argenx Presents Additional Efgartigimod Data from Global Phase 3 ADAPT Trial at the Myasthenia Gravis Foundation of America 2020 Scientific Session
October 05, 2020 at 01:00 AM EDT
October 5, 2020
“Myasthenia gravis can be a very debilitating and potentially life-threatening chronic disease in patients leading to impairments that affect a patient’s ability to complete normal daily activities, including walking, swallowing, chewing food, talking or breathing easily. Efgartigimod demonstrated in ADAPT that it is well-tolerated and that patients can experience clinically meaningful improvements in key measures of function and strength following treatment, including, in some, the achievement of minimal symptom expression. These exciting results suggest that efgartigimod as a new potential therapy for gMG patients could have a real impact on some of the daily limitations that patients face,” commented James F. Howard Jr., M.D., Professor of Neurology (Neuromuscular Disease), Medicine and Allied Health, Department of Neurology, The University of North Carolina at Chapel Hill School of Medicine and principal investigator for the ADAPT trial.
Highlights of New Data Presented at MGFA 2020 Virtual Scientific Session
Magnitude of response: Substantial proportion of efgartigimod-treated acetylcholine receptor-antibody positive (AChR-Ab+) patients showed benefit at increasing thresholds on the Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scores compared to placebo patients at week four (one week after first treatment cycle).
Repeatability of response: Similar proportion of efgartigimod-treated AChR-Ab+ patients were MG-ADL responders in the first (67.7% efgartigimod versus 29.7% placebo) and second (70.6% efgartigimod versus 25.6% placebo) treatment cycles (p<0.0001 for both cycles)
Clinical benefit in seronegative patients: Inclusion of QMG score in responder analysis showed further evidence of activity in patients where AChR antibodies were not detected (AChR-Ab-)
Key pharmacodynamic parameters: Total IgG and pathogenic autoantibody levels were reduced in efgartigimod-treated AChR-Ab+ patients throughout observation period, supporting proposed mechanism of action
Key Topline Data Previously Reported from ADAPT
Topline data from ADAPT were reported in May 2020. The trial met its primary endpoint showing 67.7% of efgartigimod-treated AChR-Ab+ gMG patients were responders on the MG-ADL score compared to 29.7% of placebo patients (p<0.0001). Responders were defined by having at least a 2-point change on the MG-ADL for at least four consecutive weeks. Efgartigimod was demonstrated to be well-tolerated with a safety profile that was comparable to placebo.
“These new data on the magnitude and repeatability of response continue to support the potential of efgartigimod as a meaningful treatment for gMG patients. ADAPT was also a broad trial which included patients with acetylcholine receptor antibodies present and those without. We were pleased to show in this presentation proof of activity in the antibody-negative patients who are often left out of clinical trials,” commented Wim Parys, M.D., Chief Medical Officer of argenx. “It is particularly gratifying to present these favorable new data at the MGFA Virtual Scientific Session, a scientific meeting solely focused on addressing unmet needs for people living with gMG. We look forward to submitting our Biologics License Application for efgartigimod to the U.S. Food and Drug Administration before the end of the year with the goal to have efgartigimod available to patients and physicians in 2021.”
Phase 3 ADAPT Trial
Efgartigimod is an investigational antibody fragment designed to reduce disease-causing immunoglobulin G (IgG) antibodies and block the IgG recycling process. Efgartigimod binds to the neonatal Fc receptor (FcRn), which is widely expressed throughout the body and plays a central role in rescuing IgG antibodies from degradation. Blocking FcRn reduces IgG antibody levels representing a logical potential therapeutic approach for several autoimmune diseases known to be driven by disease-causing IgG antibodies, including: myasthenia gravis (MG), a chronic disease that causes muscle weakness; pemphigus vulgaris (PV), a chronic disease characterized by severe blistering of the skin; immune thrombocytopenia (ITP), a chronic bruising and bleeding disease; and chronic inflammatory demyelinating polyneuropathy (CIDP), a neurological disease leading to impaired motor function.
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. More than 85% of people with MG progress to generalized MG (gMG) within 18 months, where muscles throughout the body may be affected, resulting in extreme fatigue and difficulties with facial expression, speech, swallowing, and mobility. In more life-threatening cases, MG can affect the muscles responsible for breathing. Patients with confirmed AChR antibodies account for 80-90% of the total gMG population. There are approximately 65,000 people in the United States and 20,000 people in Japan living with the disease.
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