UCB: New Positive Data on Generalized Myasthenia Gravis at ICNMD 2026

Rozanolixizumab: Prolonged Improvement of Ocular and Generalized Symptoms

A post hoc pooled analysis of final data from the Phase 3 MycarinG trial and its open-access extensions (MG0004 and MG0007) assessed the impact of repeated, symptom-driven rozanolixizumab cycles on ocular scores in 129 adult patients with gMG who received at least 2 cycles. Weighted average improvements in ocular item scores were observed from baseline to day 43 across 13 treatment cycles. A second post hoc analysis of the final pooled data evaluated individual scores from the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale and the Quantitative Myasthenia Gravis (QMG) scale in the same 129 adult patients. Improvements were consistent across the 13 cycles, encompassing ocular, bulbar, respiratory symptoms, and overall limb or motor impairment. For patients with anti-muscle-specific kinase (MuSK Ab+) antibodies, the final pooled data from MycarinG and its extensions tracked the effect of repeated, symptom-driven treatment cycles in 12 adult patients with gMG, with outcomes measured up to 13 cycles.

Pre-filled Zilucoplan: Use Validation and Sustained Benefit

A human factors validation study involving 100 adult participants (patients with or without prior injection experience, caregivers, healthcare professionals) assessed the safety and effectiveness of using the newly approved pre-filled zilucoplan pen. The buttonless pen, which encases a pre-filled syringe so that the needle is not visible, aims to facilitate self-administration for patients with gMG. According to this study, 90% of adult participants, including patients and caregivers with no prior injection experience, managed to inject correctly on their first attempt without prior training. The pre-filled zilucoplan pen recently received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) as a new additional device presentation to be used in conjunction with standard treatment for adults with gMG with anti-acetylcholine receptor (AChR Ab+) antibodies. A post hoc analysis of the Phase 3 RAISE trial and its open-access extension (RAISE-XT) evaluated changes in individual items of the MG-ADL, QMG, and the revised Myasthenia Gravis Quality of Life (MG-QoL15r) scale from baseline to week 12 in RAISE, and up to week 120 in RAISE-XT. Improvements or absence of symptoms were observed in a greater proportion of adult patients across specific gMG outcome items at week 12 compared to placebo, with these benefits maintained up to week 120.