Ambit Biosciences is a biopharmaceutical company focused on the discovery, development and commercialization of drugs to treat unmet medical needs in oncology, autoimmune and inflammatory diseases by inhibiting kinases that are important drivers for those diseases. Our pipeline currently includes three programs, each aimed at the inhibition of validated kinase targets.
Our lead drug candidate, quizartinib, is a once-daily, orally-administered, potent and selective inhibitor of FMS-like tyrosine kinase 3 (FLT3), a validated target in the treatment of AML. We plan to initiate a randomized comparative Phase 3 trial of quizartinib in relapsed/refractory AML patients with the FLT3-ITD mutation, in early 2014. The FLT3-ITD mutation acts like a “power switch” that causes leukemic cells, or blasts, to spread more aggressively and grow back more rapidly following chemotherapy, conferring an especially poor survival outcome. Quizartinib is designed to turn off this switch.
Our second drug candidate in clinical development, AC410, is a potent, selective, orally-administered, small molecule inhibitor of Janus kinase 2 (JAK2) that has potential utility for the treatment of autoimmune and inflammatory diseases.
Our third program consists of a potent and exquisitely selective small molecule compound, AC708, which inhibits the colony-stimulating factor-1 receptor (CSF1R), a receptor tyrosine kinase. This compound is in preclinical studies and has potential utility in oncology, autoimmune and inflammatory diseases. All of our drug candidates and clinical candidates have been internally discovered by us.