Nanoparticle Therapy has Potential to Reprogram the Immune System in Type 1 Diabetes

COUR Pharmaceuticals has received the okay from the FDA to begin clinical trials nanoparticle-based therapy that has the potential to address the underlying autoimmunity of type 1 diabetes, which affects about 2 million Americans. Type 1 diabetes is caused by the immune’s response to pancreatic beta cells.

The phase 1b/2a study will begin later this year to assess the safety of COUR’s CNP-103 in adults (aged 18-35) and adolescents (aged 12-17) who have Stage III or newly diagnosed (within the last 6 months) type 1 diabetes. The study will enroll 72 patients and is expected to be completed in 2027.

Dannielle Appelhans

“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,” Dannielle Appelhans, president and CEO of COUR, said in a news release. “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.”

COUR’s technology uses biodegradable nanoparticles to encapsulate four recombinant proteins known to promote islet cell destruction: preproinsulin, GAD65, IGRP, ZnT8. CNP-103 is thought to induce tolerance to these proteins and prevent islet cell destruction, allowing for maintenance of insulin production.

The nanoparticles bind to immune cells called monocytes. These cells then travel to the spleen and liver, where they undergo apoptosis. The proteins are released and are consumed by antigen presenting cells, which produces a T cell response. Data from a study in mouse model of type 1 diabetes was published in The Journal of Immunology in August 2022.

COUR has researching other potential therapies using this technology. The company currently enrolling patients in a phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in myasthenia gravis, a rare autoimmune neuromuscular disease that weakens skeletal muscles by interrupting the communication between motor neurons and muscle fibers that is required to trigger muscle contraction.

The company also has released phase 2a data for a therapy to treat patients with primary biliary cholangitis, a chronic autoimmune disease of the liver that primarily affects women. In this trial, CNP-104 demonstrated potential efficacy across multiple immunological and clinical measurements.

CNP-104 led to a slowing of disease progression in liver stiffness on Fibroscan, reaching a statistically significant decrease on day 120 in the active arms compared with placebo. It also demonstrated a favorable T cell response in pathogenic CD4 T cell populations and tolerance inducing CD8 T cells. Drug related adverse events were mild, and there were no drug related severe adverse events.

Additionally, COUR has partnered with Takeda Pharmaceuticals for its program in celiac disease, which is currently enrolling in a phase 2b trial.