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Chemical Structure| 1260075-17-9 Chemical Structure| 1260075-17-9

Structure of Mitapivat
CAS No.: 1260075-17-9

Chemical Structure| 1260075-17-9

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PKR-IN-1 is a potent human R-type pyruvate kinase (PKR) inhibitor and also shows potency for mutant PKR including R510Q PKR, R532W PKR, T384W PKR etc.

Synonyms: AG-348

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Product Details of Mitapivat

CAS No. :1260075-17-9
Formula : C24H26N4O3S
M.W : 450.55
SMILES Code : O=S(C1=C2N=CC=CC2=CC=C1)(NC3=CC=C(C(N4CCN(CC5CC5)CC4)=O)C=C3)=O
Synonyms :
AG-348
MDL No. :MFCD26936342
InChI Key :XAYGBKHKBBXDAK-UHFFFAOYSA-N
Pubchem ID :59634741

Safety of Mitapivat

GHS Pictogram:
Signal Word:Warning
Hazard Statements:H302-H315-H319-H335
Precautionary Statements:P261-P305+P351+P338

Isoform Comparison

Biological Activity

Description
Mitapivat (AG-348) functions as an orally active allosteric activator of pyruvate kinase. It enhances enzymatic activity, protein stability, and ATP levels across various PKLR genotypes, indicating its potential in restoring the function of PK (pyruvate kinase)-deficient glycolytic pathways. Mitapivat is applicable in the investigation of PK deficiency [1][2][3].

Clinical Trial:

