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Clinical Trial Results:
An Open-label Extension Study of MOM-M281-004 to Evaluate the Safety, Tolerability, and Efficacy of M281 Administered to Patients with Generalized Myasthenia Gravis

Summary
EudraCT number	2018-003618-41
Trial protocol	GB DE BE ES PL IT
Global end of trial date	22 Jun 2020

Results information
Results version number	v2(current)
This version publication date	29 Jun 2023
First version publication date	23 Dec 2021
Other versions	v1
Version creation reason

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

MOM-M281-005

ISRCTN number

US NCT number

NCT03896295

WHO universal trial number (UTN)

Sponsor organisation name

Momenta Pharmaceuticals, Inc.

Sponsor organisation address

301 Binney Street, Cambridge, United States, MA02142

Public contact

Clinical Registry group, Momenta Pharmaceuticals, Inc., ClinicalTrialsEU@its.jnj.com

Scientific contact

Clinical Registry group, Momenta Pharmaceuticals, Inc., ClinicalTrialsEU@its.jnj.com

Is trial part of an agreed paediatric investigation plan (PIP)

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Analysis stage

Final

Date of interim/final analysis

22 Jun 2020

Is this the analysis of the primary completion data?

Global end of trial reached?

Yes

Global end of trial date

22 Jun 2020

Was the trial ended prematurely?

Yes

Main objective of the trial

The main objective of the trial was to evaluate the long-term safety and tolerability of nipocalimab in subjects with generalized myasthenia gravis (gMG).

Protection of trial subjects

This study was conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki and that are consistent with Good Clinical Practice (GCP) and applicable regulatory requirements. Safety assessments included collection of adverse events (AEs) and serious AEs (SAEs), adverse events of special interests (AESIs), clinical laboratory testing (including chemistry, hematology, coagulation, and urinalysis), vital signs, physical examinations, electrocardiogram (ECG) findings, and the Columbia-Suicide Severity Rating Scale (C-SSRS).

Background therapy

Evidence for comparator

Actual start date of recruitment

11 Feb 2019

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

Germany: 2

Country: Number of subjects enrolled

Spain: 11

Country: Number of subjects enrolled

Italy: 2

Country: Number of subjects enrolled

Poland: 7

Country: Number of subjects enrolled

United States: 15

Worldwide total number of subjects

EEA total number of subjects

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

Adolescents (12-17 years)

Adults (18-64 years)

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

Screening details

Subjects from study MOM-M281-004 (NCT03772587) who completed the Day 113 visit of that study were eligible to enroll in this open-label extension study (MOM-M281-005). The Day 113 visit of MOM-M281-004 occurred approximately 8 weeks after the last dose in that study.

Period 1 title

Overall Study (overall period)

Is this the baseline period?

Yes

Allocation method

Non-randomised - controlled

Blinding used

Not blinded

Are arms mutually exclusive

Yes

Placebo-Nipocalimab

Arm description

Subjects who received placebo in MOM-M281-004 study (NCT03772587) rolled-over and received intravenous (IV) infusion of nipocalimab 30 milligrams per kilogram (mg/kg) every 4 weeks (Q4W) starting Day 1 up to 8 weeks. After 8 weeks of treatment on a stable dose of nipocalimab, the dose and/or dosing frequency could be individually adjusted, at the investigator's discretion to receive maximum dose of 60 mg/kg at a frequency of not exceeding every 2 weeks (Q2W).

Arm type

Experimental

Investigational medicinal product name

Nipocalimab

Investigational medicinal product code

Other name

Pharmaceutical forms

Injection

Routes of administration

Intravenous use

Dosage and administration details

Nipocalimab 30 mg/kg was administered once Q4W as IV infusion starting Day 1 up to 8 weeks. The dose and/or dosing frequency could be individually adjusted per investigator's discretion to receive maximum dose of 60 mg/kg at a frequency of not exceeding Q2W after 8 weeks of treatment.

Nipocalimab-Nipocalimab

Arm description

Subjects who received nipocalimab in MOM-M281-004 study (NCT03772587) rolled-over and received IV infusion of nipocalimab 30 mg/kg Q4W starting Day 1 up to 8 weeks. After 8 weeks of treatment on a stable dose of nipocalimab, the dose and/or dosing frequency could be individually adjusted, at the investigator's discretion to receive maximum dose of 60 mg/kg at a frequency of not exceeding every 2 weeks (Q2W).

Arm type

Experimental

Investigational medicinal product name

Nipocalimab

Investigational medicinal product code

Other name

Pharmaceutical forms

Injection

Routes of administration

Intravenous use

Dosage and administration details

Number of subjects in period 1	Placebo-Nipocalimab	Nipocalimab-Nipocalimab
Started	7	30
Completed	0	0
Not completed	7	30
Adverse event, serious fatal	-	1
Covid-19	7	29

Baseline characteristics

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Reporting group title

Placebo-Nipocalimab

Reporting group description

Reporting group title

Nipocalimab-Nipocalimab

Reporting group description

Reporting group values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Total
Number of subjects	7	30	37
Title for AgeCategorical
Units: subjects
Children (2-11 years)	0	0	0
Adolescents (12-17 years)	0	0	0
Adults (18-64 years)	5	20	25
From 65 to 84 years	2	10	12
85 years and over	0	0	0
Title for AgeContinuous
Units: years
arithmetic mean (standard deviation)	53.7 ± 20.39	53 ± 16.93	-
Title for Gender
Units: subjects
Female	4	18	22
Male	3	12	15

End points

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Reporting group title

Placebo-Nipocalimab

Reporting group description

Reporting group title

Nipocalimab-Nipocalimab

Reporting group description

Subject analysis set title

Nipocalimab (All Subjects)

Subject analysis set type

Full analysis

Subject analysis set description

All subjects who received placebo or nipocalimab in MOM-M281-004 study rolled-over and received IV infusion of nipocalimab 30 mg/kg Q4W starting Day 1 up to 8 weeks. After 8 weeks of treatment on a stable dose of nipocalimab, the dose and/or dosing frequency could be individually adjusted, at the investigator's discretion to receive maximum dose of 60 mg/kg at a frequency of not exceeding Q2W.

Primary: Number of Subjects with Treatment-emergent Adverse Events (TEAEs)

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End point title

Number of Subjects with Treatment-emergent Adverse Events (TEAEs) ^[1]

End point description

Number of subjects with TEAEs were reported. An adverse event (AE) is any untoward medical event that occurs in a subject administered an investigational product and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. TEAEs are defined as any AE occurring during or after the initiation of the first infusion of study drug in this study. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Up to 257 days post-baseline (Baseline is Day 1)

Notes
[1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	7	30	37
Units: subjects	4	18	22

No statistical analyses for this end point

Primary: Number of Subjects with Treatment-emergent Serious Adverse Events (SAEs)

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End point title

Number of Subjects with Treatment-emergent Serious Adverse Events (SAEs) ^[2]

End point description

An adverse event (AE) is any untoward medical event that occurs in a subject administered an investigational product and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. An SAE is defined as any AE occurring at any dose that results in any of the following outcomes: death, life-threatening AE, hospitalization or prolongation of existing hospitalization, a persistent or significant disability/incapacity, or a congenital anomaly/birth defect. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Up to 257 days post-baseline

Notes
[2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	7	30	37
Units: subjects	1	4	5

No statistical analyses for this end point

Primary: Number of Subjects with Treatment-emergent Adverse Events of Special Interest (AESIs)

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End point title

Number of Subjects with Treatment-emergent Adverse Events of Special Interest (AESIs) ^[3]

End point description

Number of subjects with treatment-emergent AESIs were reported. Severe infections and hypoalbuminemia (Grade 3 or higher according to the Common Terminology Criteria for Adverse Events [CTCAE] v5.0) were considered as AESIs. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Up to 257 days post-baseline

