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M281

Phase 2

Hemolytic Disease of the Fetus and Newborn | Small molecule | Hematology |Johnson & Johnson|Last Updated: Mar 25, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment25
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03842189A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Pregnant Women at High Risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn (HDFN)PHASE2 COMPLETED 25Apr 5, 2018Aug 5, 2024Mar 25, 202619 United States, Australia +7
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Study Endpoints
Primary Endpoints
Number of Maternal Participants With Treatment-emergent Adverse Events (TEAEs)
From baseline (Gestational Age [GA] Week 14) up to Postpartum (PP) Week 24 (up to 50 weeks)

An AE was any unfavorable and unintended sign (example, an abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, without any judgment about causality. TEAE was defined as any event occurring after the initiation of the first infusion of nipocalimab.

Number of Neonates/Infants With Adverse Events (AEs)
From Birth (PP Day 0) up to PP Week 96

An AE was any unfavorable and unintended sign (example, an abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, without any judgment about causality.

Number of Maternal Participants With Treatment-emergent Serious Adverse Events (TESAEs)
From baseline (Gestational Age [GA] Week 14) up to PP Week 24 (up to 50 weeks)

SAE was defined as any untoward medical occurrence that resulted in death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, or a congenital anomaly/birth defect. An AE was any unfavorable and unintended sign (example, an abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, without any judgment about causality. TESAEs were any SAEs occurring after the initiation of the first infusion of nipocalimab.

Number of Neonates/Infants With Serious Adverse Events (SAEs)
From Birth (PP Day 0) up to PP Week 96

SAE was defined as any untoward medical occurrence that resulted in death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, or a congenital anomaly/birth defect. An AE was any unfavorable and unintended sign (example, an abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, without any judgment about causality.

Number of Maternal Participants With Treatment-emergent Adverse Events of Special Interest (TEAESIs)
From baseline (Gestational Age [GA] Week 14) up to PP Week 24 (up to 50 weeks)

Number of maternal participants with TEAESIs were reported. An AE was any unfavorable and unintended sign (example, an abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, without any judgment about causality. All infections requiring anti-infective (that is, oral or intravenous antibacterial, antiviral, or antifungal) treatment and with hypoalbuminemia greater than or equal to (\>=) Grade 3 (less than \[\<\]20 gram per liter \[g/L\] by National Cancer Institute of Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 5.0 criteria were considered an AESI for maternal participants. TEAE was defined as any event occurring after the initiation of the first infusion of nipocalimab.

Number of Neonates/Infants With Adverse Events of Special Interest (AESIs)
From birth (PP Day 0) up to PP Week 96

Number of neonates/infants with TEAESIs were reported. An AE was any unfavorable and unintended sign (example, an abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, without any judgment about causality. All infections requiring anti-infective (that is, oral or intravenous antibacterial, antiviral, or antifungal) treatment, unexpected/unusual childhood illnesses and Immunoglobulin G (IgG) concentrations \<200 milligrams per deciliter (mg/dL) at Week 24 through Week 47 or \<300 mg/dL at Week 48 through Week 96 were considered an AESI for neonates and infants.

Maternal Participants: Absolute Value of Electrocardiogram (ECG) Parameter - Mean Ventricular Rate at Baseline
Baseline (GA Week 14)

Absolute value of ECG parameter - mean ventricular rate at baseline in maternal participants was reported. Electrocardiogram assessments included comments on whether the tracings were normal or abnormal, rhythm, presence of arrhythmia or conduction defects, morphology, any evidence of myocardial infarction, or ST segment, T Wave, and U Wave abnormalities.

Maternal Participants: Absolute Value of Electrocardiogram (ECG) Parameter - Mean Ventricular Rate at GA Week 36
GA Week 36

Absolute value of ECG parameter - mean ventricular rate at GA Week 36 in maternal participants was reported. Electrocardiogram assessments included comments on whether the tracings were normal or abnormal, rhythm, presence of arrhythmia or conduction defects, morphology, any evidence of myocardial infarction, or ST segment, T Wave, and U Wave abnormalities.

Maternal Participants: Change From Baseline in ECG Parameter- Mean Ventricular Rate
Baseline (GA Week 14) and GA Week 36

Change from baseline in ECG parameter- mean ventricular rate in maternal participants was reported. Electrocardiogram assessments included comments on whether the tracings were normal or abnormal, rhythm, presence of arrhythmia or conduction defects, morphology, any evidence of myocardial infarction, or ST segment, T Wave, and U Wave abnormalities.

