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.
AstraZeneca PLC’s rare disease unit Alexion has announced positive results with ALXN1840 in the Phase III FoCus study in Wilson disease, and the compound is expected to be submitted for marketing approval in coming months.
The promising news follows the disappointment of a week ago, when the Alexion unit reported poor results with another late-stage rare disease candidate, Ultomiris (ravulizumab), in a different condition, amyotrophic lateral sclerosis (ALS)
Private Company Edition: Deerfield’s third innovations fund will back therapeutics and other opportunities, the Gates Foundation – a sometimes funder of biotech firms – will spend $200bn over the next 20 years, and NewLimit raised a $130m series B round, among other financings.
Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.
An ‘historic economic prosperity deal’ does not include the sector.
