Clinical outcomes after first injection.
| Dose Escalation | Phase 2 Expansion | |||||||
|---|---|---|---|---|---|---|---|---|
| Placebo, N = 15 | 1 × 1011, N = 16 | 1 × 1012, N = 15 | 1 × 1013, N = 15 | Placebo, N = 16 | 1 × 1011, N = 17 | 1 × 1012, N = 17 | 1 × 1013, N = 16 | |
| AAV2-neutralizing titers prior to 1st injection, geometric mean | 1/15 | 1/41 | 1/20 | 1/58 | 1/40 | 1/29 | 1/74 | 1/23 |
| Change in target joint assessments 12 weeks after 1st injection | ||||||||
| 2-point or greater decrease in target joint swelling, n (%) | 0 | 2 (13) | 2 (13) | 4 (27) | 3 (19) | 3 (18) | 2 (12) | 3 (19) |
| 2-point or greater decrease in target joint tenderness, n (%) | 4 (27) | 3 (19) | 2 (13) | 4 (27) | 4 (25) | 2 (12) | 2 (12) | 4 (25) |
| Change in target joint patient-reported outcomes 12 weeks after 1st injection | ||||||||
| 30% or greater improvement in target joint global VAS, n (%) | NT | NT | NT | NT | 3 (19) | 9 (53) | 6 (35) | 6 (38) |
| 30% or greater improvement in target joint function VAS, n (%) | NT | NT | NT | NT | 3 (19) | 4 (24) | 6 (35) | 6 (38) |
| 2-point or greater decrease in target joint pain, n (%) | NT | NT | NT | NT | 1 (6) | 1 (6) | 2 (12) | 3 (19) |
| Change in systemic efficacy measures 12 weeks after 1st injection | ||||||||
| Swollen joints (28 count), mean ± SD | 1.4 ± 5.2 | 0.6 ± 3.5 | –1.6 ± 4.6 | –0.8 ± 1.3 | –2.2 ± 3.3 | –3.1 ± 3.8 | –2.7 ± 5.3 | –3.0 ± 4.5 |
| Tender joints (28 count), mean ± SD | –2.3 ± 5.8 | 0.0 ± 4.0 | –0.5 ± 6.8 | –0.2 ± 2.7 | –1.5 ± 7.1 | –2.5 ± 5.3 | –2.9 ± 5.0 | –2.6 ± 6.0 |
| Patient global assessment (cm), mean + SD | –0.5 ± 3.6 | –1.1 ± 1.8 | –0.7 ± 1.9 | –2.9 ± 1.1 | –0.3 ± 1.6 | –1.3 ± 1.5 | –2.5 ± 2.0 | –0.9 ± 3.4 |
| Patient assessment of pain (cm), mean ± SD | –0.0 ± 3.8 | 0.5 ± 1.7 | –0.6 ± 2.7 | –2.7 ± 1.3 | 0.1 ± 1.9 | –0.8 ± 1.4 | –2.4 ± 2.3 | –1.2 ± 2.6 |
| Health assessment questionnaire, mean ± SD | –0.1 ± 0.4 | 0.0 ± 0.2 | –0.3 ± 0.3 | –0.2 ± 0.4 | –0.0 ± 0.2 | –0.2 ± 0.3 | –0.2 ± 0.4 | –0.1 ± 0.3 |
| Physician global assessment (cm), mean ± SD | –1.4 ± 1.4 | –1.0 ± 1.4 | –0.9 ± 2.1 | –2.4 ± 0.8 | –0.7 ± 2.1 | –0.8 ± 1.2 | –2.6 ± 1.9 | –0.5 ± 1.4 |
| Erythrocyte sedimentation rate (mm/h), mean ± SD | –14 ± 17 | 2 ± 7 | –2 ± 5 | –8 ± 13 | –7 ± 9 | 9 ± 19 | –1 ± 8 | –3 ± 38 |
| Disease Activity Score, mean ± SD | –0.7 ± 0.9 | –0.2 ± 1.2 | –0.4 ± 1.0 | –0.7 ± 0.8 | –0.5 ± 0.8 | –0.4 ± 0.9 | –0.7 ± 1.2 | –0.4 ± 1.1 |
* Target joint qualified for 2nd injection if swelling the same or worse than prior to 1st injection. NT: not tested.