Drug intelligence / Profile preview

AC-430

Development stage
Phase 1
Lead developer
Daiichi Sankyo
Modality
Small Molecules
Administration
Oral
01

Overview

AC‑430 is an investigational, potent, and selective small molecule inhibitor of Janus kinase 2 (JAK2), originally developed by Ambit Biosciences (later acquired by Daiichi Sankyo). It was designed for the treatment of cancer and autoimmune diseases, including rheumatoid arthritis and leukemia. The compound is a racemic mixture of two enantiomers (AC410 and AC409), with the (R) optical isomer specifically claimed in patent literature. In preclinical studies, AC‑430 demonstrated strong activity against both wild-type JAK2 and the V617F mutant form associated with myeloproliferative neoplasms. Despite promising preclinical efficacy and tolerability at low oral doses, clinical development was discontinued after reaching Phase I trials for leukemia; it did not advance beyond preclinical stages for autoimmune disorders or inflammation[1][4][5][8].

Other names
(R)-(4-fluorophenyl)-[4-[(5-methyl-1H-pyrazol-3-yl)amino]quinazolin-2-yl]methanol1241914-87-3
02

Targets

JAK2 V617F (Janus kinase 2 V617F mutant)

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