Aerosolized Alpha-1 antitrypsin: Phase II/III final data

Final data from a double-blind, European and Canadian Phase II/III trial in 168 patients with AAT deficiency showed that twice-daily 160 mg inhaled AAT for 50 weeks missed the secondary endpoints of reducing the time to first event-based exacerbation with a severity of mild, moderate or severe (p=not significant) and of a lower rate of first severe exacerbation events (18.8% vs. 31.1%, p=not significant) vs. placebo. Additionally, inhaled AAT non-significantly reduced the rate of the first moderate exacerbation event vs. placebo (56.5% vs. 63.9%, p=not significant). In a subgroup of the most frequent exacerbators, inhaled AAT non-significantly reduced the rate of first severe exacerbation events (19.4% vs. 35.2%, p=not significant) and the rate of severe and moderate exacerbation events (44.8% vs. 51.9%, p=not significant) vs. placebo. ...