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.

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.

Top-line results from a Phase IIb study of Theravance’s potential gut-selective JAK inhibitor izencitinib have not matched expectations, and results from a similar early-stage study in Crohn’s disease with the Janssen-partnered candidate are now awaited at the end of the year or in early 2022.

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.

Boehringer Ingelheim and Eli Lilly have gained a US approval for their SGLT2 inhibitor empagliflozin in heart failure with reduced ejection fraction, which could be extended further in coming months to other types of heart failure, adding to the products competing in this previously neglected therapeutic area.

GlaxoSmithKline’s checkpoint inhibitor Jemperli has gained a second indication in the US, for the treatment of mismatch repair-deficient solid tumors, affirming the company’s drive to develop a new portfolio of cancer therapies.