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AMR Drug RepurposingSmart Screeningresearch prototype

Lonafarnib

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

Registered studies
39
Lab records
0
Pathogens at ≥40%
3 of 4

Chemical structure

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

Existing / approved use

Documented · FDA, WHO, ChEMBL

Approved for

  • Hutchinson-Gilford progeria syndrome
  • Laminopathies
  • Progeria

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

WHO therapeutic group
Various alimentary tract and metabolism products
FDA pharmacologic class
Farnesyltransferase 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 E. coli, MRSA and M. tuberculosis. It was surfaced computationally for further investigation; it is not an established treatment for these infections.

AI-predicted activity

AI prediction · nothing measured
  • MRSA63.3% AI-predicted activityAt or above the ≥40% mark this site uses to surface candidates.
  • E. coli53.6% AI-predicted activityAt or above the ≥40% mark this site uses to surface candidates.
  • K. pneumoniae31.7% AI-predicted activityBelow the ≥40% mark this site uses to surface candidates.
  • M. tuberculosis49.6% 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

39registered 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 Lonafarnib in ChEMBL Registered studies below

Docking

Computational · nothing measured

Dihydrofolate reductase ↗ (E. coli)

-7.0 kcal/mol

0better fit →-12

KPC-2 carbapenemase ↗ (K. pneumoniae)

-4.3 kcal/mol

0better fit →-12

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

-6.9 kcal/mol

0better fit →-12

Dihydrofolate reductase ↗ (MRSA)

-6.2 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 Lonafarnib 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.

  • Temozolomide and SCH66336 for Recurrent Glioblastoma Multiforme

    NCT00038493 · start 2001-09-21

    Registered record
    Registry ID
    NCT00038493
    Conditions
    Glioblastoma Multiforme
    Interventions
    Temozolomide and SCH66336
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2001-09-21
    Completion
    2005-08-01
    Enrolment
    23

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

    ConditionGlioblastoma Multiforme
    InterventionTemozolomide and SCH66336
    PhasePhase 2
    Registry statusCompleted

  • Phase II Study of SCH66336, A Farnesyltransferase Inhibitor in Chronic Myelogenous Leukemia (CML)

    NCT00038597 · start 2001-04-30

    Registered record
    Registry ID
    NCT00038597
    Conditions
    Myelogenous Leukemia, Chronic
    Interventions
    SCH66336
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2001-04-30
    Completion
    2004-05-07
    Enrolment
    13

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

    ConditionMyelogenous Leukemia, Chronic
    InterventionSCH66336
    PhasePhase 2
    Registry statusCompleted

  • SCH 66336 Plus Gemcitabine in Treating Patients With Advanced Cancer of the Urinary Tract

    NCT00006351 · start 2000-06

    Registered record
    Registry ID
    NCT00006351
    Conditions
    Bladder Cancer; Transitional Cell Cancer of the Renal Pelvis and Ureter; Urethral Cancer
    Interventions
    gemcitabine hydrochloride; lonafarnib
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2000-06
    Completion
    Not recorded
    Enrolment
    34

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

    ConditionBladder Cancer; Transitional Cell Cancer of the Renal Pelvis and Ureter; Urethral Cancer
    Interventiongemcitabine hydrochloride; lonafarnib
    PhasePhase 2
    Registry statusCompleted

  • SCH 66336 Before Surgery in Treating Patients With Colorectal Cancer That Has Metastasized to the Liver

    NCT00005030 · start 1999-09-29

    Registered record
    Registry ID
    NCT00005030
    Conditions
    Colorectal Cancer; Metastatic Cancer
    Interventions
    SCH 66336; conventional surgery
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Withdrawn
    Start
    1999-09-29
    Completion
    2000-09
    Enrolment
    0

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

    ConditionColorectal Cancer; Metastatic Cancer
    InterventionSCH 66336; conventional surgery
    PhasePhase 1
    Registry statusWithdrawn

  • A Phase IB Study Of Oral SCH 66336 Preoperatively In Patients With Head And Neck Squamous Cell Cancer

    NCT00038584 · start 1999-06

    Registered record
    Registry ID
    NCT00038584
    Conditions
    Carcinoma, Squamous Cell; Cancer of Head and Neck
    Interventions
    SCH66336
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    1999-06
    Completion
    2004-11
    Enrolment
    37

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

    ConditionCarcinoma, Squamous Cell; Cancer of Head and Neck
    InterventionSCH66336
    PhasePhase 1
    Registry statusCompleted

  • Combination Chemotherapy in Treating Patients With Advanced Cancer

    NCT00003956 · start 1999-04

    Registered record
    Registry ID
    NCT00003956
    Conditions
    Lymphoma; Unspecified Adult Solid Tumor, Protocol Specific
    Interventions
    fluorouracil; leucovorin calcium; lonafarnib
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    1999-04
    Completion
    2009-12
    Enrolment
    25

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

    ConditionLymphoma; Unspecified Adult Solid Tumor, Protocol Specific
    Interventionfluorouracil; leucovorin calcium; lonafarnib
    PhasePhase 1
    Registry statusCompleted

  • SCH 66336 With or Without Gemcitabine Followed by Surgery Compared With Surgery Alone in Treating Patients With Primary Liver Cancer

    NCT00020774 · start 1998-10

    Registered record
    Registry ID
    NCT00020774
    Conditions
    Liver Cancer
    Interventions
    gemcitabine hydrochloride; lonafarnib; conventional surgery
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Withdrawn
    Start
    1998-10
    Completion
    Not recorded
    Enrolment
    0

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

    ConditionLiver Cancer
    Interventiongemcitabine hydrochloride; lonafarnib; conventional surgery
    PhasePhase 2
    Registry statusWithdrawn

  • A Study of Lonafarnib With or Without Ritonavir in Patients With HDV

    NCT02968641

    Registered record
    Registry ID
    NCT02968641
    Conditions
    Chronic Delta Hepatitis
    Interventions
    Lonafarnib; Ritonavir
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Withdrawn
    Start
    Not recorded
    Completion
    Not recorded
    Enrolment
    0

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

    ConditionChronic Delta Hepatitis
    InterventionLonafarnib; Ritonavir
    PhasePhase 2
    Registry statusWithdrawn

  • Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid Laminopathy

    NCT03895528

    Registered record
    Registry ID
    NCT03895528
    Conditions
    Progeria; HGPS
    Interventions
    Lonafarnib
    Study type
    EXPANDED_ACCESS
    Phase
    Not recorded
    Registry status
    Approved for marketing
    Start
    Not recorded
    Completion
    Not recorded
    Enrolment
    Not recorded

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

    ConditionProgeria; HGPS
    InterventionLonafarnib
    PhaseNot recorded
    Registry statusApproved for marketing