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Journal Articles

REM-422, a Small Molecule MYB mRNA Degrader, Demonstrates Anti-Leukemic Activity As Monotherapy and in Combination with Standards of Care in Preclinical Models of AML

Samantha Levin-Furtney, Michael Thomas, Alycen M. Harney, Mengge Shan, Alexander Ivliev, Adam Stefek, Michael Cameron, Sudeep Prajapati, Michael Seiler, Dominic Reynolds, Silvia Buonamici, Charles Kung

Blood (2024) 144 (Supplement 1): 826.

https://doi.org/10.1182/blood-2024-208725

Published: 2024

Journal Articles

REM-422, a Potent, Selective, Oral Small Molecule mRNA Degrader of the MYB Oncogene, Demonstrates Anti-Tumor Activity in Mouse Xenograft Models of AML

Blood (2023) 142 (Supplement 1): 1425.

https://doi.org/10.1182/blood-2023-182676

Published: 2023

Journal Articles

A phase 1 dose escalation study of the pyruvate kinase activator mitapivat (AG-348) in sickle cell disease

Blood (2022) 140 (19): 2053–2062.

https://doi.org/10.1182/blood.2022015403

Published: 2022

Includes: Supplemental data

Journal Articles

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

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

Blood (2021) 138 (Supplement 1): 10.

https://doi.org/10.1182/blood-2021-145398

Published: 2021

Journal Articles

Pharmacodynamic Effects of AG-946, a Highly Potent Next-Generation Activator of Pyruvate Kinase, in Ex Vivo Treatment of Red Blood Cells from Sickle Cell Disease Patients

Minke A.E. Rab, Myrthe J. Van Dijk, Jennifer Bos, Brigitte A. van Oirschot, Johan Gerrits, Penelope A. Kosinski, Charles Kung, Judith J.M. Jans, Eduard J. Van Beers, Lenny Dang, Richard van Wijk

Blood (2021) 138 (Supplement 1): 2029.

https://doi.org/10.1182/blood-2021-145374

Published: 2021

Journal Articles

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

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

Blood (2021) 137 (21): 2997–3001.

https://doi.org/10.1182/blood.2020008635

Published: 2021

Includes: Supplemental data

Journal Articles

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

Blood (2020) 136 (Supplement 1): 21–22.

https://doi.org/10.1182/blood-2020-137716

Published: 2020

Journal Articles

The Pyruvate Kinase Activator Mitapivat Ameliorates Anemia and Prevents Iron Overload in a Mouse Model of Hereditary Spherocytosis

Alessandro Mattè, BSc,PhD, Wilson Anand, Enrica Federti, PhD, Charles Kung, PhD, Penelope A. Kosinski, MS, Veronica Riccardi, Iana Iatcenko, Lenny Dang, PhD, Christophe Leboeuf, Anne Janin, PhD, Carlo Brugnara, MD, Mohandas Narla, DSc, Lucia De Franceschi, MD

Blood (2020) 136 (Supplement 1): 29.

https://doi.org/10.1182/blood-2020-142232

Published: 2020

Journal Articles

Decreased Activity and Stability of Pyruvate Kinase in Hereditary Hemolytic Anemia: A Potential Target for Therapy By AG-348 (Mitapivat), an Allosteric Activator of Red Blood Cell Pyruvate Kinase

Minke A.E. Rab, MD, Brigitte A. van Oirschot, Stephanie van Straaten, MD PhD, Bart J. Biemond, MD PhD, Jennifer Bos, Penelope A. Kosinski, MS, Charles Kung, PhD, Eduard J. van Beers, MD PhD, Richard van Wijk, PhD

Blood (2019) 134 (Supplement_1): 3506.

https://doi.org/10.1182/blood-2019-129996

Published: 2019

Journal Articles

Pyruvate Kinase (PK) Protein and Enzyme Levels in the Diagnosis and Clinical Phenotype of PK Deficiency

