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

Phase III CHAMPION-ALS Study Discontinued

20 Aug 2021

• By John Davis

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.

AstraZeneca PLC’s hopes of extending the indications for Ultomiris (ravulizumab) to amyotrophic lateral sclerosis (ALS) have been set back after a failed Phase III trial of the complement C5 inhibitor, which the company hoped would bolster its rare disease ambitions following the $39bn acquisition of the drug’s originator, Alexion.

After a pre-specified interim analysis of data from the CHAMPION-ALS study, the independent data monitoring committee recommended its discontinuation because...