NMD Pharma Initiates ESTABLISH Study in Patients with Charcot-Marie-Tooth Disease

NMD Pharma Initiates ESTABLISH Study in Patients with Charcot-Marie-ToothDisease

  • The international observational study will be conducted in collaboration with Aarhus University and The Ohio State University

Aarhus, Denmark, 8 July2021NMD Pharma A/S, a biotech company developing novel therapeutics for neuromuscular disorders, today announces that it has initiated the ESTABLISH* study, an international observational study in collaboration with Aarhus University and The Ohio State University to assess neuromuscular function in patients with Charcot-Marie-Tooth (CMT) disease, an inherited neuromuscular disorder of the peripheral nerves characterized by weakness and fatigue. Aarhus University and The Ohio State University are world-leading institutions in neuromuscular disease research.

The primary aim of this study is to discover whether patients with CMT have neuromuscular junction (NMJ) transmission deficits. The hypothesis for the study is that if patients with CMT are found to have deficits in NMJ transmission, then the novel treatment approach being developed by NMD Pharma could potentially have beneficial clinical effects on neuromuscular performance in these patients. The results of this new study will be used to guide the design of a protocol for future clinical trials to assess the potential for ClC-1 inhibitors to be effective as a treatment for individuals living with CMT 1 & 2.

The international study will be led by Prof. Dr. Henning Andersen and Prof. Dr. William David Arnold at Aarhus University Hospital and The Ohio State University Wexner Medical Center, respectively, and will involve 30 subjects: 20 CMT patients and 10 healthy individuals (as controls). CMT patients will undergo electrophysiological assessments (repetitive nerve stimulation and single fiber EMG) and functional testing (involving tests of muscle strength, fatigability, dexterity, and balance) on four separate occasions over a 6-week period. The healthy individuals (controls) will undergo electrophysiological testing only.

NMD Pharma is developing first-in-class small molecule inhibitors of the muscle specific chloride ion channel, the ClC-1 ion channel. The Company has demonstrated that ClC-1 inhibition can strengthen neuromuscular transmission and ultimately skeletal muscle function and this novel treatment approach has demonstrated compelling preclinical data for Myasthenia Gravis (MG). The lead candidate, NMD670, is currently being evaluated in a combined Phase I/IIa trial to treat the symptoms of MG, which is taking place at the Centre for Human Drug Research (CHDR) in Leiden, the Netherlands.

Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, said:This study is NMD Pharma’s first coordinated international clinical trial focused on a rare neuromuscular disease such as CMT and we are delighted to be working with the teams at Aarhus University and The Ohio State University, world-leading experts in neuromuscular research.With no currently approved treatments or therapies for this debilitating disease, this study will provide us with valuable insightsintothe neuromuscular function of CMT patients and therefore the potential benefits of treating these patients with our novel treatment approach.”

Dr. Henning Andersen,Professor, Department of Neurologyat Aarhus UniversityHospital commented:CMTencompasses a group of inherited progressive and chronic peripheral neuropathies that affect approximately 1 in every 3,300 people worldwide with no approved medications toprovide a cure or alleviate symptoms. We are pleased to be working with NMD Pharma and The Ohio State University to further assess the possibility of using NMD’s ion channel inhibitor as a treatment for the symptoms of this disease.”

Dr. William David Arnold, Professor, Department of Neurology at The Ohio State University Wexner Medical Centeradded: The progressive nature of CMT means that those diagnosed with the disease will lose function and control of their legs and arms over time due to muscular weakness and atrophy, causing problems walking and running, and with balance and hand function. Therapies are desperately needed to help alleviate these life-altering symptoms and to help restore normal function for those affected.”

* Exploring SynapTic ABnormaLitIeS in Hereditary neuropathies


NMD Pharma A/S
Thomas Holm Pedersen, CEO
E-mail: contact@nmdpharma.com

Consilium Strategic Communications
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 private biotech company leading in the development of novel first-in-class therapies for severe neuromuscular disorders. The Company was incorporated as a spin-off 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 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. NMD Pharma received seed financing from Novo Seeds, Lundbeckfonden Emerge and Capnova in 2016, and in 2018 raised a €38 million Series A financing, led by new investor INKEF Capital, together with new investor Roche Venture Fund and existing investors Novo Seeds and Lundbeckfonden Emerge. Find out more about us online at http://www.nmdpharma.com/.

About the Department of Clinical Medicine: Aarhus, Denmark
Department of Clinical Medicine is Denmark’s largest health science institute conducting research in almost all medical specialities and hosting a large number of research centres.
Most of our staff are employed part-time at the department and part-time as clinical staff at Aarhus University Hospital or one of the four regional hospitals in Central Denmark Region.
The close collaboration between Aarhus University and Aarhus University Hospital ensures fast implementation of research results in clinical practice to the benefit of patients. Department of Clinical Medicine is located at Aarhus University Hospital, which has been awarded Denmark’s best hospital 13 times in a row.

About The Ohio State University
The Ohio State University Wexner Medical Center, based in Columbus, Ohio, USA, is a proud part of one of America’s largest and most comprehensive universities. On the university’s main Columbus campus, more than 56,000 students are able to choose from 14 colleges, 175 undergraduate majors and 240 master’s, doctoral and professional degree programs. Ohio State is consistently ranked as Ohio’s best and one of the nation’s top-20 public universities.
The university’s research innovations have attained world-class status, particularly in critical areas such as global climate change, cancer, infectious disease, neurosciences, advanced materials and ag-bio products that feed and fuel the world.

About NMD670
NMD670 is NMD Pharma’s lead development program. It is a first-in-class small molecule inhibitor of the muscle specific chloride ion channel, the ClC-1 ion channel. NMD Pharma has demonstrated that ClC-1 inhibition can strengthen neuromuscular transmission and ultimately skeletal muscle function and this novel treatment approach has demonstrated compelling preclinical efficacy data for MG.

About Charcot-Marie-Tooth (CMT) disease
CMT encompasses a group of hereditary sensory and motor neuropathies that cause damage to peripheral nerves. Damage caused by CMT worsens over time and can result in the loss or alteration of sensation and atrophy of muscles in the legs, feet, arms and hands. There are several types of CMT which are differentiated by their effects on neurons and inheritance patterns caused by mutations on the X chromosome - both result in either abnormalities in myelination, axonal degeneration, or both. CMT affects approximately 2.6 million individuals worldwide with symptoms appearing typically at adolescence or early adulthood, however onset of the disease can occur at any age.

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