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- || Zilbrysq (zilucoplan) / UCB
Enrollment closed: A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) - Jan 15, 2025
P3, N=31, Active, not recruiting, Sponsor: UCB Biopharma SRL
Recruiting --> Active, not recruiting
- | Journal, Benefit-risk assessment: Risk-Benefit Analysis of Novel Treatments for Patients with Generalized Myasthenia Gravis. (Pubmed Central) - Nov 13, 2024
FcRn inhibitors and complement inhibitors assessed in this study all demonstrated clinical benefit in terms of NNT as well as an acceptable safety profile in terms of NNH. Within the limitations of this meta-analysis, efgartigimod was associated with a favorable benefit-risk profile as well as a better economic value compared to ravulizumab, rozanolixizumab, and zilucoplan as treatments for anti-AChR Ab+
- Patient Characteristics Associated With Treatment Preference for Generalized Myasthenia Gravis (gMG): A Multivariate Analysis () - Nov 4, 2024 - Abstract #ISPOREU2024ISPOR_EU_1346;
Within the limitations of this meta-analysis, efgartigimod was associated with a favorable benefit-risk profile as well as a better economic value compared to ravulizumab, rozanolixizumab, and zilucoplan as treatments for anti-AChR Ab+ US adults self-report ing a physician diagnosis of AChR -Ab+ gMG completed a web-based , best
- || Zilbrysq (zilucoplan) / UCB
Trial completion: An Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Zilucoplan in Participants With Generalized Myasthenia Gravis Who Were Previously Receiving Intravenous Complement Component 5 Inhibitors (clinicaltrials.gov) - Oct 29, 2024
P3, N=26, Completed, Sponsor: UCB Biopharma SRL
US adults self-report ing a physician diagnosis of AChR -Ab+ gMG completed a web-based , best Active, not recruiting --> Completed
- || Review, Journal: Targeted Treatments for Myasthenia Gravis in Children and Adolescents. (Pubmed Central) - Oct 28, 2024
Recently, there have been several RCTs focussing on novel therapeutic strategies with potentially promising outcomes, suggesting a change in MG management over the coming years and access to more effective and faster-acting drugs for MG patients. This paper will review current and new MG treatments including efgartigimod, eculizumab, rozanolixizumab, ravulizumab, and zilucoplan, with a focus on juvenile myasthenia gravis.
- | Zilbrysq (zilucoplan) / UCB
Journal: Usability of the zilucoplan prefilled syringe for the treatment of generalized myasthenia gravis: insights from a human factors validation study. (Pubmed Central) - Oct 15, 2024
The rate of use errors was low, with pass rates for observation-based tasks ranging from 85.3% to 100.0%, and in general, after participants were instructed to refer to the IFU, the rate of use errors was reduced. This human factors study demonstrated that the zilucoplan PFS is safe and effective for intended users, including patients with gMG and their caregivers.
- || Zilbrysq (zilucoplan) / UCB
Enrollment open: A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) - Sep 20, 2024
P3, N=25, Recruiting, Sponsor: UCB Biopharma SRL
This human factors study demonstrated that the zilucoplan PFS is safe and effective for intended users, including patients with gMG and their caregivers. Not yet recruiting --> Recruiting
- || Zilbrysq (zilucoplan) / UCB
Enrollment closed: A Study to Evaluate Zilucoplan Injected Subcutaneously Either by a Prefilled Syringe or an Auto-injector in Healthy Adult Participants (clinicaltrials.gov) - Sep 20, 2024
P1, N=14, Active, not recruiting, Sponsor: UCB Biopharma SRL
Not yet recruiting --> Recruiting Recruiting --> Active, not recruiting
- || Zilbrysq (zilucoplan) / UCB
Enrollment open: A Study to Evaluate Zilucoplan Injected Subcutaneously Either by a Prefilled Syringe or an Auto-injector in Healthy Adult Participants (clinicaltrials.gov) - Aug 15, 2024
P1, N=14, Recruiting, Sponsor: UCB Biopharma SRL
Recruiting --> Active, not recruiting Not yet recruiting --> Recruiting
- || Zilbrysq (zilucoplan) / UCB
New P1 trial: A Study to Evaluate Zilucoplan Injected Subcutaneously Either by a Prefilled Syringe or an Auto-injector in Healthy Adult Participants (clinicaltrials.gov) - Jul 22, 2024
P1, N=14, Not yet recruiting, Sponsor: UCB Biopharma SRL
- | Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
Journal: Efgartigimod as Rescue Medication in a Patient with Therapy-Refractory Myasthenic Crisis. (Pubmed Central) - Jun 28, 2024
They mainly consist of complement C5 inhibitors such as eculizumab and ravulizumab and neonatal Fc receptor antagonists such as efgartigimod with the approval of more options pending, e.g., zilucoplan and rozanolixizumab...He was diagnosed with myasthenic crisis and treated with intravenous pyridostigmine, plasmapheresis (PLEX), and continued prednisone...The patient continued to improve and was stable enough for transfer to a rehabilitation facility. This case illustrates the potential of efgartigimod as a novel treatment for refractory myasthenic crises.
