AstraZeneca Drug Candidate Achieves Mixed Results in Trials for Rare Disease

By Adria Calatayud

AstraZeneca said an experimental drug for hypophosphatasia, a rare metabolic disease, achieved mixed results in three late-stage clinical trials.

The U.K. drugmaker said Tuesday that the drug, efzimfotase alfa, didn't deliver a statistically significant outcome in the primary objective of one of the studies, which evaluated the effect of the medicine on adolescents and adults with hypophosphatasia that hadn't been treated before.

Another trial in children met the primary goal by showing an improvement in bone health, AstraZeneca said. Meanwhile, the third study demonstrated the safety and tolerability of efzimfotase alfa in patients who had been previously treated and the therapeutic benefit was maintained, it added.

The drug was well tolerated across the three studies, the company said.

"Collectively, these results support the potential for efzimfotase alfa to transform the treatment paradigm for people living with this rare disease," the chief executive of AstraZeneca's rare-disease unit Alexion, Marc Dunoyer, said.

Write to Adria Calatayud at [e-mail]

(END) Dow Jones Newswires

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