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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.

  • Study to Determine Optimal Dose and Evaluate Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS)

    NCT06775041 · start 2025-01-13

    Registered record
    Registry ID
    NCT06775041
    Conditions
    Hutchinson-Gilford Progeria Syndrome
    Interventions
    Progerinin; Lonafarnib
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Active not recruiting
    Start
    2025-01-13
    Completion
    2026-03
    Enrolment
    10

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

    ConditionHutchinson-Gilford Progeria Syndrome
    InterventionProgerinin; Lonafarnib
    PhasePhase 2
    Registry statusActive not recruiting

  • Peginterferon Lambda and Lonafarnib Boosted With Ritonavir 48-Week Combination Therapy for Delta Hepatitis

    NCT05953545 · start 2024-05-22

    Registered record
    Registry ID
    NCT05953545
    Conditions
    Chronic Hepatitis Delta
    Interventions
    Peginterferon Lambda
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Withdrawn
    Start
    2024-05-22
    Completion
    2024-05-22
    Enrolment
    0

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

    ConditionChronic Hepatitis Delta
    InterventionPeginterferon Lambda
    PhasePhase 2
    Registry statusWithdrawn

  • Once Daily Dosing of Lonafarnib Co-administered With Ritonavir for Treatment of Chronic Hepatitis D Virus Infection

    NCT05229991 · start 2021-05-15

    Registered record
    Registry ID
    NCT05229991
    Conditions
    Hepatitis D, Chronic
    Interventions
    Lonafarnib; Ritonavir
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Completed
    Start
    2021-05-15
    Completion
    2025-02-05
    Enrolment
    10

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

    ConditionHepatitis D, Chronic
    InterventionLonafarnib; Ritonavir
    PhasePhase 3
    Registry statusCompleted

  • Study of the Efficacy and Safety of Lonafarnib / Ritonavir With and Without Pegylated Interferon -Alfa-2a

    NCT03719313 · start 2018-12-01

    Registered record
    Registry ID
    NCT03719313
    Conditions
    Hepatitis Delta Virus
    Interventions
    Lonafarnib; Ritonavir; PEG IFN-alfa-2a; Placebo Lonafarnib; Placebo Ritonavir
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Completed
    Start
    2018-12-01
    Completion
    2023-03-24
    Enrolment
    407

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

    ConditionHepatitis Delta Virus
    InterventionLonafarnib; Ritonavir; PEG IFN-alfa-2a; Placebo Lonafarnib +1 more
    PhasePhase 3
    Registry statusCompleted

  • Treatment of Chronic Delta Hepatitis With Lonafarnib, Ritonavir and Lambda Interferon

    NCT03600714 · start 2018-08-01

    Registered record
    Registry ID
    NCT03600714
    Conditions
    Liver Disease; Hepatitis D
    Interventions
    Peg-interferon lambda; Lonafarnib; Ritonavir
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2018-08-01
    Completion
    2020-08-11
    Enrolment
    26

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

    ConditionLiver Disease; Hepatitis D
    InterventionPeg-interferon lambda; Lonafarnib; Ritonavir
    PhasePhase 2
    Registry statusCompleted

  • Phase I/II Trial of Everolimus in Combination With Lonafarnib in Progeria

    NCT02579044 · start 2015-12

    Registered record
    Registry ID
    NCT02579044
    Conditions
    Progeria
    Interventions
    Everolimus and lonafarnib
    Study type
    INTERVENTIONAL
    Phase
    Phase 1 / Phase 2
    Registry status
    Enrolling by invitation
    Start
    2015-12
    Completion
    2027-03
    Enrolment
    80

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

    ConditionProgeria
    InterventionEverolimus and lonafarnib
    PhasePhase 1 / Phase 2
    Registry statusEnrolling by invitation

  • Titrating-Dose of Lonafarnib in Combination With Ritonavir

    NCT02527707 · start 2015-09

    Registered record
    Registry ID
    NCT02527707
    Conditions
    Chronic Delta Hepatitis
    Interventions
    lonafarnib; Ritonavir
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2015-09
    Completion
    2017-02-09
    Enrolment
    15

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

    ConditionChronic Delta Hepatitis
    Interventionlonafarnib; Ritonavir
    PhasePhase 2
    Registry statusCompleted

