FDA Clears Proof-of-concept Study for CNP-106 Immune Therapy
The Phase 1b/2a study will evaluate therapy's safety, preliminary effectiveness
COUR Pharmaceuticals is launching a Phase 1b/2a proof-of-concept study to evaluate the safety and preliminary effectiveness of its investigational therapy CNP-106, an immune-modifying nanoparticle that’s designed to reprogram the immune system in adults with myasthenia gravis (MG).
The announcement follows the U.S. Food and Drug Administration’s (FDA) approval of an investigational new drug (IND) application to begin clinical testing of CNP-106.
“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,” John J. Puisis, founder and CEO of COUR, said in a press release.
MG is an autoimmune disease caused by self-reactive antibodies that target and attack proteins that are part of neuromuscular junctions — the sites of communication between nerve and muscle cells. Immunosuppressive medications are commonly prescribed to help control symptoms, but don’t treat the disease’s root cause. Their long-term use is also linked to side effects.
“Current therapies aimed at treating MG mainly address symptoms, but do not address the underlying driver of the disease,” Richard J. Nowak, MD, an assistant professor of neurology and director of the Program for Clinical & Translational Neuromuscular Research at Yale University, said.
CNP-106 tries to prevent the immune attack on the neuromuscular junctions by reprogramming the immune system to achieve immune tolerance, or the absence of immune responses against the body’s own proteins.
The therapy is one of the lead products of COUR’s platform of immune-modifying nanoparticles that harnesses the power of regulatory T-cells. Also known as Tregs, these immune cells help keep the body’s immune system in check, dampening excessive immune and inflammatory responses.
This principle relies on the immune system’s ability to learn and “tolerate” specific antigens, particles that are capable of triggering an immune response. COUR’s platform loads MG antigens into tiny particles (nanoparticles), which, along with negative costimulating factors, instruct the immune system to recognize them as “self-proteins,” thereby preventing an immune response.
“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,” Nowak said.
In the upcoming trial, which is expected to start early next year, adult MG patients will be randomly assigned to receive CNP-106 or a placebo. Besides assessing safety and preliminary efficacy, the trial also will determine its tolerability and immunogenicity, or ability to provoke an immune response.
“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 said.