COUR Pharma Secures FDA Orphan Drug Designation for CNP-106 in Generalized Myasthenia Gravis
CHICAGO, Oct. 01, 2025 (GLOBE NEWSWIRE) -- COUR Pharma, a clinical-stage biotechnology company developing first-in-class, antigen-specific immune tolerance therapies for autoimmune diseases, announced today the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CNP-106, an investigational therapy for the treatment of generalized myasthenia gravis (gMG).
CNP-106 is a biodegradable nanoparticle that encapsulates seven acetylcholine receptor (AChR) antigens. It is being evaluated for its potential to induce antigen-specific immune tolerance in anti-AChR antibody positive generalized MG, a rare autoimmune disorder, with significant unmet need. The disease is driven by autoimmune responses that impair communication between muscles and nerves leading to serious functional limitations and, in some cases, life-threatening crisis.
“Receiving orphan drug designation for CNP-106 is an important development and regulatory milestone for our myasthenia gravis program,” said Dannielle Appelhans, President and Chief Executive Officer of COUR Pharma. “CNP-106 represents a novel tolerance-based approach aimed at addressing the underlying immune response that cause anti-AChR antibody positive gMG. As enrollment in our clinical trial for CNP-106 in gMG continues to advance, we are looking forward to sharing data readouts from our differentiated tolerance approach.”
COUR Pharma is currently enrolling people aged 18-75 years old who test positive for anti-acetylcholine receptor antibodies and have a Myasthenia Gravis Foundation of America (MGFA) Class II-IV diagnosis of gMG in a Phase 1b/2a double-blind, randomized, placebo-controlled clinical trial (NCT06106672) to evaluate the safety, tolerability, pharmacodynamics, and efficacy of multiple ascending doses of CNP-106.
About Orphan Drug Designation:
The FDA’s Office of Orphan Products Development grants orphan designation to drugs and biologics intended to treat rare diseases affecting fewer than 200,000 people in the United States. Orphan drug designation provides a drug developer with certain benefits and incentives, including a period of marketing exclusivity if regulatory approval is ultimately received for the designated indication.
About CNP-106:
CNP-106 is a biodegradable nanoparticle that encapsulates seven acetylcholine receptor (AChR) antigens. AChR+ generalized MG is a T cell-dependent B cell-mediated autoimmune disease with pathogenic antibodies directed against components of the AChR. In AChR+ generalized myasthenia gravis (gMG), activation of T cells and B cells specific to the AChR results in antibody-related impairment of AChR signalling at the neuromuscular junction impairing muscle contraction. As gMG progresses, it may lead to serious health issues, such as myasthenic crisis, tumors of the thymus gland, thyroid imbalances, and other autoimmune diseases. CNP-106 is designed to potentially induce the downregulation of AChR T cell populations and AChR antibodies, leading to improved muscle function.
About Myasthenia Gravis:
Myasthenia gravis (MG) is a chronic autoimmune disease that disrupts communication between nerves and muscles, leading to muscle weakness that can dramatically impair basic bodily functions, including vision, speech, swallowing, breathing, and mobility. As a result, people with MG often face severe limitations in daily life and, in some cases, life-threatening crises.
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 is enrolling patients in two Phase 1b/2a clinical studies: one in type 1 diabetes and the other in myasthenia gravis. COUR also expects to initiate a Phase 2b clinical study in primary biliary cholangitis, a disease in which COUR has already shown positive results in a Phase 1b/2a clinical study, in 2025. Additionally, COUR has partnered with Takeda Pharmaceuticals for its program in celiac disease, which is currently enrolling in a Phase 2b trial, and is developing an undisclosed preclinical 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
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