True North Presents Additional Data from an Ongoing Clinical Study Showing Normalization of Hemoglobin Levels with TNT009 in Severely Anemic Cold Agglutinin Disease (CAD) Patients
Oral and Poster Presentations at the 2016 American Society of Hematology Annual Meeting
South San Francisco, CA, December 3, 2016 — True North Therapeutics, a clinical stage biotechnology company developing novel product candidates that selectively inhibit the Complement system, presented updated clinical data today from an ongoing Phase 1b trial of TNT009 for the treatment of Cold Agglutinin Disease (CAD), a rare form of autoimmune hemolytic anemia. These interim clinical results further support TNT009 as a promising treatment for CAD, given the normalization of hemoglobin levels seen in severely anemic CAD patients. The data are described in oral and poster presentations by True North researchers and collaborators at the American Society of Hematology (ASH) annual meeting on December 3 – 6, 2016 in San Diego.
“We are highly encouraged by the growing body of efficacy and safety data of TNT009, enabling us to address an underserved CAD patient population,” said Nancy Stagliano, PhD, Chief Executive Officer of True North Therapeutics. “Based on these results, we are actively working with the treatment community to rapidly advance the clinical program for TNT009 as a potential new therapeutic for CAD patients.”
The additional data from the ongoing clinical study demonstrated robust activity of TNT009 in patients with CAD, as described in the oral presentation titled “TNT009 Prevents Erythrocyte C3 Fragment Opsonization and Rescues Reticulocytes from Destruction in Patients with Cold Agglutinin Disease” and the poster presentation titled “Chronic Inhibition of Complement C1s by TNT009 Produces Sustained, Complete Remission in Patients with Severe, Transfusion-Dependent Cold Agglutinin Disease (CAD).”
The clinical data were reported on six CAD patients. All patients were dosed with TNT009 in the Phase 1b trial, followed by treatment under a named patient provision for some patients. The named patient provision was requested by the treating physicians and allowed for continued access to TNT009, treating some patients for over 3 months. Among the six patients in the Phase 1b trial, one patient had an active malignancy, a lymphoma with 70% bone marrow infiltration. Highlights from the presentations at ASH include:
- All five CAD patients without an active malignancy had a sustained response with improvements in hemoglobin of approximately 4-5 g/dL, reaching hemoglobin levels of > 12g/dL.
- Patients that required transfusions immediately prior to dosing in the Phase 1b trial became transfusion-free during treatment with TNT009.
- TNT009 was safe and well-tolerated in the study, with no serious or severe drug-related adverse events.
- The clinical data support TNT009’s mechanism of inhibition of the Classical Complement pathway, by showing reductions of C3 opsonized red blood cells concurrent with improvement in hemoglobin levels.
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. Positive interim results from the ongoing Phase 1b study in patients with CAD were initially presented in June 2016 at the 21st Congress of the European Hematology Association (EHA). True North received Orphan Drug Designation for TNT009 from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
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 thrombotic events. 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 hematological disease. Because numerous other diseases are known to share the basic underlying pathological 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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