Area under the concentration-time curve limited to the end of the dosing interval. The samples included in the calculation of AUC0-tau were collected at the following times: on dosing Days 1, 3 and 5, PK samples were taken up to 30 minutes pre-dose and at 15 minutes (± 5 min), 30 minutes (± 5 min), 1 hour (± 10 min), 4 hours (± 10 min), 8 hours (± 30 min), and 24 hours (± 30 min) post-dose as well as on Day 8 pre-dose.

On Day 1 and 5 the AUC0-tau parameters did not meet lambda-z acceptance criteria for 2 patients, so they were not included. On Day 8 the AUC0-tau parameter did not meet lambda-z acceptance criteria for 1 patient, so they were not included.

Maximum observed serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5.

On Day 1, Cmax could not be estimated for 2 patients due to a) no sample provided; and b) Cmax considered unreliable due to pegcetacoplan positive pre-dose sample. On Day 3 and Day 5, Cmax could not be estimated for 1 patient due to a limited number of samples provided.

Time of maximum measured serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5.

On Day 1, Tmax could not be estimated for 2 patients due to a) no sample provided; and b) Tmax considered unreliable due to pegcetacoplan positive pre-dose sample. On Day 3 and Day 5, Tmax could not be estimated for one patient due to a limited number of samples provided.

Observed serum concentration pre-dose. From Day 8 (Week 1) and onwards, PK samples were taken pre-dose at each visit.

The number of patients in the analysis population for Ctrough decreased over the course of the study, from 11 at Week 1 to 7 at Week 14, reflecting the limited number of samples available due to study discontinuation of patients.

Absolute levels and change from baseline to Week 24 in biomarker of complement activation sC5b-9

The pharmacodynamic (PD) set includes patients in the intent-to-treat (ITT) set who receive investigational medicinal product (IMP) and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. There were also missing samples.

Occurrence and severity of treatment-emergent adverse events.

Change from baseline to Week 24 in platelets

The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.

Change from baseline to Week 24 in hemoglobin

The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.

Change from baseline to Week 24 in LDH

The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.

Absolute levels and change from baseline to Week 24 in biomarker of complement activation C3a

The PD set includes all patients in the ITT set who receive IMP and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. In some cases, there were also missing samples.

Absolute levels and change from baseline to Week 24 in biomarker of complement activation C3

The PD set includes all patients in the ITT set who receive IMP and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. In some cases, there were also missing samples.

Absolute levels and change from baseline to Week 24 in biomarker of complement activation Bb

The PD set includes all patients in the ITT set who receive IMP and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. In some cases, there were also missing samples.

Absolute levels and change from baseline to Week 24 in biomarker of complement activation C4a

The PD set includes all patients in the ITT set who receive IMP and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. In some cases, there were also missing samples.

Absolute levels and change from baseline to Week 24 in biomarker of complement activation CH50

The PD set includes all patients in the ITT set who receive IMP and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. In some cases, there were also missing samples.

Absolute levels and change from baseline to Week 24 in biomarker of complement activation AH50

The PD set includes all patients in the ITT set who receive IMP and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. In some cases, there were also missing samples.

A participant will be declared as reaching a clinical response upon improvement in laboratory markers of TMA and resolution of TMA clinical symptoms

A participant will be declared as reaching TMA response upon improvement in laboratory markers of TMA

Survival

Survival

Both clinical response sustained at week 24 and clinical response at any time during the study will be assessed.

Analysis population based on patients who achieved clinical response at any point during the study.

Both TMA response sustained at week 24 and TMA response at any time during the study will be assessed.

Analysis population based on patients who achieved TMA response at any point during the study.

Duration of clinical response sustained at week 24

Analysis population based on patients who achieved clinical response at any point during the study.

Duration of TMA response sustained at week 24

Analysis population based on patients who achieved TMA response at any point during the study.

A participant will be declared as relapsing upon appearance of laboratory markers of TMA

Only 2 patients had available assessment at the visit.

Number of participants reaching clinical response at week 12

Number of participants reaching TMA response at week 12

Change from baseline to Week 24 in haptoglobin

The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.

Change from baseline to Week 24 in indirect bilirubin

The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.

Change from baseline to Week 24 in serum creatinine

The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.

Change from baseline to Week 24 in urine protein/creatinine ratio

The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.

Change from baseline to Week 24 in systolic blood pressure. Post-dose is 30 minutes after stop of infusion. The timepoints from Week 16 onward have a single value as no infusions were administered at these study visits.

The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.

Change from baseline to Week 24 in diastolic blood pressure. Post-dose is 30 minutes after stop of infusion. The timepoints from Week 16 onward have a single value as no infusions were administered at these study visits.

The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.

Change from baseline to Week 24 in respiratory rate. Post-dose is 30 minutes after stop of infusion. The timepoints from Week 16 onward have a single value as no infusions were administered at these study visits.

The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.

Change from baseline to Week 24 in heart rate. Post-dose is 30 minutes after stop of infusion. The timepoints from Week 16 onward have a single value as no infusions were administered at these study visits.

The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.

Change from baseline to Week 24 in temperature. Post-dose is 30 minutes after stop of infusion. The timepoints from Week 16 onward have a single value as no infusions were administered at these study visits.

The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.

Occurrence of clinically significant abnormal electrocardiogram findings.

The analysis population is based on the number of patients with assessment at the visit.

Presence of antibodies to pegcetacoplan throughout treatment and follow-up periods.

Presence of antibodies to PEG throughout treatment and follow-up periods. Baseline represents the situation at treatment start.

The number of patients in the analysis population decreased over the course of the study, reflecting the limited number of samples available due to study discontinuation of patients. In some cases, there were also missing samples.

Presence of treatment-emergent anti-PEG antibodies throughout treatment and follow-up periods. Conservatively, the anti-PEG antibodies detected in a patient with a missing baseline sample have been considered as treatment-emergent.