Abstracts of 11th C1-inhibitor Deficiency & Angioedema Workshop

Table 2 Efficacy endpoints (difference, active vs placebo) for each dose level in ZENITH-1. Phase 2 trial of oral BCX7353

Endpoint^a	250 mg	500 mg	750 mg
Number of attacks, active/placebo	21/11	25/11	64/31
Change from baseline in VAS score at 4 h, mm^a	0.57	− 2.1	− 6.98***
Improved or stable VAS at 4 h, %^b	6.4	18.5	21.0*
Improved or stable VAS at 24 h, %^b	14.5	23.6	27.0*
Improved or stable symptoms at 4 h, %^b	12.6	12.4	22.3*
Improved or stable symptoms at 24 h, %^b	11.6	27.6	28.6**
Standard of care rescue treatment at 24 h, %^b	− 7.4	− 13.5	− 31.6***
No or mild symptoms at 24 hr^b	16.4	23.6	31.8***

^aLS mean difference to placebo reported. P-value generated from a mixed effect linear model including treatment, period, and sequence as fixed effects, subject within sequence as a random effect, and predose 3-symptom composite VAS score as a covariate
^bDifference to placebo in proportion of attacks achieving the endpoint reported. P-value generated from a generalised logistic model including treatment, period, and sequence as fixed effects, and subject within sequence as a random effect. Use of standard of care HAE medication = failure for endpoint
*** p < 0.005; ** p < 0.01; * p < 0.05 for active vs placebo

ISSN: 1710-1492