Live from Open Targets Platform, fetched 2026-11-11 15:27 UTC. Every row links to its source record.

STAT3 signal transducer and activator of transcription 3

ENSG00000168610 · protein coding · Open Targets profile

Signal transducer and transcription activator that mediates cellular responses to interleukins, KITLG/SCF, LEP and other growth factors (PubMed:10688651, PubMed:11294841, PubMed:12359225, PubMed:12873986, PubMed:15194700, PubMed:15653507, PubMed:16285960, PubMed:17344214, PubMed:18242580, PubMed:18782771, PubMed:22306293, PubMed:23084476, PubMed:28262505, PubMed:32929201, PubMed:38404237). Once activated, recruits coactivators, such as NCOA1 or MED1, to the promoter region of the target gene (PubMed:15653507, PubMed:16285960, PubMed:17344214, PubMed:18782771, PubMed:28262505, PubMed:32929201). May mediate cellular responses to activated FGFR1, FGFR2, FGFR3 and FGFR4 (PubMed:12873986). Upon activation of IL6ST/gp130 signaling by interleukin-6 (IL6), binds to the IL6-responsive elements identified in the promoters of various acute-phase protein genes (PubMed:12359225). Activated by IL31 through IL31RA (PubMed:15194700). Acts as a regulator of inflammatory response by regulating differentiation of naive CD4(+) T-cells into T-helper Th17 or regulatory T-cells (Treg): acetylation promotes its transcription activity and cell differentiation while deacetylation and oxidation of lysine residues by LOXL3 inhibits differentiation (PubMed:28065600, PubMed:28262505). Involved in cell cycle regulation by inducing the expression of key genes for the progression from G1 to S phase, such as CCND1 (PubMed:17344214). Mediates the effects of LEP on melanocortin production, body energy homeostasis and lactation (By similarity). May play an apoptotic role by transactivating BIRC5 expression under LEP activation (PubMed:18242580). Cytoplasmic STAT3 represses macroautophagy by inhibiting EIF2AK2/PKR activity (PubMed:23084476). Plays a crucial role in basal beta cell functions, such as regulation of insulin secretion (By similarity). Following JAK/STAT signaling activation and as part of a complex with NFATC3 and NFATC4, binds to the alpha-beta E4 promoter region of CRYAB and activates transcription in cardiomyocytes (By similarity). Involved in the oncostatin-M-mediated signaling pathway through both type I OSM receptor complex (heterodimers composed of LIFR and IL6ST) and type II OSM receptor complex (heterodimers composed of OSMR and IL6ST) (PubMed:9188471). {ECO:0000250|UniProtKB:P42227, ECO:0000269|PubMed:10688651, ECO:0000269|PubMed:11294841, ECO:0000269|PubMed:12359225, ECO:0000269|PubMed:12873986, ECO:0000269|PubMed:15194700, ECO:0000269|PubMed:15653507, ECO:0000269|PubMed:16285960, ECO:0000269|PubMed:17344214, ECO:0000269|PubMed:18242580, ECO:0000269|PubMed:18782771, ECO:0000269|PubMed:22306293, ECO:0000269|PubMed:23084476, ECO:0000269|PubMed:28065600, ECO:0000269|PubMed:28262505, ECO:0000269|PubMed:32929201, ECO:0000269|PubMed:38404237, ECO:0000269|PubMed:9188471}.

In 13 of 15 cancers, the evidence for STAT3 comes mainly from mutations found in tumours

Each row is a cancer, strongest overall evidence first. The two dots show how strong the inherited (germline) and tumour-mutation (somatic) evidence is; the line between them is the gap.

Inherited (germline) variantsMutations in tumours (somatic)

Scores run from 0 (no evidence) to 1 (strongest). A missing dot at 0 means Open Targets holds no evidence of that kind. Source: Open Targets Platform, fetched live.

Tractability

Small molecule: Structure with Ligand, High-Quality Ligand, Druggable Family
Antibody: GO CC med conf
PROTAC: Literature, Database Ubiquitination, Half-life Data, Small Molecule Binder

gnomAD loss of function constraint: o/e 0.06121300160884857 (upper bound 0.11497999727725983). Low values mean loss of function is poorly tolerated, a safety flag for inhibition.

Who runs trials of these drugs

Principal investigators and study chairs on 8 registry trials, updated in the last three years, of the most advanced drugs against STAT3 (danvatirsen), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.

