The European Medicines Agency (EMA) granted Orphan Drug Designation to True North Therapeutics' TNT009 for the treatment of autoimmune haemolytic anaemia including Cold Agglutinin Disease (CAD), in which autoantibodies target and destroy red blood cells, causing anaemia, fatigue and potentially fatal thrombosis.

“Receiving Orphan Drug Designation from the EMA is another important step forward for True North as we continue to advance development of TNT009,” said Nancy Stagliano, PhD, chief executive officer of True North. “We recently initiated a phase 1b clinical study evaluating TNT009 in patients with Cold Agglutinin Disease and look forward to reporting top-line data later this year.”

Orphan Drug Designation in the EU is granted to drugs or biologics that treat a life-threatening or chronically debilitating rare disease affecting fewer than five in 10,000 individuals in the European Union. Products receiving orphan drug designation are eligible to receive market exclusivity for a period of up to ten years, as well as development incentives such as regulatory and protocol assistance and scientific advice.

CAD is an autoimmune haemolytic anaemia in which autoantibodies target and destroy red blood cells, causing anaemia, fatigue and potentially fatal thrombosis. The prevalence of primary CAD is approximately twenty per million. There are limited treatment options available and many of these patients, despite therapy, have moderate to severe anaemia.

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. TNT009 thereby prevents downstream disease processes involving phagocytosis, inflammation, and cell lysis. With a unique mechanism of action and high target specificity, TNT009 selectively inhibits disease processes in the Classical Complement pathway while maintaining the important immune surveillance provided by the Alternative Complement Pathway and Lectin Complement Pathway. TNT009 is currently in clinical development for the treatment of Cold Agglutinin Disease (CAD), a rare disease in haematology. TNT009 is also being evaluated in three other complement-mediated rare diseases that share the same basic underlying mechanism in the haematology, transplant and dermatology settings.

True North Therapeutics is a clinical stage biotechnology company developing a pipeline of novel therapies that selectively target the complement pathway of the immune system to address fundamental mechanisms in rare diseases with high unmet need.