MEI Pharma announced that the FDA has granted Orphan Drug designation to Pracinostat for the treatment of acute myeloid leukemia (AML). Pracinostat is an orally available histone deacetylase (HDAC) inhibitor.

Pracinostat has been tested in numerous Phase 1 and Phase 2 clinical trials in advanced hematologic disorders and solid tumor indications in both adult and pediatric patients. In a Phase 1 dose-escalation trial, Pracinostat demonstrated evidence of single-agent activity in elderly AML patients, including 2 out of 14 (14%) who achieved a complete remission (CR), with durable responses persisting 206+ and 362 days, respectively.

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MEI Pharma is currently conducting a Phase 2 clinical trial of Pracinostat in combination with Vidaza (azacitidine) in elderly patients with newly diagnosed AML. Preliminary data from the open-label trial is anticipated to be released in December 2014.

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