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Balstilimab

Phase 3

Colorectal Cancer | Small molecule | Oncology |Agenus Inc.|Last Updated: Aug 14, 2026

Target and mechanism

Molecular targetPDCD1
Target classAntagonist
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedACTIVE_CONTROLLEDDMC
Total Trials1
Total Enrollment834

FDA Designations

No designations recorded

Clinical trial landscape

Balstilimab · 5 trials · 12 indications

Phase 3 2Phase 2 3
NCT07152821Botensilimab + Balstilimab vs Best Supportive Care as Therapy in Chemo-refractory, Unresectable, Colorectal AdenocarcinomaColorectal Cancer
RECRUITING834 Analytics
NCT06346197Combination of Immune Checkpoint in Locally Advanced or Metastatic MSI/dMMR Esogastric AdenocarcinomasGastric Cancer
RECRUITING132 Analytics
PHASE3RECRUITING
Botensilimab + Balstilimab vs Best Supportive Care as Therapy in Chemo-refractory, Unresectable, Colorectal Adenocarcinoma
Colorectal CancerUnlock trial analytics
PHASE3RECRUITING
Combination of Immune Checkpoint in Locally Advanced or Metastatic MSI/dMMR Esogastric Adenocarcinomas
Gastric CancerUnlock trial analytics

Study Endpoints

Primary Endpoints

Overall Survival
34 months
Survival of patients
at least 2 years

Comparison of survival of patients under Botensilimab + Balstilimab versus the standard of care FOLFOX/XELOX + nivolumab

Unacceptable toxicity rate
42 days

Unacceptable toxicity rate in cycle 1 of \< 33%

Landmark survival analyses
18 months

Overall survival and progression-free survival at 12 and 18 months, as well as median progression-free and overall survival.

Objective Response Rate (ORR)
First dose to up to 27 months

ORR is defined as the percentage of participants with complete response (CR) or partial response (PR) assessed per the Lugano criteria

Determine the progression-free survival rate
6 months

Determine the progression-free survival rate at 6 months of combination therapy with doxorubicin, AGEN1884, and AGEN2034 in anthracycline-naïve patients with advanced or metastatic soft tissue sarcomas.

Secondary Endpoints

Progression Free Survival (PFS) based on RECIST 1.1 criteria
34 months
Overall Response Rate (ORR) based on RECIST 1.1 criteria
34 months
Clinical Benefit Rate (CBR) at 6 months based on RECIST 1.1 criteria
34 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Best Supportive CareACTIVE_COMPARATOR -
Botensilimab + Balstilimab and Best Supportive CareEXPERIMENTAL -
Experimental armEXPERIMENTALPatients treated with Botensilimab + Balstilimab
Standard armACTIVE_COMPARATORo FOLFOX\*, IV, Q2W + Nivolumab 240mg, IV, Q2. \*FOLFOX = oxaliplatin 85 mg/m2 , leucovorin 400 mg/m2 , and fluorouracil 400 mg/m2 administered IV on Day 1 of each treatment cycle, and fluorouracil 1200 mg/m2 IV continuous infusion over 24 hours daily or per local standard on Days 1 and 2 of each treatment cycle, every 2 weeks. Premedication is not usually required in the first cycle. For subsequent cycles, adequate premedication may be administrated per local standard. OR * XELOX\*, IV, Q3W + Nivolumab (360 mg, IV, Q3W). XELOX = Oxaliplatin 130mg/m² IV on Day 1 of each treatment cycle + capecitabine 1000mg/m² orally twice daily on Days 1 to 14 of each treatment cycle, every 3 weeks * Treatment is recommended until disease progression, unacceptable toxicity, or up to 24 months in patients without disease progression.
Blood-brain barrier opening with concomitant BAL, BOT, DOXEXPERIMENTALPatients will undergo implantation of Sonocloud-9 after completion of radiotherapy. Within 21 days after implant, ultrasound-based BBB opening with concomitant administration of DOX (30 mg) + BAL (450 mg) every 3 weeks, and BOT (dose 1mg/kg) every 6 weeks will be initiated.
BalstilimabEXPERIMENTAL300 mg IV every 3 weeks for a maximum of 24 months
Part 1: Stage 1, Safety Lead-InEXPERIMENTALThe safety lead-in will enroll 6 participants. Balstilimab 300mg flat q3 weeks up to 2 years; Zalifrelimab 1mg/kg q6 weeks x 4; Doxorubicin 75mg/m2 q3 weeks x 2, starts at C2 The participants will complete a dose-limiting toxiciy (DLT) observation period of 9 weeks.
Part 1: Stage 2, ExpansionEXPERIMENTALBalstilimab 300mg flat q3 weeks up to 2 years; Zalifrelimab 1mg/kg q6 weeks up to 2 years; Doxorubicin 75mg/m2 q3 weeks x 6
Part 2: Stage 2, Dose 1EXPERIMENTALDoxorubicin 60mg/m2 q3 weeks x 6 doses Botensilimab 75mg q6 weeks up to two years
Stage 2, Dose 2EXPERIMENTALDoxorubicin 75mg/m2 q3 weeks x 6 doses Botensilimab 75mg q6 weeks up to two years
Stage 2, Dose 3 Combination of Doxorubicin with Botensilimab and BalstilimabEXPERIMENTALDoxorubicin 75mg/m2 q3 weeks x 6 doses Botensilimab 75mg flat q6 weeks up to 2 years Balstilimab 450mg q3 weeks up to 2 years

