Privately held biotech firm NKT Therapeutics announced that its lead therapeutic NKTT120 has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the treatment of sickle cell disease.
NKTT120 is a humanized monoclonal antibody (mAb) that works to lower the number of iNKT cells, which are considered to be key players in the inflammation and organ damage that characterize the disease.. The treatment has been shown to reduce iNKT cell-mediated inflammation. It has also previously received Orphan Drug Designation for the same indication.
Robert Mashal, CEO of NKT Therapeutics, said, “We look forward to working with the FDA on the design of our efficacy trials for NKTT120. Fast Track status will help us reach our goal of bringing this new therapy to patients as rapidly as possible.” The designation is granted to support development and speed review of promising drugs that address serious or life-threatening diseases and conditions.
The company also reported that it has completed dosing in a Phase 1B trial of the drug. The trial’s primary endpoint is safety while secondary endpoints include quality of life, effects on inflammation markers of the disease, and daily pain scores among patients.
Sickle cell disease is a group of inherited red blood cell disorders characterized by the malformation of red blood cells. The disease causes a persistent shortage of red blood cells as well as acute chest syndrome, pain, infection, and stroke. According to the Centers for Disease Control and Prevention (CDC), SCD afflicts an estimated 90,000 to 10,000 Americans.
Commenting on the drug’s mechanism of action, Joshua Field of the Blood Center of Wisconsin, said, “Much evidence exists that iNKT cells generate an inflammatory cascade that promotes and sustains the painful vascular events suffered by patients with sickle cell disease, and researchers have shown in animal models of SCD that depleting iNKT cells decreases inflammation and prevents organ damage.” The company presented the results of the Phase 1 safety and dose escalation study last year at the 55th American Society of Hematology (ASH) meeting. NKT stated it expects to present updated results of the clinical study at an upcoming major medical conference later in 2014.