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AstraZeneca PLC on Tuesday said phase-three testing of its replacement efzimfotase alfa enzyme on patients affected by hypophosphatasia yielded positive safety and efficacy results. Shares in AstraZeneca were up 0.5% to 14,770.00 pence each on Tuesday morning in London. Hypophosphatasia is a rare inherited bone disease which prevents normal bone mineralisation, leading to soft bones. In severe cases, it can be fatal in infants due to difficulty breathing from restricted thoracic capacity. Enzyme replacement therapy is the only existing treatment for the disease, caused by gene mutations resulting in deficient activity of the alkaline phosphatase enzyme. Efzimfotase alfa, or ALXN1850, was designed to offer lower injection volume and less frequent dosing compared to current treatments, closing care gaps. The clinical programme, comprising of two randomised and placebo-controlled trials in addition to a randomised, open-label, and active-controlled paediatric switch trial, has shown that the replacement enzyme was well-tolerated and had an acceptable safety profile, AstraZeneca said. The Cambridge, England-based pharmaceutical firm added that the results from the tests, which enrolled 196 patients across 22 countries, appeared to demonstrate that those who switched to the investigational enzyme from the placebo after the randomised period ‘showed clinically meaningful improvements across multiple efficacy outcomes after 24 weeks of treatment’. The company plans to share phase-three data with the medical community and global regulatory authorities. Copyright 2026 Alliance News Ltd. All Rights Reserved.
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