Safety and effectiveness of nipocalimab in adolescent participants in the open label phase 2/3 vibrance-mg clinical study

Topic:

Clinical Trials

Poster Number: P355

Author(s):

Jonathan Strober, MD, UCSF Benioff Children’s Hospital San Francisco, CA, USA, Shawn Black, PhD, Johnson & Johnson, Spring House, PA, USA, Sindhu Ramchandren, MD, MS, Johnson & Johnson, Titusville, NJ, USA, Saunder Bernes, MD, Phoenix Children's Hospital, Phoenix, AZ, USA, Akiyuki Uzawa, MD, PhD, Chiba University Hospital, Chiba, Japan, Yasuhiro Kimoto, MD, University of Miyazaki Hospital, Miyazaki, Japan, Keiko Ishigaki, MD, PhD, Department of Pediatrics, School of Medicine, Tokyo Women's Medical University Hospital, Tokyo, J, Tuan Vu, MD, Department of Neurology, University of South Florida, Tampa, FL, USA, Dan Huang, MSc, Johnson & Johnson, Neuss, Germany, Yaowei Zhu, PhD, Johnson & Johnson, Spring House, PA, USA, Hong Sun, MD, PhD, Johnson & Johnson, Titusville, NJ, USA

Background: Nipocalimab is a fully human, effectorless immunoglobulin G (IgG)1 anti-neonatal Fc receptor (FcRn) monoclonal antibody. Nipocalimab may ameliorate generalized myasthenia gravis (gMG) disease manifestations by selectively targeting FcRn IgG recycling and lowering IgG, including pathogenic autoantibodies in gMG.
Objectives: To evaluate the efficacy and safety of intravenous nipocalimab added to background standard-of-care therapy in adolescents with gMG.
Methods: Seropositive patients (ages 12 to <18 years) with gMG (Myasthenia Gravis Foundation of America [MGFA] Class II-IV) on stable therapy but inadequately controlled, were enrolled in a 24-week open-label study. Nipocalimab was administered as a 30 mg/kg intravenous (IV) loading dose followed by 15 mg/kg IV every 2 Weeks. The primary endpoint was change in total serum IgG. Secondary endpoints included change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scores. Safety was assessed. Results: Of 7 adolescents enrolled, 5 completed 24 weeks. Mean (SD) age was 14.1 (1.86) years, 6 were female, and all 7 adolescents were anti-acetylcholine receptor positive (AChR+). Mean (SD) baseline scores were 4.29 (2.430) for MG-ADL, and 12.50 (3.708) for QMG. Nipocalimab showed a statistically significant reduction in total serum IgG at week 24; the mean (SD) percent change from baseline to week 24 for total serum IgG was –68.98% (7.561). The mean (SD) change at week 24 in MG-ADL was –2.40 (0.418) and in QMG was –3.80 (2.683); 4 of 5 patients achieved minimum symptom expression (MG-ADL score 0–1) by week 24. Nipocalimab was well-tolerated; there were no serious adverse events and no discontinuations due to an adverse event. Conclusion: Nipocalimab demonstrated efficacy and safety in this 6-month trial in seropositive adolescents with gMG. © 2024 AANEM