, Feb. 28, 2014
/PRNewswire/ -- MEI Pharma, Inc. (Nasdaq: MEIP), an oncology company focused on the clinical development of novel therapies for cancer, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the Company's investigational drug Pracinostat for the treatment of acute myeloid leukemia (AML).
"We are very pleased to receive this orphan drug designation," said Daniel P. Gold, Ph.D., President and Chief Executive Officer of MEI Pharma. "AML is a particularly devastating cancer for which there are currently few broadly effective treatments. Through development of Pracinostat, we hope to address this significant unmet medical need."
The FDA's Orphan Drug Designation program provides orphan status to drugs defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Orphan designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval.
Pracinostat is an orally available histone deacetylase (HDAC) inhibitor that has been tested in a number of Phase I and Phase II clinical trials in advanced hematologic disorders and solid tumor indications in both adult and pediatric patients. Pracinostat has been generally well tolerated in more than 200 patients to date, with readily manageable side effects that are often associated with drugs of this class, such as fatigue. In a Phase I dose-escalation trial, Pracinostat demonstrated 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. MEI Pharma is currently conducting a Phase II clinical trial of Pracinostat in combination with Vidaza in elderly patients with newly diagnosed AML. Preliminary data from the open-label trial is anticipated by December 2014.
MEI Pharma owns exclusive worldwide rights to Pracinostat.
Acute myeloid leukemia (also known as acute myelogenous leukemia) is the most common acute leukemia affecting adults, and its incidence is expected to increase as the population ages. The American Cancer Society estimates about 14,590 new cases of AML per year in the U.S., with an average age of about 66. Treatment options for AML remain virtually unchanged over the past 30 years. Front line treatment consists primarily of chemotherapy, while the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology recommend Vidaza® (azacitidine) or Dacogen® (decitabine) as low intensity treatment options for AML patients over the age of 60 who are unsuited for induction chemotherapy.
About MEI Pharma
MEI Pharma, Inc. (Nasdaq: MEIP) is a San Diego-based oncology company focused on the clinical development of novel therapies for cancer. The Company's lead drug candidate is Pracinostat, a potential best-in-class, oral HDAC inhibitor currently being developed for myelodysplastic syndrome (MDS) and AML. Results from a pilot Phase II clinical trial of Pracinostat in combination with Vidaza in patients with advanced MDS showed an overall response rate of 90% (nine of 10). Data from three ongoing Phase II clinical trials of Pracinostat are expected by the end of 2014. MEI Pharma is also developing ME-344, a mitochondrial inhibitor that has shown preliminary evidence of single-agent activity in a first-in-human clinical study in patients with refractory solid tumors, including eight of 21 evaluable patients (38%) who achieved stable disease or better. In September 2013, the Company further expanded its pipeline of drug candidates with the acquisition of PWT143, a highly selective PI3-kinase delta inhibitor. For more information, go to www.meipharma.com.
Under U.S. law, a new drug cannot be marketed until it has been investigated in clinical trials and approved by the FDA as being safe and effective for the intended use. Statements included in this press release that are not historical in nature are "forward-looking statements" within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. You should be aware that our actual results could differ materially from those contained in the forward-looking statements, which are based on management's current expectations and are subject to a number of risks and uncertainties, including, but not limited to, our failure to successfully commercialize our product candidates; costs and delays in the development and/or FDA approval, or the failure to obtain such approval, of our product candidates; uncertainties or differences in interpretation in clinical trial results; our inability to maintain or enter into, and the risks resulting from our dependence upon, collaboration or contractual arrangements necessary for the development, manufacture, commercialization, marketing, sales and distribution of any products; competitive factors; our inability to protect our patents or proprietary rights and obtain necessary rights to third party patents and intellectual property to operate our business; our inability to operate our business without infringing the patents and proprietary rights of others; general economic conditions; the failure of any products to gain market acceptance; our inability to obtain any additional required financing; technological changes; government regulation; changes in industry practice; and one-time events. We do not intend to update any of these factors or to publicly announce the results of any revisions to these forward-looking statements.
SOURCE MEI Pharma, Inc.