Number of Maternal Participants With Treatment-emergent (TE) Clinically Important Laboratory and Biomarker Immunoglobulin G (IgG) Values Over Time
From baseline (Gestational Age [GA] Week 14) up to PP Week 24 (up to 50 weeks)

Laboratory parameters included hematology, chemistry, blood Lipids panel, and Immunoglobulin G (IgG) parameters. TEAE was defined as any event occurring after the initiation of the first infusion of nipocalimab. Here, HDL: high-density lipoprotein, LDL: low-density lipoprotein. The during-pregnancy value of albumin \<20 g/L was from a local laboratory and was \>=20 g/L when analyzed at the central laboratory for the same time point.

Number of Neonates or Infants With Clinically Important Laboratory and Biomarker Immunoglobulin G (IgG) Values Over Time
From Birth (PP Day 0) up to PP Week 96

Laboratory parameters included total bilirubin and biomarker included immunoglobulin G (IgG).

Maternal Participants: Absolute Value of Vital Signs - Body Temperature at Baseline
Baseline (GA Week 14)

Absolute value of vital signs - body temperature at baseline in maternal participants was reported.

Maternal Participants: Absolute Value of Vital Signs - Body Temperature at GA Week 36
GA Week 36

Absolute value of vital signs - body temperature at GA Week 36 in maternal participants was reported.

Maternal Participants: Absolute Value of Vital Signs - Body Temperature at PP Week 24
PP Week 24

Absolute value of vital signs - body temperature at PP Week 24 in maternal participants was reported.

Maternal Participants: Change From Baseline in Vital Sign - Body Temperature
Baseline (GA Week 14), GA Week 36, and PP Week 24

Change from baseline in vital signs- body temperature in maternal participants was reported.

Maternal Participants: Absolute Value of Vital Signs - Respiratory Rate at Baseline
Baseline (GA Week 14)

Absolute value of vital signs -respiratory rate at baseline in maternal participants was reported.

Maternal Participants: Absolute Value of Vital Signs - Respiratory Rate at GA Week 36
GA Week 36

Absolute value of vital signs -respiratory rate at GA Week 36 in maternal participants was reported.

Maternal Participants: Absolute Value of Vital Signs - Respiratory Rate at PP Week 24
PP Week 24

Absolute value of vital signs - respiratory rate at PP Week 24 in maternal participants was reported.

Maternal Participants: Change From Baseline in Vital Sign - Respiratory Rate
Baseline (GA Week 14), GA Week 36, and PP Week 24

Change from baseline in vital signs- respiratory rate in maternal participants was reported.

Maternal Participants: Absolute Value of Vital Signs - Pulse Rate at Baseline
Baseline (GA Week 14)

Absolute value of vital signs - pulse rate at baseline in maternal participants was reported.

Maternal Participants: Absolute Value of Vital Signs - Pulse Rate at GA Week 36
GA Week 36

Absolute value of vital signs -pulse rate at GA Week 36 in maternal participants was reported.

Maternal Participants: Absolute Value of Vital Signs -Pulse Rate at PP Week 24
PP Week 24

Absolute value of vital signs -pulse rate at PP Week 24 in maternal participants was reported.

Maternal Participants: Change From Baseline in Vital Sign - Pulse Rate
Baseline (GA Week 14), GA Week 36, and PP Week 24

Change from baseline in vital signs -pulse rate in maternal participants was reported.

Maternal Participants: Absolute Value of Vital Signs - Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) at Baseline
Baseline (GA Week 14)

Absolute value of vital signs - SBP and DBP at baseline in maternal participants was reported.

Maternal Participants: Absolute Value of Vital Signs - Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) at GA Week 36
GA Week 36

Absolute value of vital signs -SBP and DBP at GA Week 36 in maternal participants was reported.

Maternal Participants: Absolute Value of Vital Signs - Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) at PP Week 24
PP Week 24

Absolute value of vital signs - SBP and DBP at PP Week 24 in maternal participants was reported.

Maternal Participants: Change From Baseline in Vital Sign - Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)
Baseline (GA Week 14), GA Week 36, and PP Week 24

Change from baseline in vital signs -SBP and DBP in maternal participants were reported.

Maternal Participants: Absolute Value of Vital Signs - Body Weight at Baseline
Baseline (GA Week 14)

Absolute value of vital signs - body weight at baseline in maternal participants was reported.

Maternal Participants: Absolute Value of Vital Signs - Body Weight at GA Week 36
GA Week 36

Absolute value of vital signs -body weight at GA Week 36 in maternal participants was reported.

Maternal Participants: Absolute Value of Vital Signs - Body Weight at PP Week 24
PP Week 24

Absolute value of vital signs included body weight at PP Week 24 in maternal participants was reported.

Maternal Participants: Change From Baseline in Vital Sign - Body Weight
Baseline (GA Week 14), GA Week 36, and PP Week 24

Change from baseline in vital signs- body weight in maternal participants was reported.

