Trial Outcomes & Findings for Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects. (NCT NCT02588833)

NCT ID: NCT02588833

Last Updated: 2021-01-11

Results Overview

TEAEs were defined as adverse events (AEs) that occurred after dosing on Day 1 and up to 30 days after the last dose of study drug. A treatment-related TEAE is defined as a TEAE with a relationship to study drug of probably, possibly, unlikely, or unrelated. A serious adverse event (SAE) is defined as any AE that resulted in death; was life-threatening; required hospitalization or prolongation of existing hospitalization; resulted in persistent or significant incapacity or substantial disruption of ability to conduct normal life functions; was a congenital anomaly or birth defect. TEAEs were graded according to Common Terminology Criteria for Adverse Events v4.03 based on: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening; Grade 5: Death related to AE.

• Recruitment status: COMPLETED
• Study phase: PHASE1
• Target enrollment: 23 participants
• Primary outcome timeframe: From first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days.
• Results posted on: 2021-01-11

Participant Flow

This Phase 1b, pilot study was conducted at 9 sites in 5 countries (Hong Kong, Malaysia, New Zealand, Thailand and United States) in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who had not received treatment with eculizumab (Soliris®) in the past, between 01 December 2015 and 26 August 2019. A total of 22 subjects were enrolled.

Subjects could participate in more than 1 cohort. The study consisted of 3 parts: Part 1 for Cohorts 1 and 2, Part 2 (Part 2A, Part 2B and Part 2C) for Cohort 2 only and Part 3 (safety follow-up). Cohort 2 dosing was not initiated until all subjects in Cohort 1 had completed Part 1 and review of emerging safety and efficacy data. Overall, 2 cohorts of 22 unique subjects were evaluated.

Participant milestones

Measure	Cohort 1 Subjects received subcutaneous (SC) injections of pegcetacoplan 180 milligrams (mg)/day for up to 28 days.	Cohort 2 Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Part 1 STARTED	3	20
Part 1 COMPLETED	2	20
Part 1 NOT COMPLETED	1	0
Part 2A STARTED	0	20
Part 2A COMPLETED	0	18
Part 2A NOT COMPLETED	0	2
Part 2B STARTED	0	18
Part 2B COMPLETED	0	17
Part 2B NOT COMPLETED	0	1
Part 2C STARTED	0	14
Part 2C COMPLETED	0	12
Part 2C NOT COMPLETED	0	2
Part 3 (Safety Follow-up) STARTED	3	4
Part 3 (Safety Follow-up) COMPLETED	3	2
Part 3 (Safety Follow-up) NOT COMPLETED	0	2

Reasons for withdrawal

Measure	Cohort 1 Subjects received subcutaneous (SC) injections of pegcetacoplan 180 milligrams (mg)/day for up to 28 days.	Cohort 2 Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Part 1 Withdrawal by Subject	1	0
Part 2A Physician Decision	0	1
Part 2A Withdrawal by Subject	0	1
Part 2B Adverse Event	0	1
Part 2C Adverse Event	0	1
Part 2C Withdrawal by Subject	0	1
Part 3 (Safety Follow-up) Other	0	2

Baseline Characteristics

Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.

Baseline characteristics by cohort

Measure	Cohort 1 n=3 Participants Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 n=20 Participants Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.	Total n=23 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants	19 Participants n=7 Participants	22 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	9 Participants n=7 Participants	10 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	11 Participants n=7 Participants	13 Participants n=5 Participants
Race/Ethnicity, Customized White	3 Participants n=5 Participants	1 Participants n=7 Participants	4 Participants n=5 Participants
Race/Ethnicity, Customized Asian	0 Participants n=5 Participants	15 Participants n=7 Participants	15 Participants n=5 Participants
Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Maori	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Other	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	3 Participants n=5 Participants	19 Participants n=7 Participants	22 Participants n=5 Participants
Race/Ethnicity, Customized Unknown	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: From first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days.

Population: The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug.

TEAEs were defined as adverse events (AEs) that occurred after dosing on Day 1 and up to 30 days after the last dose of study drug. A treatment-related TEAE is defined as a TEAE with a relationship to study drug of probably, possibly, unlikely, or unrelated. A serious adverse event (SAE) is defined as any AE that resulted in death; was life-threatening; required hospitalization or prolongation of existing hospitalization; resulted in persistent or significant incapacity or substantial disruption of ability to conduct normal life functions; was a congenital anomaly or birth defect. TEAEs were graded according to Common Terminology Criteria for Adverse Events v4.03 based on: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening; Grade 5: Death related to AE.

Outcome measures

Measure	Cohort 1 n=3 Participants Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 n=20 Participants Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity Treatment related TEAEs	2 Participants	9 Participants
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity Serious AEs	1 Participants	6 Participants
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity TEAEs with severe intensity	0 Participants	5 Participants
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity TEAEs with life threatening intensity	0 Participants	1 Participants
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity All TEAEs	2 Participants	18 Participants
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity TEAEs leading to study drug discontinuation	1 Participants	2 Participants
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity TEAEs leading to death	0 Participants	1 Participants
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity TEAEs with mild intensity	0 Participants	3 Participants
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity TEAEs with moderate intensity	2 Participants	8 Participants

PRIMARY outcome

Timeframe: Baseline (Day 1) and Day 365.

