UCB Reports Sustained Long‑Term Efficacy of Rozanolixizumab and Zilucoplan in Generalized Myasthenia Gravis at ICNMD 2026

UCB presented new long-term data at the International Congress on Neuromuscular Diseases (ICNMD) 2026 showing sustained efficacy of rozanolixizumab and zilucoplan across multiple manifestations of generalized myasthenia gravis (gMG). The presentations included final pooled analyses from the Phase 3 MycarinG trial and its open-label extensions, updated long-term findings from the Phase 3 RAISE program, and a human factors study supporting the newly approved zilucoplan pre-filled pen.

Generalized myasthenia gravis is a chronic autoimmune disorder in which pathogenic antibodies impair neuromuscular transmission, causing fluctuating muscle weakness affecting vision, speech, swallowing, breathing, and mobility. Rozanolixizumab is a neonatal Fc receptor (FcRn) inhibitor that reduces pathogenic IgG antibodies, while zilucoplan is a once-daily subcutaneous complement C5 inhibitor approved for adults with anti-acetylcholine receptor antibody-positive (AChR Ab+) gMG.

Rozanolixizumab showed durable symptom control across repeated treatment cycles

A pooled post hoc analysis of the Phase 3 MycarinG trial (NCT03971422) and open-label extension studies MG0004 (NCT04124965) and MG0007 (NCT04650854) evaluated 129 adults who received at least two symptom-driven treatment cycles over as many as 13 cycles.

Patients experienced consistent improvements in clinician- and patient-reported ocular symptoms, including double vision and ptosis, alongside better eye-related quality of life. A second pooled analysis demonstrated sustained improvements across MG-ADL and Quantitative Myasthenia Gravis (QMG) items covering double vision, eyelid drooping, speech, chewing, swallowing, respiratory function, facial muscles, and upper- and lower-limb strength, indicating durable benefit across ocular, bulbar, respiratory, and limb manifestations.

Among 188 patients receiving at least one treatment cycle, 93.1% experienced treatment-emergent adverse events (TEAEs), most of which were mild or moderate. The most common TEAEs were headache (50.0%), diarrhoea (33.5%), and COVID-19 (21.8%). Importantly, adverse event rates did not increase with repeated treatment cycles. Although both 7 mg/kg and 10 mg/kg doses were studied, European approval is based on the approximately 7 mg/kg dose.

 Benefit extended to MuSK antibody-positive disease

Final pooled analyses also evaluated adults with muscle-specific kinase antibody-positive (MuSK Ab+) gMG. Across up to 13 treatment cycles, weighted mean MG-ADL scores improved from 10.4 to 5.2, while QMG scores improved from 16.8 to 9.4. Among 18 patients receiving at least one treatment cycle, 77.8% reported TEAEs, which were predominantly mild or moderate, supporting sustained efficacy with an acceptable safety profile in this challenging subgroup.

Long-term zilucoplan efficacy maintained through Week 120

A post hoc analysis of the Phase 3 RAISE study and ongoing RAISE-XT extension assessed adults with AChR Ab+ gMG through Week 120.

At Week 12, more patients receiving zilucoplan than placebo achieved improvement or no symptoms across MG-ADL domains, including chewing (71.4% vs 49.4%), talking (73.8% vs 57.6%), eyelid drooping (65.5% vs 48.2%), and rising from a chair (63.1% vs 43.5%). These benefits increased further by Week 120, with more than 85% of patients reporting improvement or no symptoms across key MG-ADL items. Similar long-term improvements were observed across QMG and MG-QoL15r assessments.

Pre-filled pen supported self-administration

A human factors validation study involving 100 patients, caregivers, and healthcare professionals found that 90% successfully completed their first injection with the button-free zilucoplan pre-filled pen without prior training. No new use-related safety risks were identified, and only minor revisions to the instructions for use were required. The device recently received a positive CHMP opinion as an additional presentation for adults with AChR Ab+ gMG receiving standard therapy.

Commenting on the findings, Donatello Crocetta, Global Head of Medical Affairs and Chief Medical Officer at UCB, said the data reflect the diverse symptom burden experienced by people living with gMG and reinforce the need for individualized long-term treatment strategies.

UCB is presenting 11 abstracts at ICNMD 2026, expanding evidence that rozanolixizumab and zilucoplan provide durable symptom control with manageable long-term safety while offering additional treatment flexibility for adults living with generalized myasthenia gravis.

What This Means for Patients

For people living with generalized myasthenia gravis, the new findings suggest that long-term treatment with rozanolixizumab and zilucoplan can provide sustained control of symptoms that affect everyday life, including double vision, eyelid drooping, speaking, swallowing, breathing, and muscle strength. The analyses also indicate that repeated treatment cycles with rozanolixizumab maintain their benefit without an increase in treatment-related side effects over time. In addition, the newly approved zilucoplan pre-filled pen, which enabled 90% of users to successfully self-administer their first injection without prior training, may offer greater convenience and independence for patients who prefer home-based treatment. Together, these data support more personalized long-term treatment options for adults living with generalized myasthenia gravis.

Reference

UCB presents latest data from its targeted generalized myasthenia gravis treatment portfolio at International Congress on Neuromuscular Diseases 2026 | UCB