COUR Pharmaceuticals Gains FDA Approval for IND Application of CNP-103 in Type 1 Diabetes

CHICAGO, IL, USA, February 04, 2025 – COUR Pharmaceuticals, a biotechnology company focused on developing pioneering therapies for immune-mediated diseases, has received approval from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for CNP-103. This innovative nanoparticle is being developed to target the root cause of autoimmunity in Type 1 diabetes (T1D).

Dannielle Appelhans, President and CEO of COUR, described the FDA's clearance as a significant milestone for the company. CNP-103 is the third product developed through COUR's nanoparticle platform, which aims to achieve antigen-specific immune tolerance, and it is set to proceed to clinical trials. Preclinical studies have shown that CNP-103 can halt the progression of T1D, foster a pro-regulatory environment, and minimize inflammatory cell activity. The preclinical results also suggest the potential for the preservation of β-cell function and the reversal of dysglycemia. COUR plans to launch a Phase 1b/2a trial with CNP-103 later this year.

The planned Phase 1b/2a clinical trial will evaluate the safety of CNP-103 in adults aged 18-35 and children aged 12-17 who have either Stage III or have been newly diagnosed with T1D within the past six months, provided their C-peptide levels are greater than 0.2 ng/mL.

CNP-103 is designed as a biodegradable nanoparticle incorporating four recombinant proteins linked to islet cell destruction, namely preproinsulin, GAD65, IGRP, and ZnT8. These proteins represent over 95% of the known antigens triggering Type 1 diabetes. By promoting tolerance to these proteins, COUR aims to prevent the destruction of islet cells by harmful CD4+ and CD8+ T cells. This approach aims to sustain insulin production and potentially reverse dysglycemia.

Type 1 diabetes is a progressive condition affecting more than 1.5 million people in the United States. It results from autoreactive pathogenic CD4+ and CD8+ T cells attacking insulin-producing β-cells in the pancreas, leading to their destruction. The continuous loss of β-cells causes insulin deficiency, impairing glucose regulation. Individuals with T1D experience symptoms such as frequent urination, excessive thirst, weight loss, fatigue, and may face serious complications like diabetic ketoacidosis. The broader health implications of T1D can include heart disease, kidney failure, stroke, and vision loss.

COUR Pharmaceuticals is a clinical-stage biotechnology firm dedicated to creating treatments for autoimmune diseases. The company's therapies are grounded in a proprietary platform focused on antigen-specific immune tolerance, intending to reprogram the immune system to address the fundamental causes of immune-mediated diseases. Data from several clinical and preclinical initiatives have shown COUR's products can induce antigen-specific immune tolerance, with the potential to treat a variety of autoimmune disorders.

Currently, COUR is enrolling patients in a Phase 1b/2a clinical study for Myasthenia Gravis (MG) and anticipates starting two trials in 2025: a Phase 1b/2a for Type 1 Diabetes and a Phase 2b for Primary Biliary Cholangitis (PBC), the latter of which has already shown positive outcomes in a Phase 1b/2a study. COUR is also collaborating with Takeda Pharmaceuticals on a program for Celiac Disease, now in a Phase 2b trial, and is working on a preclinical program with Genentech.