Nipocalimab shows lasting disease control in adolescents with generalized myasthenia gravis in Phase 2/3 study

Johnson & Johnson has announced promising results from their Phase 2/3 Vibrance-MG study, focusing on the treatment of generalized myasthenia gravis (gMG) in adolescents aged 12 to 17 who are antibody positive for anti-AChR. The study demonstrated that treatment with nipocalimab combined with standard care achieved sustained disease control over a 24-week period. The primary endpoint of the study, a reduction in immunoglobulin G (IgG) levels, and secondary endpoints, which include improvements in MG-ADL and QMG scores, were successfully met.

The results of this investigation will be presented at the Myasthenia Gravis Foundation of America (MGFA) Scientific Session during the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting, where Johnson & Johnson will feature 25 abstracts. Dr. Jonathan Strober, clinical services director for Child Neurology at UCSF Benioff Children's Hospital, remarked on the significance of these findings, emphasizing the importance of this investigational therapy for adolescents with gMG, noting that there is currently no approved advanced treatment option for this age group in the United States.

Myasthenia gravis, an autoimmune disorder, impacts the neuromuscular junction, impeding muscle contraction by disrupting communication between nerves and muscles. About 10% of new myasthenia gravis cases are diagnosed in adolescents, with the disease often presenting more severely in pediatric patients. Treatment with nipocalimab plus standard of care showed a significant reduction in total serum IgG by 69%, alongside improvements in MG-ADL and QMG scores, both of which measure disease activity and severity.

The study revealed that four out of five patients achieved minimal symptom expression by the end of the treatment phase, and nipocalimab was well-tolerated during the six-month period, with no serious adverse events or discontinuations due to adverse events. These findings align with previous studies in adult populations, reinforcing the potential of nipocalimab as a treatment for gMG.

Sindhu Ramchandren, Executive Medical Director for Neuroscience at Johnson & Johnson Innovative Medicine, highlighted the expanding clinical profile of nipocalimab and its potential to address the therapeutic needs of adolescents with gMG. The company has submitted applications to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) seeking approval for nipocalimab for the treatment of gMG.

The Phase 2/3 Vibrance-MG study, an ongoing open-label trial, enrolled seven adolescent participants with gMG who had an insufficient response to stable standard care therapy. Participants receive nipocalimab intravenously every two weeks over a 24-week treatment phase followed by a long-term extension phase. The primary outcomes of the study included total serum IgG reduction, safety and tolerability, and pharmacokinetics in pediatric participants with gMG, with secondary outcomes focused on changes in MG-ADL and QMG scores.

Nipocalimab, an investigational monoclonal antibody, is designed to block FcRn, reducing levels of circulating IgG antibodies, which includes autoantibodies and alloantibodies implicated in various conditions. It has received several key designations from the FDA and EMA, including Fast Track, Orphan Drug, and Breakthrough Therapy designations for multiple indications.

Johnson & Johnson's commitment to developing treatments for autoantibody-driven neurological diseases aims to transform the lives of those affected by conditions like gMG. The company continues to pursue innovations in healthcare to address unmet needs and improve patient outcomes.