Leonard Dragone, MD, PhD, Disease Area Leader at Johnson & Johnson, discusses how nipocalimab is being incorporated into generalized MG care, what long-term data show, and where J&J sees the therapy’s role evolving.
Nipocalimab (Imaavy; Johnson & Johnson) received FDA approval for the treatment of generalized myasthenia gravis in adults and pediatric patients 12 years of age and older who are anti-AChR or anti-MuSK antibody positive, and evidence supporting its use continues to grow. At the
Research presented at the meeting also offered insight into how fluctuating symptoms are influencing treatment decisions and transitions between advanced therapies in gMG. Following the meeting, NeurologyLive® caught up with Leonard Dragone, MD, PhD, Disease Area Leader for Autoantibody and Rheumatology at Johnson & Johnson, who is closely involved in the ongoing development and evidence generation program for nipocalimab across multiple conditions.
In this Q&A, Dragone discussed what J&J has learned about how nipocalimab is being used in clinical practice since approval, what neurologists should keep in mind regarding long-term safety and monitoring, and how the company sees the therapy’s role evolving, including through the PETUNIA pregnancy study and the EPIC head-to-head comparison of FcRn blockers.
Leonard Dragone, MD, PhD: Since nipocalimab’s approval for the treatment of generalized myasthenia gravis (gMG) in adults and pediatric patients 12 years of age and older who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive we're continuing to evolve our understanding of how neurologists are incorporating targeted, immunoselective therapies into individualized treatment decisions for people living with gMG.
One of the clearest themes is the importance of sustained disease control. Research presented at AANEM this year provides insight into how fluctuating symptoms and the resulting unmet need for more sustained disease control may influence treatment decisions and transitions between advanced treatments in gMG.
We're also building evidence across different patient populations. For example, two-year findings from our Phase 2/3 Vibrance-MG study of IMAAVY in adolescents demonstrated sustained disease control including improvements in daily function and muscle strength observed through Week 108, as well as reduction or discontinuation of corticosteroids in anti-AcHr+ adolescents with gMG.
We're focused on continuing to build our understanding of outcomes across the breadth of gMG, including efficacy earlier in the disease course and in patients with differing symptom severity, ocular involvement and antibody profiles.
We are continuing to collect long-term evidence for nipocalimab. The Vivacity-MG3 open-label extension (OLE) has provided up to 120 weeks of observation, showing sustained clinical improvements with a safety profile consistent with previous nipocalimab studies. Two-year findings from the Vibrance-MG study in adolescents were also consistent with the established safety profile, with no new safety signals identified.
The chronic nature of gMG means it’s important to understand whether clinical benefit can be maintained over time and how corticosteroid use may change with longer-term treament. Our Vivacity-MG3 study is helping us build that picture, including through analyses of sustained response with nipocalimab as well as corticosteroid use during the OLE.
We're also continuing to build evidence on how sustained disease control translates across the range of gMG symptoms that can affect patients’ daily lives; from ocular symptoms, such as drooping eyelids and double vision, to weakness which, can affect the limbs, speech, swallowing and breathing.These comprehensive analyses, together with long-term follow-up and real-world experience, will help us further characterize the impact of nipocalimab over time and inform individualized patient care.
Our goal has always been to help more people living with gMG achieve sustained disease control and improvements in daily function. As the treatment landscape advances, we’re focused on building the evidence to better understand how nipocalimab may address unmet need across different patient populations and clinical classification and distinct treatment needs.
One important area of special interest is pregnancy. Around 50 percent of individuals living with gMG are women, and about one in five of those women with gMG are of childbearing potential, yet there remains an important evidence gap around treatment during pregnancy. Our PETUNIA study is designed to help address this gap by collecting real-world pregnancy, maternal, neonatal and infant outcomes following exposure to IMAAVY in pregnant individuals with gMG. PETUNIA will follow reported pregnancies and, for live births, infants through one year of life.
Separately, we can draw on our scientific experience from our maternal-fetal program, where J&J is investigating nipocalimab in Phase 3 programs in hemolytic disease of the fetus and newborn (HDFN) and fetal and neonatal alloimmune thrombocytopenia (FNAIT). The Phase 2 UNITY study in HDFN, for example, has provided important fetal and neonatal pharmacokinetic data, with low to undetectable concentrations of nipocalimab observed in available samples, indicating minimal fetal drug exposure during pregnancy. While findings from these distinct diseases and studies cannot be extrapolated to pregnant patients with gMG, they contribute to a broader scientific understanding of nipocalimab in the maternal-fetal setting.
More broadly, we’re continuing to generate long-term, real-world and comparative evidence for IMAAVY in gMG to help neurologists make informed treatment decisions. The EPIC study, the first head-to-head comparison of FcRn blockers for patients with gMG in this setting, including a treatment-switch arm, is an important part of that effort. Studies like EPIC can help generate comparative evidence neurologists need to inform treatment choice and better understand which treatment may be appropriate for which patient and when.
