P2C310: Nipocalimab in Pregnancies at High Risk for Early-onset Severe Hemolytic Disease of the Fetus and Newborn (EOS-HDFN)

Saturday, October 21, 2023

12:00 PM – 1:30 PM US EDT

Location: Walter E. Washington Convention Center, Exhibit Hall A

Background: The UNITY phase 2 study evaluates the efficacy and safety of nipocalimab, a neonatal Fc receptor blocker, to reduce the risk of fetal anemia, intrauterine transfusions (IUT), and poor outcomes for pregnancies at high risk for EOS-HDFN.

Methods: This open-label, single-arm study (NCT03842189) enrolled individuals with singleton pregnancies at high risk for EOS-HDFN based on a prior obstetric history, high alloantibody titers, and a fetus with an incompatible blood type. Weekly 30 or 45 mg/kg intravenous nipocalimab was administered between 14-35 weeks GA. The primary efficacy endpoint is the proportion of participants with a live birth at ≥32 weeks GA without an IUT. Secondary endpoints for antenatal and postnatal management are reported.

Results: Of 14 pregnancies enrolled, 1 pregnancy was not included due to early elective abortion for an unrelated reason. 54% (7/13) of participants achieved the primary efficacy endpoint of a live birth at >32 weeks GA without IUTs, with a median GA at delivery of 37 1/7 weeks (range: 35 6/7-37 3/7). Twelve of 13 (92.3%) pregnancies resulted in a live birth with a median GA at delivery of 36 5/7 weeks (range: 29 2/7-37 3/7). One pregnancy resulted in fetal demise following complications of an IUT at 22 5/7 weeks GA. None of the 13 pregnancies developed fetal hydrops. Of 12 live births, 11 (92%) neonates received phototherapy and 6 (50%) received ≥1 simple transfusion in the first 12 weeks of life. No maternal or neonatal/infant deaths occurred. Four serious adverse events (SAEs) possibly related to nipocalimab occurred in 2 maternal participants. One neonate delivered at 29 weeks developed an SAE of neonatal respiratory distress.

Conclusion: Results from this first prospective interventional study demonstrate the potential for nipocalimab to impact the antenatal and postnatal management of fetal anemia in pregnancies at high risk for EOS-HDFN.

Presenting Author(s)

Shumyla Saeed-Khawaja, MD (she/her/hers)

Medical Director
Janssen Pharmaceutical Companies of Johnson & Johnson
Raritan, New Jersey

Senior Author(s)

KM

Kenneth J. Moise, Jr., MD

Professor
Dell Medical School, University of Texas, Austin, TX, USA; Comprehensive Fetal Care Center at Dell Children’s Medical Center, Austin, TX, USA
Austin, Texas

Co-Author(s)

LL

Leona E. Ling, PhD

Head of Department
Janssen Pharmaceutical Companies of Johnson & Johnson
Raritan, New Jersey
DO

Dick Oepkes, MD, PhD

Professor
Department of Obstetrics, Division of Fetal Therapy, Leiden University Medical Center
Leiden, Zuid-Holland, Netherlands
EL

Enrico Lopriore, MD, PhD

Professor
Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands
Leiden, Zuid-Holland, Netherlands
ET

Eleonor Tiblad, MD, PhD

Senior Consultant
Center for Fetal Medicine, Karolinska University Hospital, Stockholm, Sweden Clinical Epidemiology Division, Department of Medicine Solna, Karolinska Institutet, Stockholm, Sweden
Stockholm, Sodermanlands Lan, Sweden
EV

EJT (Joanne) Verweij, MD, PhD

Specialist
Department of Obstetrics, Division of Fetal Therapy, Leiden University Medical Center
Leiden, Zuid-Holland, Netherlands
JS

John Smoleniec, MD, PhD

Director
Feto-Maternal Unit, Liverpool Hospital, Liverpool, Australia
Liverpool, New South Wales, Australia
US

Ulrich J. Sachs, MD

Professor
Institute for Clinical Immunology and Transfusion Medicine, Justus Liebig University, Giessen, Germany; Department of Thrombosis and Hemostasis, Giessen University Hospital, Giessen, Germany
Geissen, Hessen, Germany
MK

Mark D. Kilby, DSc, MD

Professor
University of Birmingham, Birmingham, UK; Fetal Medicine Center, Birmingham Women’s and Children’s Foundation Trust, Birmingham, UK; Medical Genomics Research Group, Illumina, UK
Birmingham, England, United Kingdom
RM

Russell S. Miller, MD

Associate Professor
Division of Maternal-Fetal Medicine, Department of Obstetrics and Gynecology, Columbia University Irving Medical Center, New York, NY, USA; NewYork-Presbyterian Hospital, New York, NY, USA
New York, New York
RD

Roland Devlieger, MD, PhD

Gynecologist
Department of Obstetrics and Gynecology, University Hospitals KU Leuven, Leuven, Belgium
Leuven, Vlaams-Brabant, Belgium
JL

Jocelyn H. H. Leu, PharmD, PhD

Director
Janssen Pharmaceutical Companies of Johnson & Johnson
Raritan, New Jersey
AM

Arpana Mirza, MD, MPH

Project Consultant
Janssen Pharmaceutical Companies of Johnson & Johnson
Raritan, New Jersey
VS

Valerie Smith, Mstat

Associate Director
Janssen Pharmaceutical Companies of Johnson & Johnson.
Raritan, New Jersey
LS

Lisa B. Schwartz, MD

Medical Safety Officer
Janssen Pharmaceutical Companies of Johnson & Johnson.
Raritan, New Jersey
MT

May Lee Tjoa, PhD

Global Medical Affair Leader
Janssen Pharmaceutical Companies of Johnson & Johnson.
Raritan, New Jersey
YK

Yosuke Komatsu, MD, PhD

Senior Director
Janssen Pharmaceutical Companies of Johnson & Johnson.
Raritan, New Jersey
JB

James B. Bussel, MD

Professor
Department of Pediatric Oncology/Hematology, New York Presbyterian Hospital/Weill Cornell Medical College, New York, NY, USA
New York, New York