Marker Therapeutics, Inc. announced that zedenoleucel, its multi-tumor-associated antigen (multiTAA)-specific T cell product candidate, MT-401, was granted Orphan Drug Designation by the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA) for the treatment of patients with acute myeloid leukemia (AML). AML is a life-threatening and chronically debilitating disease that is rapidly progressive and fatal if untreated. Relapse rates after initial treatment are high, and the next step for eligible patients is an allogeneic hematopoietic stem cell transplant (HSCT).

Unfortunately, AML relapse after HSCT is frequent and outcomes are dismal. Patients who relapse after HSCT have an estimated median survival of less than one year (Estey and Döhner, Lancet, 2006), highlighting the urgent need for new therapies. MT-401 utilizes a novel non-genetically modified approach that recognizes multiple antigens expressed on tumor cells, thereby designed to minimize tumor escape.

MT-401 is currently being studied in a Phase 2 clinical trial for the treatment of relapsed AML following allogeneic HSCT, and was designed to specifically target four different antigens that are upregulated in AML but have limited expression on normal cells. In the European Union, orphan drug designation is granted to drugs intended for the treatment of life-threatening or chronically debilitating conditions affecting no more than five in 10,000 individuals in the European Union. Orphan drug designation by the EMA provides crucial support to expedite the development and market readiness of necessary drugs for such rare diseases.

This designation will help Marker Therapeutics continue to develop MT-401 to fill a significant void in the treatment of AML and provides Marker Therapeutics with a range of potential benefits, including ten years of market exclusivity following approval, reduced regulatory fees, and invaluable scientific advice from the EMA during the drug development phase.