Notes
[3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	7	30	37
Units: subjects	1	1	2

No statistical analyses for this end point

Primary: Number of Subjects with Treatment-emergent Abnormal Vital Signs

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End point title

Number of Subjects with Treatment-emergent Abnormal Vital Signs ^[4]

End point description

Number of subjects with treatment-emergent abnormal vital signs including pulse rate (less than or equal to [<=] 50 beats per minutes [bpm] with greater than or equal to [>=] 15 bpm decrease from baseline, >= 120 bpm with >=15 bpm increase from baseline), systolic blood pressure (SBP) (<= 90 millimeters of mercury [mmHg] with >= 20 mmHg decrease from baseline, >= 160 mmHg with >= 20 mmHg increase from baseline) and diastolic blood pressure (DBP) (<= 50 mmHg with >=15 mmHg decrease from baseline, >=100 mmHg with >=15 mmHg decrease from baseline) were reported. Safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Up to 257 days post-baseline

Notes
[4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	7	30	37
Units: subjects
PR<=50 with >=15 bpm decrease from baseline	0	0	0
PR>= 120 with >=15 bpm increase from baseline	0	0	0
SBP<=90 with >=20 mmHg decrease from baseline	0	1	1
SBP>=160 with >=20 mmHg increase from baseline	0	1	1
DBP<=50 with >=15 mmHg decrease from baseline	0	2	2
DBP>=100 with >=15 mmHg decrease from baseline	0	0	0

No statistical analyses for this end point

Primary: Number of Subjects with Abnormalities in Physical Examinations

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End point title

Number of Subjects with Abnormalities in Physical Examinations ^[5]

End point description

Number of subjects with abnormalities in physical examinations (abdomen, head, ears, eyes, nose, throat, and sinuses, lungs, neurological, skin, blood and lymphatic system, cardiovascular, chest, gastrointestinal, general appearance and musculoskeletal) were reported. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. Here, 'n' specifies the number of subjects evaluated for specific categories. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Week 12

Notes
[5] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	4	17	21
Units: subjects
Abdomen (n=4,17,21)	1	0	1
Head, Ears, Eyes, Nose, Throat, Sinuses(n=4,17,21)	0	0	0
Lungs (n=4,17,21)	0	0	0
Neurological (n=4,16,20)	0	1	1
Skin (n=4,17,21)	1	4	5

No statistical analyses for this end point

Primary: Change from Baseline in Chemistry Laboratory Parameters: Albumin and Protein

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End point title

Change from Baseline in Chemistry Laboratory Parameters: Albumin and Protein ^[6]

End point description

Change from baseline in chemistry laboratory parameters: albumin and protein were reported. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Baseline up to Week 12

Notes
[6] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	4	17	21
Units: grams per liter (g/L)
arithmetic mean (standard deviation)
Albumin	-1.5 ± 2.89	-1.2 ± 3.21	-1.3 ± 3.08
Protein	-3.5 ± 2.08	-3.8 ± 4.99	-3.7 ± 4.54

No statistical analyses for this end point

Primary: Change from Baseline in Chemistry Laboratory Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglycerides, Urate and Urea Nitrogen

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End point title

Change from Baseline in Chemistry Laboratory Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglycerides, Urate and Urea Nitrogen ^[7]

End point description

Change from baseline in chemistry laboratory parameters: bicarbonate, calcium, chloride, cholesterol, glucose, phosphate, potassium, sodium, triglycerides, urate and urea nitrogen were reported. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Baseline up to Week 12

Notes
[7] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	4	17	21
Units: millimoles per liter (mmol/L)
arithmetic mean (standard deviation)
Bicarbonate	-2.0 ± 2.45	1.6 ± 2.91	1.0 ± 3.14
Calcium	0.010 ± 0.0627	-0.018 ± 0.1005	-0.013 ± 0.0938
Chloride	0.5 ± 1.91	0.6 ± 2.85	0.6 ± 2.66
Cholesterol	0.480 ± 1.0100	0.261 ± 0.5732	0.303 ± 0.6509
Glucose	0.280 ± 0.5054	-0.258 ± 1.1401	-0.155 ± 1.0607
Phosphate	-0.018 ± 0.0971	-0.002 ± 0.1903	-0.005 ± 0.1744
Potassium	0.05 ± 0.173	0.05 ± 0.583	0.05 ± 0.526
Sodium	0.8 ± 0.96	1.4 ± 2.18	1.2 ± 2.00
Triglycerides	0.223 ± 0.5799	0.081 ± 0.3223	0.108 ± 0.3699
Urate	-0.0090 ± 0.04285	0.0174 ± 0.04790	0.124 ± 0.04716
Urea Nitrogen	-0.270 ± 1.7830	-0.105 ± 1.7292	-0.136 ± 1.6951

No statistical analyses for this end point

Primary: Change from Baseline in Chemistry Laboratory Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Creatine Kinase, Gamma Glutamyl Transferase, Lactate Dehydrogenase

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End point title

Change from Baseline in Chemistry Laboratory Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Creatine Kinase, Gamma Glutamyl Transferase, Lactate Dehydrogenase ^[8]

End point description

Change from baseline in chemistry laboratory parameters: alanine aminotransferase (ALT), alkaline phosphatase, aspartate aminotransferase (AST), creatine kinase, gamma glutamyl transferase, lactate dehydrogenase were reported. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. Here, 'n' specifies the number of subjects evaluated for specific categories. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Baseline up to Week 12

Notes
[8] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	4	17	21
Units: units per liter (U/L)
arithmetic mean (standard deviation)
ALT (n=4,17, 21)	17.0 ± 28.76	2.6 ± 5.33	5.3 ± 13.43
Alkaline Phosphatase (n=4,16, 20)	6.3 ± 2.06	5.2 ± 11.46	5.4 ± 10.23
AST (n=4,17, 21)	23.5 ± 47.67	0.9 ± 3.42	5.2 ± 20.80
Creatine Kinase (n=4,17, 21)	-17.8 ± 33.13	13.6 ± 38.34	7.7 ± 38.73
Gamma Glutamyl Transferase (n=4,17, 21)	4.3 ± 11.95	1.5 ± 13.21	2.0 ± 12.74
Lactate Dehydrogenase (n=4,16, 20)	30.0 ± 15.68	30.9 ± 58.10	30.7 ± 52.0

No statistical analyses for this end point

Primary: Change from Baseline in Chemistry Laboratory Parameters: Bilirubin, Creatinine and Direct Bilirubin

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End point title

Change from Baseline in Chemistry Laboratory Parameters: Bilirubin, Creatinine and Direct Bilirubin ^[9]

End point description

Change from baseline in chemistry laboratory parameters: bilirubin, creatinine and direct bilirubin were reported. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. Here, 'n' specifies the number of subjects evaluated for specific categories. Here, "99999" indicates that standard deviation can not be calculated for 1 subject. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Baseline up to Week 12

Notes
[9] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	4	17	21
Units: micromoles per liter (micromol/L)
arithmetic mean (standard deviation)
Bilirubin (n=4,17, 21)	0.13 ± 2.616	-0.59 ± 2.403	-0.46 ± 2.393
Creatinine (n=4,17, 21)	-9.0 ± 7.35	-2.6 ± 13.20	-3.9 ± 12.41
Direct Bilirubin (n=1,2, 3)	-1.20 ± 99999	-0.70 ± 0.283	-0.87 ± 0.359

No statistical analyses for this end point

Primary: Change from Baseline in Hematology Laboratory Parameter: Erythrocytes (Red Blood Cell)

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End point title

Change from Baseline in Hematology Laboratory Parameter: Erythrocytes (Red Blood Cell) ^[10]