Neonates/Infants: Absolute Value in Vital Signs - Body Temperature at Baseline
Baseline (PP Day 0)

Absolute value in vital signs parameter -body temperature at baseline was reported for all neonates/infants.

Neonates/Infants: Absolute Value of Vital Signs - Body Temperature at PP Week 1
PP Week 1

Absolute value of vital signs parameter -body temperature at PP Week 1 was reported for all neonates/infants.

Neonates/Infants: Absolute Value in Vital Signs - Body Temperature at PP Week 4
PP Week 4

Absolute value in vital signs parameter - body temperature at PP Week 4 was reported for all neonates/infants.

Neonates/Infants: Absolute Value in Vital Signs - Body Temperature at PP Week 24
PP Week 24

Absolute value in vital signs parameter -body temperature at PP Week 24 was reported for all neonates/infants.

Neonates/Infants: Change From Baseline in Vital Signs - Body Temperature
Baseline (PP Day 0), PP Weeks 1, 4, and 24

Change from baseline in vital signs- body temperature were reported for all neonates/infants.

Neonates/Infants: Absolute Value of Vital Signs - Body Weight at Baseline
Baseline (PP Day 0)

Absolute value of vital signs parameter- body weight at baseline was reported for all neonates/infants.

Neonates/Infants: Absolute Value of Vital Signs - Body Weight at PP Week 1
PP Week 1

Absolute value of vital signs parameter - body weight at PP Week 1 was reported for all neonates/infants.

Neonates/Infants: Absolute Value of Vital Signs - Body Weight at PP Week 4
PP Week 4

Absolute value of vital signs parameter - body weight at PP Week 4 was reported for all neonates/infants.

Neonates/Infants: Absolute Value of Vital Signs - Body Weight at PP Week 24
PP Week 24

Absolute value of vital signs parameter - body weight at PP Week 24 was reported for all neonates/infants.

Neonates/Infants: Change From Baseline in Vital Signs -Body Weight
Baseline (PP Day 0), PP Weeks 1, 4, and 24

Change from baseline in vital signs - body weight were reported for all neonates/infants.

Neonates/Infants: Absolute Value of Vital Signs - Respiratory Rate at Baseline
Baseline (PP Day 0)

Absolute value of vital signs parameter -respiratory rate at baseline was reported for all neonates/infants.

Neonates/Infants: Absolute Value of Vital Signs - Respiratory Rate at PP Week 1
PP Week 1

Absolute value of vital signs parameter - respiratory rate at PP Week 1 was reported for all neonates/infants.

Neonates/Infants: Absolute Value of Vital Signs - Respiratory Rate at PP Week 4
PP Week 4

Absolute value of vital signs parameter - respiratory rate at PP Week 4 was reported for all neonates/infants.

Neonates/Infants: Absolute Value of Vital Signs - Respiratory Rate at PP Week 24
PP Week 24

Absolute value of vital signs parameter- respiratory rate at PP Week 24 was reported for all neonates/infants.

Neonates/Infants: Change From Baseline in Vital Signs - Respiratory Rate
Baseline (PP Day 0), PP Weeks 1, 4, and 24

Change from baseline in vital signs -respiratory rate was reported for all neonates/infants.

Neonates/Infants: Absolute Value of Vital Sign - Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) at Baseline
Baseline (PP Day 0)

Absolute value of vital signs parameter - SBP and DBP at baseline were reported for all neonates/infants.

Neonates/Infants: Absolute Value of Vital Sign - Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) at PP Week 1
PP Week 1

Absolute value of vital signs parameter - SBP and DBP at PP Week 1 were reported for all neonates/infants.

Neonates/Infants: Absolute Value of Vital Sign - Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) at PP Week 4
PP Week 4

Absolute value of vital signs parameter -SBP and DBP at PP Week 4 were reported for all neonates/infants.

Neonates/Infants: Absolute Value of Vital Sign - Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) at PP Week 24
PP Week 24

Absolute value of vital signs parameter -SBP and DBP at PP Week 24 were reported for all neonates/infants.

Neonates/Infants: Change From Baseline in Vital Signs - Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)
Baseline (PP Day 0), PP Weeks 1, 4, and 24

Change from baseline in vital signs- SBP and DBP were reported for all neonates/infants.

Percentage of Maternal Participants With Intrauterine Growth Restriction (IUGR) Based on Ultrasound Assessments
Baseline (GA Week 14), GA Weeks 16, 18, 20, 22, 24, 26, 28, 30, 32, 34, and 36

Percentage of maternal participants with intrauterine growth restriction (IUGR) based on ultrasound assessments and guidelines from American College of Obstetricians and Gynecologists, and Society for Maternal-Fetal Medicine was reported. This outcome measure provided the incidence of with IUGR at delivery. IUGR is defined as weight below the 10th percentile for gestational age based on the World Health Organization (WHO) fetal growth curve.

