Astrazeneca’s Efzimfotase alfa highlighted positive results from Phase III clinical programme in hypophosphatasia

Astrazeneca announce that the efzimfotase alfa Phase III clinical programme, designed to study a broad hypophosphatasia (HPP) patient population, demonstrated positive results.

Addressing unmet needs for people living with HPP
Efzimfotase alfa is an investigational enzyme replacement therapy designed to offer lower injection volume, less frequent dosing over Strensiq and close critical gaps in care within the broader HPP patient population.

Paediatric clinical trials
The MULBERRY Phase III trial in children (2 to <12 years of age) with HPP who have not been previously treated with Strensiq,showed that efzimfotase alfa met its primary endpoint. Results demonstrated a statistically significant and clinically meaningful improvement in bone health from baseline compared to placebo, as measured by Radiographic Global Impression of Change Score at week 25.

In addition, the combination of prespecified subgroups of adolescents and adults with paediatric-onset HPP, efzimfotase alfa showed nominally statistically significant and clinically meaningful benefits in mobility, as measured by 6MWT, as well as key secondary endpoints measuring physical function and pain reduction, compared to placebo.

Eric Rush, MD, Clinical Geneticist, Children’s Mercy Hospital Kansas, Professor of Paediatrics, University of Missouri-Kansas City School of Medicine and lead principal investigator in the MULBERRY trial, said: “The results from the global MULBERRY clinical trial demonstrate efzimfotase alfa’s potential to address the underlying pathophysiology of HPP and to prevent and reverse the substantial skeletal and functional impacts of this lifelong rare disease. I am encouraged by these results and the potential for this innovative, investigational therapy to redefine care in HPP with a convenient self-administered option taken every two weeks.”

 

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Related Topics and Keywords

AstraZeneca, Efzimfotase alfa, hypophosphatasia