[October 13, 2016] |
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True North Therapeutics Receives FDA Orphan Drug Designation for TNT009 for the Treatment of Autoimmune Hemolytic Anemia, including Cold Agglutinin Disease (CAD)
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 (News - Alert)) 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 forwhich 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.
About TNT009 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.
View source version on businesswire.com: http://www.businesswire.com/news/home/20161013005741/en/
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