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2. Metabolite Bioanalysis in Drug Development: Recommendations from the IQ Consortium Metabolite Bioanalysis Working Group.

Author

Li, Wenkui, Vazvaei‐Smith, Faye, Dear, Gordon, Boer, Jason, Cuyckens, Filip, Fraier, Daniela, Liang, Yuexia, Lu, Ding, Mangus, Heidi, Moliner, Patricia, Pedersen, Mette Lund, Romeo, Andrea A., Spracklin, Douglas K., Wagner, David S., Winter, Serge, and Xu, Xiaohui

Subjects
DRUG development, CONSORTIA, CLINICAL trials, DRUG registration, INVESTIGATIONAL drugs
Abstract

The intent of this perspective is to share the recommendations of the International Consortium for Innovation and Quality in Pharmaceutical Development Metabolite Bioanalysis Working Group on the fit‐for‐purpose metabolite bioanalysis in support of drug development and registration. This report summarizes the considerations for the trigger, timing, and rigor of bioanalysis in the various assessments to address unique challenges due to metabolites, with respect to efficacy and safety, which may arise during drug development from investigational new drug (IND) enabling studies, and phase I, phase II, and phase III clinical trials to regulatory submission. The recommended approaches ensure that important drug metabolites are identified in a timely manner and properly characterized for efficient drug development. [ABSTRACT FROM AUTHOR]

Published
2024
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4. Metabolite Bioanalysis in Drug Development: Recommendations from the IQ Consortium Metabolite Bioanalysis Working Group

Author

Li, Wenkui, primary, Vazvaei‐Smith, Faye, additional, Dear, Gordon, additional, Boer, Jason, additional, Cuyckens, Filip, additional, Fraier, Daniela, additional, Liang, Yuexia, additional, Lu, Ding, additional, Mangus, Heidi, additional, Moliner, Patricia, additional, Pedersen, Mette Lund, additional, Romeo, Andrea A., additional, Spracklin, Douglas K., additional, Wagner, David S., additional, Winter, Serge, additional, and Xu, Xiaohui (Sophia), additional

Published
2023
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8. Pklr Variants Associated with Acute Pain in Sickle Cell Disease Influence ATP Concentrations in Red Blood Cells

Author

Wang, Xunde, primary, Fu, Yi-Ping, additional, Li, Quan, additional, Dunkelberger, Emily B, additional, Tegegn, Mickias, additional, Kosinski, Penelope A., additional, Mangus, Heidi, additional, Hamre, John, additional, Mbulaiteye, Sam M., additional, Prokunina-Olsson, Ludmila, additional, Chanock, Stephen J., additional, Asomaning, Nancy, additional, Jeffries, Neal, additional, Eaton, William, additional, and Thein, Swee Lay, additional

Published
2022
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10. Mitapivat (AG-348) Demonstrates Safety, Tolerability, and Improvements in Anemia, Hemolysis, Oxygen Affinity, and Hemoglobin S Polymerization Kinetics in Adults with Sickle Cell Disease: A Phase 1 Dose Escalation Study

Author

Xu, Julia Z., primary, Conrey, Anna, additional, Frey, Ingrid, additional, Gwaabe, Eveline, additional, Menapace, Laurel A, additional, Tumburu, Laxminath, additional, Lundt, Maureen, additional, Li, Quan, additional, Glass, Kristen, additional, Iyer, Varsha, additional, Mangus, Heidi, additional, Kung, Charles, additional, Dang, Lenny, additional, Kosinski, Penelope A., additional, Hawkins, Peter, additional, Jeffries, Neal, additional, Eaton, William, additional, and Thein, Swee Lay, additional

Published
2021
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12. Decreased activity and stability of pyruvate kinase in sickle cell disease: a novel target for mitapivat therapy

Author

Rab, Minke A. E., primary, Bos, Jennifer, additional, van Oirschot, Brigitte A., additional, van Straaten, Stephanie, additional, Kosinski, Penelope A., additional, Chubukov, Victor, additional, Kim, Hyeryun, additional, Mangus, Heidi, additional, Schutgens, Roger E. G., additional, Pasterkamp, Gerard, additional, Dang, Lenny, additional, Kung, Charles, additional, van Beers, Eduard J., additional, and van Wijk, Richard, additional

Published
2021
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13. Decreased activity and stability of pyruvate kinase in sickle cell disease: a novel target for mitapivat therapy

