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

From: Abstracts of 11th C1-inhibitor Deficiency & Angioedema Workshop

Endpointa 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, mma 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 hrb 16.4 23.6 31.8***
  1. 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
  2. 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
  3. *** p < 0.005; ** p < 0.01; * p < 0.05 for active vs placebo