October 13, 2016
True North Therapeutics Receives FDA Orphan Drug Designation for TNT009 for the Treatment of Autoimmune Hemolytic Anemia, Including Cold Agglutinin Disease (CAD)
South San Francisco, CA, October 13, 2016 — True North Therapeutics, a clinical stage biotechnology company developing novel product candidates that selectively inhibit the Complement system to treat certain rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the Company’s lead product candidate, TNT009 for the treatment of autoimmune hemolytic anemia, including Cold Agglutinin Disease (CAD), a form of autoimmune hemolytic anemia for which there are limited treatment options available for patients. TNT009 is currently in a Phase 1b clinical study in patients with CAD, and positive interim results from the study were announced in June 2016. True North also received Orphan Drug Designation for TNT009 from the European Medicines Agency (EMA) in February 2016.
“Receiving both U.S. and EMA 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.”
Orphan drug designation is granted by the FDA to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain incentives which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. If a product that has orphan drug designation subsequently receives the first FDA approval for the disease for which it has such designation, the product is entitled to orphan product exclusivity.
About Cold Agglutinin Disease (CAD)
CAD is 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 sixteen per million. There are limited treatment options available and many of these patients, despite therapy, have moderate to severe anemia. True North sponsors a patient registry for CAD and other autoimmune hemolytic anemias, called the COMPASS Registry (https://www.compassregistry.org/), dedicated to advancing a deeper understanding of these diseases, engaging with patients, and supporting clinical trial recruitment.
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 hematology. Because numerous other diseases are known to share the basic underlying mechanism addressed by TNT009, the potential for other indications is clear.
About True North Therapeutics
True North Therapeutics is a clinical stage biopharmaceutical company focused on the discovery, development, and commercialization of first-in-class product candidates for Complement-mediated diseases. The company’s lead product candidate, TNT009, is designed to selectively inhibit a target in the Classical Complement pathway. True North’s antibody drug development is focused on the treatment of certain rare diseases mediated by the Complement system in hematology, transplantation, and dermatology. True North Therapeutics is located in South San Francisco, California. For more information, please visit www.truenorthrx.com.
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