Generalized Myasthenia Gravis and the Possibilities with an Investigational Drug in a Clinical Trial

Living with Generalized Myasthenia Gravis

Generalized myasthenia gravis (gMG) is a rare, long-term autoimmune disease that affects how your nerves and muscles work together. In people with gMG, the signals from the nerves don’t reach the muscles properly. This makes the muscles weaker and less able to respond, causing muscle weakness that can come and go throughout the day. While current treatments may help manage the immune system, many people still experience persistent and unpredictable muscle weakness and fatigue. If this sounds like your experience, you’re not alone - and an investigational drug is being studied to help improve muscle strength and endurance.

NMD Pharma’s Treatment Approach: Targeting the Skeletal Muscles in gMG

NMD670 is an experimental oral medicine being tested to help people with generalized myasthenia gravis. It works differently than current treatments. It is a skeletal muscle-targeted therapy that is being investigated for the potential to increase muscle responsiveness to weak nerve signals and enable more consistent muscle function. It can be added to the current treatment regimen prescribed by your physician. It is an oral medication taken with a simple glass of water.

In a previously completed early phase placebo-controlled clinical trial in a small group of patients with myasthenia gravis, patients received only a single dose of NMD670 and results demonstrated indicated meaningful improvements in muscle strength for patients across multiple types of skeletal muscles (face, mouth/swallowing, arms, legs) throughout the body.1 Now, researchers are testing NMD670 in a larger clinical trial to understand the safety and efficacy of the drug in various dosing levels over 21 days.

1 https://www.nature.com/articles/d41573-024-00065-9

Join the Clinical Trial to Study the Potential Benefits and Safety of a New Investigational Skeletal Muscle-Targeted Drug

Currently there is no cure for generalized myasthenia gravis (gMG) and most people living with the disease require a combination of treatment therapies but still experience persistent and unpredictable symptoms.

The SYNAPSE-MG Study is evaluating an investigational drug called NMD670 in adults with generalized myasthenia gravis patients who are AChR or MuSK antibody positive in clinical sites located in the US, Europe and Canada. You will remain on your currently prescribed medications, and NMD670 (or placebo) will be added to your medications as part of the clinical trial.

You may be able to take part in the SYNAPSE-MG Trial if you:

  • Are 18-75 years of age

  • Have been diagnosed with myasthenia gravis with a positive AChR or MuSK antibody test meeting certain other requirements related to disease severity

  • Are able to swallow tablets

  • Can attend 6 different in-clinic visits over period of approximately 8 weeks (depending on scheduling)

Importantly, you may be able to remain on your existing physician prescribed drug therapies while in this study.

This is not a complete list of study requirements. The study doctor will review the full requirements for this study with you.

There is no cost to participate in this study, and your reasonable travel costs will be reimbursed (flight, mileage, hotel, parking, meals). Compensation may also be available for time taken for study visits or lost wages.

For more information and to request involvement in this important generalized myasthenia clinical trial, including cities/states of clinical trial sites and contact information for clinical coordinators, please visit clinicaltrials.gov or email NMD Pharma at: clinicaltrials@nmdpharma.com

NMD670 has not been approved by Regulatory Health Authorities and the safety and effectiveness of NMD670 in SMA, MG or CMT has not been established