OncoSignal
Live from Open Targets Platform, fetched 2026-11-11 21:22 UTC. Every row links to its source record.

ATP1A1 ATPase Na+/K+ transporting subunit alpha 1

ENSG00000163399 · protein coding · Open Targets profile

Catalytic subunit of the Na(+)/K(+)-ATPase pump that hydrolyzes ATP to exchange ions across the plasma membrane, exporting 3 Na(+) and importing 2 K(+) per cycle against electrochemical gradients (PubMed:29499166, PubMed:30388404, PubMed:35803952). It undergoes ATP- driven conformational changes that allow alternating binding and release of Na(+) and K(+) ions across the membrane (PubMed:35803952). This process maintains essential Na(+) and K(+) gradients for membrane potential and cellular function. Could also be part of an osmosensory signaling pathway that senses body-fluid sodium levels and controls salt intake behavior as well as voluntary water intake to regulate sodium homeostasis (By similarity). {ECO:0000250|UniProtKB:Q8VDN2, ECO:0000269|PubMed:29499166, ECO:0000269|PubMed:30388404, ECO:0000269|PubMed:35803952}.

5 of the 6 drugs recorded against ATP1A1 are approved, and 1 more reached Phase 3

Each band is a group of drugs. It runs from the kind of drug on the left to the furthest stage that drug has reached in any disease on the right. Thicker bands mean more drugs.

5 small molecule drugs at Approved: DIGITOXIN, LANATOSIDE C, DESLANOSIDE, ACETYLDIGITOXIN, DIGOXIN1 small molecule drug at Phase 3: ISTAROXIMESmall molecule 6Approved 5Phase 3 1

Approved bands are highlighted. Withdrawn means the drug was pulled from the market or from development. A combined phase counts as the later one (Phase 1/2 is shown as Phase 2). Hover a band for the drug names. Source: Open Targets Platform, fetched live.

In 15 of 15 cancers, the evidence for ATP1A1 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)
00.250.50.751aldosterone-producing adrenal cortex ad…aldosterone-producing adrenal cortex adenomaaldosterone-producing adrenal cortex adenoma: somatic 0.72gastric carcinomagastric carcinomagastric carcinoma: somatic 0.61carcinoma of liver and intrahepatic bil…carcinoma of liver and intrahepatic biliary tractcarcinoma of liver and intrahepatic biliary tract: somatic 0.61ovarian endometrioid adenocarcinoma wit…ovarian endometrioid adenocarcinoma with squamous differentiationovarian endometrioid adenocarcinoma with squamous differentiation: somatic 0.61duodenal adenocarcinomaduodenal adenocarcinomaduodenal adenocarcinoma: somatic 0.61colorectal adenocarcinomacolorectal adenocarcinomacolorectal adenocarcinoma: somatic 0.61endometrial endometrioid adenocarcinomaendometrial endometrioid adenocarcinomaendometrial endometrioid adenocarcinoma: somatic 0.61bile duct carcinomabile duct carcinomabile duct carcinoma: somatic 0.61acute myeloid leukemiaacute myeloid leukemiaacute myeloid leukemia: somatic 0.46melanomamelanomamelanoma: somatic 0.46hepatocellular carcinomahepatocellular carcinomahepatocellular carcinoma: somatic 0.46esophageal squamous cell carcinomaesophageal squamous cell carcinomaesophageal squamous cell carcinoma: somatic 0.46osteosarcomaosteosarcomaosteosarcoma: somatic 0.46pancreatic ductal adenocarcinomapancreatic ductal adenocarcinomapancreatic ductal adenocarcinoma: somatic 0.46head and neck squamous cell carcinomahead and neck squamous cell carcinomahead and neck squamous cell carcinoma: somatic 0.46

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
Approved Drug, Structure with Ligand, High-Quality Ligand, Druggable Family
Antibody
UniProt loc high conf, GO CC high conf, UniProt loc med conf, UniProt SigP or TMHMM, Human Protein Atlas loc
PROTAC
Database Ubiquitination, Half-life Data, Small Molecule Binder

gnomAD loss of function constraint: o/e 0.12197999656200409 (upper bound 0.19068999588489532). 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 127 registry trials, updated in the last three years, of the most advanced drugs against ATP1A1 (digitoxin, lanatoside c, deslanoside, acetyldigitoxin, digoxin, istaroxime), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.

49 of 124 trials of drugs against ATP1A1 have sites only in United States

Registry trials of digitoxin, lanatoside c, deslanoside, acetyldigitoxin, digoxin, istaroxime 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; 115 of 124 have all sites in one country.

