Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in sickle cell disease: a phase 2, open-label study

Note: Funding Information: Minke A.E. Rab and Richard van Wijk receive research funding from RR Mechatronics, Axcella Health Inc. and Global Blood Therapeutics (GBT). Richard van Wijk and Eduard J. van Beers are consultants for GBT. Minke A.E. Rab, Richard van Wijk and Eduard J. van Beers receive research funding and are consultants for Agios Pharmaceuticals Inc. M.H.C's institution has received investigator‐initiated research and travel grants as well as speaker fees over the years from the Netherlands Organization for Scientific Research (NWO) and Netherlands National Research Agenda (NWA), the Netherlands Organization for Health Research and Development (ZonMw), the Dutch Innovatiefonds Zorgverzekeraars, Baxter/Baxalta/Shire/ Takeda, Pfizer, Bayer Schering Pharma, CSL Behring, Sobi Biogen, Novo Nordisk, Novartis, and Nordic Pharma, and for serving as a steering board member for Roche, Bayer, and Novartis for which fees go to the Erasmus MC as an institution. Erfan Nur receives research funding from Novartis and Emmaus and participates in the advisory board of Novartis. Bart J. Biemond receives research funding from Sanquin, GBT, and Novartis and participated in the advisory boards of Novartis, GBT, Novo Nordisk, Celgene, Chiesi, and Bluebird Bio. The remaining authors declare no competing financial interests. Funding Information: The authors sincerely wish to thank the patients who participated in this study. The authors also thank all members of the SCORE consortium for recruitment and helpful comments on the study design, conduct, protocol, and manuscript. This study was supported in part by research funding from Agios Pharmaceuticals Inc., Cambridge, MA, USA.