Pharma Focus America

COUR Pharmaceuticals Announces FDA Clearance of Investigational New Drug Application for Myasthenia Gravis

Wednesday, October 26, 2022

COUR Pharmaceuticals, a clinical-stage biotechnology company developing novel immune-modifying nanoparticles designed to reprogram the immune system for the treatment of autoimmune disorders (COUR NanoParticles or CNPs), today announced that the U.S. Food and Drug Administration has cleared an Investigational New Drug (IND) application to initiate a Phase 1b/2a proof-of-concept study of COUR's investigational therapy, CNP-106. The therapy is designed to treat the underlying autoimmune causes of myasthenia gravis (MG) through antigen-specific tolerance induced by CNP-106.

"Current therapies aimed at treating MG mainly address symptoms, but do not address the underlying driver of the disease," said Richard J. Nowak, M.D., M.S., Assistant Professor of Neurology and Director of the Program for Clinical & Translational Neuromuscular Research at Yale University. "CNP-106 has the potential to halt and reverse MG by reprogramming T cells to promote immune tolerance through T regulatory cells. For the first time, we have an opportunity to address the likely seminal trigger which leads to neuromuscular junction damage and dysfunction."

MG is 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. These muscles are responsible for breathing and moving parts of the body such as the face, throat, arms and legs. According to the National Institute of Neurological Disorders and Stroke, roughly 15 to 20 percent of MG patients experience at least one myasthenia crisis that occurs when their breathing muscles are weakened to the point that a ventilator is required and may be life-threatening.

"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, Founder & CEO of COUR. "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."

The Phase 1b/2a double-blind, randomized, placebo-controlled, proof-of-concept study is expected to begin in the first quarter of 2023 and will assess the safety, tolerability, immunogenicity and preliminary efficacy of CNP-106 in a representative sample of adults suffering from MG.

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