Population: The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.

Serum chemistry assessments of LDH were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. LDH results were summarized for Cohort 2 only.

Outcome measures

Measure	Cohort 1 n=17 Participants Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Mean Change From Baseline in Lactate Dehydrogenase (LDH) at Day 365	-2105.2 units per liter (U/L) Standard Deviation 1078.79	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Day 365.

Population: The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.

Serum chemistry assessments of LDH were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. LDH results were summarized for Cohort 2 only.

Outcome measures

Measure	Cohort 1 n=17 Participants Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Mean Percentage Change From Baseline in LDH at Day 365	-84.8 percent change Standard Deviation 14.04	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Day 365.

Population: The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.

Serum chemistry assessments of haptoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Haptoglobin results were summarized for Cohort 2 only.

Outcome measures

Measure	Cohort 1 n=17 Participants Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Mean Change From Baseline in Haptoglobin at Day 365	0.066 gram per liter (g/L) Standard Deviation 0.1245	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Day 365.

Population: The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.

Serum chemistry assessments of haptoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Haptoglobin results were summarized for Cohort 2 only.

Outcome measures

Measure	Cohort 1 n=17 Participants Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Mean Percentage Change From Baseline in Haptoglobin at Day 365	166.176 percent change Standard Deviation 311.3656	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Day 365.

Population: The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.

Hematology assessments of hemoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Hemoglobin results were summarized for Cohort 2 only.

Outcome measures

Measure	Cohort 1 n=17 Participants Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Mean Change From Baseline in Hemoglobin at Day 365	3.68 g/L Standard Deviation 2.690	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Day 365.

Population: The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.

Hematology assessments of hemoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Hemoglobin results were summarized for Cohort 2 only.

Outcome measures

Measure	Cohort 1 n=17 Participants Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Mean Percentage Change From Baseline in Hemoglobin at Day 365	49.86 percent change Standard Deviation 43.254	—

SECONDARY outcome

Timeframe: Baseline (Day 1) and Day 365.

Population: The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.

The FACIT-Fatigue Scale is a 13 item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). There are 13 statements with the total score ranging from 0 to 52 and higher scores indicating better quality of life. The FACIT-Fatigue Scale results were summarized for Cohort 2 only.

Outcome measures

Measure	Cohort 1 n=17 Participants Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Day 365	7.1 score on a scale Standard Deviation 11.09	—

SECONDARY outcome

Timeframe: Baseline (Day 1) and Day 365.

Population: The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.

Hematology assessments of ARC were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. ARC results were summarized for Cohort 2 only.

Outcome measures

Measure	Cohort 1 n=17 Participants Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at Day 365	-105.9 10^9 cells/L Standard Deviation 70.28	—

SECONDARY outcome

Timeframe: Baseline (Day 1) and Day 365.

Population: The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.

Hematology assessments of ARC were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. ARC results were summarized for Cohort 2 only.

Outcome measures

Measure	Cohort 1 n=17 Participants Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Mean Percentage Change From Baseline in ARC at Day 365	-47.5 percent change Standard Deviation 26.86	—

SECONDARY outcome

Timeframe: Baseline (Day 1) and Day 365.

Population: The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.

Serum chemistry assessments of total bilirubin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the 3 parts of the treatment period. Total bilirubin results were summarized for Cohort 2 only.

Outcome measures

Measure	Cohort 1 n=17 Participants Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Mean Change From Baseline in Total Bilirubin at Day 365	-29.9 micromole per liter (umol/L) Standard Deviation 24.34	—

SECONDARY outcome

Timeframe: Baseline (Day 1) and Day 365.

Population: The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.

Serum chemistry assessments of total bilirubin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the 3 parts of the treatment period. Total bilirubin results were summarized for Cohort 2 only.

Outcome measures

Measure	Cohort 1 n=17 Participants Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Mean Percentage Change From Baseline in Total Bilirubin at Day 365	-60.9 percent change Standard Deviation 19.00	—

SECONDARY outcome

Timeframe: From Day 1 up to Day 533.

Population: The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug.

The number of on-study RBC transfusions were monitored throughout the treatment period.

Outcome measures

Measure	Cohort 1 n=3 Participants Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 n=20 Participants Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Number of Subjects Receiving Red Blood Cell (RBC) Transfusions	1 Participants	7 Participants

Adverse Events

Cohort 1

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

Cohort 2

Serious events: 6 serious events

Other events: 17 other events

Deaths: 1 deaths

Serious adverse events

Measure	Cohort 1 n=3 participants at risk Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 n=20 participants at risk Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Immune system disorders Hypersensitivity	33.3% 1/3 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	0.00% 0/20 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Abdominal neoplasm	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Blood and lymphatic system disorders Aplastic anaemia	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Blood and lymphatic system disorders Intravascular haemolysis	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Renal and urinary disorders Acute kidney injury	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Injury, poisoning and procedural complications Joint dislocation	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Respiratory, thoracic and mediastinal disorders Pneumonitis	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Blood and lymphatic system disorders Haemolysis	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	10.0% 2/20 • Number of events 3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Blood and lymphatic system disorders Paroxysmal nocturnal haemoglobinuria	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Hepatobiliary disorders Cholecystitis acute	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Gastrointestinal disorders Abdominal pain	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.

