COUR Pharmaceuticals, a biotechnology company developing novel immune-modifying nanoparticles (CNPs) to treat immune disorders, today announced the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to the Company’s investigational therapy CNP-104 for the treatment of Primary Biliary Cholangitis. CNP-104 aims to reprogram the immune system to address autoimmune causes of PBC.
“We are pleased to have received Fast Track Designation for CNP-104, which demonstrates the FDA recognizes the importance of this potential treatment for PBC and the need for improved of clinical outcomes for patients with this degenerative autoimmune disease, which currently has no treatment to stop progression of disease,” said COUR CEO John Puisis. “Based on data from another COUR autoimmune trial with a CNP platform product, our Phase 2 PBC trial is designed to show that we can stop progression of PBC disease with improved liver function. If successful in Phase 2, COUR would seek breakthrough therapy designation in PBC given that current therapies treat only symptoms.”
Fast Track Designation is an FDA process designed to facilitate the development and review of potential therapies that seek to fill an unmet medical need. The FDA created this designation to help get new therapies to patients earlier. A therapy that receives this designation is eligible for more frequent communication with the FDA and may be eligible for Accelerated Approval and Priority review, if relevant criteria are met.
The FDA accepted COUR’s investigational new drug application (IND) for CNP-201 in October 2021 and COUR is currently opening the first human dosing in a proof-of-concept (Phase 2) study for CNP-104 (NCT05104853) in early 2022 to evaluate the safety, tolerability, pharmacodynamics and efficacy of the therapy in PBC patients. CNP-104 uses COUR’s proprietary nanoparticle platform (CNP), a novel system which combines disease specific pathogenic antigens with state-of-the-art pharmaceutical nanoparticles that mimic normal removal of dead or dying liver cells from the body.
In November 2021, Ironwood Pharmaceuticals, Inc., a GI-focused healthcare company entered into a collaboration and license option agreement with COUR for the development and commercialization of CNP-104. This agreement gives Ironwood an option to acquire an exclusive license to develop and commercialize, in the U.S., COUR’s investigational therapy CNP-104 for PBC.
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 is designed to treat the root cause of immune disease, unlike traditional approaches, which only minimize symptoms using toxic immune suppression