NMD Pharma Initiates Phase 2 Study of NMD670 in Patients with Charcot-Marie-Tooth Disease Type 1 and 2

NMD Pharma Initiates Phase 2 Study of NMD670 in Patients with Charcot-Marie-Tooth Disease Type 1 and 2

Aarhus, Denmark, 18 November 2024 – NMD Pharma A/S, a clinical-stage biotech company dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, announces that it has dosed the first Charcot-Marie-Tooth (CMT) disease patient in its Phase 2a clinical trial of NMD670 following FDA IND clearance in June 2024. The first patient was dosed under the direction of Yessar Hussain, MD at the Austin Neuromuscular Center in Austin, TX in the US.

NMD670 is a first-in-class small molecule inhibitor of the skeletal muscle specific chloride ion channel 1 (CIC-1). NMD Pharma has demonstrated that CIC-1 inhibition amplifies the muscle’s responsiveness to weak signals, improving neuromuscular transmission and restores skeletal muscle function.

The Phase 2a clinical trial (NCT06482437), named SYNAPSE-CMT, is a randomized, double-blind, placebo-controlled study, evaluating the efficacy, safety, and tolerability of a twice daily oral dose of NMD670 over 21 days in 80 adult patients with any genetically confirmed CMT1 or CMT2 subtype. The study will evaluate changes in the 6-minute walk test (6MWT), time to complete the 10-meter walk/run test (10MW/R) and time to complete timed-up-and-go test (TUG), among other safety and efficacy endpoints over a 21-day study period, taking place across clinical sites in both the US and Europe.

Yessar Hussain, MD, Austin Neurological Center, stated: “Patients with CMT are very excited about entering into this study. This is the only clinical study with a clinically feasible treatment to address the muscle weakness and fatigue with a pharmaceutical therapy at the moment. The fact that this study enrols patients with all types of CMT 1 and 2 is also encouraging and motivating to the community. I am happy to be involved in this exciting clinical study.”

Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, added: “As a highly debilitating disease with no currently approved medicines or a cure, there is an urgent need for new therapeutic approaches to help improve the quality of life of patients with CMT and their families. NMD670 has demonstrated promising clinical results in a proof-of-mechanism study in patients with NMJ dysfunction in the rare neuromuscular disease myasthenia gravis, and we are confident that our novel therapeutic approach has the potential to also provide benefit to those affected by CMT. Our focus is to develop treatments that address the symptoms of CMT across multiple subtypes and provide needed muscle strength, power and endurance that can help patients gain and maintain ability to independently perform daily activities. I look forward to keeping patient communities updated on our progress.”

In June 2023, NMD Pharma announced the results of ESTABLISH¹, an international observational study of neuromuscular transmission deficits and muscle function in CMT types 1 and 2, at the Peripheral Nerve Society Annual Meeting, highlighting neuromuscular junction (NMJ) transmission deficits as an unappreciated disease characteristic in CMT.

With the initiation of this Phase 2 CMT clinical trial, NMD Pharma now has three ongoing global clinical trials investigating NMD670 across rare neuromuscular diseases characterized by a high degree of patient impact and need, including a Phase 2 study in adults living with spinal muscular atrophy (SMA) type 3 and a Phase 2b study in gMG patients.

Further information on the study can be found here: Study Details | Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease | ClinicalTrials.gov

Patients with CMT disease type 1 and type 2 in the US and Europe are encouraged to participate in the study. New clinical trial sites are being announced, starting this month and continuing until March 2025. For further information and a list of currently active clinical trial sites, please look to the ClinicalTrials.gov link about or email us at contact@nmdpharma.com.

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Contacts

NMD Pharma A/S
Thomas Holm Pedersen, CEO
E-mail: contact@nmdpharma.com

ICR Healthcare
Mary-Jane Elliott / Ashley Tapp / Lindsey Neville
E-mail: NMDPharma@icrhealthcare.com
Tel: +44 (0)20        3709 5700

About NMD Pharma
NMD Pharma A/S is a clinical-stage biotech company developing a first-in-class platform of small molecule therapies selectively targeting the skeletal muscle chloride ion channel (ClC-1) for the treatment of rare neuromuscular disorders and age-related neuromuscular diseases with high levels of patient unmet. The Company was founded on more than 15 years of muscle physiology research with a focus on regulation of skeletal muscle excitability under physical activity. NMD Pharma has built a world-leading muscle electrophysiology platform leveraging the in-depth know-how of muscle physiology and muscular disorders, small molecule modulators, enabling technologies and tools as well as in vivo pharmacology models for discovering and developing proprietary modulators of neuromuscular function. The Company has built significant clinical and development expertise as its programmes have progressed through the clinic. NMD Pharma has raised ~€155 million from investors including Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, Roche Venture Fund, and Jeito Capital. Find out more about us online at http://www.nmdpharma.com.

About NMD670
NMD670 is NMD Pharma’s lead development program. It is a first-in-class small molecule inhibitor of the skeletal muscle specific chloride ion channel 1 (CIC-1). NMD Pharma has demonstrated that CIC-1 inhibition amplifies the muscle’s responsiveness to weak signals, improving neuromuscular transmission and restores skeletal muscle function. This novel treatment approach has resulted in compelling preclinical and clinical data generalized myasthenia gravis, spinal muscular atrophy, Charcot-Marie Tooth disease, sarcopenia and holds potential in a range of other neuromuscular disorders. NMD670 has also been granted orphan-drug designation by the U.S. FDA for treatment of gMG.

About Charcot-Marie-Tooth disease (CMT)
CMT encompasses a group of hereditary sensory and motor neuropathies that cause damage to peripheral nerves and their neuromuscular junction. Damage caused by CMT worsens slowly over time and can result in substantial reductions in mobility, independence and quality of life due to muscle weakness, fatigue and atrophy across  skeletal muscle groups such as the legs, feet, arms, hands and potentially leading to diaphragm weakness and paralysis. CMT is often broadly grouped into demyelinating (CMT type 1) and axonal (CMT type 2) based on nerve conduction studies, but there are more than 160 subtypes of CMT based on their genetic causes, clinical features and progression patterns. CMT affects approximately 136,000 individuals in the United States and 3.2 million worldwide, with first symptoms typically appearing during adolescence or early adulthood.