At the time of analysis, the median follow-up was 10 months (minimum of 6 months of follow-up for all ongoing patients). Baseline demographic characteristics are described in Table 8.

Table 8: Demographic and Disease Characteristics Patient Characteristics at Entry Efficacy Population
N=444
Age
  Median, years (range) 59 (18 to 94)
Gender, n (%)
  Male 236 (53%)
Race, n (%)
  Asian 57 (13%)
  Black or African American 25 (6%)
  White 349 (79%)
  Other 13 (3%)
ECOG Performance Status, n (%)
  ECOG=0 or 1 409 (92%)
Disease History
  Median time from diagnosis to first dose, years (range) 6.1 (0.3 to 28.5)
  Resistant to Prior TKI Therapy, n (%) 374 (88%)
  Presence of one or more BCR-ABL kinase domain mutations 244 (55%)
  Prior TKI therapy– number of prior approved TKIs, n (%) 
      1 29 (7%)
      2 166 (37%)
    ≥3 249 (56%)

At the time of analysis, the median duration of Iclusig treatment was 281 days in patients with CP-CML, 286 days in patients with AP-CML, 89 days in patients with BP-CML, and 81 days in patients with Ph+ALL. Efficacy results are summarized in Table 9, and Table 10.

Table 9: Efficacy of Iclusig in Patients With Resistant or Intolerant Chronic Phase CML  Overall
(N=267) Cohort
R/I
Cohort
(N=203) T315I
Cohort
(N=64)
Cytogenetic Response   
*
Primary endpoint for CP-CML Cohorts was MCyR, which combines both complete (no detectable Ph+ cells) and partial (1% to 35% Ph+ cells in at least 20 metaphases) cytogenetic responses.
  Major * (MCyR)   
  % 54% 49% 70%
  (95% CI) (48,60) (42,56) (58,81)
    Complete (CCyR)   
    % 44% 37% 66%
    (95% CI) (38,50) (31,44) (53,77)
Primary endpoint for CP-CML Cohorts was MCyR, which combines both complete (no detectable Ph+ cells) and partial (1% to 35% Ph+ cells in at least 20 metaphases) cytogenetic responses.

In patients with CP-CML patients who achieved MCyR, the median time to MCyR was 84 days (range: 49 to 334 days). At the time of analysis, the median durations of MCyR had not yet been reached.

Table 10: Efficacy of Iclusig in Patients With Resistant or Intolerant Advanced Disease (includes R/I and T315I cohorts)  AP-CML
Overall
(N=83) BP-CML
Overall
(N=62) Ph+ ALL
Overall
(N=32)
Hematologic Response   
*
Primary endpoint for patients with AP-CML, BP-CML, and Ph+ALL was MaHR, which combines complete hematologic responses and no evidence of leukemia.
†
CHR: WBC ≤ institutional ULN, ANC ≥1000/mm3, platelets ≥100,000/mm3, no blasts or promyelocytes in peripheral blood, bone marrow blasts ≤5%, <5% myelocytes plus metamyelocytes in peripheral blood, basophils <5% in peripheral blood, No extramedullary involvement (including no hepatomegaly or splenomegaly).
  Major* (MaHR)   
  % 52% 31% 41%
  (95% CI) (41,63) (20,44) (24,59)