The US Food and Drug Administration (FDA) has cleared an Investigational New Drug (IND) application from COUR Pharmaceuticals for the initiation of a phase 1b/2a clinical trial of the company’s experimental treatment CNP-106 for myasthenia gravis (MG).
“The FDA’s acceptance of our IND application in myasthenia gravis represents COUR’s fourth IND clearance to date and expands our focus deeper into rare and severe autoimmune diseases,” said John J. Puisis, the founder and chief executive officer of COUR, in a press release from the company.
“As we continue to advance our MG program into the clinic, we look forward to demonstrating clear differentiation to existing approved therapies in durability of response and safety without the need of concomitant immunosuppressive therapies, offering a potentially groundbreaking, disease modifying treatment for patients who suffer the debilitating effects of MG,” Puisis continued.
Read more about the etiology of MG
The trial is a double-blind, randomized, placebo-controlled, proof-of-concept study that is expected to start in the first quarter of 2023. It will assess the immunogenicity, tolerability, safety, tolerability, and preliminary efficacy of CNP-106 in adult patients with MG.
CNP-106 is designed to treat the underlying autoimmune causes of MG by inducing antigen-specific tolerance. The experimental treatment “has the potential to halt and reverse MG by reprogramming T cells to promote immune tolerance through T regulatory cells,” said Richard J. Nowak, MD, MS, assistant professor of neurology at Yale University in New Haven, Connecticut.
“For the first time, we have an opportunity to address the likely seminal trigger which leads to neuromuscular junction damage and dysfunction,” he added.
MG is a rare autoimmune disease characterized by the production of autoantibodies against post-synaptic neuromuscular junction proteins. This leads to reduced transmission of electrical impulses across the neuromuscular junction, causing generalized fatigability and muscle weakness.
COUR Pharmaceuticals announces FDA clearance of investigational new drug application for myasthenia gravis. News release. COUR Pharmaceuticals; October 25, 2022.