Neoplasm
diseaseOn this page
Also known as cell process diseasedisease of cellular proliferationneoplasianeoplasm (disease)neoplastic diseaseneoplastic growthother neoplasmtumortumor diseasetumourtumour disease
Summary
Neoplasm (MONDO:0005070) is a cancer (an umbrella term covering 48 Mondo subtypes) with 2 cohort genes (17 GWAS associations across 42 studies; 2 CIViC-evidence somatic drivers; 3 ClinVar predisposition records) and 1,638 clinical trials. Top therapeutic interventions include dostarlimab, epoetin alfa, and dacomitinib anhydrous.
At a glance
- Classification: Cancer
- Umbrella term: 48 Mondo subtypes
- Cohort genes: 2
- GWAS associations: 17
- ClinVar variants: 3
- Clinical trials: 1,638
Clinical features
No curated clinical features (Orphanet) for this disease.
Identifiers
Disease identifiers
| Field | Value |
|---|---|
| Canonical name | neoplasm |
| Mondo ID | MONDO:0005070 |
| EFO | EFO:0000616 |
| MeSH | D009369 |
| DOID | DOID:14566 |
| ICD-10-CM | C00-D49, C7A-C7A, C7B-C7B |
| NCIT | C3262 |
| SNOMED CT | 55342001 |
| UMLS | C0027651 |
| MedGen | 10294 |
| Is cancer (heuristic) | yes |
Also known as: cell process disease · disease of cellular proliferation · neoplasia · neoplasm · neoplasm (disease) · neoplastic disease · neoplastic growth · other neoplasm · tumor · tumor disease · tumour · tumour disease
Data availability: 3 ClinVar variants · 17 GWAS associations (42 studies) · 1 HPO phenotype · 43 cell lines.
Disease family
An umbrella term covering 48 Mondo subtypes.
Classification path: disease › human disease › disease by etiologic mechanism › cancer or benign tumor › neoplastic disease or syndrome › neoplasm
Related subtypes (2): neoplastic syndrome, secondary neoplasm
Subtypes (48): pre-malignant neoplasm, endocrine gland neoplasm, giant cell tumor, hematopoietic and lymphoid system neoplasm, skin neoplasm, mesenchymal cell neoplasm, epidural spinal canal neoplasm, skeletal muscle neoplasm, trophoblastic neoplasm, cancer, germ cell tumor, benign neoplasm, upper aerodigestive tract neoplasm, histiocytoma, embryonal neoplasm, head and neck neoplasm, epithelial neoplasm, reproductive system neoplasm, non-seminomatous lesion, odontogenic cyst, phosphaturic mesenchymal tumor, thyroglossal duct cyst, hamartoma, mesenchymoma, mesothelial neoplasm, peritoneal neoplasm, virus associated tumor, nail tumor, respiratory tract neoplasm, spindle cell neoplasm, mixed neoplasm, urinary system neoplasm, cystic neoplasm, childhood neoplasm, melanocytic neoplasm, digestive system neoplasm, nervous system neoplasm, neoplasm of thorax, connective tissue neoplasm, bronchial adenomas/carcinoids childhood, diffuse idiopathic pulmonary neuroendocrine cell hyperplasia, erythroplakia, retroperitoneal neoplasm, cardiovascular neoplasm, dermoid or epidermoid cyst of the central nervous system, connective and soft tissue neoplasm, NTRK fusion positive cancer, RET fusion positive cancer
Genetics & variants
GWAS landscape
17 GWAS associations across 42 studies. Top hits map to 6 distinct genes (as reported by GWAS).
Top associations by p-value
| rsID | p-value | Gene | Risk allele | Odds ratio |
|---|---|---|---|---|
| rs12203592 | 4e-29 | IRF4 | C | 0.2 |
| rs1805007 | 1e-25 | MC1R | C | 0.23 |
| rs16891982 | 2e-14 | SLC45A2 | C | 0.29 |
| rs62209647 | 4e-13 | TPM3P2 - PIGPP3 | G | 0.14 |
| rs189122415 | 2e-12 | LINC03090 | T | 2.12 |
| rs140758620 | 1e-11 | TYR - NOX4 | G | 0.09 |
| rs11018578 | 4e-11 | NOX4 | T | 0.09 |
| rs189086286 | 2e-07 | FANCD2P2 | ? |
Top studies (by case count)
| Study | Lead author | Year | Cases | Controls | Title |
|---|---|---|---|---|---|
| GCST90475606 | Verma A | 2024 | 18,252 | 403,292 | Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program. |
| GCST90435628 | Zhou W | 2018 | 15,979 | 370,604 | Efficiently controlling for case-control imbalance and sample relatedness in large-scale genetic association studies. |
| GCST90475621 | Verma A | 2024 | 12,830 | 416,346 | Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program. |
| GCST90081355 | Backman JD | 2021 | 6,926 | 381,003 | Exome sequencing and analysis of 454,787 UK Biobank participants. |
| GCST90085341 | Backman JD | 2021 | 6,926 | 381,003 | Exome sequencing and analysis of 454,787 UK Biobank participants. |
| GCST90475602 | Verma A | 2024 | 6,441 | 434,830 | Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program. |
| GCST90477242 | Verma A | 2024 | 3,879 | 112,604 | Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program. |
| GCST90479824 | Verma A | 2024 | 3,879 | 112,604 | Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program. |
| GCST90079104 | Backman JD | 2021 | 3,434 | 37,571 | Exome sequencing and analysis of 454,787 UK Biobank participants. |
| GCST90083090 | Backman JD | 2021 | 3,434 | 37,571 | Exome sequencing and analysis of 454,787 UK Biobank participants. |
Variant details and genetic-evidence tiers
Tier distribution (top 50 variants)
| Tier | Variants |
|---|---|
| Tier 1: coding | 2 |
| Tier 2: splice/UTR | 1 |
| Tier 3: regulatory | 0 |
| Tier 4: intronic/intergenic | 5 |
MAF distribution
| Bucket | Variants |
|---|---|
| common (>=0.05) | 5 |
| low_freq (0.01-0.05) | 1 |
| rare (<0.01) | 1 |
| unknown | 1 |
Functional consequences
| Consequence | Count |
|---|---|
| intron_variant | 4 |
| missense_variant | 2 |
| intergenic_variant | 1 |
| 3_prime_UTR_variant | 1 |
Top variants
| rsID | Chr | Pos | Alleles | MAF | Consequence | Gene | p-value | Tier |
|---|---|---|---|---|---|---|---|---|
| rs12203592 | 6 | 396321 | C>G,T | 0.16 | intron_variant | IRF4 | 4e-29 | Tier 4: intronic/intergenic |
| rs1805007 | 16 | 89919709 | C>A,G,T | 0.082 | missense_variant | MC1R | 1e-25 | Tier 1: coding |
| rs16891982 | 5 | 33951588 | C>A,G | 0.037 | missense_variant | SLC45A2 | 2e-14 | Tier 1: coding |
| rs62209647 | 20 | 33917852 | G>C | 0.08 | intron_variant | TPM3P2 - PIGPP3 | 4e-13 | Tier 4: intronic/intergenic |
| rs189122415 | 8 | 101130834 | T>C | 0.002 | intron_variant | LINC03090 | 2e-12 | Tier 4: intronic/intergenic |
| rs140758620 | 11 | 89318601 | G>C,T | 0.306 | intergenic_variant | TYR - NOX4 | 1e-11 | Tier 4: intronic/intergenic |
| rs11018578 | 11 | 89324933 | T>C,G | 0.275 | 3_prime_UTR_variant | NOX4 | 4e-11 | Tier 2: splice/UTR |
| rs189086286 | 3 | 11868551 | C>T | intron_variant | FANCD2P2 | 2e-07 | Tier 4: intronic/intergenic |
ClinVar germline variants
3 retrieved; paginated sample, class counts are floors:
3 pathogenic
| ClinVar | Variant (HGVS) | Gene | Classification | Review |
|---|---|---|---|---|
| 560351 | NM_000051.4(ATM):c.5696dup (p.Cys1899fs) | ATM | Pathogenic | no assertion criteria provided |
| 560352 | NM_000051.4(ATM):c.2441del (p.Asp814fs) | ATM | Pathogenic | criteria provided, single submitter |
| 570837 | NM_000548.5(TSC2):c.2838-122G>A | TSC2 | Pathogenic | criteria provided, multiple submitters, no conflicts |
Genes & proteins
Mendelian disease overlap and somatic drivers
GenCC: 0 · Orphanet: 14 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0
