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AMR Drug RepurposingSmart Screeningresearch prototype

Mitapivat Sulfate

FDA-approved product (tablet, oral), marketed as Aqvesme, Pyrukynd.

Registered studies
19
Lab records
0
Pathogens at ≥40%
2 of 4

Chemical structure

Chemical structure of Mitapivat Sulfate
A drawing shows what the molecule is, not what it does.

Existing / approved use

Documented · FDA, WHO, ChEMBL

Approved for

  • Anemia
  • Familial hemolytic anemia
  • Genetic disorder

Indications ChEMBL records as approved, from FDA and DailyMed labels. Label ↗

WHO therapeutic group
Other hematological agents
FDA pharmacologic class
Pyruvate Kinase Activator FDA label ↗
Anti-infective classification
Not classified as an anti-infective.

Repurposing investigation

This medicine is being investigated here for AI-predicted antimicrobial activity against M. tuberculosis and MRSA. It was surfaced computationally for further investigation; it is not an established treatment for these infections.

AI-predicted activity

AI prediction · nothing measured
  • MRSA50.0% AI-predicted activityAt or above the ≥40% mark this site uses to surface candidates.
  • E. coli33.2% AI-predicted activityBelow the ≥40% mark this site uses to surface candidates.
  • K. pneumoniae27.0% AI-predicted activityBelow the ≥40% mark this site uses to surface candidates.
  • M. tuberculosis60.4% AI-predicted activityAt or above the ≥40% mark this site uses to surface candidates.

A model’s estimate of laboratory activity against each pathogen species. It is not clinical effectiveness.

Evidence

Clinical and experimental

Documented evidence

Clinical

19registered studies name this medicine, for any condition. See them below.

Experimental

No evidence found

No laboratory measurement against the four pathogens in the ChEMBL records loaded here.

Open Mitapivat Sulfate in ChEMBL Registered studies below

Docking

Computational · nothing measured

Dihydrofolate reductase ↗ (E. coli)

-8.2 kcal/mol

0better fit →-12

KPC-2 carbapenemase ↗ (K. pneumoniae)

-8.7 kcal/mol

0better fit →-12

Enoyl-ACP reductase (InhA) ↗ (M. tuberculosis)

-10.4 kcal/mol

0better fit →-12

Dihydrofolate reductase ↗ (MRSA)

-8.3 kcal/mol

0better fit →-12

AutoDock Vina, one experimentally solved protein per pathogen. This project’s screening target is -7.0 kcal/mol, its own mark rather than a universal cutoff. A docking score is a structural hypothesis, not proof of binding, and requires experimental validation.

Other medicines to investigate

AI prediction · nothing measured

Other repurposing candidates: approved medicines besides Mitapivat Sulfate with AI-predicted activity ≥40% against the chosen pathogen, leaving out existing antimicrobials. They are computational candidates for further investigation, not alternatives and not recommendations.

Sorted by AI-predicted activity, high to low. The order is not a ranking of which medicine is better.

Registered studies

A registration describes a study; it does not establish a positive result.

  • A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

    NCT07506863 · start 2026-09

    Registered record
    Registry ID
    NCT07506863
    Conditions
    Transfusion-dependent Alpha-Thalassemia; Transfusion-dependent Beta-Thalassemia
    Interventions
    Mitapivat Matched Placebo; Mitapivat
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Recruiting
    Start
    2026-09
    Completion
    2032-06
    Enrolment
    54

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionTransfusion-dependent Alpha-Thalassemia; Transfusion-dependent Beta-Thalassemia
    InterventionMitapivat Matched Placebo; Mitapivat
    PhasePhase 3
    Registry statusRecruiting

  • A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia

    NCT07517133 · start 2026-09

    Registered record
    Registry ID
    NCT07517133
    Conditions
    Non-Transfusion-dependent Alpha-Thalassemia; Non-Transfusion-dependent Beta-Thalassemia
    Interventions
    Mitapivat; Placebo Matching Mitapivat
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Recruiting
    Start
    2026-09
    Completion
    2032-03
    Enrolment
    45

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionNon-Transfusion-dependent Alpha-Thalassemia; Non-Transfusion-dependent Beta-Thalassemia
    InterventionMitapivat; Placebo Matching Mitapivat
    PhasePhase 3
    Registry statusRecruiting

  • A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)

    NCT07656415 · start 2026-07-21

    Registered record
    Registry ID
    NCT07656415
    Conditions
    Sickle Cell Disease
    Interventions
    Mitapivat Matched Placebo; Mitapivat
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Recruiting
    Start
    2026-07-21
    Completion
    2030-08
    Enrolment
    159

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionSickle Cell Disease
    InterventionMitapivat Matched Placebo; Mitapivat
    PhasePhase 3
    Registry statusRecruiting

  • A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy

    NCT06286046 · start 2026-03

    Registered record
    Registry ID
    NCT06286046
    Conditions
    Sickle Cell Disease; Nephropathy
    Interventions
    Mitapivat
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Withdrawn
    Start
    2026-03
    Completion
    2028-12
    Enrolment
    0

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionSickle Cell Disease; Nephropathy
    InterventionMitapivat
    PhasePhase 2
    Registry statusWithdrawn

  • To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.

