BioCryst to discuss trial results in call
BioCryst Pharmaceuticals (NASDAQ:BCRX) is up 4% premarket on average volume in response to its announcement of encouraging results in a Phase 2 clinical trial, APeX-1, assessing its next-generation killikrein inhibitor, BCX7353, as a preventative treatment to reduce the frequency of attacks in patients with hereditary angioedema (HAE).
An interim analysis was performed on 24 HAE subjects who completed 28 days of treatment (BCX7353 = 11; placebo = 13). The baseline attack rate was ~1/week and average C1 inhibitor levels were less than 20% of the normal mean (severe angioedema).
The treatment group showed a 63% reduction in attack rate (mean rate = 0.34/week) compared to 0.92/week for placebo (p=0.006). In the intent-to-treat population of 28 subjects, the mean attack rates were 0.44/week and 0.91/week, respectively (p=0.035).
Daily dosing with BCX7353 inhibited kallikrein throughout the 24-hour dosing interval with the degree of inhibition similar to that observed in healthy subjects in the Phase 1 study (same dose).
Treatment-emergent adverse events included common cold (n=3/14), diarrhea (n=4/12), flatulence (n=2/14) and fatigue (n=2/14).
The steady-state drug levels observed far exceeded the proposed therapeutic target range so APeX-1 has been amended to include a 62.5 mg once-daily regimen. The number of subjects in the 125 mg and 250 mg (both once-daily) has been increased to more fully characterize the dose response.
Management will host a conference call this morning at 9:00 ET to discuss the data.