End point description

Change from baseline in erythrocytes (red blood cell (hematology laboratory test parameter) were reported. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Baseline up to Week 12

Notes
[10] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	4	16	20
Units: 10^12 per Liter
arithmetic mean (standard deviation)	0.188 ± 0.0822	0.121 ± 0.2702	0.135 ± 0.2438

No statistical analyses for this end point

Primary: Change from Baseline in Hematology Laboratory Parameter: Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets and Leukocytes

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End point title

Change from Baseline in Hematology Laboratory Parameter: Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets and Leukocytes ^[11]

End point description

Change from baseline in hematology laboratory parameters: basophils, eosinophils, lymphocytes, monocytes, neutrophils, platelets and leukocytes were reported. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. Here 'n' specifies the number of subjects evaluated for specific categories. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Baseline up to Week 12

Notes
[11] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	4	16	20
Units: 10^9 per Liter
arithmetic mean (standard deviation)
Basophils (n=4,8,12)	-0.003 ± 0.0150	0.010 ± 0.0200	0.006 ± 0.0188
Eosinophils (n=4,13, 17)	0.015 ± 0.0465	0.013 ± 0.0776	0.014 ± 0.0702
Lymphocytes (n=4,16, 20)	-0.045 ± 0.4498	0.063 ± 0.4715	0.041 ± 0.4576
Monocytes (n=4,16, 20)	-0.105 ± 0.1964	0.059 ± 0.2008	0.027 ± 0.2061
Neutrophils (n=4,16, 20)	-0.433 ± 0.5744	0.411 ± 1.8622	0.243 ± 1.7058
Platelets (n=4,16, 20)	27.8 ± 11.81	12.3 ± 53.20	15.4 ± 47.93
Leukocytes (n=4,16, 20)	-0.568 ± 0.9214	0.567 ± 1.9956	0.340 ± 1.8694

No statistical analyses for this end point

Primary: Change from Baseline in Hematology Parameter: Erythrocytes Mean Corpuscular Hemoglobin (HGB) Concentration

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End point title

Change from Baseline in Hematology Parameter: Erythrocytes Mean Corpuscular Hemoglobin (HGB) Concentration ^[12]

End point description

Change from baseline in erythrocytes mean corpuscular HGB concentration (hematology parameter) was reported. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Baseline up to Week 12

Notes
[12] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	4	16	20
Units: grams per Liter (g/L)
arithmetic mean (standard deviation)	-6.5 ± 25.70	-2.1 ± 7.61	-3.0 ± 12.38

No statistical analyses for this end point

Primary: Change from Baseline inHematology Laboratory Parameter: Erythrocytes Mean Corpuscular Hemoglobin (HGB)

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End point title

Change from Baseline inHematology Laboratory Parameter: Erythrocytes Mean Corpuscular Hemoglobin (HGB) ^[13]

End point description

Change from baseline in erythrocytes mean corpuscular HGB (hematology laboratory parameter) were reported. The safety analysis set included all subjects who received at least 1 dose of study drug (nipocalimab). Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Baseline up to Week 12

Notes
[13] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	4	16	20
Units: picograms (pg)
arithmetic mean (standard deviation)	0.03 ± 0.873	-0.61 ± 0.813	-0.48 ± 0.842

No statistical analyses for this end point

Primary: Change from Baseline in Hematology Laboratory Parameter: Erythrocytes Mean Corpuscular Volume

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End point title

Change from Baseline in Hematology Laboratory Parameter: Erythrocytes Mean Corpuscular Volume ^[14]

End point description

Change from baseline in erythrocytes mean corpuscular volume (hematology laboratory parameter) was reported. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Baseline up to Week 12

Notes
[14] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	4	16	20
Units: femtoliters (fL)
arithmetic mean (standard deviation)	2.45 ± 6.174	-1.41 ± 3.406	-0.64 ± 4.206

No statistical analyses for this end point

Primary: Change from Baseline in Hematology Laboratory Parameter: Hematocrit

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End point title

Change from Baseline in Hematology Laboratory Parameter: Hematocrit ^[15]

End point description

Change from baseline in hematocrit values (hematology laboratory parameter) was reported. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Baseline up to Week 12

Notes
[15] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	4	16	20
Units: liter of cells per liter of blood (L/L)
arithmetic mean (standard deviation)	0.0278 ± 0.03542	0.0048 ± 0.02401	0.0094 ± 0.02725

No statistical analyses for this end point

Primary: Change from Baseline in Hematology Laboratory Parameter: Hemoglobin

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End point title

Change from Baseline in Hematology Laboratory Parameter: Hemoglobin ^[16]

End point description

Change from baseline in hemoglobin values (hematology laboratory parameter) was reported. The safety analysis set included all subjects who received at least 1 dose of study drug (nipocalimab). Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Baseline up to Week 12

Notes
[16] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	4	16	20
Units: g/L
arithmetic mean (standard deviation)	5.8 ± 4.65	0.6 ± 7.29	1.7 ± 7.06

No statistical analyses for this end point

Primary: Change from Baseline in Urinalysis Laboratory Parameter: pH

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End point title

Change from Baseline in Urinalysis Laboratory Parameter: pH ^[17]

End point description

Change from baseline in pH (urinalysis laboratory parameter) was reported. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Baseline up to Week 12

Notes
[17] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	4	17	21
Units: pH
arithmetic mean (standard deviation)	-0.8 ± 0.96	0.0 ± 1.06	-0.1 ± 1.06

No statistical analyses for this end point

Primary: Change from Baseline in Urinalysis Laboratory Parameter: Specific Gravity

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End point title

Change from Baseline in Urinalysis Laboratory Parameter: Specific Gravity ^[18]

End point description

Change from baseline in specific gravity (urinalysis laboratory parameter) was reported. The safety analysis set included all subjects who received at least 1 dose of study drug (nipocalimab). Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Baseline up to Week 12

Notes
[18] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	4	17	21
Units: ratio
arithmetic mean (standard deviation)	-0.0003 ± 0.00660	-0.0030 ± 0.00973	-0.0025 ± 0.00914

No statistical analyses for this end point

Primary: Number of Subjects with Treatment-emergent Abnormal Electrocardiograms (ECG) Values

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End point title

Number of Subjects with Treatment-emergent Abnormal Electrocardiograms (ECG) Values ^[19]

End point description

Number of subjects with treatment-emergent abnormal ECG values for variables including mean heart rate (abnormally low refers to less than or equal to [<=] 50 beats per minute [bpm], abnormally high refers greater than or equal to [>=] 120 bpm), PR interval (abnormally low refers to < 120 and abnormally high refers to >200 milliseconds [msec]), RR interval (abnormally low refers to <600 msec and abnormally high refers to >1200 msec) and QRS duration (abnormally > 120) were reported. Safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. Here 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Up to 257 days post-baseline

Notes
[19] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	7	29	36
Units: subjects
ECG Mean Heart Rate (<= 50)	0	5	5
ECG Mean Heart Rate (>=120)	0	0	0
PR Interval (<120)	0	0	0
PR Interval (> 200)	0	5	5
RR Interval (<600)	0	0	0
RR Interval (>=1200)	0	4	4
QRS Duration (>120)	1	3	4

No statistical analyses for this end point

Primary: Number of Subjects with Columbia Suicide Severity Rating Scale (C-SSRS) Scores

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End point title

Number of Subjects with Columbia Suicide Severity Rating Scale (C-SSRS) Scores ^[20]

