Trial Outcomes & Findings for A Study of RVT-1401 in Myasthenia Gravis (MG) Patients (NCT NCT03863080)

Last Updated: 2023-12-20

Results Overview

An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. SAEs were defined as any untoward medical occurrence that, at any dose: resulted in death, was life-threatening, required hospitalization or prolongation of existing hospitalization, resulted in disability/incapacity, was a congenital anomaly/birth defect, or was an important medical event that may have jeopardized the participant or may have required medical or surgical intervention to prevent one of the other outcomes listed in the definition.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

17 participants

Primary outcome timeframe

Up to Week 18

Results posted on

2023-12-20

Participant Flow

This was a Phase 2a, randomized, double-blind (DB), placebo-controlled study with an Open-Label Extension (OLE) that was designed to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RVT-1401 versus placebo in acetylcholine receptor (AChR) antibody positive myasthenia gravis (MG) participants.

A total of 17 participants were enrolled in the study. One participant completed the DB period and did not enter the OLE; this participant completed the 12-week post Double-blind Treatment Follow-up Period.

Participant milestones

Participant milestones
Measure	Double-blind Treatment Period: Placebo Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W),x 3 doses followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 340 mg/Week Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Open-label Extension Period: RVT-1401 340 mg Biweekly Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period (6 Weeks) STARTED	6	5	6	0
Double-Blind Treatment Period (6 Weeks) COMPLETED	6	5	5	0
Double-Blind Treatment Period (6 Weeks) NOT COMPLETED	0	0	1	0
Open Label Extension Period (12 Weeks) STARTED	0	0	0	15
Open Label Extension Period (12 Weeks) COMPLETED	0	0	0	9
Open Label Extension Period (12 Weeks) NOT COMPLETED	0	0	0	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Double-blind Treatment Period: RVT-1401 680 mg/Week Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Open-label Extension Period: RVT-1401 340 mg Biweekly Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period (6 Weeks) Adverse Event	1	0
Open Label Extension Period (12 Weeks) Withdrawal by Subject	0	3
Open Label Extension Period (12 Weeks) Adverse Event	0	3

Baseline Characteristics

A Study of RVT-1401 in Myasthenia Gravis (MG) Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Double-blind Treatment Period: Placebo n=6 Participants Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 340 mg/Week n=5 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment. Participants	Double-blind Treatment Period: RVT-1401 680 mg/Week n=6 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Total n=17 Participants Total of all reporting groups
Age, Continuous	41.0 Years STANDARD_DEVIATION 15.40 • n=5 Participants	56.8 Years STANDARD_DEVIATION 22.17 • n=7 Participants	70.8 Years STANDARD_DEVIATION 14.22 • n=5 Participants	56.2 Years STANDARD_DEVIATION 20.67 • n=4 Participants
Sex: Female, Male Female	4 Participants n=5 Participants	2 Participants n=7 Participants	1 Participants n=5 Participants	7 Participants n=4 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants	10 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=5 Participants	5 Participants n=7 Participants	5 Participants n=5 Participants	16 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) White	6 Participants n=5 Participants	5 Participants n=7 Participants	6 Participants n=5 Participants	17 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants

PRIMARY outcome

Timeframe: Up to Week 18

Population: Safety Analysis Set.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=6 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=6 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) Any AE	5 Participants	4 Participants	5 Participants
Double-Blind Treatment Period: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) Any SAE	0 Participants	0 Participants	1 Participants

PRIMARY outcome

Timeframe: Up to Week 18

Population: Open-label Extension (OLE) Analysis Set comprised of all participants who enrolled in the OLE study phase and received at least 1 dose of study medication in the OLE. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. TEAEs are defined as those AEs that started or worsened in severity after the initiation of study drug administration. SAEs were defined as any untoward medical occurrence that, at any dose: resulted in death, was life-threatening, required hospitalization or prolongation of existing hospitalization, resulted in disability/incapacity, was a congenital anomaly/birth defect, or was an important medical event that may have jeopardized the participant or may have required medical or surgical intervention to prevent one of the other outcomes listed in the definition.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=15 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Open-Label Extension Period: Number of Participants Reporting AEs and SAEs Any AE	10 Participants	—	—
Open-Label Extension Period: Number of Participants Reporting AEs and SAEs Any SAE	1 Participants	—	—

PRIMARY outcome

Timeframe: Up to Week 7

Population: Safety Analysis Set.

