The proportion of subjects who achieved a major molecular response was generally low (apart from those in Cohort B, where MMR rate was 50.0%). This may reflect the advanced nature of the disease as well as the short duration of follow-up. Table 23: Study 201 Efficacy results for secondary endpoints (response rates) – Duration of response Data on duration of response (for the primary endpoints) are summarised in Table 24. Table 24: Study 201 Duration of response – Cohort A Cohort B Cohort C Cohort D Cohort E Cohort F CML-CP CML-CP CML-AP CML-AP (R/I) (T315I) (R/I) (T315I) R/I T315I N 203 64 65 18 48 46 Type of MCyR MCyR MaHR MaHR MaHR MaHR response (primary endpoint) No. of 99 (48.8) 45 (70.3) 39 (60.0) 9 (50.0) 17 (35.4) 15 (32.6) subjects with response (%) No. of 5 (5.1) 1 (2.2) 17 (43.6) 5 (55.6) 7 (41.2) 10 (66.7) subjects with loss of response (%) No. of 94 (94.9) 44 (97.8) 22 (56.4) 4 (44.4) 10 (58.8) 5 (33.3) subjects censored (%) Median NR NR 289.0 174.0 (42.0 NR 126.0 (70.0 – (95% (211.0 N/A) 143.0) – – CI) 538.0) Range in 1 - 423 1 - 338 98 - 538 42 - 430 54 - 429 3- - 268 days Probability 91.6% 96.7% 73.3% 41.7% 67.7% 17.4 of remaining in response at 6 months Probability 91.6% - 49.0% 41.7% 51.6% N/A of remaining
The proportion of subjects who achieved a major molecular response was generally low (apart from those in Cohort B, where MMR rate was 50.0%). This may reflect the advanced nature of the disease as well as the short duration of follow-up. Table 23: Study 201 – Efficacy results for secondary endpoints (response rates) Duration of response Data on duration of response (for the primary endpoints) are summarised in Table 24. Table 24: Study 201 – Duration of response Cohort A Cohort B Cohort C Cohort D Cohort E Cohort F CML-CP (R/I) CML-CP (T315I) CML-AP (R/I) CML-AP (T315I) R/I T315I N 203 64 65 18 48 46 Type of response (primary endpoint) MCyR MCyR MaHR MaHR MaHR MaHR No. of subjects with response (%) 99 (48.8) 45 (70.3) 39 (60.0) 9 (50.0) 17 (35.4) 15 (32.6) No. of subjects with loss of response (%) 5 (5.1) 1 (2.2) 17 (43.6) 5 (55.6) 7 (41.2) 10 (66.7) No. of subjects censored (%) 94 (94.9) 44 (97.8) 22 (56.4) 4 (44.4) 10 (58.8) 5 (33.3) Median (95% CI) NR NR 289.0 (211.0 – 538.0) 174.0 (42.0 – N/A) NR 126.0 (70.0 – 143.0) Range in days 1 - 423 1 - 338 98 - 538 42 - 430 54 - 429 3- - 268 Probability of remaining in response at 6 months 91.6% 96.7% 73.3% 41.7% 67.7% 17.4 Probability of remaining 91.6% - 49.0% 41.7% 51.6% N/A