End point description

C-SSRS is a questionnaire designed to solicit occurrence, severity, frequency of suicide-related ideation and behaviors. Total score ranges from 1 to 10. Higher total scores indicate greater severity. Maximum score assigned for each subject summarized into one of 3 broad categories: no suicidal ideation or behavior (0), suicidal ideation (1 to 5), suicidal behavior (6 to 10). Higher score indicates more suicidal behavior/ideation. Suicidal ideation includes subjects who did not have suicidal ideation or behavior at baseline and had suicidal ideation without behavior, Suicidal behavior includes subjects who did not have suicidal ideation or behavior at baseline and had suicidal behavior at some time point post-baseline. Safety analysis set included all subjects who received at least 1 dose of nipocalimab. Number of subjects analyzed specifies all subjects who were evaluated for this endpoint. Per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Up to 257 days post-baseline

Notes
[20] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	6	30	36
Units: subjects
No Suicidal Ideation/Behavior	5	30	35
Suicidal Ideation	1	0	1
Suicidal Behavior	0	0	0

No statistical analyses for this end point

Primary: Number of Subjects with Below/Above Normal Coagulation Laboratory Values

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End point title

Number of Subjects with Below/Above Normal Coagulation Laboratory Values ^[21]

End point description

Number of subjects with at least one value above upper limit of normal (> ULN) or below the lower limit of normal (< LLN) value of coagulation parameters (activated partial thromboplastin time [APTT] and prothrombin time [PT]) were reported. The lab reference range for APTT is 25.1 to 36.5 seconds. The lab reference range for PT is 9.4 to 12.5 seconds. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. Here 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Primary

End point timeframe

Up to 257 days post-baseline

Notes
[21] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No Inferential statistical analysis was planned for this Primary Endpoint.

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	7	28	35
Units: subjects
APTT (>ULN)	2	15	17
APTT (<LLN)	0	3	3
PT (>ULN)	1	8	9
PT (<ULN)	0	1	1

No statistical analyses for this end point

Secondary: Change from Baseline in Total Myasthenia Gravis – Activities of Daily Living (MG-ADL) Score Over Time

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End point title

Change from Baseline in Total Myasthenia Gravis – Activities of Daily Living (MG-ADL) Score Over Time

End point description

MG-ADL assessed subject’s MG symptom severity and 8 functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, and eyelid droop), rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). Total score is sum of 8 function scores, ranges from 0 to 24. Higher scores indicated greater symptom severity in performing daily living activities. Full analysis set included all subjects who received at least 1 dose of nipocalimab. Here, number of subjects analyzed specifies all subjects evaluated for this endpoint, 'n' specifies number of subjects evaluated for specific timepoints,"99999" for Week 24 timepoint category indicates that standard deviation could not be calculated for 1 subject and for EoT category indicates that no/0 subjects in Placebo-Nipocalimab arm were analysed at EoT. Per planned analysis, data is reported both for individual arms and all (total) subjects.

End point type

Secondary

End point timeframe

Baseline up to Weeks 4, 8, 12, 24, End of Treatment (EoT) (up to 253 days post-baseline), Follow-up (up to 257 days post-baseline)

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	5	26	31
Units: score on a scale
arithmetic mean (standard deviation)
Week 4 (n=5,26,31)	0.6 ± 1.34	-0.6 ± 2.42	-0.4 ± 2.30
Week 8 (n=5,19,24)	-0.8 ± 3.70	-0.9 ± 2.63	-0.9 ± 2.80
Week 12 (n=4,17,21)	-0.3 ± 4.35	-1.2 ± 2.49	-1.0 ± 2.82
Week 24 (n=1,5,6)	-1.0 ± 99999	-1.2 ± 1.92	-1.2 ± 1.72
EoT (n=0,16,16)	99999 ± 99999	-2.4 ± 2.55	-2.4 ± 2.55
Follow-up (n=5,24,29)	-0.6 ± 1.14	1.2 ± 3.08	0.9 ± 2.91

No statistical analyses for this end point

Secondary: Number of Subjects With a 2-, 3-, 4-, 5-, 6-, 7-, or greater than or equal to (>=) 8-point Improvement in Total MG-ADL Score Over Time

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End point title

Number of Subjects With a 2-, 3-, 4-, 5-, 6-, 7-, or greater than or equal to (>=) 8-point Improvement in Total MG-ADL Score Over Time

End point description

Number of subjects with 2-, 3-, 4-, 5-, 6-, 7-, or >= 8-point improvement in total MG-ADL score over time were reported. MG-ADL assess subject’s MG symptom severity and 8 functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, and eyelid droop) rated on 4-point scale:0 (no impairment) to 3 (severe impairment). Total score is sum of 8 function scores ranging from 0 to 24. Higher scores indicated greater symptom severity in performing daily living activities. FAS included all subjects who received at least 1 dose of nipocalimab. Here, 'n' (number analyzed) specifies the number of subjects evaluated for specific timepoints,99999 indicates that no/0 subjects were analysed in Placebo-Nipocalimab arm. Per planned analysis, data is reported both for individual arms and all(total) subjects.

End point type

Secondary

End point timeframe

Weeks 4, 8, 12, 24, End of Treatment (EoT) (up to 253 days post-baseline), Follow-up (up to 257 days post-baseline)

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	7	30	37
Units: subjects
Week 4 (2 Point Improvement) (n=0,6,6)	99999	2	2
Week 4 (3 Point Improvement) (n=0,6,6)	99999	1	1
Week 4 (4 Point Improvement) (n=0,6,6)	99999	0	0
Week 4 (5 Point Improvement) (n=0,6,6)	99999	1	1
Week 4 (6 Point Improvement) (n=0,6,6)	99999	2	2
Week 4 (7 Point Improvement) (n=0,6,6)	99999	0	0
Week 4 (>= 8 Point Improvement) (n=0,6,6)	99999	0	0
Week 8 (2 Point Improvement) (n=2,7,9)	1	2	3
Week 8 (3 Point Improvement) (n=2,7,9)	0	4	4
Week 8 (4 Point Improvement) (n=2,7,9)	0	0	0
Week 8 (5 Point Improvement) (n=2,7,9)	0	0	0
Week 8 (6 Point Improvement) (n=2,7,9)	1	0	1
Week 8 (7 Point Improvement) (n=2,7,9)	0	0	0
Week 8 (>= 8 Point Improvement) (n=2,7,9)	0	1	1
Week 12 (2 Point Improvement) (n=1,5,6)	0	2	2
Week 12 (3 Point Improvement) (n=1,5,6)	0	0	0
Week 12 (4 Point Improvement) (n=1,5,6)	0	0	0
Week 12 (5 Point Improvement) (n=1,5,6)	0	0	0
Week 12 (6 Point Improvement) (n=1,5,6)	1	3	4
Week 12 (7 Point Improvement) (n=1,5,6)	0	0	0
Week 12 (>= 8 Point Improvement) (n=1,5,6)	0	0	0
Week 24 (2 Point Improvement) (n=0,2,2)	99999	1	1
Week 24 (3 Point Improvement) (n=0,2,2)	99999	0	0
Week 24 (4 Point Improvement) (n=0,2,2)	99999	1	1
Week 24 (5 Point Improvement) (n=0,2,2)	99999	0	0
Week 24 (6 Point Improvement) (n=0,2,2)	99999	0	0
Week 24 (7 Point Improvement) (n=0,2,2)	99999	0	0
Week 24 (>= 8 Point Improvement) (n=0,2,2)	99999	0	0
EoT (2 Point Improvement)(n=0,8,8)	99999	2	2
EoT (3 Point Improvement)(n=0,8,8)	99999	2	2
EoT (4 Point Improvement) (n=0,8,8)	99999	0	0
EoT (5 Point Improvement) (n=0,8,8)	99999	1	1
EoT (6 Point Improvement) (n=0,8,8)	99999	1	1
EoT (7 Point Improvement) (n=0,8,8)	99999	2	2
EoT (>=8 Point Improvement)(n=0,8,8)	99999	0	0
Follow-up (2 Point Improvement)(n=1,4,5)	1	3	4
Follow-up (3 Point Improvement)(n=1,4,5)	0	1	1
Follow-up (4 Point Improvement)(n=1,4,5)	0	0	0
Follow-up (5 Point Improvement)(n=1,4,5)	0	0	0
Follow-up (6 Point Improvement)(n=1,4,5)	0	0	0
Follow-up (7 Point Improvement)(n=1,4,5)	0	0	0
Follow-up (>=8 Point Improvement)(n=1,4,5)	0	0	0

