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Mebendazole

Benzimidazole derivatives · given by oral · on the market since 1995

5
Leads
31.4M
People
1
Makers
0
How abandoned
01

What it's made of

Each functional group on the molecule does a specific, predictable job.

SETS HOW LONG IT LASTS

Ester

What it does. Blood and tissue enzymes cut this bond within minutes. Whatever was attached falls off.

What it opens up. Deliberate short action, or a delivery trick — attach an ester to sneak a molecule somewhere, then let the body cut it loose at the destination.

SETS HOW LONG IT LASTS

Carbamate

What it does. Cut far more slowly than an ester. When it sits on the right enzyme it jams it for hours rather than seconds.

What it opens up. Long-lasting enzyme blockade — the basis of several nerve and muscle drugs.

CARRIES THE MAIN JOB

Imidazole ring

What it does. Slots directly onto the iron atom at the centre of haem-containing enzymes. That includes the fungal enzyme that builds cell membranes — and also the human liver's main drug-processing enzymes, which is why these compounds interfere with so many other medicines.

What it opens up. Any haem enzyme. The same ring that kills fungi also blocks human steroid synthesis, which is how an antifungal ended up used against hormone-driven disease.

02

Where it can get to

Predicted from molecular weight, lipophilicity, polar surface area and hydrogen-bond donor count. A drug can only act where it distributes, so this constrains which conditions are addressable before any biology is considered. derived

Easily
Systemic circulation
Absorbed from the gut and distributed in plasma.
Easily
Central nervous system
Crosses the blood–brain barrier.
No
Intestinal lumen only
Poorly absorbed. Acts locally in the gut with minimal systemic exposure.
Easily
Skin
Crosses the stratum corneum. Viable as a topical.
Easily
Urinary tract
Cleared unchanged in urine. Local concentration exceeds plasma.
03

What it binds — and whether we can reach it

Binding affinity is only meaningful against the plasma concentration reached at the approved dose. A target requiring higher concentrations cannot be engaged in a patient, whatever the in vitro potency. Most repurposing hypotheses fail at this step, and it is rarely checked before a trial is proposed. measured

Values are pAct — the negative logarithm of the molar concentration at which the drug half-occupies the target. Each whole step is a tenfold difference in potency: 9.0 = 1 nanomolar, 6.0 = 1 micromolar, 5.0 = 10 micromolar. Oral drugs typically reach 0.1–10 micromolar in plasma, so roughly 6.0 is the threshold below which a target cannot be reached at a tolerated dose. Figures come from DrugCentral’s curated binding data.
KDR
Kinase ·
5.44
Out of reach
ABL1
Kinase ·
5.34
Out of reach

This molecule concentrates in urine. Local concentration therefore exceeds plasma, so targets that look unreachable on a plasma reading may still be engaged. assumed

04

New uses, and who is already trying them

One card per candidate indication: the reasoning link by link, the evidence tier that governs how strongly it can be stated, and any registered trials.

5 of the 94 conditions linked to this drug’s targets have been checked against trial records so far. The rest are not ruled out — they are unchecked.

Not yet used for these — 5

chronic myelogenous leukemia, BCR-ABL1 positive

Wide open

It acts on a target linked to this disease; untested, and the direction of effect is unverified.

The condition
A chronic myeloproliferative neoplasm characterized by the expression of the BCR-ABL1 fusion gene. It presents with neutrophilic leukocytosis. It can appear at any age, but it mostly affects middle aged and older individuals. Patients usually present with fatigue, weight loss, anemia, night sweats, and splenomegaly. If untreated, it follows a biphasic or triphasic natural course; an initial indolent chronic phase which is followed by an accelerated phase, a blast phase, or both. Allogeneic stem cell transplantation
ABL1 · ABL proto-oncogene 1, non-receptor tyrosine kinase
Non-receptor tyrosine-protein kinase that plays a role in many key processes linked to cell growth and survival such as cytoskeleton remodeling in response to extracellular stimuli, cell motility and adhesion, receptor endocytosis, autophagy, DNA damage response and apoptosis. Coordinates actin remodeling through tyrosine phosphorylation of proteins controlling cytoskeleton dynamics like WASF3 (involved in branch formation); ANXA1 (involved in membrane anchoring); DBN1, DBNL, CTTN, RAPH1 and ENAH (involved in signa
Nobody has tried this
No registered trial of this drug in this condition.
What would kill it. A null trial, or pharmacokinetic data showing the drug does not reach ABL1 in the relevant tissue at tolerated doses.

acute lymphoblastic leukemia

Wide open

It acts on a target linked to this disease; untested, and the direction of effect is unverified.

