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Axatilimab

Phase 2

Chronic Graft Versus Host Disease | Monoclonal antibody | Immunology |Incyte Corporation|Last Updated: Sep 2, 2026

Target and mechanism

Molecular targetCSF1R
Target classInhibitor
ModalityMonoclonal antibody

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment49

FDA Designations

No designations recorded

Clinical trial landscape

Axatilimab · 7 trials · 22 indications

Phase 2 3Phase 1 4
NCT06663722Axatilimab in Combination With Extracorporeal Photopheresis (ECP) in Chronic Graft-versus-Host DiseaseChronic Graft Versus Host Disease
RECRUITING49 Analytics
NCT06388564A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host DiseaseChronic Graft-versus-host-disease
ACTIVE NOT_RECRUITING130 Analytics
NCT05723055Evaluating Combination of Nivolumab and Axatilimab in Patients With Relapsed/Refractory Classical Hodgkin LymphomaHodgkin Lymphoma
ACTIVE NOT_RECRUITING9 Analytics
PHASE2RECRUITING
Axatilimab in Combination With Extracorporeal Photopheresis (ECP) in Chronic Graft-versus-Host Disease
Chronic Graft Versus Host DiseaseUnlock trial analytics
PHASE2ACTIVE NOT_RECRUITING
A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease
Chronic Graft-versus-host-diseaseUnlock trial analytics
PHASE2ACTIVE NOT_RECRUITING
Evaluating Combination of Nivolumab and Axatilimab in Patients With Relapsed/Refractory Classical Hodgkin Lymphoma
Hodgkin LymphomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Best Overall Response Rate (ORR)
Up to 24 weeks

Best overall response rate (ORR) will be reported as the percentage of participants who achieve partial response (PR) or a complete response (CR) to study therapy, as defined by the 2014 National Institutes of Health (NIH) Consensus Development Project on Criteria for Clinical Trials in chronic graft-versus-host disease (cGVHD) while on study treatment.

Objective Response Rate
6 months

Defined as Complete Response (CR) or Partial Response (PR) at 6 months in the absence of new systemic therapy for cGVHD. Response assessment will be based on the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD.

Objective response rate (ORR) as measured by Best Overall Response Rate (BOR) measured by the proportion of subjects achieving a confirmed PR and CR as defined by Lugano Criteria
At the end of 6 cycles of Treatment. Each cycle is 28 days

evaluate the efficacy of combination of Axatilimab (SNDX-6352) and Nivolumab in the study population

Pharmacokinetics Parameter: Cmax of axatilimab
Pre dose and Post dose on Day 1, Post dose Days 2, 3, 4, 6, 8, 15, 22, 28 and 60

Maximum Observed Plasma Concentration of axatilimab.

Pharmacokinetics Parameter: AUC(0-t) of axatilimab
Pre dose and Post dose on Day 1, Post dose Days 2, 3, 4, 6, 8, 15, 22, 28 and 60

Area Under the concentration- time curve up to the last measurable concentration of axatilimab.

Pharmacokinetics Parameter: AUC 0-∞ of axatilimab
Pre dose and Post dose on Day 1, Post dose Days 2, 3, 4, 6, 8, 15, 22, 28 and 60

Area Under the Concentration-time Curve From 0 to Infinity of axatilimab.

Number of participants with Treatment Emergent Advers Events (TEAE's)
Up to 3 months

Adverse events reported for the first time or worsening of a pre-existing event after first dose of study drug/treatment.

Maximum Tolerated Dose (MTD)
Up to 4 weeks

MTD is determined by Bayesian optimal interval (BOIN) design, where the target toxicity rate for the MTD is 0.25 and the maximum sample size is 20.

Recommended Phase 2 Dose (RP2D)
Up to 4 weeks

If the MTD is identified as 3mg/kg, we will complete enrollment of 10 pts at the 1 mg/kg dose level, to determine if there is biological effectiveness (and clinical efficacy) at the lower dose level.

Incidence of dose limiting toxicities
Up to 42 days after the first dose of study medication

Includes hematologic and non-hematologic toxicities

Overall response rate
Up to 5 years

The number of participants whose best response (according to the 2006 International Working Group response criteria) over the efficacy analysis period is a complete response or partial response will be tallied to estimate the overall response rate and provide a 95% exact confidence interval, according to the Clopper-Pearson method.

Incidence of dose limiting toxicities (phase Ib)
From the first dose of axatilimab (day -8) to the end of cycle 1 (cycle is 28 days starting from cycle 1 day 1 [first dose of retifanlimab and paclitaxel])

Will be coded by system organ class, MedDRA preferred term, and severity grade using Common Terminology Criteria for Adverse Events (version 5.0).

Recommended phase 2 dose of the study drug combination (phase Ib)
From the first dose of axatilimab (day -8) to the end of cycle 1 (cycle is 28 days starting from cycle 1 day 1 [first dose of retifanlimab and paclitaxel])
Clinical benefit rate (phase Ib/II)
From screening assessment to the end of cycle 6 assessment (each cycle is 28 days starting from cycle 1 day 1 [first dose of retifanlimab and paclitaxel])

Defined as percentage of participants with complete response or partial response confirmed by scans at least 4 weeks apart, or stable disease for \> 4 months. Will be reported with exact 95% confidence interval.

