2025
Completion of Phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years
von Drygalski A, Gomez E, Giermasz A, Castaman G, Key N, Lattimore S, Leebeek F, Miesbach W, Recht M, Monahan P, Le Quellec S, Pipe S. Completion of Phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years. Blood Advances 2025 PMID: 40188458, DOI: 10.1182/bloodadvances.2024015291.Peer-Reviewed Original ResearchAdeno-associated virus serotype 5Etranacogene dezaparvovecHemophilia BRecombinant adeno-associated virus serotype 5FIX inhibitor developmentLiver-specific promoterPost-administrationSelf-administered infusionsSevere hemophilia BMulti-center trialYears post-administrationBleeding episodesOpen-labelSingle-doseSecondary endpointsIntravenous doseSingle-armGene therapyThrombotic complicationsYears post-treatmentSafety profileClinically significant elevationsAdverse eventsBleeding frequencyNeutralizing antibodies
2024
Prophylaxis with recombinant factor IX Fc fusion protein reduces the risk of bleeding and delays time to first spontaneous bleed event in previously untreated patients with haemophilia B: A post hoc analysis of the PUPs B‐LONG study
Nolan B, Recht M, Rendo P, Falk A, Foster M, Casiano S, Rauch A, Shapiro A. Prophylaxis with recombinant factor IX Fc fusion protein reduces the risk of bleeding and delays time to first spontaneous bleed event in previously untreated patients with haemophilia B: A post hoc analysis of the PUPs B‐LONG study. European Journal Of Haematology 2024, 113: 485-492. PMID: 38922990, DOI: 10.1111/ejh.14252.Peer-Reviewed Original ResearchConceptsRecombinant FIX Fc fusion proteinSpontaneous bleedingRisk of bleedingHemophilia BPost Hoc AnalysisUntreated patientsRecombinant factor IX Fc fusion proteinBaseline ageHoc AnalysisInitial treatment regimensMedian time to eventKaplan-Meier analysisSpontaneous bleeding eventsFc fusion proteinTime to eventProphylaxis typeBleeding eventsTreatment regimenTreatment regimensTreatment patternsTreat ODBleedingProphylaxisFixed replacementSevere casesEffect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE‐B trial 2 years after gene therapy
Itzler R, Buckner T, Leebeek F, Miller J, Recht M, Drelich D, Monahan P, Pipe S. Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE‐B trial 2 years after gene therapy. Haemophilia 2024, 30: 709-719. PMID: 38462823, DOI: 10.1111/hae.14977.Peer-Reviewed Original ResearchConceptsEQ-5D-5LPercentage of participantsQuality of lifeEtranacogene dezaparvovecNegatively affecting health-related quality of lifeHealth-related quality of lifeMinimal clinically important improvementGene therapyDisease-specific patientMeasures linear mixed modelsClinically important improvementEQ-5D-5L index valuesFactor IXGene transfer productsHRQoLLinear mixed modelsImportant improvementTrial participantsLS mean scoresMean scoreBleed protectionFamily planningB participantsTotal scoreHemophilia BEtranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial
Coppens M, Pipe S, Miesbach W, Astermark J, Recht M, van der Valk P, Ewenstein B, Pinachyan K, Galante N, Le Quellec S, Monahan P, Leebeek F, Investigators H, Castaman G, Crary S, Escobar M, Gomez E, Haley K, Hermans C, Kampmann P, Kazmi R, Key N, Klamroth R, Konkle B, Kruse-Jarres R, Lattimore S, Lemons R, Meijer K, O'Connell N, Quon D, Raheja P, Symington E, Verhamme P, Visweshwar N, von Drygalski A, Wang M, Wheeler A, White S, Young G. Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial. The Lancet Haematology 2024, 11: e265-e275. PMID: 38437857, DOI: 10.1016/s2352-3026(24)00006-1.Peer-Reviewed Original ResearchConceptsAnnualised bleeding rateLead-in periodFactor IX prophylaxisEtranacogene dezaparvovecPhase 3 trialGene therapyFactor IX activityAdverse eventsIX activityHemophilia BHemophilia B.Safety dataTreatment-related adverse eventsPost hoc efficacy analysisPost-hocTreatment-related deathsFactor IX expressionFull analysis setHemophilia B treatmentEffective therapeutic optionInfluenza-like illnessBleed protectionData cutoffBleeding phenotypePrimary endpoint
