Zilbrysq (zilucoplan) / UCB - LARVOL DELTA

A Study to Assess Zilucoplan Concentration in Breast Milk of Healthy Lactating Women (clinicaltrials.gov) - P1 | N=15 | Recruiting | Sponsor: UCB Biopharma SRL | Trial completion date: Jul 2026 ➔ Mar 2027 | Trial primary completion date: Jul 2026 ➔ Mar 2027

Trial completion date • Trial primary completion date

Comparing Traditional and Novel Therapies in Myasthenia Gravis: A Single-center Observational Study (AAN 2026) - "Traditional Therapy Group (T) patients received acetylcholinesterase inhibitors, corticosteroids, or immunosuppressants, while Novel Therapy Group (N), had biologics such as Eculizumab (Soliris), Ravulizumab (Ultomiris), Zilucoplan, or Efgartigimod (Vyvgart). Our study suggests that novel therapies may provide better symptom control but remain limited by cost and insurance barriers. Larger studies are needed to evaluate long-term efficacy, safety and cost-effectiveness."

Clinical • Observational data • CNS Disorders • Immunology • Myasthenia Gravis

Zilucoplan is effective for ravulizumab-refractory generalized myasthenia gravis with the C5 p.Arg885His variant: a case report. (PubMed, Neuromuscul Disord) - "This is the first report directly demonstrating ravulizumab nonresponse and a robust zilucoplan response in gMG with this variant. Variant-guided selection of complement inhibitors is essential, and pragmatic use of readily available CH50 testing combined with C5 genotyping may help identify potential nonresponders to eculizumab or ravulizumab."

Journal • CNS Disorders • Complement-mediated Rare Disorders • Hematological Disorders • Myasthenia Gravis • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Synergistic Clinical Improvement in a Refractory AChR-positive Non-thymomatous Generalized Myasthenia Gravis Patient Treated With Concurrent Zilucoplan and Rozanolixizumab (AAN 2026) - "However, the concurrent use of these agents has not been systematically studied, and data on potential synergistic effects are lacking.Design/ A 70-year-old female patient developed ocular myasthenia gravis in June 2023, confirmed as AChR-positive and initially responsive to prednisone and pyridostigmine...She was started on eculizumab, achieving marked clinical improvement. She was later transitioned to ravulizumab with initial stability but relapsed in early 2025 despite high-dose steroids, necessitating additional plasma exchange... This case highlights the potential synergistic benefit of dual-pathway inhibition. This combination may be feasible as zilucoplan (a macrocyclic peptide) and rozanolixizumab (a monoclonal antibody) have distinct mechanisms and no known pharmacologic interaction. Further research is needed to evaluate the safety, efficacy, and immunologic rationale of concurrent use in larger controlled studies."

Clinical • CNS Disorders • Myasthenia Gravis • Respiratory Diseases • Solid Tumor • Thymus Cancer

Early Real-life Experience on Zilucoplan for Generalized Myasthenia Gravis: ZILU25 Multicenter Observational Study (AAN 2026) - "Minimal symptomsexpression (MSE) was obtained in in 55.6% of patients at W48 with a mean reductionof the daily prednisone dose of 8.3 mg at W24. Zilucoplan was well-tolerated and effective in most patients with AChR-positive gMG. Aclinical meaningful effect was reported since the first week and was sustained after 24weeks. Further studies are needed to confirm these preliminary real-life data."

Clinical • Observational data • CNS Disorders • Immunology • Myasthenia Gravis

Sustained Minimal Symptom Expression in Generalized Myasthenia Gravis: A 120-week Post Hoc Analysis of RAISE-XT (AAN 2026) - P2, P3 | "Zilucoplan demonstrated sustained efficacy, as shown by maintenance of MSE response up to 120 weeks of treatment."

Retrospective data • CNS Disorders • Myasthenia Gravis

Bioequivalence and Effectiveness of Auto-Injector-Delivered, Self-administered Zilucoplan Compared With Pre-filled Syringe (AAN 2026) - P1, P3 | "Zilucoplan bioequivalence was established in healthy volunteers between ZLP-AI and ZLP-PFS. Self-administration with ZLP-AI achieved high complete dose delivery and self-injection satisfaction, and was well-tolerated in patients with gMG, suggesting that ZLP-AI is an effective alternative for zilucoplan administration."

