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

May 3, 2017

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.

  1. Prescient Therapeutics: FDA Grants Orphan Drug Designation for PTX-200 in Acute Myeloid Leukemia. 2017 May 1. http://ptxtherapeutics.com/wp-content/uploads/2017/05/20170501-ASX-Ann-Orphan-drug-approval-AMLPTX.pdf [Accessed 2017 May 2].
  2. Sampath D. et al. Phase I Clinical, Pharmacokinetic, and Pharmacodynamic Study of the Akt-Inhibitor Triciribine Phosphate Monohydrate in Patients with Advanced Hematologic Malignancies. Leuk Res. 2013 Nov; 37(11): 1461–1467. DOI: 10.1016/j.leukres.2013.07.034. Epub 2013 Aug 6.