AstraZeneca ends ALS trial on committee recommendation

The FTSE 100 drugmaker said the decision was based on the recommendation of the independent data monitoring committee, following its review of data from a prespecified interim analysis.

It said the committee recommended that the trial be discontinued due to lack of efficacy.

No new safety findings were observed, AstraZeneca said, and the data was consistent with the established safety profile of Ultomiris.

AstraZeneca described ALS as a “rare and fatal” neurodegenerative disease that affects motor neurons - a type of nerve cell that controls voluntary movements - in the brain and spinal cord.

“We are disappointed by this outcome and what it means for patients with this devastating disease,” said Gianluca Pirozzi, senior-vice president and head of development and safety at Alexion.

“We would like to thank the entire ALS community as well as investigators and healthcare professionals who dedicated their time and expertise to this trial.

“We continue to be confident in the potential of targeting C5 for complement-driven diseases and remain fully committed to our efforts to serve the rare disease community.”

Patients who enrolled in the trial would discontinue study medication and complete any necessary follow-up evaluations, the company said, with data from the trial to be provided to inform ongoing research.

At 0845 BST, shares in AstraZeneca were down 1.05% at 8.44p.