Zilbrysq (zilucoplan)

To treat generalized myasthenia gravis in adults who are anti-acetylcholine receptor (AChR) antibody positive Drug Trials Snapshot

FDA Approval: 10/17/2023

Research Synopsis

Related articles

Research articles about Zilbrysq (zilucoplan)

Zilbrysq (zilucoplan)

Therapies Directed Against B-Cells and Downstream Effectors in Generalized Autoimmune Myasthenia Gravis: Current Status.

London, UK

2 hours ago

1 Received

  • Myasthenia gravis is an autoimmune disease that causes muscle weakness due to autoantibodies targeting the neuromuscular junction, affecting various muscle-related proteins.
  • The disease can be categorized based on factors like age of onset, weakness patterns, autoantibody types, and thymic issues, with most patients benefiting from immunosuppressive therapies, though 15% remain difficult to treat.
  • Recent advancements in targeted B-cell therapies, including rituximab and eculizumab, have shown promise, though some studies have yielded mixed results, highlighting the need for further research and effective treatments.

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ACCORD: A Multicentre, Seamless, Phase 2 Adaptive Randomisation Platform Study to Assess the Efficacy and Safety of Multiple Candidate Agents for the Treatment of COVID-19 in Hospitalised Patients: A structured summary of a study protocol for a randomised controlled trial.

London, UK

2 hours ago

1 Received

  • The study aims to assess the safety and effectiveness of new treatment options as additional therapies for hospitalized COVID-19 patients, divided into two stages: initial screening and later confirmation of efficacy.
  • ACCORD is a flexible Phase 2 clinical trial designed to quickly evaluate various candidate agents (like bemcentinib and acalabrutinib) using a master protocol, with equal randomization and rollovers based on Stage 1 results.
  • Participants are adult COVID-19 patients in the UK, ranging from mild hospital conditions to more severe cases, and the trial will recruit a specific number of patients tailored to the results obtained in each stage.

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Progress in the therapy of myasthenia gravis: getting closer to effective targeted immunotherapies.

London, UK

2 hours ago

1 Received

  • This review updates the current strategies in immunomodulating and immunosuppressive therapies for myasthenia gravis and highlights new biologics that are either approved or on the verge of approval.
  • Recent findings include that preoperative IVIg is unnecessary for stable myasthenia gravis patients, early Rituximab treatment can lead to sustained remission in many cases, and new biologics like Zilucoplan and Efgartigimod show promise in trials.
  • The emerging therapies focus on targeted treatments aimed at specific immune pathways, indicating a positive shift towards effective management of myasthenia gravis and related neurological diseases.*

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Zilucoplan in patients with acute hypoxic respiratory failure due to COVID-19 (ZILU-COV): A structured summary of a study protocol for a randomised controlled trial.

London, UK

2 hours ago

1 Received

  • Zilucoplan is being tested for its ability to inhibit severe lung injury and enhance recovery in COVID-19 patients with breathing difficulties, focusing on improving oxygen levels.
  • The study is a phase 2 trial that is randomized and involves multiple centers in Belgium, enrolling adults who meet specific COVID-19 related criteria.
  • Participants will receive Zilucoplan alongside antibiotics, with the aim of assessing both the treatment's safety and its effectiveness in improving patients' short- and long-term health outcomes.

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Chemical synthesis and characterisation of the complement C5 inhibitory peptide zilucoplan.

London, UK

2 hours ago

1 Received

  • Zilucoplan, a peptide that inhibits the complement component C5, is in phase III clinical trials for treating myasthenia gravis (MG).
  • The chemical synthesis of zilucoplan has not been documented in published literature until now.
  • The study outlines the synthesis method and confirms that the produced zilucoplan effectively blocks C5a production induced by lipopolysaccharides in human blood.

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Complement Inhibitor Therapy for Myasthenia Gravis.

London, UK

2 hours ago

1 Received

  • - Complement activation plays a major role in the pathology of myasthenia gravis (MG), particularly in patients with acetylcholine receptor antibody-positive MG, where the damaging membrane attack complex (MAC) is found at neuromuscular junctions.
  • - Research shows that animals with experimental MG rely on an active complement system for developing muscle weakness, and mice lacking specific complement regulatory proteins face increased destruction at neuromuscular junctions.
  • - Eculizumab, a drug targeting the C5 component of complement, has proven effective in clinical trials, leading to its FDA approval for treating acetylcholine receptor antibody-positive MG, while newer complement inhibitors are being developed and tested for efficacy.

