Johnson & Johnson’s (J&J) investigational FcRn blocker nipocalimab has demonstrated sustained disease control in adolescents with the rare autoimmune disease generalised myasthenia gravis (gMG), according to phase 2/3 results shared by the company.
The ongoing Vibrance-MG study has been evaluating the candidate in gMG patients aged 12 to 17 years who have had an insufficient clinical response to ongoing, stable standard of care (SOC) therapy.
In myasthenia gravis, autoantibodies target proteins at the neuromuscular junction, disrupting the ability of the nerves to stimulate the muscles and resulting in weakness. The disease affects an estimated 700,000 people worldwide, with 85% of these patients experiencing the more extensive form of the disease, gMG.
Approximately 10% of new myasthenia gravis cases are diagnosed in adolescents and the severity of gMG in paediatric patients is heightened. Other than symptomatic treatments, there are currently no approved FcRn blockers that may address the root cause of the disease for this patient population in the US.
Findings from Vibrance-MG, which will be presented at this year’s American Association of Neuromuscular and Electrodiagnostic Medicine Annual Meeting, demonstrated reductions in immunoglobulin G (IgG) in adolescents who received nipocalimab infusions for 24 weeks.
Nipocalimab plus SOC resulted in a 69% reduction in total serum IgG, meeting the study’s primary endpoint. Two secondary endpoints assessing measures of disease activity were also achieved, with four of five patients experiencing minimum symptom expression by the end of their treatment phase.
The data is consistent with findings from the pivotal study of nipocalimab in adult patients with gMG, J&J said, adding that it has already submitted regulatory applications to the US Food and Drug Administration and European Medicines Agency for nipocalimab in gMG.
Sindhu Ramchandren, executive medical director, neuroscience, J&J Innovative Medicine, said: “The Vibrance-MG data adds to the expanding clinical profile of nipocalimab and highlight its potential for adolescents living with gMG who are in need of new treatments.”