Table 2.
Summary of treatment-related AEs occurring in more than 10% of patients (all treated patients)*
| Once-daily molibresib dose |
||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Treatment-related AEs, No. (%) |
Grade 3 and 4 treatment-related AEs, No. (%) |
|||||||||||
| 2–16 mg | 30 mg | 60 mg | 80 mg | 100 mg | Total | 2–16 mg | 30 mg | 60 mg | 80 mg | 100 mg | Total | |
| (n = 11)† | (n = 4) | (n = 9) | (n = 32) | (n = 9) | (n = 65) | (n = 11) | (n = 4) | (n = 9) | (n = 32) | (n = 9) | (n = 65) | |
| Any event | 5 (45) | 2 (50) | 7 (78) | 31 (97) | 9 (100) | 54 (83) | — | — | 1 (11) | 23 (72) | 7 (78) | 31 (48) |
| Thrombocytopenia | — | 1 (25) | 3 (33) | 22 (69) | 7 (78) | 33 (51) | — | — | 1 (11) | 16 (50) | 7 (78) | 24 (37) |
| Nausea | 2 (18) | 2 (50) | 2 (22) | 15 (47) | 6 (67) | 27 (42) | — | — | — | 2 (6) | — | 2 (3) |
| Decreased appetite | 1 (9) | 1 (25) | 1 (11) | 11 (34) | 4 (44) | 18 (28) | — | — | — | 3 (9) | — | 3 (5) |
| Diarrhea | — | — | 1 (11) | 12 (38) | 2 (22) | 15 (23) | — | — | — | 1 (3) | — | 1 (2) |
| Vomiting | 2 (18) | — | 1 (11) | 9 (28) | 3 (33) | 15 (23) | — | — | — | — | — | — |
| Anemia | — | — | — | 12 (38) | 2 (22) | 14 (22) | — | — | — | 5 (16) | — | 5 (8) |
| Dysgeusia | — | 1 (25) | 3 (33) | 6 (19) | 4 (44) | 14 (22) | — | — | — | — | — | — |
| Fatigue | — | — | 1 (11) | 8 (25) | 4 (44) | 13 (20) | — | — | — | 1 (3) | — | 1 (2) |
| Blood bilirubin increased | — | — | — | 7 (22) | 4 (44) | 11 (17) | — | — | — | 2 (6) | 1 (11) | 3 (5) |
| Asthenia | — | — | — | 10 (31) | — | 10 (15) | — | — | — | 3 (9) | — | 3 (5) |
| AST increased | — | — | — | 5 (16) | 2 (22) | 7 (11) | — | — | — | — | 1 (11) | 1 (2) |
| Rash | — | — | 2 (22) | 4 (13) | 1 (11) | 7 (11) | — | — | — | — | — | — |
*
Including all grade 3 and 4 treatment-related AEs occurring in more than one patient. AE = adverse event; AST = aspartate aminotransferase; DLT = dose-limiting toxicities; PD = progressive disease.
†
Early PD among patients enrolled at the lowest dose levels caused more than one enrollment to achieve one DLT-evaluable patient. The 3 + 3 rule was triggered at 16 mg after a grade 2 AE of nausea was documented.