Inaxaplin information

Inaxaplin
Clinical data
Other names	VX-147
Legal status
Legal status	Investigational;
Identifiers
	IUPAC name 3-[5,7-Difluoro-2-(4-fluorophenyl)-1H-indol-3-yl]-N-[(3S,4R)-4-hydroxy-2-oxopyrrolidin-3-yl]propanamide;
CAS Number	2446816-88-0;
PubChem CID	147289591;
ChemSpider	115009344;
UNII	S2SJ2RVZ6Y;
ChEMBL	ChEMBL5083170;
Chemical and physical data
Formula	C21H18F3N3O3
Molar mass	417.388 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES C1[C@H]([C@@H](C(=O)N1)NC(=O)CCC2=C(NC3=C2C=C(C=C3F)F)C4=CC=C(C=C4)F)O;
	InChI InChI=1S/C21H18F3N3O3/c22-11-3-1-10(2-4-11)18-13(14-7-12(23)8-15(24)19(14)27-18)5-6-17(29)26-20-16(28)9-25-21(20)30/h1-4,7-8,16,20,27-28H,5-6,9H2,(H,25,30)(H,26,29)/t16-,20+/m1/s1; Key:CTXLPYZCBOVVQK-UZLBHIALSA-N;

Inaxaplin (VX-147) is a small-molecule apolipoprotein L1 inhibitor developed by Vertex Pharmaceuticals for APOL1-mediated kidney disease. In preliminary studies the drug has shown promise in treating people with kidney disease and multiple gain of function mutations on the APOL1 gene.^[1]^[2]

^ Gbadegesin, Rasheed; Lane, Brandon (August 2023). "Inaxaplin for the treatment of APOL1-associated kidney disease". Nature Reviews Nephrology. 19 (8): 479–480. doi:10.1038/s41581-023-00721-0. PMC 10461697. PMID 37106136.
^ Egbuna, Ogo; Zimmerman, Brandon; Manos, George; Fortier, Anne; Chirieac, Madalina C.; Dakin, Leslie A.; Friedman, David J.; Bramham, Kate; Campbell, Kirk; Knebelmann, Bertrand; Barisoni, Laura; Falk, Ronald J.; Gipson, Debbie S.; Lipkowitz, Michael S.; Ojo, Akinlolu; Bunnage, Mark E.; Pollak, Martin R.; Altshuler, David; Chertow, Glenn M. (16 March 2023). "Inaxaplin for Proteinuric Kidney Disease in Persons with Two APOL1 Variants". New England Journal of Medicine. 388 (11): 969–979. doi:10.1056/NEJMoa2202396. PMID 36920755. S2CID 257534251.

[1] Gbadegesin, Rasheed; Lane, Brandon (August 2023). "Inaxaplin for the treatment of APOL1-associated kidney disease". Nature Reviews Nephrology. 19 (8): 479–480. doi:10.1038/s41581-023-00721-0. PMC 10461697. PMID 37106136.

[2] Egbuna, Ogo; Zimmerman, Brandon; Manos, George; Fortier, Anne; Chirieac, Madalina C.; Dakin, Leslie A.; Friedman, David J.; Bramham, Kate; Campbell, Kirk; Knebelmann, Bertrand; Barisoni, Laura; Falk, Ronald J.; Gipson, Debbie S.; Lipkowitz, Michael S.; Ojo, Akinlolu; Bunnage, Mark E.; Pollak, Martin R.; Altshuler, David; Chertow, Glenn M. (16 March 2023). "Inaxaplin for Proteinuric Kidney Disease in Persons with Two APOL1 Variants". New England Journal of Medicine. 388 (11): 969–979. doi:10.1056/NEJMoa2202396. PMID 36920755. S2CID 257534251.