On 1 stMay 2017, the U.S. Food and Drug Administration(FDA) granted Orphan Drug Designation to PTX-200 for the treatment of patients with Acute Myeloid Leukemia (AML). 1
Akt, a serine/threonine protein kinase, is constitutively phosphorylated and hyper-activated in several diseases including AML. High levels of phosphorylated Akt is associated with adverse outcomes in AML patients. 2 PTX-200(triciribine phosphate monohydrate) is a potent small molecule inhibitor that specifically inhibits Akt by a different mechanism, and so is comparably safer, compared to other non-specific kinase inhibitors.
Currently, PTX-200 is being explored in a phase 1/2 study ( NCT02930109), which is aiming to assess the safety and efficacy of PTX-200 in combination with cytarabine in patients with relapsed or refractory AML.