Somatic driver evidence (intOGen + CIViC, cohort fanout)
| Gene | intOGen role | Cancer types | CIViC |
|---|---|---|---|
| TSC2 | LoF | ANGS,BLCA,CHOL,CHRCC,ESCA,HCC,PANCREAS,PANET,SCLC,STAD | CIViC #47 |
| ATM | LoF | BLCA,BRCA,CCRCC,CHOL,CLLSLL,COAD,COADREAD,ESCA,HCC,LUAD,LUSC,MEL,NSCLC,PAAD,PANCREAS,PANET,PCM,PLMESO,PRAD,PROSTATE,STAD,UCEC,UTUC,WDTC | CIViC #69 |
Orphanet rare-disease linkage (cohort genes)
| Gene | Orphanet ID | Rare disease |
|---|---|---|
| TSC2 | Orphanet:210159 | Adult hepatocellular carcinoma |
| TSC2 | Orphanet:269001 | Isolated focal cortical dysplasia type IIa |
| TSC2 | Orphanet:269008 | Isolated focal cortical dysplasia type IIb |
| TSC2 | Orphanet:538 | Lymphangioleiomyomatosis |
| TSC2 | Orphanet:805 | Tuberous sclerosis complex |
| TSC2 | Orphanet:88924 | Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis |
| ATM | Orphanet:100 | Ataxia-telangiectasia |
| ATM | Orphanet:1331 | Familial prostate cancer |
| ATM | Orphanet:145 | Hereditary breast and/or ovarian cancer syndrome |
| ATM | Orphanet:227535 | Hereditary breast cancer |
| ATM | Orphanet:370109 | Ataxia-telangiectasia variant |
| ATM | Orphanet:440437 | Familial colorectal cancer Type X |
| ATM | Orphanet:52416 | Mantle cell lymphoma |
| ATM | Orphanet:67038 | B-cell chronic lymphocytic leukemia |
Cohort genes → proteins
2 cohort genes, 2 distinct canonical proteins.
Evidence partition
| Subset | Genes |
|---|---|
| multi_evidence | 2 |
Cohort genes (full)
| Symbol | HGNC | Ensembl | UniProt | Name | Evidence |
|---|---|---|---|---|---|
| TSC2 | HGNC:12363 | ENSG00000103197 | P49815 | Tuberin | clinvar |
| ATM | HGNC:795 | ENSG00000149311 | Q13315 | Serine-protein kinase ATM | clinvar |
Cohort function summary
Lead sentence per gene, UniProt-curated.
| Symbol | Protein name | Function (lead sentence) |
|---|---|---|
| TSC2 | Tuberin | Catalytic component of the TSC-TBC complex, a multiprotein complex that acts as a negative regulator of the canonical mTORC1 complex, an evolutionarily conserved central nutrient sensor that stimulates anabolic reactions and macromolecule… |
| ATM | Serine-protein kinase ATM | Serine/threonine protein kinase which activates checkpoint signaling upon double strand breaks (DSBs), apoptosis and genotoxic stresses such as ionizing ultraviolet A light (UVA), thereby acting as a DNA damage sensor. |
Protein-family classification
Druggable: 1 · Difficult: 0 · Unknown: 1 · Druggable fraction: 0.5
Family distribution
Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.
| Family | Genes | Fold | FDR |
|---|---|---|---|
| Kinase | 1 | 13.9× | 0.142 |
| Other/Unknown | 1 | 0.9× | 0.805 |
Per-gene assignment
| Symbol | Family | Druggable? | EC | InterPro (top 3) |
|---|---|---|---|---|
| TSC2 | Other/Unknown | no | Rap/Ran_GAP_dom, Tuberin, ARM-like | |
| ATM | Kinase | yes | 2.7.11.1 | PI3/4_kinase_cat_dom, PIK-rel_kinase_FAT, FATC_dom |
Expression context
Cohort genes with no expression data: 0.
2 cohort genes are a single-cell marker in ≥1 SCXA experiment.
Breadth distribution (Bgee present_calls)
| Bucket | Genes |
|---|---|
| narrow (1-5 tissues) | 0 |
| moderate (6-20) | 0 |
| broad (>20) | 2 |
| unknown | 0 |
Top tissues across cohort
| Tissue | Cohort genes |
|---|---|
| cerebellar cortex | 1 |
| cerebellar hemisphere | 1 |
| right hemisphere of cerebellum | 1 |
| calcaneal tendon | 1 |
| colonic epithelium | 1 |
| corpus callosum | 1 |
Per-gene tissue summary (top 30)
| Symbol | Bgee breadth | FANTOM5 breadth | SCXA | Top tissues |
|---|---|---|---|---|
| TSC2 | 282 | ubiquitous | marker | right hemisphere of cerebellum, cerebellar hemisphere, cerebellar cortex |
| ATM | 286 | ubiquitous | marker | calcaneal tendon, colonic epithelium, corpus callosum |
Protein interactions among cohort
Intra-cohort edges: 0.
Hub genes (top 10 by interactor count)
| Symbol | Interactor count |
|---|---|
| ATM | 7,383 |
| TSC2 | 4,135 |
Structural data
PDB: 2 · AlphaFold-only: 0 · No structure: 0
Cohort genes with PDB structures (top 30)
| Symbol | UniProt | PDB entries |
|---|---|---|
| ATM | Q13315 | 14 |
| TSC2 | P49815 | 2 |
Function
Pathway analysis
Distinct Reactome pathways touched by cohort: 67. Enrichment computed across 2 evidence-associated genes (2 with Reactome annotation).
Pathways by enrichment
Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 2 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.
| Pathway | Cohort genes | Fold | FDR | Sample cohort genes |
|---|---|---|---|---|
| Macroautophagy | 2 | 115.3× | 0.005 | TSC2, ATM |
| Inhibition of TSC complex formation by AKT (PKB) | 1 | 1142.0× | 0.012 | TSC2 |
| Sensing of DNA Double Strand Breaks | 1 | 951.7× | 0.012 | ATM |
| TP53 Regulates Transcription of Caspase Activators and Caspases | 1 | 475.8× | 0.012 | ATM |
| Pexophagy | 1 | 475.8× | 0.012 | ATM |
| Defective homologous recombination repair (HRR) due to PALB2 loss of function | 1 | 475.8× | 0.012 | ATM |
| AKT phosphorylates targets in the cytosol | 1 | 407.9× | 0.012 | TSC2 |
| Diseases of DNA Double-Strand Break Repair | 1 | 407.9× | 0.012 | ATM |
| Defective homologous recombination repair (HRR) due to BRCA2 loss of function | 1 | 407.9× | 0.012 | ATM |
| Stabilization of p53 | 1 | 380.7× | 0.012 | ATM |
| p53-Dependent G1 DNA Damage Response | 1 | 356.9× | 0.012 | ATM |
| p53-Dependent G1/S DNA damage checkpoint | 1 | 356.9× | 0.012 | ATM |
| G1/S DNA Damage Checkpoints | 1 | 335.9× | 0.012 | ATM |
| Resolution of D-Loop Structures | 1 | 317.2× | 0.012 | ATM |
| Diseases of DNA repair | 1 | 285.5× | 0.012 | ATM |
| TP53 Regulates Transcription of Cell Death Genes | 1 | 271.9× | 0.012 | ATM |
| TP53 Regulates Transcription of Genes Involved in Cytochrome C Release | 1 | 271.9× | 0.012 | ATM |
| Regulation of TP53 Activity through Methylation | 1 | 271.9× | 0.012 | ATM |
| Regulation of TP53 Expression and Degradation | 1 | 259.6× | 0.012 | ATM |
| DNA Double Strand Break Response | 1 | 237.9× | 0.012 | ATM |
| Impaired BRCA2 binding to PALB2 | 1 | 228.4× | 0.012 | ATM |
| Constitutive Signaling by AKT1 E17K in Cancer | 1 | 211.5× | 0.012 | TSC2 |
| Defective homologous recombination repair (HRR) due to BRCA1 loss of function | 1 | 211.5× | 0.012 | ATM |
| Defective HDR through Homologous Recombination Repair (HRR) due to PALB2 loss of BRCA1 binding function | 1 | 211.5× | 0.012 | ATM |
| Defective HDR through Homologous Recombination Repair (HRR) due to PALB2 loss of BRCA2/RAD51/RAD51C binding function | 1 | 211.5× | 0.012 | ATM |
| Cellular response to heat stress | 1 | 196.9× | 0.012 | ATM |
| Energy dependent regulation of mTOR by LKB1-AMPK | 1 | 196.9× | 0.012 | TSC2 |
| Resolution of D-loop Structures through Synthesis-Dependent Strand Annealing (SDSA) | 1 | 196.9× | 0.012 | ATM |
| Homologous DNA Pairing and Strand Exchange | 1 | 190.3× | 0.012 | ATM |
| Homology Directed Repair | 1 | 154.3× | 0.013 | ATM |
GO biological processes by enrichment
Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 2 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.
