Trial Outcomes & Findings for Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy (NCT NCT04811716)

NCT ID: NCT04811716

Last Updated: 2025-04-08

Results Overview

Open Label Treatment Period (OLTP)

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 24 participants
• Primary outcome timeframe: Through Week 28
• Results posted on: 2025-04-08

Participant Flow

Twenty-four participants were screened and randomized.

Participant milestones

Measure	Pozelimab Q2W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Overall Study STARTED	12	12
Overall Study COMPLETED	10	12
Overall Study NOT COMPLETED	2	0

Reasons for withdrawal

Measure	Pozelimab Q2W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Overall Study Withdrawal by Subject	2	0

Baseline Characteristics

Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy

Baseline characteristics by cohort

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.	Total n=24 Participants Total of all reporting groups
Age, Continuous	41.4 Years STANDARD_DEVIATION 16.89 • n=93 Participants	53.2 Years STANDARD_DEVIATION 16.38 • n=4 Participants	47.3 Years STANDARD_DEVIATION 17.34 • n=27 Participants
Sex: Female, Male Female	5 Participants n=93 Participants	6 Participants n=4 Participants	11 Participants n=27 Participants
Sex: Female, Male Male	7 Participants n=93 Participants	6 Participants n=4 Participants	13 Participants n=27 Participants
Race/Ethnicity, Customized Hispanic or Latino	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	12 Participants n=93 Participants	12 Participants n=4 Participants	24 Participants n=27 Participants
Race/Ethnicity, Customized White	1 Participants n=93 Participants	1 Participants n=4 Participants	2 Participants n=27 Participants
Race/Ethnicity, Customized Black or African American	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race/Ethnicity, Customized Asian	10 Participants n=93 Participants	11 Participants n=4 Participants	21 Participants n=27 Participants
Race/Ethnicity, Customized American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race/Ethnicity, Customized Not Reported	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants

PRIMARY outcome

Timeframe: Through Week 28

Population: Safety analysis set (SAF) included all randomized participants who received any amount of study drug and was based on the treatment received (as treated); Here 'number analyzed' = the number of evaluable participants at a specified timepoint

Open Label Treatment Period (OLTP)

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) Participants with any TEAE	66.7 Percentage of participants	41.7 Percentage of participants
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) Participants with serious TEAE	16.7 Percentage of participants	0 Percentage of participants
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) Participants with severe TEAE	16.7 Percentage of participants	0 Percentage of participants

SECONDARY outcome

Timeframe: End of treatment period, approximately 28 Weeks

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint

OLTP Pre-treatment (mean of LDH values prior to combination dosing); End-of-treatment (mean of LDH value at week 24- through week 28); percentage of change in Upper Limit of Normal (xULN).

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percent Change of Lactate Dehydrogenase (LDH) From Pre-treatment to End-of-treatment Period	2.93 Percentage of change Standard Deviation 36.575	3.65 Percentage of change Standard Deviation 13.608

SECONDARY outcome

Timeframe: Baseline (Day 1) through Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' is the number of evaluable participants at a specified timepoint

OLTP Adequate control of hemolysis is defined as LDH values ≤1.5 × Upper limit of normal (ULN) from baseline (day 1) to week 28

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants Maintaining Adequate Control of Hemolysis From Baseline (Day 1) Through Week 28	75.0 Percentage of participants	91.7 Percentage of participants

SECONDARY outcome

Timeframe: Week 4 through Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint

OLTP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants Maintaining Adequate Control of Hemolysis From Week 4 Through Week 28	83.3 Percentage of Participants	91.7 Percentage of Participants

SECONDARY outcome

Timeframe: Day 1 through Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint

OLTP; Adequate control at a visit is defined as having LDH <=1.5 x ULN at that visit