- |||| Zilbrysq (zilucoplan) / UCB
New P3 trial: A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) - Jun 24, 2024
P3, N=25, Not yet recruiting, Sponsor: UCB Biopharma SRL
- | Zilbrysq (zilucoplan) / UCB
New P3 trial: ziMyG+: An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) - May 29, 2024
P3, N=8, Not yet recruiting, Sponsor: UCB Biopharma SRL
- | Zilbrysq (zilucoplan) / UCB
Trial completion date: An Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Zilucoplan in Participants With Generalized Myasthenia Gravis Who Were Previously Receiving Intravenous Complement Component 5 Inhibitors (clinicaltrials.gov) - May 15, 2024
P3, N=26, Active, not recruiting, Sponsor: UCB Biopharma SRL
This case illustrates the potential of efgartigimod as a novel treatment for refractory myasthenic crises. Trial completion date: Jul 2024 --> Nov 2024
- | Enrollment closed: HEALEY ALS Platform Trial - Master Protocol (clinicaltrials.gov) - May 14, 2024
P2/3, N=1500, Active, not recruiting, Sponsor: Merit E. Cudkowicz, MD
Trial completion date: Jul 2024 --> Nov 2024 Recruiting --> Active, not recruiting
- | Zilbrysq (zilucoplan) / UCB
Journal: Long-term safety and efficacy of zilucoplan in patients with generalized myasthenia gravis: interim analysis of the RAISE-XT open-label extension study. (Pubmed Central) - Apr 19, 2024
P3
Additional long-term data will be available in future analyses. ClinicalTrials.gov identifier: NCT04225871 (https://clinicaltrials.gov/ct2/show/NCT04225871).
- || Empaveli (pegcetacoplan SC) / Apellis, SOBI
Review, Journal: Complement inhibition in paroxysmal nocturnal hemoglobinuria: From biology to therapy. (Pubmed Central) - Apr 16, 2024
Upstream inhibition of complement cascade was also explored with the anti-C3 pegcetacoplan, and with the alternative pathway inhibitors iptacopan (anti-factor B) and danicopan (anti-factor D)...Additionally, both anti-C5 and upstream inhibitors do not resolve the unmet need of pharmacodynamic BTH events due to complement amplifying conditions such as infections, traumas, and surgery. In this review, we will recapitulate PNH physiopathology, clinical presentation, and diagnosis and describe available and developing drugs that will lead to a precision medicine approach for this rare though heterogenous disease.
- | Zilbrysq (zilucoplan) / UCB
Journal: Zilucoplan (Zilbrysq) for myasthenia gravis. (Pubmed Central) - Apr 9, 2024
In this review, we will recapitulate PNH physiopathology, clinical presentation, and diagnosis and describe available and developing drugs that will lead to a precision medicine approach for this rare though heterogenous disease. No abstract available
- ||||| Zilbrysq (zilucoplan) / UCB
Enrollment change, Trial completion date: An Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Zilucoplan in Participants With Generalized Myasthenia Gravis Who Were Previously Receiving Intravenous Complement Component 5 Inhibitors (clinicaltrials.gov) - Mar 26, 2024
P3, N=26, Active, not recruiting, Sponsor: UCB Biopharma SRL
No abstract available N=20 --> 26 | Trial completion date: Mar 2024 --> Jul 2024
- Zilbrysq (zilucoplan) / UCB
Enrollment open: A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) - Mar 12, 2024
P2/3, N=8, Recruiting, Sponsor: UCB Biopharma SRL
N=20 --> 26 | Trial completion date: Mar 2024 --> Jul 2024 Not yet recruiting --> Recruiting
- Evidence Gap Analysis of the Burden of Illness and Treatment of Myasthenia Gravis () - Mar 8, 2024 - Abstract #ISPOR2024ISPOR_2036;
Besides traditional non-specific immunosuppressive agents, recently available biologics (e.g., eculizumab, ravulizumab, efgartigimod, rozanolixizumab, zilucoplan) are increasingly used in MG to target specific components of the immune pathway. We identified several gaps in the literature, including the need for comprehensive studies to advance our understanding of the epidemiology, humanistic and economic burden, as well as the evolving treatment pathways in MG.