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Zilucoplan: An Investigational Complement C5 Inhibitor for the Treatment of Acetylcholine Receptor Autoantibody-Positive Generalized Myasthenia Gravis.

London, UK

2 hours ago

1 Received

  • * Research is focusing on the complement cascade's role in gMG, leading to the investigation of a new treatment called zilucoplan, which specifically inhibits a component of the complement system to reduce muscle weakness.
  • * In clinical trials, zilucoplan has shown promising results in inhibiting complement activity, indicating potential as a new treatment option for patients with gMG, subject to further phase 3 studies for efficacy and safety.

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Quantitative evaluation of drug efficacy in the treatment of myasthenia gravis.

London, UK

2 hours ago

1 Received

  • The study evaluated the effects of drugs and placebo on myasthenia gravis (MG) patients, focusing on quantitative myasthenia gravis score (QMGs) and MG activities of daily living score (MG-ADLs).
  • It included data from 12 articles and 13 trials involving 673 participants, assessing various immunosuppressants and targeted therapies.
  • Results showed that eculizumab was the most effective in reducing QMGs, while efgartigimod excelled in lowering MG-ADLs, and the placebo effect strengthened over time alongside improved daily living abilities in patients who had thymectomy.

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Pharmacotherapy of Generalized Myasthenia Gravis with Special Emphasis on Newer Biologicals.

London, UK

2 hours ago

1 Received

  • - Myasthenia gravis (MG) is an autoimmune disorder affecting muscle function, with treatments traditionally relying on acetylcholinesterase inhibitors and corticosteroids, though newer therapies are emerging.
  • - Conventional treatments can be slow to act and have significant side effects, whereas new biologic agents like eculizumab and efgartigimod offer faster, targeted immunotherapy with fewer adverse effects.
  • - Ongoing research includes various agents targeting different mechanisms, but there are concerns like the risk of Neisseria meningitis with complement inhibitors, emphasizing the need for vaccinations.

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Paroxysmal nocturnal hemoglobinuria: advances in the understanding of pathophysiology, diagnosis, and treatment.

London, UK

2 hours ago

1 Received

  • Recent advancements in the understanding of paroxysmal nocturnal hemoglobinuria (PNH) have improved diagnostics and therapies, focusing on the unique survival of PNH stem cells against cell death.
  • Changes in immune-related proteins (cytokines and chemokines) among PNH patients suggest a link to autoimmune processes and cell death mechanisms.
  • The review discusses current diagnostic methods, treatment options (like C5 inhibitors and stem cell transplantation), and introduces new experimental drugs, emphasizing the significance of tailored treatment plans for better disease outcomes.

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Role of complement, anti-complement therapeutics, and other targeted immunotherapies in myasthenia gravis.

London, UK

2 hours ago

1 Received

  • Patients with myasthenia gravis (MG) often struggle with current treatments, highlighting the need for new therapies.
  • The paper focuses on emerging target-specific immunotherapies, particularly anti-complement treatments and monoclonal antibodies that target FcRn and B cells, showing promise for long-term remission.
  • Expert opinion suggests that these therapies, including Eculizumab and Rituximab, can effectively reduce harmful antibodies and improve patient outcomes, potentially revolutionizing MG treatment strategies.

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Efficacy and safety of the investigational complement C5 inhibitor zilucoplan in patients hospitalized with COVID-19: an open-label randomized controlled trial.

London, UK

2 hours ago

1 Received

  • A multicenter randomized controlled trial evaluated the efficacy and safety of the C5 inhibitor zilucoplan in hospitalized COVID-19 patients with systemic inflammation and hypoxemia.
  • Out of 81 participants, those receiving zilucoplan showed greater improvement in oxygen levels compared to the control group, with a mortality rate of 9% versus 21% at day 28.
  • Although the results were not statistically significant, findings suggest potential benefits of C5 inhibition for respiratory function and clinical outcomes, warranting further investigation in larger studies.

