Biocryst has bagged a win with its latest oral hereditary angioedema candidate, BCX7353 – kind of. The Apex-2 trial, testing the project as a preventative therapy, met its primary endpoint. But a 44% reduction in HAE attacks with the higher 150mg dose of BCX7353, although statistically significant, fell well short of the 87% reduction seen with the market leader, Takeda’s Takhzyro, in its phase III study. This raises the question of whether any patient would want to take a chance on Biocryst’s once-daily pill, despite its convenience advantage over subcutaneous Takhzyro, particularly since HAE attacks can be fatal. And, with BCX7353 linked with diarrhoea and liver toxicity, it is not without risks either. Biocryst investors did not see any reason for optimism, sending the group’s stock down 51% this morning. EvaluatePharma sellside consensus forecasts BCX7353 sales of $287m in 2024, and according to the EvaluatePharma Vision module Biocryst has ploughed $235m into its clinical development. The company is also evaluating BCX7353 as an acute HAE therapy, with the pivotal Zenith-2 trial set to begin this summer, but after today's setback hopes here cannot be high.
|The hereditary angioedema landscape|
|Product||Company||Description||Setting||Status||2024e sales ($m)|
|Takhzyro||Takeda||SC kallikrein antibody||Prophylaxis||Marketed||1,764|
|Ruconest||Pharming||IV complement factor C1 convertase inhibitor||Acute||Marketed||509|
|KVD900||Kalvista||Oral plasma kallikrein inhibitor||Acute||Phase II||411|
|Haegarda||CSL||SC complement factor C1 convertase inhibitor||Prophylaxis||Marketed||362|
|BCX7353||Biocryst||Oral kallikrein 1 inhibitor||Prophylaxis/acute||Phase III||287|