COUR Pharma has achieved a significant milestone in type 1 diabetes research by dosing the first patient in a Phase 1b/2a clinical trial evaluating CNP-103, a novel tolerogenic nanoparticle therapy designed to address the underlying autoimmunity associated with type 1 diabetes (T1D). The announcement marks a potential breakthrough in treating a condition that affects more than 1.5 million individuals in the United States.
Novel Approach to Autoimmune Intervention
CNP-103 represents a first-in-class approach to type 1 diabetes treatment, moving beyond symptom management to directly target the autoimmune processes driving the disease. "The initiation of this trial is important for patients, as we believe our approach to antigen-specific immune tolerance offers the potential to surpass symptom management and instead directly address the underlying autoimmunity present in people with T1D," said Dannielle Appelhans, President and Chief Executive Officer of COUR.
The therapy consists of biodegradable nanoparticles encapsulating four recombinant proteins: preproinsulin, GAD65, IGRP, and ZnT8. These proteins collectively cover more than 95% of known antigens driving type 1 diabetes. By inducing tolerance to these proteins, COUR aims to prevent further islet cell destruction by pathogenic CD4+ and CD8+ T cells, maintain insulin production at clinically important levels, and potentially reverse dysglycemia.
Targeting an Unmet Medical Need
The Phase 1b/2a study (NCT06783309) specifically focuses on stage 3 type 1 diabetes patients, representing a critical gap in current treatment options. "While CNP-103 has potential across stages 1 through 3 in T1D and potentially in later-stage patients in combination with islet cell transplants, our initial focus is on stage 3, for which there are no FDA-approved disease-modifying therapies to combat autoimmune β-cell destruction," Appelhans explained.
The trial will assess the safety of CNP-103 in adults aged 18-35 and pediatric patients aged 12-17 who have stage 3 newly diagnosed type 1 diabetes within the last six months and C-peptide levels greater than 0.2 nmol/L. Beyond safety evaluation, the study will examine CNP-103's ability to preserve islet cell function through the maintenance of C-peptide levels.
Understanding Type 1 Diabetes Pathophysiology
Type 1 diabetes is a progressive autoimmune disorder caused by autoreactive pathogenic CD4+ and CD8+ T cells that target insulin-producing β-cells in the islets of Langerhans. This autoimmune attack leads to progressive β-cell loss, resulting in insulin deficiency and inability to regulate glucose levels effectively.
Patients with type 1 diabetes experience symptoms including frequent urination, excessive thirst, weight loss, fatigue, and potentially life-threatening diabetic ketoacidosis. The broader health impacts extend to cardiovascular complications, kidney failure, stroke, and vision loss, highlighting the urgent need for disease-modifying interventions.
Expanding Pipeline and Strategic Partnerships
COUR Pharma's approach extends beyond type 1 diabetes, with the company currently enrolling patients in two Phase 1b/2a clinical studies: one in myasthenia gravis and the current type 1 diabetes trial. The company expects to initiate a Phase 2b study in primary biliary cholangitis in 2025, building on positive results from a previous Phase 1b/2a study in this indication.
Strategic collaborations further strengthen COUR's pipeline, including a partnership with Takeda Pharmaceuticals for a celiac disease program currently enrolling in a Phase 2b trial, and an undisclosed preclinical program in development with Genentech. These partnerships demonstrate industry confidence in COUR's proprietary antigen-specific immune tolerance platform.
The company's therapeutic approach is designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases, with data from multiple clinical and preclinical programs demonstrating the ability to induce antigen-specific immune tolerance across a wide range of autoimmune conditions.
