7 minute read
Mar. 13, 2024

Inaxaplin: A Breakthrough APOL1 Channel Inhibitor for a Genetically Defined Kidney Disease

inaxaplin (VX-147)

oral, apolipoprotein L1 channel inhibitor Ph. II/III candidate for chronic kidney disease HTS/MetID-enabled optimization New England Journal of Medicine, March 2023 Vertex Pharmaceuticals, Boston, MA


Inaxaplin (VX-147), developed by Vertex, is an inhibitor of APOL1 (apolipoprotein L1) channel activity currently in a Ph. II/III pivotal study for the treatment of chronic kidney disease caused by specific variants of the APOL1 gene. It was recently granted Breakthrough Therapy designation by the FDA and PRIME designation by the EMA. The discovery story, which is an excellent case study for the use of metabolite ID (MetID), was presented at the 2023 SCI/RSC 22nd Med. Chem. Symposium in Cambridge, UK. Inaxaplin has been called “the most important genomic-driven drug discovery for [chronic kidney disease] this century”, acting on a target with a fascinating human genetic validation story.



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