Duration of Treatment 7.8 7.1 - median (range) mths (2.6-13.6) (1.1-13.4) % Subjects with 35% 41.9% 0.7% 41.3% [33.2%, ≥ reduction in spleen 49.2%]1 volume at Week 24 p<0.00012 Duration of reduction in 48.1 Not - spleen volume median applicable weeks3 % Subjects with 50% 45.9% 5.3% 40.6% [?, ?] ≥ reduction in symptom p<0.00015 score4 at Week 24 EORTC QLQ-C30 +12.3 -3.4 15.76 [?, ?] score mean – change at Week 24 1 Newcombe-Wilson method without continuity correction CE R1, p.68. 2 Fisher’s exact test.3 – estimate in subjects with 35% reduction in spleen volume at week 24. 4 Night sweats, itch, ≥ abdominal discomfort, pain under left ribs, feeling of fullness, muscle bone pain scored 0-10 daily from Week -1 to Week 24 using Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 developed by the sponsor and agreed with the US FDA. 5 test. 6 Difference < 10 not clinically significant. ? χ2 Sponsor requested to provide data in Pre-ACPM Response. 23: #352 Results Intent-to-Treat Population – Ruxolitinib BAT Difference n=144 n=72 [95% CI] Duration of Treatment median 11.8 10.4 - (range) months (0.5-17.3) (0.03-15.4) % Subjects with 35% 28.5% 0.0% 28.5% ≥ reduction in spleen volume at [?, ?] Week 48 p<0.00011 % Subjects with 35% 31.9% 0.0% 31.9% ≥ reduction in spleen volume at [?, ?] Week 24 p<0.00011 Duration of reduction in spleen 48 Not - volume median weeks3 assessable EORTC QLQ-C30 quality-of-life ? ? ?4 score mean change at Week 48 – [?, ?]
Duration of Treatment median (range) mths 7.8 (2.6-13.6) 7.1 (1.1-13.4) - % Subjects with ≥ 35% reduction in spleen volume at Week 24 41.9% 0.7% 41.3% [33.2%, 49.2%]1 p<0.00012 Duration of reduction in spleen volume median weeks3 48.1 Not applicable - % Subjects with ≥ 50% reduction in symptom score4 at Week 24 45.9% 5.3% 40.6% [?, ?] p<0.00015 EORTC QLQ-C30 score – mean change at Week 24 +12.3 -3.4 15.76 [?, ?] 1 Newcombe-Wilson method without continuity correction – CE R1, p.68. 2 Fisher’s exact test.3 estimate in subjects with ≥ 35% reduction in spleen volume at week 24. 4 Night sweats, itch, abdominal discomfort, pain under left ribs, feeling of fullness, muscle bone pain scored 0-10 daily from Week -1 to Week 24 using Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 developed by the sponsor and agreed with the US FDA. 5 χ2 test. 6 Difference < 10 not clinically significant. ? Sponsor requested to provide data in Pre-ACPM Response. 23: #352 Results – Intent-to-Treat Population Ruxolitinib n=144 BAT n=72 Difference [95% CI] Duration of Treatment median (range) months 11.8 (0.5-17.3) 10.4 (0.03-15.4) - % Subjects with ≥ 35% reduction in spleen volume at Week 48 28.5% 0.0% 28.5% [?, ?] p<0.00011 % Subjects with ≥ 35% reduction in spleen volume at Week 24 31.9% 0.0% 31.9% [?, ?] p<0.00011 Duration of reduction in spleen volume median weeks3 48 Not assessable - EORTC QLQ-C30 quality-of-life score – mean change at Week 48 ? ? ?4 [?, ?]
auspar-ruxolitinib-140121.docx