Table 2.
Summary Table of Efficacy Outcomes
| Outcome | Cohort 1 (n = 40) |
Cohort 2 (n = 45) |
||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| HR Positive (n = 21) |
HR Negative (n = 19) |
Total (n = 40) |
HR Positive (n = 26) |
HR Negative (n = 19) |
Total (n = 45) |
|||||||
| No. (%) | 95% CI | No. (%) | 95% CI | No. (%) | 95% CI | No. (%) | 95% CI | No. (%) | 95% CI | No. (%) | 95% CI | |
| Response (RECIST v1.0) | ||||||||||||
| Confirmed CR | 2 (9.5) | 1 (5.3) | 3 (7.5) | 0 (0) | 0 (0) | 0 (0) | ||||||
| Confirmed PR | 6 (28.6) | 11 (57.8) | 17 (42.5) | 6 (23.1) | 4 (21.1) | 10 (22.2) | ||||||
| Unconfirmed PR | 1 (4.8) | 1 (5.3) | 2 (5.0) | 4 (15.4) | 0 (0) | 4 (8.9) | ||||||
| SD ≥ 24 weeks | 3 (14.3) | 0 (0) | 3 (7.5) | 5 (19.2) | 3 (15.8) | 8 (17.8) | ||||||
| SD < 24 weeks | 2 (9.5) | 2 (10.5) | 4 (10.0) | 5 (19.2) | 8 (42.1) | 13 (28.8) | ||||||
| PD | 6 (28.6) | 4 (21.1) | 10 (25.0) | 6 (23.1) | 4 (21.1) | 10 (22.2) | ||||||
| PD in CNS by RECIST | 1* (4.8) | 0 (0) | 1 (2.5) | 1 (3.8) | 0 (0) | 1 (2.2) | ||||||
| Clinical PD | 0 (0) | 1 (5.3) | 1 (2.5) | 1 (3.8) | 1 (5.3) | 2 (4.4) | ||||||
| Symptomatic deterioration | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | ||||||
| Not evaluated | 1 (4.8) | 0 (0) | 1 (2.5) | 0 (0) | 0 (0) | 0 (0) | ||||||
| Confirmed ORR† (CR + PR) | 8 (38.1) | 18.1 to 61.6 | 12 (63.2) | 38.4 to 83.7 | 20 (50.0) | 33.8 to 66.2 | 6 (23.1) | 9.0 to 43.6 | 4 (21.1) | 6.1 to 45.6 | 10 (22.2) | 11.3 to 37.3 |
| CBR (CR + PR + SD ≥ 24 weeks) | 11 (52.4) | 29.8 to 74.3 | 12 (63.2) | 38.4 to 83.7 | 23 (57.5) | 40.9 to 73.0 | 11 (42.3) | 23.4 to 63.1 | 7 (36.8) | 16.3 to 61.6 | 18 (40.0) | 25.7 to 55.7 |
| Time to event outcomes | ||||||||||||
| Median PFS, months | 6.5 | 3.7 to 16.6 | 8.8 | 3.9 to inf | 7.4 | 3.9 to 9.3 | 5.4 | 3.6 to 7.6 | 5.1 | 3.7 to inf | 5.3 | 3.7 to 6.7 |
| Median OS, months | 40.0 | 33.3 to inf | Not reached | — | Not reached | — | 26.1 | 23.8 to 41.4 | 36.0 | 30.7 to inf | 31.6 | 25.9 to 39.8 |
NOTE. Data were analyzed separately for cohorts 1 and 2 and by hormone receptor (HR) status. Patients who achieved both an unconfirmed partial response (PR) and stable disease (SD) ≥ 24 weeks are included in the group with SD ≥ 24 weeks (n = 2).
Abbreviations: CBR, clinical benefit rate; CR, complete response; inf, infinity; ORR, objective response rate; OS, overall survival; PD, progressive disease; PFS, progression-free survival.
*
One patient with both CNS and non-CNS PD.
†
CIs for ORR are based on the two-stage design.