Alnylam Pharmaceuticals Heading Toward A Key Trial Read-Out

Any day now Alnylam Pharmaceuticals (NASDAQ:ALNY) investors will get a key piece of information – the efficacy of its key drug Onpattro in the treatment of hereditary ATTR amyloidosis (or hATTR) characterized by cardiomyopathy (or hATTR-CM). Success in the APOLLO-B study could more than quadruple sales of this drug, not to mention presage successful outcomes for the HELIOS-B study of follow-on compound Amvuttra (vutrisiran), while failure would represent a significant risk to the share price and future value of the ATTR franchise.

The data that management have presented over the years suggest that Onpattro should hit the mark in APOLLO-B, but there’s a reason that the FDA and clinicians insist on clinical trials and prior signals of efficacy and post-hoc analyses aren’t substitutes for a positive statistically-significant outcome. I believe that Alnylam shares are meaningfully undervalued below $200/share, but investors buying in ahead of the APOLLO-B data must appreciate the risk of a sharp correction if the study results don’t live up to expectations.

 

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Alnylam Pharmaceuticals Heading Toward A Key Trial Read-Out