2024
US Clinical Practice Experience with Eculizumab in Myasthenia Gravis: Acute Clinical Events and Healthcare Resource Utilization
Nowak R, Habib A, Klink A, Muppidi S, Parthan A, Sader S, Balanean A, Gajra A, Howard J. US Clinical Practice Experience with Eculizumab in Myasthenia Gravis: Acute Clinical Events and Healthcare Resource Utilization. Drugs - Real World Outcomes 2024, 11: 593-601. PMID: 39470958, PMCID: PMC11589080, DOI: 10.1007/s40801-024-00457-8.Peer-Reviewed Original ResearchHealthcare resource utilizationAssociated healthcare resource utilizationRescue therapy useEculizumab treatmentMyasthenia gravisMG exacerbationEculizumab initiationTherapy useMethodsA retrospective chart reviewTerminal complement inhibitor eculizumabRetrospective chart review analysisRates of healthcare resource utilizationComplement inhibitor eculizumabGeneralized myasthenia gravisRetrospective chart reviewChart review analysisTreatment of patientsUS adultsEculizumab therapyAcute clinical eventsMG crisisRescue therapyChart reviewEculizumabTherapy administrationUnited States clinical practice experience with eculizumab in myasthenia gravis: symptoms, function, and immunosuppressant therapy use
Habib A, Klink A, Muppidi S, Parthan A, Sader S, Balanean A, Gajra A, Nowak R, Howard J. United States clinical practice experience with eculizumab in myasthenia gravis: symptoms, function, and immunosuppressant therapy use. Journal Of Neurology 2024, 271: 6114-6126. PMID: 39052039, PMCID: PMC11377470, DOI: 10.1007/s00415-024-12569-w.Peer-Reviewed Original ResearchMinimal symptom expression’Minimal manifestation statusGeneralized myasthenia gravisEculizumab initiationEculizumab treatmentTotal scoreMG-ADLPrednisone dosageMyasthenia gravisMethodsA retrospective chart reviewComplement C5 inhibitor eculizumabSteroid-sparing effectAssociated with sustained improvementOpen-label extensionEffectiveness of eculizumabImmunosuppressive therapy useConcomitant medication useRetrospective chart reviewNonsteroidal immunosuppressive therapyMyasthenia Gravis-ActivitiesTreatment-refractoryC5 inhibitor eculizumabDiscontinue prednisoneImmunosuppressive therapySymptom expression
2023
Safety and Efficacy of Nipocalimab in Patients With Generalized Myasthenia Gravis
Antozzi C, Guptill J, Bril V, Gamez J, Meuth S, Nowak R, Quan D, Sevilla T, Jouvin M, Jin J, Karcher K, Ramchandren S, Sun H, Ling L, Zhu Y, Arroyo S, Group F. Safety and Efficacy of Nipocalimab in Patients With Generalized Myasthenia Gravis. Neurology 2023, 102: e207937. PMID: 38165333, PMCID: PMC10962909, DOI: 10.1212/wnl.0000000000207937.Peer-Reviewed Original ResearchConceptsTreatment-emergent adverse eventsGeneralized myasthenia gravisSignificant dose responseAdverse eventsMG-ADLDay 57Myasthenia gravisDose responseMyasthenia Gravis ActivitiesPrimary safety endpointPrimary efficacy endpointSerious adverse eventsPhase 2 studyReceptor monoclonal antibodyDaily living (IADL) total scoreClinical trial registrationMG-ADL scoreDose-dependent reductionEudraCT numberQ2W groupEfficacy endpointPlacebo groupSafety endpointCare therapyReceptor autoantibodiesSubcutaneous batoclimab in generalized myasthenia gravis: Results from a Phase 2a trial with an open‐label extension
Nowak R, Breiner A, Bril V, Allen J, Khan S, Levine T, Jacobs D, Sahagian G, Siddiqi Z, Xu J, Macias W, Benatar M, Adams L, Genge A, Habib A, Hinton J, Holmlund T, Jacobs D, Lange D, Nicolle M, Phan H, Silvestri N, Small G, Yegiaian S. Subcutaneous batoclimab in generalized myasthenia gravis: Results from a Phase 2a trial with an open‐label extension. Annals Of Clinical And Translational Neurology 2023, 11: 194-206. PMID: 38062618, PMCID: PMC10791011, DOI: 10.1002/acn3.51946.Peer-Reviewed Original ResearchAnti-acetylcholine receptor antibodiesGeneralized myasthenia gravisTotal immunoglobulin GMyasthenia gravisImmunoglobulin G subclassesReceptor antibodiesOpen-label extension studyG subclassesImmunoglobulin GMyasthenia Gravis ActivitiesPatient-administered therapyPreliminary clinical benefitsQuantitative Myasthenia GravisOpen-label extensionPlacebo-controlled trialPhase 2a trialWeekly subcutaneous injectionsReceptor monoclonal antibodyMyasthenia Gravis QualityStudy support further investigationSupport further investigationMyasthenia Gravis CompositeEligible patientsPrimary endpointSecondary endpointsChange in concomitant therapies for generalized myasthenia gravis in patients receiving eculizumab: A retrospective analysis of registry data