| GO term | Cohort genes | Fold | FDR | Sample cohort genes |
|---|---|---|---|---|
| negative regulation of TORC1 signaling | 2 | 324.1× | 8e-04 | TSC2, ATM |
| establishment of RNA localization to telomere | 1 | 4213.0× | 0.003 | ATM |
| establishment of protein-containing complex localization to telomere | 1 | 4213.0× | 0.003 | ATM |
| positive regulation of telomerase catalytic core complex assembly | 1 | 4213.0× | 0.003 | ATM |
| heart development | 2 | 78.8× | 0.003 | TSC2, ATM |
| regulation of cell cycle | 2 | 74.6× | 0.003 | TSC2, ATM |
| pre-B cell allelic exclusion | 1 | 2808.7× | 0.004 | ATM |
| cellular response to nitrosative stress | 1 | 2808.7× | 0.004 | ATM |
| peptidyl-serine autophosphorylation | 1 | 1685.2× | 0.005 | ATM |
| negative regulation of telomere capping | 1 | 1685.2× | 0.005 | ATM |
| regulation of telomere maintenance via telomerase | 1 | 1404.3× | 0.005 | ATM |
| positive regulation of telomere maintenance via telomere lengthening | 1 | 1404.3× | 0.005 | ATM |
| lipoprotein catabolic process | 1 | 1203.7× | 0.005 | ATM |
| V(D)J recombination | 1 | 1053.2× | 0.005 | ATM |
| meiotic telomere clustering | 1 | 936.2× | 0.005 | ATM |
| female meiotic nuclear division | 1 | 842.6× | 0.005 | ATM |
| regulation of insulin receptor signaling pathway | 1 | 842.6× | 0.005 | TSC2 |
| histone mRNA catabolic process | 1 | 842.6× | 0.005 | ATM |
| cellular response to X-ray | 1 | 842.6× | 0.005 | ATM |
| DNA double-strand break processing | 1 | 766.0× | 0.005 | ATM |
| negative regulation of mitophagy | 1 | 766.0× | 0.005 | TSC2 |
| anoikis | 1 | 648.1× | 0.006 | TSC2 |
| regulation of autophagosome assembly | 1 | 561.7× | 0.006 | ATM |
| pexophagy | 1 | 526.6× | 0.006 | ATM |
| regulation of cellular response to heat | 1 | 526.6× | 0.006 | ATM |
| positive regulation of DNA damage response, signal transduction by p53 class mediator | 1 | 495.6× | 0.006 | ATM |
| replicative senescence | 1 | 495.6× | 0.006 | ATM |
| negative regulation of B cell proliferation | 1 | 468.1× | 0.006 | ATM |
| oocyte development | 1 | 468.1× | 0.006 | ATM |
| cellular response to stress | 1 | 421.3× | 0.007 | ATM |
Therapeutics
Drugs indicated for this disease
300 approved, 136 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.
| Drug | Development status |
|---|---|
| Abarelix | Approved (phase 4) |
| Abemaciclib | Approved (phase 4) |
| Abiraterone | Approved (phase 4) |
| Acalabrutinib | Approved (phase 4) |
| Adagrasib | Approved (phase 4) |
| Aflibercept | Approved (phase 4) |
| Aldesleukin | Approved (phase 4) |
| Alectinib | Approved (phase 4) |
| Alitretinoin | Approved (phase 4) |
| Allopurinol | Approved (phase 4) |
| Alpelisib | Approved (phase 4) |
| Altretamine | Approved (phase 4) |
| Aminoglutethimide | Approved (phase 4) |
| Aminolevulinic Acid | Approved (phase 4) |
| Amivantamab | Approved (phase 4) |
| Amsacrine | Approved (phase 4) |
| Anagrelide | Approved (phase 4) |
| Anastrozole | Approved (phase 4) |
| Apalutamide | Approved (phase 4) |
| Aprepitant | Approved (phase 4) |
| Arsenic Trioxide | Approved (phase 4) |
| Asciminib | Approved (phase 4) |
| Asparaginase | Approved (phase 4) |
| Atezolizumab | Approved (phase 4) |
| Avapritinib | Approved (phase 4) |
| Avelumab | Approved (phase 4) |
| Axitinib | Approved (phase 4) |
| Azacitidine | Approved (phase 4) |
| Belantamab Mafodotin | Approved (phase 4) |
| Belinostat | Approved (phase 4) |
| Belzutifan | Approved (phase 4) |
| Bendamustine | Approved (phase 4) |
| Bevacizumab | Approved (phase 4) |
| Bexarotene | Approved (phase 4) |
| Bicalutamide | Approved (phase 4) |
| Binimetinib | Approved (phase 4) |
| Bleomycin | Approved (phase 4) |
| Blinatumomab | Approved (phase 4) |
| Bortezomib | Approved (phase 4) |
| Brentuximab Vedotin | Approved (phase 4) |
| Brigatinib | Approved (phase 4) |
| Buserelin | Approved (phase 4) |
| Busulfan | Approved (phase 4) |
| Cabazitaxel | Approved (phase 4) |
| Cabozantinib | Approved (phase 4) |
| Calaspargase Pegol | Approved (phase 4) |
| Capecitabine | Approved (phase 4) |
| Capivasertib | Approved (phase 4) |
| Capmatinib | Approved (phase 4) |
| Carboplatin | Approved (phase 4) |
| Carfilzomib | Approved (phase 4) |
| Carmustine | Approved (phase 4) |
| Catumaxomab | Approved (phase 4) |
| Celecoxib | Approved (phase 4) |
| Cemiplimab | Approved (phase 4) |
| Ceritinib | Approved (phase 4) |
| Cetuximab | Approved (phase 4) |
| Chlorambucil | Approved (phase 4) |
| Cisplatin | Approved (phase 4) |
| Cladribine | Approved (phase 4) |
| Clofarabine | Approved (phase 4) |
| Clonidine | Approved (phase 4) |
| Cobimetinib | Approved (phase 4) |
| Copanlisib | Approved (phase 4) |
| Crizotinib | Approved (phase 4) |
| Cytarabine | Approved (phase 4) |
| Dabrafenib | Approved (phase 4) |
| Dacarbazine | Approved (phase 4) |
| Dactinomycin | Approved (phase 4) |
| Daratumumab | Approved (phase 4) |
| Darbepoetin Alfa | Approved (phase 4) |
| Darolutamide | Approved (phase 4) |
| Dasatinib Anhydrous | Approved (phase 4) |
| Daunorubicin | Approved (phase 4) |
| Decitabine | Approved (phase 4) |
| Denileukin Diftitox | Approved (phase 4) |
| Dexamethasone | Approved (phase 4) |
| Diethylstilbestrol | Approved (phase 4) |
| Diethylstilbestrol Diphosphate | Approved (phase 4) |
| Dinutuximab | Approved (phase 4) |
| Dostarlimab | Approved (phase 4) |
| Doxorubicin | Approved (phase 4) |
| Dronabinol | Approved (phase 4) |
| Durvalumab | Approved (phase 4) |
| Duvelisib | Approved (phase 4) |
| Efbemalenograstim Alfa | Approved (phase 4) |
| Eflapegrastim | Approved (phase 4) |
| Elapegademase | Approved (phase 4) |
| Elotuzumab | Approved (phase 4) |
| Elranatamab | Approved (phase 4) |
| Encorafenib | Approved (phase 4) |