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants With Adequate Control of Hemolysis at Each Visit Day 1 Through Week 28 Baseline Visit (Day 1)	100.0 Percentage of participants	100.0 Percentage of participants
Percentage of Participants With Adequate Control of Hemolysis at Each Visit Day 1 Through Week 28 Pre-treatment	100.0 Percentage of participants	100.0 Percentage of participants
Percentage of Participants With Adequate Control of Hemolysis at Each Visit Day 1 Through Week 28 Week 1	100.0 Percentage of participants	100.0 Percentage of participants
Percentage of Participants With Adequate Control of Hemolysis at Each Visit Day 1 Through Week 28 Week 2	100.0 Percentage of participants	100.0 Percentage of participants
Percentage of Participants With Adequate Control of Hemolysis at Each Visit Day 1 Through Week 28 Week 4	83.0 Percentage of participants	100.0 Percentage of participants
Percentage of Participants With Adequate Control of Hemolysis at Each Visit Day 1 Through Week 28 Week 6	92.0 Percentage of participants	100.0 Percentage of participants
Percentage of Participants With Adequate Control of Hemolysis at Each Visit Day 1 Through Week 28 Week 8	100.0 Percentage of participants	100.0 Percentage of participants
Percentage of Participants With Adequate Control of Hemolysis at Each Visit Day 1 Through Week 28 Week 10	100.0 Percentage of participants	100.0 Percentage of participants
Percentage of Participants With Adequate Control of Hemolysis at Each Visit Day 1 Through Week 28 Week 12	100.0 Percentage of participants	92.0 Percentage of participants
Percentage of Participants With Adequate Control of Hemolysis at Each Visit Day 1 Through Week 28 Week 16	100.0 Percentage of participants	100.0 Percentage of participants
Percentage of Participants With Adequate Control of Hemolysis at Each Visit Day 1 Through Week 28 Week 20	100.0 Percentage of participants	100.0 Percentage of participants
Percentage of Participants With Adequate Control of Hemolysis at Each Visit Day 1 Through Week 28 Week 24	100.0 Percentage of participants	100.0 Percentage of participants
Percentage of Participants With Adequate Control of Hemolysis at Each Visit Day 1 Through Week 28 Week 28	92.0 Percentage of participants	100.0 Percentage of participants

SECONDARY outcome

Timeframe: Baseline (Day 1) through Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint

OLTP; Normalization of LDH was defined as LDH ≤1.0 × ULN at each visit

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1) Through Week 28 Baseline (Day 1)	92.0 Percentage of participants	75.0 Percentage of participants
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1) Through Week 28 Pre-treatment	92.0 Percentage of participants	83.0 Percentage of participants
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1) Through Week 28 Week 1	100.0 Percentage of participants	92.0 Percentage of participants
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1) Through Week 28 Week 2	92.0 Percentage of participants	83.0 Percentage of participants
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1) Through Week 28 Week 4	83.0 Percentage of participants	83.0 Percentage of participants
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1) Through Week 28 Week 6	83.0 Percentage of participants	75.0 Percentage of participants
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1) Through Week 28 Week 8	100.0 Percentage of participants	82.0 Percentage of participants
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1) Through Week 28 Week 10	100.0 Percentage of participants	83.0 Percentage of participants
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1) Through Week 28 Week 12	92.0 Percentage of participants	83.0 Percentage of participants
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1) Through Week 28 Week 16	92.0 Percentage of participants	75.0 Percentage of participants
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1) Through Week 28 Week 20	100.0 Percentage of participants	92.0 Percentage of participants
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1) Through Week 28 Week 24	100.0 Percentage of participants	92.0 Percentage of participants
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1) Through Week 28 Week 28	83.0 Percentage of participants	83.0 Percentage of participants

SECONDARY outcome

Timeframe: Baseline (Day 1) through Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint

OLTP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Average LDH (U/L) Based on Area Under the Curve (AUC) From OLTP Baseline (Day 1) Through Week 28	238.18 Units per Liter (U/L) Standard Deviation 44.006	244.42 Units per Liter (U/L) Standard Deviation 39.085

