IMPDH1 inosine monophosphate dehydrogenase 1
ENSG00000106348 · protein coding · Open Targets profile
Catalyzes the conversion of inosine 5'-phosphate (IMP) to xanthosine 5'-phosphate (XMP), the first committed and rate-limiting step in the de novo synthesis of guanine nucleotides, and therefore plays an important role in the regulation of cell growth. Could also have a single-stranded nucleic acid-binding activity and could play a role in RNA and/or DNA metabolism. It may also have a role in the development of malignancy and the growth progression of some tumors.
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.
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 Withdrawn means the drug was pulled from the market or from development.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.
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.
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, Med-Quality Pocket, Druggable Family
- Antibody
- GO CC high conf
- PROTAC
- Database Ubiquitination, Half-life Data, Small Molecule Binder
gnomAD loss of function constraint: o/e 0.5577700138092041 (upper bound 0.7236999869346619). 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 400 registry trials, updated in the last three years, of the most advanced drugs against IMPDH1 (mycophenolate mofetil, mizoribine, thioguanine, merimepodib, mycophenolic acid, mycophenolate, AVN-944), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Dolores GrossoThomas Jefferson University · 6 of 6 trials Phase 2+
- Neal FlomenbergThomas Jefferson University · 5 of 5 trials Phase 2+
- Usama GergisSidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University · 5 of 5 trials Phase 2+
- Christopher G KanakryNational Cancer Institute (NCI) · 4 of 5 trials Phase 2+Ph1/Ph2NCT05436418 · The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantationbrief
- Rachel B. SalitFred Hutch/University of Washington Cancer Consortium · 4 of 4 trials Phase 2+Ph2NCT04370301 · Reduced Intensity Haploidentical Transplantation for the Treatment of Primary or Secondary Myelofibrosisbrief
- Christian P. LarsenEmory University · 3 of 3 trials Phase 2+
- Dimana DimitrovaNational Cancer Institute (NCI) · 3 of 3 trials Phase 2+
- Kenneth NewellEmory University · 3 of 3 trials Phase 2+
- Mary HorowitzCenter for International Blood and Marrow Transplant Research · 3 of 3 trials Phase 2+
- Peter S. HeegerIcahn School of Medicine at Mount Sinai · 3 of 3 trials Phase 2+
- Phuong VoFred Hutchinson Cancer Center · 3 of 3 trials Phase 2+Ph1/Ph2NCT03670966 · 211At-BC8-B10 Followed by Donor Stem Cell Transplant in Treating Patients With Relapsed or Refractory High-Risk Acute Leukemia or Myelodysplastic Syndromebrief
- Danielle E Pregent-ArnoldNational Cancer Institute (NCI) · 2 of 2 trials Phase 2+
- Flavio VincentiUniversity of California at San Francisco · 2 of 2 trials Phase 2+
- Fredrik JuhlinITB-Med LLC · 2 of 2 trials Phase 2+
- Heather J SymonsChildren's Oncology Group · 2 of 2 trials Phase 2+
Registry trials of mycophenolate mofetil, mizoribine, thioguanine, merimepodib, mycophenolic acid, mycophenolate, AVN-944 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; 314 of 369 have all sites in one country.
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. 31 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
241 trials of drugs against IMPDH1 were stopped early; 15 of the 200 most recent give no reason
Terminated, withdrawn and suspended interventional trials of mycophenolate mofetil, mizoribine, thioguanine, merimepodib, mycophenolic acid, mycophenolate, AVN-944, 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 25 most recent of 241.
- withdrawn · 2026-10-15 · Brigham and Women's Hospital · matched by the registry's drug synonyms“Attempts to renew our protocol have not been accepted. Also, composition of the team and institutional goals have changed.”
- terminated · 2026-09-24 · National Institute of Allergy and Infectious Diseases (NIAID) · Mycophenolate mofetil“Slow accrual within enrollment time period: projected accrual goal not achieved.”
- terminated · 2026-09-24 · National Institute of Allergy and Infectious Diseases (NIAID) · matched by the registry's drug synonyms“Absence of equipoise on the basis of predetermined stopping rules.”
- terminated · 2026-09-24 · National Institute of Allergy and Infectious Diseases (NIAID) · matched by the registry's drug synonyms“Secondary to safety concerns plus change in Campath® (alemtuzumab) availability.”
- terminated · 2026-09-24 · National Institute of Allergy and Infectious Diseases (NIAID) · mycophenolate mofetil“Safety: Stopping rule not met.”
