Efficacy end points at week 12 and week 24 (MMRM analysis)
| Change from baseline . | Danicopan-danicopan . | Placebo-danicopan∗ . | Treatment difference (danicopan-danicopan and placebo-danicopan) . | |||
|---|---|---|---|---|---|---|
| Week 12 . | Week 24 . | Week 12 . | Week 24 . | Week 12 . | P value . | |
| Hb levels, g/dL,† LSM (SEM) | 2.8 (0.2) | 2.9 (0.3) | 0.5 (0.3) | 2.3 (0.3) | 2.3 (0.4) | <.0001 |
| LDH, U/L,‡ LSM (SEM) | –25.6 (7.9) | –23.6 (11.3) | –16.9 (11.4) | 1.0 (12.4) | –8.7 (13.8) | .5306 |
| ARC, ×109/L,§ LSM (SEM) | –92.5 (8.2) | –87.9 (7.8) | –0.8 (11.8) | –53.6 (11.7) | –91.7 (14.3) | <.0001 |
| Total bilirubin||, μmol/L, LSM (SEM) | –11.6 (1.5) | –11.0 (2.2) | –1.4 (2.2) | –6.3 (2.9) | –10.1 (2.6) | .0002 |
| FACIT-Fatigue scores,¶ LSM (SEM) | 8.1 (0.9) | 6.2 (1.0) | 2.4 (1.3) | 5.6 (1.9) | 5.8 (1.6) | .0004 |
| Change from baseline . | Danicopan-danicopan . | Placebo-danicopan∗ . | Treatment difference (danicopan-danicopan and placebo-danicopan) . | |||
|---|---|---|---|---|---|---|
| Week 12 . | Week 24 . | Week 12 . | Week 24 . | Week 12 . | P value . | |
| Hb levels, g/dL,† LSM (SEM) | 2.8 (0.2) | 2.9 (0.3) | 0.5 (0.3) | 2.3 (0.3) | 2.3 (0.4) | <.0001 |
| LDH, U/L,‡ LSM (SEM) | –25.6 (7.9) | –23.6 (11.3) | –16.9 (11.4) | 1.0 (12.4) | –8.7 (13.8) | .5306 |
| ARC, ×109/L,§ LSM (SEM) | –92.5 (8.2) | –87.9 (7.8) | –0.8 (11.8) | –53.6 (11.7) | –91.7 (14.3) | <.0001 |
| Total bilirubin||, μmol/L, LSM (SEM) | –11.6 (1.5) | –11.0 (2.2) | –1.4 (2.2) | –6.3 (2.9) | –10.1 (2.6) | .0002 |
| FACIT-Fatigue scores,¶ LSM (SEM) | 8.1 (0.9) | 6.2 (1.0) | 2.4 (1.3) | 5.6 (1.9) | 5.8 (1.6) | .0004 |
All measures were significantly improved with danicopan compared with placebo at week 12. Randomization was stratified by transfusion history (>2 or ≤2 transfusions within 6 months of screening), screening Hb (<8.5 g/dL or ≥8.5 g/dL), baseline Hb, and study visit Hb.
MMRM, mixed model for repeated measures.
After week 12, participants receiving placebo were switched to danicopan treatment.
Week 12: danicopan, n = 57; placebo, n = 28 and week 24: danicopan, n = 50; placebo, n = 26.
Week 12: danicopan, n = 56; placebo, n = 28 and week 24: danicopan, n = 54; placebo, n = 26.
Week 12: danicopan, n = 57; placebo, n = 26 and week 24: danicopan, n = 50; placebo, n = 26.
Week 12: danicopan, n = 57; placebo, n = 29 and week 24: danicopan, n = 55; placebo, n = 27.
Week 12: danicopan, n = 56; placebo, n = 28 and week 24: danicopan, n = 52; placebo, n = 27.