NMD670 / NMD Pharma - LARVOL DELTA

NMD Pharma presents preclinical data highlighting increased innervation of skeletal muscle with ClC-1 ion channel inhibition treatment in neuromuscular disease model (GlobeNewswire) - "Martin Brandhøj Skov...delivered the oral presentation on the positive effects of NMD Pharma’s orally bioavailable small molecule ClC-1 inhibitor in a muscle-specific kinase (MuSK) myasthenia gravis (MG) rat model on improved disease symptoms at the conference. The study showed that NMD Pharma’s small molecule ClC-1 inhibitor had a positive effect on electromyography (EMG), leading to higher body weight, increased survival, and increased muscle and respiratory function. In addition to the alleviation of disease symptoms and improved function, the structural integrity of the neuromuscular junction was improved, with animals treated with a ClC-1 inhibitor demonstrating an increase in the number of fully innervated muscle fibers from 21% (vehicle) to 46% (treated with ClC-1 inhibitor)."

Preclinical • Myasthenia Gravis

SYNAPSE-SMA: Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (clinicaltrials.gov) - P2 | N=54 | Recruiting | Sponsor: NMD Pharma A/S | Trial completion date: Jul 2025 ➔ Jan 2026 | Trial primary completion date: Jul 2025 ➔ Jan 2026

Trial completion date • Trial primary completion date • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases • SMN1 • SMN2

NMD Pharma Announces FDA Orphan Drug Designation Granted to NMD670 for the Treatment of Patients with Charcot-Marie-Tooth Disease (GlobeNewswire) - "NMD Pharma A/S...today announces that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) for NMD670, a novel, oral, small molecule inhibitor of the skeletal muscle-specific chloride ion channel ClC-1, for the treatment of Charcot-Marie-Tooth disease (CMT)."

Orphan drug • Genetic Disorders

Neuromuscular transmission deficits in patients with CMT and ClC-1 inhibition in CMT animal models. (PubMed, Ann Clin Transl Neurol) - "Our study suggests that NMJ dysfunction contributes to muscle dysfunction in patients with CMT 1 and 2. Furthermore, our preclinical data provide proof-of-mechanism for recovery of muscle function with ClC-1 inhibition in CMT mouse models. Collectively, these findings suggest that targeting NMJ dysfunction with ClC-1 inhibitors could enhance muscle function in CMT patients, warranting further clinical trials."

Journal • Preclinical • Fatigue • Genetic Disorders • Pain

Safety, Pharmacokinetics, and Pharmacodynamics of a First-in-Class ClC-1 Inhibitor to Enhance Muscle Excitability: Phase I Randomized Controlled Trial. (PubMed, Clin Pharmacol Ther) - "NMD670 is a first-in-class inhibitor of skeletal muscle-specific chloride channel ClC-1, developed to improve muscle weakness and fatigue in neuromuscular diseases...Importantly, the results of this study indicate pharmacological target engagement at well-tolerated dose levels in healthy subjects; firstly, because myotonia was an expected exaggerated on-target pharmacological effect, and secondly, because the effects on MVRC indicate increased muscle cell excitability. This study in healthy subjects indicates proof-of-mechanism and provides a solid base for translation to patients with neuromuscular diseases."

Clinical • Journal • P1 data • PK/PD data • CNS Disorders • Fatigue

SYNAPSE-SMA: Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (clinicaltrials.gov) - P2 | N=54 | Recruiting | Sponsor: NMD Pharma A/S | Trial completion date: Dec 2024 ➔ Jul 2025 | Trial primary completion date: Dec 2024 ➔ Jul 2025

Trial completion date • Trial primary completion date • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases • SMN1

SYNAPSE-CMT: Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease (clinicaltrials.gov) - P2 | N=80 | Recruiting | Sponsor: NMD Pharma A/S | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders

SYNAPSE-MG: Safety and Efficacy of 3 Dose Levels of NMD670 in Adult Patients With Myasthenia Gravis (clinicaltrials.gov) - P2 | N=84 | Recruiting | Sponsor: NMD Pharma A/S | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Myasthenia Gravis

SYNAPSE-CMT: Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease (clinicaltrials.gov) - P2 | N=80 | Not yet recruiting | Sponsor: NMD Pharma A/S

New P2 trial • Genetic Disorders

SYNAPSE-MG: Safety and Efficacy of 3 Dose Levels of NMD670 in Adult Patients With Myasthenia Gravis (clinicaltrials.gov) - P2 | N=84 | Not yet recruiting | Sponsor: NMD Pharma A/S

New P2 trial • CNS Disorders • Myasthenia Gravis

SYNAPSE-SMA: Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (clinicaltrials.gov) - P2 | N=54 | Recruiting | Sponsor: NMD Pharma A/S | Trial completion date: Sep 2024 ➔ Dec 2024 | Trial primary completion date: Sep 2024 ➔ Dec 2024

Trial completion date • Trial primary completion date • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases • SMN1