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AstraZeneca's Ultomiris Disappoints In ALS, Setting Back Rare Disease Ambitions

Phase III CHAMPION-ALS Study Discontinued

Executive Summary

AstraZeneca is discontinuing a Phase III trial of the long-acting complement inhibitor it gained via its Alexion acquisition in amyotrophic lateral sclerosis because of lack of efficacy in an interim analysis. Interest is now turning to antisense and cell therapy candidates from other companies that are in late-stage development for the condition.

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