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Enzyme Replacement

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AstraZeneca cites high placebo response for failed bone disease trial
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AstraZeneca cites high placebo response for failed bone disease trial

AstraZeneca reported that its Phase 3 trial for an enzyme replacement therapy targeting hypophosphatasia, a rare genetic bone disease, failed to meet its primary endpoints. The company attributed the failure to a high placebo response, suggesting that the study design rather than the drug's efficacy was the primary issue. This setback follows recent Phase 3 misses for other AstraZeneca therapies, including its ATTR-CM silencer.