No statistical analyses for this end point

Secondary: Change from Baseline in Total Quantitative Myasthenia Gravis (QMG) Score Over Time

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End point title

Change from Baseline in Total Quantitative Myasthenia Gravis (QMG) Score Over Time

End point description

The QMG test was used to assess the subject’s strength. The quantitative results of each of the 13 strength components were mapped to a 4-point scale where 0 equals to (=) none, 1= mild, 2= moderate and 3= severe. The total score is the sum of the 13 scale scores and ranges from 0 to 39. Higher scores indicated more severe impairment. The full analysis set (FAS) included all subjects who received at least 1 dose of study drug nipocalimab. Here 'n' specifies the number of subjects evaluated for specific timepoints. Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. Here "99999" for follow-up timepoint category indicates that standard deviation could not be calculated for 1 subject and for Week 24 and EoT category indicates that no/ 0 subjects in the Placebo-Nipocalimab group were analysed at Weeks 24 and EoT. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Secondary

End point timeframe

Baseline up to Weeks 4, 8, 12, 24, End of Treatment (EoT) (up to 253 days post-baseline), follow-up (up to 257 days post-baseline)

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	2	9	11
Units: score on a scale
arithmetic mean (standard deviation)
Week 4 (n=2,9, 11)	1.5 ± 6.36	-1.3 ± 2.60	-0.8 ± 3.28
Week 8 (n=2,7,9)	-3.0 ± 5.66	1.0 ± 1.53	0.1 ± 2.98
Week 12 (n=2,6,8)	-0.5 ± 3.54	-1.2 ± 2.23	-1.0 ± 2.33
Week 24 (n=0,3,3)	99999 ± 99999	-2.7 ± 1.53	-2.7 ± 1.53
EoT (n=0,7,7)	99999 ± 99999	-2.4 ± 4.12	-2.4 ± 4.12
Follow-up (n=1,7,8)	-3.0 ± 99999	-1.6 ± 2.07	-1.8 ± 1.98

No statistical analyses for this end point

Secondary: Change from Baseline in Total Revised Myasthenia Gravis Quality of Life – 15 Scale (MG-QoL15r) Score Over Time

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End point title

Change from Baseline in Total Revised Myasthenia Gravis Quality of Life – 15 Scale (MG-QoL15r) Score Over Time

End point description

MG-QoL15r was used to assess subject’s limitations related to living with MG. Each of 15 questions were rated by subject on a 3-point scale (0= Not at all, 1= somewhat, 2=very much) based on a recall period of “over past few weeks”. Total score is the sum of 15 question scores and ranges from 0 to 30. Higher scores indicated more limitation. FAS included all subject who received at least 1 dose of study drug nipocalimab. Here 'n' specifies the number of subjects evaluated for specific timepoints. Here "99999" for Week 24 timepoint category indicates that standard deviation could not be calculated for 1 subject and for EoT category indicates that no/ 0 subjects in the Placebo-Nipocalimab arm were analysed. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Secondary

End point timeframe

Baseline up to Weeks 4, 8, 12, 24, End of Treatment (EoT) (up to 253 days post-baseline), Follow-up (up to 257 days post-baseline)

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	7	30	37
Units: score on a scale
arithmetic mean (standard deviation)
Week 4 (n=5,26,31)	1.4 ± 3.85	-2.2 ± 4.53	-1.6 ± 4.57
Week 8 (n=5,19,24)	-0.4 ± 3.97	-1.9 ± 4.34	-1.6 ± 4.23
Week 12 (n= 4,17,21)	-1.0 ± 6.27	-3.4 ± 4.70	-2.9 ± 4.95
Week 24 (1, 5,6)	-3.0 ± 99999	-1.4 ± 1.52	-1.7 ± 1.51
EoT (n=0,16,16)	99999 ± 99999	-3.7 ± 5.28	-3.7 ± 5.28
Follow-up (n=6, 24,30)	-1.3 ± 5.72	0.1 ± 3.11	-0.2 ± 3.69

No statistical analyses for this end point

Secondary: Change from Baseline in Clinical Global Impression of Severity (CGI-S) Scores Over Time

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End point title

Change from Baseline in Clinical Global Impression of Severity (CGI-S) Scores Over Time

End point description

CCGI-S scale is clinician’s global assessment of illness severity of MG and rated by answering on 8-point scale. Subject is assessed on severity of illness according to: 0=not performed; 1=normal, not at all ill; 2=borderline illness; 3=mildly ill; 4=moderately ill; 5=markedly ill; 6=severely ill; 7=among most extremely ill patients. Higher scores indicated more severity of illness. Values of 0 (not assessed) were excluded from analysis. FAS included all subjects who received at least 1 dose of study drug nipocalimab. Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint, 'n' specifies number of subjects evaluated for specific timepoints, "99999" for Week 24 timepoint category indicates that standard deviation could be calculated for 1 subject and for EoT category indicates that no/ 0 subjects in Placebo-Nipocalimab group analysed. Per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Secondary

End point timeframe

Baseline up to Weeks 4, 8, 12, 24, End of Treatment (EoT) (up to 253 days post-baseline), Follow-up (up to 257 days post-baseline)

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	5	26	31
Units: score on a scale
arithmetic mean (standard deviation)
Week 4 (n=5, 26,31)	-0.2 ± 0.84	-0.3 ± 0.60	-0.3 ± 0.63
Week 8 (n=5,19,24)	-1.0 ± 0.71	-0.6 ± 0.90	-0.7 ± 0.86
Week 12 (n=4,17,21)	-0.5 ± 1.00	-0.5 ± 0.80	-0.5 ± 0.81
Week 24 (n=1,5,6)	0.0 ± 99999	-0.6 ± 0.89	-0.5 ± 0.84
EoT (n=0,16,16)	99999 ± 99999	-0.7 ± 0.95	-0.7 ± 0.95
Follow-up (n=5,21,26)	-1.0 ± 1.22	-0.1 ± 0.73	-0.3 ± 0.88

No statistical analyses for this end point

Secondary: Number of Subjects with Improvement of Illness Over Time based on Clinical Global Impression of Improvement (CGI-I) Scale Score

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End point title

Number of Subjects with Improvement of Illness Over Time based on Clinical Global Impression of Improvement (CGI-I) Scale Score

End point description

Number of subjects with improvement of illness based on CGI-I scale score over time were reported. The CGI-I scale is the clinician/physician’s global assessment of the change in severity of the patient’s gMG since starting this study. The rating is given on a 7-point scale with lower scores indicating greater improvement (1= Very much improved; 2 = Much improved; 3 = Minimally improved; 4 = No change; 5 =Minimally worse; 6 = Much worse; 7 = Very much worse. Values of 0 (not assessed) were excluded from analysis. Higher score indicates more severity. The FAS included all subjects who received at least 1 dose of study drug nipocalimab. Here, 'n' specifies the number of subjects evaluated for specific timepoints. Per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Secondary

End point timeframe

Weeks 4, 8, 12, 24, End of Treatment (EoT) (up to 253 days post-baseline), Follow-up (up to 257 days post-baseline)

