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news.True North Therapeutics has secured Orphan Drug Designation from the US Food and Drug Administration (FDA) for TNT009 to treat autoimmune hemolytic anemia, including Cold Agglutinin Disease (CAD).
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CAD is an autoimmune hemolytic anemia in which autoantibodies target and destroy red blood cells, resulting in anemia, fatigue and potentially fatal thrombosis.
TNT009 selectively inhibits C1s, a classical pathway-specific enzyme. It is currently in clinical development for the treatment of rare diseases in hematology, transplantation, and dermatology.
It inhibits the dysregulation of the Classical pathway and maintains the key immune surveillance offered by the other two Complement system pathways, the Alternative pathway and Lectin pathway.
In June this year, the company unveiled positive interim results from a Phase 1b clinical study in patients with CAD.
Earlier this year, the European Medicines Agency granted orphan drug designation for TNT009.
True North Therapeutics CEO Nancy Stagliano said: “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.
“We look forward to continuing development of TNT009 for CAD and reporting additional Phase 1b clinical data when available.”
True North has a product engine that has the capacity to generate a pipeline of novel monoclonal antibodies for the treatment of rare diseases.
The company is investigating other treatment options for a range of Classical Complement-mediated diseases.
It is developing additional monoclonal antibodies to inhibit Classical Complement activity with other molecular targets and pharmacologic properties.
Image: CAD is an autoimmune hemolytic anemia in which autoantibodies target and destroy red blood cells. Photo: courtesy of Toeytoey/FreeDigitalPhotos.net.