Wednesday, January 10, 2018

Alnylam Intensifies Its Focus On Rare Disease

Alnylam (ALNY) had an active 2017, with its first new drug application (or NDA) submitted to the FDA and multiple clinical read-outs, and 2018 is likely to be no less busy. This new year should see the company get its first FDA approval and begin its first commercial launch, in additional to more incremental data on several programs, including data that could potentially support an NDA filing for givosiran before year-end. Moreover, management has not been shy about leveraging good news to raise funds, leaving the company with close to $1.7 billion in cash to start the year.

Alnylam remains a risky biotech. While approval for its lead drug seems highly likely, the FDA could still surprise the company and there are still relevant questions about how the drug will fare in the real world. The company’s pipeline likewise still contains ample risk, though Alnylam has actively worked to identify biomarkers that can help point toward efficacy relatively early in development. With upside close to $140, there are still valid reasons to own Alnylam, but this is now a well-liked stock with a robust market cap that is many years away from generating the sort of earnings to support that optimism.

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Alnylam Intensifies Its Focus On Rare Disease

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