Vital signs including systolic blood pressure (SBP), diastolic blood pressure (DBP), pulse rate and temperature were measured after resting for at least 5 minutes in a semi-supine position.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=6 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=6 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period: Number of Participants With Clinically Significant Changes in Vital Signs	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Up to Week 18

Population: OLE Analysis Set. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

Vital signs including SBP, DBP, pulse rate and temperature were measured after resting for at least 5 minutes in a semi-supine position.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=6 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=4 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Open-label Extension Period: Number of Participants With Clinically Significant Changes in Vital Signs	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Up to Week 7

Population: Safety Analysis Set.

Clinical laboratory parameters included clinical chemistry, hematology and urinalysis.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=6 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=6 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-blind Treatment Period: Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Up to Week 18

Population: OLE Analysis Set. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

Clinical laboratory parameters included clinical chemistry, hematology and urinalysis.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=6 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=4 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Open-label Extension Period: Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Up to Week 7

Population: Safety Analysis Set.

Twelve-lead ECG was performed after 5 minutes of rest in the supine position.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=6 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=6 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period: Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG)	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Up to Week 18

Population: OLE Analysis Set. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

Twelve-lead ECG was performed after 5 minutes of rest in the supine position.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=6 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=4 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Open-label Extension Period: Number of Participants With Clinically Significant Changes in ECG	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Baseline (Day 1) and Up to Week 7

Population: Full Analysis Set comprised of all randomized participants who received at least 1 dose of randomized study medication with at least 1 valid post-treatment value. Only those participants with data available at specified time points has been presented.

Serum samples were collected for the analysis of total immunoglobulin G. Percent change from Baseline was calculated as the mean value at the specified time frame (Week 7) minus the Baseline value, divided by the Baseline value x 100.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=5 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-blind Treatment Period: Percent Change From Baseline in Levels of Total Immunoglobulin G (IgG)	-3.17 Percent change Standard Error 2.97	-59.49 Percent change Standard Error 3.15	-77.66 Percent change Standard Error 2.92

PRIMARY outcome

Timeframe: Baseline (Day 1) and Up to Week 7

Population: Full Analysis Set. Only those participants with data available at specified time points has been presented.

Serum samples were collected for the analysis of IgG 1, 2, 3 and 4 levels. Percent change from Baseline was calculated as the mean value at the specified time frame (Week 7) minus the Baseline value, divided by the Baseline value x 100.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=5 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=4 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-blind Treatment Period: Percent Change From Baseline in IgG Subclasses 1, 2, 3 and 4 IgG1	-3.3 Percent change Standard Error 3.67	-65.9 Percent change Standard Error 4.02	-76.3 Percent change Standard Error 3.98
Double-blind Treatment Period: Percent Change From Baseline in IgG Subclasses 1, 2, 3 and 4 IgG2	-2.2 Percent change Standard Error 5.35	-54.7 Percent change Standard Error 5.65	-63.6 Percent change Standard Error 5.60
Double-blind Treatment Period: Percent Change From Baseline in IgG Subclasses 1, 2, 3 and 4 IgG3	-2.1 Percent change Standard Error 4.69	-68.2 Percent change Standard Error 5.05	-82.9 Percent change Standard Error 5.12
Double-blind Treatment Period: Percent Change From Baseline in IgG Subclasses 1, 2, 3 and 4 IgG4	-3.1 Percent change Standard Error 5.68	-48.0 Percent change Standard Error 6.08	-64.7 Percent change Standard Error 6.10

PRIMARY outcome

Timeframe: Baseline (Day 1) and Week 7

Population: Full Analysis Set. Only those participants with data available at definite timepoints has been presented.

Serum samples were collected for the analysis of Anti-AChR-IgG. Percent change from Baseline was calculated as the mean value at the specified time frame (Week 7) minus the Baseline value, divided by the Baseline value x 100.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=5 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=4 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period: Percent Change From Baseline in Anti-acetylcholine Receptor Immunoglobulin G (Anti-AChR-IgG) at Week 7	15.92 Percent change Standard Error 12.41	-46.66 Percent change Standard Error 13.64	-85.28 Percent change Standard Error 12.27

SECONDARY outcome

Timeframe: Pre-dose; Day 1, Day 3, Day 5, Day 8, Day 15, Day 22, Day 29, Day 36, Day 38, Day 40, Week 7, Week 8

Population: Pharmacokinetic (PK) Analysis Set comprised of all participants who underwent PK sampling and had evaluable concentration-time data for analysis. As PK analyses were not conducted, a PK Analysis Set was not presented.

