AC220 – A Best-In-Class FLT-3 Inhibitor for AML

AC220, Ambit’s lead product candidate, entered the clinic in 2006 for the treatment of Acute Myeloid Leukemia (AML), the most common type of blood cancer in adults. AC220 targets the kinase FLT3, which is mutated and constitutively activated in 25-40 percent of patients, causing poor prognosis and decreased response to existing treatments including chemotherapy and stem cell treatments.

AC220 binds potently and specifically to FLT3 with sub-nanomolar inhibition of FLT3 phosphorylation in an AML cell line derived from human patients. In preclinical studies, AC220 has shown a superior pharmacokinetic and safety profile, with no significant inhibition of the five human CYP isoforms. AC220 is orally bioavailable and has been shown to induce tumor regression in a xenograft model at low doses.