Alexion, AstraZeneca Rare Disease, the rare disease business unit of British-Swedish biopharmaceutical company AstraZeneca PLC (STO:AZN, LON:AZN, Nasdaq:AZN), on Thursday announced that gefurulimab, its investigational complement C5 inhibitor, met all primary and secondary endpoints in the PREVAIL Phase III trial for adults with anti-acetylcholine receptor antibody-positive generalised myasthenia gravis (gMG).

Gefurulimab, a dual-binding nanobody, is designed for once-weekly, subcutaneous self-administration. The treatment binds to the C5 protein in the terminal complement cascade, aiming to reduce immune system attacks.

PREVAIL enrolled 260 patients across 20 countries in a double-blind, placebo-controlled, randomised design. The study showed statistically significant and clinically meaningful improvement in Myasthenia Gravis Activities of Daily Living (MG-ADL) total scores at 26 weeks, compared to placebo.

The therapy demonstrated significant reduction in disease severity and was well-tolerated, with no new safety signals and a safety profile consistent with other C5 inhibitors.

Gefurulimab has received Orphan Drug Designation in the United States for the treatment of myasthenia gravis.

Data from the trial will be presented at an upcoming medical meeting and shared with global regulatory authorities.