Nipocalimab cuts generalized myasthenia gravis symptoms in phase 2 trial
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Key takeaways:
- Nipocalimab brought dose-dependent improvements to symptoms of generalized myasthenia gravis.
- The drug had a similar incidence of treatment-emergent adverse events compared with placebo.
Nipocalimab showed evidence of dose-dependent, statistically significant symptom reductions in patients with generalized myasthenia gravis, while remaining generally safe and well-tolerated, in a phase 2 trial.
The finding in favor of the fully human monoclonal antibody comes as existing treatments for generalized myasthenia gravis are associated with persistent symptoms and side effects that limit their use, Carlo Antozzi, MD, and colleagues wrote in Neurology.
The study enrolled 68 adult patients with generalized myasthenia gravis who had inadequate response to standard-of-care therapy. While continuing that therapy, the patients were randomly assigned in a 1:1:1:1 ratio to undergo one of the following treatments:
- IV placebo dose every 2 weeks;
- 5 mg/kg nipocalimab once every 4 weeks;
- 30 mg/kg nipocalimab once every 4 weeks;
- 60 mg/kg nipocalimab every 2 weeks; or
- a single 60 mg/kg nipocalimab dose.
The primary efficacy endpoint was mean change from baseline in Myasthenia Gravis-Activities of Daily Living total score. Across 57 days, there was a statistically significant linear trend of improved mean scores (P = .03) in the groups on sustained nipocalimab regimens, as well as the placebo group.
All nipocalimab groups combined had a similar overall incidence of treatment-emergent adverse events (83.3%) compared with placebo (78.6%), as well as infections (33.3% vs. 21.4%, respectively).
“All dose groups demonstrated acceptable safety and tolerability profile, with no clinically significant safety signals identified,” Antozzi and colleagues wrote. “Based on the analysis of safety, tolerability and clinical endpoints from this phase 2 study, a phase 3 confirmatory study for nipocalimab in the treatment of [generalized myasthenia gravis] is ongoing.”