AstraZeneca’s hopes of adding amyotrophic lateral sclerosis (ALS) to the list of approved indications for the newly acquired blockbuster drug have fallen through.The pharmaceutical giant announced on Friday that it is stop Key research on ALS treatment after reviewing the data early shows that experimental treatments are unlikely to work.
The decision to discontinue the Phase 3 study of the drug Ultomiris was based on the findings of the independent data monitoring committee. No new safety issues were observed in clinical trials, but the committee recommended that the study be stopped due to lack of efficacy.
As part of the $39 billion acquisition of Alexion Pharmaceuticals, AstraZeneca added Ultomiris to its drug pipeline. Alexion has achieved success by developing and commercializing drugs that solve the uncontrolled activation of the complement system (part of the immune system). Ultomiris is an antibody designed to block a protein of the complement system called C5. Disorders of the complement system have been found in a variety of central nervous system diseases. Alexion had hoped that blocking C5 would have an impact on ALS.
Ultomiris was first approved in 2018 for the treatment of adults with rare blood disease paroxysmal nocturnal hemoglobinuria (PNH). The following year, the FDA approved an intravenous drug for another rare disease, atypical hemolytic uremic syndrome. The latest approval of the drug was in June, which is an endorsement for the treatment of PNH in children and adolescents.
Last year, Ultramiris generated more than $1 billion in sales for Alexion. In addition to ALS, the company also evaluated the drug as a treatment for hospitalized COVID-19 patients requiring mechanical ventilation. In January, due to lack of efficacy, the study suspended the recruitment of certain COVID-19 patients. The company is also testing the drug in thrombotic microangiopathies, which are blood clots in small blood vessels in the body that may occur after hematopoietic stem cell transplantation. The third phase of research for this indication started in December last year.
AstraZeneca Announce Alexion’s acquisition agreement at the end of last year.This transaction is fully At the end of last month, Alexion became a new rare disease department under AstraZeneca. In addition to Ultomiris, Alexion also brought AstraZeneca clinical stage projects for metabolic disorders, cancer, and blood and kidney diseases.
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