UCB, a global biopharmaceutical company, announced it will present comprehensive new clinical data for its two approved generalized myasthenia gravis (gMG) therapies at the 2025 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting and Myasthenia Gravis Foundation of America (MGFA) Scientific Session, taking place October 29 – November 1, 2025, in San Francisco.
The company will showcase 18 abstracts highlighting patient outcomes with rozanolixizumab (RYSTIGGO) and zilucoplan (ZILBRYSQ), including new insights on corticosteroid-sparing strategies, quality of life improvements, and long-term safety and effectiveness data.
Key Clinical Data Presentations
Rozanolixizumab (RYSTIGGO) Studies
The presentations will feature an oral presentation focusing on data from the double-blind Phase 3 MycarinG study and its open-label extensions, including a comprehensive analysis of corticosteroid dose tapering during treatment with rozanolixizumab in patients with gMG. Additional rozanolixizumab data includes a new comprehensive analysis of treatment response from the Phase 3 MycarinG study and its open-label extensions, offering valuable information on efficacy in clinical settings.
Long-term safety data will be presented through a further analysis of the Phase 3 MycarinG study exploring the long-term safety of cyclic rozanolixizumab treatment in patients with gMG to evaluate its potential for sustained therapeutic use.
Zilucoplan (ZILBRYSQ) Research
Post hoc data from the Phase 3 RAISE-XT trial will examine zilucoplan's impact on health-related quality-of-life items, including more severe symptoms such as difficulty speaking. The presentations will also include new data on the long-term effectiveness of zilucoplan, focusing on severe exacerbations in gMG based on predictive modeling from the Phase 3 RAISE and RAISE-XT clinical trials and a U.S. real-world database.
From the RAISE-XT 120-week follow-up, new post-hoc data will examine the long-term effect of zilucoplan treatment on fatigue, a significant symptom for gMG patients.
Real-World Evidence and Patient Engagement
The conference presentations will include an analysis exploring user characteristics and adherence patterns among individuals living with myasthenia gravis, leveraging data from the HumaMG app to provide valuable real-world insights into patient management and engagement.
"Empowering patients with innovative solutions is at the core our purpose at UCB," said Donatello Crocetta, Global Head of Medical Affairs & Chief Medical Officer at UCB. "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."
Disease Background and Treatment Landscape
Generalized myasthenia gravis is a rare autoimmune disease with a global prevalence of 100–350 cases per 1 million people. People living with gMG can experience a variety of symptoms, including severe muscular weakness that can result in double vision, drooping eyelids, difficulty with swallowing, chewing and talking, as well as life-threatening weakness of the muscles of respiration.
In gMG, pathogenic autoantibodies can impair synaptic transmission at the neuromuscular junction by targeting specific proteins on the post-synaptic membrane. This disrupts the ability of the nerves to stimulate the skeletal muscle and results in a weaker contraction.
Therapeutic Mechanisms and Approvals
Rozanolixizumab is a subcutaneously administered, humanized monoclonal antibody that specifically binds with high affinity to human neonatal Fc receptor (FcRn). It has been designed to block the interaction of FcRn and Immunoglobulin G (IgG), accelerating the catabolism of antibodies and reducing the concentration of pathogenic IgG autoantibodies.
Zilucoplan is a once-daily subcutaneous injection, self-administered peptide inhibitor of complement component 5 (C5 inhibitor). As the only self-administered C5 inhibitor targeted therapy for gMG, zilucoplan may inhibit complement-mediated damage to the neuromuscular junction through its targeted mechanism of action.
Both therapies have received regulatory approvals across multiple regions. Rozanolixizumab-noli was approved by the FDA in June 2023 for the treatment of gMG in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody-positive. Zilucoplan was approved by the U.S. FDA in October 2023 for the treatment of gMG in adult patients who are anti-acetylcholine receptor antibody positive.
Additional Conference Activities
UCB is hosting a panel discussion entitled 'Continuing the Conversation - Subpopulations with Barriers to Care in Myasthenia Gravis' on October 29, 2025, and a UCB-sponsored Industry Therapeutic Update on expert perspectives on two targeted generalized myasthenia gravis treatments on October 31, 2025.
The company is also collaborating with the MG community on the recently launched 'Faces of MG' campaign, seeking to elevate the voices of those deeply connected to the disease and to foster understanding of the invisible impact it has on people's lives.
