Ignaseclant (NMD670) is an inhibitor of skeletal muscle-specific chloride channel protein 1 (ClC-1) that amplifies the skeletal muscle’s responsiveness to weak signals. Ignaseclant is being developed for the treatment of neuromuscular diseases, including myasthenia gravis (MG), in which neuromuscular junction transmission is impaired. Despite available treatments, MG patients continue to experience muscle weakness and fatigue.
In a published proof-of-mechanism study in 12 MG patients with mild symptoms, a single-dose administration of NMD670 led to significant and clinically meaningful improvements in muscle strength as assessed with the Quantitative MG (QMG) score. NMD670 was shown to be safe and well tolerated thus supporting further clinical development for this indication.
Therefore, a dose-finding study in AchR and MuSK positive adult patients with MG was initiated in late 2024. The aim of this study is to evaluate the efficacy and safety of three dose levels of NMD670, administered twice daily for 21 days compared with placebo. Eligible participants are adult male and female aged 18-75 with generalized MG, MGFA class II-IV, MG Activities of Daily Living (MG-ADL) ≥ 5 and QMG ≥ 9 at screening and being currently enrolled at 38 sites across Europe and North America. Endpoints include changes in clinician-reported outcomes (QMG, MG-ADL, MG Composite), and patient-reported outcomes (MG Quality of Life 15 revised , and Neuro Qol Fatigue Short Form). Key updates on study status and detailed study design will be provided by NMD Pharma A/S at MDA Clinical and Scientific Conference in March 2026 in Orlando, Florida.