AstraZeneca PLC on Sunday said results from its phase three trial of Efzimfotase alfa showed improvement but failed to achieve statistical significance.
The Cambridge, England-based pharmaceuticals firm said the drug demonstrated a numerical improvement in its primary endpoint of the six-minute walk test in adolescents over the age of 12 and adults.
The trial was carried out in patients with hypophosphatasia who had not been treated with Strensiq at week 25, compared to placebo. Hypophosphatasia is a genetic metabolic condition that leads to weak bones, fatigue and muscle weakness.
The six-minute walk test measures the total distance covered by a patient walking on a flat surface over a six-minute period.
AstraZeneca said the test achieved a mean difference of 17.05 metres from the baseline in the overall patient population compared to those who received the placebo.
However, it said the lack of statistical significance in the results was due to better-than-expected performance from the placebo group.
Kathryn Dahir, lead investigator in the "Hickory" phase three trial, said: "For adolescents and adults, the chronic and progressive disease burden of HPP can have far-reaching impact on physical function and emotional wellbeing, potentially impairing people's ability to live independently, form connections and sustain employment.
"These results from the Hickory phase III trial signal benefits across multiple clinically relevant endpoints and represent an important step toward potentially bringing this advanced enzyme replacement therapy to a broad range of people who may benefit."
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