The latest data from the Angioedema Prophylaxis 1 (APeX-1) trial suggest the investigational treatment BCX7353 substantially reduces the frequency of attacks in hereditary angioedema.
The drug is administered orally once daily, offering benefits over the parenterally-administered treatments currently available or in development, while avoiding the toxicities associated with attenuated androgens.
The international, placebo-controlled trial evaluated four doses of BCX7353 (62.5 mg, 125 mg, 250 mg, and 350 mg once daily) over a 28-day period in patients with type I or II hereditary angioedema with a history of at least two angioedema attacks per month.
Among the 72 participants who completed the trial, the rate of attacks with placebo was 0.95 per week. BCX7353 reduced the rate by 45.5 per cent at 350 mg (P=.006); 44.6 per cent at 250 mg, (P=0.01); 73.8 per cent at 125 mg (P<.001 and per cent at mg>
Gastrointestinal adverse events, predominantly of grade 1, were the most commonly reported adverse events, particularly in the two highest BCX7353 dose groups.
The findings are published in the New England Journal of Medicine.