SECONDARY outcome

Timeframe: Week 4 through Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint

OLTP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Average LDH (U/L) Based on Area Under the Curve (AUC) From OLTP Week 4 Through Week 28	202.49 U/L Standard Deviation 38.272	211.28 U/L Standard Deviation 34.280

SECONDARY outcome

Timeframe: Baseline (Day 1) through Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint

OLTP

Breakthrough hemolysis is defined as an increase in LDH with concomitant signs or symptoms associated with hemolysis:

• An increase in LDH occurs when:

• LDH ≥2 × ULN if pre-treatment LDH is ≤1.5 × ULN or
• LDH ≥2 × ULN after initial achievement of LDH ≤1.5 × ULN if pre-treatment LDH is >1.5 × ULN Signs or symptoms should correspond to those known to be associated with intravascular hemolysis due to Paroxysmal nocturnal hemoglobinuria (PNH) limited to the following: new onset or worsening fatigue, headache, dyspnea, hemoglobinuria, abdominal pain, scleral icterus, erectile dysfunction, chest pain, confusion, dysphagia, anemia including hemoglobin value significantly lower (ie, ≥2g/dL decrease) compared to patient's known baseline hemoglobin values, and thrombotic event.

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants With Breakthrough Hemolysis From Baseline (Day 1) Through Week 28	8.3 Percentage of participants	0.0 Percentage of participants

SECONDARY outcome

Timeframe: Baseline (Day 1) through Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint

OLTP Hemoglobin stabilization was defined as participants who did not receive an RBC transfusion and had no decrease in hemoglobin level of ≥2 grams per deciLiter (g/dL).

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants With Hemoglobin Stabilization From Baseline (Day 1) Through Week 28	75.0 Percentage of participants	91.7 Percentage of participants

SECONDARY outcome

Timeframe: Baseline (Day 1) to Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint

OLTP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=11 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Change in Hemoglobin Levels From Baseline (Day 1) Through Week 28	-1.3 grams per Liter (g/L) Standard Deviation 13.77	10.3 grams per Liter (g/L) Standard Deviation 7.41

SECONDARY outcome

Timeframe: Baseline (Day 1) to Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint

OLTP Not requiring a red blood cell (RBC) transfusion as per protocol algorithm

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants With Transfusion Avoidance From Baseline (Day 1) Through Week 28	83.3 Percentage of participants	100.0 Percentage of participants

SECONDARY outcome

Timeframe: Baseline (Day 1) to Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint

OLTP Rate of RBCs transfused is defined as number of events per person-years of treatment. For each participant, the participant-years are the time from first dose date to week 28 (or early terminations visit if subject discontinued the study early) in the OLTP.

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Rate of Red Blood Cells (RBCs) Transfused From Baseline (Day 1) to Week 28	1.284 Events per person-years of treatment Interval 0.169 to 9.76	NA Events per person-years of treatment No per-protocol transfusions occurred in the Q4W arm

SECONDARY outcome

Timeframe: Baseline (Day 1) to Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint

OLTP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Number of Per-Protocol RBC Units Transfused From Baseline (Day 1) Through Week 28	1.5 Units Standard Deviation 3.73	0.0 Units Standard Deviation 0.00

SECONDARY outcome

Timeframe: Baseline (Day 1) to Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint

OLTP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Change in Total Complement Hemolysis Activity Assay (CH50) From Baseline (Day 1) Through Week 28	0.0 Units per milliliter (U/mL) Standard Deviation 0.0	-0.1 Units per milliliter (U/mL) Standard Deviation 0.29

SECONDARY outcome

Timeframe: Baseline (Day 1) to Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint

OLTP FACIT fatigue is a 13-item scale and for each item 4 is not at all fatigued to 0 very much fatigued. Higher FACIT-Fatigue scores indicate less fatigue (scores range from 0-52). A 5-point change is considered clinically meaningful.