2023
Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy
Reiss U, Davidoff A, Tuddenham E, Chowdary P, McIntosh J, Riddell A, Pie A, Batty P, Calvert J, Mangles S, Mahlangu J, Haley K, Recht M, Shen Y, Halka K, Zhou J, Kang G, Okhomina V, Morton C, Nathwani A. Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy. Blood 2023, 142: 1056. DOI: 10.1182/blood-2023-186891.Peer-Reviewed Original ResearchAnnualized bleeding rateSelf-complementary adeno-associated virusGene therapySevere hemophilia BFactor IXFactor IX activityAdverse eventsHemophilia BAdeno-associated virus gene therapyFollow-upTransient elevation of liver transaminasesLung adenocarcinoma in situMedian annualized bleeding rateElevation of liver transaminasesTreatment-related adverse eventsHigh vector dosesYears of follow-upSustained clinical benefitMedian Follow-UpAdenocarcinoma in situAdeno-associated virusFactor IX inhibitorsFirst-in-humanLiver-specific promoterDose-dependent increaseOC 52.5 Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec in Severe/Moderately Severe Hemophilia B: A Post Hoc Responder Analysis of Participants Who Received Full Dose and Responded to Treatment
Pipe S, Leebeek F, Recht M, Key N, Lattimore S, Castaman G, Coppens M, Li Y, Monahan P, Miesbach W. OC 52.5 Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec in Severe/Moderately Severe Hemophilia B: A Post Hoc Responder Analysis of Participants Who Received Full Dose and Responded to Treatment. Research And Practice In Thrombosis And Haemostasis 2023, 7: 100448. DOI: 10.1016/j.rpth.2023.100448.Peer-Reviewed Original ResearchOC 52.1 Assessing the Safety Profile of AMT-060 and Etranacogene Dezaparvovec Gene Therapies Across Clinical Trials in People with Severe/Moderately Severe Hemophilia B
Miesbach W, Recht M, Key N, Guillen-Gonzalez R, Sivamurthy K, Monahan P, Pipe S. OC 52.1 Assessing the Safety Profile of AMT-060 and Etranacogene Dezaparvovec Gene Therapies Across Clinical Trials in People with Severe/Moderately Severe Hemophilia B. Research And Practice In Thrombosis And Haemostasis 2023, 7: 100456. DOI: 10.1016/j.rpth.2023.100456.Peer-Reviewed Original ResearchStable and durable factor IX levels in hemophilia B patients over 3 years post etranacogene dezaparvovec gene therapy
von Drygalski A, Gomez E, Giermasz A, Castaman G, Key N, Lattimore S, Leebeek F, Miesbach W, Recht M, Gut R, Dolmetsch R, Monahan P, Le Quellec S, Pipe S. Stable and durable factor IX levels in hemophilia B patients over 3 years post etranacogene dezaparvovec gene therapy. Blood Advances 2023, 7: 5671-5679. PMID: 36490302, PMCID: PMC10539871, DOI: 10.1182/bloodadvances.2022008886.Peer-Reviewed Original ResearchConceptsAdeno-associated virus serotype 5Adverse eventsEtranacogene dezaparvovecHemophilia BRecombinant adeno-associated virus serotype 5Self-limiting adverse eventsFIX inhibitor developmentSecondary end pointsFIX replacement therapyLiver-specific promoterHemophilia B patientsSelf-administered infusionsSevere hemophilia BElective surgical proceduresAMT-061Open-labelB patientsBleeding episodesSingle-doseIntravenous doseMulticenter trialReplacement therapySingle-armGene therapyClinically significant elevationsGene Therapy with Etranacogene Dezaparvovec for Hemophilia B