    NCT07055243 · start 2025-06-26

    Registered record
    Registry ID
    NCT07055243
    Conditions
    Anemia
    Interventions
    Mitapivat
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Recruiting
    Start
    2025-06-26
    Completion
    2027-06-30
    Enrolment
    9

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionAnemia
    InterventionMitapivat
    PhasePhase 2
    Registry statusRecruiting

  • Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants

    NCT06648824 · start 2024-10-17

    Registered record
    Registry ID
    NCT06648824
    Conditions
    Healthy Participants
    Interventions
    Mitapivat; Midazolam
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2024-10-17
    Completion
    2024-12-03
    Enrolment
    20

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionHealthy Participants
    InterventionMitapivat; Midazolam
    PhasePhase 1
    Registry statusCompleted

  • Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies

    NCT05935202 · start 2023-12-21

    Registered record
    Registry ID
    NCT05935202
    Conditions
    Hereditary Red Blood Cell Disorder (Disorder)
    Interventions
    Mitapivat sulfate
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Not yet recruiting
    Start
    2023-12-21
    Completion
    2026-12-15
    Enrolment
    25

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionHereditary Red Blood Cell Disorder (Disorder)
    InterventionMitapivat sulfate
    PhasePhase 2
    Registry statusNot yet recruiting

  • A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study

    NCT05777993 · start 2023-07-06

    Registered record
    Registry ID
    NCT05777993
    Conditions
    Pyruvate Kinase Deficiency; Anemia, Hemolytic; Sickle Cell Disease; Thalassemia
    Interventions
    Mitapivat
    Study type
    INTERVENTIONAL
    Phase
    Phase 4
    Registry status
    Enrolling by invitation
    Start
    2023-07-06
    Completion
    2029-08
    Enrolment
    59

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionPyruvate Kinase Deficiency; Anemia, Hemolytic; Sickle Cell Disease; Thalassemia
    InterventionMitapivat
    PhasePhase 4
    Registry statusEnrolling by invitation

  • A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With Normal Hepatic Function

    NCT05610657 · start 2023-01-10

    Registered record
    Registry ID
    NCT05610657
    Conditions
    Moderate Hepatic Impairment
    Interventions
    Mitapivat
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2023-01-10
    Completion
    2023-07-21
    Enrolment
    20

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionModerate Hepatic Impairment
    InterventionMitapivat
    PhasePhase 1
    Registry statusCompleted

  • A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period

    NCT05144256 · start 2022-06-08

    Registered record
    Registry ID
    NCT05144256
    Conditions
    Pediatric Pyruvate Kinase Deficiency; Pediatric Hemolytic Anemia
    Interventions
    Mitapivat; Mitapivat-matching placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Active not recruiting
    Start
    2022-06-08
    Completion
    2029-06
    Enrolment
    49

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionPediatric Pyruvate Kinase Deficiency; Pediatric Hemolytic Anemia
    InterventionMitapivat; Mitapivat-matching placebo
    PhasePhase 3
    Registry statusActive not recruiting

  • A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period

    NCT05175105 · start 2022-06-06

    Registered record
    Registry ID
    NCT05175105
    Conditions
    Pediatric Pyruvate Kinase Deficiency; Pediatric Hemolytic Anemia
    Interventions
    Mitapivat; Mitapivat-matching placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Active not recruiting
    Start
    2022-06-06
    Completion
    2030-01
    Enrolment
    30

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionPediatric Pyruvate Kinase Deficiency; Pediatric Hemolytic Anemia
    InterventionMitapivat; Mitapivat-matching placebo
    PhasePhase 3
    Registry statusActive not recruiting

  • A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

    NCT05031780 · start 2022-02-11

    Registered record
    Registry ID
    NCT05031780
    Conditions
    Sickle Cell Disease
    Interventions
    Mitapivat; Mitapivat-matching placebo; Mitapivat-matching placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 2 / Phase 3
    Registry status
    Active not recruiting
    Start
    2022-02-11
    Completion
    2030-02
    Enrolment
    286

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionSickle Cell Disease
    InterventionMitapivat; Mitapivat-matching placebo; Mitapivat-matching placebo
    PhasePhase 2 / Phase 3
    Registry statusActive not recruiting

  • A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

    NCT04770753 · start 2021-12-20

    Registered record
    Registry ID
    NCT04770753
    Conditions
    Non-Transfusion-dependent Alpha-Thalassemia; Non-Transfusion-dependent Beta-Thalassemia
    Interventions
    Placebo Matching Mitapivat; Mitapivat
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Active not recruiting
    Start
    2021-12-20
    Completion
    2028-12
    Enrolment
    194

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionNon-Transfusion-dependent Alpha-Thalassemia; Non-Transfusion-dependent Beta-Thalassemia
    InterventionPlacebo Matching Mitapivat; Mitapivat
    PhasePhase 3
    Registry statusActive not recruiting

  • A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

    NCT04770779 · start 2021-11-30

    Registered record
    Registry ID
    NCT04770779
    Conditions
    Transfusion-dependent Alpha-Thalassemia; Transfusion-dependent Beta-Thalassemia
    Interventions
    Placebo Matching Mitapivat; Mitapivat
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Active not recruiting
    Start
    2021-11-30
    Completion
    2029-06
    Enrolment
    258

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionTransfusion-dependent Alpha-Thalassemia; Transfusion-dependent Beta-Thalassemia
    InterventionPlacebo Matching Mitapivat; Mitapivat
    PhasePhase 3
    Registry statusActive not recruiting

  • A Study to Compare the Pharmacokinetics and Safety of Mitapivat 100 mg Tablet Formulation With Mitapivat 2 × 50 mg Tablet Formulation in Healthy Adult Participants

    NCT04696393 · start 2021-01-06

    Registered record
    Registry ID
    NCT04696393
    Conditions
    Healthy Volunteers
    Interventions
    Mitapivat tablet
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2021-01-06
    Completion
    2021-03-19
    Enrolment
    26

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionHealthy Volunteers
    InterventionMitapivat tablet
    PhasePhase 1
    Registry statusCompleted