Efficacy assessment summary (modified intent-to-treat population; n=43)
| Parameter | VX-509 | |||
|---|---|---|---|---|
| Placebo (n=12) | 100 mg qd (n=11) | 200 mg qd (n=10) | 300 mg qd (n=10) | |
| DAS28-CRP | ||||
| Baseline, n | 12 | 11 | 10 | 10 |
| Mean (SD) | 6.3 (1.0) | 5.4 (0.7) | 5.8 (0.9) | 6.1 (0.9) |
| Week 12, n | 11 | 10 | 8 | 8 |
| Mean (SD) | 5.6 (1.9) | 3.9 (1.5) | 4.0 (1.4) | 4.1 (1.9) |
| Change, n | 11 | 10 | 8 | 8 |
| Mean (SD) | −0.8 (1.2) | −1.5 (1.3) | −2.0 (1.5) | −2.3 (1.6) |
| DAS28-ESR | ||||
| Baseline, n | 12 | 11 | 10 | 10 |
| Mean (SD) | 7.1 (0.8) | 6.0 (0.9) | 6.3 (1.1) | 6.6 (1.3) |
| Week 12, n | 11 | 10 | 8 | 9 |
| Mean (SD) | 6.4 (1.7) | 4.7 (1.7) | 4.3 (1.6) | 4.2 (2.3) |
| Change, n | 11 | 10 | 8 | 9 |
| Mean (SD) | −0.8 (1.2) | −1.2 (1.4) | −2.1 (1.3) | −2.4 (1.5) |
| RAMRIS synovitis score | ||||
| Baseline, n | 12 | 11 | 10 | 10 |
| Mean (SD) | 14.7 (6.4) | 12.9 (5.7) | 12.2 (6.4) | 13.0 (5.5) |
| Week 12, n | 8 | 10 | 8 | 7 |
| Mean (SD) | 16.3 (6.9) | 10.8 (6.6) | 8.3 (4.2) | 8.2 (3.7) |
| Change, n | 8 | 10 | 8 | 7 |
| Mean (SD) | 0.9 (1.7) | −2.5 (3.3) | −4.1 (6.0) | −5.2 (4.1) |
| RAMRIS osteitis score* | ||||
| Baseline, n | 11 | 11 | 8 | 10 |
| Mean (SD) | 9.5 (8.2) | 9.5 (8.0) | 9.5 (8.5) | 8.7 (7.3) |
| Week 12, n | 8 | 8 | 8 | 5 |
| Mean (SD) | 10.6 (9.8) | 6.9 (8.3) | 8.6 (6.2) | 4.8 (4.8) |
| Change, n | 8 | 8 | 6 | 5 |
| Mean (SD) | 0.7 (1.2) | −0.5 (3.4) | −2.8 (6.2) | −3.2 (2.8) |
| RAMRIS erosion score | ||||
| Baseline, n | 12 | 11 | 10 | 10 |
| Mean (SD) | 23.7 (29.3) | 24.3 (20.5) | 17.5 (12.1) | 29.1 (29.1) |
| Week 12, n | 8 | 10 | 8 | 7 |
| Mean (SD) | 13.3 (8.3) | 21.2 (18.5) | 19.2 (12.9) | 20.7 (21.0) |
| Change, n | 8 | 10 | 8 | 7 |
| Mean (SD) | −0.1 (0.2) | 0.1 (0.6) | 0 (0.3) | −0.1 (0.2) |
| RAMRIS joint space narrowing score | ||||
| Baseline, n | 12 | 11 | 10 | 10 |
| Mean (SD) | 30.3 (21.5) | 36.4 (20.5) | 30.6 (15.8) | 35.8 (15.8) |
| Week 12, n | 8 | 10 | 8 | 7 |
| Mean (SD) | 25.8 (14.1) | 33.7 (20.1) | 32.6 (14.6) | 30.8 (14.3) |
| Change, n | 8 | 10 | 8 | 7 |
| Mean (SD) | 0.8 (1.5) | −0.5 (1.9) | −1.6 (3.5) | −1.4 (3.4) |
*For RAMRIS osteitis score at baseline, placebo n=11, VX-509 100 mg qd n=11, VX-509 200 mg qd n=8 and VX-509 300 mg qd n=10. Because of the quality of the imaging, the MRI had to be repeated in some patients to assess the RAMRIS osteitis score. However, in some cases, the repeat MRI assessment of osteitis occurred outside the visit window, and therefore was not used in the analysis, since it may not represent the true treatment effect.
CRP, C reactive protein; DAS28-CRP, disease activity score 28 using CRP (four components); ESR, erythrocyte sedimentation rate; RAMRIS, Rheumatoid Arthritis MRI scoring system.