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news.The US Food and Drug Administration (FDA) has granted orphan drug designation to MEI Pharma's investigational drug Pracinostat for the treatment of acute myeloid leukemia (AML).
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MEI Pharma is a US-based oncology firm focused on the clinical development of novel therapies for cancer.
Pracinostat is an orally available histone deacetylase (HDAC) inhibitor that has been tested in several Phase I and Phase II clinical trials in advanced hematologic disorders and solid tumor indications in both adult and pediatric patients.
MEI Pharma president and chief executive officer Daniel Gold said the company is happy to receive this orphan drug designation.
"AML is a particularly devastating cancer for which there are currently few broadly effective treatments," Gold said.
"Through development of Pracinostat, we hope to address this significant unmet medical need."
To date, Pracinostat has been generally well tolerated in over 200, with readily manageable side effects that are often associated with drugs of this class, such as fatigue.
In a Phase I dose-escalation trial, the drug showed evidence of single-agent activity in elderly AML patients, including two out of 14 (14%) who achieved a complete remission (CR), with durable responses persisting 206+ and 362 days, respectively.
The company is currently carrying out an open-label Phase II clinical trial of Pracinostat in combination with Vidaza in elderly patients with newly diagnosed AML and the preliminary data is anticipated by December 2014.