Kathryn Addonizio, BA, Hanny Al-Samkari, MD, Bertil Glader, MD PhD, D Holmes Morton, MD, Satheesh Chonat, MD, Alexis A. Thompson, MD MPH, Kevin H. M. Kuo, MDMSc,FRCPC, Yaddanapudi Ravindranath, MBBS, Heng Wang, MD PhD, Jennifer A. Rothman, MD, Janet L. Kwiatkowski, MD MSCE, Charles Kung, PhD, Penelope A. Kosinski, MS, Hasan Al-Sayegh, MS, Wendy B. London, PhD, Rachael F. Grace, MD

Blood (2019) 134 (Supplement_1): 3515.

https://doi.org/10.1182/blood-2019-125248

Published: 2019

Journal Articles

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

Charles Kung, PhD, Penelope A. Kosinski, MS, Heidi Mangus, BS, Lei Hua, PhD, Gary Connor, Michelle Mobilia, BS, Karen Sullivan, BA, Sasha Frye, BS, Marie-Hélène Jouvin, MD, Rachael F. Grace, MD, Bertil Glader, MD, Chris Bowden, MD

Blood (2018) 132 (Supplement 1): 3621.

https://doi.org/10.1182/blood-2018-99-111166

Published: 2018

Journal Articles

Tibetan Enriched PKLR Variant Is Beneficial to High Altitude Adaption By Improving Oxygen Delivery

Jihyun Song, PhD, Virginia Abello, MD, Ricardo Amaru, MD, Adelina Sergueeva, MD, Jainagul Isakova, Penelope A. Kosinski, MS, Brigitte A. van Oirschot, Victor R. Gordeuk, MD, Charles Kung, PhD, Richard van Wijk, PhD, Josef T. Prchal, MD

Blood (2018) 132 (Supplement 1): 1027.

https://doi.org/10.1182/blood-2018-99-117389

Published: 2018

Journal Articles

Results Update from the DRIVE PK Study: Effects of AG-348, a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency

Blood (2017) 130 (Supplement 1): 2194.

https://doi.org/10.1182/blood.V130.Suppl_1.2194.2194

Published: 2017

Journal Articles

AG-348 enhances pyruvate kinase activity in red blood cells from patients with pyruvate kinase deficiency

Blood (2017) 130 (11): 1347–1356.

https://doi.org/10.1182/blood-2016-11-753525

Published: 2017

Includes: Supplemental data

Journal Articles

Population Pharmacokinetics and Pharmacodynamics of AG-519, a Pyruvate Kinase Activator for the Treatment of Pyruvate Kinase Deficiency, in Human Healthy Volunteers

Kha Le, Marvin Cohen, Ann J Barbier, Elizabeth Merica, Charles Kung, Penelope A Kosinski, Scott Biller, Hua Yang

Blood (2016) 128 (22): 1263.

https://doi.org/10.1182/blood.V128.22.1263.1263

Published: 2016

Journal Articles

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Doses of AG-519, an Allosteric Activator of Pyruvate Kinase-R, in Healthy Subjects

Ann J Barbier, Susan Bodie, Gary Connor, Elizabeth Merica, Charles Kung, Kha Le, Hua Yang, Penelope A Kosinski, Lee Silverman, Zheng (Jason) Yuan, Chris Bowden, Marvin Cohen

Blood (2016) 128 (22): 1264.

https://doi.org/10.1182/blood.V128.22.1264.1264

Published: 2016

Journal Articles

Characterization of Metabolic Response to AG-348, an Allosteric Activator of Red Cell Pyruvate Kinase, in Healthy Volunteers and Pyruvate Kinase Deficiency Patients

Victor Chubukov, Kendall Johnson, Penelope A Kosinski, Michelle Clasquin, Abhishek Jha, Hyeryun Kim, Thomas P Roddy, Elizabeth Merica, Ann J Barbier, Lenny Dang, Lee Silverman, Charles Kung

Blood (2016) 128 (22): 2452.