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=8 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=9 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Change in Fatigue as Measured by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale From Baseline (Day 1) Through Week 28	-6.0 Score on a scale Standard Deviation 10.09	-2.0 Score on a scale Standard Deviation 4.36

SECONDARY outcome

Timeframe: Baseline (Day 1) to Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint

OLTP EORTC QLQ-C30 is a 30-question tool used to assess the overall quality of life (QoL) in cancer participants. Items contributing to the GHS/QoL, were scored 1 ("very poor") to 7 ("excellent"). A linear transformation was applied to the raw scores so that transformed score lies between 0 to 100. A higher score indicates better global health status/functioning and a negative change from baseline indicated less improvement.

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=8 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=9 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Change in Global Health Status/Quality of Life Scale (GHS/QoL) on the European Organization for Research and Treatment of Cancer: Quality-of-Life Questionnaire Core 30 Items (EORTC QLQ-C30) From Baseline (Day 1) Through Week 28	-9.4 Score on a scale Standard Deviation 23.33	-1.9 Score on a scale Standard Deviation 13.03

SECONDARY outcome

Timeframe: Baseline (Day 1) to Week 28

Population: Full analysis set (FAS) = included all randomized participants who received any amount of study drug \& had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint

OLTP; EORTC QLQ-C30 is a 30-question tool used to assess the overall quality of life (QoL) in cancer participants. Items contributing to the GHS/QoL, were scored 1 ("very poor") to 7 ("excellent"). A linear transformation was applied to the raw scores so that transformed score lies between 0 to 100. A higher score indicates better global health status/functioning and a negative change from baseline indicated less improvement.

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=8 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=9 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Change in Physical Function (PF) Scores on the EORTC QLQ-C30 From Baseline (Day 1) Through Week 28	-6.7 Score on a scale Standard Deviation 19.19	0.7 Score on a scale Standard Deviation 9.09

SECONDARY outcome

Timeframe: On Week 28

Population: The pharmacokinetic (PK) analysis set includes all treated participants who received any amount of study drug (SAF) and who had at least 1 non-missing analyte measurement following the first dose of combination treatment. The PK analysis set is based on the actual treatment received.

OLTP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Concentrations of Total Pozelimab in Serum on Week 28	131 mg/L Standard Deviation 74.8	58.2 mg/L Standard Deviation 31.5

SECONDARY outcome

Timeframe: On Week 28

Population: The pharmacokinetic (PK) analysis set includes all treated participants who received any amount of study drug (SAF) and who had at least 1 non-missing analyte measurement following the first dose of combination treatment. The PK analysis set is based on the actual treatment received.

OLTP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Concentrations of Cemdisiran in Plasma on Week 28	NA mg/L Standard Deviation NA There were no quantifiable concentration at this time point	NA mg/L Standard Deviation NA There were no quantifiable concentration at this time point

SECONDARY outcome

Timeframe: On Week 28

Population: The pharmacokinetic (PK) analysis set includes all treated participants who received any amount of study drug (SAF) and who had at least 1 non-missing analyte measurement following the first dose of combination treatment. The PK analysis set is based on the actual treatment received.

OLTP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Concentrations of Total C5 on Week 28	0 mg/L Interval 0.0 to 13.8	0 mg/L Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: Up to Week 52 (OLTP [ Week 0 - Week 28] + OLEP [Week 28 - Week 52])

Population: Anti-Drug Antibodies (ADA) Analysis Set; Here 'n' = the number of evaluable participants at a certain timepoint

OLTP and OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Number of Participants With Pozelimab Anti-Drug Antibody (ADA) Responses Over Time Treatment-Boosted Response	0 Participants	0 Participants
Number of Participants With Pozelimab Anti-Drug Antibody (ADA) Responses Over Time Treatment-Emergent Response	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to Week 52 (OLTP [ Week 0 - Week 28] + OLEP [Week 28 - Week 52])

