Posted by Michael Wonder on 01 Aug 2016
MEI Pharma's pracinostat receives breakthrough therapy designation from FDA for treatment in combination with azacitidine of patients with newly diagnosed acute myeloid leukaemia unfit for intensive chemotherapy
1 August 2016 - MEI Pharma announced today that the U.S. FDA has granted breakthrough therapy designation for the investigational drug pracinostat in combination with azacitidine for the treatment of patients with newly diagnosed acute myeloid leukaemia who are ≥75 years of age or unfit for intensive chemotherapy.
In addition, agreement has been reached with the FDA on the company's proposed Phase III study design.
The breakthrough therapy designation is supported by data from a Phase II study of Pracinostat plus azacitidine in elderly patients with newly diagnosed AML, not candidates for induction chemotherapy, which showed a median overall survival of 19.1 months and a complete response (CR) rate of 42% (21 of 50 patients). These data compare favorably to a Phase III study of azacitidine (AZA-AML-0011), which showed a median overall survival of 10.4 months with azacitidine alone and a CR rate of 19.5% in a similar patient population. The combination of Pracinostat and azacitidine was generally well tolerated, with no unexpected toxicities. The most common grade 3 4 treatment-emergent adverse events included febrile neutropenia, thrombocytopenia, anemia and fatigue.
