COUR Pharmaceuticals Secures FDA Orphan Drug Designation for CNP-104 in Primary Biliary Cholangitis
CHICAGO, Jan. 08, 2025 (GLOBE NEWSWIRE) — COUR Pharmaceuticals, a clinical-stage biotechnology company developing first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, has secured Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for CNP-104 for the treatment of primary biliary cholangitis (PBC).
“Receiving Orphan Drug Designation for CNP-104 underscores its potential to become the first disease-modifying treatment for individuals with PBC,” said Dannielle Appelhans, President and Chief Executive Officer of COUR. “This designation follows our presentation of positive topline data from the Phase 2a clinical trial of CNP-104 in PBC at The Liver Meeting® 2024. Notably, in addition to demonstrating favorable T cell responses among treated participants, CNP-104 slowed disease progression, as evidenced by a statistically significant reduction in liver stiffness measured by FibroScan by day 120 of the study period. We are now collaborating with our distinguished clinical advisors and key opinion leaders to advance CNP-104 to the next phase of clinical development.”
The FDA grants ODD status to encourage the development of treatments for rare diseases affecting less than 200,000 individuals in the United States. This designation qualifies sponsors for various incentives, including tax credits for clinical trials, exemption from user fees, and the potential for seven years of market exclusivity following approval. CNP-104 additionally received Fast Track Designation from the FDA in January 2022, which would make it eligible for Accelerated Approval and Priority Review if specific criteria are met.
About CNP-104:
CNP-104 is a biodegradable nanoparticle encapsulating the E2 component of the mitochondrial pyruvate dehydrogenase complex (PDC), a key autoantigen in PBC. CNP-104 aims to address the root cause of PBC by inducing tolerance to pathogenic activated PDC-E2 T-cells that drive inflammation in bile ducts, leading to improvement in clinical outcomes of liver health.
About PBC:
PBC is a chronic, life-threatening autoimmune disease of the liver that disproportionately affects women, and is a leading cause of liver transplants in this population. PBC is characterized by impaired bile flow (cholestasis) and the accumulation of toxic bile acids in the liver, which can lead to fibrosis, cirrhosis and ultimately liver failure, necessitating a transplant. Symptoms such as fatigue and pruritus (severe itching) significantly affect patients’ quality of life. While the exact cause of PBC is unknown, an autoimmune response to the E2 component of the pyruvate dehydrogenase complex (PDC), is thought to be highly associated with the development of the disease.
About COUR Pharmaceuticals:
COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune diseases. COUR’s first-in-class 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 is currently enrolling patients in a Phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in Myasthenia Gravis and developing a product candidate in Type 1 Diabetes in addition to having completed first-in-human studies in Celiac Disease (partnered with Takeda Pharmaceuticals), and Primary Biliary Cholangitis. Additionally, COUR is developing an undisclosed pre-clinical stage program in collaboration with Genentech.
For more information, please visit www.courpharma.com.
Contacts
For Investor Relations
Brian Bock, Chief Financial Officer
bbock@courpharma.com
For Media
Jason Braco
jbraco@lifescicomms.com
