FDA Approves NMD Pharma's Phase 2 Trial of NMD670 for Charcot-Marie-Tooth Disease

June 18, 2024 – NMD Pharma, an innovative biotech firm specializing in treatments for severe neuromuscular diseases, has secured approval from the US Food and Drug Administration (FDA) to commence a Phase 2 clinical trial, dubbed SYNAPSE-CMT, for its drug candidate NMD670 in patients with Charcot-Marie-Tooth (CMT) disease types 1 and 2. NMD670 is a pioneering small molecule inhibitor targeting the ClC-1 chloride ion channel in skeletal muscles.

The upcoming Phase 2 trial will be a randomized, double-blind, placebo-controlled study. It aims to assess the efficacy, safety, and tolerability of NMD670, administered orally twice daily over a 21-day period. Approximately 80 adult patients with genetically confirmed CMT1 or CMT2 will participate in the study, which will evaluate various clinical endpoints such as the 6-minute walk test, the 10-meter walk/run test, and the timed-up-and-go test. The trial sites will be located in both the US and Europe, with patient enrollment expected to begin shortly.

In June 2023, NMD Pharma unveiled findings from the ESTABLISH study, an international observational study on neuromuscular junction function in CMT types 1 and 2, presented at the Peripheral Nerve Society Annual Meeting. The study indicated that dysfunction at the neuromuscular junction is a significant yet underrecognized characteristic in CMT patients, with varying degrees of signal transmission deficits correlating with disease severity and motor function.

Thomas Holm Pedersen, CEO of NMD Pharma, stated, "Charcot-Marie-Tooth disease is a profoundly debilitating condition with no existing treatments. The ESTABLISH study's insights into neuromuscular junction deficits give us confidence that NMD670 could offer meaningful benefits by improving skeletal muscle function in CMT patients."

Allison Moore, CEO of the Hereditary Neuropathy Foundation (HNF), commented, "NMD Pharma's focus on addressing symptoms across multiple CMT subtypes is crucial for improving patient outcomes. Their efforts bring hope and potential relief to those grappling with this challenging condition."

Cleary Simpson, CEO of the CMT Research Foundation (CMTRF), added, "The initiation of Phase 2 trials for NMD670 marks an exciting development for the CMT community, promising potential improvements in muscle function for affected individuals."

Sue Bruhn, CEO of the Charcot-Marie-Tooth Association (CMTA), expressed, "We are proud to partner with NMD Pharma. Their innovative approach aligns with our mission to find effective treatments for CMT, and FDA clearance for the NMD670 Phase 2 trial is a pivotal step forward."

By the end of 2024, NMD Pharma aims to have three ongoing Phase 2 trials utilizing its skeletal muscle activation therapy for rare neuromuscular diseases. These include studies on spinal muscular atrophy (SMA) type 3, generalized myasthenia gravis (gMG), and now, Charcot-Marie-Tooth disease types 1 and 2.

About NMD Pharma
NMD Pharma A/S is committed to developing small molecule therapies targeting the ClC-1 chloride ion channel in skeletal muscles to treat severe neuromuscular disorders. Founded on extensive research in muscle physiology, NMD Pharma has created a leading platform for discovering and developing modulators of neuromuscular function. The company has advanced its programs through clinical development and secured significant investment from notable firms, including Novo Holdings and the Roche Venture Fund.

About NMD670
NMD670, the leading program of NMD Pharma, is a first-in-class small molecule inhibitor of the ClC-1 chloride ion channel. It has demonstrated potential in enhancing neuromuscular transmission and restoring muscle function, showing promising results in preclinical and clinical studies for conditions like myasthenia gravis and spinal muscular atrophy.

About Charcot-Marie-Tooth Disease (CMT)
CMT encompasses hereditary neuropathies that damage peripheral nerves, leading to muscle weakness, fatigue, and atrophy. With over 160 subtypes, CMT affects approximately 136,000 individuals in the US and 3.2 million globally, typically manifesting symptoms in adolescence or early adulthood.

NMD Pharma's advancement to Phase 2 clinical trials for NMD670 signifies a significant step toward addressing the unmet needs of CMT patients, offering hope for improved treatments and quality of life.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!