CNS Disorders • Myasthenia Gravis

Rapid Symptomatic Improvement and Long-Term Minimal Symptom Expression in a Myasthenia Gravis Patient Treated With Zilucoplan Without Maintenance Cholinesterase Inhibitors, Corticosteroids, or Immunosuppressants: A Case Report. (PubMed, Cureus) - "Clinical stability was sustained for at least one and a half years without concomitant cholinesterase inhibitors, corticosteroids, or other immunosuppressants. This case suggests that zilucoplan may represent an effective and durable treatment option for generalized MG."

Journal • CNS Disorders • Immunology • Myasthenia Gravis

Strong Execution Fueling Sustained Company Growth (UCB Press Release) - "Revenue in 2025 increased to € 7 741 million (+26%; +29% CER1) and net sales went up to € 7 388 million (+32%; +35% CER1). This growth was driven by the strong, consistent growth of UCB’s growth drivers: BIMZELX, EVENITY, FINTEPLA, RYSTIGGO and ZILBRYSQ, thanks to strong execution."

Sales • Ankylosing Spondylitis • CNS Disorders • Hidradenitis Suppurativa • Myasthenia Gravis • Osteoporosis • Psoriasis • Psoriatic Arthritis

Zilucoplan for Severe gMG Exacerbations (clinicaltrials.gov) - P3 | N=15 | Recruiting | Sponsor: Miriam Freimer | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Myasthenia Gravis

HEALEY ALS Platform Trial - Master Protocol (clinicaltrials.gov) - P2/3 | N=1500 | Recruiting | Sponsor: Merit E. Cudkowicz, MD | Active, not recruiting ➔ Recruiting | Trial primary completion date: Nov 2027 ➔ Jul 2027

Enrollment open • Trial primary completion date • Amyotrophic Lateral Sclerosis • CNS Disorders

Letter to editor: Early real-life experience on zilucoplan for generalized myasthenia gravis: ZILU25 multicenter observational study. (PubMed, J Neurol Sci) - No abstract available

Journal • Observational data • CNS Disorders • Myasthenia Gravis

Assessing the Value Contribution of Vyvgart® (Efgartigimod Alfa) in the Treatment of Generalized Myasthenia Gravis with Acetylcholine Receptor Antibody in Spain Through Multi-criteria Decision Analysis. (PubMed, Adv Ther) - "Efgartigimod has been recognised as a valuable option for gMG AChR+ treatment in Spain by a multidisciplinary panel of experts through the application of MCDA, receiving higher scores compared with ravulizumab, zilucoplan, and rozanolixizumab."

Journal • CNS Disorders • Immunology • Myasthenia Gravis • Rare Diseases

Letter to the editor: Early real-life experience on Zilucoplan for generalized myasthenia gravis: ZILU25 multicenter observational study. (PubMed, J Neurol Sci) - No abstract available

Journal • Observational data • CNS Disorders • Myasthenia Gravis

Authors' reply to comment letter on ZILU25 study. (PubMed, J Neurol Sci) - No abstract available

Journal • CNS Disorders • Myasthenia Gravis

Comments on ZILU25: Real-world efficacy of Zilucoplan in generalized myasthenia gravis. (PubMed, J Neurol Sci) - No abstract available

Journal • Real-world evidence • CNS Disorders • Myasthenia Gravis

Novel Therapies for Generalized Myasthenia Gravis: Insights Into FcRn and Complement Inhibition. (PubMed, Am J Ther) - "These therapies offer personalized, effective options for MG management, though challenges in cost and access remain. Future research should address long-term outcomes and biomarkers."

Journal • Review • CNS Disorders • Immunology • Infectious Disease • Myasthenia Gravis

HEALEY ALS Platform Trial - Master Protocol (clinicaltrials.gov) - P2/3 | N=1500 | Active, not recruiting | Sponsor: Merit E. Cudkowicz, MD | Trial completion date: Apr 2026 ➔ Aug 2028 | Trial primary completion date: Jul 2025 ➔ Nov 2027

Trial completion date • Trial primary completion date • Amyotrophic Lateral Sclerosis • CNS Disorders

Body weight distribution of US patients with myasthenia gravis. (PubMed, J Med Econ) - "The dosing requirements of certain therapies (efgartigimod intravenous, nipocalimab, ravulizumab, rozanolixizumab, zilucoplan) are based on patient's body weight. The annual cost of health-care-provider-administered treatment for patients with gMG was estimated at $223,272-$648,960 depending on drug and body weight; annual cost was highest for patients who weighed >80 kg (up to $648,960 versus up to $467,044 for patients who weighed <80 kg). Our findings demonstrate that patients with MG may be heavier on average than previously assumed, which may influence the cost-effectiveness of gMG therapies with weight-based dosing."

