UCB Highlights Robust Data on Generalized Myasthenia Gravis Therapies at 15th MGFA International Conference, Reinforcing Commitment to Patient-Centered Innovation
UCB,a global biopharmaceutical leader specializing in immunology and neurology, has announced the presentation of a comprehensive suite of data from its generalized myasthenia gravis (gMG) treatment portfolio at the 15th Myasthenia Gravis Foundation of America (MGFA) International Conference. Held from May 13–15, 2025, in The Hague, The Netherlands, the event serves as a premier global platform for advancing clinical knowledge and collaborative efforts in treating this rare, chronic autoimmune neuromuscular disease.
Spanning a total of 21 abstracts, UCB’s contributions reflect a multifaceted commitment to both scientific innovation and the lived experiences of patients. The company’s presentations include pivotal findings from late-stage clinical trials, long-term extension studies, real-world evidence research, and patient preference analyses. Together, the data showcase the evolving treatment landscape of gMG and emphasize UCB’s strategic focus on precision-targeted therapies that deliver improved outcomes and empower patients in their disease management journey.
The company is presenting new findings for two of its FDA- and EMA-approved therapies for adults with generalized myasthenia gravis: RYSTIGGO®▼ (rozanolixizumab), a subcutaneous anti-FcRn monoclonal antibody, and ZILBRYSQ®▼ (zilucoplan), a once-daily, subcutaneous, self-administered peptide inhibitor of complement C5. These innovative therapies target distinct immunopathological pathways involved in gMG, offering differentiated therapeutic options to address patient heterogeneity in disease presentation and progression.
“Our presence at the 15th MGFA International Conference reaffirms UCB’s longstanding commitment to improving the lives of people with generalized myasthenia gravis,” said Dr. Donatello Crocetta, Chief Medical Officer at UCB. “We are particularly excited to present new long-term data on rozanolixizumab’s efficacy and safety, and to share findings showing the early and sustained response observed with zilucoplan. These insights reflect not only the clinical potential of our therapies but also our patient-centered research philosophy.”
Pivotal and Real-World Data Offer New Insights into gMG Management
Among the most anticipated presentations from UCB at the MGFA Conference are final pooled analyses of the MycarinG Phase 3 clinical trial and its open-label extension (OLE) studies. These analyses evaluate the long-term efficacy and safety of rozanolixizumab over multiple symptom-driven treatment cycles in adult patients with generalized MG. The findings underscore the therapeutic consistency and tolerability of rozanolixizumab in repeated administration settings, reinforcing its role as a flexible treatment option tailored to the episodic nature of gMG symptoms.1,2
Complementing this data set is a 120-week post hoc analysis of the RAISE-XT study, which evaluated the durability of clinical responses to zilucoplan. The analysis reveals that patients treated with zilucoplan demonstrated both early and sustained improvements in muscle strength and function, supporting its utility as a long-term treatment strategy in generalized MG. The ability to maintain clinical benefit over an extended period is especially relevant given the relapsing-remitting course of the disease and the unmet need for therapies that can offer continuous disease control.3
Another presentation of note is from the MG0020 study, an open-label, crossover Phase 3 trial that assessed patient experiences with self-administered rozanolixizumab. The study evaluated not only clinical safety and tolerability but also patient preferences and quality-of-life outcomes. Results from MG0020 offer a unique perspective on how treatment delivery mode affects patient autonomy, convenience, and overall satisfaction—factors that are increasingly important in the management of chronic conditions.4
Advancing Real-World Evidence with the Vitaccess Real MG Registry
In a landmark step toward expanding real-world understanding of myasthenia gravis, UCB is also unveiling details of its support for the newly launched Vitaccess Real MG (VRMG) Registry, a global initiative designed to capture the experiences and outcomes of people living with gMG in everyday clinical settings. UCB is both the funder and the first subscriber to this ambitious international patient registry.
The VRMG Registry will provide valuable longitudinal insights on treatment patterns, symptom evolution, comorbidity burden, and healthcare resource utilization. It will also allow stakeholders—including clinicians, researchers, and policymakers—to identify trends and gaps in care across diverse patient populations.5 UCB’s involvement underscores its leadership in incorporating the patient voice into therapeutic development and healthcare decision-making processes.
“By supporting the Vitaccess Real MG Registry, we aim to bridge the gap between clinical trial results and real-world patient experiences,” Dr. Crocetta added. “This initiative aligns with our mission to go beyond clinical endpoints and create tangible improvements in patients’ day-to-day lives.”
Measuring What Matters: Functional and Ocular Outcomes
UCB will also present an analysis evaluating the impact of zilucoplan on Myasthenia Gravis Activities of Daily Living (MG-ADL) scores and Quantitative Myasthenia Gravis (QMG) ocular subdomain scores from both the RAISE and RAISE-XT studies. These measures are critical indicators of disease burden, as gMG often affects ocular function and daily capabilities such as speaking, swallowing, and breathing.
The analysis shows that zilucoplan provides clinically meaningful improvements in both functional and ocular domains, highlighting its comprehensive benefit in addressing the multifactorial symptoms of gMG. This evidence is particularly important for clinicians seeking a holistic approach to disease control that goes beyond generalized muscle strength.6
Comparative Effectiveness Research: Zilucoplan Versus Other C5 Inhibitors
In a bid to further differentiate its C5 inhibitor, UCB is also presenting a matching-adjusted indirect comparison (MAIC) study comparing zilucoplan to eculizumab and ravulizumab, two other therapies approved for the treatment of gMG that also target the complement C5 pathway.
The MAIC methodology adjusts for baseline patient characteristics to enable a more equitable comparison across different clinical trial populations. While indirect, the analysis offers preliminary insights into the relative efficacy and safety profiles of these therapies. Results suggest that zilucoplan may offer comparable or improved outcomes in certain patient subgroups, potentially making it a preferred option for patients seeking a convenient, self-administered treatment.7
A Deepening Commitment to Myasthenia Gravis Patients
The data presented by UCB at the 15th MGFA International Conference reflect more than just scientific achievement—they exemplify a commitment to reshaping the treatment paradigm in generalized myasthenia gravis. By advancing therapies that offer long-term control, early onset of action, patient flexibility, and robust real-world relevance, UCB is reinforcing its position as a pioneer in the rare neuromuscular disease space.
As the company continues to invest in clinical development, patient engagement, and data generation across diverse settings, its vision for gMG treatment continues to evolve. The convergence of robust clinical trial data and real-world evidence shared this week in The Hague may help shape the next generation of standards in managing this complex, often debilitating autoimmune disease.
