AZ's Rare Disease Unit Reports Positive Phase III Results For Copper-Binding ALXN1840
Effective In Wilson Disease
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.
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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.
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.
Some analysts are suggesting that AstraZeneca is getting hold of a "scarce and high-quality asset" in Alexion for a very reasonable price and shareholders could demand more than the $175 per share offer on the table.