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August 20, 2021

AstraZeneca scraps Alexion drug trial for ALS after data disappoints

By Natalie Missakian

Drug giant AstraZeneca is pulling the plug on a late-stage clinical trial of Alexion’s drug Ultomiris in patients diagnosed with the deadly neuromuscular disease amyotrophic lateral sclerosis, or ALS.

An independent monitoring committee recommended stopping the trial after an interim review found Ultomiris lacked efficacy against the disease, the UK-based drug giant said in an update Friday.

The setback comes a month after New Haven-born Alexion officially became AstraZeneca’s Boston-based rare disease unit following a $39 billion buyout.

“We are disappointed by the outcome and what it means for patients with this devastating disease,” said Dr. Gianluca Pirozzi, Alexion’s head of development and safety. “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.”

Commonly known as Lou Gehrig’s disease, ALS is marked by the degeneration of nerve cells in the brain and spinal cord that control muscles in the body. While there are drugs on the market to slow the disease’s progression, there is no cure. People with the disease eventually lose the ability to speak, eat, move and breathe.

Alexion’s Phase 3 trial had enrolled 382 adults with ALS at approximately 90 trial sites in North America, Europe and Asia. The “lack of efficacy” finding was based on a commonly used scale of ALS functioning, the company said.

Trial volunteers are being told to discontinue the study medication and complete any necessary follow-up evaluations, AstraZeneca said. Data from the trial will be used to inform ongoing research, according to the drug maker.

Ultomiris is already approved to treat the rare blood disorders PNH (paroxysmal nocturnal hemoglobinuria) and aHUS (atypical hemolytic uremic syndrome) in the U.S., European Union and Japan.

Alexion employs roughly 500 people at its 100 College St. research facility in downtown New Haven.

Contact Natalie Missakian at news@newhavenbiz.com

 

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