Population: Anti-Drug Antibodies (ADA) Analysis Set; Here 'n' = the number of evaluable participants at a certain timepoint

OLTP and OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Number of Participants With Cemdisiran Anti-Drug Antibody (ADA) Responses Over Time Treatment-Boosted Response	0 Participants	0 Participants
Number of Participants With Cemdisiran Anti-Drug Antibody (ADA) Responses Over Time Treatment-Emergent Response	0 Participants	1 Participants

SECONDARY outcome

Timeframe: Through Week 28

Population: Safety analysis set (SAF) included all randomized participants who received any amount of study drug and was based on the treatment received (as treated); Here 'n' = the number of evaluable participants at a specified timepoint

OLTP No participants received dose intensification during the study; Therefore, assessment of the safety of pozelimab + cemdisiran combination therapy in participants requiring dose intensification was not conducted.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 24e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint

Optional Open-Label Extension Period (OLEP)

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=22 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Change of LDH From Baseline (Day 1e) to Week 24e	-18.2 U/L Standard Deviation 108.40	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 24e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint

OLEP; Percentage of change for units per liter (U/L)

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=22 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percent Change of LDH From OLEP Baseline (Day 1e) to Week 24e	-0.7 Percentage of change Standard Deviation 20.60	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=22 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Change of LDH From Baseline (Day 1e) to Week 52e	-14.5 U/L Standard Deviation 105.90	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=22 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percent Change of LDH From Baseline (Day 1e) to Week 52e	0.2 Percentage of change Standard Deviation 19.49	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) through Week 24e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants Maintaining Adequate Control of Hemolysis From Baseline (Day 1e) Through Week 24e	95.7 Percentage of Participants	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) through Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants Maintaining Adequate Control of Hemolysis From Baseline (Day 1e) Through Week 52e	95.7 Percentage of Participants	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) through Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP Adequate control at a visit is defined as having LDH <=1.5 x ULN at that visit

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants With Adequate Control of Hemolysis at Each Visit From Baseline (Day 1e) Through Week 52e OLEP Baseline (Day 1e)	96.0 Percentage of participants	—
Percentage of Participants With Adequate Control of Hemolysis at Each Visit From Baseline (Day 1e) Through Week 52e Week 8e	100.0 Percentage of participants	—
Percentage of Participants With Adequate Control of Hemolysis at Each Visit From Baseline (Day 1e) Through Week 52e Week 16e	100.0 Percentage of participants	—
Percentage of Participants With Adequate Control of Hemolysis at Each Visit From Baseline (Day 1e) Through Week 52e Week 24e	100.0 Percentage of participants	—
Percentage of Participants With Adequate Control of Hemolysis at Each Visit From Baseline (Day 1e) Through Week 52e Week 32e	100.0 Percentage of participants	—
Percentage of Participants With Adequate Control of Hemolysis at Each Visit From Baseline (Day 1e) Through Week 52e Week 40e	100.0 Percentage of participants	—
Percentage of Participants With Adequate Control of Hemolysis at Each Visit From Baseline (Day 1e) Through Week 52e Week 52e	100.0 Percentage of participants	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) through week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1e) Through Week 52e Week 24e	82.0 Percentage of participants	—
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1e) Through Week 52e Week 32e	86.0 Percentage of participants	—
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1e) Through Week 52e OLEP Baseline (Day 1e)	87.0 Percentage of participants	—
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1e) Through Week 52e Week 8e	83.0 Percentage of participants	—
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1e) Through Week 52e Week 16e	82.0 Percentage of participants	—
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1e) Through Week 52e Week 40e	82.0 Percentage of participants	—
Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1e) Through Week 52e Week 52e	82.0 Percentage of participants	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) through Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Average LDH (U/L) Based on Area Under the Curve (AUC) From OLEP Baseline (Day 1e) Through Week 52e	154.63 U/L Standard Deviation 38.231	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) through Week 24e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants With Breakthrough Hemolysis From Baseline (Day 1e) Through Week 24e	4.3 Percentage of Participants	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) through Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants With Breakthrough Hemolysis From Baseline (Day 1e) Through Week 52e	4.3 Percentage of participants	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) through Week 24e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP Participants who did not receive RBC transfusion and had no decrease in hemoglobin levels

