Retifanlimab

Summary

Retifanlimab (CHEMBL4298037) is an approved antibody (ATC L01FF10) targeting PDCD1; indicated across 24 conditions including neoplasm and head and neck squamous cell carcinoma.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Antibody
• ATC class: L01FF10
• Targets: 1 (PDCD1)
• Indications: 24 conditions
• Clinical trials: 66

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4298037
Name	Retifanlimab
Type	Antibody
Max phase	4
ATC	L01FF10

Also known as: AEX-1188, AEX1188, INCMGA-00012, INCMGA-0012, Incmga00012, INCMGA00012, MGA-012, Mga012, MGA012, Retifanlimab, Retifanlimab-dlwr, Zynyz

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
PDCD1	programmed cell death 1 (CD279)	Binding	8.82	0%	Q15116

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

Aggregated over 1 target gene(s): PDCD1.

Top Reactome pathways

3 total, by targets touching each:

Pathway	Targets	Genes
Co-inhibition by PD-1	1	PDCD1
Potential therapeutics for SARS	1	PDCD1
PD-L1(CD274) glycosylation and translocation to plasma membrane	1	PDCD1

Dominant GO biological processes

GO term	Targets
B cell apoptotic process	1
adaptive immune response	1
negative regulation of tolerance induction	1
negative regulation of T cell mediated immune response to tumor cell	1
negative regulation of B cell apoptotic process	1
apoptotic process	1
humoral immune response	1
negative regulation of inflammatory response	1
regulation of immune response	1
negative regulation of immune response	1
negative regulation of T cell receptor signaling pathway	1
negative regulation of T cell activation	1
positive regulation of T cell apoptotic process	1
regulatory T cell apoptotic process	1
immune system process	1

Indications & clinical

Indications

5 approved indications. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
neoplasm	4	MONDO:0005070	EFO:0000616
head and neck squamous cell carcinoma	4	MONDO:0010150	EFO:0000181
squamous cell carcinoma	4	MONDO:0005096	EFO:0000707
cutaneous neuroendocrine carcinoma	4	MONDO:0019210	EFO:1001471
non-small cell lung carcinoma	3	MONDO:0005233	EFO:0003060

16 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
glioblastoma	2	MONDO:0018177	EFO:0000519
breast neoplasm	2	MONDO:0021100	MONDO:0007254
triple-negative breast carcinoma	2	MONDO:0005494	EFO:0005537
urothelial carcinoma	2	MONDO:0040679	EFO:0008528
carcinoid syndrome	2	MONDO:0100347	EFO:1000852
soft tissue sarcoma	2	MONDO:0018078	EFO:1001968
endometrium neoplasm	2	MONDO:0021251	MONDO:0011962
sarcoma	2	MONDO:0005089	EFO:0000691
exocrine pancreatic carcinoma	2	MONDO:0005192	EFO:0002618
neuroendocrine neoplasm	2	MONDO:0019496	EFO:1001901
head and neck cancer	2	MONDO:0005627	EFO:0006859
breast carcinoma	2	MONDO:0004989	EFO:0000305
dedifferentiated liposarcoma	2	MONDO:0020563	EFO:0003085
paraganglioma	2	MONDO:0000448	EFO:1000453
melanoma	1	MONDO:0005105	EFO:0000756
pancreatic ductal adenocarcinoma	1	MONDO:0005184	MONDO:0005184

3 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 66.

