NMD Pharma announced that they have published new clinical and preclinical data that provides evidence that targeting neuromuscular junction deficits could potentially improve muscle function in CMT patients.

The peer-reviewed paper titled “Neuromuscular Transmission Deficits in patients with CMT and ClC-1 Inhibition in CMT animal Models” describes the data from ESTABLISH, an observational clinical study in CMT Type 1 and 2 patients and healthy patients, which revealed that CMT patients have neuromuscular junction transmission dysfunction that correlates with disease severity. Additionally, preclinical studies demonstrated that treatment with NMD Pharma’s drug candidate, NMD670, corrected neuromuscular junction deficits and led to improved muscle contractile function in two different mouse models of CMT. NMD670 is a first-in-class small molecule inhibitor of ClC-1, a skeletal muscle specific chloride ion channel.

These findings offer encouraging evidence that targeting ClC-1 using NMD670 could be an effective therapeutic option for improving muscle function in CMT patients. NMD Pharma has an ongoing Phase 2a clinical trial testing the effectiveness of NMD670 in patients with CMT Type 1 and Type 2. NMD Pharma announced that they dosed the first CMT patient with NMD670 in November 2024.

“We are encouraged by these published results from NMD Pharma, which demonstrate that neuromuscular junction dysfunction is a significant disease characteristic in CMT patients and that targeting neuromuscular junction transmission with NMD Pharma’s drug candidate, NMD670, can restore muscle function in CMT mouse models,” said Dr. Riann Egusquiza, CMTRF Director of Research. “Together, these findings highlight NMD670 as a promising therapeutic option for CMT patients. We eagerly await the results of their ongoing clinical trial evaluating the effectiveness of NMD670 in CMT patients.”

To date, CMTRF has not funded this project, but is very pleased by its success. CMTRF Scientific Advisory Board Member Dr. William David Arnold and his team collaborated with NMD Pharma in co-authoring this paper.

Read the full release here.