Journal • CNS Disorders • Immunology • Myasthenia Gravis

Cellular and humoral vaccination response under immunotherapies-German consensus on vaccination strategies in neurological autoimmune diseases. (PubMed, Ther Adv Neurol Disord) - "The specific humoral and cellular response to vaccination can be compromised under alemtuzumab, azathioprine, cladribine, cyclophosphamide, CD19/CD20 antibodies (inebilizumab, ocrelizumab, ofatumumab, rituximab, ublituximab), dimethyl fumarate/diroximel fumarate, FcRn inhibitors (efgartigimod, rozanolixizumab), complement C5 inhibitors (eculizumab, ravulizumab, zilucoplan), interleukin-6 receptor antibodies (tocilizumab, satralizumab), intravenous immunoglobulins, long-term steroid administration, methotrexate, mitoxantrone, mycophenolate mofetil, tacrolimus, teriflunomide, tumor necrosis factor-α blockers, and sphingosine-1-phosphate receptor modulators (fingolimod, ozanimod, ponesimod, siponimod), as well as after autologous stem cell transplantation...However, the humoral and cellular vaccination response may be impaired under immunotherapy necessitating close monitoring. Here, we provide applicable recommendations to optimize immunization for individuals receiving..."

Journal • CNS Disorders • Immunology • Infectious Disease • Oncology • Transplantation • IL6R

Human in vitro neuromuscular junction model to functionally dissect the pathogenic mechanism of anti-AChR autoantibody-positive myasthenia gravis. (PubMed, BMC Pharmacol Toxicol) - "These findings offer a mechanistic rationale for the observed clinical response in patients with anti-AChR autoantibody-positive myasthenia gravis treated with C5 inhibitors. The in vitro neuromuscular junction model provides a robust platform for studying the mechanistic pathways of complement-mediated damage and evaluating therapeutic interventions for myasthenia gravis."

Journal • Preclinical • CNS Disorders • Immunology • Myasthenia Gravis

Real-world data on breakthrough hemolysis in patients with paroxysmal nocturnal hemoglobinuria treated with proximal and terminal complement inhibitors. (ASH 2025) - "Four patients were treated with factor D inhibitor danicopan(n=3) or iptacopan (n=1) concurrently with ravulizumab.A total of 49 BTH events were identified; while on eculizumab (26 events, 12 patients), ravulizumab (14events, 7 patients), ravulizumab and danicopan (6 events, 3 patients), pegcetacoplan (1 event, 1 patient),zilucoplan (2 events, 2 patients), and no events while on iptacopan. Twelve BTH events were incidentally detected during routine CIadministration; more frequent lab draws may be beneficial for patients with chronic hemolysis. Moredata is needed to understand the BTH rates of different CIs and guide clinicians in BTH management."

Clinical • Real-world • Real-world evidence • Anemia • Aplastic Anemia • Breast Cancer • Complement-mediated Rare Disorders • Infectious Disease • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Solid Tumor • CD55 • CD59 • HP

Discovery of Zilucoplan: A Complement C5 Inhibitor for Treatment of Anti-Acetylcholine Receptor (AChR) Antibody-Positive Generalized Myasthenia Gravis (gMG). (PubMed, J Med Chem) - "In Ribosome Display and Related Technologies: Methods and Protocols; Douthwaite, J. A., Jackson, R. H., Eds.; Springer: New York, 2012; pp 367-390.) against C5 and approved for the treatment of gMG. We highlight the key steps and rationale behind the peptide medicinal chemistry optimization of the initial screening hits, that led to improved potency, stability, and pharmacokinetic properties."

Journal • CNS Disorders • Myasthenia Gravis

Effectiveness and Safety of Zilucoplan Treatment for French Patients With Myasthenia Gravis Over 3 Months (clinicaltrials.gov) - P=N/A | N=55 | Recruiting | Sponsor: Centre Hospitalier Universitaire de Nice | Not yet recruiting ➔ Recruiting | Trial primary completion date: Nov 2025 ➔ Feb 2026

Enrollment open • Trial primary completion date • CNS Disorders • Myasthenia Gravis

Early real-life experience on Zilucoplan for generalized myasthenia gravis: ZILU25 multicenter observational study. (PubMed, J Neurol Sci) - "Zilucoplan was well-tolerated and effective in most patients with AChR-positive gMG. A clinical meaningful effect was reported since the first week and was sustained after 24 weeks. Further studies are needed to confirm these preliminary real-life data."

Journal • Observational data • CNS Disorders • Immunology • Myasthenia Gravis