Sites only in this groupSites here and elsewhere
United StatesUnited States: 49 trials with sites only hereUnited States: 7 trials with sites here and elsewhere56ChinaChina: 26 trials with sites only hereChina: 2 trials with sites here and elsewhere28EuropeEurope: 20 trials with sites only hereEurope: 8 trials with sites here and elsewhere28JapanJapan: 0 trials with sites only hereJapan: 1 trials with sites here and elsewhere1OtherOther: 21 trials with sites only hereOther: 7 trials with sites here and elsewhere28

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. 3 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.

20 trials of drugs against ATP1A1 were stopped early; 2 give no reason

Terminated, withdrawn and suspended interventional trials of digitoxin, lanatoside c, deslanoside, acetyldigitoxin, digoxin, istaroxime, 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 20 most recent of 20.

  • terminated · 2026-10-21 · University of Nebraska · Digoxin
    “Due to low accrual and futility”
  • terminated · 2026-09-29 · Yale University · Intravenous digoxin
    “Slow accrual”
  • terminated · 2026-09-10 · CAMC Health System · matched by the registry's drug synonyms
    “Low enrollment and PI retired.”
  • terminated · 2026-05-22 · AMAG Pharmaceuticals, Inc. · AMAG-423 (digoxin immune fab)
    “Following a recommendation from the Data and Safety Monitoring Board (DSMB), the study was stopped early for futility. There were no safety concerns raised.”
  • withdrawn · 2025-04-22 · ViiV Healthcare · matched by the registry's drug synonyms
    “Sponsor decision”
  • withdrawn · 2025-04-01 · Turning Point Therapeutics, Inc. · Digoxin
    “Insufficient enrollment”
  • terminated · 2025-02-20 · Damascus University · Digoxin+furosemide+captopril
    “lack of necessary equipment”
  • withdrawn · 2024-09-20 · University of Illinois Chicago · Digoxin
    “Study Not Funded”
  • withdrawn · 2023-02-22 · The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School · Digoxin tablet
    “Administrative decision to terminate.”
  • withdrawn · 2022-12-01 · Scripps Health · matched by the registry's drug synonyms
    “no participants enrolled”
  • terminated · 2021-03-16 · Fox Chase Cancer Center · Digoxin
    “Slow accrual”
  • terminated · 2019-03-26 · Rennes University Hospital · matched by the registry's drug synonyms
    No reason given
  • terminated · 2019-03-20 · Maruho Co., Ltd. · matched by the registry's drug synonyms
    “Lack of effect in interim analysis”
  • terminated · 2019-02-15 · Aultman Health Foundation · matched by the registry's drug synonyms
    “Limited resources available to met accrual goal.”
  • withdrawn · 2018-05-15 · University of Texas Southwestern Medical Center · Digoxin Combination
    “No subjects consented for enrollment on this study.”
  • terminated · 2018-03-29 · University of Louisville · Erlotinib plus Digoxin
    “Interim analysis revealed that only 1 patient had a partial response.”
  • terminated · 2018-02-22 · Western Regional Medical Center · Digoxin
    “PI Leaving Site”
  • withdrawn · 2015-03-10 · Boehringer Ingelheim · Digoxin
    No reason given
  • withdrawn · 2014-10-23 · Debiopharm International SA · Istaroxime
    “The study was not started due to a re-evaluation of the istaroxime development program”
  • withdrawn · 2014-10-23 · Debiopharm International SA · Istaroxime
    “The study was not started due to a re-evaluation of the istaroxime development program”
Paediatric incentives and obligations for ATP1A1's drugs

For digitoxin, lanatoside c, deslanoside, acetyldigitoxin, digoxin, istaroxime: 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.

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 6 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.

DrugTypeHighest stageIndicationsSource records
DIGITOXINSmall moleculeapprovalcardiovascular disorder, cardiac arrhythmia, heart failure, cystic fibrosisOpen Targets · ChEMBL
LANATOSIDE CSmall moleculeapprovalcardiovascular disorderOpen Targets · ChEMBL
DESLANOSIDESmall moleculeapprovalcardiovascular disorder, cardiac arrhythmia, heart failureOpen Targets · ChEMBL
ACETYLDIGITOXINSmall moleculeapprovalcardiovascular disorder, congestive heart failureOpen Targets · ChEMBL
DIGOXINSmall moleculephase 4heart failure, atrial fibrillation, congestive heart failure, cardiovascular disorder, cardiac arrhythmia, Supraventricular tachycardia, atrial flutter, preeclampsia, +23 moreOpen Targets · ChEMBL
ISTAROXIMESmall moleculephase 2 3heart disorder, heart failure, ShockOpen Targets · ChEMBL