COUR Pharmaceuticals' Phase 2a CNP-104 Data on Primary Biliary Cholangitis Accepted for Liver Meeting® 2024

COUR Pharmaceuticals, a clinical-stage biotechnology company, is set to unveil detailed safety and efficacy findings from its Phase 2a study of the drug CNP-104, aimed at treating Primary Biliary Cholangitis (PBC). These significant findings will be presented through a late-breaking poster at The Liver Meeting® 2024, organized by the American Association for the Study of Liver Disease (AASLD) in San Diego, California, from November 15-18. The presentation, titled "Tolerogenic treatment with CNP-104 results in regulation of Th17 cells slowing progression of PBC on liver stiffness," will be delivered by Dr. Paul M. Peloso, COUR’s Chief Medical Officer, on November 18, 2024, from 1:00 to 2:00 p.m. PT, under publication number 5039.

CNP-104 is a biodegradable nanoparticle containing the E2 component of the mitochondrial pyruvate dehydrogenase complex (PDC), a critical autoantigen in PBC. This novel drug targets the root cause of PBC by inducing tolerance to pathogenic PDC-E2 T-cells, which are responsible for inflammation in bile ducts. The study has shown that CNP-104 can potentially slow the progression of the disease, as evidenced by a statistically significant decrease in liver stiffness compared to a placebo. This promising outcome could lead to improved liver health for patients suffering from PBC.

The development of CNP-104 is particularly noteworthy as it was granted Fast Track Designation by the United States Food and Drug Administration (FDA) in January 2022. This designation is crucial as it might enable the drug to qualify for Accelerated Approval and Priority Review, provided it meets certain criteria. These regulatory advantages could expedite the drug’s availability to patients who need it most.

COUR Pharmaceuticals specializes in creating first-in-class therapies designed to reprogram the immune system to tackle the fundamental causes of autoimmune diseases. The company’s proprietary platform focuses on inducing antigen-specific immune tolerance. Their research has demonstrated the potential to treat various autoimmune conditions through this innovative approach.

In addition to its work on PBC, COUR Pharmaceuticals is actively enrolling patients in a phase 1b/2a double-blind, placebo-controlled, multicenter clinical study for Myasthenia Gravis. The company is also developing a product candidate for Type 1 Diabetes and has completed first-in-human studies for Celiac Disease, in collaboration with Takeda Pharmaceuticals. These initiatives highlight COUR’s commitment to addressing unmet medical needs in the field of autoimmune diseases.

The upcoming presentation at The Liver Meeting® 2024 provides an excellent platform for COUR Pharmaceuticals to showcase the potential of CNP-104 in managing PBC. The detailed results are eagerly anticipated by the medical community, given the drug’s ability to significantly reduce liver stiffness and its implications for slowing disease progression. This development marks a significant step forward in the treatment of PBC and underscores COUR’s innovative approach to tackling autoimmune diseases at their root cause.

The findings from the Phase 2a study are expected to pave the way for further research and development, potentially transforming the treatment landscape for patients with PBC. As COUR Pharmaceuticals continues to advance its clinical programs, the medical community and patients alike await the broader implications of their research, which promises to bring new hope to those affected by autoimmune diseases.