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants With Hemoglobin Stabilization From Baseline (Day 1e) Through Week 24e	78.3 Percentage of participants	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) through Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP Participants who did not receive RBC transfusion and had no decrease in hemoglobin levels

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants With Hemoglobin Stabilization From Baseline (Day 1e) Through Week 52e	69.6 Percentage of participants	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 24e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=21 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Change in Hemoglobin Levels From Baseline (Day 1e) to Week 24e	0.8 g/L Standard Deviation 15.63	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=22 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Change in Hemoglobin Levels From Baseline (Day 1e) to Week 52e	-0.6 g/L Standard Deviation 12.60	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) through Week 24e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP Not requiring a RBC transfusion as per protocol algorithm

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants With Per-Protocol Transfusion Avoidance From Baseline (Day 1e) Through Week 24e	87.0 Percentage of Participants	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP Not requiring a RBC transfusion as per protocol algorithm

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants With Per-Protocol Transfusion Avoidance From Baseline (Day 1e) Through Week 52e	87.0 Percentage of Participants	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 24e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Rate of RBCs Transfused From Baseline (Day 1e) to Week 24e	0.591 Events per person-years of treatment Interval 0.143 to 2.445	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Rate of RBCs Transfused From Baseline (Day 1e) to Week 52e	0.506 Events per person-years of treatment Interval 0.092 to 2.773	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 24e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Number of Units of RBCs Transfused From Baseline (Day 1e) to Week 24e	0.4 Units Standard Deviation 1.47	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Number of Units of RBCs Transfused From Baseline (Day 1e) to Week 52e	0.9 Units Standard Deviation 3.95	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 16e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Change in CH50 From Baseline (Day 1e) to Week 16e	0.0 U/mL Standard Deviation 0.00	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 24e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint; No CH50 samples were collected at Week 24e

OLEP

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=22 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Change in CH50 From Baseline (Day 1e) to Week 52e	0.0 U/mL Standard Deviation 0.21	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 16e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint; Since all participants had 0 values at baseline for CH50, the percentage change was not appropriate and undefined. Therefore, this endpoint was not able to be calculated.

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percent Change in CH50 From Baseline (Day 1e) to Week 16e	NA Percentage of change All participants had values of 0 at baseline for CH50, the percentage change was not appropriate and undefined	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 24e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint; Since all participants had 0 values at baseline for CH50, the percentage change was not appropriate and undefined. Therefore, this endpoint was not able to be calculated.

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percent Change in CH50 From Baseline (Day 1e) to Week 24e	NA Percentage of change All participants had values of 0 at baseline for CH50, the percentage change was not appropriate and undefined	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint; Since all participants had 0 values at baseline for CH50, the percentage change was not appropriate and undefined. Therefore, this endpoint was not able to be calculated.

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percent Change in CH50 From Baseline (Day 1e) to Week 52e	NA Percentage of change All participants had values of 0 at baseline for CH50, the percentage change was not appropriate and undefined	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP; The FACIT-Fatigue is a 13-item, self-administered assessment of an individual's level of fatigue during their usual daily activities over the past week. This questionnaire is part of the FACIT measurement system, a compilation of questions measuring health-related QoL in participants with cancer and other chronic illnesses. The FACIT-Fatigue items are measured with a 5-point Likert scale ranging from 0 (not at all) to 4 (very much). Scores range from 0 to 52, with higher scores indicating less fatigue. A 5-point change is considered clinically meaningful.

