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NMD Pharma A/S has obtained clearance for its investigational new drug (IND) utility from the US Meals and Drug Administration (FDA) to advance NMD670 right into a part II medical examine in sufferers of spinal muscular atrophy (SMA) sort 3. 

NMD Pharma is a Danish medical stage biotech firm growing first-in-class, small molecule ClC-1 inhibitors for neuromuscular issues.

The medical trial is a randomized, double-blind, placebo-controlled, two-way crossover examine to guage the efficacy, security, and tolerability of NMD670 in ambulatory grownup sufferers with SMA sort 3. The examine is a global multicenter examine and can embody websites in North America and Europe, with first dosing of sufferers anticipated to happen in Q1 2023.

NMD670 is a first-in-class small molecule inhibitor of the muscle particular chloride ion channel, the ClC-1 ion channel. NMD Pharma has pre-clinically demonstrated that ClC-1 inhibition can improve neuromuscular transmission and in the end skeletal muscle operate. In October, NMD introduced constructive topline knowledge from NMD670 single dose in a part I/IIa examine in sufferers with myasthenia gravis (MG) confirming security, tolerability and preliminary efficacy knowledge in topics affected by neuromuscular issues. 

Primarily based on these preclinical and medical knowledge, it’s anticipated that this novel method may very well be helpful within the therapy of sufferers affected by SMA.

Thomas Holm Pedersen, chief govt officer of NMD Pharma, mentioned: “Receiving IND clearance to start out our first medical trial in SMA sufferers with NMD670 displays the progress of NMD Pharma’s medical improvement into new indications and geographies and comes solely two months after we reported profitable part I/IIa knowledge on NMD670 in myasthenia gravis. SMA is a uncommon illness and regardless of latest therapy advances, there may be nonetheless a considerable unmet medical must alleviate weak point and fatigue in these sufferers.”

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