Neoplasms

Nervous System Diseases

Immune System Diseases

All Conditions

Musculoskeletal Diseases

Symptoms and General Pathology

Rare Diseases

Myasthenia Gravis

Muscle Weakness

Paresis

Asthenia

Muscular Diseases

Neuromuscular Manifestations

Neurologic Manifestations

Syndrome

Paraneoplastic Syndromes

Paraneoplastic Syndromes, Nervous System

Nervous System Neoplasms

Autoimmune Diseases

Autoimmune Diseases of the Nervous System

Neurodegenerative Diseases

Neuromuscular Junction Diseases

Neuromuscular Diseases

All Drugs and Chemicals

Complement System Proteins

Study participants will be treated with subcutaneous zilucoplan (0.3mg/kg/day)

zilucoplan (RA101495)

UCB Cares

The purpose of the study is to evaluate the safety and tolerability of switching from intravenous (IV) complement component 5 (C5) inhibitors to subcutaneous (SC) Zilucoplan in study participants with generalized myasthenia gravis (gMG)

An Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Zilucoplan in Participants With Generalized Myasthenia Gravis Who Were Previously Receiving Intravenous Complement Component 5 Inhibitors

A Phase 3b, Multicenter, Open-Label, Single-Arm Study to Evaluate the Safety, Tolerability, and Efficacy of Zilucoplan in Participants With Generalized Myasthenia Gravis Switching From Intravenous Complement Component 5 Inhibitors to Subcutaneous Zilucoplan

Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

Treatment-emergent adverse events (TEAEs) are any untoward medical incidence in a subject during administered study treatment, whether or not these events are related to study treatment.

The Myasthenia Gravis-Activities of Daily Living (MG-ADL) profile provides an assessment of myasthenia gravis (MG) symptom severity and measures 8 items on a 0-3 scale, with 0 being the least severe. The total sum of the 8 items represents the MG-ADL score. The MG-ADL score can range from 0 (least severe) to 24 (most severe).

The Quantitative Myasthenia Gravis (QMG) is a standardized and validated quantitative strength scoring system and measures 13 items on a 0-3 scale, with 0 being the least severe. The total sum of the 13 items represents the QMG score. The QMG score can range from 0 (least severe) to 39 (most severe)

Treatment-emergent serious adverse events (serious TEAEs) are any untoward medical incidence in a subject during administered study treatment, whether or not these events are related to study treatment and additionally are emergent untoward medical occurrence that at any dose:

* Results in death
* Is life-threatening
* Requires in patient hospitalisation or prolongation of existing hospitalisation
* Results in persistent disability/incapacity
* Is a congenital anomaly or birth defect
* Important medical events

Incidence of study withdrawals based to pre-defined reasons in the Protocol.

UCB Biopharma SRL

The RAISE-XT study is an open-label extension study to evaluate the long-term efficacy, safety, and tolerability of zilucoplan in subjects with gMG who have previously participated in a qualifying Ra Pharmaceuticals sponsored zilucoplan study.

Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis

A Phase 3, Multicenter, Open-Label Extension Study of Zilucoplan in Subjects With Generalized Myasthenia Gravis

A treatment-emergent adverse event is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication.

The MG-ADL profile provides an assessment of MG symptom severity and measures 8 items on a 0-3 scale, with 0 being the least severe. The total sum of the 8 items represents the ADL score. The ADL score can range from 0 (least severe) to 24 (most severe).

The QMG test is a standardized quantitative strength scoring system and measures 13 items on a 0-3 scale, with 0 being the least severe. The total sum of the 13 items represents the QMG score. The QMG score can range from 0 (least severe) to 39 (most severe).

The MGC is a 10-item scale that has been used to measure the clinical status of patients with Myasthenia Gravis (MG) in order to evaluate treatment response. The MGC has 4-point Likerttype Scale response options ranging from 0 to 2, 3, 4, 5, 6 or 9 according to the item (weighted response options). The total score is the sum of all items (range 0-50) where higher scores indicate more severe impairment due to the disease.

