 to $110.79.