- Zilbrysq (zilucoplan) / UCB
Safety, Efficacy, and Patient Preference for Subcutaneous Zilucoplan in Myasthenia Gravis After Switching from Intravenous Complement Component 5 Inhibitors: An Interim Analysis of a Phase 3b Study (Colorado Convention Center | Exhibit Hall B-E) - Mar 8, 2024 - Abstract #AAN2024AAN_4246;
P3
In this interim analysis, switching from IV C5 inhibitors to SC zilucoplan did not raise any safety concerns. After 12 weeks, data showed trends towards higher treatment satisfaction, symptom improvement, and preference for zilucoplan over IV C5 inhibitors.
- Zilbrysq (zilucoplan) / UCB
Long-term Safety and Efficacy of Zilucoplan in Myasthenia Gravis: Additional Interim Analyses of RAISE-XT (Colorado Convention Center | Bluebird 2E-H) - Mar 8, 2024 - Abstract #AAN2024AAN_2887;
P2, P3
In this interim analysis of RAISE-XT, zilucoplan demonstrated a favorable long-term safety profile. Efficacy was sustained through to Week 96 in patients who had previously received zilucoplan and was demonstrated in those who switched from placebo.
- | Rystiggo (rozanolixizumab) / UCB
Journal: Rozanolixizumab (Rystiggo) for myasthenia gravis. (Pubmed Central) - Mar 2, 2024
Efficacy was sustained through to Week 96 in patients who had previously received zilucoplan and was demonstrated in those who switched from placebo. No abstract available
- | Zilbrysq (zilucoplan) / UCB
Journal: Improvement of fatigue in generalised myasthenia gravis with zilucoplan. (Pubmed Central) - Feb 24, 2024
P3
No abstract available Treatment with zilucoplan demonstrated statistical and clinically meaningful improvements in fatigue scores and severity versus placebo during RAISE, which were sustained to Week 60 in RAISE-XT.
- || Review, Journal: 2023 FDA TIDES (Peptides and Oligonucleotides) Harvest. (Pubmed Central) - Feb 24, 2024
Furthermore, Rett syndrome has found its first-ever core symptoms treatment, which is also peptide-based. Here, we analyze the TIDES approved in 2023 on the basis of their chemical structure, medical target, mode of action, administration route, and common adverse effects.
- || Clinical, Retrospective data, Review: The Efficacy and Safety of Different Targeted Drugs for the Treatment of Generalized Myasthenia Gravis: A Systematic Review and Bayesian Network Meta-analysis. (Pubmed Central) - Feb 22, 2024
Eculizumab was ranked second, and nipocalimab had the worst efficacy. With the exception of batoclimab,
- || Review, Journal: Myasthenia Gravis Treatment: From Old Drugs to Innovative Therapies with a Glimpse into the Future. (Pubmed Central) - Jan 29, 2024
The aim of this review is to provide a detailed overview of the pre- and post-marketing evidence on the five pharmacological treatments most recently approved for the treatment of MG, by identifying both preclinical and clinical studies registered in clinicaltrials.gov. A description of the molecules currently under evaluation for the treatment of MG is also provided.
- Zilbrysq (zilucoplan) / UCB
Trial initiation date: A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) - Nov 27, 2023
P2/3, N=8, Not yet recruiting, Sponsor: UCB Biopharma SRL
A description of the molecules currently under evaluation for the treatment of MG is also provided. Initiation date: Sep 2023 --> Jan 2024
- | Journal: What Is in the Neuromuscular Junction Literature? (Pubmed Central) - Nov 18, 2023
The notion of using fast-acting therapies early in the course of MG is addressed. The last sections center on MG and Lambert-Eaton myasthenic syndrome as a consequence of immune checkpoint inhibitor therapy.
