NMD Pharma announces poster and oral presentations on skeletal muscle targeted ClC-1 therapy at two leading neuromuscular disease conferences

2024-03-01

临床2期临床结果

NMD Pharma announces poster and oral presentations on skeletal muscle targeted ClC-1 therapy at two leading neuromuscular disease conferences ClC-1 is a chloride ion channel specifically expressed in skeletal muscle cells that normally dampens muscle fiber excitability and is involved in regulating muscle fiber excitability during exercise. ClC-1 inhibition could be a possible mechanism to improve muscle fiber activation to address muscle weakness and fatigue in neuromuscular diseases. Presentations include new preclinical data on CIC-1 inhibition and a design and status update on the ongoing Phase 2, SYNAPSE-SMA, trial of NMD670 in spinal muscular atrophy type 3. Aarhus, Denmark, 1 March 2024 – NMD Pharma A/S, a clinical-stage biotech dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, today announces that it will present two posters at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference 2024 and one poster and one oral presentation at the 4th Scientific International Congress on SMA 2024. The presentations will provide a clinical study design and status update on the Phase II SYNAPSE-SMA trial of NMD670 in ambulatory adults with spinal muscular atrophy type 3. The SYNAPSE-SMA study is a proof-of-concept, Phase II, randomized, double-blind, placebo-controlled, 2-way crossover study to evaluate the efficacy, safety, and tolerability of NMD670. The study is an international multicenter study including sites in North America and Europe and the first subject was enrolled in September 2023. In addition, NMD Pharma will also be presenting a poster outlining pre-clinical results elucidating the mechanism of action for CIC-1 inhibition and its potential to improve neuromuscular transmission in neuromuscular diseases with transmission failure. Jorge A. Quiroz, EVP, and Chief Medical Officer at NMD Pharma: “We are looking forward to sharing updates on the ongoing Phase 2 SYNAPSE-SMA trial for NMD670, along with further insights into the mechanism of ClC-1 inhibition and its potential to enhance neuromuscular transmission improving muscle weakness and fatigue.” “There remains a significant unmet need for new treatments for severe neuromuscular diseases, including patients with SMA. NMD670 has already demonstrated positive clinical study results in a Phase I/II study in patients with myasthenia gravis (MG), and we are confident that it could also be beneficial for the treatment of patients affected by SMA.” The MDA Conference is taking place in Orlando, Florida from 3-6 March. Details of NMD Pharma’s poster presentations are below: Poster number: S114 Location: Poster Exhibit Hall, Hilton Orlando Date and time: Sunday March 3, 6:00pm – 8:00pm ET Poster number: M278 Location: Poster Exhibit Hall, Hilton Orlando Date and time: Monday March 4, 6:00pm – 8:00pm ET The 4th Scientific International Congress on SMA is taking place in Ghent, Belgium, from 14-16 March. : NMD Pharma be presenting one poster and one oral presentation at the congress. Details of NMD Pharma’s oral presentations are below: Oral Presentation: “ClC-1 inhibitor ClC-1 inhibitor compound improves neuromuscular transmission and enhances skeletal muscle function in pre-clinical animal models of neuromuscular dysfunction” Location: Main Auditorium Date and Time: Saturday 16th March 9:30am CET -END- Contacts NMD Pharma A/S Thomas Holm Pedersen, CEO E-mail: contact@nmdpharma.com ICR Consilium Mary-Jane Elliott / Ashley Tapp / Lindsey Neville E-mail: NMDPharma@consilium-comms.com Tel: +44 (0)20 3709 5700 About NMD Pharma NMD Pharma A/S is a clinical-stage biotech company developing a first-in-class platform of small molecule therapies selectively targeting the skeletal muscle chloride ion channel (ClC-1) for the treatment of severe neuromuscular disorders. The Company was founded on more than 15 years of muscle physiology research with a focus on regulation of skeletal muscle excitability under physical activity. NMD Pharma has built a world-leading muscle electrophysiology platform leveraging the in-depth know-how of muscle physiology and muscular disorders, small molecule modulators, enabling technologies and tools as well as in vivo pharmacology models for discovering and developing proprietary modulators of neuromuscular function. The Company has built significant clinical and development expertise as its programmes have progressed through in the clinic. NMD Pharma has raised ~€155 million from investors including Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, Roche Venture Fund, and Jeito Capital. Find out more about us online at .