TGFB1 transforming growth factor beta 1
ENSG00000105329 · protein coding · Open Targets profile
Transforming growth factor beta-1 proprotein: Precursor of the Latency-associated peptide (LAP) and Transforming growth factor beta-1 (TGF-beta-1) chains, which constitute the regulatory and active subunit of TGF-beta-1, respectively. {ECO:0000269|PubMed:29109152, ECO:0000303|PubMed:27252363}.
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 Withdrawn means the drug was pulled from the market or from development.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
- Structure with Ligand, Med-Quality Pocket, Druggable Family
- Antibody
- Advanced Clinical, UniProt loc high conf, GO CC high conf, UniProt SigP or TMHMM
- PROTAC
- Database Ubiquitination, Half-life Data, Small Molecule Binder
- Other modalities
- Approved Drug
gnomAD loss of function constraint: o/e 0.43417999148368835 (upper bound 0.6370900273323059). 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 82 registry trials, updated in the last three years, of the most advanced drugs against TGFB1 (luspatercept, bintrafusp alfa, fresolimumab, LY-2382770), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Bristol-Myers SquibbBristol-Myers Squibb · 6 of 6 trials Phase 2+Ph3NCT04717414 · An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell Transfusionsbrief
- James L GulleyNational Cancer Institute (NCI) · 3 of 3 trials Phase 2+Ph1/Ph2NCT03493945 · Phase I/II Study of Immunotherapy Combination BN-Brachyury Vaccine, M7824, N-803 and Epacadostat (QuEST1)brief
- Hoyoung MaengNational Cancer Institute (NCI) · 2 of 2 trials Phase 2+
- Qifa LiuNanfang Hospital, Southern Medical University · 2 of 2 trials Phase 2+
- Abhishek A. MangaonkarMayo Clinic in Rochester · 1 of 1 trials Phase 2+
- Anne Sophie KubaschUniversity Leipzig · 1 of 1 trials Phase 2+
- Annemiek ME WalenkampUniversity Medical Center Groningen · 1 of 1 trials Phase 2+
- Arun RajanNational Cancer Institute (NCI) · 1 of 1 trials Phase 2+
- Bing BingPeking Union Medical College Hospital · 1 of 1 trials Phase 2+
- Charalampos FloudasNational Cancer Institute (NCI) · 1 of 1 trials Phase 2+
- Chi Leung ChiangThe University of Hong Kong · 1 of 1 trials Phase 2+
- Guillermo Garcia-ManeroM.D. Anderson Cancer Center · 1 of 1 trials Phase 2+
- Hoyoung M MaengNational Cancer Institute (NCI) · 1 of 1 trials Phase 2+
- Hussein A TawbiM.D. Anderson Cancer Center · 1 of 1 trials Phase 2+
- Jeevan ShettyCelgene Corporation · 1 of 1 trials Phase 2+
Registry trials of luspatercept, bintrafusp alfa, fresolimumab, LY-2382770 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; 49 of 76 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. 6 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
25 trials of drugs against TGFB1 were stopped early
Terminated, withdrawn and suspended interventional trials of luspatercept, bintrafusp alfa, fresolimumab, LY-2382770, metelimumab, 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 25.
- terminated · 2026-09-03 · Forma Therapeutics, Inc. · matched by the registry's drug synonyms“Futility assessment concluded low likelihood of the trial showing meaningful patient benefit.”
- suspended · 2026-07-17 · Mikkael Sekeres, MD · Luspatercept“Funding Sponsor decision”
- withdrawn · 2026-06-04 · Peking Union Medical College Hospital · Luspatercept“This is a duplicate registration. The research plan and content have already been updated in another entry.”
- terminated · 2026-03-18 · M.D. Anderson Cancer Center · Bintrafusp Alfa“Insufficient accrual”
- terminated · 2025-11-17 · Mayo Clinic · Bintrafusp Alfa“EMD Serono recommendation based on three other studies that failed to show benefit for bintrafusp alfa”
- terminated · 2024-11-07 · M.D. Anderson Cancer Center · Bintrafusp Alfa“The sponsor decided to no longer develop the drug and stop support of studies using the drug.”
- terminated · 2024-09-19 · Mayo Clinic · Luspatercept“Slow accrual”
- terminated · 2024-04-18 · GlaxoSmithKline · Bintrafusp alfa“Primary investigator withdrew the participant due to an SAE”
- withdrawn · 2024-04-02 · National Cancer Institute (NCI) · Bintrafusp alfa“Closed by study team, no subjects enrolled.”
- terminated · 2024-01-16 · EMD Serono Research & Development Institute, Inc. · matched by the registry's drug synonyms“Based on recommendations by an external Independent data Monitoring Committee (IDMC), Sponsor decided to discontinue this clinical study due to a low likelihood of achieving superiority in the efficacy endpoints versus standard of care.”
