AstraZeneca reported that efzimfotase alfa demonstrated improvements in bone health at week 25 in treatment-naive patients with hypophosphatasia enrolled in the Phase III MULBERRY trial. The study evaluated the investigational therapy in children with the rare metabolic bone disorder who had not previously received treatment with asfotase alfa.
According to the company, MULBERRY met its primary endpoint, showing statistically significant and clinically meaningful improvement in bone health as assessed by the radiographic global impression of change score compared with placebo at week 25. Secondary endpoints, including measures of rickets severity, physical function and motor proficiency, also supported the benefit of efzimfotase alfa in the pediatric population studied.
The MULBERRY trial is part of a broader Phase III program of efzimfotase alfa in hypophosphatasia that includes additional placebo-controlled and active-controlled studies across children, adolescents and adults. AstraZeneca said the overall program was designed to assess efficacy and safety across different age groups and disease presentations of hypophosphatasia.
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