The MG-QOL15r is a 15-item survey that was designed to assess quality of life in patients with MG. The MG-QoL has 3-point Likert Scale response options ranging from 0 to 2. The MGQoL15r score can range from 0 to 30, where higher scores indicate more severe impact of the disease on aspects of the patient's life.

Ra Pharmaceuticals, Inc.

Placebo

The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with generalized Myasthenia Gravis (gMG). Subjects will be randomized in a 1:1:1 ratio to receive daily SC doses of 0.1 mg/kg RA101495, 0.3 mg/kg RA101495, or matching placebo for 12 weeks.

Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis

A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of RA101495 in Subjects With Generalized Myasthenia Gravis

The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for myasthenia gravis (MG). The scale consists of 13 individual assessments, each scored on a 0-3 point scale (i.e., 0=none, 1=mild, 2=moderate, and 3=severe). The total score is the sum of the individual scores with a range of 0-39. Higher scores are representative of more severe impairment.

The MG-ADL is a brief 8-item survey designed to evaluate MG symptom severity. The scale consists of 8 items each scored on a 0-3 point scale (i.e., 0=none, 1=mild, 2=moderate, and 3=severe). The total score is the sum of the 8 individual scores with range of 0-24. Higher scores are associated with more severe symptoms of MG.

The MG-QOL15r is a 15-item survey that was designed to assess quality of life in participants with MG. The survey consisted of 15 questions and the corresponding responses were each scored on a 0-2 point scale (0=Not much at all, 1=Somewhat, 2=Very Much). The total score is the sum of the 15 individual item scores with a range of 0-30. Higher scores indicate more severe impact of the disease on aspects of the participant's life.

The MG Composite is a 10-item scale that has been used to measure the clinical status of participants with MG, in order to evaluate treatment response. It consists of 10 items which included ptosis (score range=0 to 3), double vision on lateral gaze left/right/both (score range=0 to 4), eye closure (score range=0 to 2), talking (score range=0 to 6), chewing (score range=0 to 6), swallowing (score range=0 to 6), breathing (score range=0 to 9), neck flexion or extension (score range=0 to 4), shoulder abduction (score range=0 to 5) and hip flexion (score range= 0 to 5). The total score is the sum of the 10 individual scores with a range of 0-50. Higher scores in the MG Composite indicate more severe impairment due to the disease.

The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consists of 13 individual assessments, each scored on a 0-3 point scale (i.e., 0=none, 1=mild, 2=moderate, and 3=severe). The total score is the sum of the individual scores with a range of 0-39. Higher scores are representative of more severe impairment.

Percentage of participants who used at least 1 dose of rescue medication were reported.

An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A serious adverse event (SAE) is any untoward medical occurrence that at any dose resulted in death, life-threatening, significant or persistent disability/incapacity, congenital anomaly/birth defect, important medical event, initial inpatient hospitalization or prolongation of hospitalization.

A BioTek ELx800 automated microplate reader is used to measure the optical density at 415 nanometer (nm) of human plasma samples to calculate the percent lysis of sheep erythrocytes that has occurred as a result of total complement activity. The measure of complement activity is determined by the degree of hemolysis of the erythrocytes.

Blood samples were collected from participants to assess Complement Component 5C levels.

RA102758 and RA103488 are the metabolites of RA101495.

Cmax is defined as the maximum observed plasma concentration.

Tmax is defined as the time to observe maximum plasma concentration.

Ra Pharmaceuticals

Treatment emergent adverse events (TEAEs) are defined as an AE that occurs after a treatment start date or an AE that increases in severity after treatment start date. In this study, AEs were planned to be reported for Main portion and Main + Extension portion only.

Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion.

Main Portion: RA101495 0.1 mg/kg

Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion

Main Portion: RA101495 0.3 mg/kg

Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion.

Main Portion: Placebo

Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants who completed main portion and were eligible to enter in the extension period, continued receiving RA101495 0.1 mg/kg up to 48 weeks (until protocol amendment v3.0) in the extension portion.

