AstraZeneca (AZN) reported positive results from its Phase 3 trial of gefurulimab, showing "statistically significant and clinically meaningful improvements" in patients' ability to perform daily activities with generalized Myasthenia Gravis, a chronic autoimmune neuromuscular disorder.
The trial met its primary endpoint, demonstrating a significant improvement in the Myasthenia Gravis Activities of Daily Living score compared to placebo by week 26, with benefits visible as early as week one, the company said Thursday in a statement.
Gefurulimab also met all secondary endpoints, including improvements in the Quantitative Myasthenia Gravis score at weeks four and 26, further supporting its therapeutic potential, the company said. The trial was conducted by AstraZeneca's Alexion rare disease unit.
The treatment is a self-administered, once-weekly injection using a dual-binding nanobody that targets the C5 protein, a key driver of inflammation in the immune system. AstraZeneca said it is tolerated, with a safety profile similar to existing C5 inhibitors. Most reported side effects were mild to moderate, including injection site reactions, headache, and back pain, the company said.
AstraZeneca shares rose 0.4% in recent Thursday trading.
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