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=21 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Change in Fatigue as Measured by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale From Baseline (Day 1e) to Week 52e	-0.9 Score on a scale Standard Deviation 5.95	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP; EORTC QLQ-C30 is a 30-question tool used to assess the overall quality of life (QoL) in cancer participants. Items contributing to the GHS/QoL, were scored 1 ("very poor") to 7 ("excellent"). A linear transformation was applied to the raw scores so that transformed score lies between 0 to 100. A higher score indicates better global health status/functioning and a negative change from baseline indicated less improvement.

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=21 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Change in GHS/QoL on the EORTC QLQ-C30 From Baseline (Day 1e) to Week 52e	6.0 Score on a scale Standard Deviation 13.73	—

SECONDARY outcome

Timeframe: Baseline (Day 1e) to Week 52e

Population: OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint

OLEP; EORTC QLQ-C30 is a 30-question tool used to assess the overall quality of life (QoL) in cancer participants. Items contributing to the GHS/QoL, were scored 1 ("very poor") to 7 ("excellent"). A linear transformation was applied to the raw scores so that transformed score lies between 0 to 100. A higher score indicates better global health status/functioning and a negative change from baseline indicated less improvement.

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=21 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Change in PF Scores on the EORTC QLQ-C30 From Baseline (Day 1e) to Week 52e	-0.3 Score on a scale Standard Deviation 3.93	—

SECONDARY outcome

Timeframe: Up to Week 52

Population: The OLEP SAF includes all participants who participated in the OLEP who received any amount of study drug in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=23 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Percentage of Participants With TEAEs Up to Week 52 Participants with any TEAE	73.9 Percentage of participants	—
Percentage of Participants With TEAEs Up to Week 52 Participants with serious TEAE	8.7 Percentage of participants	—
Percentage of Participants With TEAEs Up to Week 52 Participants with severe TEAE	4.3 Percentage of participants	—

SECONDARY outcome

Timeframe: On Week 52

Population: The OLEP pharmacokinetic (PK) analysis set includes all participants who participated in the OLEP who received any amount of study drug in the OLEP and who had at least 1 non-missing analyte measurement following the first dose of study drug in the OLEP.

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Concentrations of Total Pozelimab in Serum on Week 52	63.4 mg/L Standard Deviation 35.6	58.6 mg/L Standard Deviation 28.7

SECONDARY outcome

Timeframe: On Week 52

Population: The OLEP pharmacokinetic (PK) analysis set includes all participants who participated in the OLEP who received any amount of study drug in the OLEP and who had at least 1 non-missing analyte measurement following the first dose of study drug in the OLEP.

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Concentrations of Total C5 on Week 52	0 mg/L Interval 0.0 to 8.05	NA mg/L There were no quantifiable concentration at this time point

SECONDARY outcome

Timeframe: On Week 52

Population: The OLEP pharmacokinetic (PK) analysis set includes all participants who participated in the OLEP who received any amount of study drug in the OLEP and who had at least 1 non-missing analyte measurement following the first dose of study drug in the OLEP.

OLEP

Outcome measures

Measure	Pozelimab Q2W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	Pozelimab Q4W + Cemdisiran n=12 Participants Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.
Concentrations of Cemdisiran in Plasma on Week 52	NA mg/L Standard Deviation NA There were no quantifiable concentration at this time point	NA mg/L Standard Deviation NA There were no quantifiable concentration at this time point

