In May 2024, several significant transactions worth millions and billions of dollars took place to advance next-generation neuroplastogens, KRAS G12C inhibitors, Pompe disease treatments, and molecular glues. We also highlight positive clinical readouts for hot targets such as tetrameric transthyretin (TTR) stabilizers, Hedgehog pathway inhibitors, plasma kallikrein inhibitors, dipeptidyl peptidase 1 (DPP-1) inhibitors, and PCSK9 inhibitors. Here's a recap of the most notable news highlights from May 2024!

Cerevance’s CVN293 is an oral, CNS-penetrant, selective inhibitor of potassium efflux-mediated NLRP3-inflammasome activation in microglia for the treatment of neurodegenerative disorders. Cerevance’s NETSseq platform was used to discover a microglia-specific potassium efflux channel, KCNK13, which allows modulation of the NLRP3-inflammasome in the CNS without affecting peripheral innate immunity. Read the full article to discover highlights on the CNS-penetration of highly polar compounds and how particle size can be key to oral bioavailability

To select April’s Molecules of the Month, our team evaluated hundreds of molecules from thousands of papers, press releases, conference presentations, and other materials. Here we’ve curated a roundup of more than 60 additional molecules that caught our interest from April, along with highlights we found most notable.

In April 2024, patent disclosures continued to highlight the swift progress in targeting key therapeutic areas. The Drug Hunter team has carefully compiled a searchable database containing over 200 patents relevant to drug discovery. This resource is enhanced with detailed annotations and highlights some of the most promising molecules and targets, such as GPR84 antagonists, brain-penetrant TYK2 inhibitors, and tricyclic urea JAK2 V617F inhibitors, among others. This compilation serves as a valuable tool for drug hunters to stay abreast of the latest trends in drug discovery.

In the aftermath of the thalidomide tragedy, the FDA advised excluding females in the childbearing age from early-stage clinical trials. However, this policy resulted in the prolonged underrepresentation of women in clinical research, limiting knowledge about how drugs affect genders differently. Recent policy changes, including the NIH's emphasis on considering sex as a biological variable, aim to reduce these disparities by promoting more diverse clinical trial participation. This article explores the current landscape of endometriosis therapies and how it relates to women’s healthcare.