Here is STAT’s biotech scorecard, our regular ledger of stock-moving biotech events, for the third quarter:
The readout of Alnylam Pharmaceuticals’ APOLLO-B Phase 3 study is one of the biggest clinical trial catalysts of the year. The study is attempting to expand the use of Alnylam’s approved RNA-interference medicine Onpattro to encompass a significantly larger group of patients with transthyretin amyloidosis cardiomyopathy, or ATTR-CM, a progressive heart disease caused by the buildup of a toxic protein. APOLLO-B is also high risk, given the uncertainties raised last year when a similar study conducted by BridgeBio delivered negative results.
In November 2019, a novel combination pill from Karuna Therapeutics reduced the acute psychosis and related symptoms experienced by patients with schizophrenia in a small mid-stage clinical trial. This quarter, Karuna is attempting to replicate and confirm the efficacy of its drug called KarXT in a larger Phase 3 study called EMERGENT-2. A new treatment for schizophrenia with a meaningful benefit-risk profile could generate billions of dollars in sales.
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