24th Jul 2025 14:10
(Alliance News) - AstraZeneca PLC on Thursday said its investigational complement C5 inhibitor gefurulimab showed statistically significant results in treating adults with a form of generalised myasthenia gravis, an autoimmune neuromuscular disease.
The Cambridge, England-based pharmaceutical company said phase 3 trial results showed gefurulimab met all its primary and secondary endpoints.
The trial was in adults with anti-acetylcholine receptor antibody-positive generalised myasthenia gravis, which is a rare chronic autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness. It can cause slurred speech, double vision, with symptoms becoming more severe over time, including extreme fatigue, difficulty swallowing, choking and respiratory failure.
Gefurulimab binds to the C5 protein in the terminal complement cascade, a part of the body's immune system. AstraZeneca explains: "When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Gefurulimab's concurrent binding to serum albumin provides an extended half-life, enabling once-weekly dosing."
AstraZeneca said its inhibitor demonstrated a clinically meaningful reduction in disease severity at week 26, when compared to a placebo.
Marc Dunoyer, chief executive officer at Alexion, AstraZeneca Rare Disease, said: "Building on Alexion's pioneering leadership in gMG, these positive results from the Prevail Phase III trial demonstrate the potential for gefurulimab to offer rapid and sustained disease control for this patient community. These data, reflecting patient participation across 20 countries, reinforce the established safety profile and efficacy of C5 inhibition and show the potential for gefurulimab as a first line biologic, with the convenience of a self-administered option."
AstraZeneca shares were up 2.7% to 10,958.20 pence each on Thursday afternoon in London, giving it a market capitalisation of GBP169.45 billion.
By Tom Budszus, Alliance News slot editor
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