Revusiran Termination A Setback For Alnylam, But Platform-Wide Problems Unlikely

Following, Alnylam Pharmaceuticals, Inc.ALNY announcing the termination of a late-stage study of Revusiran, citing mortality imbalance, Jefferies said it strongly believes the issue might be program- or disease-specific rather than being overall technology platform-related.

Gena Wang, an analyst at Jefferies noted that the development of Revusiran was discontinued due to newly reported peripheral neuropathy in a Phase 2 OLE and mortality imbalance in the ENDEAVOR Phase III trial in FAC. Revusirn is an investigational RNAi Therapy for treating transthyretin mediated amyloidosis, which is a slowly progressive condition characterized by the build-up of abnormal deposits of a protein called amyloid.

Related Link: What Went Wrong With Alnylam's Revusiran Program?

Although the analyst sees limited read-through to the Patisiran in FAP, she believes the risk to the TTRsc02 program is high. The increased risk to the TTRsc02 is premised on a trio of factors, including having the same siRNA sequence as revusiran, a lack of drug activity for revusiran and the analyst's doubts over the validity of having 6MWT as primary endpoints.

However, Jefferies believes the toxicity could be program/disease specific, as all other clinical programs, except Patisiran, uses 2nd generation GaINAC. Additionally, the rapid progression of FAC and the inconclusive relationship between drug and neuropathy/mortality were also offered as reasons for the firm's belief.

Jefferies lowered its price target on Alnylam shares to $58 from $60 but maintains its Buy rating.

At last check, shares of Alnylam were down a whopping 47.25 percent at $37.08.

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