Biopharmaceutical company AstraZeneca plc (STO:AZN) (LON:AZN) (NYSE:AZN) on Tuesday reported positive results from its global Phase III clinical programme evaluating efzimfotase alfa for hypophosphatasia, a rare metabolic disease.

The programme included three trials across 196 patients in 22 countries, covering paediatric, adolescent and adult populations.

In the MULBERRY trial, efzimfotase alfa achieved statistically significant and clinically meaningful improvements in bone health in treatment-naive children, meeting its primary endpoint. The CHESTNUT trial demonstrated safety, tolerability and maintenance of therapeutic benefit in paediatric patients transitioning from prior treatment.

The HICKORY trial in adolescents and adults showed numerical improvement but did not meet the primary endpoint, although clinically meaningful benefits were observed in predefined subgroups and secondary measures. Long-term extension data indicated continued improvements across key endpoints.

Efzimfotase alfa was generally well tolerated across all studies.

Data from the trials will be presented at a forthcoming medical meeting and submitted to regulatory authorities.