TABLE 2.
Summary of Key Efficacy End Points in Final Analysis on the Basis of RECIST Assessments
| Efficacy End Point | All Efficacy-Evaluable Patients (n = 31) | Patients With Alterations in TSC1 (n = 5)a | Patients With Alterations in TSC2 (n = 9)a |
|---|---|---|---|
| Overall response rate, No. (%) | 12 (38.7) | — | — |
| 95% CI | 21.8 to 57.8 | — | — |
| Best overall response (confirmed), No. (%) | |||
| Complete response | 2 (6.5) | 0 | 2 (22.2) |
| Partial response | 10 (32.3) | 1 (20.0) | 6 (66.7) |
| Stable disease | 16 (51.6) | 3 (60.0) | 1 (11.1) |
| Progressive disease | 3 (9.7) | 1 (20.0) | 0 |
| Disease control rate,b No. (%) | 22 (71.0) | — | — |
| 95% CI | 52.0 to 85.8 | — | — |
| Median DOR, months | 39.7 | 5.6 | 51.7 |
| 95% CI | 6.5 to NR | NE | 6.5 to NR |
| Median PFS, months | 10.6 | 5.5 | 53.1 |
| 95% CI | 5.5 to 41.2 | 1.4 to NR | 10.6 to NR |
| Median OS, monthsc | 53.1 | 31.6 | NR |
| 95% CI | 22.2 to NR | 3.8 to NR | 53.1 to NR |
NOTE. Percentages are rounded and are based on the number of patients with positive, negative, or unevaluable biomarker status at screening.
Abbreviations: DOR, duration of response; NE, not evaluable; NR, not reached; OS, overall survival; PFS, progression-free survival.
a
Of the 25 patients with tissue sufficient for next-generation sequencing, 14 patients had inactivating alterations in TSC1 or TSC2.
b
Percentage of patients with a confirmed objective response or with stable disease of ≥12-week duration.
c
The median time from first dose to last contact during OS follow-up was 22.0 months (range, 1-66).