M2N Bio, an investment partner in Pacylex Pharmaceuticals, said that the PCLX-001 drug to treat patients with acute myeloid leukemia (AML) received U.S. FDA orphan drug designation (ODD) on Saturday.

PCLX-001 is the first N-myristoyltransferase (NMT) inhibitor in its class undergoing phase 1 clinical development by Pacylex in Canada. PCLX-001 is the first small-molecular NMT inhibitor developed by the University of Dundee’s Drug Discovery Unit in the U.K. 

PCLX-001 showed a 10-fold or more potent effect than the two drugs currently used to treat blood cancer, Imbruvica (ingredient: ibrutinib) and Sprycel (dasatinib), in tests using in vitro cultured cancer cells.

According to M2N, the ODD includes benefits such as clinical cost support, 50 percent tax reduction in clinical trial costs in the U.S., advice on trial plans for FDA marketing approval, shortened FDA approval review and guaranteed market exclusivity for seven years from authorization.

“Accordingly, this IND approval drug for AML treatments has now been added as an orphan drug, which reflects expectations from U.S. health authorities for future development, said a Pacylex official.

PCLX-001 is currently being studied in four hospitals in Canada in patients with non-Hodgkin's lymphoma (NHL) and solid cancer. In the U.S., IND submission has been completed with the FDA to study PCLX-001 in AML patients.

Additionally, the University of Texas MD Anderson Cancer Center will receive $1.4 million to develop an AML treatment for PCLX-001 from the U.S. Department of Defense.

"While pursuing the expansion of indications for PCLX-001, we were notified last weekend that PCLX-001 had been designated as an orphan drug for AML by the FDA," said an M2N Bio official. "We will continue to invest in companies specializing in developing anti-cancer drugs at home and abroad like Pacylex and Green Fire Bio."