AstraZeneca (LSE:AZN) released results from three Phase III clinical studies evaluating efzimfotase alfa, an experimental treatment for hypophosphatasia (HPP), a rare inherited disorder that affects bone development.
The MULBERRY study, which involved treatment-naive children aged 2 to 12, achieved its primary objective. Patients receiving efzimfotase alfa showed a statistically significant improvement in bone health versus placebo at week 25, measured using the Radiographic Global Impression of Change score.
Results from the CHESTNUT trial showed that pediatric patients who transitioned from the current therapy Strensiq to efzimfotase alfa were able to maintain treatment benefits. The study also indicated that the new therapy demonstrated acceptable safety and tolerability.
However, the HICKORY trial, which evaluated adolescents and adults aged 12 and above, did not meet its primary endpoint. The study failed to show a statistically significant improvement in the Six-Minute Walk Test compared with placebo. AstraZeneca said this was largely due to stronger-than-anticipated outcomes in the placebo group among adults with late-onset HPP. Despite this, the treatment delivered nominally significant improvements in fatigue across the overall study population and showed positive effects on mobility and physical functioning in predefined subgroups of patients with pediatric-onset disease.
In total, the clinical programme enrolled 196 participants across 22 countries, marking the first trials designed to include both pediatric-onset and adult-onset HPP patients.
Efzimfotase alfa is being developed as an enzyme replacement therapy intended to reduce treatment burden by requiring smaller injection volumes and less frequent dosing than the currently available therapy Strensiq.
AstraZeneca said the findings will be presented at an upcoming medical conference and will also be submitted to regulators worldwide. The therapy was developed by Alexion, AstraZeneca’s rare disease division.
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