Pulley M, Macwan S, Nowak R, Mozaffar T, Rodrigues E, Korideck H, Werneburg B, Narayanaswami P. Change in concomitant therapies for generalized myasthenia gravis in patients receiving eculizumab: A retrospective analysis of registry data. Journal Of The Neurological Sciences 2023, 455: 121092. DOI: 10.1016/j.jns.2023.121092.Peer-Reviewed Original ResearchChange in Concomitant Therapies for Generalized Myasthenia Gravis in Patients Receiving Eculizumab: a Retrospective Analysis of Registry Data (P1-5.002)
Pulley M, Macwan S, Nowak R, Mozaffar T, Rodrigues E, Korideck H, Werneburg B, Narayanaswami P. Change in Concomitant Therapies for Generalized Myasthenia Gravis in Patients Receiving Eculizumab: a Retrospective Analysis of Registry Data (P1-5.002). Neurology 2023, 100 DOI: 10.1212/wnl.0000000000202372.Peer-Reviewed Original Research
2022
Real-world experience of eculizumab treatment for generalized myasthenia gravis: initial data from a registry of patients with generalized myasthenia gravis in the USA (S25.006)
Narayanaswami P, Cutter G, Nowak R, Greene E, Muppidi S, Mozaffar T, Rodrigues E, Korideck H, Howard J. Real-world experience of eculizumab treatment for generalized myasthenia gravis: initial data from a registry of patients with generalized myasthenia gravis in the USA (S25.006). Neurology 2022, 98 DOI: 10.1212/wnl.98.18_supplement.1320.Peer-Reviewed Original ResearchGeneralized myasthenia gravisMyasthenia gravisRegistry of patientsEculizumab treatmentReal-world experiments
2021
Topline Results of a Phase 2 Study of Subcutaneous IMVT-1401 in Patients with Generalized Myasthenia Gravis (1417)
Benatar M, Breiner A, Bril V, Nowak R, Dunn I, Jacobs A. Topline Results of a Phase 2 Study of Subcutaneous IMVT-1401 in Patients with Generalized Myasthenia Gravis (1417). Neurology 2021, 96 DOI: 10.1212/wnl.96.15_supplement.1417.Peer-Reviewed Original ResearchVivacity-MG: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Nipocalimab Administered to Adults with Generalized Myasthenia Gravis (2157)
Guptill J, Antozzi C, Bril V, Gamez J, Meuth S, Blanco J, Nowak R, Quan D, Sevilla T, Szczudlik A, Hegarty B, Jouvin M, Jin J, Arroyo S. Vivacity-MG: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Nipocalimab Administered to Adults with Generalized Myasthenia Gravis (2157). Neurology 2021, 96 DOI: 10.1212/wnl.96.15_supplement.2157.Peer-Reviewed Original Research
2020
Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis
Howard JF, Nowak RJ, Wolfe GI, Freimer ML, Vu TH, Hinton JL, Benatar M, Duda PW, MacDougall JE, Farzaneh-Far R, Kaminski HJ, Barohn R, Dimachkie M, Pasnoor M, Farmakidis C, Liu T, Colgan S, Benatar M, Bertorini T, Pillai R, Henegar R, Bromberg M, Gibson S, Janecki T, Freimer M, Elsheikh B, Matisak P, Genge A, Guidon A, David W, Habib A, Mathew V, Mozaffar T, Hinton J, Hewitt W, Barnett D, Sullivan P, Ho D, Howard J, Traub R, Chopra M, Kaminski H, Aly R, Bayat E, Abu-Rub M, Khan S, Lange D, Holzberg S, Khatri B, Lindman E, Olapo T, Sershon L, Lisak R, Bernitsas E, Jia K, Malik R, Lewis-Collins T, Nicolle M, Nowak R, Sharma A, Roy B, Nye J, Pulley M, Berger A, Shabbir Y, Sachdev A, Patterson K, Siddiqi Z, Sivak M, Bratton J, Small G, Kohli A, Fetter M, Vu T, Lam L, Harvey B, Wolfe G, Silvestri N, Patrick K, Zakalik K, Duda P, MacDougall J, Farzaneh-Far R, Pontius A, Hoarty M. Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis. JAMA Neurology 2020, 77: 582-592. PMID: 32065623, PMCID: PMC7042797, DOI: 10.1001/jamaneurol.2019.5125.Peer-Reviewed Original ResearchConceptsGeneralized myasthenia gravisSecondary end pointsDaily living scoreTolerability profileEnd pointLiving scoreMyasthenia gravisClinical effectsPlacebo-controlled phase 2 clinical trialKey secondary efficacy end pointsKey secondary end pointSecondary efficacy end pointsSevere Generalized Myasthenia GravisQuantitative Myasthenia Gravis ScorePhase 2 clinical trialAcetylcholine receptor autoantibodiesEfficacy end pointMyasthenia gravis scoreMG activityComplement component 5Disease-specific variablesQuality of lifeComplete complement inhibitionBroader populationDaily SC