Table 1.

Baseline characteristics

CharacteristicDanicopan-danicopan (n = 57)Placebo-danicopan (n = 29)
Age, mean (min-max), y 52.8 (20-82) 52.9 (29-77) 
Sex, n (%)   
Female 34 (59.6) 20 (69.0) 
Male 23 (40.4) 9 (31.0) 
Hb, mean (SD), g/dL 7.7 (0.9) 7.9 (1.0) 
ARC, mean (SD), ×109/L 247.6 (97.2) 222.7 (115.4) 
LDH, mean (SD), U/L 304.0 (123.6) 286.4 (93.1) 
FACIT-Fatigue scores, mean (SD) 34.0 (11.3) 31.7 (11.0) 
Participants requiring transfusion instances 6 months before screening, n (%) 56 (98.2) 28 (96.6) 
Number of transfusion instances 6 months before screening, median (range) 2.0 (0-8) 3.0 (0-8) 
C5 inhibitor, n (%)   
Ravulizumab 36 (63.2) 15 (51.7) 
Eculizumab 21 (36.8) 14 (48.3) 
Duration of C5 inhibitor therapy, mean (SD), y 5.1 (3.6) 6.1 (4.2) 
Duration of disease, mean (SD), y 10.0 (9.7) 11.0 (9.5) 
Proportion of type III PNH RBC population§, mean (SD), % 57.6 (26.1) 53.7 (28.2) 
C3 deposition on type III PNH RBCs§, mean (SD), % 33.4 (18.2) 30.4 (16.0) 
Medical history of aplastic anemia||, n (%) 18 (31.6) 8 (27.6) 
Medical history of myelodysplastic syndrome||, n (%) 2 (3.5) 3 (10.3) 
CharacteristicDanicopan-danicopan (n = 57)Placebo-danicopan (n = 29)
Age, mean (min-max), y 52.8 (20-82) 52.9 (29-77) 
Sex, n (%)   
Female 34 (59.6) 20 (69.0) 
Male 23 (40.4) 9 (31.0) 
Hb, mean (SD), g/dL 7.7 (0.9) 7.9 (1.0) 
ARC, mean (SD), ×109/L 247.6 (97.2) 222.7 (115.4) 
LDH, mean (SD), U/L 304.0 (123.6) 286.4 (93.1) 
FACIT-Fatigue scores, mean (SD) 34.0 (11.3) 31.7 (11.0) 
Participants requiring transfusion instances 6 months before screening, n (%) 56 (98.2) 28 (96.6) 
Number of transfusion instances 6 months before screening, median (range) 2.0 (0-8) 3.0 (0-8) 
C5 inhibitor, n (%)   
Ravulizumab 36 (63.2) 15 (51.7) 
Eculizumab 21 (36.8) 14 (48.3) 
Duration of C5 inhibitor therapy, mean (SD), y 5.1 (3.6) 6.1 (4.2) 
Duration of disease, mean (SD), y 10.0 (9.7) 11.0 (9.5) 
Proportion of type III PNH RBC population§, mean (SD), % 57.6 (26.1) 53.7 (28.2) 
C3 deposition on type III PNH RBCs§, mean (SD), % 33.4 (18.2) 30.4 (16.0) 
Medical history of aplastic anemia||, n (%) 18 (31.6) 8 (27.6) 
Medical history of myelodysplastic syndrome||, n (%) 2 (3.5) 3 (10.3) 

Max, maximum; min, minimum.

LDH reference range, 135 to 330 U/L.

Duration from initial C5 inhibitor therapy to first dose of study drug.

Duration from diagnosis to informed consent.

§

CD55/CD59.

||

Collected as part of participant medical history before enrollment.

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