Table 2.

Efficacy of olipudase alfa in patients with acid sphingomyelinase deficiency types B or A/B

Treatment (no. of pts)	LSM percent change from baseline to week 52 [baseline values]
	% predicted DLCOa	Spleen volumea	Liver volume	Platelet count
In randomized, double-blind, phase 2/3 ASCEND trial in adult pts [24]
Olipudase alfa (18)	+ 21.97 [49.4 %]	− 39.45 [11.7 MN]	− 28.06 [1.4 MN]	+ 16.82 [107.2 × 109/L]
Placebo (18)	+ 2.96 [48.5%]	+ 0.48 [11.2 MN]	− 1.47 [1.6 MN]	+ 2.49 [115.6 × 109/L]
Difference	+ 19.0**	− 39.9***	− 26.6***	+ 14.3*
In single-arm, open-label, phase 1/2 ASCEND-Peds trial in paediatric pts [25]
Overall (20)	+ 33†† [54.8%]	− 49.2††† [19 MN]	− 40.6††† [2.7 MN]	+ 34.03††† [137.4 × 109/L]
Adolescent (4)	+ 28b	− 46.9†††	− 41††b	+ 45.01 [98.93 × 109/L]
Child (9)	+ 35††b	− 46.0†††	− 37†††b	+ 30.67† [148.82 × 109/L]
Infant/early child (7)	NA	− 54.6†††	− 45†††b	+ 31.76† [145.66 × 109/L]

DL_CO lung diffusing capacity for carbon monoxide, LSM least squares mean, MN multiples of normal, NA not applicable, pts patients, SRS splenomegaly-related score

*p ≤ 0.05, **p ≤ 0.01, ***p ≤ 0.001 vs placebo; ^†p ≤ 0.05, ^††p ≤ 0.01, ^†††p ≤ 0.001 vs baseline

^aPrimary endpoints. In the USA, the spleen endpoint was spleen volume combined with SRS (see main text for SRS description); there was no significant difference between olipudase alfa and placebo group for SRS at week 52

^bData extracted from graph