Data Presented at AANEM 2026 Highlight Sustained Symptom Control and High Adherence for Participants with gMG Treated With Zilbrysq
KEY TAKEAWAYS
- Participants reported taking 99.2% of expected zilucoplan doses during the RAISE-XT open-label extension, providing feasibility data for long-term daily self-administration.
- Nearly 30% of patients receiving nonsteroidal immunosuppressive therapy at baseline discontinued treatment or reduced the dose by at least 50% during long-term follow-up.
- Improvements in fatigue persisted through 120 weeks, while patient-reported assessments showed high satisfaction and confidence with self-injection.
New long-term data presented at the 2026 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting provided additional inssight into sustained disease control and treatment burden with Zilbrysq (zilucoplan; UCB, Atlanta, GA) treatment in adults with anti–acetylcholine receptor antibody-positive (AChR+) generalized myasthenia gravis (gMG). Findings presented during a UCB-sponsored Industry Therapeutic Update included outcomes through 120 weeks across minimal symptom expression (MSE), concomitant therapy use, adherence, fatigue, and self-administration.
The findings were reported from the phase 3 RAISE clinical trial (NCT04115293) and its open-label extension, RAISE-XT (NCT04225871). RAISE evaluated once-daily subcutaneous Zilbrysq treatment vs placebo in adults with AChR+ gMG, with change in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at week 12 as the primary efficacy endpoint. Participants could subsequently receive Zilbrysq in RAISE-XT, providing follow-up through week 120. Zilbrysq is a complement C5 inhibitor approved by the Food and Drug Administration (FDA) for adults with AChR+ gMG.
Insights from the Presented Data
- Among participants who achieved MSE after starting Zilbrysq, the median proportion of observed time subsequently spent in MSE was 80.8% through week 120.
- Among patients receiving corticosteroids at RAISE baseline, 41.5% discontinued treatment or reduced the dose by at least 50%. For nonsteroidal immunosuppressive therapy, the corresponding proportion was 29.8%.
- Patients reported taking a mean 99.2% of expected Zilbrysq doses during RAISE-XT, and improvements in Neuro-QoL Short Form–Fatigue scores were observed through week 120.
- Patient-reported findings added context around daily self-administration. At week 60, median scores exceeded 8 of 10 across measures including satisfaction, confidence with self-injection, willingness to continue treatment, and ease of use.
How This May Influence Clinical Practice
For neurologists treating patients with AChR+ gMG, the findings provide longer-term information relevant to treatment selection and follow-up. Sustained MSE and observed reductions in background corticosteroid and immunosuppressive therapy may inform discussions about disease control and treatment burden, although the analyses do not establish that concomitant therapy can be reduced in every patient. High adherence and favorable self-administration assessments also provide practical information when discussing whether daily subcutaneous treatment fits an individual patient’s preferences and circumstances.
Zilucoplan carries a Boxed Warning for serious meningococcal infections, making appropriate vaccination and infection-risk management important components of treatment.
Sources
- Howard JF Jr, Dimachkie M. Complement inhibition for patients with anti-AChR Ab+ generalized myasthenia gravis (gMG). Presented at: 2026 American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting; September 30, 2026; Orlando, FL. Industry Therapeutic Update sponsored by UCB, Inc.
- UCB. Data presented at 2026 AANEM Annual Meeting and MGFA Scientific Session. Press release. Published September 29, 2026.