NMD Pharma A/S has acquired clearance for its investigational new drug (IND) software from the US Meals and Drug Administration (FDA) to advance NMD670 right into a part II medical research in sufferers of spinal muscular atrophy (SMA) kind 3.
NMD Pharma is a Danish medical stage biotech firm growing first-in-class, small molecule ClC-1 inhibitors for neuromuscular problems.
The medical trial is a randomized, double-blind, placebo-controlled, two-way crossover research to guage the efficacy, security, and tolerability of NMD670 in ambulatory grownup sufferers with SMA kind 3. The research is a global multicenter research 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 finally skeletal muscle perform. In October, NMD introduced constructive topline information from NMD670 single dose in a part I/IIa research in sufferers with myasthenia gravis (MG) confirming security, tolerability and preliminary efficacy information in topics affected by neuromuscular problems.
Based mostly on these preclinical and medical information, it’s anticipated that this novel method could possibly be helpful within the remedy of sufferers affected by SMA.
Thomas Holm Pedersen, chief govt officer of NMD Pharma, stated: “Receiving IND clearance to begin our first medical trial in SMA sufferers with NMD670 displays the progress of NMD Pharma’s medical growth into new indications and geographies and comes solely two months after we reported profitable part I/IIa information on NMD670 in myasthenia gravis. SMA is a uncommon illness and regardless of latest remedy advances, there may be nonetheless a considerable unmet medical must alleviate weak spot and fatigue in these sufferers.”