ZNT8

CHICAGO, IL, USA I February 04, 2025 I COUR Pharmaceuticals, a clinical-stage biotechnology company developing first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for CNP-103, a nanoparticle in development to address the underlying autoimmunity of Type 1 diabetes (T1D). “Clearance of the IND application for CNP-103 is a notable moment for COUR, as CNP-103 will be our third proprietary autoimmune disease program developed using our nanoparticle platform for antigen-specific immune tolerance to enter the clinic,” said Dannielle Appelhans, President and Chief Executive Officer of COUR. “In preclinical studies to date, CNP-103 has demonstrated the ability to stop T1D disease progression while creating an enhanced pro-regulatory environment with reduced inflammatory cell activity. These findings lead us to believe that CNP-103 could potentially preserve β-cell function and reverse dysglycemia, which are results that have also been observed in preclinical models. We expect to initiate a Phase 1b/2a first-in-human trial of CNP-103 later this year.” The Phase 1b/2a study will assess the safety of CNP-103 in adults (aged 18-35) and pediatrics (aged 12-17) who have Stage III or newly diagnosed (within the last 6 months) T1D as well as C-peptide >0.2 ng/mL. About CNP-103: CNP-103 is a biodegradable nanoparticle encapsulating four recombinant proteins known to promote islet cell destruction: preproinsulin, GAD65, IGRP, ZnT8. Notably, these proteins cover >95% of known antigens driving Type 1 diabetes. By inducing tolerance to these proteins, COUR aims to prevent islet cell destruction by pathogenic CD4+ and CD8+ T cells, allowing for maintenance of insulin production and potential reversal of dysglycemia. About T1D: T1D is a progressive disorder impacting >1.5 million individuals in the U.S. and is caused by autoreactive pathogenic CD4+ and CD8+ T cells targeting insulin-producing β-cells in the islets of Langerhans, causing destruction. Progressive β-cell loss results in insulin deficiency and inability to regulate glucose. People who live with T1D experience symptoms that include acutely frequent urination, excessive thirst, weight loss, fatigue, and potentially life-threatening diabetic ketoacidosis. Broader health impacts of T1D include heart disease, kidney failure, stroke, and vision loss. 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 enrolling patients in a Phase 1b/2a clinical study in Myasthenia Gravis (MG) and expects to initiate two trials in 2025: a Phase 1b/2a in Type 1 Diabetes and a Phase 2b in Primary Biliary Cholangitis (PBC), a disease in which COUR has already shown positive results in a Phase 1b/2a study. 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 . SOURCE: COUR Pharmaceuticals