First-in-Class Small Molecule Drug Approvals of 2022
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2023 Novel Large Molecule FDA Drug Approvals
Recently, we summarized the novel small molecule drug approvals of 2023 (34/55). The remaining 21 molecular entities approved were large molecules like proteins and oligonucleotides, accounting for nearly 38% of the total approvals. Notably, seven of these large molecules have earned the prestigious Breakthrough Therapy designation, relative to the two small molecules that share this recognition. A summary of approvals for large molecule drugs, accompanied by links to FDA labels and additional information pertaining to the drug's discovery and originators, appears here.
KIN-3248: Overcoming FGFR Resistance with a Next-Generation Pan-FGFR Inhibitor
KIN-3248 (Kinnate Biopharma) is a next-generation, irreversible, pan-fibroblast growth factor receptor inhibitor (FGFRi) in a first-in-human (FIH) dose-escalation study (NCT05242822) in adults with advanced tumors harboring FGFR2 and FGFR3 gene alterations. The program is an excellent case study for rational covalent drug design to address kinase mutations while maintaining sufficient PK properties for oral daily dosing in humans.
KRAS Roundup: Twelve G12C Clinical Molecules to Know
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.
How GLP-1 Receptor Agonists Evolved from Diabetes Treatments to Weight Loss Treatments
Peptidic GLP-1R agonists have received significant media coverage over the past year for their astounding efficacy in several indications. GLP-1R agonists now appear to be efficacious in reducing heart disease risk and in treating certain chronic kidney diseases, expanding their likely future use. This review by Oliver Philps provides an overview of GLP-1 agonists and where they fit in the treatment of diabetes, how the peptide drugs evolved over time to today’s weight loss medications, and what to expect next...
A Bear Bile Acid Goes Bust: Taurursodiol + Phenylbutyrate
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.
