A new Phase 2 clinical trial sheds light on nipocalimab, an investigational drug that could revolutionize the treatment of Hemolytic Disease of the Fetus and Newborn (HDFN) by reducing the need for risky intrauterine blood transfusions.
Promising data from a new drug trial suggests a potential breakthrough in the treatment of Hemolytic Disease of the Fetus and Newborn (HDFN). An investigational drug, nipocalimab, could significantly delay or even prevent anemia and the necessity for intrauterine blood transfusions in high-risk pregnancies, offering a safer alternative to current methods.
The findings of this Phase 2 clinical trial were recently published in The New England Journal of Medicine.
HDFN occurs when the blood types of a mother and her fetus are incompatible, resulting in the maternal immune system attacking the fetus’s red blood cells, which can lead to severe anemia.
Currently, the standard treatment involves an average of four ultrasound-guided intrauterine blood transfusions during pregnancy. These procedures carry significant risks, including fetal death, premature rupture of membranes and preterm birth.
Kenneth Moise Jr., the lead investigator and maternal fetal medicine specialist, underscored the potential impact of nipocalimab.
“If further studies support using nipocalimab to treat HDFN, it will make treating the fetus in these pregnancies safer and easier for pregnant moms,” Moise, who is also a professor at Dell Medical School at The University of Texas at Austin, said in a news release.
This Phase 2 trial, dubbed the UNITY study, monitored 13 pregnant women who had either experienced a fetal loss or required early intrauterine transfusions in prior pregnancies due to HDFN. Knowing the current fetuses were at high risk, these mothers received intravenous nipocalimab from 14 to 35 weeks of gestation.
Remarkably, more than half of the participants — 54% — had live births at or after 32 weeks without necessitating any transfusions. Some newborns did not require transfusions post-birth either. No cases of fetal hydrops, a severe form of HDFN that markedly lowers survival rates, were reported among the babies.
Nipocalimab functions by blocking the maternal antibodies from crossing the placenta and reducing their levels in the mother’s bloodstream, thereby averting the destruction of the fetus’s red blood cells.
Moise highlighted the broader implications of this drug.
“Nipocalimab is the only drug in development with the potential to treat a variety of alloimmune diseases that affect the fetus, such as fetal/neonatal alloimmune thrombocytopenia and immune-mediated congenital heart block,” he added.
Beyond its potential in maternal-fetal medicine, nipocalimab shows promise in treating a range of autoantibody diseases including rheumatoid arthritis and myasthenia gravis, further underlining its significant scope.
Building on the success of the UNITY study, Johnson & Johnson has launched a Phase 3 pivotal trial, known as AZALEA, to further evaluate nipocalimab’s safety and efficacy. This randomized controlled trial began enrolling participants in late 2023 and is underway in maternal fetal centers globally, with Moise leading the Central Texas segment.
The results of these studies could herald a new era in the treatment of HDFN, sparing countless mothers and babies from the risks associated with current therapeutic methods.