Table 9: Observed Value and Percentage Change from Baseline to Week 52 in Key Endpoints in Adult Patients with ASMD Type B, A/B on XENPOZYME or Placebo (Trial 1)
	Placebo	XENPOZYME	Difference [95% CI]
Nominal p value:
* p value = 0.0003; † p value <0.0001; ‡ p value= 0.0280
DLco
n	18	13
Mean % predicted DLco at baseline (SD)	48.5 (10.8)	49.1 (9.7)	NA
n	17	12
Mean % predicted DLco at Week 52 (SD)	49.9 (11.1)	59.4 (9.6)	NA
n	17	12
LS Mean Percent change in % predicted DLco at Week 52 (SE)	3.0 (3.3)	23.9 (3.8)	20.9 (5.0)* [10.6, 31.2]
Spleen volume
n	18	13
Mean Spleen Volume (MN) at baseline (SD)	11.2 (3.8)	11.5 (4.7)	NA
n	17	13
Mean Spleen Volume (MN) at Week 52 (SD)	11.2 (4.2)	7.2 (3.9)	NA
n	17	13
LS Mean Percent change in Spleen Volume (in MN) at Week 52 (SE)	0.5 (2.62)	-38.9 (3.0)	-39.4 (4.0)† [-47.6, -31.2]
Liver volume
n	18	13
Mean Liver Volume (MN) at baseline (SD)	1.6 (0.5)	1.4 (0.3)	NA
n	17	12
Mean Liver Volume (MN) at Week 52 (SD)	1.6 (0.5)	1.0 (0.2)	NA
n	17	12
LS Mean Percent change in Liver Volume from baseline to Week 52 (SE)	-1.8 (2.7)	-26.5 (3.2)	-24.7 (4.2)† [-33.4, -16.1]
Platelet count
n	18	13
Mean Platelet Count (109/L) at baseline (SD)	115.6 (36.3)	109.3 (30.6)	NA
n	16	13
Mean Platelet Count (109/L) at Week 52 (SD)	120.2 (43.2)	126.4 (29.0)	NA
n	16	13
LS Mean Percent change in Platelet Count from baseline to Week 52 (SE)	2.7 (4.5)	18.3 (5.0)	+15.6 (6.7) ‡ [1.8, 29.4]

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