Percentage of Maternal Participants With Abnormal Amniotic Fluid Values: Amniotic Fluid Index (AFI) at Baseline
Baseline (GA Week 14)

Percentage of maternal participants with abnormal amniotic fluid values: amniotic fluid index (AFI) at baseline was reported. The amniotic fluid volume abnormality was categorized as an AFI \<5 centimeter (cm) or \>24 cm.

Percentage of Maternal Participants With Abnormal Amniotic Fluid Values: AFI at GA Week 26
GA Week 26

Percentage of maternal participants with abnormal amniotic fluid values: AFI at GA Week 26 was reported. The amniotic fluid volume abnormality was categorized as an AFI \<5 cm or \>24 cm.

Percentage of Maternal Participants With Abnormal Amniotic Fluid Values: AFI at GA Week 36
GA Week 36

Percentage of maternal participants with abnormal amniotic fluid values: AFI at GA Week 36 was reported. The amniotic fluid volume abnormality was categorized as an AFI \<5 cm or \>24 cm.

Percentage of Maternal Participants With Abnormal Amniotic Fluid Values: Maximum Vertical Pocket (MVP) at Baseline
From Baseline (GA Week 14)

Percentage of maternal participants with abnormal amniotic fluid values: maximum vertical pocket (MVP) at baseline was reported. The amniotic fluid volume abnormality was categorized as MVP \<2 cm or \>8 cm.

Percentage of Maternal Participants With Abnormal Amniotic Fluid Values: MVP at GA Week 18
GA Week 18

Percentage of maternal participants with abnormal amniotic fluid values: MVP at GA Week 18 was reported. The amniotic fluid volume abnormality was categorized as MVP \<2 cm or \>8 cm.

Percentage of Maternal Participants With Abnormal Amniotic Fluid Values: MVP at GA Week 22
GA Week 22

Percentage of maternal participants with abnormal amniotic fluid values: MVP at GA Week 22 was reported. The amniotic fluid volume abnormality was categorized as MVP \<2 cm or \>8 cm.

Number of Neonates/Infants With Appearance, Pulse, Grimace Response, Activity, Respiration (Apgar) Score
1, 5, and 10 minutes after birth at PP Day 0

Number of neonates/infants with Apgar score from 1 to 10 minutes of life were reported. The system provided a standardized assessment for infants after delivery. The Apgar score comprises five components: 1) color, 2) heart rate, 3) reflexes, 4) muscle tone, and 5) respiration, each of which is given a score of 0, 1, or 2. The score is reported at 1 minute and 5 minutes after birth for all infants, and at 5-minute intervals thereafter until 20 minutes for infants with a score less than 7. This is using an Apgar scale which ranges from minimum total score of 0 and maximum total score of 10, with higher score representing a better outcome.

Number of Maternal Participants With Concomitant Medications and Therapies
From baseline (Gestational Age [GA] Week 14) up to PP Week 24 (up to 50 weeks)

Number of maternal participants with concomitant medications and therapies were reported.

Number of Neonates/Infants With Concomitant Medications and Therapies
From birth (PP Day 0) up to PP Week 96

Number of neonates/infants with concomitant medications and therapies were reported.

Percentage of Maternal Participants With Live Birth at or After Gestational Age (GA) Week 32 and Without an Intrauterine Transfusion (IUT) Throughout Their Entire Pregnancies
From baseline (GA Week 14) up to GA Week 37

Percentage of maternal participants with live birth at or after GA Week 32 and without an IUT throughout their entire pregnancies were reported.

Secondary Endpoints
Percentage of Maternal Participants With Live Birth
From baseline (GA Week 14) up to GA Week 37
Percentage of Maternal Participants Without an Intrauterine Transfusion (IUT) Before Gestational Age (GA) Week 24
From baseline (GA Week 14) up to GA Week 24
Maternal Participants : Gestational Age (GA) at First Intrauterine Transfusion (IUT)
From baseline (GA Week 14) up to GA Week 37
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
M281EXPERIMENTAL -
Interventions
NameTypeDescription
M281DRUGParticipants will receive once weekly intravenous (IV) infusions of M281
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Eligibility Criteria
Age Range18 Years — N/A
SexFEMALE
Healthy VolunteersNo
Study Sites19

Inclusion Criteria: * Approximately 15 eligible participants and their offspring will be enrolled * Each participant must meet all of the following criteria to be enrolled in the study: * Female and greater than or equal to (\>=)18 years of age * Pregnant to an estimated gestational age of between ...

Countries:United StatesAustraliaBelgiumCanadaGermanyNetherlandsSpainSwedenUnited Kingdom
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