| Enfortumab Vedotin | Approved (phase 4) |
| Entrectinib | Approved (phase 4) |
| Enzalutamide | Approved (phase 4) |
| Epcoritamab | Approved (phase 4) |
| Epirubicin | Approved (phase 4) |
| Erdafitinib | Approved (phase 4) |
| Eribulin | Approved (phase 4) |
| Ethinyl Estradiol | Approved (phase 4) |
| Ethiodized Oil | Approved (phase 4) |
| Etoposide | Approved (phase 4) |
| Everolimus | Approved (phase 4) |
| Exemestane | Approved (phase 4) |
| Fedratinib | Approved (phase 4) |
| Fentanyl | Approved (phase 4) |
| Filgrastim | Approved (phase 4) |
| Floxuridine | Approved (phase 4) |
| Fludarabine Phosphate | Approved (phase 4) |
| Fluorouracil | Approved (phase 4) |
| Fluoxymesterone | Approved (phase 4) |
| Flutamide | Approved (phase 4) |
| Fruquintinib | Approved (phase 4) |
| Fulvestrant | Approved (phase 4) |
| Futibatinib | Approved (phase 4) |
| Gefitinib | Approved (phase 4) |
| Gemcitabine | Approved (phase 4) |
| Gemtuzumab Ozogamicin | Approved (phase 4) |
| Gilteritinib | Approved (phase 4) |
| Glasdegib | Approved (phase 4) |
| Glatiramer Acetate | Approved (phase 4) |
| Glofitamab | Approved (phase 4) |
| Goserelin | Approved (phase 4) |
| Granisetron | Approved (phase 4) |
| Histrelin | Approved (phase 4) |
| Hydrocortisone | Approved (phase 4) |
| Hydroxyurea | Approved (phase 4) |
| INTERFERON ALFA-2A | Approved (phase 4) |
| INTERFERON ALFA-2B | Approved (phase 4) |
| INTERFERON ALFACON-1 | Approved (phase 4) |
| INTERFERON BETA-1A | Approved (phase 4) |
| INTERFERON BETA-1B | Approved (phase 4) |
| INTERFERON GAMMA-1B | Approved (phase 4) |
| Ibrutinib | Approved (phase 4) |
| Idarubicin | Approved (phase 4) |
| Idecabtagene Vicleucel | Approved (phase 4) |
| Idelalisib | Approved (phase 4) |
| Ifosfamide | Approved (phase 4) |
| Infigratinib | Approved (phase 4) |
| Inotuzumab Ozogamicin | Approved (phase 4) |
| Interferon Alfa | Approved (phase 4) |
| Interferon Beta | Approved (phase 4) |
| Ipilimumab | Approved (phase 4) |
| Irinotecan | Approved (phase 4) |
| Isatuximab | Approved (phase 4) |
| Ixabepilone | Approved (phase 4) |
| Ixazomib | Approved (phase 4) |
| Larotrectinib | Approved (phase 4) |
| Lenograstim | Approved (phase 4) |
| Lenvatinib | Approved (phase 4) |
| Letrozole | Approved (phase 4) |
| Leuprolide | Approved (phase 4) |
| Lipegfilgrastim | Approved (phase 4) |
| Lomustine | Approved (phase 4) |
| Loncastuximab Tesirine | Approved (phase 4) |
| Lorlatinib | Approved (phase 4) |
| Lurbinectedin | Approved (phase 4) |
| Margetuximab | Approved (phase 4) |
| Masitinib | Approved (phase 4) |
| Masoprocol | Approved (phase 4) |
| Mavorixafor | Approved (phase 4) |
| Mechlorethamine | Approved (phase 4) |
| Medroxyprogesterone Acetate | Approved (phase 4) |
| Megestrol Acetate | Approved (phase 4) |
| Melphalan | Approved (phase 4) |
| Melphalan Flufenamide | Approved (phase 4) |
| Methotrexate | Approved (phase 4) |
| Methyl Aminolevulinate | Approved (phase 4) |
| Midostaurin | Approved (phase 4) |
| Mirvetuximab Soravtansine | Approved (phase 4) |
| Mitomycin | Approved (phase 4) |
| Mitotane | Approved (phase 4) |
| Mitoxantrone | Approved (phase 4) |
| Mobocertinib | Approved (phase 4) |
| Mogamulizumab | Approved (phase 4) |
| Molgramostim | Approved (phase 4) |
| Mosunetuzumab | Approved (phase 4) |
| Moxetumomab Pasudotox | Approved (phase 4) |
| Nabilone | Approved (phase 4) |
| Naxitamab | Approved (phase 4) |
| Necitumumab | Approved (phase 4) |
| Nelarabine | Approved (phase 4) |
| Nilutamide | Approved (phase 4) |
| Niraparib | Approved (phase 4) |
| Nivolumab | Approved (phase 4) |
| Obinutuzumab | Approved (phase 4) |
| Ofatumumab | Approved (phase 4) |
| Olaparib | Approved (phase 4) |
| Olaratumab | Approved (phase 4) |
| Olmutinib | Approved (phase 4) |
| Omacetaxine Mepesuccinate | Approved (phase 4) |
| Ondansetron | Approved (phase 4) |
| Oprelvekin | Approved (phase 4) |
| Osimertinib | Approved (phase 4) |
| Oxaliplatin | Approved (phase 4) |
| PEGINTERFERON ALFA-2A | Approved (phase 4) |
| PEGINTERFERON ALFA-2B | Approved (phase 4) |
| PEGINTERFERON BETA-1A | Approved (phase 4) |
| Paclitaxel | Approved (phase 4) |
| Pacritinib | Approved (phase 4) |
| Padeliporfin | Approved (phase 4) |
| Palbociclib | Approved (phase 4) |
| Panitumumab | Approved (phase 4) |
| Panobinostat | Approved (phase 4) |
| Pegademase Bovine | Approved (phase 4) |
| Pegaspargase | Approved (phase 4) |
| Pegfilgrastim | Approved (phase 4) |
| Pembrolizumab | Approved (phase 4) |
| Pemetrexed | Approved (phase 4) |
| Pemigatinib | Approved (phase 4) |
| Pentostatin | Approved (phase 4) |
| Pertuzumab | Approved (phase 4) |
| Pexidartinib | Approved (phase 4) |
| Pipobroman | Approved (phase 4) |
| Pirtobrutinib | Approved (phase 4) |
| Pixantrone | Approved (phase 4) |
| Plerixafor | Approved (phase 4) |
| Plicamycin | Approved (phase 4) |
| Polatuzumab Vedotin | Approved (phase 4) |
| Polyestradiol Phosphate | Approved (phase 4) |
| Porfimer Sodium | Approved (phase 4) |
| Pralatrexate | Approved (phase 4) |
| Pralsetinib | Approved (phase 4) |
| Prednisone | Approved (phase 4) |
| Procarbazine | Approved (phase 4) |
| ROPEGINTERFERON ALFA-2B | Approved (phase 4) |
| Raltitrexed | Approved (phase 4) |
| Ramucirumab | Approved (phase 4) |
| Regorafenib | Approved (phase 4) |
| Relugolix | Approved (phase 4) |
| Repotrectinib | Approved (phase 4) |
| Retifanlimab | Approved (phase 4) |
| Ripretinib | Approved (phase 4) |
| Rituximab | Approved (phase 4) |
| Romidepsin | Approved (phase 4) |
| Rucaparib | Approved (phase 4) |
| Ruxolitinib | Approved (phase 4) |
| Sacituzumab Govitecan | Approved (phase 4) |
| Sargramostim | Approved (phase 4) |
| Selinexor | Approved (phase 4) |
| Selpercatinib | Approved (phase 4) |