Blood samples were collected for the analysis of Pharmacokinetic parameter AUC (0-168h).

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=5 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=6 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period: Area Under the Concentration-time Curve From Time 0 to 168 Hours (AUC0-168h) of RVT-1401	NA Hours*microgram per milliliter Geometric Coefficient of Variation NA Not applicable (NA) indicates data was not available as all concentration values were below the lower limit of quantification.	NA Hours*microgram per milliliter Geometric Coefficient of Variation NA NA indicates data was not available as all concentration values were below the lower limit of quantification.	—

SECONDARY outcome

Timeframe: Pre-dose; Day 1, Day 3, Day 5, Day 8, Day 15, Day 22, Day 29, Day 36, Day 38, Day 40, Week 7, Week 8, Week 9, Week 12 and Week 14

Population: Pharmacokinetic Analysis Set. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

Pharmacokinetic parameters were not estimated because the sparse PK sampling schedule did not allow for accurate estimates of these parameters.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=5 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=4 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Open-label Extension Period: AUC0-168h of RVT-1401	NA Hours*microgram per milliliter Geometric Coefficient of Variation NA NA indicates data was not available as all concentration values were below the lower limit of quantification.	NA Hours*microgram per milliliter Geometric Coefficient of Variation NA NA indicates data was not available as all concentration values were below the lower limit of quantification.	—

SECONDARY outcome

Timeframe: Pre-dose; Day 1, Day 3, Day 5, Day 8, Day 15, Day 22, Day 29, Day 36, Day 38, Day 40, Week 7, Week 8

Population: Pharmacokinetic Analysis Set.

Blood samples were collected for the analysis of Pharmacokinetic parameter Cmax.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=5 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=6 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period: Maximum Concentration (Cmax) of RVT-1401	NA micrograms per milliliter Geometric Coefficient of Variation NA NA indicates data was not available as all concentration values were below the lower limit of quantification.	NA micrograms per milliliter Geometric Coefficient of Variation NA NA indicates data was not available as all concentration values were below the lower limit of quantification.	—

SECONDARY outcome

Timeframe: Pre-dose; Day 1, Day 3, Day 5, Day 8, Day 15, Day 22, Day 29, Day 36, Day 38, Day 40, Week 7, Week 8, Week 9, Week 12 and Week 14

Population: Pharmacokinetic Analysis Set. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

Blood samples were collected for the analysis of Pharmacokinetic parameter Cmax.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=5 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=4 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Open-label Extension Period: Cmax of RVT-1401	NA Micrograms per milliliter Geometric Coefficient of Variation NA NA indicates data was not available as all concentration values were below the lower limit of quantification.	NA Micrograms per milliliter Geometric Coefficient of Variation NA NA indicates data was not available as all concentration values were below the lower limit of quantification.	—

SECONDARY outcome

Timeframe: Pre-dose

Population: Pharmacokinetic Analysis Set. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

Blood samples were planned to be collected at indicated time points to measure the concentration of RVT-1401 pre-dose (Ctrough) as an assessment of the pharmacokinetic RVT-1401.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=5 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=6 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period: Trough Concentrations (Ctrough) of RVT-1401	NA Milligrams per liter Standard Deviation NA NA indicates data could not be calculated for double-blind treatment period due to high proportion of non-quantifiable values.	NA Milligrams per liter Standard Deviation NA NA indicates data could not be calculated for double-blind treatment period due to high proportion of non-quantifiable values.	—

SECONDARY outcome

Timeframe: Pre-dose

Population: Pharmacokinetic Analysis Set. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.