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	5	26	31
Units: subjects
Week 4 (Very much worse) (n=5,26, 31)	0	0	0
Week 4 (Much worse) (n=5,26,31)	0	0	0
Week 4 (Minimally worse) (n=5,26,31)	1	2	3
Week 4 (No change) (n=5,26,31)	1	7	8
Week 4 (Minimally improved) (n=5,26,31)	2	12	14
Week 4 (Much improved) (n=5,26,31)	0	5	5
Week 4 (Very much improved) (n=5,26,31)	1	0	1
Week 8 (Very much worse) (n=5,19,24)	0	0	0
Week 8 (Much worse) (n=5,19,24)	0	0	0
Week 8 (Minimally worse) (n=5,19,24)	0	2	2
Week 8 (No change) (n=5,19,24)	0	3	3
Week 8 (Minimally improved) (n=5,19,24)	2	6	8
Week 8 (Much improved) (n=5,19,24)	2	8	10
Week 8 (Very much improved) (n=5,19,24)	1	0	1
Week 12 (Very much worse) (n=4,17,21)	0	0	0
Week 12 (Much worse) (n=4,17,21)	0	0	0
Week 12 (Minimally worse) (n=4,17,21)	0	2	2
Week 12 (No change) (n=4,17,21)	2	1	3
Week 12 (Minimally improved) (n=4,17,21)	1	3	4
Week 12 (Much improved) (n=4,17,21)	1	10	11
Week 12 (Very much improved) (n=4,17,21)	0	1	1
Week 24 (Very Much worse) (n=1,5,6)	0	0	0
Week 24 (Much worse) (n=1,5,6)	0	0	0
Week 24 (Minimally worse) (n=1,5,6)	0	0	0
Week 24 (No change) (n=1,5,6)	1	0	1
Week 24 (Minimally improved) (n=1,5,6)	0	1	1
Week 24 (Much improved) (n=1,5,6)	0	4	4
Week 24 (Very Much improved) (n=1,5,6)	0	0	0
EoT (Very Much worse) (n=0,16,16)	99999	0	0
EoT (Much worse) (n=0,16,16)	99999	0	0
EoT (Minimally worse) (n=0,16,16)	99999	0	0
EoT (No change) (n=0,16,16)	99999	1	1
EoT (Minimally improved) (n=0,16,16)	99999	7	7
EoT (Much improved) (n=0,16,16)	99999	7	7
EoT (Very much improved) (n=0,16,16)	99999	1	1
Follow-up (Very Much worse) (n=5,21, 26)	0	0	0
Follow-up (Much worse) (n=5,21, 26)	1	2	3
Follow-up (Minimally worse)(n=5,21,26)	0	3	3
Follow-up (No change) (n=5,21,26)	0	5	5
Follow-up (Minimally improved) (n=5,21,26)	0	8	8
Follow-up (Much improved) (n=5,21,26)	3	2	5
Follow-up (Very much improved) (n=5,21,26)	1	1	2

No statistical analyses for this end point

Secondary: Number of Subjects with Change from Baseline in Myasthenia Gravis Foundation of America (MGFA) Classification Score Over Time

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End point title

Number of Subjects with Change from Baseline in Myasthenia Gravis Foundation of America (MGFA) Classification Score Over Time

End point description

MGFA assessed subject’s MG severity, identifies subgroup subject with MG sharing distinct clinical features or severity of disease: Class I (ocular MG), classes II, III and IV generalized MG with mild, moderate, severe disease, respectively; Class V MG crisis. Separate subclasses under classes II, III, IV designed: ‘a’ if predominant weakness affecting limb/axial weakness or both; ‘b’ if predominant weakness affecting oropharyngeal or respiratory muscles or both. Lower roman numerals mean less severity. Changes in MGFA classification (regardless of subclass) categorized as ‘Improved’, ‘Same’, or ‘Worsened’. FAS included all subjects who received at least 1 dose of nipocalimab. Number of subjects analyzed specifies subjects evaluated for this endpoint, 'n' specifies number of subjects evaluated for specific timepoint category, '99999' indicates 0/no subjects analysed in respective arm for EoT. As planned, data is reported both for individual arms and all (total) subjects.

End point type

Secondary

End point timeframe

Weeks 8, 24 and End of Treatment (EoT) (up to 253 days post-baseline)

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	4	9	13
Units: subjects
Week 8 (Improved) (n=4,9,13)	1	5	6
Week 8 (Same) (n=4,9,13)	3	3	6
Week 8 (Worsened) (n=4,9,13)	0	1	1
Week 24 (Improved) (n=1,1, 2)	0	0	0
Week 24 (Same) (n=1,1, 2)	1	1	2
Week 24 (Worsened) (n=1,1, 2)	0	0	0
EoT (Improved) (n=0,8, 8)	99999	4	4
EoT (Same) (n=0,8,8)	99999	3	3
EoT (Worsened) (n=0,8,8)	99999	1	1

No statistical analyses for this end point

Secondary: Number of Subjects with Anti-drug Antibodies (ADA) to Nipocalimab

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End point title

Number of Subjects with Anti-drug Antibodies (ADA) to Nipocalimab

End point description

Number of subjects with ADA to nipocalimab were reported. The presence of ADA to nipocalimab in serum was determined by a sensitive and drug-tolerant electrochemiluminescence immunoassay (ECLIA) method. The FAS included all subjects who received at least 1 dose of study drug nipocalimab. Here 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. As planned, data is reported both for individual arms and all (total) subjects.

End point type

Secondary

End point timeframe

Up to 257 days post-baseline

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	6	29	35
Units: subjects	1	16	17

No statistical analyses for this end point

Secondary: Number of Subjects with Neutralizing Antibodies (NAbs) to Nipocalimab

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End point title

Number of Subjects with Neutralizing Antibodies (NAbs) to Nipocalimab

End point description

Number of subjects with NAbs were reported. Neutralizing antibodies to nipocalimab were assessed using a non-cell based competitive ligand binding ECLIA assay. Here, 'N' (number of subjects analyzed) specifies all subjects who were positive to antibodies to nipocalimab. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Secondary

End point timeframe

Up to 257 days post-baseline

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	1	16	17
Units: subjects	0	4	4

No statistical analyses for this end point

Secondary: Change from Baseline in Serum Immunoglobulin (Ig)G Concentration Over Time

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End point title

Change from Baseline in Serum Immunoglobulin (Ig)G Concentration Over Time

End point description

Change from baseline in serum immunoglobulin (Ig)G concentration over time was reported. The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab. Here, 'N' (number of subjects analyzed) specifies all subjects who were evaluated for this endpoint. Here 'n' specifies the number of subjects evaluated for specific timepoints. Here, '99999' indicates that no/0 subjects in the Placebo-Nipocalimab group were analysed at Week 36 and '99999' for Week 24 category indicates standard deviation can not be calculated for 1 subject. As per planned analysis, data is reported for individual arms and for all (total) subjects both.

End point type

Secondary

End point timeframe

Baseline to Weeks 2, 4, 8, 12, 24, up to 253 days post-baseline, up to 257 days post-baseline

End point values	Placebo-Nipocalimab	Nipocalimab-Nipocalimab	Nipocalimab (All Subjects)
Number of subjects analysed	5	27	32
Units: g/L
arithmetic mean (standard deviation)
Week 2 (n=5,27,32)	-6.888 ± 0.6104	-5.457 ± 1.2510	-5.681 ± 1.2804
Week 4 (n=5,26, 31)	-3.234 ± 1.1858	-2.796 ± 0.9728	-2.866 ± 1.0014
Week 8 (n=5,19, 24)	-2.996 ± 1.3773	-3.122 ± 1.3466	-3.095 ± 1.3235
Week 12 (n=4,17, 21)	-3.243 ± 1.6378	-3.639 ± 2.2627	-3.563 ± 2.1269
Week 24 (n=1,5, 6)	-3.870 ± 99999	-4.072 ± 1.2826	-4.038 ± 1.1502
253 days post-baseline (n=0,14,14)	99999 ± 99999	-3.494 ± 1.208	-3.494 ± 1.9208
257 days post-baseline (n=5,16, 21)	-1.150 ± 0.5687	-0.020 ± 1.6162	-0.289 ± 1.5056

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

Up to 257 days post-baseline

Adverse event reporting additional description

The safety analysis set included all subjects who received at least 1 dose of study drug nipocalimab.

