NMOSD Drug Associated with Favorable Outcomes in Individuals with Generalized Myasthenia Gravis

Results presented at the American Academy of Neurology (AAN) 2025 Annual Meeting demonstrate the efficacy and safety of Uplizna (inebilizumab-cdon; Amgen, Dublin, Ireland) for the treatment of individuals with acetylcholine receptor-positive (AChR+) or muscle-specific kinase-positive (MuSK+) generalized myasthenia gravis (gMG).

The randomized, parallel-group, phase 3 MINT trial (NCT04524272) included 238 adults with gMG who were randomized 1:1 to receive either intravenous (IV) Uplizna 300 mg or placebo. Treatment was administered on day 1, day 15, and week 26 (AChR+ group only) of the randomized control period, which lasted 52 weeks for individuals with AChR+ gMG and 26 weeks for individuals with MuSK+ gMG. The primary endpoint was the change in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at week 26 for the combined population. Secondary end points included changes in Quantitative Myasthenia Gravis (QMG) scores and subgroup analyses for the AChR+ and MuSK+ gMG populations.

At week 26, individuals treated with Uplizna demonstrated the following:

• Greater improvement in MG-ADL scores (-4.2) compared with placebo (-2.2) (difference, -1.9; 95% CI, -2.9 to -1.0; P<.001)
• Greater improvement in QMG scores (-4.8) compared with placebo (-2.3) (difference, -2.5; 95% CI, -3.8 to -1.2; P<.001)

Adverse events occurred in 80.7% of Uplizna-treated individuals vs 73.1% of those who received placebo, and serious adverse events occurred in 8.4% of Uplizna-treated individuals vs 13.4% of those who received placebo.

Uplizna is Food and Drug Administration (FDA)-approved for the treatment of adult patients with anti-aquaporin-4 antibody positive (AQP4+) neuromyelitis optica spectrum disorder (NMOSD) and has been granted orphan drug designation by the FDA for the treatment of gMG.

"I'm looking forward to further examining the 52-week MINT data with my colleagues in the neurology community at AAN," said Richard J. Nowak, MD, MS, global principal study investigator and director of the Myasthenia Gravis Clinic at Yale University. "These results showed that Uplizna consistently relieved burdensome symptoms and improved activities of daily living for gMG patients."