AstraZeneca Announces Positive Phase III Results for Gefurulimab in Generalized Myasthenia Gravis

On July 24, AstraZeneca announced that Gefurulimab, a bispecific nanobody for adult patients with generalized myasthenia gravis (gMG), demonstrated significant efficacy in the PREVAIL Phase III clinical trial, bringing statistically and clinically meaningful improvements in functional daily activity capabilities for patients.

Generalized Myasthenia Gravis (gMG) is a rare, disabling, chronic autoimmune neuromuscular disorder that leads to loss of muscle function and severe weakness. Individuals with gMG may initially experience symptoms such as slurred speech, double vision, drooping eyelids, and muscle weakness. As the disease progresses, symptoms become more severe, including extreme fatigue, difficulty swallowing, choking, and even respiratory failure. The symptoms of this condition fluctuate rapidly and are unpredictable, causing disability that affects nearly every aspect of a patient’s life, making early intervention and sustained disease control critical treatment goals.

Gefurulimab, as a complement C5 inhibitor, is a novel dual-binding nanobody optimized for subcutaneous self-administration. It is currently being developed for the treatment of anti-acetylcholine receptor antibody-positive (AChR-Ab+) gMG. This investigational drug works by binding to the C5 protein in the terminal complement cascade, which is part of the human immune system. Uncontrolled activation of the complement cascade can lead to an overreaction of the body, attacking its own healthy cells. Gefurulimab's simultaneous binding to serum albumin extends its half-life, allowing for once-weekly dosing. Gefurulimab has received orphan drug designation in the United States for the treatment of myasthenia gravis.

This trial recruited 260 patients across 20 countries globally, all of whom had been diagnosed with myasthenia gravis for at least three months, tested positive for anti-AChR autoantibodies through serological testing, and met the Myasthenia Gravis Foundation of America clinical classification levels II to IV. The trial results showed,Gefurulimab met its primary endpoint and all secondary endpoints, demonstrating statistically significant and clinically meaningful improvement in the total score of the Myasthenia Gravis Activities of Daily Living Scale (MG-ADL) from baseline at week 26 compared to placebo.The MG-ADL total score is a patient-reported scale used to assess the patient's ability to perform daily activities. During the trial, Gefurulimab was well-tolerated, with a safety profile consistent with previous C5 inhibitors in gMG trials, and no new safety signals were observed.

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