Pralsetinib is a highly potent, selective RET inhibitor targeting oncogenic
ARROW (75 sites in 11 countries; NCT03037385) consists of a phase I dose escalation to establish the recommended phase II dose (400 mg once daily [QD] orally), and phase II expansion cohorts defined by tumour type and/or RET alteration. Primary phase II objectives are overall response rate (ORR; blinded independent central review per RECIST v1.1) and safety. We report efficacy for response-evaluable pts (REP) with
In 79 REP with
Pralsetinib demonstrated potent and durable clinical activity in
NCT03037385.
Medical writing support was provided by Miriam Cohen, PhD, and editorial support was provided by Sinead Stewart, all of Paragon, Knutsford, UK, supported by Blueprint Medicines Corporation, Cambridge, MA according to Good Publication Practice guidelines.
Blueprint Medicines Corporation.
Blueprint Medicines Corporation.
M. Hu: Advisory/Consultancy: Blueprint Medicines Corporation; Advisory/Consultancy: Eli Lilly & Co; Advisory/Consultancy: Loxo Oncology; Advisory/Consultancy: Veracyte. V. Subbiah: Research grant/Funding (self): Roche/Genentech; Research grant/Funding (self): Novartis; Research grant/Funding (self): Bayer; Research grant/Funding (self): GSK; Research grant/Funding (self): Nanocarrier; Research grant/Funding (self): Vegenics; Research grant/Funding (self): Celgene; Research grant/Funding (self): Northwest Biotherapeutics; Research grant/Funding (self): Berghealth; Research grant/Funding (self): Incyte; Research grant/Funding (self): Fujifilm; Research grant/Funding (self): PharmaMar; Research grant/Funding (self): D3; Research grant/Funding (self): Pfizer; Research grant/Funding (self): Multivir; Research grant/Funding (self): Amgen; Research grant/Funding (self): AbbVie; Research grant/Funding (self): Alfa-sigma; Research grant/Funding (self): Agensys; Research grant/Funding (self): Boston Biomedical; Research grant/Funding (self): Idera Pharma; Research grant/Funding (self): Inhibrx; Research grant/Funding (self): Exelixis; Research grant/Funding (self): Blueprint Medicines Corporation; Research grant/Funding (self): Loxo Oncology; Research grant/Funding (self): MedImmune; Research grant/Funding (self): Altum; Research grant/Funding (self): Dragonfly Therapeutics; Research grant/Funding (self): Takeda; Research grant/Funding (self): National Comprehensive Cancer Network. L.J. Wirth: Advisory/Consultancy: Bayer; Advisory/Consultancy: Blueprint Medicines Corporation; Advisory/Consultancy: Cue Biopharma; Advisory/Consultancy: Eisai; Advisory/Consultancy: Exelixis; Advisory/Consultancy: Genentech; Advisory/Consultancy: Lilly; Advisory/Consultancy: Loxo Oncology; Advisory/Consultancy: Merck; Advisory/Consultancy: Rakuten Medical. M. Schuler: Advisory/Consultancy: AstraZeneca; Advisory/Consultancy: Boehringer Ingelheim; Advisory/Consultancy: Bristol-Myers Squibb; Advisory/Consultancy: Novartis; Advisory/Consultancy: Roche; Advisory/Consultancy: Takeda; Advisory/Consultancy: MorphoSys. A.S. Mansfield: Honoraria (institution): AbbVie; Honoraria (institution): AstraZeneca; Honoraria (institution): BMS; Honoraria (institution): Roche/Genentech; Travel/Accommodation/Expenses: Novartis; Research grant/Funding (institution): Verily Research; Non-remunerated activity/ies: Mesothelioma Applied Research Foundation. M.S. Brose: Honoraria (self), Research grant/Funding (institution): Exelixis; Advisory/Consultancy, Research grant/Funding (institution): Blueprint Medicines Corporation; Advisory/Consultancy, Research grant/Funding (institution): Lilly; Honoraria (self), Advisory/Consultancy: Bayer; Honoraria (self), Advisory/Consultancy: Eisai; Honoraria (self): Raffo; Advisory/Consultancy: Loxo. G. Curigliano: Advisory/Consultancy, Speaker Bureau/Expert testimony: Roche; Advisory/Consultancy, Speaker Bureau/Expert testimony: Seattle Genetics; Speaker Bureau/Expert testimony: Novartis; Advisory/Consultancy, Speaker Bureau/Expert testimony: Lilly; Advisory/Consultancy, Speaker Bureau/Expert testimony: Pfizer; Advisory/Consultancy, Speaker Bureau/Expert testimony: Foundation Medicine; Advisory/Consultancy, Speaker Bureau/Expert testimony: NanoString; Advisory/Consultancy, Speaker Bureau/Expert