Author

Rab, Minke A. E., Bos, Jennifer, van Oirschot, Brigitte A., van Straaten, H.A.S. (Stéphanie), Kosinski, Penelope A., Chubukov, Victor, Kim, Hyeryun, Mangus, Heidi, Schutgens, Roger E. G., Pasterkamp, Gerard, Dang, Lenny, Kung, Charles, van Beers, Eduard J., van Wijk, Richard, Rab, Minke A. E., Bos, Jennifer, van Oirschot, Brigitte A., van Straaten, H.A.S. (Stéphanie), Kosinski, Penelope A., Chubukov, Victor, Kim, Hyeryun, Mangus, Heidi, Schutgens, Roger E. G., Pasterkamp, Gerard, Dang, Lenny, Kung, Charles, van Beers, Eduard J., and van Wijk, Richard

Published
2021

14. Decreased activity and stability of pyruvate kinase in sickle cell disease: a novel target for mitapivat therapy

Author

Unit Opleiding Aios, Circulatory Health, CDL Rode cel, Poli Van Creveldkliniek Medisch, Infection & Immunity, CDL Onderzoek Pasterkamp, CDL Arcadia, Rab, Minke A E, Bos, Jennifer, van Oirschot, Brigitte, Straaten, Stephanie van, Kosinski, Penelope A, Chubukov, Victor, Kim, Hyeryun, Mangus, Heidi, Schutgens, Roger E G, Pasterkamp, Gerard, Dang, Lenny, Kung, Charles, van Beers, Eduard J, van Wijk, Richard, Unit Opleiding Aios, Circulatory Health, CDL Rode cel, Poli Van Creveldkliniek Medisch, Infection & Immunity, CDL Onderzoek Pasterkamp, CDL Arcadia, Rab, Minke A E, Bos, Jennifer, van Oirschot, Brigitte, Straaten, Stephanie van, Kosinski, Penelope A, Chubukov, Victor, Kim, Hyeryun, Mangus, Heidi, Schutgens, Roger E G, Pasterkamp, Gerard, Dang, Lenny, Kung, Charles, van Beers, Eduard J, and van Wijk, Richard

Published
2021

15. Phase 1 Multiple Ascending Dose Study of Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Mitapivat (AG-348) in Subjects with Sickle Cell Disease

Author

Xu, Julia Z., primary, Conrey, Anna, additional, Frey, Ingrid, additional, Nichols, James, additional, Menapace, Laurel A, additional, Tumburu, Laxminath, additional, Lequang, Timothy, additional, Li, Quan, additional, Dunkelberger, Emily, additional, Henry, Eric, additional, Cellmer, Troy, additional, Iyer, Varsha, additional, Mangus, Heidi, additional, Kung, Charles, additional, Dang, Lenny, additional, Kosinski, Penelope A., additional, Hawkins, Peter, additional, Jeffries, Neal, additional, Eaton, William, additional, and Thein, Swee Lay, additional

Published
2020
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18. Genotype-Response Correlation in DRIVE PK, a Phase 2 Study of AG-348 in Patients with Pyruvate Kinase Deficiency

Author

Kung, Charles, primary, Kosinski, Penelope A., additional, Mangus, Heidi, additional, Hua, Lei, additional, Connor, Gary, additional, Mobilia, Michelle, additional, Sullivan, Karen, additional, Frye, Sasha, additional, Jouvin, Marie-Hélène, additional, Grace, Rachael F., additional, Glader, Bertil, additional, and Bowden, Chris, additional

Published
2018
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19. Interim Analyses from the Beacon Trial: A Phase 2, Randomized, Open-Label Trial of Bitopertin in Erythropoietic Protoporphyria

Author

Ross, Gayle, Stewart, Peter, Mensing, George, Chin, Melanie, Howell, Haley, Mangus, Heidi, and Savage, William

Abstract

Introduction:Erythropoietic protoporphyria (EPP) is associated with accumulation of photoreactive protoporphyrin IX (PPIX) in the skin and other organs, causing debilitating phototoxic skin reactions following exposure to sunlight, and potentially life-threatening protoporphyric hepatopathy in some patients. Reduction of PPIX is associated with amelioration of disease in the settings of hematopoietic stem cell transplant, pregnancy, and extracorporeal photoinactivation.

Published
2023
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