CSF2RB colony stimulating factor 2 receptor subunit beta
ENSG00000100368 · protein coding · Open Targets profile
Cell surface receptor that plays a role in immune response and controls the production and differentiation of hematopoietic progenitor cells into lineage-restricted cells. Acts by forming an heterodimeric receptor through interaction with different partners such as IL3RA, IL5RA or CSF2RA (PubMed:1495999). In turn, participates in various signaling pathways including interleukin-3, interleukin-5 and granulocyte-macrophage colony-stimulating factor/CSF2 pathways. In unstimulated conditions, interacts constitutively with JAK1 and ligand binding leads to JAK1 stimulation and subsequent activation of the JAK- STAT pathway (PubMed:9516124). {ECO:0000269|PubMed:1495999, ECO:0000269|PubMed:9516124}.
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 Withdrawn means the drug was pulled from the market or from development.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
- High-Quality Pocket
- Antibody
- Advanced Clinical, GO CC high conf, UniProt SigP or TMHMM
- PROTAC
- Database Ubiquitination
- Other modalities
- Advanced Clinical
gnomAD loss of function constraint: o/e 0.3276199996471405 (upper bound 0.46783000230789185). 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 152 registry trials, updated in the last three years, of the most advanced drugs against CSF2RB (sargramostim, mavrilimumab, cibinetide), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Brian KushnerMemorial Sloan Kettering Cancer Center · 3 of 3 trials Phase 2+
- David AviganBeth Israel Deaconess Medical Center · 2 of 2 trials Phase 2+
- Frank S HodiECOG-ACRIN Cancer Research Group · 2 of 2 trials Phase 2+
- Hun Jungjhun@astonsci.com · 2 of 2 trials Phase 2+
- Mark RanalliNationwide Children's Hospital · 2 of 2 trials Phase 2+
- Natasha HunterFred Hutch/University of Washington Cancer Consortium · 2 of 2 trials Phase 2+
- Robert FenstermakerRoswell Park Cancer Institute · 2 of 2 trials Phase 2+
- Tianhong LiVA Northern California Health Care System, Mather, CA · 2 of 2 trials Phase 2+
- Duane MitchellUniversity of Florida · 1 of 4 trials Phase 2+
- David KirnJennerex Biotherapeutics · 1 of 2 trials Phase 2+
- Jasmeet KaurRoswell Park Cancer Institute · 1 of 2 trials Phase 2+
- Alice L YuChildren's Oncology Group · 1 of 1 trials Phase 2+
- Alice P ChenNational Cancer Institute (NCI) · 1 of 1 trials Phase 2+
- Ami V DesaiChildren's Oncology Group · 1 of 1 trials Phase 2+
- Arshed QuyyumiEmory University · 1 of 1 trials Phase 2+
Registry trials of sargramostim, mavrilimumab, cibinetide 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; 128 of 149 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. 3 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
112 trials of drugs against CSF2RB were stopped early; 23 give no reason
Terminated, withdrawn and suspended interventional trials of sargramostim, mavrilimumab, cibinetide, 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 112.
- terminated · 2026-10-30 · Tufts Medical Center · matched by the registry's drug synonyms“Futility”
- withdrawn · 2026-10-09 · Georgetown University · matched by the registry's drug synonyms“Sponsor Decision”
- terminated · 2026-08-05 · Beth Israel Deaconess Medical Center · matched by the registry's drug synonyms“Withdrawal of funding”
- suspended · 2026-06-29 · University of California, Davis · Sargramostim“Drugs unavailable”
- terminated · 2026-04-23 · University of Florida · matched by the registry's drug synonyms“due to lack of accrual/feasibility to complete accrual due to competing trials”
- terminated · 2025-10-29 · University of Florida · matched by the registry's drug synonyms“Not feasible to accrue due to competing studies”
- withdrawn · 2025-08-07 · Mayo Clinic · Aerosol Sargramostim“Lack of funding”
- terminated · 2025-08-06 · University of Florida · matched by the registry's drug synonyms“Terminated by mutual agreement by sponsor and institution.”