Assessment type

Non-systematic

Dictionary name

MedDRA

Dictionary version

23.1

Reporting group title

Nipocalimab-Nipocalimab

Reporting group description

Reporting group title

Placebo-Nipocalimab

Reporting group description

Subjects who received placebo in MOM-M281-004 study rolled-over and received intravenous (IV) infusion of nipocalimab 30 milligrams per kilogram (mg/kg) every 4 weeks (Q4W) starting Day 1 up to 8 weeks. After 8 weeks of treatment on a stable dose of nipocalimab, the dose and/or dosing frequency could be individually adjusted, at the investigator's discretion to receive maximum dose of 60 mg/kg at a frequency of not exceeding every 2 weeks (Q2W).

Serious adverse events	Nipocalimab-Nipocalimab	Placebo-Nipocalimab
Total subjects affected by serious adverse events
subjects affected / exposed	4 / 30 (13.33%)	1 / 7 (14.29%)
number of deaths (all causes)	1	0
number of deaths resulting from adverse events	1	0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Gliosarcoma
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0
deaths causally related to treatment / all	1 / 1	0 / 0
Nervous system disorders
Myasthenia Gravis
subjects affected / exposed	2 / 30 (6.67%)	0 / 7 (0.00%)
occurrences causally related to treatment / all	0 / 2	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Infections and infestations
Coronavirus Infection
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Covid-19 Pneumonia
subjects affected / exposed	0 / 30 (0.00%)	1 / 7 (14.29%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 2%

Non-serious adverse events	Nipocalimab-Nipocalimab	Placebo-Nipocalimab
Total subjects affected by non serious adverse events
subjects affected / exposed	17 / 30 (56.67%)	4 / 7 (57.14%)
General disorders and administration site conditions
Asthenia
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Feeling Hot
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Oedema Peripheral
subjects affected / exposed	3 / 30 (10.00%)	1 / 7 (14.29%)
occurrences all number	4	1
Pyrexia
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Peripheral Swelling
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Reproductive system and breast disorders
Adenomyosis
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Menorrhagia
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Respiratory, thoracic and mediastinal disorders
Aphonia
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Cough
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Dyspnoea
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Increased Viscosity of Upper Respiratory Secretion
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Oropharyngeal Pain
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Psychiatric disorders
Depressed Mood
subjects affected / exposed	0 / 30 (0.00%)	1 / 7 (14.29%)
occurrences all number	0	1
Investigations
Lymphocyte Count Decreased
subjects affected / exposed	2 / 30 (6.67%)	0 / 7 (0.00%)
occurrences all number	3	0
Hepatic Enzyme Increased
subjects affected / exposed	0 / 30 (0.00%)	1 / 7 (14.29%)
occurrences all number	0	1
Blood Potassium Decreased
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	2	0
Blood Immunoglobulin G Decreased
subjects affected / exposed	4 / 30 (13.33%)	1 / 7 (14.29%)
occurrences all number	4	1
Blood Creatine Phosphokinase Increased
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	3	0
Neutrophil Count Increased
subjects affected / exposed	2 / 30 (6.67%)	0 / 7 (0.00%)
occurrences all number	2	0
Platelet Count Decreased
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
White Blood Cell Count Increased
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Injury, poisoning and procedural complications
Contusion
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Ligament Sprain
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Foreign Body
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Foot Fracture
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Skin Laceration
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	2	0
Wound
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Nervous system disorders
Paraesthesia
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Neuropathy Peripheral
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Myasthenia Gravis
subjects affected / exposed	0 / 30 (0.00%)	1 / 7 (14.29%)
occurrences all number	0	1
Headache
subjects affected / exposed	2 / 30 (6.67%)	0 / 7 (0.00%)
occurrences all number	5	0
Blood and lymphatic system disorders
Iron Deficiency Anaemia
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Ear and labyrinth disorders
Vertigo Positional
subjects affected / exposed	0 / 30 (0.00%)	1 / 7 (14.29%)
occurrences all number	0	1
Eye disorders
Conjunctival Haemorrhage
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Conjunctival Hyperaemia
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Gastrointestinal disorders
Dyspepsia
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Nausea
subjects affected / exposed	2 / 30 (6.67%)	0 / 7 (0.00%)
occurrences all number	2	0
Toothache
subjects affected / exposed	0 / 30 (0.00%)	1 / 7 (14.29%)
occurrences all number	0	1
Vomiting
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	2	0
Diarrhoea
subjects affected / exposed	2 / 30 (6.67%)	0 / 7 (0.00%)
occurrences all number	2	0
Skin and subcutaneous tissue disorders
Rash Maculo-Papular
subjects affected / exposed	0 / 30 (0.00%)	1 / 7 (14.29%)
occurrences all number	0	1
Musculoskeletal and connective tissue disorders
Pain in Extremity
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Osteoporosis
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Arthralgia
subjects affected / exposed	1 / 30 (3.33%)	1 / 7 (14.29%)
occurrences all number	1	1
Infections and infestations
Nasopharyngitis
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Herpes Zoster
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Oral Herpes
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Otitis Media
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Viral Upper Respiratory Tract Infection
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Upper Respiratory Tract Infection
subjects affected / exposed	2 / 30 (6.67%)	0 / 7 (0.00%)
occurrences all number	2	0
Sinusitis
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Pharyngitis
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0
Metabolism and nutrition disorders
Vitamin B6 Deficiency
subjects affected / exposed	1 / 30 (3.33%)	0 / 7 (0.00%)
occurrences all number	1	0

More information

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Were there any global substantial amendments to the protocol? Yes

03 Dec 2018

The first amendment dated: 03-dec-20218 included following changes: added a medical monitor review of any patient who received rescue therapy during the proof-of-concept (POC) study, before enrollment in this study; deleted text that implied last infusion is given at early termination; added potential risks and plans for mitigation section; added/revised to include a medical monitor review of any patient who received rescue therapy during the POC study, clarification of abstinence, length of abstinence following last statement, acceptable methods of contraception, clarification regarding infection, and clarification of diagnosed Gilbert’s disease; revised patient position when measuring blood pressure and window of time when assessments are to be started; added event of pregnancy to individual patient’s study drug stopping rules; added a required approval by regulatory authority(ies) before resuming drug.

01 Aug 2019

The second amendment, dated 01 August 2019, included the following changes: The Clinical Global Impression of Change (CGI-C) is now referred to as the Clinical Global Impression of Improvement (CGI-I); removed all references to the infusion duration. A post infusion safety observation period will only be required for a minimum of the first 2 infusions. Home infusions may be allowed for every other infusion. Deleted Table 2. Section 6 Study Assessments and Procedures, Table 2 Schedule of Assessments Before and After Study Drug Infusion; Added hypoalbuminemia as an AE of special interest; Modified the criterion for exclusionary elevated creatine kinase (CK); Modified exclusion #2 for clarity. Modified exclusion #3 for consistency with the text in Section 6.1.7, C-SSRS and to provide greater clarity to study sites regarding assessment of suicidal ideation/behavior. Modified exclusion #14 to permit patients who have moderate increases in CK at screen, the opportunity to repeat the assessment and potentially qualify for the study if the Investigator believes the elevation is not of pathological origin; added new text regarding the Infusion Manual; added text about allowed nipocalimab dose adjustments. Removed all references to the infusion duration. A post infusion safety observation period will only be required for a minimum of the first 2 infusions; added text regarding the option for home infusions; added text about labeling of nipocalimab supplies; added a Week 10 visit for subjects on a every 2 weeks (Q2W) regimen (with the same assessments as for a regular Q2W or every 4 weeks [Q4W] visit), and clarified that visits every 12 weeks (Q12W) applies to both regimens; added the time frame for reporting a pregnancy in the female partner of a male patient; Converted the CGI-S and CGI-I to 8-point scales (formerly 7-point scales).