Blood samples were planned to be collected at indicated time points to measure the concentration of RVT-1401 pre-dose (Ctrough) as an assessment of the pharmacokinetic RVT-1401.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=5 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=4 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Open-label Extension Period: Ctrough of RVT-1401	NA Milligrams per liter Standard Deviation NA NA indicates data could not be calculated for Open-label treatment period due to high proportion of non-quantifiable values.	NA Milligrams per liter Standard Deviation NA NA indicates data could not be calculated for Open-label treatment period due to high proportion of non-quantifiable values.	—

SECONDARY outcome

Timeframe: Baseline (Day 1) and at Week 2, Week 3, Week 4, Week 5, Week 6 (Day 36) and Week 7

Population: Full Analysis Set. Only those participants with data available at specified time points has been represented (represented by n=X in the category titles).

The QMG score is a physician-reported outcome measure was used to assess MG disease severity and pattern of deficits based on quantitative testing of affected muscle groups. The scale comprised of 13 test items that were graded on a scale of 0 (no myasthenic findings) to 3 (maximal myasthenic deficits). The total sum across all 13 items represents the QMG score. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease. Baseline was defined as the last non-missing value prior to the date (time) of first dose of study drug. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=6 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=6 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-blind Treatment Period: Change From Baseline in the Quantitative Myasthenia Gravis Score (QMG) Score Week 2, n=6,5,6	-3.3 Scores on a scale Standard Deviation 2.16	-2.8 Scores on a scale Standard Deviation 5.36	-1.7 Scores on a scale Standard Deviation 3.14
Double-blind Treatment Period: Change From Baseline in the Quantitative Myasthenia Gravis Score (QMG) Score Week 3, n=6,5,6	-2.5 Scores on a scale Standard Deviation 2.74	-4.6 Scores on a scale Standard Deviation 7.70	-2.5 Scores on a scale Standard Deviation 2.95
Double-blind Treatment Period: Change From Baseline in the Quantitative Myasthenia Gravis Score (QMG) Score Week 4, n=6,5,5	-2.7 Scores on a scale Standard Deviation 2.73	-3.6 Scores on a scale Standard Deviation 6.43	-3.6 Scores on a scale Standard Deviation 4.16
Double-blind Treatment Period: Change From Baseline in the Quantitative Myasthenia Gravis Score (QMG) Score Week 5, n=6,5,5	-2.7 Scores on a scale Standard Deviation 2.73	-3.2 Scores on a scale Standard Deviation 6.80	-3.2 Scores on a scale Standard Deviation 3.70
Double-blind Treatment Period: Change From Baseline in the Quantitative Myasthenia Gravis Score (QMG) Score Week 6 (Day 36), n=6,5,5	-3.0 Scores on a scale Standard Deviation 3.22	-4.0 Scores on a scale Standard Deviation 6.75	-4.4 Scores on a scale Standard Deviation 3.71
Double-blind Treatment Period: Change From Baseline in the Quantitative Myasthenia Gravis Score (QMG) Score Week 7, n=5,5,5	-1.8 Scores on a scale Standard Deviation 3.27	-4.0 Scores on a scale Standard Deviation 6.82	-3.8 Scores on a scale Standard Deviation 4.76

SECONDARY outcome

Timeframe: Baseline (Day 1) to Week 7

Population: Full Analysis Set. Only those participants with data available at specified time points has been presented.

The response is defined as improvement from Baseline on the QMG score by =\> 3 points. The QMG score is a physician-reported outcome measure was used to assess MG disease severity and pattern of deficits based on quantitative testing of affected muscle groups. The scale comprised of 13 test items that were graded on a scale of 0 (no myasthenic findings) to 3 (maximal myasthenic deficits). The total sum across all 13 items represents the QMG score. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease. Baseline was defined as the last non-missing value prior to the date (time) of first dose of study drug. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=5 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period: Percentage of Participants With an Improvement/ Response on the QMG Score From Baseline	20.0 Percentage of participants	40.0 Percentage of participants	60.0 Percentage of participants

SECONDARY outcome

Timeframe: Baseline (Day 1) and at Week 2, Week 3, Week 4, Week 5, Week 6 (Day 36), Week 7

Population: Full Analysis Set. Only those participants with data available at specified time points has been presented.

