Alnylam Discontinues Development for Key Drug; Lowering Our Fair Value Estimate
We are lowering our fair value estimate for Alnylam significantly, to $50 per share from $90, after the unexpected discontinuation of phase 3 revusiran development. Revusiran is an RNAi drug for the treatment of familial amyloid cardiomyopathy, a hereditary disease where abnormal protein deposits gather in the heart and lead to heart failure. Before the announcement, revusiran was a key revenue driver in our Alnylam model. A higher incidence of patient deaths in the treatment arm of the revusira...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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