Main and Extension Portion: RA101495 0.1 mg/kg Before Switch to 0.3 mg/kg

Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants who completed main portion and were eligible to enter in the extension period, continued receiving RA101495 0.1 mg/kg up to Week 48 (until protocol amendment v3.0). Following implementation of protocol amendment v3.0, and upon appropriate reconsent, all study participants ongoing in the extension portion who had previously received the RA101495 0.1mg/kg/day dose switched to receive the RA101495 0.3mg/kg/day until RA101495 is approved and available in the territory, or the sponsor terminates development of RA101495 for gMG. In countries where RA101495 is not approved or marketed, but in which sponsored clinical studies had been conducted, participants received RA101495 through a compassionate use pathway in the extension portion (up to 122 weeks).

Main and Extension Portion: RA101495 0.1 mg/kg After Switch to 0.3 mg/kg

Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. At the end of the treatment period in the main portion, participants received the same dose of study drug in the extension portion until RA101495 is approved and available in the territory, or the sponsor terminates development of RA101495 for gMG. In countries where RA101495 is not approved or marketed, but in which sponsored clinical studies had been conducted, participants received RA101495 through a compassionate use pathway in the extension portion (up to 122 weeks).

Main and Extension Portion: RA101495 0.3 mg/kg

RA101495 0.1 mg/kg

Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. At the end of the treatment period in the main portion, participants received the same dose of study drug in the extension portion until RA101495 is approved and available in the territory, or the sponsor terminates development of RA101495 for generalized myasthenia gravis (gMG). In countries where RA101495 is not approved or marketed, but in which sponsored clinical studies had been conducted, participants received RA101495 through a compassionate use pathway in the extension portion (up to 122 weeks).

RA101495 0.3 mg/kg

Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion. At the end of the treatment period in the main portion, participants were randomized to receive either RA101495 0.1 mg/kg (up to 48 weeks) or RA101495 0.3 mg/kg (up to 122 weeks) in the extension portion, as a subcutaneous injection once daily.

Total

Age, Categorical

Age, Continuous

Sex: Female, Male

Asian

Black or African American

White

Race/Ethnicity, Customized

Intent-to-Treat (ITT) population included all randomized participants.

Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.

RA101495 0.1 mg/kg (mITT)

Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.

RA101495 0.3 mg/kg (mITT)

Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the mITT population which included all participants in the ITT population who had received at least 1 dose of study drug.

Placebo (mITT)

The modified ITT (mITT) population included all participants in the ITT population who had received at least 1 dose of study drug.

Main Portion: Change From Baseline to Week 12 in Quantitative Myasthenia Gravis (QMG) Score

The mITT population included all participants in the ITT population who had received at least 1 dose of study drug.

Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) Scale

Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life 15r (MG-QOL15r) Survey

Main Portion: Change From Baseline to Week 12 in the MG Composite Scale Total Score

Main Portion: Percentage of Participants With >= 3-point Reduction in QMG Total Score at Week 12

Main Portion: Percentage of Participants Who Required Rescue Therapy Over the 12-week Treatment Period

SAEs

Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the safety set (SS) population which included all participants who had received at least 1 dose of study drug (i.e., mITT Population), with participants to be analyzed based on the actual treatment received.

RA101495 0.1 mg/kg (SS)

Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the SS population which included all participants who had received at least 1 dose of study drug (i.e., mITT Population), with participants to be analyzed based on the actual treatment received.

RA101495 0.3 mg/kg (SS)

Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the SS population which included all participants who had received at least 1 dose of study drug (i.e., mITT Population), with participants to be analyzed based on the actual treatment received.

Placebo (SS)

The safety population included all participants who had received at least 1 dose of study drug (i.e., mITT Population), with participants to be analyzed based on the actual treatment received.

Main Portion: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed PD population which included all participants in mITT population who had at least 1 evaluable PD assessment.

RA101495 0.1 mg/kg (PD)

Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed PD population which included all participants in mITT population who had at least 1 evaluable PD assessment.

RA101495 0.3 mg/kg (PD)

Participants received placebo matched to RA101495 as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed PD population which included all participants in mITT population who had at least 1 evaluable PD assessment.

Placebo (PD)

The pharmacodynamic (PD) population included all participants in mITT Population who had at least 1 evaluable PD assessment.

