Correlation of DS events with clinical response
| Population . | AG221-C-001 monotherapy . | AG221-AML-004 monotherapy . | AG221-AML-005 AZA combo∗ . | AG120-221-C-001 7 + 3 combo† . | All studies pooled . | |||
|---|---|---|---|---|---|---|---|---|
| ND AML n = 39 n (%) . | R/R AML n = 280 n (%) . | R/R AML n = 157 n (%) . | ND AML n = 74 n (%) . | ND AML n = 93 n (%) . | R/R AML n = 437 n (%) . | ND AML n = 206 n (%) . | Total AML N = 643 n (%) . | |
| Patients with ≥1 DS event | 5 (12.8) | 33 (11.8) | 14 (8.9) | 13 (17.6) | 2 (2.2) | 47 (10.8) | 20 (9.7) | 67 (10.4) |
| Achieved ORR‡,§ | 0 | 16 (48.5) | 8 (57.1) | 9 (69.2) | 2 (100.0) | 24 (51.1) | 11 (55.0) | 35 (52.2) |
| Before onset of first DS event‡ | 0 | 3 (9.1) | 0 | 1 (7.7) | 0 | 3 (6.4) | 1 (5.0) | 4 (6.0) |
| After onset of first DS event‡ | 0 | 15 (45.5) | 8 (57.1) | 8 (61.5) | 2 (100.0) | 23 (48.9) | 10 (50.0) | 33 (49.3) |
| Achieved CR/CRi‡ | 0 | 8 (24.2) | 2 (14.3) | 6 (46.2) | 1 (50.0) | 10 (21.3) | 7 (35.0) | 17 (25.4) |
| Before onset of first DS event‡ | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| After onset of first DS event‡ | 0 | 8 (24.2) | 2 (14.3) | 6 (46.2) | 1 (50.0) | 10 (21.3) | 7 (35.0) | 17 (25.4) |
| Achieved CR/CRp‡ | 0 | 11 (33.3) | 2 (14.3) | 7 (53.8) | 2 (100.0) | 13 (27.7) | 9 (45.0) | 22 (32.8) |
| Before onset of first DS event‡ | 0 | 1 (3.0) | 0 | 0 | 0 | 1 (2.1) | 0 | 1 (1.5) |
| After onset of first DS event‡ | 0 | 10 (30.3) | 2 (14.3) | 6 (46.2) | 2 (100.0) | 12 (25.5) | 8 (40.0) | 20 (29.9) |
| Achieved CR/CRh‡ | NA | 9 (27.3) | 3 (21.4) | 7 (53.8) | 2 (100.0) | 12 (25.5) | NA | NA |
| Before onset of first DS event‡ | NA | 0 | 0 | 0 | 0 | 0 | NA | NA |
| After onset of first DS event‡ | NA | 9 (27.3) | 3 (21.4) | 7 (53.8) | 2 (100.0) | 12 (25.5) | NA | NA |
| Patients with no DS event | 34 (87.2) | 247 (88.2) | 143 (91.1) | 61 (82.4) | 91 (97.8) | 390 (89.2) | 186 (90.3) | 576 (89.6) |
| Achieved ORR§,‖ | 12 (35.3) | 95 (38.5) | 56 (39.2) | 45 (73.8) | 77 (84.6) | 151 (38.7) | 134 (72.0) | 285 (49.5) |
| Achieved CR/CRi‖ | 7 (20.6) | 49 (19.8) | 35 (24.5) | 38 (62.3) | 58 (63.7) | 84 (21.5) | 103 (55.4) | 187 (32.5) |
| Achieved CR/CRp‖ | 8 (23.5) | 61 (24.7) | 35 (24.5) | 36 (59.0) | 57 (62.6) | 96 (24.6) | 101 (54.3) | 197 (34.2) |
| Achieved CR/CRh‖ | NA | 54 (21.9) | 37 (25.9) | 36 (59.0) | 57 (62.6) | 91 (23.3) | NA | NA |
| Population . | AG221-C-001 monotherapy . | AG221-AML-004 monotherapy . | AG221-AML-005 AZA combo∗ . | AG120-221-C-001 7 + 3 combo† . | All studies pooled . | |||
|---|---|---|---|---|---|---|---|---|
| ND AML n = 39 n (%) . | R/R AML n = 280 n (%) . | R/R AML n = 157 n (%) . | ND AML n = 74 n (%) . | ND AML n = 93 n (%) . | R/R AML n = 437 n (%) . | ND AML n = 206 n (%) . | Total AML N = 643 n (%) . | |
| Patients with ≥1 DS event | 5 (12.8) | 33 (11.8) | 14 (8.9) | 13 (17.6) | 2 (2.2) | 47 (10.8) | 20 (9.7) | 67 (10.4) |
