According to a story from Euroinvestor, the biotechnology company argenx recently announced that it has gotten correspondence from Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) in regards to the design of the company’s Phase 3 trial, which will test its investigational product ARGX-113. This therapy is in development for the treatment of generalized myathenia gravis. Argenx is committed to the development of antibody based drugs for the treatment of cancer and severe autoimmune diseases.
About Myasthenia Gravis
Myasthenia gravis is a long term neuromuscular disease which is most characterized by weakness of the skeletal muscles, which can vary in severity. This is an autoimmune disease, meaning that an improper response from the immune system damages critical receptors that allow for nerve signals to move muscles. Symptoms can often appear abruptly and without warning. They include drooping eyelids, double vision, difficulty breathing, problems chewing, speaking, and swallowing, weakness of face and neck muscles, and worsening muscle weakness throughout the day. Pregnancy can make symptoms worse. Treatment can involve a variety of medications, plasmapharesis, and surgery to remove the thymus. Improvements in treatment mean that most patients can manage the disease well enough to live fairly normal lives. To learn more about myasthenia gravis, click here.
About The Phase 3 Trial
The feedback from the PMDA clears the way forward for the global Phase 3 trial, which is projected to involve a total of 150 myasthenia gravis patients. The trial will include patients from the US, Japan, and Europe and the results will determine whether the drug will obtain marketing authorization in Japan. The placebo controlled trial will test a 10mg/kg dose of ARGX-113 for a period of 26 weeks.
ARGX-113 is intended for the treatment of any autoimmune disease that is linked to IgG antibodies. The therapy works by causing these antibodies to deplete themselves, which should eventually cause the immune response that is causing the disease to stop.
Argenx plans to begin the clinical trial before the end of the year. Positive results from the trial would also be used for the company’s Biologics License Application to the US FDA. While treatment options for myasthenia gravis have improved greatly over the years, there still remains a significant unmet need for alternative approaches that can help patients who do not respond as well to currently available treatments.