The FDA grants PTX-200 Orphan Drug Designation for the treatment of AML

On 1^st May 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.