
Assessing Clinical Deterioration With Gefurulimab in the PREVAIL Trial: James Winkley, MD
The chairman of the Neurology Service Line at Baptist Health in Kentucky discusses PREVAIL findings on clinical deterioration, rescue therapy use, and hospitalization with gefurulimab in generalized myasthenia gravis. [WATCH TIME: 3 minutes]
WATCH TIME: 3 minutes
Generalized myasthenia gravis (gMG) is a chronic autoimmune disorder characterized by fluctuating muscle weakness that can produce substantial impairment in daily functioning and, during periods of worsening, lead to exacerbations requiring rescue treatment or hospitalization. Complement activation plays an important pathogenic role in patients with anti-acetylcholine receptor antibody-positive (AChR-Ab+) gMG, providing a therapeutic target for agents designed to interrupt the complement cascade.
Gefurulimab is an investigational dual-binding nanobody designed to inhibit complement component 5 and administered by once-weekly subcutaneous self-injection. In the phase 3 PREVAIL trial, 260 adults with AChR-Ab+ gMG were randomly assigned to gefurulimab or placebo for 26 weeks. Gefurulimab significantly improved MG-ADL scores by 4.2 points from baseline compared with 2.6 points for placebo, with significant improvements also observed on the Quantitative Myasthenia Gravis scale.1
“We saw 16 require rescue therapy in the placebo arm versus seven in the treatment arm, and that was statistically significant. For hospitalizations, 12 patients required MG-related hospitalization in the placebo arm versus three in the treatment arm.”
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In an interview with NeurologyLive®, Winkley discussed how these additional findings complement PREVAIL's primary efficacy results. He detailed differences in clinical deterioration between the treatment groups and examined whether gefurulimab treatment translated into reductions in rescue interventions, including IVIg, plasma exchange, and high-dose corticosteroids, as well as gMG-related hospitalizations.
REFERENCES
1. Gwathmey KG, Saccà F, Howard JF, et al. Efficacy and safety of gefurulimab in generalized myasthenia gravis: the PREVAIL phase 3 randomized clinical trial. JAMA Neurol. 2026;83(9):841-850. doi:10.1001/jamaneurol.2026.2333.
2. Winkley J. Clinical deterioration, rescue therapy, and hospitalization among patients with generalized myasthenia gravis (gMG): results from the PREVAIL trial of gefurulimab. Presented at: 2026 Myasthenia Gravis Foundation of America Scientific Session; September 29, 2026; Orlando, FL. Abstract 108.
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