The condition
Leukemia with an acute onset, characterized by the presence of lymphoblasts in the bone marrow and the peripheral blood. It includes the acute B lymphoblastic leukemia and acute T lymphoblastic leukemia.
ABL1 · ABL proto-oncogene 1, non-receptor tyrosine kinase
Non-receptor tyrosine-protein kinase that plays a role in many key processes linked to cell growth and survival such as cytoskeleton remodeling in response to extracellular stimuli, cell motility and adhesion, receptor endocytosis, autophagy, DNA damage response and apoptosis. Coordinates actin remodeling through tyrosine phosphorylation of proteins controlling cytoskeleton dynamics like WASF3 (involved in branch formation); ANXA1 (involved in membrane anchoring); DBN1, DBNL, CTTN, RAPH1 and ENAH (involved in signa
Nobody has tried this
No registered trial of this drug in this condition.
What would kill it. A null trial, or pharmacokinetic data showing the drug does not reach ABL1 in the relevant tissue at tolerated doses.

neoplasm

Tried in people

It showed an effect in a human trial that was not followed up.

The condition
A benign or malignant tissue growth resulting from uncontrolled cell proliferation. Benign neoplastic cells resemble normal cells without exhibiting significant cytologic atypia, while malignant cells exhibit overt signs such as dysplastic features, atypical mitotic figures, necrosis, nuclear pleomorphism, and anaplasia. Representative examples of benign neoplasms include papillomas, cystadenomas, and lipomas; malignant neoplasms include carcinomas, sarcomas, lymphomas, and leukemias.
KDR · kinase insert domain receptor
Tyrosine-protein kinase that acts as a cell-surface receptor for VEGFA, VEGFC and VEGFD. Plays an essential role in the regulation of angiogenesis, vascular development, vascular permeability, and embryonic hematopoiesis. Promotes proliferation, survival, migration and differentiation of endothelial cells. Promotes reorganization of the actin cytoskeleton. Isoforms lacking a transmembrane domain, such as isoform 2 and isoform 3, may function as decoy receptors for VEGFA, VEGFC and/or VEGFD. Isoform 2 plays an impor
Phase 3 has run
A Phase 3 trial exists. Read its result before anything else.
NCT02201381 Study of the Safety, Tolerability and Ef · Ph 3
+10 more on ClinicalTrials.gov
What would kill it. A null result in the completed Phase 3.

hepatocellular carcinoma

Animal models only

It reduces disease in animal models; untested in humans.

The condition
A malignant tumor that arises from hepatocytes. Hepatocellular carcinoma is relatively rare in the United States but very common in all African countries south of the Sahara and in Southeast Asia. Most cases are seen in patients over the age of 50 years, but this tumor can also occur in younger individuals and even in children. Hepatocellular carcinoma is more common in males than females and is associated with hepatitis B, hepatitis C, chronic alcohol abuse and cirrhosis. Serum elevation of alpha-fetoprotein occur
KDR · kinase insert domain receptor
Tyrosine-protein kinase that acts as a cell-surface receptor for VEGFA, VEGFC and VEGFD. Plays an essential role in the regulation of angiogenesis, vascular development, vascular permeability, and embryonic hematopoiesis. Promotes proliferation, survival, migration and differentiation of endothelial cells. Promotes reorganization of the actin cytoskeleton. Isoforms lacking a transmembrane domain, such as isoform 2 and isoform 3, may function as decoy receptors for VEGFA, VEGFC and/or VEGFD. Isoform 2 plays an impor
Phase 1 only
Safety and dose-finding studies exist. No efficacy data.
NCT04443049 To Study the Effects of Addition of Mebe · NA · 170 people
What would kill it. A null trial, or pharmacokinetic data showing the drug does not reach KDR in the relevant tissue at tolerated doses.

gastrointestinal stromal tumor

Wide open

It acts on a target linked to this disease; untested, and the direction of effect is unverified.

The condition
Gastrointestinal stromal tumor (GIST) is the most common mesenchymal neoplasm of the gastrointestinal (GI) tract, typically presenting in adults over the age of 40 (mean age 63), and only rarely in children, in various regions of the GI tract, most commonly the stomach or small intestine but also less commonly in the esophagus, appendix, rectum and colon. GISTs can be asymptomatic or present with various non-specific signs, depending on the location and size of tumor, such as loss of appetite, anemia, weight loss,
KDR · kinase insert domain receptor
Tyrosine-protein kinase that acts as a cell-surface receptor for VEGFA, VEGFC and VEGFD. Plays an essential role in the regulation of angiogenesis, vascular development, vascular permeability, and embryonic hematopoiesis. Promotes proliferation, survival, migration and differentiation of endothelial cells. Promotes reorganization of the actin cytoskeleton. Isoforms lacking a transmembrane domain, such as isoform 2 and isoform 3, may function as decoy receptors for VEGFA, VEGFC and/or VEGFD. Isoform 2 plays an impor
Nobody has tried this
No registered trial of this drug in this condition.
What would kill it. A null trial, or pharmacokinetic data showing the drug does not reach KDR in the relevant tissue at tolerated doses.
05

Why nobody will fund it

Only one company makes this. With that many manufacturers the price has fallen to production cost. A company funding a trial could not recover it — competitors could market the same molecule for the new indication immediately, without contributing.