- Zilbrysq (zilucoplan) / UCB
Phase classification: An Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Zilucoplan in Participants With Generalized Myasthenia Gravis Who Were Previously Receiving Intravenous Complement Component 5 Inhibitors (clinicaltrials.gov) - Nov 13, 2023
P3, N=20, Active, not recruiting, Sponsor: UCB Biopharma SRL
The last sections center on MG and Lambert-Eaton myasthenic syndrome as a consequence of immune checkpoint inhibitor therapy. Phase classification: P3b --> P3
- || Zilbrysq (zilucoplan) / UCB
Enrollment closed: An Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Zilucoplan in Participants With Generalized Myasthenia Gravis Who Were Previously Receiving Intravenous Complement Component 5 Inhibitors (clinicaltrials.gov) - Nov 1, 2023
P3b, N=20, Active, not recruiting, Sponsor: UCB Biopharma SRL
Phase classification: P3b --> P3 Recruiting --> Active, not recruiting
- || Soliris (eculizumab) / AstraZeneca, Ultomiris IV (ravulizumab-cwvz IV) / AstraZeneca, Zilbrysq (zilucoplan) / UCB
Review, Journal: Role of complement in myasthenia gravis. (Pubmed Central) - Oct 24, 2023
Complement therapies were initially studied and approved for paroxysmal nocturnal hemoglobinuria and in the past decade, those have also been studied in myasthenia gravis. The currently available randomized control trial and real-world data on the efficacy and safety of the approved and investigational complement therapies are summarized in this review.
- | Zilbrysq (zilucoplan) / UCB
New P2/3 trial: A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) - Sep 28, 2023
P2/3, N=8, Not yet recruiting, Sponsor: UCB Biopharma SRL
- | Zilbrysq (zilucoplan) / UCB
Enrollment open: A first in human study assessing the safety and pharmacokinetics (blood levels) of RA101495. (EUDRACT) - Sep 4, 2023
P1, N=40, Recruiting, Sponsor: Ra Pharmaceuticals, Inc.
The currently available randomized control trial and real-world data on the efficacy and safety of the approved and investigational complement therapies are summarized in this review. Completed --> Recruiting
- | zilucoplan (RA101495) / UCB
Journal: Zilucoplan, a macrocyclic peptide inhibitor of human complement component 5, uses a dual mode of action to prevent terminal complement pathway activation. (Pubmed Central) - Aug 29, 2023
Our findings demonstrate that zilucoplan uses a dual mode of action to potently inhibit the activation of C5 and terminal complement pathway including wild-type and clinical R885 variants that do not respond to eculizumab treatment. These data may be relevant to the clinically demonstrated benefits of zilucoplan.
- | Journal: Advancements in Targeted Therapies for Generalized Acetylcholine Receptor Antibody Positive Myasthenia Gravis: Beginnings of a Paradigm Shift. (Pubmed Central) - Aug 14, 2023
Compared to traditional immune modulatory therapies (steroids, non-steroidal immunosuppressive agents), these agents have more specific target engagement via inhibition of complement activation (eculizumab, ravulizumab, zilucoplan) or blockade of IgG recycling via the neonatal Fc receptor (FcRn; efgartigimod, rozanolixizumab)...B-cell targeting with rituximab was safe and well-tolerated but with less impressive efficacy in two phase 2 studies...A paradigm shift is afoot. The challenge is figuring out where these therapies may fit in gMG treatment algorithms.
- || zilucoplan (RA101495) / UCB
P2 data, Journal: Phase II trials of zilucoplan in paroxysmal nocturnal hemoglobinuria. (Pubmed Central) - Aug 3, 2023
The challenge is figuring out where these therapies may fit in gMG treatment algorithms. Not available.
- ||||| Zilbrysq (zilucoplan) / UCB
Trial completion: HEALEY ALS Platform Trial - Regimen A Zilucoplan (clinicaltrials.gov) - Jul 27, 2023
P2/3, N=162, Completed, Sponsor: Merit E. Cudkowicz, MD
Not available. Active, not recruiting --> Completed
- || Retrospective data, Review, Journal: Efficacy of Innovative Therapies in Myasthenia Gravis: Systematic Review, Meta-Analysis and Network Meta-Analysis. (Pubmed Central) - May 19, 2023
With the limitations of this meta-analysis, including efficacy time-points, FcRn treatments showed a short-term higher effect on the QMG. Real-life studies with long-term measurements are needed to confirm our results.
