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AMR Drug RepurposingSmart Screeningresearch prototype

Midostaurin

FDA-approved product (capsule, oral), marketed as Midostaurin, Rydapt.

Registered studies
50+
Lab records
0
Pathogens at ≥40%
2 of 4

Chemical structure

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

Existing / approved use

Documented · FDA, WHO, ChEMBL

Approved for

  • Acute myeloid leukemia
  • Mast-cell leukemia
  • Mastocytosis
  • Neoplasm
  • Systemic mastocytosis

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

WHO therapeutic group
Other protein kinase inhibitors
FDA pharmacologic class
Kinase Inhibitor 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
  • MRSA50.9% AI-predicted activityAt or above the ≥40% mark this site uses to surface candidates.
  • E. coli35.5% AI-predicted activityBelow the ≥40% mark this site uses to surface candidates.
  • K. pneumoniae28.8% AI-predicted activityBelow the ≥40% mark this site uses to surface candidates.
  • M. tuberculosis55.2% 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

50+registered studies name this medicine, for any condition. The registry search keeps the first 50, so there may be more. See them below.

Experimental

No evidence found

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

Open Midostaurin in ChEMBL Registered studies below

Docking

Computational · nothing measured

Dihydrofolate reductase ↗ (E. coli)

-9.3 kcal/mol

0better fit →-12

KPC-2 carbapenemase ↗ (K. pneumoniae)

-6.1 kcal/mol

0better fit →-12

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

-8.0 kcal/mol

0better fit →-12

Dihydrofolate reductase ↗ (MRSA)

-9.4 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 Midostaurin 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.

  • Study of Iomab-B vs. Conventional Care in Older Subjects With Active, Relapsed or Refractory Acute Myeloid Leukemia

    NCT02665065 · start 2016-06

    Registered record
    Registry ID
    NCT02665065
    Conditions
    Acute Myeloid Leukemia; Leukemia, Acute Myeloid; Myeloid Leukemia, Acute; Leukemia, Myeloid, Acute; Acute Myelogenous Leukemia; Leukemia, Acute Myelogenous; Myelogenous Leukemia, Acute; AML; Bone Marrow Transplant
    Interventions
    Iomab-B; Conventional Care; HCT
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Active not recruiting
    Start
    2016-06
    Completion
    2026-12
    Enrolment
    153

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

    ConditionAcute Myeloid Leukemia; Leukemia, Acute Myeloid; Myeloid Leukemia, Acute; Leukemia, Myeloid, Acute +5 more
    InterventionIomab-B; Conventional Care; HCT
    PhasePhase 3
    Registry statusActive not recruiting

  • Cladribine, Idarubicin, Cytarabine, and Venetoclax in Treating Patients With Acute Myeloid Leukemia, High-Risk Myelodysplastic Syndrome, or Blastic Phase Chronic Myeloid Leukemia

    NCT02115295 · start 2014-05-19

    Registered record
    Registry ID
    NCT02115295
    Conditions
    Acute Biphenotypic Leukemia; Acute Myeloid Leukemia; Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Blasts 10 Percent or More of Bone Marrow Nucleated Cells; Blasts 10 Percent or More of Peripheral Blood White Cells; de Novo Myelodysplastic Syndrome; Myelodysplastic Syndrome; Previously Treated Myelodysplastic Syndrome; Recurrent Acute Myeloid Leukemia; Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Refractory Acute Myeloid Leukemia; Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Secondary Acute Myeloid Leukemia; Untreated Adult Acute Myeloid Leukemia
    Interventions
    Cladribine; Cytarabine; Gilteritinib; Idarubicin; Laboratory Biomarker Analysis; Midostaurin; Venetoclax
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Recruiting
    Start
    2014-05-19
    Completion
    2030-05-31
    Enrolment
    508

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

    ConditionAcute Biphenotypic Leukemia; Acute Myeloid Leukemia; Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Blasts 10 Percent or More of Bone Marrow Nucleated Cells +10 more
    InterventionCladribine; Cytarabine; Gilteritinib; Idarubicin +3 more
    PhasePhase 2
    Registry statusRecruiting

  • A Safety and Efficacy Study of LGH447 in Patients With Acute Myeloid Leukemia (AML) or High Risk Myelodysplastic Syndrome (MDS)

