NMD Pharma begins phase 2 trial for spinal muscular atrophy treatment

Betsy Goodfellow | September 27, 2023 | News story | Research and Development Musculo-skeletal disorder, NMD Pharma, clinical trial, spinal muscular atrophy 

NMD Pharma has announced that the first patient has been dosed in its phase 2 clinical trial for its CIC-1 inhibitor NMD670 in patients with spinal muscular atrophy (SMA).

The phase 2 trial is randomised, double-blind and placebo-controlled and aims to assess the efficacy, safety and tolerability of twice-daily oral dosing of NMD670 for 21 days in ambulatory patients with SMA Type 3.

The drug is a first-in-class small molecule inhibitor of the muscle-specific chloride, the CIC-1 ion channel. It has already demonstrated positive results in a phase 1/2 study in patients with myasthenia gravis (MG).

Jorge A Quiroz, EVP and chief medical officer of NMD Pharma, commented: “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 MG and we are confident that it could also be beneficial for the treatment of patients affected by SMA.”

Thomas Holm Pedersen, CEO of NMD Pharma, added: “Despite great advances in the therapeutic armamentarium of therapies to treat SMA, patients can still experience muscle weakness and impaired mobility. This represents a significant unmet need for new interventions that can help patients independently perform daily activities and better manage the symptoms of SMA. We are very pleased to announce the dosing of the first patient in this phase 2 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.”

Betsy Goodfellow

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