Molecule Spotlight: Risdiplam, the First Approved Small Molecule Splicing Modulator for Spinal Muscular Atrophy

In this update of small molecules in the news for February 2023, we will cover recent: - Molecules in Motion : clinical trial updates and FDA filings - Negative Readouts : halted, discontinued or altered clinical trials - Million-Dollar Molecules : recent M&A’s, IPOs, and other fundraising - Potential Approvals : recent NDA filings and [...]

This article compiles 20+ recent small molecules of general interest in the news in April 2023, with structures where they are available. Capacity Bio’s MAS Receptor Agonists with $35M Raised for FIH Clinical Trials MAS modulator from WO2022165189 example 107 New start-up Capacity Bio backed by RA Capital, Insight Partners, and Remiges [...]

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.

In 2021 there were: 50 novel drugs approved by the FDA 27 that CDER considers first-in-class (FIC) 16 first-in-class small molecules (according to CDER) These drugs are the first in their therapeutic class to have a particular mechanism of action [...]

Billion-Dollar Molecules: Etavopivat, an Allosteric PKR Kinase Activator for Sickle Cell Disease Abstract. Novo Nordisk has acquired Forma Therapeutics for $1.1B for its primary investigational drug, etavopivat. Etavopivat, a past Molecule of the Month , is an oral, once-daily allosteric activator of pyruvate kinase-R (PKR), which is [...]