| Selumetinib | Approved (phase 4) |
| Sipuleucel-T | Approved (phase 4) |
| Sirolimus | Approved (phase 4) |
| Sonidegib | Approved (phase 4) |
| Sotorasib | Approved (phase 4) |
| Streptozocin | Approved (phase 4) |
| Sugemalimab | Approved (phase 4) |
| Sunitinib | Approved (phase 4) |
| TECHNETIUM TC 99M SULFUR COLLOID | Approved (phase 4) |
| Tafasitamab | Approved (phase 4) |
| Tagraxofusp | Approved (phase 4) |
| Talquetamab | Approved (phase 4) |
| Tamoxifen | Approved (phase 4) |
| Tarlatamab | Approved (phase 4) |
| Tasonermin | Approved (phase 4) |
| Tazemetostat | Approved (phase 4) |
| Tebentafusp | Approved (phase 4) |
| Teclistamab | Approved (phase 4) |
| Tegafur | Approved (phase 4) |
| Temoporfin | Approved (phase 4) |
| Temozolomide | Approved (phase 4) |
| Temsirolimus | Approved (phase 4) |
| Teniposide | Approved (phase 4) |
| Tepotinib | Approved (phase 4) |
| Testosterone | Approved (phase 4) |
| Testosterone Cypionate | Approved (phase 4) |
| Testosterone Enanthate | Approved (phase 4) |
| Testosterone Undecanoate | Approved (phase 4) |
| Thioguanine | Approved (phase 4) |
| Thiotepa | Approved (phase 4) |
| Tislelizumab | Approved (phase 4) |
| Tisotumab Vedotin | Approved (phase 4) |
| Toripalimab | Approved (phase 4) |
| Trabectedin | Approved (phase 4) |
| Trametinib | Approved (phase 4) |
| Trastuzumab | Approved (phase 4) |
| Trastuzumab Deruxtecan | Approved (phase 4) |
| Trastuzumab Emtansine | Approved (phase 4) |
| Tremelimumab | Approved (phase 4) |
| Treosulfan | Approved (phase 4) |
| Tretinoin | Approved (phase 4) |
| Trifluridine | Approved (phase 4) |
| Triptorelin | Approved (phase 4) |
| Tucatinib | Approved (phase 4) |
| Umbralisib | Approved (phase 4) |
| Uracil Mustard | Approved (phase 4) |
| Valrubicin | Approved (phase 4) |
| Vandetanib | Approved (phase 4) |
| Vemurafenib | Approved (phase 4) |
| Venetoclax | Approved (phase 4) |
| Vinblastine | Approved (phase 4) |
| Vincristine | Approved (phase 4) |
| Vindesine | Approved (phase 4) |
| Vinorelbine | Approved (phase 4) |
| Vismodegib | Approved (phase 4) |
| Vorinostat | Approved (phase 4) |
| Zanidatamab | Approved (phase 4) |
| Zanubrutinib | Approved (phase 4) |
| Zinc Oxide | Approved (phase 4) |
| Zolbetuximab | Approved (phase 4) |
| 6-O-BENZYLGUANINE | Phase 3 (in late-stage trials) |
| ALBINTERFERON ALFA-2B | Phase 3 (in late-stage trials) |
| Acetaminophen | Phase 3 (in late-stage trials) |
| Aclarubicin | Phase 3 (in late-stage trials) |
| Adenosine | Phase 3 (in late-stage trials) |
| Afatinib | Phase 3 (in late-stage trials) |
| Amphotericin B | Phase 3 (in late-stage trials) |
| Amrubicin | Phase 3 (in late-stage trials) |
| Apixaban | Phase 3 (in late-stage trials) |
| Arginine Hydrochloride | Phase 3 (in late-stage trials) |
| Atomoxetine | Phase 3 (in late-stage trials) |
| Aumolertinib | Phase 3 (in late-stage trials) |
| Bcg Vaccine | Phase 3 (in late-stage trials) |
| Belotecan | Phase 3 (in late-stage trials) |
| Benmelstobart | Phase 3 (in late-stage trials) |
| Bermekimab | Phase 3 (in late-stage trials) |
| Bosutinib | Phase 3 (in late-stage trials) |
| Bupivacaine | Phase 3 (in late-stage trials) |
| CEPEGINTERFERON ALFA-2B | Phase 3 (in late-stage trials) |
| Cediranib | Phase 3 (in late-stage trials) |
| Datopotamab Deruxtecan | Phase 3 (in late-stage trials) |
| Degarelix | Phase 3 (in late-stage trials) |
| Dexmedetomidine | Phase 3 (in late-stage trials) |
| Donepezil | Phase 3 (in late-stage trials) |
| Edrecolomab | Phase 3 (in late-stage trials) |
| Efaproxiral | Phase 3 (in late-stage trials) |
| Eflornithine | Phase 3 (in late-stage trials) |
| Elacestrant | Phase 3 (in late-stage trials) |
| Eltrombopag | Phase 3 (in late-stage trials) |
| Emactuzumab | Phase 3 (in late-stage trials) |
| Enalapril | Phase 3 (in late-stage trials) |
| Enoxaparin Sodium | Phase 3 (in late-stage trials) |
| Entinostat | Phase 3 (in late-stage trials) |
| Epacadostat | Phase 3 (in late-stage trials) |
| Epoetin Beta | Phase 3 (in late-stage trials) |
| Erlotinib | Phase 3 (in late-stage trials) |
| Escitalopram | Phase 3 (in late-stage trials) |
| Estramustine | Phase 3 (in late-stage trials) |
| Etirinotecan Pegol | Phase 3 (in late-stage trials) |
| Fenretinide | Phase 3 (in late-stage trials) |
| Fluconazole | Phase 3 (in late-stage trials) |
| Fosaprepitant | Phase 3 (in late-stage trials) |
| Fotemustine | Phase 3 (in late-stage trials) |
| Ghrelin | Phase 3 (in late-stage trials) |
| Gimistotug | Phase 3 (in late-stage trials) |
| Ginseng, American | Phase 3 (in late-stage trials) |
| Glutamine | Phase 3 (in late-stage trials) |
| Heparin | Phase 3 (in late-stage trials) |
| Icotinib | Phase 3 (in late-stage trials) |
| Imatinib | Phase 3 (in late-stage trials) |
| Imetelstat | Phase 3 (in late-stage trials) |
| Iniparib | Phase 3 (in late-stage trials) |
| Ipatasertib | Phase 3 (in late-stage trials) |
| Itraconazole | Phase 3 (in late-stage trials) |
| Ivabradine | Phase 3 (in late-stage trials) |
| Ketamine | Phase 3 (in late-stage trials) |
| Lanreotide | Phase 3 (in late-stage trials) |
| Lapatinib | Phase 3 (in late-stage trials) |
| Lazertinib | Phase 3 (in late-stage trials) |
| Leniolisib | Phase 3 (in late-stage trials) |
| Levobupivacaine | Phase 3 (in late-stage trials) |
| Lonidamine | Phase 3 (in late-stage trials) |
| Melatonin | Phase 3 (in late-stage trials) |
| Methadone | Phase 3 (in late-stage trials) |
| Methoxyflurane | Phase 3 (in late-stage trials) |
| Methylphenidate | Phase 3 (in late-stage trials) |
| Metoclopramide | Phase 3 (in late-stage trials) |
| Mitoguazone | Phase 3 (in late-stage trials) |
| Momelotinib | Phase 3 (in late-stage trials) |
| Morphine | Phase 3 (in late-stage trials) |
