Charcot-Marie-Tooth Association (CMTA) Alliance Partner, NMD Pharma, announced the publication of comprehensive data sets demonstrating for the first time the ability of CIC-1 inhibition to restore muscle function in patients suffering from neuromuscular junction (NMJ) transmission deficits. These data are published in the journal Science Translational Medicine. While the study was done in Myasthenia Gravis (MG), the data has important implications for CMT.

ClC-1 is a skeletal muscle specific chloride ion channel that plays important roles for regulating neuromuscular transmission and muscle fiber excitability during exercise. According to the company, its compound, NMD670, is a ClC-1 inhibiting small molecule that targets muscle to improve strength and endurance.

What Does This Mean for CMT?

NMD Pharma previously found through a study of patients who had CMT1 or CMT2—demyelinating or axonal, respectively, that neuromuscular junction (NMJ) transmission deficits affect some who have CMT and that these deficits correlate with disease severity.

Building on the reported success of NMD670 to improve disease symptoms associated with NMJ transmission deficits seen in myasthenia gravis (MG), NMD Pharma is planning future Phase 2 clinical trials in CMT.

“This paper summarizes years of work here at NMD Pharma and Aarhus University, firstly establishing neuromuscular transmission deficits in patients impacted by myasthenia gravis and then demonstrating that ClC-1 inhibition can reverse these deficits and improve the muscle function both in animal models and humans,” said Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma. “These data provide important proof-of-mechanism as we progress our Phase 2 trial in spinal muscular atrophy and plan for the initiation of two other Phase 2 trials in AChR and MuSK antibody positive myasthenia gravis and Charcot-Marie-Tooth disease this year.”

“As we witness groundbreaking advancements in the treatment of myasthenia gravis, we’re filled with hope for the potential translational impact on CMT,” said Katherine Forsey, Ph.D., CMTA chief research officer. “NMD Pharma’s pioneering research not only sheds light on novel therapeutic avenues but also underscores the interconnectedness of neuromuscular disorders. We eagerly anticipate the potential benefits this research may bring to individuals living with CMT, marking a significant step forward in the CMTA-STAR mission to accelerate research to bring treatments to CMT.”

This news from our Alliance Partner suggests their experimental compound, NMD670, has the potential to improve symptoms and overall quality-of-life for our community members who have CMT whether demyelinating or axonal. The CMTA continues to follow the progress of NMD Pharma’s NMD670 study with great interest and we support our Alliance Partner as they plan a clinical trial in CMT.

Register With Patients as Partners in Research Today

Not yet registered with Patients as Partners in Research? Click the button below to register today! Registering today ensures you are the first in line when NMD is ready to start enrolling community members in its planned CMT study. Registering also will ensure you are kept up to date on all the latest news and developments in CMT research. Registration is free, secure, and all who have CMT are invited to join.

Patients as Partners Registration

Published: March 26, 2024