2024
A Phase 2B, Open-Label Multicenter Study of Tebapivat (AG-946), a Potent Pyruvate Kinase Activator, in Patients with Anemia Due to Lower-Risk Myelodysplastic Syndromes
Zeidan A, Sekeres M, Al-Samkari H, Carraway H, DeZern A, Mittelman M, Little M, Beynon V, Dai X, Sommakia S, Despotovic J, Patel P, Dibacco M, Fattizzo B, Stein E, Sallman D, Kulasekararaj A, Platzbecker U, Fenaux P. A Phase 2B, Open-Label Multicenter Study of Tebapivat (AG-946), a Potent Pyruvate Kinase Activator, in Patients with Anemia Due to Lower-Risk Myelodysplastic Syndromes. Blood 2024, 144: 6708-6708. DOI: 10.1182/blood-2024-203073.Peer-Reviewed Original ResearchLower-risk MDSErythropoiesis-stimulating agentsLower-risk myelodysplastic syndromesRed blood cell unitsMyelodysplastic syndromeProportion of patientsDose levelsRed blood cellsIPSS-RTransfusion burdenOpen-labelFirst doseRevised International Prognostic Scoring SystemHistory of acute myeloid leukemiaMDS mouse modelsMedian duration of responsePhase 2bInternational Prognostic Scoring SystemOpen-label multicenter studyAssociated with increased morbidityBone marrow neoplasmsHigh transfusion burdenIPSS-R scoreLow transfusion burdenDose level 1
2023
Equitable Care for Severe Hemophilia_A: Distributional Cost-Effectiveness of Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in Patients with Severe Hemophilia_A in the United States
Ito S, Potnis K, Allen C, Richmond R, Waldron C, Viswanathan G, Bona R, Goshua G. Equitable Care for Severe Hemophilia_A: Distributional Cost-Effectiveness of Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in Patients with Severe Hemophilia_A in the United States. Blood 2023, 142: 492. DOI: 10.1182/blood-2023-190420.Peer-Reviewed Original ResearchIncremental cost-effectiveness ratioSevere hemophilia A.Quality-adjusted life expectancySevere haemophilia AHemophilia AFactor VIIIDistributional cost-effectiveness analysisHemophilia A.Chronic arthropathyOpen-label multicenter studyCoagulation factor replacementLifelong prophylactic treatmentCare of patientsCost-effectiveness ratioUSD/QALYProbabilistic sensitivity analysesCost-effectiveness analysisPackage insert informationDeterministic sensitivity analysesWeekly prophylaxisPrimary outcomeSecondary outcomesWeekly dosingFactor replacementIntracranial hemorrhage
2020
Masterkey-01: Phase I/II, open-label multicenter study to assess safety, tolerability, pharmacokinetics, and antitumor activity of BDTX-189, an inhibitor of allosteric ErbB mutations, in patients with advanced solid malignancies.
Hamilton E, Patel M, Rodon J, Hong D, Schram A, Janne P, LoRusso P, Sachdev J, Ou S, Buck E, O'Connor M, Waters N, Witt K, Cook C. Masterkey-01: Phase I/II, open-label multicenter study to assess safety, tolerability, pharmacokinetics, and antitumor activity of BDTX-189, an inhibitor of allosteric ErbB mutations, in patients with advanced solid malignancies. Journal Of Clinical Oncology 2020, 38: tps3665-tps3665. DOI: 10.1200/jco.2020.38.15_suppl.tps3665.Peer-Reviewed Original ResearchAntitumor activityStandard therapyPreliminary efficacyERBB mutationsOpen-label multicenter studySignificant unmet need existsPhase I/IIHER2 tyrosine kinase inhibitorPhase 2 doseAdvanced solid malignanciesAdequate drug exposureMetastatic solid tumorsPreliminary antitumor activityExon 20 insertionsOncogenic driver mutationsTyrosine kinase inhibitorsUnmet need existsAssessment of safetyCurrent eGFRExpansion cohortMulticenter studyPharmacodynamic effectsSolid malignanciesDrug exposureCohort 1
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