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, 11 October 2022 –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 ClC-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. NMD Pharma’s biomarker driven approach to indication selection, patient stratification and monitoring of response combined with NMD670’s 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 CLC-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 ClC-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 ClC-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.”