NMD Pharma to present NMD670 clinical data from a Phase I/IIa trial in myasthenia gravis at the 28th International Annual Congress of the World Muscle Society


NMD Pharma to present NMD670 clinical data from a Phase I/IIa trial in myasthenia gravis at the 28th International Annual Congress of the World Muscle Society

  • NMD Pharma will show the first ever clinical proof-of-mechanism for the novel concept of ClC-1 inhibition with NMD670 in treatment of neuromuscular diseases demonstrated in patients with myasthenia gravis
  • The data provides clinical validation of the ability of CIC-1 inhibition to restore neuromuscular function leading to clinically meaningful effects with a single dose of NMD670
  • This data has been instrumental in the design of the recently initiated Phase II clinical trial of NMD670 in patients with type 3 spinal muscular atrophy

Aarhus, Denmark, 3 October 2023 – NMD Pharma A/S, a clinical-stage biotech company developing first-in-class, small molecule ClC-1 inhibitors directly targeting muscle function within neuromuscular disorders, today announces that it will present data from a Phase I/IIa clinical trial of NMD670 in patients with myasthenia gravis (MG) in a poster presentation at the 28th International Annual Congress of the World Muscle Society.


The World Muscle Society Congress is taking place at the Charleston Convention Center in Charleston, South Carolina, US from 3-7 October. Details of NMD Pharma’s poster presentation are below:

Poster number: P272
Location: Ballroom A-C
Date and time: Thursday, 5 October from 3:30 pm to 4:30 pm EDT.

These results build on the positive topline data announced in October 2022, which established the first clinical proof-of-mechanism of NMD Pharma’s novel CIC-1 chloride channel inhibitor approach in patients impacted by MG. NMD670 was found to be safe and well tolerated in patients, and administration of a single dose of the Company’s lead development programme was associated with clinically and statistically significant improvements in the Quantitative Myasthenia Gravis Total Score. In addition, up to 50% of patients were responders to NMD670 according to pre-specified criteria, as early as 3 hours after the administration of a single dose.

The clinical validation of NMD Pharma’s CIC-1 inhibitor approach achieved in this Phase I/IIa study in patients with MG was used to plan the Company’s Phase II clinical trial of NMD670 in ambulatory patients with spinal muscular atrophy (SMA) Type 3, dosing the first patient in September 2023.

“The International Congress of the World Muscle Society is one of the most significant scientific gatherings in our field, globally. We eagerly anticipate the opportunity to share data from our clinical trial involving patients with myasthenia gravis,” remarked Jorge A. Quiroz, EVP, and Chief Medical Officer at NMD Pharma. He added, “These foundational results mark a significant milestone for NMD Pharma, serving as our initial proof-of-mechanism for CIC-1 inhibition, which holds relevance across a wide spectrum of diseases linked to neuromuscular dysfunction.”



NMD Pharma A/S
Dan Brennan, SVP, Corporate and Commercial Strategy
E-mail: [email protected]

Consilium Strategic Communications
Mary-Jane Elliott / Ashley Tapp / Lindsey Neville
E-mail: [email protected]
Tel: +44 (0)20 3709 5700

About NMD Pharma
NMD Pharma A/S, is a clinical-stage biotech company leading in the development of novel first-in-class therapies for severe neuromuscular disorders. The Company was incorporated as a spin-out from Aarhus University, Denmark in 2015 and 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 its in-depth know-how of muscle physiology and muscular disorders and is developing a pipeline of ClC-1 inhibitors for the treatment of patients with neuromuscular disorders including myasthenia gravis and spinal muscular atrophy. Positive top-line data reported from a Phase I/II clinical trial of lead program NMD670 in myasthenia gravis has provided clinical validation of ClC-1 inhibition to restore neuromuscular function. NMD Pharma has raised ~€80 million from investors including Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, Roche Venture Fund, and Jeito Capital. Find out more about us at http://www.nmdpharma.com/.

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