Phase distribution

Phase	Trials
PHASE2	27
PHASE1	19
PHASE1/PHASE2	12
PHASE3	4
PHASE2/PHASE3	2
EARLY_PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04205812	PHASE3	ACTIVE_NOT_RECRUITING	Platinum-Based Chemotherapy With/Without INCMGA00012, an Anti-PD-1 Antibody, in Non-Small Cell Lung Cancer
NCT06072612	PHASE3	RECRUITING	Study of the Bria-IMT Regimen and CPI vs Physicians’ Choice in Advanced Metastatic Breast Cancer.
NCT04082364	PHASE2/PHASE3	COMPLETED	Combination Margetuximab, Retifanlimab, Tebotelimab, and Chemotherapy Phase 2/3 Trial in HER2+ Gastric/GEJ Cancer
NCT04129320	PHASE2/PHASE3	WITHDRAWN	Enoblituzumab Plus MGA012 or MGD013 in Squamous Cell Carcinoma of the Head and Neck
NCT04203511	PHASE3	WITHDRAWN	INCMGA00012 in Combination With Chemoradiation in Participants With Stage III Non-Small Cell Lung Cancer (POD1UM-301)
NCT04472429	PHASE3	COMPLETED	Carboplatin-paclitaxel With Retifanlimab or Placebo in Participants With Locally Advanced or Metastatic Squamous Cell Anal Carcinoma (POD1UM-303/InterAACT 2).
NCT04225039	PHASE2	ACTIVE_NOT_RECRUITING	Anti-GITR/Anti-PD1/Stereotactic Radiosurgery, in Recurrent Glioblastoma
NCT04374305	PHASE2	RECRUITING	Innovative Trial for Understanding the Impact of Targeted Therapies in NF2-Related Schwannomatosis (INTUITT-NF2)
NCT04438824	PHASE2	ACTIVE_NOT_RECRUITING	Palbociclib and INCMGA00012 in People With Advanced Liposarcoma
NCT04445844	PHASE2	ACTIVE_NOT_RECRUITING	INCMGA00012 and Pelareorep for the Treatment of Metastatic Triple Negative Breast Cancer, IRENE Study
NCT04463771	PHASE2	ACTIVE_NOT_RECRUITING	Safety and Efficacy of Retifanlimab (INCMGA00012) Alone or in Combination With Other Therapies in Participants With Advanced or Metastatic Endometrial Cancer Who Have Progressed on or After Platinum-based Chemotherapy.
NCT04494009	PHASE2	ACTIVE_NOT_RECRUITING	PD-1 Inhibitor INCMGA00012 as Consolidation Therapy After Definitive Concurrent Chemoradiotherapy
NCT04577014	PHASE1/PHASE2	RECRUITING	Retifanlimab (Anti-PD-1 Antibody) With Gemcitabine and Docetaxel in Patients With Advanced Soft Tissue Sarcoma
NCT04968106	PHASE2	ACTIVE_NOT_RECRUITING	Neoadjuvant Chemotherapy and Retifanlimab in Patients With Selected Sarcomas (TORNADO)
NCT05177133	PHASE2	RECRUITING	Anti-PD-1 and CapOx for the First-line Treatment of dMMR Esophagogastric Cancer (AuspiCiOus)
NCT05287113	PHASE2	ACTIVE_NOT_RECRUITING	Study of Retinfanlimab in Combination With INCAGN02385 and INCAGN02390 as First-Line Treatment in Participants With PD-L1-Positive (CPS ≥ 1) Recurrent/Metastatic Squamous Cell Carcinoma of the Head and Neck
NCT05345002	PHASE2	RECRUITING	All-Trans Retinoic Acid (ATRA) Plus PD-1 Inhibition in Recurrent IDH-Mutant Glioma
NCT05455697	PHASE1/PHASE2	RECRUITING	Tafasitamab, Retifanlimab, and Rituximab in Combination With Standard Therapy for the Treatment of Diffuse Large B-cell Lymphoma
NCT05594290	PHASE2	ACTIVE_NOT_RECRUITING	Chemo-immunotherapy in Patients With Resectable Merkel Cell Carcinoma Prior to Surgery
NCT06056895	PHASE2	ACTIVE_NOT_RECRUITING	Triple Immune Checkpoint Inhibition for Advanced or Metastatic PD-(L)1 Refractory Merkel Cell Carcinoma
