Alnylam Pharmaceuticals Sheds Some Light On Its Expanding Early-Stage Opportunities

Although I hadn’t planned writing two articles on Alnylam Pharmaceuticals (ALNY) in close succession, the early approval of Givlaari and the subsequent R&D meeting both argued for a write-up, as both are significant to the long-term investment story.

Alnylam’s Nov 22 R&D Day was perhaps more evolutionary than revolutionary, but it confirmed what I believe are some key points to the story – management is attentive to the need to become a profitable enterprise, the company’s ongoing investments in basic chemistry continue to pay dividends, and the company has established a strong R&D platform that could produce multiple new drug candidates every year.

With expanded information on a handful of early-stage programs, I’ve raised my fair value estimate by a small amount, but the key drivers remain the commercial uptake of Onpattro, the success of trials that could enable Onpattro and vitusiran to compete in the larger AATR cardiomyopathy and mixed phenotype markets, the launch of Givlaari, and path to commercialization for lumasiran, inclisiran, and fitusiran.

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Alnylam Pharmaceuticals Sheds Some Light On Its Expanding Early-Stage Opportunities