The MG-ADL is an 8-item, participant-reported outcome measure that assessed Myasthenia Gravis symptoms and their effects on activities of daily living, with each response graded from 0 (normal) to 3 (most severe). The MG-ADL score was calculated by totaling the rating for each of the 8 items. Total MG-ADL scores range from 0 to 24 with higher scores indicating more severe disease. Baseline was defined as the last non-missing value prior to the date (time) of first dose of study drug. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=6 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=6 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Activities of Daily Living (MG-ADL) Score Week 2	-1.7 Scores on a scale Standard Deviation 2.34	-3.2 Scores on a scale Standard Deviation 6.61	-0.7 Scores on a scale Standard Deviation 0.82
Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Activities of Daily Living (MG-ADL) Score Week 3	-0.8 Scores on a scale Standard Deviation 2.04	-3.8 Scores on a scale Standard Deviation 5.97	-1.7 Scores on a scale Standard Deviation 1.97
Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Activities of Daily Living (MG-ADL) Score Week 4	-0.8 Scores on a scale Standard Deviation 2.64	-3.2 Scores on a scale Standard Deviation 6.38	-2.0 Scores on a scale Standard Deviation 2.35
Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Activities of Daily Living (MG-ADL) Score Week 5	-1.5 Scores on a scale Standard Deviation 3.02	-3.2 Scores on a scale Standard Deviation 6.65	-2.0 Scores on a scale Standard Deviation 2.35
Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Activities of Daily Living (MG-ADL) Score Week 6 (Day 36)	-1.2 Scores on a scale Standard Deviation 3.19	-5.2 Scores on a scale Standard Deviation 7.09	-3.4 Scores on a scale Standard Deviation 3.36
Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Activities of Daily Living (MG-ADL) Score Week 7	-0.2 Scores on a scale Standard Deviation 2.71	-4.4 Scores on a scale Standard Deviation 7.06	-3.2 Scores on a scale Standard Deviation 3.11

SECONDARY outcome

Timeframe: Baseline (Day 1) to Week 7

Population: Full Analysis Set. Only those participants with data available at specified time points has been presented.

The response was defined as improvement (decrease) from Baseline on the MG-ADL score by =\> 2 points. The MG-ADL is an 8-item, participant-reported outcome measure that assessed Myasthenia Gravis symptoms and their effects on activities of daily living, with each response graded from 0 (normal) to 3 (most severe). The MG-ADL score was calculated by totaling the rating for each of the 8 items. Total MG-ADL scores range from 0 to 24 with higher scores indicating more severe disease. Baseline was defined as the last non-missing value prior to the date (time) of first dose of study drug.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=6 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period: Percentage of Participants With an Improvement/ Response on the MG-ADL Score	33.3 Percentage of participants	60.0 Percentage of participants	60.0 Percentage of participants

SECONDARY outcome

Timeframe: Baseline (Day 1) and at Week 2, Week 3, Week 4, Week 5, Week 6 (Day 36), Week 7

Population: Full Analysis Set. Only those participants with data available at specified timepoints has been presented.

The MGC was developed by selecting the best performing items from 3 commonly used Myasthenia Gravis specific scales (QMG, Myasthenia Gravis manual muscle test, and MG-ADL) and is comprised of 10 functional domains: 3 ocular, 3 bulbar, 1 respiratory, 1 neck, and 2 limb items. The total score ranges from 0 (no myasthenic findings) to 50 (maximal myasthenic deficits). The scale measures symptoms and signs of MG in these domains incorporating both physician and participant-reported test items. Higher scores correlate with clinical worsening of the disease. Baseline was defined as the last non-missing value prior to the date (time) of first dose of study drug. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=6 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=6 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Composite Score (MGC) Score Week 2	-2.3 Scores on a scale Standard Deviation 2.94	-7.0 Scores on a scale Standard Deviation 9.27	-3.0 Scores on a scale Standard Deviation 3.10
Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Composite Score (MGC) Score Week 3	-1.3 Scores on a scale Standard Deviation 5.20	-8.8 Scores on a scale Standard Deviation 9.98	-2.3 Scores on a scale Standard Deviation 2.88
Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Composite Score (MGC) Score Week 4	-2.0 Scores on a scale Standard Deviation 8.10	-7.2 Scores on a scale Standard Deviation 10.40	-4.0 Scores on a scale Standard Deviation 6.60
Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Composite Score (MGC) Score Week 5	-4.3 Scores on a scale Standard Deviation 7.50	-7.0 Scores on a scale Standard Deviation 10.07	-2.8 Scores on a scale Standard Deviation 4.97
Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Composite Score (MGC) Score Week 6 (Day 36)	-2.5 Scores on a scale Standard Deviation 8.04	-10.4 Scores on a scale Standard Deviation 9.32	-6.6 Scores on a scale Standard Deviation 5.13
Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Composite Score (MGC) Score Week 7	-0.8 Scores on a scale Standard Deviation 7.33	-9.0 Scores on a scale Standard Deviation 10.12	-7.0 Scores on a scale Standard Deviation 5.34

SECONDARY outcome

Timeframe: Baseline (Day 1) to Week 7

Population: Full Analysis Set. Only those participants with data available at specified time points has been presented.