NCT06149481	PHASE1/PHASE2	RECRUITING	Phase I/II Study of the Combination Immunotherapy Regimen: SX-682, TriAdeno Vaccine, Retifanlimab and IL-15 Agonist N-803 (STAR15) for Metastatic Colorectal Cancer (mCRC)
NCT06160206	PHASE2	RECRUITING	Retifanlimab with Bevacizumab and Hypofractionated Radiotherapy for the Treatment of Recurrent Glioblastoma
NCT06320405	PHASE1/PHASE2	RECRUITING	Axatilimab in Combination With Retifanlimab and Paclitaxel for the Treatment of Patients With Advanced or Metastatic Solid Tumors
NCT06389799	PHASE2	RECRUITING	A Phase 2, Open Label Study of PEmigatinib and REtifanlimab in Advanced Dedifferentiated LIposarcoma (PERELI)
NCT06873789	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	A Study to Evaluate INCB177054 in Participants With Select Advanced or Metastatic Solid Tumors
NCT07219576	PHASE1/PHASE2	RECRUITING	Retifanlimab and Ruxolitinib In Solid Malignancies
NCT07425054	PHASE2	NOT_YET_RECRUITING	HPV ctDNA Response-Adapted Chemoradiation +/- Retifanlimab Treatment in Advanced-Stage Anal Cancer
NCT07468136	PHASE1/PHASE2	NOT_YET_RECRUITING	Retifanlimab With or Without Difluoromethylornithine for the Treatment of Progressive High Grade Gliomas
NCT07472322	PHASE1/PHASE2	NOT_YET_RECRUITING	ASTX727 & Retifanlimab-dlwr for Advanced Merkel Cell After Progression on Anti-PD-(L)1
NCT02393248	PHASE1/PHASE2	TERMINATED	Open-Label, Dose-Escalation Study of Pemigatinib in Subjects With Advanced Malignancies - (FIGHT-101)
NCT03328026	PHASE1/PHASE2	COMPLETED	Combination Study of SV-BR-1-GM With Retifanlimab
NCT03531632	PHASE1/PHASE2	COMPLETED	MGD007 Combined With MGA012 in Relapsed/Refractory Metastatic Colorectal Cancer
NCT03532295	PHASE2	COMPLETED	Retifanlimab and Epacadostat in Combination With Radiation and Bevacizumab in Patients With Recurrent Gliomas
NCT03597295	PHASE2	COMPLETED	A Study of INCMGA00012 in Squamous Carcinoma of the Anal Canal Following Platinum-Based Chemotherapy (POD1UM-202)
NCT03599713	PHASE2	COMPLETED	A Study of INCMGA00012 in Metastatic Merkel Cell Carcinoma (POD1UM-201)
NCT03679767	PHASE2	COMPLETED	A Study of INCMGA00012 in Participants With Selected Solid Tumors (POD1UM-203)
NCT04116073	PHASE2	COMPLETED	INCMGA00012 in Patients With Previously Treated Unresectable or Metastatic Adenosquamous Pancreatic or Ampullary Cancer
NCT04231981	PHASE2	COMPLETED	Efficacy of INCMGA00012 in Penile Squamous Cell Carcinoma (ORPHEUS)
NCT04586244	PHASE2	TERMINATED	An Umbrella Study to Determine the Safety and Efficacy of Various Monotherapy or Combination Therapies in Neoadjuvant Urothelial Carcinoma
NCT04634825	PHASE2	TERMINATED	Enoblituzumab Plus Retifanlimab or Tebotelimab in Head and Neck Cancer
NCT04776876	PHASE2	WITHDRAWN	Retifanlimab (INCMGA00012) and Telotristat Ethyl for the Treatment of Advanced Neuroendocrine Tumors and Carcinoid Syndrome
NCT04949191	PHASE2	TERMINATED	The Purpose of the Study is to Continue to Provide Pemigatinib to Patients With Advanced Malignancies.
NCT05239182	PHASE2	TERMINATED	9-ING-41 Plus Retifanlimab and Gemcitabine/Nab-Paclitaxel in Patients With Advanced Pancreatic Adenocarcinoma
NCT05359692	PHASE2	WITHDRAWN	INCAGN01876 in Combination With Immunotherapy in Participants With Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma
NCT06939036	PHASE1/PHASE2	TERMINATED	Study of 225Ac-SS0110 in Subjects With ES-SCLC or MCC (SANTANA-225 )
