Johnson & Johnson has announced positive safety and efficacy results in a phase 2 trial evaluating nipocalimab against early-onset severe hemolytic disease of the fetus and newborn.
The phase 2 open-label UNITY clinical trial has shown positive results for treating early-onset severe (EOS) hemolytic disease of the fetus and newborn (HDFN) risk in pregnant individuals using nipocalimab (Johnson & Johnson).
HDFN is when fetal red blood cells are attacked by alloantibodies that crossed the placenta from a pregnant individual’s immune system. In certain cases, the disease is life threatening, and can lead to conditions such as hyperbilirubinemia, neonatal jaundice, and fetal anemia.
Currently, there are no nonsurgical EOS HDFN interventions available in the United States, making repeated intrauterine transfusions (IUTs) often necessary to treat patients. This procedure is invasive and associated with increased risks of premature birth and fetal mortality.
Nipocalimab is an antibody designed for use in patients at high risk of EOS HDFN. It lowers the circulation of immunoglobulin G antibodies by blocking the Fc receptor (FcRn), reducing EOS HDFN risk. Nipocalimab is the only FcRN currently being developed for patients with high HDFN risk.
The UNITY trial was conducted to evaluate the safety, efficacy, and pharmacokinetics of nipocalimab in pregnant patients with a high EOS HDFN risk. Participants wereRhD (D) or Kell (K) alloimmunized and had singleton pregnancies with a high risk of EOS HDFN.
Intravenous infusions were given to patients once per week. Live birth at 32 weeks’ gestation or greater without the need for an IUT during pregnancy was the primary outcome of the study, with safety monitoring occurring during a 24-week follow-up period after delivery for maternal patients, and up to 96 weeks of follow-up for infants.
The primary outcome was reached by 54% of patients receiving nipocalimab, compared to 10% in the data used for reference. The mean gestational age at delivery in patients reaching the endpoint was 37 and 1/7 weeks.
Safety data was also positive, with adverse events not deviating from those commonly reported during pregnancy. Most serious adverse events (SAEs) were related to HDFN or other conditions associated with pregnancy. There were 2 patients completing live births with SAEs linked to treatment, one of which was a subchorionic hematoma, and the other premature placenta separation.
Overall, positive safety and efficacy was observed in patients receiving nipocalimab. This indicates nipocalimab may be used as a non-surgical HDFN option in the future.
New phase 2 data demonstratepotential benefit of nipocalimab for pregnant individuals at high risk of early-onset severe hemolytic disease of the fetus and newborn (HDFN). The Janssen Pharmaceutical Companies of Johnson & Johnson. June 26, 2023. Accessed June 26, 2023. https://www.prnewswire.com/news-releases/new-phase-2-data-demonstrate-potential-benefit-of-nipocalimab-for-pregnant-individuals-at-high-risk-of-early-onset-severe-hemolytic-disease-of-the-fetus-and-newborn-hdfn-301863068.html