David Hong
M.D. Anderson Cancer Center · 1 of 1 trials Phase 2+
Ph1/Ph2NCT02499328 · Study to Assess MEDI4736 With Either AZD9150 or AZD5069 in Advanced Solid Tumors & Relapsed Metastatic Squamous Cell Carcinoma of Head & Neck brief
David S Hong
M.D. Anderson Cancer Center · 1 of 1 trials Phase 2+
Ph2NCT02983578 · Danvatirsen and Durvalumab in Treating Patients With Advanced and Refractory Pancreatic, Non-Small Cell Lung Cancer, and Mismatch Repair Deficient Colorectal Cancer brief
John Heymach
M.D. Anderson Cancer Center · 1 of 1 trials Phase 2+
Ph2NCT03334617 · Phase II Umbrella Study of Novel Anti-cancer Agents in Participants With NSCLC Who Progressed on an Anti-PD-1/PD-L1 Containing Therapy brief
Nabil Saba
Emory University · 1 of 1 trials Phase 2+
Ph2NCT05814666 · Activity and Safety of Danvatirsen and Pembrolizumab in HNSCC brief
Aditi Shastri
Montefiore Medical Center · 0 of 1 trials Phase 2+
Ph1NCT05986240 · Danvatirsen Monotherapy Followed by Combination With Venetoclax in Relapsed/Refractory MDS & AML brief
Chih-Hsin Yang
National Taiwan University Hospital · 0 of 1 trials Phase 2+
Ph1NCT03819465 · A Study of Novel Anti-cancer Agents in Patients With Previously Untreated NSCLC brief
Hendrik-Tobias Arkenau
Sarah Cannon Research Institute, UK · 0 of 1 trials Phase 2+
Ph1NCT02546661 · Open-Label, Randomised, Multi-Drug, Biomarker-Directed, Phase 1b Study in Pts w/ Muscle Invasive Bladder Cancer brief
Melissa Johnson
Tennessee Oncology · 0 of 1 trials Phase 2+
Ph1NCT03421353 · AZD9150 Plus Durvalumab Alone or in Combination With Chemotherapy in Patients With Advanced, Solid Tumours and in Patients With Non-Small-Cell Lung Cancer brief
Sandip Patel
UCSD Morres Cancer Center · 0 of 1 trials Phase 2+
Ph1NCT03819465 · A Study of Novel Anti-cancer Agents in Patients With Previously Untreated NSCLC brief
Thomas Powles
Barts Cancer Center, Barts and The London School of Medicine and Denistry · 0 of 1 trials Phase 2+
Ph1NCT02546661 · Open-Label, Randomised, Multi-Drug, Biomarker-Directed, Phase 1b Study in Pts w/ Muscle Invasive Bladder Cancer brief

3 of 8 trials of drugs against STAT3 have sites only in United States

Registry trials of danvatirsen updated in the last three years, counted by the country group of their listed sites. A trial with sites in several groups counts in each; 3 of 8 have all sites in one country.

Sites only in this groupSites here and elsewhere

Site counts are not patients per country, and trials registered only outside ClinicalTrials.gov are not covered. Where sites are is a fact about the record, not a judgement of its quality. Europe is the EU and EEA, the UK and Switzerland. Source: ClinicalTrials.gov, fetched live.

2 trials of drugs against STAT3 were stopped early

Terminated, withdrawn and suspended interventional trials of danvatirsen, newest update first, live from ClinicalTrials.gov. Each reason is quoted exactly as the registry gives it; we do not sort reasons into safety, efficacy or business. Dates are the last registry update. Showing the 2 most recent of 2.

Ph2NCT05814666 brief why it stopped
terminated · 2026-01-30 · Flamingo Therapeutics NV · Danvatirsen
“Interim analysis was negative”
Ph2NCT02417753 brief why it stopped
terminated · 2017-05-15 · National Cancer Institute (NCI) · matched by the registry's drug synonyms
“Study was closed because we could not find these types of patients.”

Paediatric incentives and obligations for STAT3's drugs

For danvatirsen: what EMA and FDA publish about paediatric plans, orphan status and pediatric approvals, and how many registered studies take children. Each line is a yes, no or not published fact from the record linked under it; none of it is a forecast.

FDA pediatric target list: yes · STAT3 is on the list. Membership alone neither requires nor waives a pediatric study.
Rule: Target is on FDA's Pediatric Molecular Target List (relevant targets). Source: FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01)), 2026-11-11
EU paediatric investigation plan: no · No EMA paediatric investigation plan decision names danvatirsen
Rule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA data download (dated snapshot), 2026-11-11
EU orphan designation: no · No EU orphan designation names danvatirsen
Rule: An EU orphan designation lists the drug as its active substance (exact name) or belongs to a medicine that does; withdrawn and expired designations are listed with their status. Source: EMA data download (dated snapshot), 2026-11-11
Authorised in the EU: no · EMA lists no centrally authorised medicine of danvatirsen
Rule: An EMA medicine record lists the drug as its active substance or INN, exact name; national-only authorisations are not in this file. Source: EMA data download (dated snapshot), 2026-11-11
FDA orphan designation: not published · FDA's orphan designation database turns away automated requests, so this app does not read it; search it by hand at the link
Rule: Would be: an FDA orphan designation names the drug. Source: FDA orphan designations search
FDA pediatric oncology approval: no · FDA's table lists no pediatric approval of danvatirsen
Rule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-11
Registered studies open to children: no · No interventional study of danvatirsen includes patients under 18
Rule: The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age. Source: ClinicalTrials.gov search, 2026-11-11

Rare pediatric disease voucher. FDA, content current as of 29 Apr 2026: “As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029. FDA may not award any PRVs under this program after September 30, 2029.” Voucher sale prices are not shown here; they appear only as company-reported news. FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs

All 1 drugs and clinical candidates as a table

Each drug, stage and indication links to its Open Targets record, where the trials and approvals behind it are listed. Salt forms (for example a mesylate) are listed as Open Targets records them, as separate entries.

Drug	Type	Highest stage	Indications	Source records
DANVATIRSEN	Oligonucleotide	phase 2	ovarian cancer, colorectal cancer, pancreatic neoplasm, non-small cell lung carcinoma, malignant pancreatic neoplasm, head and neck squamous cell carcinoma, diffuse large B-cell lymphoma, hepatocellular carcinoma, +4 more	Open Targets · ChEMBL