Vonoprazan, Tapinarof, and Tirzepatide: FDA Novel Drug Approvals in May 2022

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 [...]

This October roundup features key clinical updates, including FDA approvals, significant trial results for major drug candidates, the initiation of new clinical trials, and updates on halted studies.

This summary of major clinical updates from November 2024 covers FDA approvals, the initiation of new clinical trials, notable trial outcomes for key drug candidates, and news on paused and discontinued trials and programs.

Relyvrio, developed by Amylyx Pharmaceuticals, is a combination treatment of sodium phenylbutyrate and taurursodiol, approved by the FDA in 2022 for ALS (“Lou Gehrig’s disease”). The approval was controversial, with an Advisory Committee initially voting against approval based on Ph. II data. Now, Ph. III data have confirmed the drug is no better than placebo in ALS, turning this phenotypic drug discovery story from two undergrads into a post-mortem highlighting the importance of clearly defined molecular mechanisms of action. This article reviews what happened and what we can learn.

There continue to be opportunities for another KRAS(G12C) inhibitor to prove itself best-in-class.

This roundup highlights twelve KRAS(G12C) molecules to know, with links to recent case studies on Drug Hunter going in more detail.