CMTA-STAR Alliance Partner NMD Pharma A/S has announced the publication of foundational findings in the Annals of Clinical and Translational Neurology. The study highlights neuromuscular junction (NMJ) dysfunction as a characteristic in Charcot-Marie-Tooth disease (CMT) types 1 and 2, identifying its impact on muscle function and disease severity. This research laid the groundwork for NMD Pharma’s SYNAPSE-CMT Phase II clinical trial, which is now actively recruiting participants.
NMD Pharma’s publication, which reports the results from its ESTABLISH-CMT observational study, describes how NMJ dysfunction affects CMT patients regardless of their genotype. According to the company, treatment with the company’s novel CIC-1 inhibitor NMD670 improved muscle function in preclinical models, marking a critical step toward developing therapies for the CMT community.
“This data provides further evidence of the crucial role of neuromuscular junction dysfunction in CMT patients,” Thomas Holm Pedersen, CEO of NMD Pharma A/S. “It is very promising that NMD670 improves muscle contractile function in animal models of CMT, and we have already started evaluating the effect of NMD670 in a Phase 2 clinical trial initiated in November 2024.”
The trial, currently recruiting at multiple sites globally, welcomed its first participant on November 18, 2024, who received their first dose under the direction of Yessar M. Hussain, MD, CMTA Centers of Excellence clinic director at the Austin Neuromuscular Center in Austin, Texas.
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Published on: December 18, 2024