How abandoned it is0 / 100
0 to 100, calculated from manufacturer count, remaining patent or exclusivity, and whether any reformulation route to exclusivity is still open. 100 means no route to commercial return exists. The weights behind it are unsourced.
THE ONE WAY A COMPANY COULD STILL OWN IT
topicalinjectableinhaledmodified-release

A new route or dosage form qualifies for a 505(b)(2) application, which carries three years of exclusivity for that formulation. It is the only remaining route to exclusivity on a generic molecule.

06

What it would cost to find out

Derived, not quoted. Events needed is 4(zα+zβ)²/ln(HR)²; enrolment is that divided by the event rate and follow-up. The 1/ln(HR)² term dominates: halving the detectable effect size roughly quadruples enrolment. derived

0.75
Lower = bigger effect = smaller trial
8.0%
2 yr
$32K
Unsourced placeholder
379
Events needed
3K
Patients
$93M
Trial cost
assumed
$8.45
Per person reached
assumed

Cost per person reached is the relevant metric for a non-commercial funder, since there is no revenue to set against the outlay.

07

Ask for ideas nobody has listed

Reasons from the functional groups and distribution above, excluding conditions already listed. Output is hypotheses for falsification, not findings. Not medical advice.

08

Who might pay for the trial

Trials of off-patent drugs are rarely funded by venture capital or pharma. They are paid for by government grants and disease foundations. Here is who has funded work on this molecule before, what federal mechanisms are open now, and which foundations fund trials in the disease areas it touches. measured

Federal grants that name this medicine
37 grants on NIH RePORTER
3R01AI182301-03S1(NIAID · National Institute of Allergy & Infectious Diseases)Active
$126K (2026)
Genomic approaches to define hookworm population diversity and deworming drug response
PI: Cappello, Michael · Yale University, New Haven, CT · through 2029-01
5R01AI182301-03(NIAID · National Institute of Allergy & Infectious Diseases)Active
$587K (2026)
Genomic approaches to define hookworm population diversity and deworming drug response
PI: Cappello, Michael · Yale University, New Haven, CT · through 2029-01
2P01CA066996-26(NCI · National Cancer Institute)Concluded
$389K (2025)
Molecular basis for microtubule-destabilization dependent MYB degradation
PI: Ebert, Benjamin Levine · Dana-Farber Cancer Inst, Boston, MA · through 2030-08
Open federal programmes that would fund this work

Open notices with an explicit mandate to fund work on approved or off-patent molecules.

RFA-CA-25-033National Institutes of HealthForecast — not open yet
Glioblastoma Therapeutics Network (GTN; U19 Clinical Trial Required)
RFA-FD-25-020Food and Drug Administrationcloses 05/16/2028
Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet
FOR-FD-25-020Food and Drug AdministrationForecast — not open yet
Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R
FOR-FD-25-001Food and Drug AdministrationForecast — not open yet
Clinical Trials Addressing Unmet Needs of Rare Neurodegenerative Diseases (R01
RFA-TR-25-002National Institutes of Healthcloses 07/02/2027
Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not
PAR-25-374National Institutes of Healthcloses 05/07/2028
Translational Bioinformatics and Experimental Approaches to Advance Drug Repos
HT942526PRCRPCTADefense Health Agency Contracting Activity -closes 10/05/2026
DoW Peer Reviewed Cancer, Clinical Trial Award
PAR-26-049National Institutes of HealthForecast — not open yet
Establishing Pediatric CNS Pharmacodynamic Measures as Tools to Enable Psychia
Showing 8 of 990 currently open under this category on Grants.gov, matched to the disease areas this drug touches. derived Browse all 990 on Grants.gov ↗
Funders beyond the federal system

Grants.gov lists US federal money. Many off-patent drug trials are supported by disease foundations and nonprofits. These are matched to the body systems this drug touches. assumed

Matched by disease area, not by molecule — a foundation appearing here funds work in a field this drug touches.
09

Who manufactures it

Companies holding an approved abbreviated application for this molecule in the United States, from the FDA Orange Book. Supply already exists at scale, so a trial would not need a manufacturing programme — but any of these could also be a partner, or an objector.

Impax Laboratories Inc
1
Manufacturers
companies with their own approval
1
Approved products
distinct strengths and forms
1
Routes
oral
1995
Oldest product still sold
approval year of the earliest product still on the market
ALSO SOLD AS
EMVERM

Nothing on this page is medical advice. Candidate uses are unproven hypotheses derived from public data; established uses are listed for verification only. Figures carry their provenance inline — anything marked assumed or derived is not a measurement.

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