The response was defined as improvement (decrease) from Baseline on the MGC score by =\> 3 points. The MGC was developed by selecting the best performing items from 3 commonly used Myasthenia Gravis specific scales (QMG, Myasthenia Gravis manual muscle test, and MG-ADL) and is comprised of 10 functional domains: 3 ocular, 3 bulbar, 1 respiratory, 1 neck, and 2 limb items. The total score ranges from 0 (no myasthenic findings) to 50 (maximal myasthenic deficits). The scale measures symptoms and signs of MG in these domains incorporating both physician and participant-reported test items. Higher scores correlate with clinical worsening of the disease. Baseline was defined as the last non-missing value prior to the date (time) of first dose of study drug.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=5 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period: Percentage of Participants With an Improvement on the MGC Score	40.0 Percentage of participants	60.0 Percentage of participants	60.0 Percentage of participants

SECONDARY outcome

Timeframe: Baseline (Day 1) and at Week 4 and Week 7

Population: Full Analysis Set. Only those participants with data available at specified time points has been presented.

The MG-QOL15r is a participant-reported questionnaire designed to assess how a participant's Myasthenia Gravis affects different aspects related to their quality of life. The scale includes 15 items that are graded on a scale of 0 to2; the total across is the sum of all 15 items and represents the MG-QOL15r score. The range of the MG-QOL15r score is 0 - 30. Higher scores indicate worse outcomes. Baseline was defined as the last non-missing value prior to the date (time) of first dose of study drug. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=6 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=4 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Quality of Life 15 Revised Score (MG-QOL 15r) Score Week 4	-3.5 Scores on a scale Standard Deviation 3.83	-6.0 Scores on a scale Standard Deviation 9.77	1.6 Scores on a scale Standard Deviation 5.03
Double-Blind Treatment Period: Change From Baseline in the Myasthenia Gravis Quality of Life 15 Revised Score (MG-QOL 15r) Score Week 7	-2.3 Scores on a scale Standard Deviation 3.93	-6.6 Scores on a scale Standard Deviation 10.55	2.0 Scores on a scale Standard Deviation 4.42

SECONDARY outcome

Timeframe: Up to Week 7

Population: Full Analysis Set.

The serum levels of anti-RVT-1401 antibodies were determined. All samples that were potentially positive were analyzed with the confirmation assay where presence of anti-RVT-1401 was confirmed; the therapeutic antibody was used to compete with the analytical responses of anti-drug antibody (ADA) to assess specificity of screened positive samples.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=6 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=6 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Double-Blind Treatment Period: Number of Participants Reporting Confirmed Positive Anti-RVT-1401 Antibodies	2 Participants	1 Participants	1 Participants

SECONDARY outcome

Timeframe: Up to Week 18

Population: Full Analysis Set.

The serum levels of anti-RVT-1401 antibodies were determined. All samples that were potentially positive were analyzed with the confirmation assay where presence of anti-RVT-1401 was confirmed; the therapeutic antibody was used to compete with the analytical responses of ADA to assess specificity of screened positive samples.

Outcome measures

Outcome measures
Measure	Double-blind Treatment Period: RVT-1401 340 mg/Week n=6 Participants Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=5 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.	Double-blind Treatment Period: RVT-1401 680 mg/Week n=4 Participants Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Open-Label Extension Period: Number of Participants Reporting Confirmed Positive Anti-RVT-1401 Antibodies	1 Participants	0 Participants	0 Participants

Adverse Events

Double-blind Treatment Period: Placebo

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Double-blind Treatment Period: RVT-1401 340 mg/Week

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Double-blind Treatment Period: RVT-1401 680 mg/Week

