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Cat. No. Product Name CAS No. Information



Danicopan (ACH-4471, ACH-0144471) is a highly potent, orally active Factor D inhibitor with Kd of 0.54 nM, inhibits the proteolytic activity of purified Factor D against substrate Factor B in complex with C3b, blocking production of Bb fragment IC50 of 15 nM; significantly reduces complement-mediated hemolysis at concentrations as low as 10 nM in cells from paroxysmal nocturnal hemoglobinuria patients, decreases C3 fragment deposition on paroxysmal nocturnal hemoglobinuria erythrocytes; blocks alternative pathway activity when administered orally to cynomolgus monkeys.

Other Indication

Phase 2 Clinical




AG-348 is a first-in-class, allosteric activator of Pyruvate kinase (PK) with mean AC50 of 29 nM (PK-R), activates a spectrum of recombinant mutant PK-R enzymes (R532W, R510Q); AG-348 can increases the activity of wild-type and mutant PK enzymes in biochemical assays and in patient red blood cells treated ex vivo; demonstrates enhanced glycolytic pathway activity in PK-deficient RBCs that contain a variety of mutations.

Other Indication

Phase 3 Clinical




A4250 (A-4250, Odevixibat) is a small molecule ileal bile acid transporter (IBAT) inhibitor for the treatment of cholestatic liver diseases including progressive familial intrahepatic cholestasis and NASH.

Other Indication

Phase 3 Clinical




LAU159 is a potent, functionally selective positive modulator of α6β3γ2 GABAA receptor.

Other Indication

Phase 3 Clinical




Elamipretide (SS-31, MTP-131, Bendavia) is a novel cell-permeable antioxidant tetrapeptide (D-Arg-dimethylTyr-Lys-Phe-NH2) that targets inner mitochondrial membrane and prevents oxidative damage of neuronal cells and other cell types; prevents mitochondrial depolarization, reduces islet cell apoptosis, increases islet cell yield, and improves posttransplantation function.

Other Indication

Phase 3 Clinical


GSK 2793660


GSK 2793660(GSK2793660) is a potent, selective, irreversible and orally bioavailable cathepsin C inhibitor, but not the activity of downstream neutrophil serine proteases, for the treatment of cystic fibrosis, non-cystic fibrosis bronchiectasis, ANCA-associated vasculitis and bronchiectasis.

Other Indication

Phase 1 Discontinued


GSK598809 hydrochloride


GSK598809 hydrochloride is a potent, selective, CNS penetrant and orally bioavailable dopamine D3 receptor antagonist with pKi of 8.9; displays >100-fold selectivity over D2 and H1 receptors; significantly attenuates the expression of conditioned place preference (CPP) to nicotine and cocaine; potentiates the hemodynamic effects of cocaine in dogs, also increases mean arterial blood pressure and heart rate in vivo.

Other Indication

Phase 2 Clinical

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