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 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. Quizartinib is currently under clinical development in patients with relapsed/refractory acute myeloid leukemia (AML) and in newly diagnosed AML patients in combination with chemotherapy as well as maintenance following a hematopoietic stem cell transplantation (HSCT).
We plan to initiate a randomized comparative Phase 3 trial of quizartinib in relapsed/refractory AML patients with the FLT3-ITD mutation, in the second quarter 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.
In addition to quizartinib, an additional candidate for clinical development is, 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.
Another program candidate 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.