Other adverse events

Measure	Cohort 1 n=3 participants at risk Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.	Cohort 2 n=20 participants at risk Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.
Infections and infestations Upper respiratory tract infection	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	25.0% 5/20 • Number of events 6 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Infections and infestations Pharyngitis	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	10.0% 2/20 • Number of events 2 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Infections and infestations Hordeolum	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 2 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Infections and infestations Nasal abscess	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Infections and infestations Nasopharyngitis	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Infections and infestations Ophthalmic herpes zoster	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Infections and infestations Paronychia	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Infections and infestations Urinary tract infection	33.3% 1/3 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	0.00% 0/20 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Respiratory, thoracic and mediastinal disorders Epistaxis	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	15.0% 3/20 • Number of events 3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Respiratory, thoracic and mediastinal disorders Cough	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Respiratory, thoracic and mediastinal disorders Nasal congestion	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Respiratory, thoracic and mediastinal disorders Pleural effusion	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Respiratory, thoracic and mediastinal disorders Productive cough	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
General disorders Injection site erythema	33.3% 1/3 • Number of events 4 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	20.0% 4/20 • Number of events 5 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
General disorders Pyrexia	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	10.0% 2/20 • Number of events 2 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
General disorders Injection site induration	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
General disorders Injection site pain	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
General disorders Injection site swelling	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
General disorders Oedema peripheral	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 2 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Gastrointestinal disorders Abdominal pain upper	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Gastrointestinal disorders Constipation	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Gastrointestinal disorders Diarrhoea	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Gastrointestinal disorders Gingival bleeding	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Gastrointestinal disorders Mouth ulceration	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Gastrointestinal disorders Vomiting	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Investigations Electrocardiogram QT prolonged	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	15.0% 3/20 • Number of events 4 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Investigations Alanine aminotransferase increased	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Investigations Blood alkaline phosphatase increased	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Investigations Blood lactate dehydrogenase increased	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Investigations Gamma-glutamyltransferase increased	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Investigations Liver function test abnormal	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Investigations Serum ferritin decreased	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Skin and subcutaneous tissue disorders Rash maculo-papular	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	10.0% 2/20 • Number of events 2 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Skin and subcutaneous tissue disorders Dry skin	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Skin and subcutaneous tissue disorders Erythema	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Skin and subcutaneous tissue disorders Petechiae	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Skin and subcutaneous tissue disorders Purpura	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Skin and subcutaneous tissue disorders Rash	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Skin and subcutaneous tissue disorders Skin hypopigmentation	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 6 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Blood and lymphatic system disorders Neutropenia	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	15.0% 3/20 • Number of events 4 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Blood and lymphatic system disorders Haemolysis	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 2 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Blood and lymphatic system disorders Thrombocytopenia	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	10.0% 2/20 • Number of events 2 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Blood and lymphatic system disorders Anaemia	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 5 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Blood and lymphatic system disorders Febrile neutropenia	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Blood and lymphatic system disorders Haemolytic anaemia	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Renal and urinary disorders Acute kidney injury	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Renal and urinary disorders Chromaturia	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	10.0% 2/20 • Number of events 4 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Renal and urinary disorders Haematuria	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	10.0% 2/20 • Number of events 2 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Renal and urinary disorders Chronic kidney disease	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Renal and urinary disorders Haemoglobinuria	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 2 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Injury, poisoning and procedural complications Contusion	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	10.0% 2/20 • Number of events 3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Injury, poisoning and procedural complications Ligament sprain	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Injury, poisoning and procedural complications Post procedural haemorrhage	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Injury, poisoning and procedural complications Road traffic accident	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Metabolism and nutrition disorders Hypokalaemia	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	20.0% 4/20 • Number of events 4 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Metabolism and nutrition disorders Hypoalbuminaemia	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Metabolism and nutrition disorders Hypocalcaemia	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Nervous system disorders Dizziness	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	10.0% 2/20 • Number of events 3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Nervous system disorders Hypoaesthesia	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Nervous system disorders Lethargy	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Eye disorders Cataract	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 2 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Eye disorders Conjunctival haemorrhage	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Eye disorders Dry eye	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Musculoskeletal and connective tissue disorders Pain in extremity	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Hepatobiliary disorders Cholangitis	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Hepatobiliary disorders Hepatic steatosis	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Vascular disorders Haematoma	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Vascular disorders Hypotension	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Cardiac disorders Bradycardia	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
Reproductive system and breast disorders Uterine haemorrhage	0.00% 0/3 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.	5.0% 1/20 • Number of events 1 • TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.

Additional Information

Apellis Clinical Trial Information Line

Apellis Pharmaceuticals, Inc

Phone: 1-833-284-6361

Email: clinicaltrials@apellis.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place