Pipe S, Leebeek F, Recht M, Key N, Castaman G, Miesbach W, Lattimore S, Peerlinck K, Van der Valk P, Coppens M, Kampmann P, Meijer K, O’Connell N, Pasi K, Hart D, Kazmi R, Astermark J, Hermans C, Klamroth R, Lemons R, Visweshwar N, von Drygalski A, Young G, Crary S, Escobar M, Gomez E, Kruse-Jarres R, Quon D, Symington E, Wang M, Wheeler A, Gut R, Liu Y, Dolmetsch R, Cooper D, Li Y, Goldstein B, Monahan P. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. New England Journal Of Medicine 2023, 388: 706-718. PMID: 36812434, DOI: 10.1056/nejmoa2211644.Peer-Reviewed Original ResearchConceptsAnnualized bleeding rateLead-in periodAdeno-associated virus 5Factor IX prophylaxisGene therapyHemophilia BBleeding rateEtranacogene dezaparvovecFactor IX activityMonth 7Factor IX replacementPhase 3 studyIX activityNeutralizing antibody titersFactor IX concentrateOpen-labelRate ratiosSafety profileEfficacy measuresAdverse eventsPrevent bleedingNeutralizing antibodiesNoninferiority marginNoninferiority analysisNoninferiorityDurability of bleeding protection and Factor IX activity in individuals with and without adeno-associated virus serotype 5 neutralising antibodies (Titres <1:700) in the phase 3 HOPE-B trial of etranacogene dezaparvovec gene therapy for haemophilia B
Pipe W, Leebeek W, Recht M, Key S, Lattimore S, Castaman G, Cooper D, Verweij S, Dolmetsch R, Tarrant J, Li Y, Monahan E, Miesbach W. Durability of bleeding protection and Factor IX activity in individuals with and without adeno-associated virus serotype 5 neutralising antibodies (Titres <1:700) in the phase 3 HOPE-B trial of etranacogene dezaparvovec gene therapy for haemophilia B. Hämostaseologie 2023, 43: s45-s45. DOI: 10.1055/s-0042-1760524.Peer-Reviewed Original Research
2022
Durability of Bleeding Protection and Factor IX Activity Levels Are Demonstrated in Individuals with and without Adeno-Associated Virus Serotype 5 Neutralizing Antibodies (Titers <1:700) with Comparable Safety in the Phase 3 HOPE-B Clinical Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B
Pipe S, Leebeek F, Recht M, Key N, Lattimore S, Castaman G, Cooper D, Verweij S, Dolmetsch R, Tarrant J, Li Y, Monahan P, Miesbach W. Durability of Bleeding Protection and Factor IX Activity Levels Are Demonstrated in Individuals with and without Adeno-Associated Virus Serotype 5 Neutralizing Antibodies (Titers <1:700) with Comparable Safety in the Phase 3 HOPE-B Clinical Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B. Blood 2022, 140: 4904-4906. DOI: 10.1182/blood-2022-166745.Peer-Reviewed Original ResearchAssociation of factor expression levels with health-related quality of life and direct medical costs for people with haemophilia B
Burke T, Shaikh A, Ali T, Li N, Curtis R, Diego D, Recht M, Sannie T, Skinner M, O’Hara J. Association of factor expression levels with health-related quality of life and direct medical costs for people with haemophilia B. Journal Of Medical Economics 2022, 25: 386-392. PMID: 35253589, DOI: 10.1080/13696998.2022.2049552.Peer-Reviewed Original ResearchConceptsDirect medical costsHemophilia BHealth-related quality of lifeMedical costsHealth-related qualityMedical recordsQuality of lifeGene therapy trialsFactor replacement therapyRetrospective cross-sectional analysisBody mass indexBlood-borne virusesEQ-5D questionnaireFunctional cureEQ-5D scoresConsecutive patientsTherapy trialsReplacement therapyMass indexCross-sectional analysisI-IIPatientsAssociation of increasesOffice visitsCost of care
2021
Reduced Dosing Frequency Following a Switch to Rix-FP for the Treatment of Hemophilia B: Results from the Athn 2 Study
