First-in-Class Small Molecule Drug Approvals of 2022
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Leniolisib: The First PI3K Inhibitor First Approved Outside of Oncology
Leniolisib is a selective PI3Kδ inhibitor that received approval in March 2023 from the FDA as a first-in-class treatment for activated PI3K-δ syndrome (APDS), a genetic immunodeficiency disorder caused by PI3Kδ activation. The leniolisib discovery program is notable for several reasons: it is a first approval for a PI3K inhibitor outside of oncology; the first approved drug for APDS; has a novel chemotype that appears to avoid safety issues that led to black box warnings seen with other PI3K molecules; and it overcame a polymorph issue with an early lead that led to inconsistent exposures.
Patent Highlights: Allosteric AR Modulators, Glycogen Synthase Inhibitors, and More
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KIN-3248: Overcoming FGFR Resistance with a Next-Generation Pan-FGFR Inhibitor
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How GLP-1 Receptor Agonists Evolved from Diabetes Treatments to Weight Loss Treatments
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Billion-Dollar Molecules: Etavopivat, an Allosteric PKR Kinase Activator for Sickle Cell Disease
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