Ra Pharma is committed to patients with rare, complement-mediated diseases.

What Is Generalized Myasthenia Gravis (gMG)?


Ra Pharma is committed to patients with rare, complement-mediated diseases.

What Is Generalized Myasthenia Gravis (gMG)?


Myasthenia gravis (MG) is a rare, chronic, autoimmune, neuromuscular condition characterized by weakness of voluntary muscles that increases with exercise and recovers with rest. Generalized myasthenia gravis (gMG) is a severe form of MG that can include weakness of multiple muscle groups such the limbs, neck, diaphragm, and eye muscles.

How Voluntary Muscles Work

How Voluntary Muscles Work

Let's review how voluntary muscles work. Voluntary muscles in the body are controlled by nerve signals, or neurotransmitters, from the brain. The area where the nerve signals from the brain meet the muscle fiber is called the neuromuscular junction. The neuromuscular junction contains many receptors called acetylcholine receptors. These receptors are essential for initiating muscle movement.

When signals from the brain travel through the nerve, the nerve endings release a neurotransmitter called acetylcholine into the neuromuscular junction. The acetylcholine binds to acetylcholine receptors to stimulate the muscle fibers and enable them to contract.

Normal neuromuscular junction
Damaged neuromuscular junction

What Causes gMG?

What Causes gMG?

In people with gMG, the immune system doesn't function correctly. Instead of helping the body fight off sickness by attacking intruders like bacteria and viruses, the body makes antibodies against the acetylcholine receptors. These antibodies switch on another part of the immune system, called the complement cascade, and damage the neuromuscular junction. As a result, the neuromuscular junction can no longer function and nerve impulses are not transmitted to the muscle, causing weakness.

Damaged neuromuscular junction

What Are the Symptoms of gMG?

What Are the Symptoms of gMG?


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Muscle weakness in patients with gMG characteristically becomes worse with repeated use, and then may get better after the muscle is rested.

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The respiratory muscles (i.e., diaphragm) can be affected, which can cause life-threatening crisis and possibly require treatment in a hospital or ICU.

For more information about MG and gMG, please visit the other websites listed here.

What Are Current Treatment Options for gMG?

What Are Current Treatment Options for gMG?


Right now, doctors can use several treatments to alleviate symptoms of gMG. Although these treatments may help improve muscle weakness, most of these treatments have been developed for other illnesses and have not been specifically designed nor approved to treat gMG. Various side effects, from mild to serious, can occur from use of some of these treatments. We recommend that you speak to your physician for details.

If those treatments do not work, doctors sometimes try adding an intravenous complement inhibitor. The goal of Ra Pharma's ongoing research is to develop a self-administered, subcutaneous complement inhibitor designed to be used early in treatment, which we believe has the potential to provide benefit to many more patients.

Zilucoplan


Zilucoplan* is a small peptide (or a tiny protein) which we believe has the potential to benefit many more patients. Zilucoplan's size is designed to allow for self-administration at home as a daily injection.

In December 2018, Ra Pharma completed a Phase 2 clinical trial evaluating zilucoplan for gMG, in which zilucoplan achieved the primary and key secondary endpoints.

Ra Pharma is now enrolling eligible people with gMG into a global Phase 3 study, called the RAISE study.

*Zilucoplan is an investigational drug and its safety and effectiveness are currently being studied. Zilucoplan has not been approved by the FDA for the treatment of gMG or any other disease or condition.

To see if you could qualify for this study, click here.

For more information about the RAISE study, click here.

Want to learn more about clinical trials? Visit our "Resources" section to learn more.

RAISE

What Are the Goals of the RAISE Study?

What Are the Goals of the RAISE Study?

The main purpose of the RAISE study is to test how well zilucoplan works and how safe it is for people with gMG.

RAISE Study Design

The RAISE study is a placebo-controlled study with a 12-week study treatment period. Participants will be randomly assigned to receive either zilucoplan or placebo. Study participants will continue their standard of care treatment** during the study and, in addition, will receive study drug as a once-daily, self-administered (at home) subcutaneous injection.

Participation in the study will last approximately 16 weeks and would include approximately 7 study visits. Participants will have an option to continue to receive zilucoplan in a long-term extension study (the RAISE-XT study) after completing this clinical trial.

**Some standard of care treatments, such as IVIG and Soliris, may be not be allowed while on this study. Please contact us for more information.

Who Is Eligible to Participate in This Study?

If you are between the ages of 18 and 74 and have a diagnosis of gMG, you may be eligible to participate in the RAISE study.

Your doctor will need to see if you meet these and other eligibility criteria before you can be enrolled.

Key Eligibility Criteria

  • Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IV ]
  • Positive serology for acetylcholine receptor (AChR) binding autoantibodies
  • Stable doses of current gMG medications during the study
  • No thymectomy within 12 months prior to baseline or scheduled to occur during the 12-week study
  • No treatment with plasma exchange within 4 weeks

Locations

Locations

The RAISE study will enroll approximately 130 subjects in around 80 sites around the globe.

For participants who do not live near a study location, Ra Pharma may reimburse for costs associated with travel, if needed.

London, Ontario
Montreal, Quebec
Nice, France
Paris, France
Strasbourg, France
Essen, Germany
Freiburg, Germany
Göttingen, Germany
Halle, Germany
Munich, Germany
Tubingen, Germany
Milan, Italy
Rome, Italy
Hanamaki, Japan
Nagasaki, Japan
Sapporo, Japan
Sendai, Japan
Tokyo, Japan
Bergen, Norway
Oslo, Norway
Barcelona, Spain
Bilbao, Spain
Madrid, Spain
Oxford, United Kingdom
Sheffield, United Kingdom
Augusta, Georgia
Austin, Texas
Boston, Massachusetts
Buffalo, New York
Chapel Hill, North Carolina
Charleston, South Carolina
Cleveland, Ohio
Columbia, Missouri
Columbus, Ohio
Dallas, Texas
Detroit, Michigan
Durham, North Carolina
East Lansing, Michigan
Jacksonville, Florida
Kansas City, Kansas
Las Vegas, Nevada
Los Angeles, California
Milwaukee, Wisconsin
Minneapolis, Minnesota
Mobile, Alabama
New York, New York
Orange, California
Phoenix, Arizona
Pittsburgh, Pennsylvania
Portland, Oregon
Salt Lake City, Utah
Tampa, Florida
Washington, D.C.
New Haven, Connecticut

Resources

Resources

Our commitment to gMG does not end with research.

The Ra Pharma team is dedicated to the gMG community. We are working to spread awareness of the disease by partnering in local fundraising walks and events and providing information about our work on the disease that can easily be shared.

For more details about MG and gMG or to become more involved in the community, visit the following:

To learn more about clinical trials, visit the following:

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