NMD Pharma cleared for spinal muscular atrophy trial

spinal muscular atrophy

NMD Pharma A/S has received clearance for its investigational new drug (IND) application from the US Food and Drug Administration (FDA) to advance NMD670 into a phase II clinical study in patients of spinal muscular atrophy (SMA) type 3. 

NMD Pharma is a Danish clinical stage biotech company developing first-in-class, small molecule ClC-1 inhibitors for neuromuscular disorders.

The clinical trial is a randomized, double-blind, placebo-controlled, two-way crossover study to evaluate the efficacy, safety, and tolerability of NMD670 in ambulatory adult patients with SMA type 3. The study is an international multicenter study and will include sites in North America and Europe, with first dosing of patients expected to take place in Q1 2023.

NMD670 is a first-in-class small molecule inhibitor of the muscle specific chloride ion channel, the ClC-1 ion channel. NMD Pharma has pre-clinically demonstrated that ClC-1 inhibition can enhance neuromuscular transmission and ultimately skeletal muscle function. In October, NMD announced positive topline data from NMD670 single dose in a phase I/IIa study in patients with myasthenia gravis (MG) confirming safety, tolerability and initial efficacy data in subjects suffering from neuromuscular disorders. 

Based on these preclinical and clinical data, it is expected that this novel approach could be beneficial in the treatment of patients suffering from SMA.

Thomas Holm Pedersen, chief executive officer of NMD Pharma, said: “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 I/IIa data on NMD670 in myasthenia gravis. 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.”

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