AstraZeneca says efzimfotase alfa may meet targets after mixed trial results

AstraZeneca reported mixed late-stage trial results for efzimfotase alfa: two infant studies were positive while an adult/adolescent study did not meet its primary endpoint. The company said the drug was well tolerated, plans to submit data to regulators, and expects broader market availability than its predecessor with reported peak sales of $3–5 billion.

AstraZeneca reported mixed results from three late-stage clinical trials of efzimfotase alfa, an experimental treatment for hypophosphatasia. Two trials in infants showed positive outcomes, while a separate study in adults and adolescents did not achieve its primary objective. The company said the medicine was well tolerated and announced plans to submit the trial data to regulators as soon as possible. Key facts: - Three late-stage trials were completed: two infant studies with positive results, and one adult/adolescent study that missed its primary endpoint. - AstraZeneca reported the drug was well tolerated across the three studies. - The company said it will submit the trial data to regulators promptly. - AstraZeneca's rare-disease unit said efzimfotase alfa could be launched in more markets than Strensiq and reported potential annual peak sales of $3 billion to $5 billion. Summary: The mixed results leave efzimfotase alfa with confirmed positive findings in infant studies and a non-significant outcome in an adult/adolescent trial. AstraZeneca plans regulatory submissions and reports an aim to expand market availability compared with its predecessor, with reported peak sales of $3–5 billion.