True North Therapeutics Presents Data in Patients with Cold Agglutinin Disease (CAD) Supporting Patient Profiles for TNT009 Phase 1b Study
Data from Longitudinal Study Demonstrating Strong Correlation Between Complement Activity and Abnormal Hematologic Profiles
Study Results Presented at 2015 ASH Annual Meeting in Orlando, FL
South San Francisco, CA, December 7, 2015 — True North Therapeutics, a clinical stage biotechnology company developing novel therapies that selectively inhibit the Complement system to treat rare diseases, today announced new human data from a longitudinal study characterizing the complement activity and hematological profiles of blood samples from Cold Agglutinin Disease (CAD) patients. These data, presented at the American Society of Hematology 2015 annual meeting held December 5-8, 2015 in Orlando, demonstrated a clear relationship between complement activity and hemolysis/anemia in the vast majority of patients. The study results demonstrate that patients with the strongest signatures of in vivo complement activity are those that exhibit the most severe signs of anemia and hemolysis, and are therefore those in greatest need for treatment. The results here will help guide the assessment of patients for participation in clinical studies. True North plans to initiate a Phase 1b clinical study with TNT009, its lead complement inhibitor, in CAD patients by year-end 2015.
“We are building on True North’s deep understanding of the biology of CAD, a rare disease that lacks comprehensive characterization in contemporary clinical practice, as part of our effort to assess and select patients who are most in need of treatment for our Phase 1b trial with TNT009,” said Nancy Stagliano, PhD, Chief Executive Officer of True North. “The successful identification of such CAD patients creates a unique opportunity to evaluate proof-of-concept early in the clinical development of TNT009, as we believe these patients are most likely to achieve a robust clinical signal given their markers of anemia and hemolysis.”
In a poster titled, “Therapeutic Rationale and Clinical Development of TNT009, an Upstream Classical Pathway Inhibitor, for Cold Agglutinin Disease,” True North researchers describe the use of longitudinal blood samples from patients with CAD to identify those patients with abnormal complement levels and to track associated hemolysis and anemia. This research demonstrated that increased complement activity in patient samples was directly associated with markers of anemia and hemolysis. In supportive analyses, blood samples from CAD patients treated ex vivo with TNT009 showed inhibited complement activation on human red blood cells, further supporting the role of TNT009’s therapeutic potential in CAD patients.
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 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.
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