COUR Pharma Announces Positive One-Year Results from the Phase 2a Study of CNP-104 in Primary Biliary Cholangitis

• CNP-104 improved key cholestasis endpoints at one year, including a composite serum alkaline phosphatase (ALP) and bilirubin biochemical response
• Durable effects observed across liver stiffness, prognostic risk markers, and liver function following only two doses of CNP-104 administered one week apart
• Full one-year dataset to be presented at future scientific meetings
  
  

CHICAGO, March 03, 2026 (GLOBE NEWSWIRE) -- COUR Pharma, a clinical-stage biotechnology company developing first-in-class, antigen-specific immune tolerance therapies for autoimmune diseases, today announced positive one-year results from its Phase 2a study evaluating CNP-104 as a potential treatment for patients with Primary Biliary Cholangitis (PBC).

“CNP-104 demonstrated robust antigen-specific immune modulation consistent with restoration of immune tolerance in PBC, as shown in the previously announced day 120 analysis,” said Dannielle Appelhans, Chief Executive Officer of COUR Pharma. “The one-year data confirms continued stabilization in liver stiffness measurements and validated prognostic risk scores, while newly demonstrating achievement of established composite biochemical response criteria at twelve months. Together, these results spanning both positive fibrotic and cholestatic biomarkers support the potential of CNP-104 as a novel therapeutic option of PBC and inform our ongoing evaluation of the program’s development strategy, including potential collaboration with a strategic partner.”

Following two loading doses of CNP-104, one-year findings show durable clinical effects including:

• Separation of CNP-104 treated patients from placebo on a composite biochemical response (defined as ALP <1.67× upper level of normal (ULN), ≥15% reduction from baseline, and total bilirubin ≤ ULN)
• Improvements in albumin, a marker of liver synthetic function
• Sustained statistically significant separation of liver stiffness versus placebo, a key independent marker of disease progression¹
• Improvement in the UK-PBC prognostic risk score and associated components (ALP, bilirubin, alanine aminotransferase, albumin, platelets)
• Favorable safety and tolerability profile through one year of follow-up, with all drug related adverse events graded as mild or moderate (grade 1 or 2), with no drug related severe adverse events (SAEs) reported
  

The Phase 2a first-in-human, double-blind, placebo controlled clinical trial (NCT05104853) was designed to assess the safety, tolerability, pharmacodynamics, and efficacy of two doses of CNP-104 administered one week apart in patients aged 18-75 who were unresponsive to treatment with ursodeoxycholic acid (UDCA) and/or obeticholic acid (OCA)². The previously reported 120-day primary study period analysis demonstrated modulation of the antigen specific Th17 T-cell axis, together with statistically significant clinical differentiation in liver stiffness measurement assessed by transient elastography (FibroScan^®).

Detailed one-year results from the Phase 2a study of CNP-104 will be submitted for presentation at a future scientific conference.

About CNP-104

CNP-104 is an investigational biodegradable nanoparticle encapsulating the E2 component of the mitochondrial pyruvate dehydrogenase complex (PDC-E2), a key autoantigen in PBC. CNP-104 aims to address the root cause of PBC by inducing tolerance to pathogenic PDC-E2 T cells that drive bile duct inflammation and progressive liver injury. CNP-104 has been granted Orphan Drug Designation as well as Fast Track Designation by the United States Food and Drug Administration (FDA).

About PBC
Primary biliary cholangitis (PBC), formerly known as primary biliary cirrhosis, is a chronic, progressive autoimmune liver disease that primarily affects women between 40 and 60 years of age. The disease is characterized by loss of tolerance to mitochondrial antigens and T-lymphocyte-mediated destruction of small intrahepatic bile ducts.
This immune-driven injury leads to cholestasis, progressive fibrosis, cirrhosis, and eventual liver failure, with some patients ultimately requiring liver transplantation. Symptoms such as fatigue and pruritus can significantly impair quality of life.

About COUR Pharma
COUR Pharma is a clinical-stage biotechnology company developing first-in-class therapies to treat patients with autoimmune diseases. COUR’s therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune diseases.

COUR has three proprietary clinical-stage programs - type 1 diabetes, myasthenia gravis and primary biliary cholangitis, and is currently enrolling patients in a Phase 1b/2a clinical study in type 1 diabetes. Additionally, COUR has two partnered programs, one with Takeda Pharmaceuticals for celiac disease and with Genentech for an undisclosed program.

Contacts

For Investor Relations
Brian Bock, Chief Financial Officer
bbock@courpharma.com

For Media
Owen Blaschak
oblaschak@lifescicomms.com

References

1. Corpechot, Christophe et al. “Liver stiffness measurement by vibration-controlled transient elastography improves outcome prediction in primary biliary cholangitis.” Journal of hepatology vol. 77,6 (2022): 1545-1553.doi:10.1016/j.jhep.2022.06.017
2. Study to Evaluate the Safety, Tolerability, PDs, and Efficacy of CNP-104 in Subjects with Primary Biliary Cholangitis. https://clinicaltrials.gov/study/NCT05104853
   
   

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