NCT Number Conditions Phases Recruitment Completion Date Locations
NCT06286046 Sickle Cell Disease|Nephropath... More >>y Less << PHASE2 NOT_YET_RECRUITING 2025-12-27 -
NCT04000165 Sickle Cell Disease EARLY_PHASE1 COMPLETED 2021-06-21 National Institutes of Health ... More >>Clinical Center, Bethesda, Maryland, 20892, United States Less <<
NCT05777993 Pyruvate Kinase Deficiency|Ane... More >>mia, Hemolytic Less << PHASE4 ENROLLING_BY_INVITATION 2025-08-29 McMaster Centre for Transfusio... More >>n Medicine McMaster University, Hamilton, Ontario, L8S3Z5, Canada|Tohoku University Hospital, Sendai, Miyagi, 980-8574, Japan|Kyoto Katsura Hospital, Kyoto, 615-8256, Japan|Toho University - Omori Medical Center, Ota-Ku, 143-8541, Japan Less <<
NCT02476916 Pyruvate Kinase Deficiency PHASE2 ACTIVE_NOT_RECRUITING 2025-03-25 Stanford University, Palo Alto... More >>, California, 94304, United States|Boston Children's Hospital, Boston, Massachusetts, 02215, United States|Wayne State University School of Medicine - Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|New York Presbyterian Hospital- Weil Cornell Medical College, New York, New York, 10065, United States|Central Pennsylvania Clinic, Belleville, Pennsylvania, 17004, United States|Children Hospital of Philadelphia (CHOP), Philadelphia, Pennsylvania, 19104, United States|University of Utah, Salt Lake City, Utah, 84113, United States|University Health Network, Toronto, Ontario, M5G 2C4, Canada|H?pital Henri Mondor, Créteil, Ile-de-France, 94010, France|H?pital Saint-Vincent de Paul, Lille, Nord, 59000, France|UOC Oncoematologia Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano, 20122, Italy|Universitair Medisch Centrum Utrecht, Utrecht, 3584 CX, Netherlands|Hammersmith Hospital, London, W12 0NN, United Kingdom Less <<
NCT03559699 Pyruvate Kinase Deficiency|Ane... More >>mia, Hemolytic Less << PHASE3 COMPLETED 2020-11-12 UCSF Benioff Children's Hospit... More >>al, Oakland, California, 94609, United States|Emory University, Atlanta, Georgia, 30322, United States|Massachusetts General Hospital, Boston, Massachusetts, 02115, United States|Seattle Cancer Care Alliance, Seattle, Washington, 98109, United States|Toronto General Hospital, University Health Network, Toronto, M5G 2C4, Canada|University of Copenhagen, Herlev Hospital, Herlev, 2730, Denmark|Hopital Universitaire Henri Mondor, Créteil, 94010, France|H?pital de la Timone, Marseille, Cedex 5, 13385, France|St James's Hospital Department of Haematology, Dublin, Ireland|Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milano, 20122, Italy|AORN Cardarelli, Napoli, 80131, Italy|Ospedale Galliera, Napoli, 80131, Italy|AOU Policlinico, Università della Campania "Luigi Vanvitelli", Napoli, 80138, Italy|Universitair Medisch Centrum Utrecht, Utrecht, 3584, Netherlands|Department of Paediatrics and Thalassaemia Center, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, 10700, Thailand|Addenbrooke's Hospital, Cambridge, CB2 0QQ, United Kingdom|Imperial College Healthcare NHS Trust, Hammersmith Hospital, London, W12 0NN, United Kingdom|University College London, London, WC1E 6BT, United Kingdom|Manchester Royal Infirmary, Manchester, M13 9WL, United Kingdom Less <<
NCT05610657 Moderate Hepatic Impairment PHASE1 COMPLETED 2023-07-21 Orange County Research Center ... More >>(OCRC), Tustin, California, 92780, United States|Clinical Pharmacology of Miami (CPMI), Miami, Florida, 33014, United States|Orlando Clinical Research Center (OCRC), Orlando, Florida, 32809, United States Less <<
NCT03853798 Pyruvate Kinase Deficiency PHASE3 COMPLETED 2024-07-03 Phoenix Children's Hospital, P... More >>hoenix, Arizona, 85016, United States|UCSF Benioff Children's Hospital, Oakland, Oakland, California, 95609, United States|Emory-Children's Center, Atlanta, Georgia, 30322, United States|Indiana Hemophilia & Thrombosis Center Inc., Indianapolis, Indiana, 46260, United States|Massachusetts General Hospital, Boston, Massachusetts, 02114, United States|Boston Children's Hospital, Boston, Massachusetts, 02115, United States|Wayne State University School of Medicine, Detroit, Michigan, 48201, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States|Houston Methodist Research Institute, Houston, Texas, 77030, United States|University of Utah, Salt Lake City, Utah, 84113, United States|Seattle Cancer Care Alliance, Seattle, Washington, 98195, United States|UNICAMP - Hemocentro, S?o Paulo, 13083-878, Brazil|McMaster University, Hamilton, Ontario, L8N3Z5, Canada|Herlev University Hospital, Herlev, 2730, Denmark|CHU Hopitaux de Bordeaux - H?pital Saint-André, Bordeaux, 33000, France|CHU H?pital Henri Mondor, Créteil, 94010, France|Hospital La Timone, Marseille, 13385, France|Institut Universitaire du Cancer de Toulouse - Oncopole, Toulouse, 31100, France|Charite - UB - CVK - Medizinische Klinik, Berlin, 10117, Germany|Universit?tsklinik Würzburg, Würzburg, 97080, Germany|St James's Hospital, Dublin 8, Ireland|Ospedale Galliera, Genova, 16128, Italy|Osp Maggiore Policlinico Milano, Milano, 20122, Italy|AORN Cardarelli, Napoli, 00165, Italy|Università della Campania "Luigi Vanvitelli", Napoli, 80318, Italy|Mie University Hospital, Tsu-shi, Mie, 514-8507, Japan|Tohoku University Hospital, Sendai, Miyagi, 980-8574, Japan|Kyoto Katsura Hospital, Kyoto, 615-8256, Japan|Kansai Medical University, Dep. of Pediatrics, Hirakata Hospital, Osaka, 573-1010, Japan|Toho University Omori Medical Center, Tokyo, 8541, Japan|Yeungnam University Hospital, Daegu, 705-703, Korea, Republic of|Van Creveldkliniek, Utrecht, 3508 GA, Netherlands|Hospital. U. Vall d'Hebron, Barcelona, 08035, Spain|Hospital Universitario La Paz, Madrid, 28046, Spain|Htal Clínico Universitario Virgen de la Arrixaca., Murcia, 30120, Spain|Centre Hospitalier Universitaire Vaudois (CHUV), Vaud (Lausanne), 1011, Switzerland|Faculty of Medicine Siriraj Hospital, Bangkok, 10700, Thailand|Hacettepe University Faculty of Medicine, Ankara, 06100, Turkey|Addenbrooke's Hospital, Cambridge, CB2 0QQ, United Kingdom|Imperial College Healthcare NHS Trust, London, W2 INY, United Kingdom|University College London, London, WC1E 6BT, United Kingdom Less <<
NCT03692052 Thalassemia PHASE2 ACTIVE_NOT_RECRUITING 2030-09-30 UCSF Benioff Children's Hospit... More >>al Oakland, Oakland, California, 94609, United States|Massachusetts General Hospital, Boston, Massachusetts, 02114, United States|University Health Network (Toronto General Hospital), Toronto, Ontario, M5G 2C4, Canada|Imperial College Healthcare NHS Trust (Hammersmith Hospital), London, W12 0HS, United Kingdom Less <<
NCT04610866 Sickle Cell Disease|Hemolytic ... More >>Anemia Less << PHASE1|PHASE2 ACTIVE_NOT_RECRUITING 2028-02-28 National Institutes of Health ... More >>Clinical Center, Bethesda, Maryland, 20892, United States Less <<

Protocol

Bio Calculators
Preparing Stock Solutions 1mg 5mg 10mg

1 mM

5 mM

10 mM

2.22mL

0.44mL

0.22mL

11.10mL

2.22mL

1.11mL

22.20mL

4.44mL

2.22mL

Dissolving Methods
Please choose the appropriate dissolution scheme according to your animal administration guide.For the following dissolution schemes, clear stock solution should be prepared according to in vitro experiments, and then cosolvent should be added in turn:

in order to ensure the reliability of the experimental results, the clarified stock solution can be properly preserved according to the storage conditions; The working fluid for in vivo experiment is recommended to be prepared now and used on the same day;

The percentage shown in front of the following solvent refers to the volume ratio of the solvent in the final solution; If precipitation or precipitation occurs in the preparation process, it can be assisted by heating and/or ultrasound.
Protocol 1
Protocol 2

References

 

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