SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--True North Therapeutics, a clinical stage biotechnology company developing novel therapies for Complement-mediated rare diseases, today announced clinical progress with its lead program TNT009, a first-in-class anti-C1s monoclonal antibody. True North has completed a Phase 1a clinical study evaluating single and multiple ascending doses of TNT009 in healthy volunteers and has initiated a Phase 1b study in patients with Cold Agglutinin Disease (CAD). Nancy Stagliano, Ph.D. President and CEO of True North Therapeutics will present the results of the Phase 1a study validating TNT009’s proof-of-mechanism along with the overall development plan for TNT009 at the 34th Annual J.P. Morgan Healthcare Conference on Tuesday, January 12, 2016 at 11:00 a.m. PT.
Data from the Phase 1a trial demonstrated proof-of-mechanism for TNT009’s inhibition of the Classical Complement pathway via a blood-based pharmacodynamic assay, with no relevant adverse events. Based on these data, True North has initiated a Phase 1b study in patients with CAD, an autoimmune hemolytic anemia in which autoantibodies target and destroy red blood cells, causing anemia, fatigue and potentially fatal thrombosis. In addition to CAD, TNT009 is being evaluated in the Phase 1b trial in three other Complement-mediated rare diseases, across hematology, organ transplant and dermatology settings that share the same basic underlying dysregulation of the Classical Complement pathway. Top-line data from the Phase 1b CAD study is expected in mid-2016, demonstrating the safety and tolerability of TNT009 in CAD patients, target engagement using blood-based biomarkers, and proof-of-concept through modulation of blood parameters.
“We are encouraged by these Phase 1a data demonstrating clinical proof-of-mechanism for TNT009 and look forward to reporting top-line Phase 1b data later this year from which we would expect to observe clinical proof-of-concept signals in CAD patients,” said Nancy Stagliano, PhD, Chief Executive Officer of True North. “Given the inadequate treatment options available to CAD patients, a significant unmet need remains for patients suffering from moderate to severe anemia that can be life-threatening. By targeting upstream in the Classical Complement pathway, TNT009 has the potential to provide more effective treatment options for these patients by minimizing the anemia and hemolysis associated with CAD.”
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 twenty per million. There are limited treatment options available and many of these patients, despite therapy, have moderate to severe anemia.
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. TNT009 is also being evaluated in three other complement-mediated rare diseases that share the same basic underlying mechanism in the hematology, transplant and dermatology settings.
About True North Therapeutics
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. The company’s lead drug candidate, TNT009, offers a novel approach for treating Complement-mediated diseases by selectively inhibiting a target in the Classical Complement pathway. True North’s antibody drug development is focused on the treatment of 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.