Helsinn, a Swiss pharmaceutical group focused on building quality cancer care products, and MEI Pharma, an oncology company focused on the clinical development of novel therapies for cancer,  announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to pracinostat, an investigational drug candidate currently in a phase 3 study in combination with azacitidine for the treatment of acute myeloid leukemia (AML) in adult patients unfit to induction chemotherapy.

The orphan drug designation is based on the scarcity of treatments for patients suffering from AML and on positive phase 2 study results (see below) that were presented at the American Society of Hematology (ASH) Annual Meeting, in December 2016.

The EMA orphan drug designation is a status assigned to a medicine intended for use against a rare condition in the European Union (prevalence of the condition in the European Union must not be more than 5 in 10,000). The designation allows pharmaceutical companies to benefit from incentives offered by the EU to develop a medicine for the treatment, prevention or diagnosis of a disease that is life threatening or a chronically debilitating rare disease. These include 10 years of market exclusivity once approved, alongside a range of other regulatory advantages.

Riccardo Braglia, Helsinn Group vice chairman and chief executive officer, commented: “Helsinn is pleased with the decision of the EMA to grant orphan drug designation to Pracinostat. This decision encourages us to continuously dedicate significant resources to accelerate our clinical trial program, with a goal of helping patients who are fighting rare and difficult-to-treat diseases, such as AML and, at present, have very few treatment options. Following the positive Phase 2 clinical trials of Pracinostat for patients with AML, Helsinn has recently initiated the Phase 3 program.”

“This designation from the EMA recognizes the potential that Pracinostat holds in addressing a significant unmet need for those suffering with AML,” said Daniel P. Gold, Ph.D., president and chief executive officer of MEI Pharma. “This represents another important milestone in the global development strategy for Pracinostat.”

1st patient randomized in phase 3 trial: In August of 2017, Helsinn and MEI Pharma announced that the first patient has been dosed in the pivotal phase 3 study of the investigational agent Pracinostat in combination with Azacitidine in adults with newly diagnosed acute myeloid leukemia (AML) who are unfit to receive intensive induction chemotherapy. The primary endpoint of the study is overall survival. Secondary endpoints include, among others, morphologic complete remission (CR) rate, cytogenetic complete remission and complete remission without minimal residual disease.

Phase 2 study results for Pracinostat for the treatment of AML: Results from a phase 2 open-label, single-arm, multicenter study of pracinostat and azacitidine in 50 patients aged = 65 years with newly diagnosed AML not eligible for induction chemotherapy showed a median overall survival of 19.1 (95%CI: 10.0-26.5) months, one-year survival of 62% and a CR rate of 42%. These results were presented at the American Society of Hematology (ASH) Annual Meeting in December 2016.