AstraZeneca's Ultomiris Disappoints In ALS, Setting Back Rare Disease Ambitions
Phase III CHAMPION-ALS Study Discontinued
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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AstraZeneca’s recently-acquired Alexion unit is paying $150m upfront to buy the remainder of Caelum Biosciences and its late-stage therapy for light chain amyloidosis, a rare disease in which misfolded amyloid proteins build up in organs throughout the body, including the heart and kidneys.
Alexion/AstraZeneca has announced positive top-line results from the Phase III study of ALXN1840 in patients with the rare genetic disorder, Wilson disease, including in patients already on standard-of-care therapies. The compound is ahead of two candidate gene therapies from other companies at early-stage clinical development.
The company's first-in-class FcRn antagonist, which has an FDA action date in December, is now being reviewed by the EMA and although the regulator rejected a request for an accelerated assessment of efgartigimod for generalized myasthenia gravis, commercial teams are being built up in Europe.