Serious events: 1 serious events

Other events: 5 other events

Deaths: 0 deaths

Open-label Extension Period: RVT-1401 340 mg Biweekly

Serious events: 1 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Double-blind Treatment Period: Placebo n=6 participants at risk Participants were randomized to receive Placebo during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment. Participants	Double-blind Treatment Period: RVT-1401 340 mg/Week n=5 participants at risk Participants were randomized to receive 340 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment. Participants	Double-blind Treatment Period: RVT-1401 680 mg/Week n=6 participants at risk Participants were randomized to receive 680 mg/week during the 6-week double blind period. Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 Q2W x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment. Participants	Open-label Extension Period: RVT-1401 340 mg Biweekly n=15 participants at risk Participants who completed DB Treatment period entered an OLE where they received 340 mg RVT-1401 every 2 weeks (Q2W) x 3 doses, followed by a 6-week follow-up period where the participant did not receive any study treatment.
Infections and infestations Herpes simplex	0.00% 0/6 • From Baseline and Up to Week 18 Adverse events and Serious adverse events were collected in Safety Analysis Set in Double-Blind Treatment Period and in OLE Analysis Set in Open-Label Extension Period. Safety Analysis Set comprised of all participants who received at least 1 dose of study medication. OLE Analysis Set comprised of all participants who enrolled in the OLE study phase and received at least 1 dose of study medication in the OLE.	0.00% 0/5 • From Baseline and Up to Week 18 Adverse events and Serious adverse events were collected in Safety Analysis Set in Double-Blind Treatment Period and in OLE Analysis Set in Open-Label Extension Period. Safety Analysis Set comprised of all participants who received at least 1 dose of study medication. OLE Analysis Set comprised of all participants who enrolled in the OLE study phase and received at least 1 dose of study medication in the OLE.	16.7% 1/6 • From Baseline and Up to Week 18 Adverse events and Serious adverse events were collected in Safety Analysis Set in Double-Blind Treatment Period and in OLE Analysis Set in Open-Label Extension Period. Safety Analysis Set comprised of all participants who received at least 1 dose of study medication. OLE Analysis Set comprised of all participants who enrolled in the OLE study phase and received at least 1 dose of study medication in the OLE.	0.00% 0/15 • From Baseline and Up to Week 18 Adverse events and Serious adverse events were collected in Safety Analysis Set in Double-Blind Treatment Period and in OLE Analysis Set in Open-Label Extension Period. Safety Analysis Set comprised of all participants who received at least 1 dose of study medication. OLE Analysis Set comprised of all participants who enrolled in the OLE study phase and received at least 1 dose of study medication in the OLE.
Nervous system disorders Myasthenia gravis	0.00% 0/6 • From Baseline and Up to Week 18 Adverse events and Serious adverse events were collected in Safety Analysis Set in Double-Blind Treatment Period and in OLE Analysis Set in Open-Label Extension Period. Safety Analysis Set comprised of all participants who received at least 1 dose of study medication. OLE Analysis Set comprised of all participants who enrolled in the OLE study phase and received at least 1 dose of study medication in the OLE.	0.00% 0/5 • From Baseline and Up to Week 18 Adverse events and Serious adverse events were collected in Safety Analysis Set in Double-Blind Treatment Period and in OLE Analysis Set in Open-Label Extension Period. Safety Analysis Set comprised of all participants who received at least 1 dose of study medication. OLE Analysis Set comprised of all participants who enrolled in the OLE study phase and received at least 1 dose of study medication in the OLE.	0.00% 0/6 • From Baseline and Up to Week 18 Adverse events and Serious adverse events were collected in Safety Analysis Set in Double-Blind Treatment Period and in OLE Analysis Set in Open-Label Extension Period. Safety Analysis Set comprised of all participants who received at least 1 dose of study medication. OLE Analysis Set comprised of all participants who enrolled in the OLE study phase and received at least 1 dose of study medication in the OLE.	6.7% 1/15 • From Baseline and Up to Week 18 Adverse events and Serious adverse events were collected in Safety Analysis Set in Double-Blind Treatment Period and in OLE Analysis Set in Open-Label Extension Period. Safety Analysis Set comprised of all participants who received at least 1 dose of study medication. OLE Analysis Set comprised of all participants who enrolled in the OLE study phase and received at least 1 dose of study medication in the OLE.

Other adverse events

Additional Information

Central Study Contact

Immunovant, Inc

Phone: 1-800-797-0414

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place