NMD Pharma A/S announces that the U.S. Food and Drug Administration has granted orphan-drug designation for NMD670, its lead product candidate in development for treatment of myasthenia gravis.
FDA grants Orphan Drug Designation for NMD670 for the Treatment of Myasthenia Gravis
Aarhus, Denmark, 27September2022 – NMD Pharma A/S, a clinical stage biotech company developing first-in-class ClC-1 inhibitors for neuromuscular disorders, today announces that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation (ODD) for NMD670, its lead product candidate in development for treatment of myasthenia gravis (MG).
NMD670 is a first-in-class small molecule inhibitor of the skeletal muscle specific chloride ion channel (ClC-1). The ODD was designated on compelling preclinical efficacy data with NMD670 in animal models of MG demonstrating that ClC-1 inhibition with NMD670 enhances neuromuscular transmission and restores skeletal muscle function. NMD670 has recently completed a combined Phase I/IIa clinical trial to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NMD670 with results anticipated imminently.
Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, commented:“Myasthenia gravisis a rare neuromuscular disease with a highunmet medical need for innovative new treatments to addressthe debilitating symptoms of muscle weakness and fatigue. Receiving orphan drug designation for our lead product candidate, NMD670, isa significant milestone for NMD Pharmaas it further strengthens our belief in this novel treatment approach and helps to progressourinvestigational drug to patients.”
ODD is granted by the FDA to a drug or biologic intended to treat a rare disease or condition affecting fewer than 200,000 people in the United States.
Further information on the ongoing Phase I/IIa study of NMD670 in MG can be found on the International Clinical Trials Registry: https://trialsearch.who.int/Trial2.aspx?TrialID=NL8692.
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Contacts
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 invivo pharmacology models for discovering and developing proprietary modulators of neuromuscular function. NMD Pharma received initial seed financing in 2016 and have since raised ~€80 million from investors including Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, Roche Venture Fund, and Jeito Capital. Find out more about us online at http://www.nmdpharma.com/.
About NMD670
NMD670 is NMD Pharma’s lead development program. It is a first-in-class small molecule inhibitor of the skeletal muscle specific chloride ion channel (ClC-1). NMD Pharma has demonstrated that ClC-1 inhibition enhances neuromuscular transmission and restores skeletal muscle function, and this novel treatment approach has demonstrated compelling preclinical efficacy data in animal models of myasthenia gravis (MG) and a range of other neuromuscular disorders.
About Myasthenia Gravis (MG)
MG is a rare and chronic autoimmune disease where IgG antibodies disrupt communication between nerves and muscles causing debilitating and potentially life-threatening muscle weakness. It commonly affects the muscles that control the eyes and eyelids, facial expressions, chewing, swallowing, and speaking but in most patients it eventually impacts most skeletal muscles. More than 85% of people with MG progress to generalized MG (gMG) within 18 months and it can be life-threatening when affecting the muscles responsible for breathing. There are approximately 100,000 people in the European Union, 65,000 people in the United States and 20,000 people in Japan living with the disease.