- suspended · 2026-09-10 · Leland Metheny · Mycophenolate Mofetil“insufficient funding”
- withdrawn · 2026-09-03 · Mayo Clinic · Mycophenolate Mofetil“Sponsor declined to fund study due to inability to agree on IP.”
- terminated · 2026-06-10 · National Institute of Allergy and Infectious Diseases (NIAID) · matched by the registry's drug synonyms“The study was closed due to continued enrollment hurdles.”
- terminated · 2026-05-27 · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Mycophenolate Mofetil“The stopping rule was met and hence the study was closed”
- terminated · 2026-05-14 · Fred Hutchinson Cancer Center · Mycophenolate Mofetil“Study was terminated prior to enrolling protocol target accrual goal due to declining accrual and Data Safety Monitoring Board recommendations.”
- suspended · 2026-05-07 · Wondercel Biotech (ShenZhen) · matched by the registry's drug synonyms“Because the implementation of the new regulation policy by the Chinese authority”
- suspended · 2026-05-07 · Wondercel Biotech (ShenZhen) · matched by the registry's drug synonyms“Because the implementation of the new regulation policy by the Chinese authority”
- suspended · 2026-05-07 · Wondercel Biotech (ShenZhen) · matched by the registry's drug synonyms“Because the implementation of the new regulation policy by the Chinese authority”
- terminated · 2026-05-06 · Mats Remberger · matched by the registry's drug synonyms“Too slow recruitment”
- terminated · 2026-04-14 · University Hospital, Rouen · matched by the registry's drug synonyms“Difficulty in enrolling new patients”
- suspended · 2026-03-30 · University of Chicago · Mycophenolate Mofetil“Enrollment on hold pending amendment”
- terminated · 2026-03-12 · ITB-Med LLC · Mycophenolate Mofetil (MMF)“After enrolment was complete, and sufficient data was collected to fully characterize the highest dose cohort, study follow up was terminated by the sponsor for non-safety related reasons.”
- withdrawn · 2026-02-27 · Janssen Research & Development, LLC · matched by the registry's drug synonyms“Business Decision”
- withdrawn · 2026-02-24 · Ossium Health, Inc. · Mycophenolate Mofetil“Change in Study Design”
- terminated · 2026-02-10 · Aurinia Pharmaceuticals Inc. · matched by the registry's drug synonyms“DSMB recommendation”
- terminated · 2025-11-10 · University of Southern California · Mycophenolate mofetil (MMF) or Mycophenolic acid (MPA)“Research contract expired”
- terminated · 2025-10-21 · Incyte Corporation · matched by the registry's drug synonyms“Study terminated by sponsor”
- terminated · 2025-09-09 · National Institute of Allergy and Infectious Diseases (NIAID) · Mycophenolate Mofetil, Placebo for Mycophenolate Mofetil“The study was closed due to slow enrollment.”
- terminated · 2025-07-25 · LMU Klinikum · Mycophenolate Mofetil, Mycophenolate Mofetil Placebo“Premature end of recruitment due to lack of eligible patients. Within two years of recruitment only nine patients could be randomized out of 130 required patients as per trial protocol.”
- terminated · 2025-06-29 · M.D. Anderson Cancer Center · Mycophenolate Mofetil“PI Request”
Paediatric incentives and obligations for IMPDH1's drugs
For mycophenolate mofetil, mizoribine, thioguanine, merimepodib, mycophenolic acid, mycophenolate, AVN-944: 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: no · IMPDH1 is not on the listRule: 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 mycophenolate mofetil, mizoribine, thioguanine, merimepodib, mycophenolic acid, mycophenolate, avn-944Rule: 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 mycophenolate mofetil, mizoribine, thioguanine, merimepodib, mycophenolic acid, mycophenolate, avn-944Rule: 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: yes · CellCept: authorised since 14 Feb 1996; Mycophenolate mofetil Teva: authorised since 21 Feb 2008; Myfenax: authorised since 21 Feb 2008; Myclausen: authorised since 7 Oct 2010Rule: 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 linkRule: Would be: an FDA orphan designation names the drug. Source: FDA orphan designations search
- FDA pediatric oncology approval: yes · Thioguanine [Tabloid], date not given: Remission induction and remission consolidation treatment of acute nonlymphocytic leukemiasRule: 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: yes · 455 interventional studies of mycophenolate mofetil, mizoribine, thioguanine, merimepodib, mycophenolic acid, mycophenolate, avn-944 include patients under 18; 87 are recruiting, not yet recruiting or activeRule: 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 9 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.