| Motixafortide | Phase 3 (in late-stage trials) |
| Mycophenolate Mofetil | Phase 3 (in late-stage trials) |
| Nadroparin Calcium | Phase 3 (in late-stage trials) |
| Navitoclax | Phase 3 (in late-stage trials) |
| Neratinib | Phase 3 (in late-stage trials) |
| Nilotinib | Phase 3 (in late-stage trials) |
| Nintedanib | Phase 3 (in late-stage trials) |
| Nirogacestat | Phase 3 (in late-stage trials) |
| Ociperlimab | Phase 3 (in late-stage trials) |
| Olanzapine | Phase 3 (in late-stage trials) |
| Onartuzumab | Phase 3 (in late-stage trials) |
| Oportuzumab Monatox | Phase 3 (in late-stage trials) |
| Orelabrutinib | Phase 3 (in late-stage trials) |
| Oxandrolone | Phase 3 (in late-stage trials) |
| Oxycodone | Phase 3 (in late-stage trials) |
| P-Toluenesulfonamide | Phase 3 (in late-stage trials) |
| Paclitaxel Poliglumex | Phase 3 (in late-stage trials) |
| Palifermin | Phase 3 (in late-stage trials) |
| Palonosetron | Phase 3 (in late-stage trials) |
| Pamiparib | Phase 3 (in late-stage trials) |
| Parsaclisib | Phase 3 (in late-stage trials) |
| Patritumab Deruxtecan | Phase 3 (in late-stage trials) |
| Pazopanib | Phase 3 (in late-stage trials) |
| Pelabresib | Phase 3 (in late-stage trials) |
| Pirarubicin | Phase 3 (in late-stage trials) |
| Plitidepsin | Phase 3 (in late-stage trials) |
| Ponatinib | Phase 3 (in late-stage trials) |
| Prolgolimab | Phase 3 (in late-stage trials) |
| Propofol | Phase 3 (in late-stage trials) |
| Quizartinib | Phase 3 (in late-stage trials) |
| Ramosetron | Phase 3 (in late-stage trials) |
| Ranimustine | Phase 3 (in late-stage trials) |
| Remifentanil | Phase 3 (in late-stage trials) |
| Ridaforolimus | Phase 3 (in late-stage trials) |
| Rivaroxaban | Phase 3 (in late-stage trials) |
| Rociletinib | Phase 3 (in late-stage trials) |
| Sabatolimab | Phase 3 (in late-stage trials) |
| Satraplatin | Phase 3 (in late-stage trials) |
| Semustine | Phase 3 (in late-stage trials) |
| Serplulimab | Phase 3 (in late-stage trials) |
| Sevoflurane | Phase 3 (in late-stage trials) |
| Sitravatinib | Phase 3 (in late-stage trials) |
| Sodium Chloride | Phase 3 (in late-stage trials) |
| Sorafenib | Phase 3 (in late-stage trials) |
| Surufatinib | Phase 3 (in late-stage trials) |
| Surzebiclimab | Phase 3 (in late-stage trials) |
| Talazoparib | Phase 3 (in late-stage trials) |
| Tapentadol | Phase 3 (in late-stage trials) |
| Thalidomide | Phase 3 (in late-stage trials) |
| Thrombin | Phase 3 (in late-stage trials) |
| Thymopentin | Phase 3 (in late-stage trials) |
| Tiragolumab | Phase 3 (in late-stage trials) |
| Tivozanib | Phase 3 (in late-stage trials) |
| Topotecan | Phase 3 (in late-stage trials) |
| Toremifene | Phase 3 (in late-stage trials) |
| Tranexamic Acid | Phase 3 (in late-stage trials) |
| Trastuzumab Duocarmazine | Phase 3 (in late-stage trials) |
| Triphosphate | Phase 3 (in late-stage trials) |
| Trofosfamide | Phase 3 (in late-stage trials) |
| Tucidinostat | Phase 3 (in late-stage trials) |
| Valacyclovir | Phase 3 (in late-stage trials) |
| Varenicline | Phase 3 (in late-stage trials) |
| Veliparib | Phase 3 (in late-stage trials) |
| Vinflunine | Phase 3 (in late-stage trials) |
| Vintafolide | Phase 3 (in late-stage trials) |
| Vosaroxin | Phase 3 (in late-stage trials) |
Earlier-phase candidates (phase 2, investigational — efficacy not yet established): ANTINEOPLASTON A10, Alemtuzumab, Alteplase, Anidulafungin, Armodafinil, Aspirin, Atorvastatin, Azithromycin, Becotatug Vedotin, Belrestotug, Brenetafusp, Bromelains, Buparlisib, CPI 613, Cadonilimab, Calcium, Calcium Carbonate, Camrelizumab, Cannabidiol, Cannabinol, Carvedilol, Catequentinib, Ceralasertib, Cholecalciferol, Cobicistat, Colistin, Cridanimod, Curcumin, Cyclosporine, Dacomitinib Anhydrous, Dactolisib, Dalpiciclib, Denosumab, Diphenhydramine, Domvanalimab, Dovitinib, Empagliflozin, Endostatin, Endostatin, N-Terminal-Mggshhhhh, Enlonstobart, Envafolimab, Enzastaurin, Epoetin Alfa, Epratuzumab, Etentamig, Famitinib, Feladilimab, Felbinac, Firmonertinib, Fish Oil, Fludarabine, Fluzoparib, Fosbretabulin, Fosfomycin, Fospropofol, Furosemide, Gabapentin, Gadobutrol, Gadofosveset, Gadopentetate Dimeglumine, Gemigliptin, Gentamicin, Gimeracil, Glucarpidase, Golcadomide, Gotistobart, Hyaluronidase, Hydralazine, Hydrocodone, Hydroxychloroquine, Ibcasertib, Icosapent, Idasanutlin, Ifinatamab Deruxtecan, Imlunestrant, Inavolisib, Inosine, Interferon, Itacitinib, Ivosidenib, Izalontamab, KN-026, Ketoconazole, L-Citrulline, Leronlimab, Levocarnitine, Lidocaine, Linrodostat, Linsitinib, Linvoseltamab, Loperamide, Magrolimab, Mangafodipir, Metformin, Methylprednisolone, Monalizumab, Moxifloxacin, Netupitant, Niacin, Nisevokitug, Octreotide, Odronextamab, Opnurasib, Orteronel, Oteracil, Oxygen, Palazestrant, Pasireotide, Pectin, Penpulimab, Pentoxifylline, Pimicotinib, Protirelin, Psilocybin, Pucotenlimab, Pyrotinib, Relatlimab, Revumenib, Ribociclib, Rilvegostomig, Ritonavir, Rivoceranib, Ropivacaine, Rovalpituzumab Tesirine, Ruserontinib, Sacituzumab Tirumotecan, Saracatinib, Saruparib, Sigvotatug Vedotin, Siltuximab, Silybin A, Sintilimab, Sodium Bicarbonate, Sonrotoclax, Sotatercept, Sovleplenib, Spironolactone, Tacrolimus Anhydrous, Talabostat, Tanespimycin, Thymalfasin, Tildrakizumab, Tilsotolimod, Tirabrutinib, Tocilizumab, Tovorafenib, Tozinameran, Tramadol, Trastuzumab Envedotin, Trastuzumab Vedotin, Tromethamine, Vatalanib, Vedolizumab, Vepdegestrant, Vitamin E, Volasertib, Volrustomig, Zanzalintinib, Zimberelimab.
Drug target analysis
Approved (phase 4): 1 · Phase ≥3: 1 · Phased (≥1): 1 · Undrugged: 1
Druggability breadth: 2 of 2 evidence-associated genes (100%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).