Journeycake J, Cheng D, Chrisentery-Singleton T, Desai V, von Drygalski A, Fedor C, Hirsh N, Patel B, Raffini L, Recht M, Sidinio R, Wang M, Zhang X, Neufeld E. Reduced Dosing Frequency Following a Switch to Rix-FP for the Treatment of Hemophilia B: Results from the Athn 2 Study. Blood 2021, 138: 1039. DOI: 10.1182/blood-2021-144617.Peer-Reviewed Original ResearchEntity's Board of DirectorsExtended dosing intervalRIX-FPDosing intervalHemophilia treatment centersDosing frequencyCSL BehringProspective armRetrospective armOnce-weeklyHemophilia BHemophilia B.Half-life recombinant factorTreatment switchUS hemophilia treatment centersRange of therapeutic optionsFrequent dosing regimenPrescribed dosing frequencyTreatment of hemophilia BFactor replacement productsWeeks prior to enrollmentAmerican ThrombosisSevere hemophilia BBaseline demographic dataReduced dosing frequencyProphylaxis with rFIXFc Reduces the Frequency and Delays Time to First Spontaneous Bleed Event in Previously Untreated Patients with Hemophilia B: A Post Hoc Analysis of the PUPs B-LONG Trial
Nolan B, Recht M, Rendo P, Falk A, Foster M, Casiano S, Rauch A, Shapiro A. Prophylaxis with rFIXFc Reduces the Frequency and Delays Time to First Spontaneous Bleed Event in Previously Untreated Patients with Hemophilia B: A Post Hoc Analysis of the PUPs B-LONG Trial. Blood 2021, 138: 498. DOI: 10.1182/blood-2021-148765.Peer-Reviewed Original ResearchSpontaneous bleeding eventsEntity's Board of DirectorsBleeding eventsPost Hoc AnalysisHemophilia BYears of agePPX treatmentCSL BehringKaplan-MeierHalf-life recombinant factorTreatment of bleeding eventsPrevention of bleeding eventsHoc AnalysisSmall patient groupsFIX replacement therapyFactor IXKaplan-Meier (KM) survival analysisStandard of careAdvisory CommitteeOD therapyBleeding patternsUntreated patientsPrimary endpointOD regimensProlonged bleedingPatient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment
Witkop M, Morgan G, O'Hara J, Recht M, Buckner T, Nugent D, Curtis R, O'Mahony B, Skinner M, Mulhern B, Cawson M, Ali T, Sawyer E, Li N. Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment. Haemophilia 2021, 27: 769-782. PMID: 34310811, PMCID: PMC9290457, DOI: 10.1111/hae.14383.Peer-Reviewed Original ResearchConceptsTherapy attributesGene therapyHemophilia AHemophilia BHemophilia gene therapyFactor replacement treatmentPatient preference studiesHaemophilia typeReplacement treatmentSevere haemophiliaAnnual bleedsBleeding rateClinical trialsSide effectsHemophiliaTreatment burdenPatientsPatient preferencesBleedingTherapyDoseDaily activitiesMale adultsTreatmentPatient panelFinal results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B
Nolan B, Klukowska A, Shapiro A, Rauch A, Recht M, Ragni M, Curtin J, Gunawardena S, Mukhopadhyay S, Jayawardene D, Winding B, Fischer K, Liesner R. Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B. Blood Advances 2021, 5: 2732-2739. PMID: 34242387, PMCID: PMC8288672, DOI: 10.1182/bloodadvances.2020004085.Peer-Reviewed Original ResearchConceptsRecombinant factor IX Fc fusion proteinHemophilia BUntreated patientsBleeding episodesFamily history of inhibitorsExposure daysTreatment of bleeding episodesLow-titer inhibitorsMedian dosing intervalHigh-titer inhibitorsAverage weekly doseHistory of inhibitorsSecondary end pointsPhase 3 studyAdverse event profilePrimary end pointMedian ABRBleed resolutionOpen-labelWeekly dosesDosing intervalEvent profileAdverse eventsBleeding rateEvaluate safetyAdult lifetime cost of hemophilia B management in the US: payer and societal perspectives from a decision analytic model