| Achieved ORR‡,§ | 0 | 16 (48.5) | 8 (57.1) | 9 (69.2) | 2 (100.0) | 24 (51.1) | 11 (55.0) | 35 (52.2) |
| Before onset of first DS event‡ | 0 | 3 (9.1) | 0 | 1 (7.7) | 0 | 3 (6.4) | 1 (5.0) | 4 (6.0) |
| After onset of first DS event‡ | 0 | 15 (45.5) | 8 (57.1) | 8 (61.5) | 2 (100.0) | 23 (48.9) | 10 (50.0) | 33 (49.3) |
| Achieved CR/CRi‡ | 0 | 8 (24.2) | 2 (14.3) | 6 (46.2) | 1 (50.0) | 10 (21.3) | 7 (35.0) | 17 (25.4) |
| Before onset of first DS event‡ | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| After onset of first DS event‡ | 0 | 8 (24.2) | 2 (14.3) | 6 (46.2) | 1 (50.0) | 10 (21.3) | 7 (35.0) | 17 (25.4) |
| Achieved CR/CRp‡ | 0 | 11 (33.3) | 2 (14.3) | 7 (53.8) | 2 (100.0) | 13 (27.7) | 9 (45.0) | 22 (32.8) |
| Before onset of first DS event‡ | 0 | 1 (3.0) | 0 | 0 | 0 | 1 (2.1) | 0 | 1 (1.5) |
| After onset of first DS event‡ | 0 | 10 (30.3) | 2 (14.3) | 6 (46.2) | 2 (100.0) | 12 (25.5) | 8 (40.0) | 20 (29.9) |
| Achieved CR/CRh‡ | NA | 9 (27.3) | 3 (21.4) | 7 (53.8) | 2 (100.0) | 12 (25.5) | NA | NA |
| Before onset of first DS event‡ | NA | 0 | 0 | 0 | 0 | 0 | NA | NA |
| After onset of first DS event‡ | NA | 9 (27.3) | 3 (21.4) | 7 (53.8) | 2 (100.0) | 12 (25.5) | NA | NA |
| Patients with no DS event | 34 (87.2) | 247 (88.2) | 143 (91.1) | 61 (82.4) | 91 (97.8) | 390 (89.2) | 186 (90.3) | 576 (89.6) |
| Achieved ORR§,‖ | 12 (35.3) | 95 (38.5) | 56 (39.2) | 45 (73.8) | 77 (84.6) | 151 (38.7) | 134 (72.0) | 285 (49.5) |
| Achieved CR/CRi‖ | 7 (20.6) | 49 (19.8) | 35 (24.5) | 38 (62.3) | 58 (63.7) | 84 (21.5) | 103 (55.4) | 187 (32.5) |
| Achieved CR/CRp‖ | 8 (23.5) | 61 (24.7) | 35 (24.5) | 36 (59.0) | 57 (62.6) | 96 (24.6) | 101 (54.3) | 197 (34.2) |
| Achieved CR/CRh‖ | NA | 54 (21.9) | 37 (25.9) | 36 (59.0) | 57 (62.6) | 91 (23.3) | NA | NA |
Data from ND or R/R AML populations, and AML safety population.
AML, acute myeloid leukemia; CRi, morphologic CR with incomplete neutrophil recovery; CRp, morphologic CR with incomplete platelet recovery; d, day(s); DS, differentiation syndrome; MLFS, morphologic leukemia-free state; N/A, not available; ND, newly diagnosed; ORR, overall response ratio; PR, partial remission; R/R, relapsed or refractory.
AML, acute myeloid leukemia; d, day(s); DS, differentiation syndrome; ND, newly diagnosed; R/R, relapsed or refractory; ORR, overall response ratio.
Patients were treated with enasidenib (100 mg or 200 mg per day) in combination with azacitidine 75 mg/m2 per day.
Patients were treated with enasidenib (100 mg once daily) in combination with cytarabine (200 mg/m2 per day for 7 days) and either daunorubicin (60 mg/m2 per day for 3 days) or idarubicin (12 mg/m2 per day for 3 days).
Percentage was based on number of patients in the safety AML population with >1 DS event of any grade.
ORR: CR + CRi + CRp + PR + MLFS.
Percentage was based on number of patients in the safety AML population without a DS event.