Morningstar | ALNY Updated Forecasts and Estimates from 17 Jun 2019
Alnylam Pharmaceuticals is a pioneer in designing therapeutics based on RNA interference, a mechanism that occurs naturally in cells and silences overexpressed genes, which often cause protein misfolding and/or protein accumulation. Alnylam seeks to capitalize on the therapeutic potential of RNAi by creating small interfering RNA that can treat disease at the genetic level. If siRNA reaches its intended target, it can silence the gene that it contains code for, making it a potentially game-changing technology for tough-to-treat diseases caused by genetic mutations.After over a decade of research, Onpattro, the first RNAi therapy, was approved in 2018 for polyneuropathy in hereditary ATTR amyloidosis, positioning Alnylam as a leader among RNA-based biotechs. With the potential for label expansion into cardiomyopathy and wild-type ATTR amyloidosis, we believe the company's largest opportunity now is in ATTR amyloidosis, with Onpattro and next-generation vutrisiran. With several late-stage candidates in its abundant pipeline, we believe Alnylam is one of only a few biotechnology companies with a systematic methodology for identifying targets and developing drug candidates. Aside from Onpattro, its only approved drug, Alnylam has several candidates in the pipeline, including givosiran for acute hepatic porphyria (AHP), lumasiran for primary hyperoxaluria Type 1 (PH1), inclisiran for hypercholesterolemia (partnered with The Medicines Company), and fitusiran for hemophilia. Alnylam competes with a few other biotechs that focus on RNA-based therapies, including Ionis, which received approval in polyneuropathy in hATTR amyloidosis for Tegsedi about two months after Alnylam. Onpattro's safety profile is more attractive than that of Tegsedi, but we expect both companies to produce next-generation candidates that could look more competitive.Alnylam Pharmaceuticals is a commercial-stage biopharmaceutical company developing therapeutics based on RNA interference (RNAi). RNAi is a biological pathway within cells for sequence-specific silencing and regulation of gene expression. The company has two RNAi-based medicines, ONPATTRO? (patisiran) and GIVLAARI? (givosiran). ONPATTRO is approved by the United States Food and Drug Administration (FDA) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults and has also been approved in the European Union, Japan, Canada and Switzerland. GIVLAARI is approved by FDA for the treatment of adults with acute hepatic porphyria.
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