- withdrawn · 2025-04-02 · University of Colorado, Denver · Sargramostim for Injection“Difficulties enrolling participants due to study design”
- terminated · 2025-04-01 · Emory University · matched by the registry's drug synonyms“Accrual goal met”
- terminated · 2024-10-04 · Belfast Health and Social Care Trust · matched by the registry's drug synonyms“Expiry of study drug - no replacement available.”
- terminated · 2024-08-21 · Hoffmann-La Roche · matched by the registry's drug synonyms“The study was terminated by the sponsor following interim analysis as the primary endpoint of iDFS crossed the pre-specified futility boundary of hazard ratio > 1 in the Intent-to-Treat (ITT) population.”
- terminated · 2024-07-03 · Savara Inc. · matched by the registry's drug synonyms“SAV006-03 was initiated before the evaluation of the IMPALA study (NCT02702180) results. Considering these results and authority advice there would not be adequate efficacy and safety data from SAV006-03 and the study was terminated.”
- terminated · 2024-06-17 · Aston Sci. Inc. · matched by the registry's drug synonyms“Difficulty in recruiting patients”
- terminated · 2024-04-08 · Mustafa Khasraw, MBChB, MD, FRCP, FRACP · matched by the registry's drug synonyms“Resource shortage”
- terminated · 2024-03-18 · Y-mAbs Therapeutics · matched by the registry's drug synonyms“Due to business priorities”
- terminated · 2024-03-04 · Kite, A Gilead Company · matched by the registry's drug synonyms“Development program terminated and the decision was not due to any safety concerns.”
- terminated · 2024-01-29 · Masonic Cancer Center, University of Minnesota · sargramostim“Slow Accrual”
- terminated · 2023-11-24 · Binhui Biopharmaceutical Co., Ltd. · matched by the registry's drug synonyms“Strategy adjustment”
- terminated · 2023-09-18 · University of Nebraska · sargramostim“Genitope suspend drug development by decision made by the FDA March 6, 2008.”
- withdrawn · 2023-09-15 · Partner Therapeutics, Inc. · Sargramostim“Study did not commence.”
- withdrawn · 2023-06-02 · Milton S. Hershey Medical Center · Sargramostim 0.04 mcg/kg/dose, Sargramostim 0.2mcg/kg/dose, Sargramostim 1 mcg/kg/dose“Clinical Hold FDA”
- withdrawn · 2023-05-22 · Dana-Farber Cancer Institute · Sargramostim (GM-CSF)“Study terminated by sponsor”
- terminated · 2023-01-10 · Savara Inc. · matched by the registry's drug synonyms“Recruitment for trial stopped early due to limitations from COVID-19, additionally changes in the standard of care for CF patients during the study period resulted in confounding of the primary endpoint.”
- terminated · 2022-12-15 · Marker Therapeutics, Inc. · matched by the registry's drug synonyms“Futility analysis did not support continuation of the trial”
Paediatric incentives and obligations for CSF2RB's drugs
For sargramostim, mavrilimumab, cibinetide: 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 · CSF2RB 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 · EMA/PE/0000232765 (sargramostim, Treatment of pulmonary alveolar proteinosis): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 14 Apr 2025; EMA/PE/0000232765 (sargramostim, Treatment of pulmonary alveolar proteinosis): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 14 Apr 2025; EMEA-003568-PIP01-23 (sargramostim, Treatment of acute radiation syndrome): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 27 Mar 2024Rule: 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/3/24/2928 positive 28 Jun 2024: Treatment of pulmonary alveolar proteinosis; EU/3/16/1721 positive 29 Aug 2016: Prevention of graft loss in pancreatic islet transplantation; EU/3/13/1191 positive 7 Oct 2013: Treatment of sarcoidosisRule: 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 · Imreplys: authorised since 21 Aug 2025, exceptional circumstancesRule: 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 Imreplys, 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 sargramostim, mavrilimumab, cibinetideRule: 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 · 172 interventional studies of sargramostim, mavrilimumab, cibinetide include patients under 18; 28 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 3 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.