Company: AZN
Filing Date: 2025-07-24
Form Type: 6-K
Source: 0001654954-25-008413
Chunk: 1

Company: ASTRAZENECA PLC
Filing Date: 2025-07-24
Form: 6-K
Chunk 1
---
asthenia Gravis Activities of Daily Living (MG-ADL) total score at week 26 compared to placebo.

gMG is a rare, debilitating, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness. 1 Those living with gMG may initially experience slurred speech, double vision, droopy eyelids and weakness, with symptoms becoming more severe as the disease progresses, including extreme fatigue, difficulty swallowing, choking and respiratory failure. 2,3

Kelly Gwathmey, MD, Associate Professor of Neurology, Chief of Neuromuscular Division, Virginia Commonwealth University, Richmond, VA, Vice Chair of the MGFA Medical & Scientific Advisory Council and principal investigator in the trial, said: "Rapidly fluctuating symptoms and the unpredictable disability associated with gMG can affect nearly every aspect of a patient's life, making early intervention and sustained disease control a critical treatment goal. A once-weekly, self-administered C5 treatment option would offer patients greater convenience and independence in managing their condition, empowering them to have more control over their therapy."

Marc Dunoyer, Chief Executive Officer, 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."

Gefurulimab was well-tolerated, and the safety profile was consistent with previous trials of C5 inhibitors in gMG with no new safety signals observed. These data will be presented at a forthcoming medical meeting and shared with global regulatory authorities.

#### Notes

#### gMG
gMG is a rare autoimmune disorder characterised by loss of muscle function and severe muscle weakness. 1

Eighty-five percent of people with gMG are AChR antibody-positive meaning they produce specific antibodies (anti-AChR) that bind to signal receptors at the neuromuscular junction (NMJ), the connection point between nerve cells and the muscles they control. 4 This binding activates the complement system, causing the immune system to attack the NMJ, leading to inflammation and a breakdown in communication between the brain and the muscles.