    NCT02078609 · start 2014-03-20

    Registered record
    Registry ID
    NCT02078609
    Conditions
    AML and High Risk MDS
    Interventions
    LGH447; LGH447 + midostaurin
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2014-03-20
    Completion
    2019-04-18
    Enrolment
    70

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

    ConditionAML and High Risk MDS
    InterventionLGH447; LGH447 + midostaurin
    PhasePhase 1
    Registry statusCompleted

  • Midostaurin in Indolent Systemic Mastocytosis

    NCT01920204 · start 2013-08

    Registered record
    Registry ID
    NCT01920204
    Conditions
    Indolent Systemic Mastocytosis
    Interventions
    Midostaurin,
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Unknown
    Start
    2013-08
    Completion
    2015-05
    Enrolment
    20

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

    ConditionIndolent Systemic Mastocytosis
    InterventionMidostaurin,
    PhasePhase 2
    Registry statusUnknown

  • Trial to Assess the Efficacy of Midostaurin (PKC412) in Patients With c-KIT or FLT3-ITD Mutated t(8;21) AML

    NCT01830361 · start 2013-03-13

    Registered record
    Registry ID
    NCT01830361
    Conditions
    Acute Myeloid Leukemia
    Interventions
    midostaurin (PKC412)
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2013-03-13
    Completion
    2019-10-30
    Enrolment
    18

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

    ConditionAcute Myeloid Leukemia
    Interventionmidostaurin (PKC412)
    PhasePhase 2
    Registry statusCompleted

  • PK and Safety of Midostaurin in Subjects With Impaired Hepatic Function and Subjects With Normal Hepatic Function

    NCT01429337 · start 2011-03-07

    Registered record
    Registry ID
    NCT01429337
    Conditions
    Hepatic Impairment
    Interventions
    Midostaurin; Midostaurin
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2011-03-07
    Completion
    2020-05-09
    Enrolment
    43

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

    ConditionHepatic Impairment
    InterventionMidostaurin; Midostaurin
    PhasePhase 1
    Registry statusCompleted

  • PKC412 and 5-Azacytidine

    NCT01202877 · start 2011-03

    Registered record
    Registry ID
    NCT01202877
    Conditions
    Leukemia
    Interventions
    5-azacytidine; PKC412
    Study type
    INTERVENTIONAL
    Phase
    Phase 1 / Phase 2
    Registry status
    Completed
    Start
    2011-03
    Completion
    2015-07
    Enrolment
    54

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

    ConditionLeukemia
    Intervention5-azacytidine; PKC412
    PhasePhase 1 / Phase 2
    Registry statusCompleted

  • Cladribine Based Induction Therapy With All-Trans Retinoic Acid and Midostaurin in Relapsed/Refractory AML

    NCT01161550 · start 2010-11

    Registered record
    Registry ID
    NCT01161550
    Conditions
    Leukemia, Myeloid, Acute
    Interventions
    Granulocyte colony-stimulating factor (G-CSF); Cladribine; Cytarabine; All-Trans Retinoic Acid (ATRA); Midostaurin
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2010-11
    Completion
    2012-08
    Enrolment
    11

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

    ConditionLeukemia, Myeloid, Acute
    InterventionGranulocyte colony-stimulating factor (G-CSF); Cladribine; Cytarabine; All-Trans Retinoic Acid (ATRA) +1 more
    PhasePhase 1
    Registry statusCompleted

  • Phase I Combination of Midostaurin, Bortezomib, and Chemo in Relapsed/Refractory Acute Myeloid Leukemia

    NCT01174888 · start 2010-08

    Registered record
    Registry ID
    NCT01174888
    Conditions
    Acute Myeloid Leukemia; Acute Myeloid Leukemia With Multilineage Dysplasia Following; Myelodysplastic Syndrome; Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities; Adult Acute Myeloid Leukemia With Del(5q); Adult Acute Myeloid Leukemia With Inv(16)(p13; q22); Adult Acute Myeloid Leukemia With t(15; 17)(q22; q12); Adult Acute Myeloid Leukemia With t(16; 16)(p13; q22); Adult Acute Myeloid Leukemia With t(8; 21)(q22; q22); Recurrent Adult Acute Myeloid Leukemia; Secondary Acute Myeloid Leukemia
    Interventions
    midostaurin; Bortezomib; mitoxantrone hydrochloride; etoposide; cytarabine
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2010-08
    Completion
    2016-05
    Enrolment
    34

