Infection with the malaria parasite, Plasmodium falciparum, is a leading cause of fatality in the tropical regions of the world, with over 240M infections and >600k deaths each year. Currently, over half of the world population is at risk of infection and with the rise of resistance against current treatments, the need for new antimalarials is clear. At the ACS Fall 2024 conference in Denver, CO, Novartis outlined the structure and discovery story of NVP-IWY357, a novel antimalarial that has no cross-resistance to current drugs and the potential to achieve a single-dose cure.

AstraZeneca recently disclosed the structure and discovery journey of their oral small molecule PCSK9 inhibitor, AZD0780, during the ACS Fall 2024 First Time Disclosures session in Denver. Discover how the team identified a fragment hit targeting a previously unexplored pocket, verified its novel mechanism of action, and successfully advanced it into a clinical compound now in Ph. II trials for patients with dyslipidemia.

Vactosertib is an orally bioavailable TGFβ type I receptor kinase inhibitor, that has demonstrated safety, tolerability, and clinical efficacy in combination with pomalidomide in a Ph. Ib trial for RRMM.

ONL1204 is ONL Therapeutics' small peptide inhibitor of the Fas receptor for IRD.

Compound 20 is a galactose-based monosaccharide Gal-3 inhibitor from Bristol Myers Squibb that shows potential in targeting fibrotic diseases.