  • Treatment of Chronic Delta Hepatitis With Lonafarnib and Ritonavir

    NCT02511431 · start 2015-07-29

    Registered record
    Registry ID
    NCT02511431
    Conditions
    Hepatitis D
    Interventions
    Lonafarnib; Ritonavir; Placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2015-07-29
    Completion
    2017-02-23
    Enrolment
    22

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

    ConditionHepatitis D
    InterventionLonafarnib; Ritonavir; Placebo
    PhasePhase 2
    Registry statusCompleted

  • Lonafarnib Boosted With Ritonavir With and Without Peginterferon Alfa-2a (PEG IFN-a) in HDV (LOWR-2)

    NCT02430194 · start 2014-12

    Registered record
    Registry ID
    NCT02430194
    Conditions
    Chronic Hepatitis D Infection
    Interventions
    lonafarnib; ritonavir; Pegylated interferon-alfa-2a
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2014-12
    Completion
    2017-06-15
    Enrolment
    55

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

    ConditionChronic Hepatitis D Infection
    Interventionlonafarnib; ritonavir; Pegylated interferon-alfa-2a
    PhasePhase 2
    Registry statusCompleted

  • Lonafarnib With and Without Ritonavir in HDV (LOWR-1)

    NCT02430181 · start 2014-11

    Registered record
    Registry ID
    NCT02430181
    Conditions
    Chronic Hepatitis D Infection
    Interventions
    lonafarnib; peginterferon alfa-2a; ritonavir
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2014-11
    Completion
    2015-11
    Enrolment
    21

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

    ConditionChronic Hepatitis D Infection
    Interventionlonafarnib; peginterferon alfa-2a; ritonavir
    PhasePhase 2
    Registry statusCompleted

  • Lonafarnib for Chronic Hepatitis D

    NCT01495585 · start 2011-12

    Registered record
    Registry ID
    NCT01495585
    Conditions
    Hepatitis D
    Interventions
    Lonafarnib; Placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2011-12
    Completion
    2015-04
    Enrolment
    14

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

    ConditionHepatitis D
    InterventionLonafarnib; Placebo
    PhasePhase 2
    Registry statusCompleted

  • Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria

    NCT00916747 · start 2009-08

    Registered record
    Registry ID
    NCT00916747
    Conditions
    Progeria
    Interventions
    Lonafarnib, Zoledronic Acid, and Pravastatin
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Unknown
    Start
    2009-08
    Completion
    2023-12
    Enrolment
    85

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

    ConditionProgeria
    InterventionLonafarnib, Zoledronic Acid, and Pravastatin
    PhasePhase 2
    Registry statusUnknown

  • Predicting Response and Toxicity in Patients Receiving Lonafarnib for Breast Cancer

    NCT01232881 · start 2009-08

    Registered record
    Registry ID
    NCT01232881
    Conditions
    Breast Cancer
    Interventions
    Tumor Sample; Serum Sample
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Terminated
    Start
    2009-08
    Completion
    2010-11
    Enrolment
    27

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

    ConditionBreast Cancer
    InterventionTumor Sample; Serum Sample
    PhaseNot recorded
    Registry statusTerminated

  • A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria

    NCT00879034 · start 2009-03

    Registered record
    Registry ID
    NCT00879034
    Conditions
    Progeria; Hutchinson-Gilford Syndrome
    Interventions
    Lonafarnib; Zoledronic Acid; Pravastatin
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2009-03
    Completion
    2009-04
    Enrolment
    5

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

    ConditionProgeria; Hutchinson-Gilford Syndrome
    InterventionLonafarnib; Zoledronic Acid; Pravastatin
    PhasePhase 2
    Registry statusCompleted

  • Lonafarnib in Metastatic Breast Cancer

    NCT00773474 · start 2008-10

    Registered record
    Registry ID
    NCT00773474
    Conditions
    Metastatic Breast Cancer
    Interventions
    Lonafarnib
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Terminated
    Start
    2008-10
    Completion
    2010-11
    Enrolment
    29

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

    ConditionMetastatic Breast Cancer
    InterventionLonafarnib
    PhasePhase 2
    Registry statusTerminated