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

    ConditionAcute Myeloid Leukemia; Acute Myeloid Leukemia With Multilineage Dysplasia Following; Myelodysplastic Syndrome; Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities +14 more
    Interventionmidostaurin; Bortezomib; mitoxantrone hydrochloride; etoposide +1 more
    PhasePhase 1
    Registry statusCompleted

  • Combination of Decitabine and Midostaurin in Patients Older Than 60 With Newly Diagnosed or Relapsed Refractory Acute Myeloid Leukemia

    NCT01130662 · start 2010-03

    Registered record
    Registry ID
    NCT01130662
    Conditions
    Acute Myeloid Leukemia
    Interventions
    combination therapy using decitabine and midostaurin
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2010-03
    Completion
    2011-10
    Enrolment
    16

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

    ConditionAcute Myeloid Leukemia
    Interventioncombination therapy using decitabine and midostaurin
    PhasePhase 1
    Registry statusCompleted

  • A Study of the Safety and Preliminary Efficacy of Oral Midostaurin (PKC412) in Relapsed or Refractory Pediatric Leukemia

    NCT00866281 · start 2009-09

    Registered record
    Registry ID
    NCT00866281
    Conditions
    Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia
    Interventions
    midostaurin
    Study type
    INTERVENTIONAL
    Phase
    Phase 1 / Phase 2
    Registry status
    Terminated
    Start
    2009-09
    Completion
    2014-09
    Enrolment
    22

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

    ConditionAcute Myeloid Leukemia; Acute Lymphoblastic Leukemia
    Interventionmidostaurin
    PhasePhase 1 / Phase 2
    Registry statusTerminated

  • RAD001 in Combination With PKC412 in Patients With Relapsed, Refractory or Poor Prognosis AML or MDS

    NCT00819546 · start 2009-01

    Registered record
    Registry ID
    NCT00819546
    Conditions
    Acute Myeloid Leukemia; Myelodysplastic Syndrome
    Interventions
    RAD001; PKC412
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Active not recruiting
    Start
    2009-01
    Completion
    2026-12
    Enrolment
    29

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

    ConditionAcute Myeloid Leukemia; Myelodysplastic Syndrome
    InterventionRAD001; PKC412
    PhasePhase 1
    Registry statusActive not recruiting

  • Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia

    NCT00782067 · start 2008-10-13

    Registered record
    Registry ID
    NCT00782067
    Conditions
    Leukemia
    Interventions
    Midostaurin (PKC412)
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2008-10-13
    Completion
    2017-08-24
    Enrolment
    116

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

    ConditionLeukemia
    InterventionMidostaurin (PKC412)
    PhasePhase 2
    Registry statusCompleted

  • Phase 2 Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia

    NCT00233454 · start 2005-03

    Registered record
    Registry ID
    NCT00233454
    Conditions
    Systemic Mastocytosis, Aggressive (ASM); Leukemia, Mast Cell; Hematological Non-mast Cell Lineage Disease (AHNMD)
    Interventions
    Midostaurin
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2005-03
    Completion
    2011-04-16
    Enrolment
    26

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

    ConditionSystemic Mastocytosis, Aggressive (ASM); Leukemia, Mast Cell; Hematological Non-mast Cell Lineage Disease (AHNMD)
    InterventionMidostaurin
    PhasePhase 2
    Registry statusCompleted

  • PKC412, Daunorubicin, and Cytarabine in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia

    NCT00093600 · start 2004-02

    Registered record
    Registry ID
    NCT00093600
    Conditions
    Acute Myeloid Leukemia (AML)
    Interventions
    cytarabine; daunorubicin hydrochloride; midostaurin
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2004-02
    Completion
    2011-06
    Enrolment
    69

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

    ConditionAcute Myeloid Leukemia (AML)
    Interventioncytarabine; daunorubicin hydrochloride; midostaurin
    PhasePhase 1
    Registry statusCompleted