Adverse Events

OLTP_Pozelimab Q2W + Cemdisiran

Serious events: 3 serious events

Other events: 8 other events

Deaths: 0 deaths

OLTP_Pozelimab Q4W + Cemdisiran

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

OLEP_Pozelimab Q4W + Cemdisiran

Serious events: 2 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Measure	OLTP_Pozelimab Q2W + Cemdisiran n=12 participants at risk Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	OLTP_Pozelimab Q4W + Cemdisiran n=12 participants at risk Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.	OLEP_Pozelimab Q4W + Cemdisiran n=23 participants at risk In the open-label extension period (OLEP), participants received a regimen of pozelimab SC Q4W + cemdisiran SC, regardless of their treatment assignment in the OLTP.
Blood and lymphatic system disorders Haemolysis	0.00% 0/12 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	4.3% 1/23 • Number of events 1 • From first dose up to Week 52
Gastrointestinal disorders Anal fistula	0.00% 0/12 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	4.3% 1/23 • Number of events 1 • From first dose up to Week 52
Gastrointestinal disorders Large intestine polyp	0.00% 0/12 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	4.3% 1/23 • Number of events 1 • From first dose up to Week 52
Infections and infestations COVID-19	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Infections and infestations Gastroenteritis	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Infections and infestations Upper respiratory tract infection	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52

Other adverse events

Measure	OLTP_Pozelimab Q2W + Cemdisiran n=12 participants at risk Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP.	OLTP_Pozelimab Q4W + Cemdisiran n=12 participants at risk Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP.	OLEP_Pozelimab Q4W + Cemdisiran n=23 participants at risk In the open-label extension period (OLEP), participants received a regimen of pozelimab SC Q4W + cemdisiran SC, regardless of their treatment assignment in the OLTP.
General disorders Injection site reaction	16.7% 2/12 • Number of events 6 • From first dose up to Week 52	16.7% 2/12 • Number of events 4 • From first dose up to Week 52	13.0% 3/23 • Number of events 4 • From first dose up to Week 52
General disorders Chest pain	0.00% 0/12 • From first dose up to Week 52	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/12 • From first dose up to Week 52	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Musculoskeletal and connective tissue disorders Musculoskeletal chest pain	0.00% 0/12 • From first dose up to Week 52	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Musculoskeletal and connective tissue disorders Myalgia	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Musculoskeletal and connective tissue disorders Muscle spasms	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Gastrointestinal disorders Abdominal pain	0.00% 0/12 • From first dose up to Week 52	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Gastrointestinal disorders Anal fistula	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Gastrointestinal disorders Haemorrhoidal haemorrhage	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Gastrointestinal disorders Nausea	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Infections and infestations COVID-19	16.7% 2/12 • Number of events 2 • From first dose up to Week 52	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	17.4% 4/23 • Number of events 4 • From first dose up to Week 52
Infections and infestations Chlamydial infection	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Infections and infestations Subcutaneous abscess	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Infections and infestations Upper respiratory tract infection	16.7% 2/12 • Number of events 2 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	17.4% 4/23 • Number of events 5 • From first dose up to Week 52
Investigations Free haemoglobin present	0.00% 0/12 • From first dose up to Week 52	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Investigations Electrocardiogram ST segment depression	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Investigations Transaminases increased	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Blood and lymphatic system disorders Anaemia	8.3% 1/12 • Number of events 2 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Blood and lymphatic system disorders Breakthrough haemolysis	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Blood and lymphatic system disorders Haemolysis	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Ear and labyrinth disorders Inner ear disorder	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52
Renal and urinary disorders Paroxysmal nocturnal haemoglobinuria	8.3% 1/12 • Number of events 1 • From first dose up to Week 52	0.00% 0/12 • From first dose up to Week 52	0.00% 0/23 • From first dose up to Week 52

Additional Information

Clinical Trials Administrator

Regeneron Pharmaceuticals, Inc.

Phone: 844-734-6643

Email: clinicaltrials@regeneron.com

Results disclosure agreements

• Principal investigator is a sponsor employee The investigator has the right to independently publish study results from the investigator's site after a multi-center publication, or a defined period after the completion of the study by all sites. The investigator must provide the sponsor a copy of any such publication derived from the study for review and comment in advance of any submission, and delay publication, if requested, to allow the Sponsor to preserve its proprietary rights.
• Publication restrictions are in place

Restriction type: OTHER