testimony: Samsung; Advisory/Consultancy, Speaker Bureau/Expert testimony: Celltrion; Non-remunerated activity/ies: Ellipsis; Speaker Bureau/Expert testimony: BMS; Speaker Bureau/Expert testimony: MSD; Advisory/Consultancy: Mylan. S. Leboulleux: Advisory/Consultancy: Lilly; Advisory/Consultancy: Bayer; Speaker Bureau/Expert testimony: Eisai. V.W. Zhu: Honoraria (self): AstraZeneca; Honoraria (self): Roche/Foundation Medicine; Honoraria (self): Roche/Genentech; Honoraria (self): Takeda; Shareholder/Stockholder/Stock options: TP Therapeutics. B. Keam: Advisory/Consultancy, Research grant/Funding (self): AstraZeneca; Advisory/Consultancy, Research grant/Funding (self): MSD Oncology; Advisory/Consultancy: ABL Bio; Advisory/Consultancy: Genexine; Advisory/Consultancy: Cellid; Advisory/Consultancy: Ono Pharmaceutical. I. Matos: Research grant/Funding (self): Roche. C-C. Lin: Advisory/Consultancy: Blueprint Medicines Corporation; Advisory/Consultancy: Boehringer Ingelheim; Honoraria (self), Advisory/Consultancy: Daiichi Sankyo; Honoraria (self), Advisory/Consultancy: Novartis; Advisory/Consultancy: Takeda; Honoraria (self): BMS; Honoraria (self): Roche; Travel/Accommodation/Expenses: BeiGene; Travel/Accommodation/Expenses: Eli Lilly. D. Adkins: Advisory/Consultancy: Lilly; Advisory/Consultancy: Pfizer; Advisory/Consultancy: Celgene; Advisory/Consultancy: Merck; Advisory/Consultancy: CUE; Advisory/Consultancy: Aduro; Advisory/Consultancy: KURA; Advisory/Consultancy: Oncolys; Advisory/Consultancy: Enzychyme; Advisory/Consultancy: Matrix; Advisory/Consultancy: Celldex. C.S. Baik: Advisory/Consultancy, Research grant/Funding (self): AstraZeneca; Research grant/Funding (self): Celgene; Research grant/Funding (self): Novartis; Research grant/Funding (self): MedImmune; Research grant/Funding (self): SWOG; Research grant/Funding (self): Genentech; Research grant/Funding (self): Loxo Oncology; Research grant/Funding (self): Pfizer; Research grant/Funding (self): Spectrum Pharmaceuticals; Research grant/Funding (self): Blueprint Medicines Corporation; Research grant/Funding (self): Daiichi Sankyo; Research grant/Funding (self): Rain Therapeutics; Research grant/Funding (self): AbbVie; Research grant/Funding (self): TP Therapeutics; Research grant/Funding (self): Lilly Oncology. G. Lopes: Advisory/Consultancy, Research grant/Funding (institution), Travel/Accommodation/Expenses: Pfizer; Advisory/Consultancy, Research grant/Funding (self): AstraZeneca; Honoraria (self), Travel/Accommodation/Expenses: Boehringer Ingelheim; Travel/Accommodation/Expenses: E R Squibb Sons; Research grant/Funding (institution), Travel/Accommodation/Expenses: Janssen; Research grant/Funding (institution): MSD; Research grant/Funding (institution): EMD Serono; Research grant/Funding (institution): Blueprint Medicines Corporation; Research grant/Funding (institution): Tesaro; Research grant/Funding (institution): Bavarian Nordic; Research grant/Funding (institution): Novartis; Research grant/Funding (institution): G1 Therapeutics; Research grant/Funding (institution): Adaptimmune; Research grant/Funding (institution): BMS; Research grant/Funding (institution): GSK; Research grant/Funding (institution): AbbVie; Research grant/Funding (institution): Rgenix; Research grant/Funding (institution): Roche; Research grant/Funding (institution): Genentech; Research grant/Funding (institution): Lilly. D. Sarker: Honoraria (self): Pfizer; Honoraria (self): Bayer; Advisory/Consultancy: Ipsen; Honoraria (self): Novartis; Advisory/Consultancy: Eisai; Advisory/Consultancy: Surface Oncology; Advisory/Consultancy: MSD Oncology. H. Zhang, C. Turner: Shareholder/Stockholder/Stock options, Full/Part-time employment: Blueprint Medicines Corporation. M. Taylor: Advisory/Consultancy, Speaker Bureau/Expert testimony: BMS; Advisory/Consultancy, Speaker Bureau/Expert testimony: Eisai; Advisory/Consultancy: Array BioPharma; Advisory/Consultancy: Loxo Oncology; Advisory/Consultancy: Bayer; Advisory/Consultancy: ArQule; Advisory/Consultancy: Blueprint Medicines Corporation; Advisory/Consultancy: Novartis; Advisory/Consultancy: Sanofi/Genzyme; Speaker Bureau/Expert testimony: Merck. All other authors have declared no conflicts of interest.