Danish biotechnology company NMD Pharma has dosed the first patient in a Phase II clinical trial of NMD670 to treat spinal muscular atrophy (SMA).
The multicentre, double-blind study is being carried out at various sites across North America, Europe and other continents.
It is designed to assess the safety, tolerability and efficacy of the ClC-1 inhibitor NMD670 in ambulatory adult patients with SMA Type III.
NMD Pharma CEO Thomas Holm Pedersen said: “We are very pleased to announce the dosing of the first patient in this Phase II study.
“This represents a significant milestone for NMD Pharma as we further expand our pipeline into new indications, and I look forward to keeping the patient communities and market updated as we progress.”
Patients in the trial will be given NMD670 orally twice a day for 21 days.
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The study’s primary outcome measure is the change from baseline in the distance walked in six minutes against placebo.
NMD670 previously demonstrated improvements in neuromuscular transmission and skeletal muscle function in several animal models of neuromuscular diseases.
NMD Pharma executive vice-president and chief medical officer Jorge Quiroz said: “SMA is a rare neuromuscular disorder characterised by severe muscle weakness and fatigue that greatly affects the quality of life of patients and their families.
“NMD670 has already been shown to be safe and efficacious in a proof of mechanism study in patients with myasthenia gravis and we are confident that it could also be beneficial for the treatment of patients affected by SMA.”
Earlier this year, NMD Pharma initiated a Phase I clinical trial of its ClC-1 inhibitor NMD1343 in healthy subjects to treat rare neuromuscular diseases in the UK.
The study assessed NMD1343’s safety, tolerability and pharmacokinetics in patients who were given either a single dose of the drug or a matching placebo.