argenx announces publication of Phase 3 ADAPT trial results of efgartigimod for the treatment of generalized myasthenia gravis in The Lancet Neurology
Results from ADAPT showed treatment with efgartigimod provided clinically meaningful improvements in strength and quality of life measures in generalized myasthenia gravis (gMG)
Efgartigimod is under
“Myasthenia gravis can have a devastating impact on a person's life and independence, potentially affecting one's ability to swallow, speak, walk and even breathe. In addition, each patient experiences the course of MG differently, which can make disease management unpredictable,” said
The ADAPT trial met its primary endpoint demonstrating significantly more acetylcholine receptor-antibody positive (AChR-Ab+) gMG patients were responders on the Myasthenia Gravis Activities of Daily Living (MG-ADL) score following treatment with efgartigimod compared with placebo (67.7% vs. 29.7%; p<0.0001). Responders were defined as having at least a two-point improvement sustained for four or more consecutive weeks on the MG-ADL score. Additionally, 40% of patients treated with efgartigimod achieved minimal symptom expression defined as MG-ADL scores of zero (symptom free) or one, compared to 11.1% of patients who received placebo. Among AChR-Ab+ responders, 84.1% showed clinically meaningful improvement on the MG-ADL score within the first two weeks of treatment. The safety profile of efgartigimod was comparable to placebo.
After completing ADAPT, 90% of participants entered ADAPT-plus, an ongoing three-year open-label extension study evaluating the long-term safety and tolerability of efgartigimod. In total across ADAPT and ADAPT-plus, at least 118 patients have been on efgartigimod therapy for 12 months or more.
“The publication of the ADAPT results provides an exciting opportunity to share these data with the clinical community as we aim to introduce a new treatment option for gMG patients. gMG is a chronic, debilitating and potentially life-threatening disease where both the disease symptoms and side effects from current therapies can cause significant impairment on a person’s life,” said
Phase 3 ADAPT Trial
The Phase 3 ADAPT trial was a randomized, double-blind, placebo-controlled, multi-center, global trial evaluating the safety and efficacy of efgartigimod in patients with gMG. A total of 167 adult patients with gMG in
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
Myasthenia gravis (MG) is a rare and chronic autoimmune disease, often 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. There are approximately 65,000 people in
argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancer. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx is evaluating efgartigimod in multiple serious autoimmune diseases. argenx is also advancing several earlier stage experimental medicines within its therapeutic franchises. argenx has offices in
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Joke Comijn (EU)
The contents of this announcement include statements that are, or may be deemed to be, “forward-looking statements.” These forward-looking statements can be identified by the use of forward-looking terminology, including the terms “believes,” “could,” “estimates,” “anticipates,” “expects,” “intends,” “plan,” “may,” “will,” or “should” and include statements argenx makes concerning the Prescription Drug User Fee Act (PDUFA) target action date of