https://doi.org/10.1182/blood.V128.22.2452.2452

Published: 2016

Journal Articles

Effects of AG-348, a Pyruvate Kinase Activator, on Anemia and Hemolysis in Patients with Pyruvate Kinase Deficiency: Data from the DRIVE PK Study

Blood (2016) 128 (22): 402.

https://doi.org/10.1182/blood.V128.22.402.402

Published: 2016

Journal Articles

Population Pharmacokinetics and Pharmacodynamics of AG-348 in Healthy Human Volunteers Guide Dose Selection for the Treatment of Pyruvate Kinase Deficiency

Kha Le, PhD, Marvin Cohen, Yue Chen, Hyeryun Kim, Bruce Silver, Sam Agresta, MD, Elizabeth Merica, Charles Kung, Penelope Kosinski, Lee Silverman, Scott Biller, Hua Yang, PhD

Blood (2015) 126 (23): 3336.

https://doi.org/10.1182/blood.V126.23.3336.3336

Published: 2015

Journal Articles

DRIVE PK: A Phase 2 Trial of AG-348 in Patients with Pyruvate Kinase Deficiency

Ann Barbier, Bruce Silver, Elizabeth Merica, Marvin Cohen, Charles Kung, Hua Yang, PhD, Rachael F. Grace, MD, Bertil Glader, MD PhD, Sam Agresta, MD

Blood (2015) 126 (23): 4548.

https://doi.org/10.1182/blood.V126.23.4548.4548

Published: 2015

NARROW

REM-422, a Small Molecule MYB mRNA Degrader, Demonstrates Anti-Leukemic Activity As Monotherapy and in Combination with Standards of Care in Preclinical Models of AML

REM-422, a Potent, Selective, Oral Small Molecule mRNA Degrader of the MYB Oncogene, Demonstrates Anti-Tumor Activity in Mouse Xenograft Models of AML

A phase 1 dose escalation study of the pyruvate kinase activator mitapivat (AG-348) in sickle cell disease

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

Pharmacodynamic Effects of AG-946, a Highly Potent Next-Generation Activator of Pyruvate Kinase, in Ex Vivo Treatment of Red Blood Cells from Sickle Cell Disease Patients

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

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

The Pyruvate Kinase Activator Mitapivat Ameliorates Anemia and Prevents Iron Overload in a Mouse Model of Hereditary Spherocytosis

Decreased Activity and Stability of Pyruvate Kinase in Hereditary Hemolytic Anemia: A Potential Target for Therapy By AG-348 (Mitapivat), an Allosteric Activator of Red Blood Cell Pyruvate Kinase

Pyruvate Kinase (PK) Protein and Enzyme Levels in the Diagnosis and Clinical Phenotype of PK Deficiency

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

Tibetan Enriched PKLR Variant Is Beneficial to High Altitude Adaption By Improving Oxygen Delivery

Results Update from the DRIVE PK Study: Effects of AG-348, a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency

AG-348 enhances pyruvate kinase activity in red blood cells from patients with pyruvate kinase deficiency

Population Pharmacokinetics and Pharmacodynamics of AG-519, a Pyruvate Kinase Activator for the Treatment of Pyruvate Kinase Deficiency, in Human Healthy Volunteers

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Doses of AG-519, an Allosteric Activator of Pyruvate Kinase-R, in Healthy Subjects

Characterization of Metabolic Response to AG-348, an Allosteric Activator of Red Cell Pyruvate Kinase, in Healthy Volunteers and Pyruvate Kinase Deficiency Patients

Effects of AG-348, a Pyruvate Kinase Activator, on Anemia and Hemolysis in Patients with Pyruvate Kinase Deficiency: Data from the DRIVE PK Study

Population Pharmacokinetics and Pharmacodynamics of AG-348 in Healthy Human Volunteers Guide Dose Selection for the Treatment of Pyruvate Kinase Deficiency

DRIVE PK: A Phase 2 Trial of AG-348 in Patients with Pyruvate Kinase Deficiency

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American Society of Hematology

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American Society of Hematology

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