NMD Pharma received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to progress the NMD670 inhibitor into a phase 2 clinical study in patients suffering from spinal muscular atrophy (SMA) type 3.

NMD Pharma A/S, a clinical-stage biotech company founded in 2015 and headquartered in Aarhus, Denmark, provides life-transforming therapies for people with neuromuscular diseases. It is the leading company that utilizes ClC-1 chloride ion channel inhibitors for various neuromuscular disorders, and currently, it has one clinical-stage product candidate for the treatment of myasthenia gravis (MG). 

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The phase 2 clinical trial is a double-blind, randomized, placebo-controlled, 2-way crossover study to assess the safety, efficacy, and supportability of NMD670 inhibitors in adult patients experiencing SMA Type 3. The proposed study is an international multicenter study, including sites in Europe and North America. The first dosing of NMD670 inhibitor for the patients is expected to take place in quarter 1 of 2023.

“Receiving IND clearance to start our first clinical trial in SMA patients with NMD670 reflects the progress of NMD Pharma’s clinical development into new indications and geographies and comes only two months after we reported successful Phase 1/2a data on NMD670 in myasthenia gravis,” said Thomas Holm Pedersen, chief executive officer of NMD Pharma.

Spinal muscular atrophy (SMA) is a collection of genetic diseases defined by the progressive destruction of the nerve cells controlling vital skeletal muscle activity, resulting in weakness and fatigue. SMA is caused by the defects in both copies of the survival motor neuron one gene (SMN1) on chromosome 5q. Chromosome 5q-associated SMA is usually classified into types 1 to 4 (0 to 4 sometimes) and depends on the onset’s age. Type 1 (0) SMA is reported to be the most severe form of the disease, where the symptoms appear at birth or in infancy (before birth, type 0).

NMD670 is a high-quality small molecule inhibitor of the muscle-specific chloride ion channel, i.e., the ClC-1 ion channel. NMD pharma has preclinically exhibited that ClC-1 inhibition can increase neuromuscular transmission and restores skeletal muscle function. The proposed novel treatment approach has shown convincing preclinical efficacy data in the animal models of MG, SMA, and other neuromuscular disorders. Moreover, it demonstrated interesting clinical efficacy in Phase 2a clinical proof of mechanism study in MG. Furthermore, NMD670 has been recently granted the orphan-drug designation by the FDA for treating MG.

“SMA is a rare disease, and despite recent treatment advances, there is still a substantial unmet medical need to alleviate weakness and fatigue in these patients,” Dr. Pedersen concluded. 


NMD pharma receives IND clearance to start a phase II clinical trial of NMD670 for the treatment of symptoms of spinal muscular atrophy type 3. NMD Pharm; December 14, 2022.