AstraZeneca's investigational enzyme replacement therapy, efzimfotase alfa, did not meet its primary endpoint in the Phase III HICKORY trial. The trial evaluated the therapy in adults and adolescents with hypophosphatasia (HPP), a rare metabolic bone disease. The study's primary goal was a statistically significant improvement in the six-minute walk test (6MWT) compared to placebo, which the therapy failed to achieve.
AstraZeneca attributed the outcome to a better-than-expected performance in the placebo group. Despite missing the primary endpoint, efzimfotase alfa demonstrated a numerical improvement in walking distance. It also showed nominally significant improvements in a key secondary endpoint measuring fatigue.
The US FDA has granted the therapy a Priority Review, based on the overall clinical program, which includes other successful trials in pediatric patients. A decision is anticipated in the first half of 2027.