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Prevention of Anti-HMGCR Immune-Mediated Necrotising Myopathy by C5 Complement Inhibition in a Humanised Mouse Model.

London, UK

2 hours ago

1 Received

  • Immune-mediated necrotising myopathy (IMNM) is linked to specific antibodies that activate the complement system, prompting muscle damage.
  • Zilucoplan, a potential treatment that inhibits complement component C5, was tested in mouse models to assess its effectiveness in preventing muscle strength loss caused by IMNM.
  • Results showed that early use of zilucoplan significantly prevented muscle damage and strength loss in specific mouse strains, whereas its effectiveness was less pronounced when given after the onset of the disease.

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New immune horizons in therapeutics and diagnostic approaches to Preeclampsia.

London, UK

2 hours ago

1 Received

  • Hypertensive disorders of pregnancy (HDP) affect 5%-10% of pregnancies globally, with four main types identified, including preeclampsia (PE), which significantly contributes to maternal and fetal deaths worldwide.
  • Preeclampsia is a complex disorder usually diagnosed after 20 weeks of pregnancy and is linked to issues in the immune system and inadequate blood flow to the placenta, causing various complications.
  • Current research suggests that targeting the immune system with specific medications could provide new treatment options for preeclampsia, as the immune dysfunction plays a key role in its development.

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Zilucoplan in immune-mediated necrotising myopathy: a phase 2, randomised, double-blind, placebo-controlled, multicentre trial.

London, UK

2 hours ago

1 Received

  • Immune-mediated necrotizing myopathy (IMNM) is an autoimmune condition causing muscle weakness and high creatine kinase levels, characterized by specific autoantibodies, with no approved treatments currently available.
  • A study named IMNM01 was conducted to evaluate the drug zilucoplan, targeting complement C5, as a potential treatment for adults with anti-HMGCR or anti-SRP positive IMNM.
  • Results showed no significant difference in muscle enzyme levels after eight weeks of treatment with zilucoplan compared to placebo, indicating that the drug did not lead to clinically meaningful improvements.

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Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study.

London, UK

2 hours ago

1 Received

  • Generalised myasthenia gravis is a rare and difficult-to-manage disease that has a high treatment burden, highlighting the need for better therapies; zilucoplan, a self-administered treatment, was tested in this context.
  • The RAISE trial was a phase 3 study conducted across 75 sites in multiple countries, comparing zilucoplan with a placebo in patients diagnosed with AChR-positive myasthenia gravis over a 12-week period.
  • Results indicated that patients receiving zilucoplan experienced a significant reduction in their MG-ADL scores compared to those on placebo, suggesting it may be an effective treatment option.

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Corrigendum: Zilucoplan, a macrocyclic peptide inhibitor of human complement component 5, uses a dual mode of action to prevent terminal complement pathway activation.

London, UK

2 hours ago

1 Received

  • The article DOI: 10.3389/fimmu.2023.1213920 has been updated to correct previous information.
  • The correction addresses inaccuracies in data or findings presented in the original publication.
  • Readers are encouraged to refer to the updated version for accurate and reliable information.

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Zilucoplan, a macrocyclic peptide inhibitor of human complement component 5, uses a dual mode of action to prevent terminal complement pathway activation.

London, UK

2 hours ago

1 Received

  • The complement system plays a crucial role in the immune response, and its abnormal activation can lead to various diseases; zilucoplan, a macrocyclic peptide, effectively inhibits the activation of complement component C5.
  • Research methods used to study zilucoplan's effectiveness included surface plasmon resonance, hemolysis assays, and ELISA to analyze its binding properties and impact on C5 activation, particularly for clinical variants with R885 polymorphisms.
  • Results showed that zilucoplan binds strongly to C5, prevents its cleavage and the formation of the cytolytic membrane attack complex, and works effectively against C5 variants that do not respond to other treatments, indicating its potential for rapid clinical application.

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Role of complement in myasthenia gravis.

London, UK

2 hours ago

1 Received

  • * Patients often experience varying degrees of muscle weakness that can become severe and life-threatening if it affects muscles necessary for breathing and swallowing.
  • * The article discusses how complement system involvement contributes to the damage at the neuromuscular junction and reviews current therapies targeting this pathway, highlighting their efficacy and safety based on recent studies.

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