Novo Seeds Portfolio Company NMD Pharma Reports Positive Top-Line Phase I/IIa Data in Myasthenia Gravis

• Data provide first clinical proof of mechanism of action and clinical utility of NMD Pharma's lead asset NMD670 in Myasthenia Gravis
• The clinical validation provides a unique translational platform and informs new clinical studies in additional indications characterized by neuromuscular dysfunction
• Novo Seeds seeded and funded the company to develop novel treatments for rare neuromuscular diseases that cause debilitating and potentially life-threatening muscle weakness

COPENHAGEN, Denmark, Oct. 11, 2022 /PRNewswire/ -- Novo Holdings, a leading international life sciences investor, announces that its portfolio company NMD Pharma, a clinical stage biotech company developing first-in-class, small molecule CIC-1 inhibitors for neuromuscular disorders, reported positive top-line results from a Phase I/IIa clinical trial for its lead asset, NMD670.

NMD670, which recently received orphan drug designation by the FDA for treatment of myasthenia gravis (MG), was found to be safe and well tolerated in healthy volunteers and patients and showed clinically significant improvements. MG is a rare and chronic autoimmune disease that causes debilitating and potentially life-threatening muscle weakness due to impaired neuromuscular transmission. It commonly affects the muscles that control the eyes and the face, and it eventually impacts most skeletal muscles. The condition can be life-threatening when affecting the muscles responsible for breathing.

Novo Seeds, the company creation arm of Novo Holdings, played an instrumental role in founding and seeding NMD Pharma in 2015, and has since then been a cornerstone investor for the company.

Morten Graugaard Døssing, Board Director of NMD Pharma and Partner at Novo Holdings, said: "The clinical validation of NMD's scientific approach which resulted in clinical meaningful effects provides a unique translational platform to address many rare diseases with large unmet medical needs characterized by neuromuscular dysfunction. Our biomarker driven approach to indication selection, patient stratification and monitoring of response combined with a rapid onset of efficacy provides a compelling value proposition. We have been working collaboratively alongside CEO Thomas Holm Pedersen and the NMD Pharma team even before the company was founded and are incredibly proud of the progress the team has made to date. I look forward to continuing supporting the company alongside our fellow investors and progress its portfolio of CIC-1 inhibitors for the treatment of patients suffering from rare neuromuscular diseases."

Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, said: "These trial results represent an important milestone for NMD Pharma as they provide the first clinical proof of mechanism for our novel CIC-1 inhibitor treatment approach. With these data we complete an important journey from conceptualizing a new treatment concept to obtaining clinical proof of mechanism, and further establish the relevance of pursuing the development of CIC-1 inhibitors across a range of diseases associated with neuromuscular dysfunction. I would like to thank the NMD Pharma team for their many contributions to making this trial a success and, most importantly, the patients who participated in the study."

About Novo Holdings A/S

Novo Holdings A/S is a private limited liability company wholly owned by the Novo Nordisk Foundation. It is the holding company of the Novo Group, comprising Novo Nordisk A/S and Novozymes A/S, and is responsible for managing the Novo Nordisk Foundation's assets.

Novo Holdings is recognised as a leading international life science investor, with a focus on creating long-term value. As a life science investor, Novo Holdings provides seed and venture capital to development-stage companies and takes significant ownership positions in growth and well-established companies. Novo Holdings also manages a broad portfolio of diversified financial assets. Further information:www.novoholdings.dk.

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 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 (CIC-1). NMD Pharma has demonstrated that CIC-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. NMD670 has recently been granted orphan-drug designation (ODD) by the U.S. Food and Drug Administration (FDA) for treatment of MG.

About the Study

The Phase I part included single ascending dose and multiple ascending doses in 67 healthy volunteers. The Phase IIa part was a single dose, proof of mechanism, randomized, placebo-controlled, double-blind, three-way cross-over study, investigating the safety and pharmacodynamic effects of NMD670 at two dose levels in 12 patients with MG.

NMD670 was safe and well tolerated in healthy volunteers and patients. Administration of single doses of NMD670 was associated with clinically significant improvements in the Quantitative Myasthenia Gravis Score with up to 50% of the patients meeting pre-specified responder criterion. Electrophysiological endpoints demonstrated target engagement and restoration of neuromuscular transmission that in patients were associated with increases in muscle strength and function. The trial took place at the Centre for Human Drug Research (CHDR) in Leiden in the Netherlands. Further information on the study can be found on the International Clinical Trials Registry: https://trialsearch.who.int/Trial2.aspx?TrialID=NL8692. 

NMD Pharma will publish the full trial data in a peer reviewed journal and present the findings at a leading industry conference over the coming months.

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.