Main Portion: Change From Baseline in Sheep Red Blood Cell (sRBC) Lysis Assay at Week 12 (Pre-dose)

Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in main portion. Participants formed PD population which included all participants in mITT population who had at least 1 evaluable PD assessment.

The PD population included all participants in mITT Population who had at least 1 evaluable PD assessment.

Main Portion: Change From Baseline in C5 Levels at Week 12 (Pre-dose)

RA101495- Day 1: 1 hour postdose

RA101495- Day 1: 3 hours postdose

RA101495- Day 1: 6 hours postdose

RA101495- Week 1: Pre-dose

RA101495- Week 2: Pre-dose

RA101495- Week 4: Pre-dose

RA101495- Week 8: Pre-dose

RA101495- Week 12: Pre-dose

RA102758- Day 1: 1 hour postdose

RA102758- Day 1: 3 hours postdose

RA102758- Day 1: 6 hours postdose

RA102758- Week 1: Pre-dose

RA102758- Week 2: Pre-dose

RA102758- Week 4: Pre-dose

RA102758- Week 8: Pre-dose

RA102758- Week 12: Pre-dose

RA103488- Day 1: 1 hour postdose

RA103488- Day 1: 3 hours postdose

RA103488- Day 1: 6 hours postdose

RA103488- Week 1: Pre-dose

RA103488- Week 2: Pre-dose

RA103488- Week 4: Pre-dose

RA103488- Week 8: Pre-dose

RA103488- Week 12: Pre-dose

Participants received RA101495 0.1 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the PK population which included all participants in mITT population who had at least 1 evaluable PK assessment.

RA101495 0.1 mg/kg (PK)

Participants received RA101495 0.3 mg/kg as a subcutaneous injection once daily for 12 weeks in the main portion. Participants formed the PK population which included all participants in mITT population who had at least 1 evaluable PK assessment.

RA101495 0.3 mg/kg (PK)

The pharmacokinetic (PK) population included all participants in mITT population who had at least 1 evaluable PK assessment. Here "Number Analyzed" signifies number of participants who were evaluable at specified time points. 1 Participant (who was randomized to Placebo) mistakenly received Zilucoplan on Day 1 following a dispensing error (which was documented as a minor PD). Due to data confidentiality reasons, the results have not been reported for the placebo arm.

Main Portion: Plasma Concentration of RA101495 and Its Major Metabolites

The PK Population included all participants in mITT population who had at least 1 evaluable PK assessment. 1 Participant (who was randomized to Placebo) mistakenly received Zilucoplan on Day 1 following a dispensing error (which was documented as a minor PD). Due to data confidentiality reasons, the results have not been reported for the placebo arm.

Main Portion: Maximum Plasma Concentration (Cmax) on Day 1

Main Portion: Time Corresponding to Cmax (Tmax) on Day 1

RA102758/RA101495- Day 1: Pre-dose

RA102758/RA101495- Day 1: 1 hour postdose

RA102758/RA101495- Day 1: 3 hours postdose

RA102758/RA101495- Day 1: 6 hours postdose

RA102758/RA101495- Week 1: Pre-dose

RA102758/RA101495- Week 2: Pre-dose

RA102758/RA101495- Week 4: Pre-dose

RA102758/RA101495- Week 8: Pre-dose

RA102758/RA101495- Week 12: Pre-dose

RA103488/RA101495- Day 1: Pre-dose

RA103488/RA101495- Day 1: 1 hour postdose

RA103488/RA101495- Day 1: 3 hours postdose

RA103488/RA101495- Day 1: 6 hours postdose

RA103488/RA101495- Week 1: Pre-dose

RA103488/RA101495- Week 2: Pre-dose

RA103488/RA101495- Week 4: Pre-dose

RA103488/RA101495- Week 8: Pre-dose

RA103488/RA101495- Week 12: Pre-dose

The PK Population included all participants in mITT population who had at least 1 evaluable PK assessment. Here "Number Analyzed" signifies number of participants who were evaluable at specified time points. 1 Participant (who was randomized to Placebo) mistakenly received Zilucoplan on Day 1 following a dispensing error (which was documented as a minor PD). Due to data confidentiality reasons, the results have not been reported for the placebo arm.