Interventions

NameTypeDescription
Best Supportive CareOTHERMeasures designed to provide palliation of symptoms and improve quality of life as much as possible
BalstilimabDRUG450mg IV every 3 weeks
BotensilimabDRUG75mg IV every 6 weeks x 4 doses
Folfox ProtocolDRUGoxaliplatin 85 mg/m2 , leucovorin 400 mg/m2 , and fluorouracil 400 mg/m2 administered IV on Day 1 of each treatment cycle, and fluorouracil 1200 mg/m2 IV continuous infusion over 24 hours daily or per local standard on Days 1 and 2 of each treatment cycle, every 2 weeks.Treatment is recommended until disease progression, unacceptable toxicity, or up to 24 months in patients without disease progression.
XELOXDRUGOxaliplatin 130mg/m² IV on Day 1 of each treatment cycle + capecitabine 1000mg/m² orally twice daily on Days 1 to 14 of each treatment cycle, every 3 weeks. Treatment is recommended until disease progression, unacceptable toxicity, or up to 24 months in patients without disease progression.
NivolumabDRUG240mg, IV, Q2. Treatment is recommended until disease progression, unacceptable toxicity, or up to 24 months in patients without disease progression.
Liposomal DoxorubicinDRUGLiposomal Doxorubicin 30 mg IV over 30 minutes every 3 weeks
Sonocloud-9 (SC-9)DEVICEDevice activation of 9 ultrasound emitters during IV injection of microbubbles every 3 weeks
ZalifrelimabDRUGZalifrelimab (AGEN1884) is a fully human monoclonal immunoglobulin G1 κ subclass (IgG1κ) antibody that specifically recognizes cytotoxic T lymphocyte-associated protein 4 (CTLA-4, also known as CD152). Zalifrelimab (AGEN1884) is being developed as a monotherapy for cancer indications with potential for future development in combination with other immunotherapies.
DoxorubicinDRUGDoxorubicin is the standard of care first-line therapy for most subtypes of metastatic soft tissue sarcomas. Doxorubicin monotherapy administered at 75 mg/m2 has resulted in objective response rate of 14%, and median progression-free survival of 4.6 months, and another study reported progression-free survival at 6 months of 46.3%, with a median PFS of 5.8 months, and best objective response rate of 19%.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites55

Inclusion Criteria: * Must have histologically confirmed colorectal adenocarcinoma that is not deficient mismatch repair (dMMR) or microsatellite instability-high (MSI-H). * Received and failed all prior available therapies, such that the standard of care for the patient would be best supportive ca...

Countries:CanadaFranceUnited StatesArmenia
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Recent Changes (Last 90 Days)

LOWAug 14, 2026NCT05864534lastUpdatePostDate: changed
LOWAug 14, 2026NCT05864534lastUpdatePostDate: changed
LOWJun 29, 2026NCT07152821lastUpdatePostDate: changed
LOWJun 29, 2026NCT07152821lastUpdatePostDate: changed
LOWJun 25, 2026NCT06346197lastUpdatePostDate: changed
LOWJun 25, 2026NCT06346197lastUpdatePostDate: changed
LOWJun 25, 2026NCT06346197lastUpdatePostDate: changed
LOWJun 11, 2026NCT07152821lastUpdatePostDate: changed
LOWJun 11, 2026NCT07152821lastUpdatePostDate: changed
LOWJun 9, 2026NCT07152821lastUpdatePostDate: changed
LOWJun 9, 2026NCT07152821lastUpdatePostDate: changed
LOWJun 9, 2026NCT07152821lastUpdatePostDate: changed
LOWJun 9, 2026NCT07152821lastUpdatePostDate: changed

Frequently asked questions about Balstilimab

What is Balstilimab used for?

Balstilimab is an investigational oncology therapy being studied for gastric cancer, colorectal cancer, lymphoma, metastatic soft tissue sarcoma, and newly diagnosed glioblastoma. It is in Phase 3 clinical development for certain indications. The drug is being evaluated in clinical trials for these cancer types.

What does Balstilimab target?

Balstilimab targets PDCD1, also known as programmed cell death protein 1, and acts as an antagonist. By blocking PDCD1, it is designed to modulate immune responses in cancer treatment. This mechanism is being investigated across multiple oncology indications.

Who makes Balstilimab?

Balstilimab is being developed by Agenus Inc., a biopharmaceutical company traded on NASDAQ under the ticker AGEN. The company is conducting clinical trials to evaluate the drug's safety and efficacy in various cancer types.

What phase is Balstilimab in?

Balstilimab is in Phase 3 clinical development for gastric cancer and colorectal cancer. It is also being studied in Phase 2 trials for lymphoma and newly diagnosed glioblastoma. The drug remains investigational and has not been approved by regulatory authorities.

What clinical trials is Balstilimab in?

Balstilimab is being evaluated in several clinical trials, including NCT07152821, a Phase 3 study in colorectal cancer with 834 participants, and NCT06346197, a Phase 3 trial in gastric cancer. Phase 2 trials include NCT05891821 for lymphoma and NCT05864534 for glioblastoma.

Is Balstilimab the same as botensilimab?

No, Balstilimab and botensilimab are different drugs. Balstilimab targets PDCD1, while botensilimab is a separate agent. They are being studied in combination, such as in the Phase 3 trial NCT07152821 for colorectal cancer, but they are distinct investigational therapies.