U.S. FDA Accepts COUR Pharmaceuticals Investigational New Drug Application (IND) for a Proof-of-Concept Study for the Treatment of Primary Biliary Cholangitis (PBC)

CHICAGO, Oct. 4, 2021 /PRNewswire/ -- COUR Pharmaceuticals, a biotechnology company developing novel immune-modifying nanoparticles (CNPs) to treat immune disorders today announced the U.S. FDA accepted an investigational new drug application (IND) for a proof-of-concept study of COUR's investigatory therapy CNP-104 to address autoimmune causes of Primary Biliary Cholangitis (PBC).

COUR's nanoparticle platform (CNP) is a novel, proprietary system combining disease specific pathogenic antigens with state-of-the-art pharmaceutical nanoparticles that mimic normal removal of dead or dying cells from the body. This mimicry initiates a tolerance signal to the immune system during which disease specific antigens encapsulated within the particle are presented to the T cells responsible for driving inflammation and immune dysfunction. Once disease specific T cells are presented with both the tolerance signal and the disease-causing antigen, the T cells are reprogramed to either turn off and become non-active or to induce regulatory T cells that promote immune tolerance, thus facilitating Immune Reprograming by CNPs.

PBC affects an estimated 130,000 people in the United States, primarily women. Currently, healthcare professionals and patients rely on symptomatic therapies with limited results to slow down the progression of PBC but do not address its inherent cause, autoimmunity to PDC-E2 and the inflammatory cellular infiltrate in the liver. This proof of concept trial will assess the safety and tolerability of CNP-104 in adults with PBC and aims to find the optimal dose level that strikes the best balance between safety and treatment activity. CNP-104 will be administered via intravenous infusion and harnesses the patient's own immune system to induce tolerance to PDC-E2.

"COUR's proprietary CNP system has demonstrated the opportunity to address a wide range of conditions across the spectrum of immune disorders," said COUR CEO John Puisis. "CNP-104 eliminates the need for chronic treatment and offers the potential for better clinical outcomes, allowing a greater quality of life for PBC patients and their families." 

"Current therapies aimed at treating PBC address symptoms, but do not address the underlying cause of the disease," said M. Eric Gershwin, M.D., MACP, MACR, Distinguished Professor of Medicine at University of California, Davis. "CNP-104 has the potential to halt and reverse PBC by reprogramming T cells to promote immune tolerance through T regulatory cells. For the first time, patients have an opportunity to address progressive liver dysfunction and damage."

COUR's lead product for celiac disease, CNP-101, in partnership with Takeda, is the first demonstration of antigen-specific immune cell reprogramming in humans, in any autoimmune condition.

Trials for CNP-104 for the treatment of PBC are set to begin in Q4 2021.

About COUR Pharmaceuticals
COUR Pharmaceuticals is developing first-in-class therapies designed to reprogram the immune system to achieve antigen-specific tolerance for immune-mediated disease. COUR's platform of immune-modifying nanoparticles treats the root cause of immune disease, unlike traditional approaches, which only minimize symptoms using toxic immune suppression. COUR's lead product for celiac disease, partnered with Takeda Pharmaceutical Company, is the first demonstration of induction of antigen-specific T cell immune reprogramming in any autoimmune disease. Data from clinical and preclinical settings demonstrate the opportunity for the COUR nanoparticle platform to address a wide range of immune and inflammatory conditions. The underlying technology was acquired from Northwestern University and draws from more than 30 years of research by the laboratory of Stephen D. Miller, Ph.D., the Judy E. Guggenheim Research Professor of Microbiology-Immunology.

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SOURCE COUR Pharmaceuticals