Main Portion: Metabolites (RA102758 and RA103488) to Parent Ratio

Participants received RA101495 0.1 milligram/kilogram (mg/kg) as a subcutaneous injection once daily for 12 weeks in the main portion. Participants who completed main portion and were eligible to enter in the extension portion, continued receiving RA101495 0.1 mg/kg up to 48 weeks (until protocol amendment v3.0) in the extension portion (EP).

Following implementation of protocol amendment v3.0, and upon appropriate reconsent, all study participants ongoing in the extension portion who had previously received the RA101495 0.1mg/kg/day dose switched to receive the RA101495 0.3mg/kg/day until RA101495 is approved and available in the territory, or the sponsor terminates development of RA101495 for gMG. In countries where RA101495 is not approved or marketed, but in which sponsored clinical studies had been conducted, participants received RA101495 through a compassionate use pathway in the extension portion (up to 122 weeks).

RA101495 0.1 mg/kg (Before Switch) to 0.3 mg/kg (After Switch)

Main Portion (12 Weeks)

EP:Before Switch to 0.3 mg/kg(Week13-48)

EP:After Switch to 0.3 mg/kg(Week48-122)

The RAISE study is a multicenter, randomized, double-blind, placebo controlled study to confirm the efficacy, safety, and tolerability of zilucoplan in subjects with generalized Myasthenia Gravis. Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg zilucoplan or placebo for 12 weeks.

Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis

A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis

Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed;in this case and to protect participants, individual patient-level data would not be made available.

The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. A decrease from Baseline score indicated improvement.

The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. A decrease from Baseline score indicated improvement.

The total MGC score was sum of responses to 10 individual items : 1. Ptosis upward gaze (0 to 3), 2. Double vision on lateral gaze, left or right (0, to 4), 3. Eye closure (0 to 2), 4. Talking (0 to 6), 5. Chewing (0 to 6), 6. Swallowing \[0 to 6\], 7. Breathing (0 to 9), 8. Neck flexion or extension (0 to 4), 9. Shoulder abduction (0 to 5), 10. Hip flexion (0 to 5). The higher score for each item indicated severity. The total score ranged 0 to 50 with higher score indicative of severe disease activity). A decrease from Baseline score showed improvement.

The MG-QoL15r is a 15-item patient-reported outcome measure designed to assess quality of life in patients with MG. Each item in the scale scored on a 0 to 2-point scale (0=Not much at all, 1=Somewhat, 2=Very much). The total score was the sum of the 15 individual item scores, ranging from 0 to 30. Higher scores indicated more severe impact of the disease on aspects of the patient's life. A decrease from Baseline score indicated improvement.

Time to first receipt of rescue therapy over the 12-week treatment period (in days) was defined as the date of first rescue therapy use minus date of first Investigational Medicinal Product (IMP) + 1.

Percentage of Participants achieving MSE was defined as achieving a MG-ADL value of a 0 (No MG symptoms) or 1 (Mild MG symptoms) at Week 12 and not having taken rescue therapy. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the missing at Random (MAR) assumption.

Percentage of participants achieving a ≥ 3-point reduction in MG-ADL Score at Week 12 without rescue therapy were reported. The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored on a 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption.

Percentage of participants achieving a ≥5-point reduction in QMG Score without rescue therapy at Week 12 were reported. The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption.

A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.

Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.

Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.

Zilucoplan 0.3 mg/kg

American Indian/Alaska native

Black

The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
An Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Zilucoplan in Participants With Generalized Myasthenia Gravis Who Were Previously Receiving Intravenous Complement Component 5 Inhibitors	NCT05514873	Generalized Mya...	zilucoplan (RA1...	18 Years - 85 Years	UCB Pharma
Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis	NCT04225871	Generalized Mya...	zilucoplan (RA1...	18 Years -	UCB Pharma
Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis	NCT03315130	Generalized Mya...	zilucoplan (RA1... Placebo	18 Years - 85 Years	UCB Pharma
Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis	NCT04115293	Myasthenia Grav...	zilucoplan (RA1... Placebo	18 Years - 74 Years	UCB Pharma

Spots Global Cancer Trial Database for zilucoplan (ra101495)