Genes with an approved drug
The molecule shown is one approved compound that hits the gene — not necessarily a drug of choice or one indicated for this disease.
| Symbol | Example approved molecule |
|---|---|
| ATM | AMIODARONE HYDROCHLORIDE |
Top cohort targets by molecule count
| Symbol | Molecules | Max phase |
|---|---|---|
| ATM | 35 | 4 |
| TSC2 | 0 | 0 |
Drugs targeting cohort genes (top 30)
| Molecule | Max phase | Targets in cohort |
|---|---|---|
| AMIODARONE HYDROCHLORIDE | 4 | ATM |
| FURAZOLIDONE | 4 | ATM |
| ESTRADIOL ACETATE | 4 | ATM |
| NAFTIFINE HYDROCHLORIDE | 4 | ATM |
| METHYSERGIDE MALEATE | 4 | ATM |
| AMITRIPTYLINE HYDROCHLORIDE | 4 | ATM |
| XYLOMETAZOLINE HYDROCHLORIDE | 4 | ATM |
| FLUVOXAMINE MALEATE | 4 | ATM |
| ESTRADIOL VALERATE | 4 | ATM |
| PERMETHRIN | 4 | ATM |
| MITOTANE | 4 | ATM |
| TICLOPIDINE HYDROCHLORIDE | 4 | ATM |
| ENOXIMONE | 4 | ATM |
| METHYLENE BLUE ANHYDROUS | 4 | ATM |
| DITHIAZANINE IODIDE | 4 | ATM |
| ETHACRYNIC ACID | 4 | ATM |
| SECNIDAZOLE | 4 | ATM |
| MENADIONE | 4 | ATM |
| FENOFIBRATE | 4 | ATM |
| DIPYRIDAMOLE | 4 | ATM |
| DACTOLISIB | 3 | ATM |
| STREPTONIGRIN | 2 | ATM |
| CALCIMYCIN | 2 | ATM |
| ENPIROLINE | 2 | ATM |
| OXACEPROL | 2 | ATM |
| TOLONIUM CHLORIDE | 2 | ATM |
| ESTRADIOL BENZOATE | 2 | ATM |
| BERZOSERTIB | 2 | ATM |
| LARTESERTIB | 2 | ATM |
| ALTHIAZIDE | 2 | ATM |
Bioactivity and enzyme data
Enzyme cohort genes (≥1 EC): 1.
Cohort genes with ChEMBL bioactivity (full, sorted by assay count)
| Symbol | Assays | Type breakdown |
|---|---|---|
| ATM | 240 | Binding:233, Functional:5, ADMET:2 |
| TSC2 | 1 | Binding:1 |
Cohort enzymes (BRENDA EC)
| Symbol | EC numbers | Names |
|---|---|---|
| ATM | 2.7.11.1 | non-specific serine/threonine protein kinase |
Cohort genes with high screening signal
≥100 ChEMBL assays — a studied-ness signal; see Therapeutics for approved-drug status.
| Symbol | ChEMBL assays |
|---|---|
| ATM | 240 |
Pharmacogenomics
Cohort genes with a PharmGKB record: 2; with CPIC/DPWG dosing guidelines: 0.
No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).
Drug repurposing candidates
30 approved/phased drugs hit cohort targets but don’t yet appear in disease-level clinical trials. Target-inhibition rationale is strongest for cancer driver genes; a bioactivity hit is a screening signal, not a treatment claim.
| Compound | Max phase | Cohort target (bioactivity) |
|---|---|---|
| AMIODARONE HYDROCHLORIDE | 4 | ATM |
| FURAZOLIDONE | 4 | ATM |
| ESTRADIOL ACETATE | 4 | ATM |
| NAFTIFINE HYDROCHLORIDE | 4 | ATM |
| METHYSERGIDE MALEATE | 4 | ATM |
| AMITRIPTYLINE HYDROCHLORIDE | 4 | ATM |
| XYLOMETAZOLINE HYDROCHLORIDE | 4 | ATM |
| FLUVOXAMINE MALEATE | 4 | ATM |
| ESTRADIOL VALERATE | 4 | ATM |
| PERMETHRIN | 4 | ATM |
| MITOTANE | 4 | ATM |
| TICLOPIDINE HYDROCHLORIDE | 4 | ATM |
| ENOXIMONE | 4 | ATM |
| METHYLENE BLUE ANHYDROUS | 4 | ATM |
| DITHIAZANINE IODIDE | 4 | ATM |
| ETHACRYNIC ACID | 4 | ATM |
| SECNIDAZOLE | 4 | ATM |
| MENADIONE | 4 | ATM |
| FENOFIBRATE | 4 | ATM |
| DIPYRIDAMOLE | 4 | ATM |
| DACTOLISIB | 3 | ATM |
| STREPTONIGRIN | 2 | ATM |
| CALCIMYCIN | 2 | ATM |
| ENPIROLINE | 2 | ATM |
| OXACEPROL | 2 | ATM |
| TOLONIUM CHLORIDE | 2 | ATM |
| ESTRADIOL BENZOATE | 2 | ATM |
| BERZOSERTIB | 2 | ATM |
| LARTESERTIB | 2 | ATM |
| ALTHIAZIDE | 2 | ATM |
Druggability pyramid
Cohort genes binned by druggability tier (high → low):
| Tier | Definition | Genes | Symbols |
|---|---|---|---|
| A | Approved (phase 4 drug) | 1 | ATM |
| B | Phased (≥1) drug, not yet approved | 0 | |
| C | Druggable family + PDB, no drug | 0 | |
| D | Druggable family + AlphaFold only, no drug | 0 | |
| E | Difficult family or no structure, no drug | 1 | TSC2 |
Undrugged target profiles
1 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).
| Symbol | ChEMBL assays | Drugged partners (top 3) |
|---|---|---|
| TSC2 | 1 | — |
Clinical trials & evidence
Clinical trials
Clinical trials: 1,638.