NCT04387084	PHASE1	ACTIVE_NOT_RECRUITING	Short-term Fasting Prior to PD-1/PD-L1 Inhibitor Therapy for of Advanced or Metastatic Skin Malignancy
NCT04470024	PHASE1	RECRUITING	Phase Ib Trial of Multivalent Autophagosome Vaccine With or Without GITR Agonist, With Anti-PD-1 Immunotherapy in HNSCC
NCT05083754	PHASE1	RECRUITING	Carmustine Wafer in Combination With Retifanlimab and Radiation With/Without Temozolomide in Subjects With Glioblastoma
NCT05743595	PHASE1	ACTIVE_NOT_RECRUITING	Neoantigen-based Personalized DNA Vaccine With Retifanlimab PD-1 Blockade Therapy in Patients With Newly Diagnosed, Unmethylated Glioblastoma
NCT06179160	PHASE1	RECRUITING	A Study to Evaluate INCB161734 in Participants With Advanced or Metastatic Solid Tumors With KRAS G12D Mutation
NCT06896188	PHASE1	RECRUITING	9-ING-41 Combined With Retifanlimab, Plus Modified FOLFIRINOX for Patients With Advanced Pancreatic Adenocarcinoma (RiLEY)
NCT06959537	PHASE1	RECRUITING	A Phase Ib Study of Novel Combination (New) of Low Dose Oral CyclophoSphamide (s) to Potentiate Axatilimab (A) + Retifanlimab (R) in Treating Metastatic Triple Negative Breast Cancer (TNBC)
NCT02475213	PHASE1	COMPLETED	Safety Study of Enoblituzumab (MGA271) in Combination With Pembrolizumab or MGA012 in Refractory Cancer
NCT03059823	PHASE1	COMPLETED	A Phase 1 Study of INCMGA00012 in Patients With Advanced Solid Tumors
NCT03406949	PHASE1	COMPLETED	MGD009/MGA012 Combination in Relapsed/Refractory Cancer
NCT03522142	PHASE1	TERMINATED	A Study Exploring the Safety and Tolerability of INCB081776 in Participants With Advanced Malignancies
NCT03589651	PHASE1	COMPLETED	INCMGA00012 in Combination With Other Therapies in Patients With Advanced Solid Tumors
NCT03910530	PHASE1	COMPLETED	A Study of INCMGA00012, INCB001158, and the Combination in Japanese Participants With Advanced Solid Tumors
NCT03920839	PHASE1	WITHDRAWN	INCMGA00012 Plus Chemotherapy in Participants With Advanced Solid Tumors (POD1UM-105)
NCT04580485	PHASE1	COMPLETED	INCB106385 Alone or in Combination With Immunotherapy in Advanced Solid Tumors
NCT04799431	PHASE1	WITHDRAWN	Neoantigen-Targeted Vaccine Combined With Anti-PD-1 Antibody for Patients With Stage IV MMR-p Colon and Pancreatic Ductal Cancer
NCT04989387	PHASE1	TERMINATED	Study of INCA 0186 in Subjects With Advanced Solid Tumors
NCT05440942	PHASE1	COMPLETED	Combined MEK, STAT3 and PD-1 Inhibition in Metastatic Pancreatic Ductal Adenocarcinoma
NCT06290622	PHASE1	WITHDRAWN	PD-1, LAG-3 and TIM-3 Checkpoint Blockade in DLBCL
NCT06610682	EARLY_PHASE1	RECRUITING	A Trial to Evaluate CSF ctDNA and Safety of Plixorafenib Alone or With Retifanlimab in Patients With BRAF-altered Glioma
NCT06910137	Not specified	AVAILABLE	An Early Access Program Guideline to Provide Access to Retifanlimab (INCMGA00012) Together With Carboplatin and Paclitaxel for Squamous Carcinoma of the Anal Canal (SCAC)

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

4 molecules share ≥1 primary target. Top 4 by shared-target count:

Molecule	Source	Status	Shared targets
PYRVINIUM	ChEMBL	Phase 4 (approved)	PDCD1
RIFABUTIN	ChEMBL	Phase 4 (approved)	PDCD1
Gefitinib	PubChem	Approved	PDCD1
Pomalidomide	PubChem	Approved	PDCD1