Phase 3 MG0020 Trial Highlights Success of Self-Administered Rozanolixizumab for Generalized Myasthenia Gravis

New data from the open-label, crossover phase 3 MG0020 study (NCT05681715) of rozanolixizumab (Rystiggo; UCB) presented at the 2025 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) meeting, held October 29 to November 1, in San Francisco, California, showed that all patients with generalized myasthenia gravis (gMG) in the trial successfully self-administered the therapy, with the clinical response and safety profile being consistent prior studies.^1,2

Among 62 patients with gMG enrolled, researchers randomized 55 participants. Investigators reported that all patients successfully self-administered rozanolixizumab at each visit. Reductions from baseline in total immunoglobulin G (IgG) and Myasthenia Gravis–Activities of Daily Living (MG-ADL) scores of at least 2.0 points were sustained during self-administration. Regardless of sequence, authors noted that most patients preferred self-administration (n= 35) over health care provider administration (n = 5) during training whereas 9 patients reported no preference from those randomized.

"At UCB, we are deeply proud to present a robust range of data at this year’s AANEM Annual Meeting and MGFA Scientific Session, demonstrating our commitment to elevating the lives of patients with gMG through shared decision-making and continuous collaboration with healthcare professionals,” Kimberly, Moran, PhD, SVP & Head, US Rare Diseases at UCB, said in a statement.³ "People living with gMG face unpredictable symptoms that can greatly impact their ability to perform daily activities. Expanding the body of evidence generation of the risks and benefits of our gMG targeted therapies is central to our mission of delivering meaningful, sustainable clinical improvements to address the unique challenges these patients encounter.”

Presented by lead author Rachana K. Gandhi Mehta, MBBS, the MG0020 study explored patient self administration of rozanolixizumab using manual push and syringe driver methods in adults with gMG. Participants in the trial received once-weekly rozanolixizumab for 18 weeks including a 6-week training period followed by 1:1 randomization to Sequence 1 (syringe driver then manual push) or Sequence 2 (manual push then syringe driver) for 6 weeks per method.

Published in the Journal of Neurology, the primary end point was successful self-administration, defined as correct infusion site selection, subcutaneous administration, and delivery of the intended dose at weeks 12 and 18. Secondary assessments included treatment-emergent adverse events (TEAEs), changes in total IgG, MG-ADL scores, patient preference, and patient experience measured by the infusion version of the Self-Injection Assessment Questionnaire (SIAQ).

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The trial design was originally presented at the 2024 AANEM meeting, held October 15-18, in Savannah, Georgia also by Mehta, an assistant professor of neurology at the Wake Forest School of Medicine.⁴ In an interview with NeurologyLive^® during the meeting, Mehta discussed the study more in detail and its unique build. She provided clinical insight on how the study came to be and the advantages the syringe driver may bring. Furthermore, Mehta commented on how this study speaks to the progress in treating patients with gMG.

Additional findings from the study showed that the majority of patients preferred manual push (n = 25) to syringe driver (n = 17); 7 patients had no preference from those that were randomized. Furthermore, the mean pretreatment SIAQ scores ranged from 5.8 to 8.6 (0 to 10 scale) whereas the mean post treatment SIAQ scores 7.5 to 9.3, suggesting a positive self administration experience. Authors noted that TEAEs occurred in 75.8% (n = 47) of patients enrolled, with most of the events reported being considered mild or moderate.

"Empowering patients with innovative solutions is at the core our purpose at UCB," Donatello Crocetta, global head of medical affairs and chief medical officer at UCB, said in a statement.³ "Our presentations at the AANEM Annual Meeting and MGFA Scientific Session showcase our focus on developing treatment options that not only highlight clinical management but also quality of life, providing meaningful outcomes that make a difference for those living with gMG."

Rozanolixizumab was first approved by the FDA in June 2023 as a new treatment for both anti-acetylcholine receptor or anti-muscle-specific tyrosine kinase antibody positive gMG, the most common subtypes of the disease. The basis for the approval was data from the phase 3 MycarinG study (NCT03971422), in which rozanolixizumab-treated patients showed significant reductions in the primary end point of MG-ADL scores (P <.001) over a 43-day period.^5,6

In the MycarinG study, 200 patients were randomized into three groups to receive rozanolixizumab 7 mg/kg (n = 66), 10 mg/kg (n = 67), or placebo (n = 67) for 6 weeks, followed by an 8-week observation period. The treatment demonstrated significant effects on secondary endpoints, including the Quantitative Myasthenia Gravis (QMG) score, which measures muscle weakness. By day 43, the QMG total score showed statistically significant improvements in the rozanolixizumab groups, with point changes of –5.4 and –6.7 in the 7 mg/kg and 10 mg/kg groups, respectively, compared to –1.9 in the placebo group (P < .001).

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REFERENCES
1. Gandhi Mehta RK, Antonzzi C, Berkowicz T, et al. Patient Preferences and Experience With Self-Administration of Rozanolixizumab in Generalized Myasthenia Gravis: The MG0020 Study. Presented at: 2025 AANEM; October 29 to November 1; San Francisco, California. Abstract 16.
2. Bril V, Antozzi C, Berkowicz T, et al. Self-administration of rozanolixizumab via manual push and infusion pump methods in patients with generalised myasthenia gravis: a randomised, phase 3, open-label, crossover study. J Neurol. 2025;272(10):686. Published 2025 Oct 11. doi:10.1007/s00415-025-13420-6
3. UCB to unveil new data for RYSTIGGO® (rozanolixizumab-noli) and ZILBRYSQ®(zilucoplan) for gMG at the 2025 AANEM Annual Meeting and MGFA Scientific Session. News release. October 29, 2025. Accessed October 29, 2025. https://www.ucb-usa.com/stories-media/UCB-U-S-News/detail/article/ucb-to-unveil-new-data-for-rystiggo-rozanolixizumab-noli-and-and-zilbrysq-zilucoplan-for-gmg-at-the-2025-aanem-annual-meeting-and-mgfa-scientific-session
4. Mehta RG, Bril V, Antozzi C, et al. Self-administration of subcutaneous rozanolixizumab in patients with generalized myasthenia gravis: clinical study design. Presented at: 2024 AANEM Annual Meeting; October 15-18; Savannah, GA. ABSTRACT 164
5. UCB announces US FDA approval of Rystiggo (rozanolixizumab-noli) for the treatment of adults with generalized myasthenia gravis. News release. June 27, 2023. Accessed October 29, 2025. https://www.prnewswire.com/news-releases/ucb-announces-us-fda-approval-of-rystiggo-rozanolixizumab-noli-for-the-treatment-of-adults-with-generalized-myasthenia-gravis-301864023.html
6. Bril V, Druzdz A, Grosskreutz J, et al. Safety and efficacy of rozanolixizumab in patients with generalized myasthenia gravis (MycarinG): a randomized, double-blind, placebo-controlled, adaptive phase 3 study. Lancet Neurol. 2023;22(5):383-394. doi:10.1016/S1474-4422(23)00077-7.