Drug to Treat Autoimmune Hemolytic Anemia Granted FDA Orphan Drug Status

True North Therapeutics’ TNT009 for the treatment of autoimmune hemolytic anemia, including cold agglutinin disease (CAD), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). TNT009 is currently in clinical development for the treatment of CAD, an autoimmune hemolytic anemia in which autoantibodies target and destroy red blood cells, causing anemia, fatigue and potentially fatal thrombosis. The prevalence of primary CAD is approximately 16 per million, and there are limited treatment options available.

TNT009 is a first-in-class monoclonal antibody that selectively inhibits the classical complement pathway by targeting C1s, a serine protease within the C1- complex in the complement pathway of the immune system, thus preventing downstream disease processes involving phagocytosis, inflammation and cell lysis. It has a unique mechanism of action and high target specificity that selectively inhibits disease processes in the classical complement pathway while maintaining the immune surveillance provided by the alternative complement pathway and lectin complement pathway.

“Receiving both U.S. and EMA [European Medicines Agency] Orphan Drug designation for TNT009 is an important milestone in the development of much-needed new treatment options for patients with CAD,” said Nancy Stagliano, PhD, chief executive officer of True North. “We look forward to continuing development of TNT009 for CAD and reporting additional Phase 1b clinical data when available.”