NMD Pharma reports the Charcot-Marie-Tooth results

NMD Pharma A/S, a clinical-stage biotech company that developed a first-in-class small molecule ClC-1 inhibitor for neuromuscular disorders, has presented results from ESTABLISH1, an observational study of neuromuscular function in patients with the Charcot-Marie-Gigi inherited neurological condition ( CMT) types 1 and 2.

The results were revealed in a poster presentation at the Peripheral Nerve Society Annual Meeting (PNS) in Copenhagen, Denmark.

The results of this study establish, for the first time, neuromuscular junction (NMJ) transmission deficit as a novel disease characteristic in patients with CMT types 1 and 2. The degree of NMJ transmission deficit in patients with CMT is associated with disease severity as assessed by various clinical measures of strength and muscle function.

The study also provides information on the tolerability and reliability of clinical and electrophysiological outcomes that the company says will assist the selection of relevant outcomes in future clinical trials.

This study compared electrophysiological assessments (single-fiber electromyography (EMG) and repetitive nerve stimulation) and clinical trials (involving tests of muscle strength, fatigue, agility, and balance) in 21 patients with CMT types 1 and 2, and 10 patients of healthy age. subjects. The international study was led by Henning Andersen, Aarhus University Hospital, and William David Arnold, NextGen Precision Health, University of Missouri.

Andersen, Department of Neurology at Aarhus University Hospital, said: “CMT is a very debilitating disease with no approved treatments to cure or relieve symptoms in patients. Here we show that patients with CMT are characterized by a deficit of the neuromuscular junction to transmit signals effectively from nerves to muscles and that the degree of transmission failure correlates with the functional abilities of the patients. This opens the door for targeting transmission failure pharmacology to improve symptoms in patients with CMT.”

Arnold, executive director at NextGen Precision Health, University of Missouri added: “We are pleased to report identifying aspects of the disease that may represent a breakthrough in the identification of new treatments for CMT.”

Jorge A. Quiroz, chief medical officer of NMD Pharma, said: “With no currently approved treatment or therapy for this debilitating disease, uncovering failure of NMJ transmission as a novel disease mechanism in CMT is an important milestone for the development of new therapies including our approach. new ClC-1 inhibitor treatment. I want to thank the Aarhus and Ohio teams for their tremendous contribution to the success of this study and, most importantly, the patients and families who participated in the Establish study.”

NMD Pharma plans to publish the full set of study results in a peer-reviewed journal.

NMD Pharma is developing NMD670, a first-in-class small molecule inhibitor for the muscle-specific chloride ion channel, the ClC-1 ion channel. NMD Pharma pre-clinically demonstrated that ClC-1 inhibition can improve neuromuscular transmission and, ultimately, skeletal muscle function and NMD670 has shown positive results in phase I and evidence of mechanism studies in patients with myasthenia gravis confirming safety, tolerability, and early onset. efficacy data in subjects suffering from neuromuscular disorders.

In December, NMD Pharma was cleared for a spinal muscular atrophy trial. In October, he published positive results from a myasthenia gravis study.