- withdrawn · 2023-12-27 · M.D. Anderson Cancer Center · Bintrafusp Alfa“Per Sponsor Request-no longer manufacturing the study drug”
- terminated · 2023-11-14 · EMD Serono Research & Development Institute, Inc. · matched by the registry's drug synonyms“Based on a review of data conducted by the Independent Data Monitoring Committee (IDMC), Sponsor decided to discontinue this study as the study was unlikely to achieve the primary objective of overall survival.”
- terminated · 2023-08-01 · M.D. Anderson Cancer Center · Bintrafusp Alfa“PI Request”
- terminated · 2023-04-10 · EMD Serono Research & Development Institute, Inc. · Bintrafusp alfa“The study was prematurely discontinued by the sponsor due to probability of success which was too low to justify the continuation of recruitment.”
- terminated · 2023-01-17 · Olivia Newton-John Cancer Research Institute · matched by the registry's drug synonyms“Efficacy finding”
- terminated · 2022-10-31 · M.D. Anderson Cancer Center · Bintrafusp Alfa“Due to the fact that the sponsor decided not to move forward with the development of M7824”
- terminated · 2022-07-28 · M.D. Anderson Cancer Center · Bintrafusp Alfa“Per PI Request”
- terminated · 2022-05-23 · AIO-Studien-gGmbH · Bintrafusp alfa“Based on results of another phase II study with bintrafusp alfa in combination with gemcitabine plus cisplatin, that was discontinued as it was unlikely to meet the primary endpoint of OS, the recruitment for this study was stopped prematurely.”
- terminated · 2022-05-18 · National Cancer Institute (NCI) · matched by the registry's drug synonyms“Study closed to accrual due to the worsening risk: benefit ratio for participants receiving bintrafusp alfa (M7824).”
- terminated · 2022-04-06 · UNICANCER · bintrafusp alfa“Sponsor decision following information on cases of hyperprogression and early toxicities with bintrafusp alfa in other studies”
- terminated · 2022-03-22 · National Cancer Institute (NCI) · Bintrafusp alfa (M7824)“Because of the low accrual and the safety concerns we closed the study.”
- terminated · 2022-01-11 · National Cancer Institute (NCI) · matched by the registry's drug synonyms“One participant was accrued, and the study was stopped due to new safety data from the company for M7824 and slow accrual.”
- withdrawn · 2021-12-17 · National Cancer Institute (NCI) · Bintrafusp alfa“Due to notification from EMD Serono, for Bintrafusp alfa (M7824) regarding an observed increase in the frequently of early progression and death.”
- terminated · 2019-09-17 · Eli Lilly and Company · matched by the registry's drug synonyms“Interim Assessment: Lack of Efficacy”
- terminated · 2014-12-08 · John Mascarenhas · matched by the registry's drug synonyms“Early termination when the drug was no longer made available by the pharmaceutical company due to unanticipated management and administrative decisions.”
Paediatric incentives and obligations for TGFB1's drugs
For luspatercept, bintrafusp alfa, fresolimumab, LY-2382770, metelimumab: 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 · TGFB1 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: yes · EMEA-001521-PIP03-22 (Luspatercept, Treatment of alpha-thalassaemia): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 27 Oct 2023; EMEA-001521-PIP01-13-M06 (Luspatercept, Treatment of beta-thalassaemia;Treatment of myelodysplastic syndromes): PM: decision on the application for modification of an agreed PIP, 13 May 2022; EMEA-002586-PIP01-19-M01 (bintrafusp alfa, Treatment of all conditions included in the category of malignant neoplasms (except central nervous system tumours, haematopoietic and lymphoid tissue neoplasms)): PM: decision on the application for modification of an agreed PIP, 17 Mar 2021; EMEA-001521-PIP02-18 (Luspatercept, Treatment of myelofibrosis): W: decision granting a waiver in all age groups for all conditions or indications, 15 Aug 2018Rule: 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: yes · EU/3/14/1300 positive 29 Jul 2014: Treatment of beta thalassaemia intermedia and major; EU/3/14/1331 positive 22 Aug 2014: Treatment of myelodysplastic syndromes; EU/3/20/2255 positive 28 Feb 2020: Treatment of myelofibrosis; EU/3/11/882 withdrawn 5 Aug 2011: Treatment of focal segmental glomerulosclerosisRule: 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 · Reblozyl: authorised since 25 Jun 2020, orphan medicineRule: 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 EPAR Reblozyl, 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: no · FDA's table lists no pediatric approval of luspatercept, bintrafusp alfa, fresolimumab, ly-2382770, metelimumabRule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-12
- Registered studies open to children: yes · 2 interventional studies of luspatercept, bintrafusp alfa, fresolimumab, ly-2382770, metelimumab include patients under 18; 2 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, 2026-11-12
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 5 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.