James Howard Jr., MD, Professor of Neurology at the University of North Carolina at Chapel Hill, discusses results from the ADAPT SERON clinical trial of efgartigimod IV in patients with seronegative generalized myasthenia gravis (gMG). Data from this trial was pivotal in efgartigimod’s recent FDA approval.
MG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Common symptoms include weakness of the muscles that control the eye and eyelid, facial expressions, chewing, talking, and swallowing. The condition results from a defect in the transmission of nerve impulses to muscles due to the presence of antibodies against the acetylcholine receptor. The exact reason this occurs is not known.
At the American Academy of Neurology (AAN) 2026 meeting, data was presented on the phase 3 ADAPT SERON clinical trial evaluating the safety and efficacy of efgartigimod IV in adults with anti-MuSK-Ab positive, anti-LRP4-Ab positive, and/or triple seronegative gMG. Efgartigimod alfa is a first-in-class human IgG1 antibody fragment that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating IgG autoantibodies.
In the double-blinded, placebo-controlled part A, adult patients were randomized to receive 4 once-weekly infusions of 10 mg/kg efgartigimod IV or placebo followed by a 5-week follow-up period. Part B included an open-label extension.
Topline results included 119 participants, 58 of whom received efgartigimod IV and 61 received placebo. The change in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score from baseline to week 4 was significantly different between efgartigimod IV (−3.35) and placebo (−1.90) groups. Incremental improvements in MG-ADL total scores were observed during part B over subsequent treatment cycles across all three subgroups. Efgartigimod IV was well tolerated, with no new safety signals observed.
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To learn more about MG and other rare neurological conditions, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/
