Johnson & Johnson has announced significant findings from their Phase 2 UNITY study of nipocalimab for the treatment of pregnant individuals at high risk for early onset severe hemolytic disease of the fetus and newborn (HDFN). The results, published in The New England Journal of Medicine, indicate that nipocalimab can delay or prevent severe fetal anemia and improve birth outcomes.
HDFN occurs when the maternal immune system produces antibodies against fetal red blood cells, causing anemia and other complications. Severe cases often require repeated IUTs, which carry significant risks. Nipocalimab, an investigational monoclonal antibody, is designed to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies, preventing transplacental transfer of harmful maternal antibodies.
Nipocalimab has received several key designations from the FDA and EMA, including Fast Track and Orphan Drug statuses, highlighting its potential as a groundbreaking treatment for HDFN and related conditions.
The Phase 2 UNITY study, a multicenter, open-label, single-arm trial, assessed the safety and efficacy of intravenous nipocalimab from 14-35 weeks of gestation in pregnancies at high risk for recurrent early onset severe (EOS) HDFN. The study met its primary endpoint with 54% of participants achieving a live birth at or after 32 weeks of gestation without needing an intrauterine transfusion (IUT). This is a notable improvement over the historical benchmark of 10%.
The study demonstrated that nipocalimab could establish a protective effect against severe fetal anemia, reducing the need for invasive IUTs and other in-utero procedures, which are associated with high treatment burdens and risks to fetal life.
Dr. Kenneth J. Moise Jr., lead study investigator and Professor at the Department of Women’s Health, Dell Medical School, University of Texas at Austin, commented, “The Phase 2 data published in the NEJM are encouraging, as the results support the potential of nipocalimab in the treatment of pregnant individuals with a history of severe HDFN, helping to establish a path forward for further development in this disease in a larger scale Phase 3 study.”
Nipocalimab is currently the only therapy in clinical development for HDFN, a serious condition caused by maternal-fetal blood type incompatibility, leading to fetal anemia and other severe complications, according to Johnson & Johnson.
The company is progressing with the AZALEA Phase 3 pivotal study, which is currently enrolling patients to further assess the efficacy and safety of nipocalimab in pregnancies at risk for severe HDFN. Additionally, a Phase 3 study is being conducted for fetal and neonatal alloimmune thrombocytopenia (FNAIT), a related condition where maternal antibodies attack fetal platelets.
Katie Abouzahr, M.D., Vice President, Autoantibody Diseases and Maternal-Fetal Immunology Disease Area Leader at Johnson & Johnson, emphasized: “The data published in the NEJM underscore the potential of nipocalimab to address the high unmet medical need in severe HDFN, a serious, life-threatening and rare condition in which no other therapies in clinical development exist.”
