Table 2.

Summary of change in biomarkers from baseline to week 2 at 8 am in Study 201 in participants not taking concomitant dexamethasone therapy

	Poor Disease Control				Good Disease Control
	Cohort A			Cohort C	Cohort A	Cohort B
	200 mg QD n = 6	600 mg QD n = 6	1000 mg QD n = 6	100 mg BID n = 5	200 mg QD n = 1	200 mg BID n = 5
ACTH
Baseline, GM (CV%) [n]	501.3 (105.3) [4]	473.1 (88.3) [5]	473.1 (88.3) [5]	333.2 (95.5) [5]	112.7 (NA) [1]	23.89 (278.2) [5]
Week 2, GM (CV%)	266.2 (43.8)	192.2 (62.6)	444.4 (113.8)	238.6 (298.8)	187.4 (NA)	22.98 (232.4)
% change (95% CI)	−46.9 (−85.2, 91.1)	−59.4 (−83.9, 2.3)	−6.1 (−29.9, 25.8)	−28.4 (−76.0, 114.1)	66.3 (NA)	−3.8 (−36.7, 46.3)
17-OHP
Baseline, GM (CV%) [n]	6629.5 (154.4) [5]	8910.5 (96.9) [5]	6778.5 (128.8) [6]	6582.2 (113.5) [5]	11249.0 (NA) [1]	2137.1 (46.3) [4]
Week 2, GM (CV%)	5545.2 (211.8)	8483.9 (188.8)	6800.1 (248.7)	4061.3 (188.2)	9786.0 (NA)	1266.2 (81.8)
% change (95% CI)	−16.4 (−42.2, 21.1)	−4.8 (−50.2, 81.9)	0.3 (−44.1, 80.1)	−38.3 (−77.4, 68.5)	−13.0 (NA)	−40.8 (−83.5, 112.9)
A4
Baseline, GM (CV%) [n]	405.6 (234.9) [5]	425.4 (188.2) [6]	425.4 (188.2) [6]	248.3 (167.2) [5]	533.0 (NA) [1]	69.5 (296.9) [5]
Week 2, GM (CV%)	332.2 (184.8)	322.7 (296.6)	332.5 (378.0)	193.6 (180.8)	499.0 (NA)	76.5 (216.6)
% change (95% CI)	−18.1 (−42.1, 15.9)	−24.2 (−50.5, 16.2)	−21.8 (−56.0, 38.9)	−22.0 (−53.5, 31.0)	−6.4 (NA)	10.0 (−59.2, 196.3)

No participant in Cohort B qualified as having poor disease control. Participants with poor disease control had baseline adrenocorticotropic hormone (ACTH) and androstenedione (A4) levels that were more than twice the upper limit of normal. Participants with good disease control had baseline ACTH and A4 that were less than twice the upper limit of normal.

Abbreviations: 17-OHP, 17-hydroxyprogesterone; BID, twice daily; CV, coefficient of variation; GM, geometric mean; QD, once daily.