Dr. James F. Howard Jr. leads groundbreaking study in myasthenia gravis treatment
The NC TraCS Clinical & Translational Research Center (CTRC) is providing space and highly skilled nursing staff for the treatment visits for the ADAPT SERON study; and the Director of Nursing aides with protocol implementation and continuing logistical support throughout the study.
Dr. James F. Howard Jr., Professor of Neurology at the University of North Carolina at Chapel Hill School of Medicine, has played a pivotal role as Principal Investigator in the ADAPT SERON study—marking a major advancement in the treatment of generalized myasthenia gravis (gMG).
The study, conducted by global immunology company argenx, demonstrated that VYVGART® (efgartigimod alfa-fcab) significantly improves disease activity in patients with AChR-Ab seronegative gMG.
"The results of the ADAPT SERON study, the largest study to date of AChR-Ab seronegative gMG, confirm that VYVGART now has the potential to be a targeted, effective, safe, and necessary treatment for patients living with gMG, regardless of autoantibody status," said 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 SERON trial. "Paired with our existing knowledge, these data demonstrate that pathogenic IgGs are underlying drivers of gMG across patient subtypes. This is a critical advancement in the management of this debilitating and unpredictable disease for patients with limited treatment options."
The ADAPT SERON trial is the first global Phase 3 study to show clinically meaningful improvements across all three AChR seronegative subtypes—MuSK+, LRP4+, and triple seronegative. With a statistically significant primary endpoint (p=0.0068), the findings support a planned submission to the FDA for label expansion by the end of 2025. Dr. Howard also served as the global lead for the primary phase 3 trial that led to regulatory approval of this drug in the US, Europe, Japan, and China.
Dr. Howard emphasized that the data reinforce the role of pathogenic IgGs as underlying drivers of gMG across subtypes, offering new hope for patients who previously had few therapeutic options.
About the ADAPT SERON Study
The ADAPT SERON study looked at how well the medication VYVGART works for people with a rare form of myasthenia gravis (gMG) who don't have the common AChR antibodies. This includes people with MuSK+, LRP4+, or no detectable antibodies at all (triple seronegative).
- Who was in the study?
119 adults from around the world who had been diagnosed with gMG and were already on stable treatment. - What happened in the study?
Participants were randomly given either VYVGART or a placebo (a treatment with no active medicine) through weekly infusions for 4 weeks. After that, they were monitored for 5 weeks. - What was measured?
The main goal was to see if VYVGART helped improve daily activities affected by gMG—like speaking, chewing, breathing, and walking—using a tool called the MG-ADL score. - What happened next?
Everyone in the study had the chance to continue receiving VYVGART in an open-label phase, where all participants knew they were getting the real treatment.
Originally published at med.unc.edu