Li N, Sawyer E, Maruszczyk K, Guzauskas G, Slomka M, Burke T, Martin A, O’Hara J, Stevenson M, Recht M. Adult lifetime cost of hemophilia B management in the US: payer and societal perspectives from a decision analytic model. Journal Of Medical Economics 2021, 24: 363-372. PMID: 33591884, DOI: 10.1080/13696998.2021.1891088.Peer-Reviewed Original ResearchConceptsStandard half-lifeExtended half-lifeSevere HBFactor IXHemophilia BAssociated health resource utilizationUS third-party payerFactor IX prophylaxisBleeding-related complicationsLifetime costsFixed treatmentStandard of careChronic joint damageTreatment costsSocietal perspectiveHealth resource utilizationHalf-lifeDecision-analytic modelClotting factor IXBleeding eventsTreatment paradigmTreatment optionsCongenital disorderProphylaxisLifetime horizon
2020
First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies
Pipe S, Recht M, Key N, Leebeek F, Castaman G, Lattimore S, Van Der Valk P, Peerlinck K, Coppens M, O'Connell N, Pasi J, Kampmann P, Meijer K, von Drygalski A, Young G, Hermans C, Astermark J, Klamroth R, Lemons R, Visweshwar N, Crary S, Kazmi R, Symington E, Escobar M, Gomez E, Kruse-Jarres R, Kotowski A, Quon D, Wang M, Wheeler A, Sawyer E, Verweij S, Colletta V, Bajma N, Gut R, Miesbach W. First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies. Blood 2020, 136: lba-6. DOI: 10.1182/blood-2020-143560.Peer-Reviewed Original ResearchAdeno-associated virus serotype 5Entity's Board of DirectorsEtranacogene dezaparvovecInvestigational gene therapyCSL BehringHemophilia BAdverse eventsPersonal feesGene therapyFrequent treatment-related adverse eventsClinical studiesPre-existing neutralizing antibodiesTreatment-related adverse eventsLead-InGene therapy clinical studiesPre-existing NAbsAnnualized bleeding rateGene therapy trialsInfusion-related reactionsPhase 2b studyPhase 3 studyLead-in periodUse of corticosteroidsInfluenza-like illnessLiver-specific promoterEtranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial
von Drygalski A, Giermasz A, Castaman G, Key N, Lattimore S, Leebeek F, Miesbach W, Recht M, Gomez E, Gut R, Pipe S. Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial. Blood 2020, 136: 13. DOI: 10.1182/blood-2020-139295.Peer-Reviewed Original ResearchEntity's Board of DirectorsEtranacogene dezaparvovecInvestigational gene therapyLiver-specific promoterAMT-061Gene therapyHemophilia BCSL BehringAdverse eventsNormal rangeWeek 6FIX activity levelsRecords of bleedingYears of follow-upPhase 2b trialAmeliorate disease severityYears of follow-up dataWeeks post-dosingFollow-up dataMulti-center trialNational Hemophilia FoundationAdvisory CommitteeHemophilia B subjectsSustained FIX activityFixing activityThe impact of extended half‐life factor concentrates on prophylaxis for severe hemophilia in the United States
Malec L, Cheng D, Witmer C, Jaffray J, Kouides P, Haley K, Sidonio R, Johnson K, Recht M, White G, Croteau S, Ragni M. The impact of extended half‐life factor concentrates on prophylaxis for severe hemophilia in the United States. American Journal Of Hematology 2020, 95: 960-965. PMID: 32356313, DOI: 10.1002/ajh.25844.Peer-Reviewed Original ResearchConceptsAnnualized bleeding rateHemophilia Joint Health ScoreSevere hemophilia AHemophilia treatment centersSevere hemophilia BStandard half-lifeQuality of lifeSevere haemophiliaNon-factor therapiesClinical practiceCohort of patientsJoint Health ScoreNon-factor productsHalf-lifeHemophilia AHalf-life factorHealth scoresHemophilia BBleeding rateHemophiliaProphylaxisPatientsUnited StatesEHL products
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