Phase distribution (across all retrieved trials)
| Phase | Trials |
|---|---|
| PHASE1 | 640 |
| PHASE2 | 191 |
| PHASE1/PHASE2 | 107 |
| PHASE3 | 68 |
| PHASE4 | 35 |
| Not specified | 34 |
| EARLY_PHASE1 | 19 |
| PHASE2/PHASE3 | 6 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT05440240 | PHASE4 | RECRUITING | Percutaneous Needle Fasciotomy +/- Corticosteroid Injection for Dupuytren’s Contracture |
| NCT06706921 | PHASE4 | RECRUITING | 18F-Fluciclovine PET/CT Impact on Predicting Clinical Outcome of 177Lu-PSMA-617 Therapy in Patients With Prostate Cancer |
| NCT00050635 | PHASE4 | COMPLETED | STOP Trial - Sandostatin LAR Depot Trial for the Optimum Prevention of Chemotherapy Induced Diarrhea |
| NCT00117039 | PHASE4 | COMPLETED | A Study to Evaluate the Effectiveness of Aranesp® for Cancer Patients With Anemia |
| NCT00216541 | PHASE4 | COMPLETED | A Study of the Safety and Effectiveness of Epoetin Alfa on Hemoglobin Levels and Blood Transfusions in Cancer Patients Receiving Chemotherapy |
| NCT00230217 | PHASE4 | COMPLETED | Study of Rasburicase as Treatment or Prevention of Hyperuricemia Associated With Tumor Lysis Syndrome in Patients With Relapsed or Refractory Lymphoma, Leukemia, or Solid Tumor Malignancy |
| NCT00283465 | PHASE4 | COMPLETED | A Study of the Effectiveness and Safety of Treatment With Epoetin Alfa on Hemoglobin Levels, Red Blood Cell Transfusions, and Quality of Life in Patients With Cancer Receiving Platinum-containing Chemotherapy |
| NCT00588835 | PHASE4 | TERMINATED | Pharmacokinetic Study on the Addition of Aprepitant to Cisplatin - Etoposide Treatment in Lung Cancer Patients |
| NCT00630084 | PHASE4 | COMPLETED | Peginterferon Plus Ribavirin for Hepatitis C Patients Concomitant With Malignancy Other Than Hepatocellular Carcinoma |
| NCT00684463 | PHASE4 | COMPLETED | Palonosetron in Moderately and Highly Emetogenic Chemotherapy Induced Nausea and Vomiting (Study P04935)(COMPLETED) |
| NCT00687011 | PHASE4 | COMPLETED | Palonosetron Plus Dexamethasone in Moderately Emetogenic Chemotherapy Induced Nausea and Vomiting (Study P04594) |
| NCT00894153 | PHASE4 | UNKNOWN | p53 Gene Therapy for Head and Neck Malignant Tumors in Advanced Stage |
| NCT01394991 | PHASE4 | COMPLETED | A Safety Study of Epoetin Alfa in Patients With Cancer Who Have Chemotherapy-Related Anemia |
| NCT01427400 | PHASE4 | UNKNOWN | The Use of Botulinum Toxin A in Two-Stage Tissue Expander/ Implant Breast Reconstruction |
| NCT01471756 | PHASE4 | COMPLETED | Improving Complete Endoscopic Mucosal Resection (EMR) of Colorectal Neoplasia |
| NCT01481831 | PHASE4 | COMPLETED | Efficacy and Safety of Palonosetron Hydrochloride in the Prevention of Nausea and Vomiting |
| NCT01523912 | PHASE4 | COMPLETED | Radiofrequency Ablation for the Treatment of Gastric Dysplasia |
| NCT01588184 | PHASE4 | COMPLETED | An Extension Study to Provide Continued Bevacizumab Therapy to Participants With Solid Tumors Who Were Previously Enrolled in a Roche/Genentech Sponsored Study |
| NCT01708850 | PHASE4 | COMPLETED | Study in Cancer Patients With Central Line Associated Clots in the Upper Extremity Treated With Rivaroxaban (Catheter 2) |
| NCT01739764 | PHASE4 | COMPLETED | An Extension (Rollover) Study of Vemurafenib in Participants With BRAF V600 Mutation-Positive Malignancies Previously Enrolled in an Antecedent Vemurafenib Protocol |
| NCT01789281 | PHASE4 | COMPLETED | Everolimus Roll-over Protocol for Patients Who Have Completed a Previous Novartis-sponsored Everolimus Study. |
| NCT01802996 | PHASE4 | UNKNOWN | Evaluate the Efficacy of Magnesium Isoglycyrrhizinate in the Prevention of Chemotherapy Related Acute Liver Injury |
| NCT01862198 | PHASE4 | COMPLETED | The Usefulness of Endoscopic Ultrasound-guided Biliary Drainage With a Newly Designed Hybrid Metallic Stent |
| NCT01894828 | PHASE4 | UNKNOWN | Nutritional Supplementation in Patients With no Signs of Malnutrition |
| NCT02052024 | PHASE4 | COMPLETED | Myobloc Atrophy Study |
| NCT02081469 | PHASE4 | COMPLETED | Prophylaxis of CHB Patients With Malignant Tumor Receiving Chemotherapy |
| NCT02532634 | PHASE4 | COMPLETED | Ramosetron, Aprepitant, and Dexamethasone Versus Palonosetron, Aprepitant, and Dexamethasone |
| NCT02581176 | PHASE4 | COMPLETED | Apixaban as Treatment of Venous Thrombosis in Patients With Cancer: The CAP Study |
| NCT03333655 | PHASE4 | COMPLETED | Study to Explore the Mechanism of Acquired Immune Escape In Participants With Metastatic Cancer Progressing on CPI Therapy |
| NCT03513757 | PHASE4 | COMPLETED | Dexmedetomidine and Propofol for Pediatric MRI Sedation |
| NCT03895502 | PHASE4 | COMPLETED | Optimal Duration of Anticoagulation Therapy for Isolated Distal Deep Vein Thrombosis in Patients With Cancer Study |
| NCT04724460 | PHASE4 | COMPLETED | Optimal Duration of Anticoagulation Therapy for Low-risk Pulmonary Embolism Patients With Cancer |
| NCT05505006 | PHASE4 | UNKNOWN | Management of Preoperative Anaemia in Surgical Oncology |
| NCT05660694 | PHASE4 | COMPLETED | Comparison of Triamcinolone With Pentoxifylline and Vitamin- E Efficacy in the Treatment of Stage 2 and 3 Oral Submucous Fibrosis |
| NCT06005259 | PHASE4 | UNKNOWN | Effect of Spironolactone in the Prevention of Anthracycline-induced Cardiotoxicity (SPIROTOX) |
| NCT02261415 | PHASE3 | ACTIVE_NOT_RECRUITING | The HeLiX (Hemorrhage During Liver Resection: traneXamic Acid) Trial |
| NCT03194893 | PHASE3 | ACTIVE_NOT_RECRUITING | A Rollover Study of Alectinib in Patients With Anaplastic Lymphoma Kinase (ALK)-Positive or Rearranged During Transfection (RET)-Positive Cancer |
| NCT03435796 | PHASE2/PHASE3 | RECRUITING | Long-Term Follow-up Protocol for Participants Treated With Gene-Modified T Cells |
| NCT03961841 | PHASE3 | NOT_YET_RECRUITING | Perioperative Combination Chemotherapy Versus Chemoradiation for Locally Advanced EGJ Adenocarcinoma |
| NCT03981796 | PHASE3 | ACTIVE_NOT_RECRUITING | A Study to Evaluate Dostarlimab Plus Carboplatin-paclitaxel Versus Placebo Plus Carboplatin-paclitaxel in Participants With Recurrent or Primary Advanced Endometrial Cancer |
Drugs tested across these trials (top 30)
| Molecule | Max phase | Trials referencing |
|---|---|---|
| DOSTARLIMAB | 4 | 16 |
| EPOETIN ALFA | 4 | 13 |
| DACOMITINIB ANHYDROUS | 4 | 10 |
| NIRAPARIB | 4 | 9 |
| AXITINIB | 4 | 7 |
| NINTEDANIB | 4 | 6 |
| VEMURAFENIB | 4 | 6 |
| ABEMACICLIB | 4 | 5 |
| ALECTINIB | 4 | 5 |
| PALONOSETRON | 4 | 5 |
| AFLIBERCEPT | 4 | 4 |
| COBIMETINIB | 4 | 4 |
| COPANLISIB | 4 | 4 |
| NERATINIB | 4 | 4 |
| PALBOCICLIB | 4 | 4 |
| TEPROTUMUMAB | 4 | 4 |
| AFATINIB | 4 | 3 |
| BELANTAMAB MAFODOTIN | 4 | 3 |
| BOSUTINIB | 4 | 3 |
| ERDAFITINIB | 4 | 3 |
| INDOCYANINE GREEN | 4 | 3 |
| ISATUXIMAB | 4 | 3 |
| LENVATINIB | 4 | 3 |
| RUCAPARIB | 4 | 3 |
| TRABECTEDIN | 4 | 3 |
| VISMODEGIB | 4 | 3 |
| ALPELISIB | 4 | 2 |
| APREPITANT | 4 | 2 |
| BELINOSTAT | 4 | 2 |
| BETAMETHASONE | 4 | 2 |
Related Atlas pages
- Cohort genes: TSC2, ATM
- Drugs: Dostarlimab, Epoetin Alfa, Dacomitinib, Niraparib, Axitinib, Nintedanib, Vemurafenib, Abemaciclib, Alectinib, Palonosetron, Aflibercept, Cobimetinib, Copanlisib, Neratinib, Palbociclib, Teprotumumab, Afatinib, Belantamab Mafodotin, Bosutinib, Erdafitinib, Indocyanine Green, Isatuximab, Lenvatinib, Rucaparib, Trabectedin, Vismodegib, Alpelisib, Aprepitant, Belinostat, Betamethasone