- | Trial completion date, Trial primary completion date: HEALEY ALS Platform Trial - Master Protocol (clinicaltrials.gov) - May 16, 2023
P2/3, N=1500, Recruiting, Sponsor: Merit E. Cudkowicz, MD
Real-life studies with long-term measurements are needed to confirm our results. Trial completion date: Dec 2025 --> Apr 2026 | Trial primary completion date: Apr 2025 --> Jul 2025
- ||| Zilbrysq (zilucoplan) / UCB
Trial completion date, Trial primary completion date: RAISE-XT: Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis (clinicaltrials.gov) - May 10, 2023
P3, N=200, Active, not recruiting, Sponsor: Ra Pharmaceuticals, Inc.
Trial completion date: Dec 2025 --> Apr 2026 | Trial primary completion date: Apr 2025 --> Jul 2025 Trial completion date: Apr 2024 --> Jun 2026 | Trial primary completion date: Apr 2024 --> Jun 2026
- zilucoplan (RA101495) / UCB
Response to zilucoplan in the Phase 3 RAISE study in patients with generalised myasthenia gravis (A5) - Apr 21, 2023 - Abstract #EAN2023EAN_1650;
- zilucoplan (RA101495) / UCB
Long-term safety, efficacy & self-injection satisfaction with zilucoplan in myasthenia gravis: RAISE-XT interim analysis (A4) - Apr 21, 2023 - Abstract #EAN2023EAN_1537;
- ||| zilucoplan (RA101495) / UCB
P3 data, Journal: Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study. (Pubmed Central) - Apr 19, 2023
P3
Zilucoplan is a new potential treatment option for a broad population of patients with AChR-positive generalised myasthenia gravis. The long-term safety and efficacy of zilucoplan is being assessed in an ongoing open-label extension study.
- | Enrollment change, Trial completion date, Trial primary completion date: HEALEY ALS Platform Trial - Master Protocol (clinicaltrials.gov) - Apr 10, 2023
P2/3, N=1200, Recruiting, Sponsor: Merit E. Cudkowicz, MD
The long-term safety and efficacy of zilucoplan is being assessed in an ongoing open-label extension study. N=800 --> 1200 | Trial completion date: Dec 2023 --> Dec 2025 | Trial primary completion date: Apr 2023 --> Apr 2025
- zilucoplan (RA101495) / UCB
Efficacy and Safety of Zilucoplan for the Treatment of COVID-19 in Hospitalized Patients: Part of the COMMUNITY Platform Trial (Walter E. Washington Convention Center, Area H, Hall C (Lower Level)) - Mar 25, 2023 - Abstract #ATS2023ATS_4921;
P3
Early termination of the sub-protocol COV-01-005 meant that the planned statistical analysis was not conducted, and no conclusions on efficacy can be drawn. However, the safety data appear to support the favorable safety and good tolerability profile of zilucoplan at the administered dose.
- zilucoplan (RA101495) / UCB
RAISE-XT: An interim analysis of safety and efficacy in an open-label extension study of zilucoplan in patients with myasthenia gravis (Both in-person and online) - Mar 12, 2023 - Abstract #AAN2023AAN_1226;
P2, P3
Responder rates improved from extension study baseline in both the zilucoplan and placebo-switch groups. Funding: UCB Pharma.
- zilucoplan (RA101495) / UCB
Subgroup outcomes from RAISE: A randomized, Phase 3 trial of zilucoplan in generalized myasthenia gravis (Both in-person and online) - Mar 12, 2023 - Abstract #AAN2023AAN_1225;
P3
Conclusions Daily subcutaneous zilucoplan demonstrated consistent improvements in MG-specific efficacy outcomes irrespective of disease severity or duration. Funding: UCB Pharma.
- |||||| zilucoplan (RA101495) / UCB
Clinical: NEW RESEARCH—A phase 2 multicentre RCT of zilucoplan shows that C5 inhibition does not appear to be an efficacious treatment modality for people with immune-mediated necrotising myopathy https://t.co/aTmoFFu2W5 @mzdimachkie @drhectorchinoy @DrIagoPinal (Twitter) - Jan 25, 2023