Were there any global interruptions to the trial? Yes

Date	Interruption	Restart date
17 Apr 2020	Study was originally halted due to the COVID-19 pandemic. The study was later terminated prematurely due to the continuing COVID-19 pandemic, and because as the subjects will have the option to enter into an open-label extension portion of a planned future study. It was not due to safety concerns.	-

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

None reported

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Clinical trials

Clinical Trial Results: An Open-label Extension Study of MOM-M281-004 to Evaluate the Safety, Tolerability, and Efficacy of M281 Administered to Patients with Generalized Myasthenia Gravis

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Number of Subjects with Treatment-emergent Adverse Events (TEAEs)

Primary: Number of Subjects with Treatment-emergent Adverse Events (TEAEs)

Primary: Number of Subjects with Treatment-emergent Serious Adverse Events (SAEs)

Primary: Number of Subjects with Treatment-emergent Serious Adverse Events (SAEs)

Primary: Number of Subjects with Treatment-emergent Adverse Events of Special Interest (AESIs)

Primary: Number of Subjects with Treatment-emergent Adverse Events of Special Interest (AESIs)

Primary: Number of Subjects with Treatment-emergent Abnormal Vital Signs

Primary: Number of Subjects with Treatment-emergent Abnormal Vital Signs

Primary: Number of Subjects with Abnormalities in Physical Examinations

Primary: Number of Subjects with Abnormalities in Physical Examinations

Primary: Change from Baseline in Chemistry Laboratory Parameters: Albumin and Protein

Primary: Change from Baseline in Chemistry Laboratory Parameters: Albumin and Protein

Primary: Change from Baseline in Chemistry Laboratory Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglycerides, Urate and Urea Nitrogen

Primary: Change from Baseline in Chemistry Laboratory Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglycerides, Urate and Urea Nitrogen

Primary: Change from Baseline in Chemistry Laboratory Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Creatine Kinase, Gamma Glutamyl Transferase, Lactate Dehydrogenase

Primary: Change from Baseline in Chemistry Laboratory Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Creatine Kinase, Gamma Glutamyl Transferase, Lactate Dehydrogenase

Primary: Change from Baseline in Chemistry Laboratory Parameters: Bilirubin, Creatinine and Direct Bilirubin

Primary: Change from Baseline in Chemistry Laboratory Parameters: Bilirubin, Creatinine and Direct Bilirubin

Primary: Change from Baseline in Hematology Laboratory Parameter: Erythrocytes (Red Blood Cell)

Primary: Change from Baseline in Hematology Laboratory Parameter: Erythrocytes (Red Blood Cell)

Primary: Change from Baseline in Hematology Laboratory Parameter: Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets and Leukocytes

Primary: Change from Baseline in Hematology Laboratory Parameter: Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets and Leukocytes

Primary: Change from Baseline in Hematology Parameter: Erythrocytes Mean Corpuscular Hemoglobin (HGB) Concentration

Primary: Change from Baseline in Hematology Parameter: Erythrocytes Mean Corpuscular Hemoglobin (HGB) Concentration

Primary: Change from Baseline inHematology Laboratory Parameter: Erythrocytes Mean Corpuscular Hemoglobin (HGB)

Primary: Change from Baseline inHematology Laboratory Parameter: Erythrocytes Mean Corpuscular Hemoglobin (HGB)

Primary: Change from Baseline in Hematology Laboratory Parameter: Erythrocytes Mean Corpuscular Volume

Primary: Change from Baseline in Hematology Laboratory Parameter: Erythrocytes Mean Corpuscular Volume

Primary: Change from Baseline in Hematology Laboratory Parameter: Hematocrit

Primary: Change from Baseline in Hematology Laboratory Parameter: Hematocrit

Primary: Change from Baseline in Hematology Laboratory Parameter: Hemoglobin

Primary: Change from Baseline in Hematology Laboratory Parameter: Hemoglobin

Primary: Change from Baseline in Urinalysis Laboratory Parameter: pH

Primary: Change from Baseline in Urinalysis Laboratory Parameter: pH

Primary: Change from Baseline in Urinalysis Laboratory Parameter: Specific Gravity

Primary: Change from Baseline in Urinalysis Laboratory Parameter: Specific Gravity

Primary: Number of Subjects with Treatment-emergent Abnormal Electrocardiograms (ECG) Values

Primary: Number of Subjects with Treatment-emergent Abnormal Electrocardiograms (ECG) Values

Primary: Number of Subjects with Columbia Suicide Severity Rating Scale (C-SSRS) Scores

Primary: Number of Subjects with Columbia Suicide Severity Rating Scale (C-SSRS) Scores

Primary: Number of Subjects with Below/Above Normal Coagulation Laboratory Values

Primary: Number of Subjects with Below/Above Normal Coagulation Laboratory Values

Secondary: Change from Baseline in Total Myasthenia Gravis – Activities of Daily Living (MG-ADL) Score Over Time

Secondary: Change from Baseline in Total Myasthenia Gravis – Activities of Daily Living (MG-ADL) Score Over Time

Secondary: Number of Subjects With a 2-, 3-, 4-, 5-, 6-, 7-, or greater than or equal to (>=) 8-point Improvement in Total MG-ADL Score Over Time

Secondary: Number of Subjects With a 2-, 3-, 4-, 5-, 6-, 7-, or greater than or equal to (>=) 8-point Improvement in Total MG-ADL Score Over Time

Secondary: Change from Baseline in Total Quantitative Myasthenia Gravis (QMG) Score Over Time

Secondary: Change from Baseline in Total Quantitative Myasthenia Gravis (QMG) Score Over Time

Secondary: Change from Baseline in Total Revised Myasthenia Gravis Quality of Life – 15 Scale (MG-QoL15r) Score Over Time

Secondary: Change from Baseline in Total Revised Myasthenia Gravis Quality of Life – 15 Scale (MG-QoL15r) Score Over Time

Secondary: Change from Baseline in Clinical Global Impression of Severity (CGI-S) Scores Over Time

Secondary: Change from Baseline in Clinical Global Impression of Severity (CGI-S) Scores Over Time

Secondary: Number of Subjects with Improvement of Illness Over Time based on Clinical Global Impression of Improvement (CGI-I) Scale Score

Secondary: Number of Subjects with Improvement of Illness Over Time based on Clinical Global Impression of Improvement (CGI-I) Scale Score

Secondary: Number of Subjects with Change from Baseline in Myasthenia Gravis Foundation of America (MGFA) Classification Score Over Time

Secondary: Number of Subjects with Change from Baseline in Myasthenia Gravis Foundation of America (MGFA) Classification Score Over Time

Secondary: Number of Subjects with Anti-drug Antibodies (ADA) to Nipocalimab

Secondary: Number of Subjects with Anti-drug Antibodies (ADA) to Nipocalimab

Secondary: Number of Subjects with Neutralizing Antibodies (NAbs) to Nipocalimab

Secondary: Number of Subjects with Neutralizing Antibodies (NAbs) to Nipocalimab

Secondary: Change from Baseline in Serum Immunoglobulin (Ig)G Concentration Over Time

Secondary: Change from Baseline in Serum Immunoglobulin (Ig)G Concentration Over Time

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
An Open-label Extension Study of MOM-M281-004 to Evaluate the Safety, Tolerability, and Efficacy of M281 Administered to Patients with Generalized Myasthenia Gravis