Secondary Endpoints

Proportion of participants experiencing treatment-related adverse events (AEs)
Up to 15 months
Proportion of participants experiencing serious adverse events (SAEs)
Up to 15 months
Change in cumulative dose of corticosteroid usage
Baseline, 24 weeks, 1 year
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Axatilimab in combination with ECP GroupEXPERIMENTALParticipants in this group will receive Axatilimab in combination with extracorporeal photopheresis (ECP) therapy for up to seven (7) four-week cycles. Total participation duration is about 15 months.
Treatment Group AEXPERIMENTALAxatilimab will be administered at a protocol defined starting dose plus ruxolitinib at a protocol defined starting dose.
Treatment Group BEXPERIMENTALRuxolitinib will be administered at a protocol defined starting dose.
Treatment Group CEXPERIMENTALCorticosteroids alone will be administered at a protocol defined starting dose.
Treatment: All PatientsEXPERIMENTALNivolumab 480 mg IV Q4 weeks Axatilimab (SNDX 6532) dose (3mg/kg IV) Q4 weeks. If DLT criteria are met, Axatilimab dosing will be reduced to 2mg/kg IV Q4W for the remainder of patients on the study. The combination will be continued until progression/toxicity up to a maximum of 12 cycles.
Cohort AEXPERIMENTALAxatilimab will be administered at a protocol defined starting dose administered as an IV infusion.
Cohort BEXPERIMENTALAxatilimab will be administered at a protocol defined starting dose administered as an SC injection.
Cohort CEXPERIMENTALAxatilimab will be administered at a protocol defined starting dose administered as an SC injection.
Cohort DEXPERIMENTALAxatilimab will be administered at a protocol defined starting dose administered as an SC injection.
Axatilimab + OlaparibEXPERIMENTALA Bayesian Optimal Interval design will be used to establish the maximum tolerated dose of Axatilimab. Dose reduction and escalation will be per protocol. Participants will complete: * Baseline visit, tumor biopsy, and imaging * Imaging every 8 weeks * Window Phase: --Predetermined dose of Olaparib 2x daily, taken 12 hours apart for 14 days * Combination Treatment Phase: * Cycle 1 through Cycle 2: * Day 1 of 28 day cycle: Tumor biopsy * Days 1 and 15 of 28 day Cycle: Predetermined dose of Axalitimab 1x daily * Days 1 through 28 of 28 day cycle Predetermined dose of Olaparib 2x daily, 12 hours apart * Tumor biopsy after two weeks of Olaparib and at the end of Cycle 2 * Cycle 3 through End of Treatment: * Days 1 and 15 of 28 Day Cycle: Predetermined dose of Axalitimab 1x daily * Predetermined dose of Olaparib 2x daily, taken 12 hours apart * End of Treatment Visit with imaging * Follow Up: Every 6 months for 3 years. Imaging will be every 10 weeks.
Phase I (Axatilimab)EXPERIMENTALPatients receive axatilimab IV over 30 minutes on days 1 and 15 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. At time of phase Ib completion, patients with clinical improvement may transition to phase II. Patients undergo bone marrow biopsy and aspiration and blood sample collection throughout the study.
Phase II (Axatilimab and azacitidine)EXPERIMENTALPatients receive axatilimab IV over 30 minutes on days 1 and 15 and azacitidine IV over 10-40 minutes or SC on days 1-7 of each cycle. Cycles repeat every 28 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity. Patients who achieve PR or better may continue for up to 24 total cycles. Patients who achieve less than PR receive 2 additional cycles and, if PR or better is achieved, may complete up to 24 total cycles. Patients undergo bone marrow biopsy and aspiration and blood sample collection throughout the study.
Treatment (axatilimab, retifanlimab, paclitaxel)EXPERIMENTALPatients receive axatilimab IV over 30 minutes on day -8, prior to cycle 1. Beginning in cycle 1 day 1, patients receive axatilimab IV over 30 minutes on days 8 and 21 of each cycle, retifanlimab IV over 30-60 minutes on day 1 of each cycle, and paclitaxel IV over 60 minutes on days 1, 8, and 15 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients also undergo tumor biopsy, CT scan, and blood sample collection throughout the study and may undergo MRI and/or PET scan throughout the study.

Interventions

NameTypeDescription
AxatilimabBIOLOGICALAxatilimab will be administered intravenously (IV) at a dose of 0.3 mg/kg, beginning as a pre-phase dose two weeks prior to initiation of Extracorporeal Photopheresis (ECP) therapy. Thereafter, Axatilimab will be administered with a frequency of one treatment session bi-weekly during each treatment cycle.
Extracorporeal PhotopheresisPROCEDUREMandatory ECP therapy will be administered at a frequency of two treatment sessions per week during Cycles 1 through 3, two treatment bi-weekly during Cycles 4 through 6, and two treatments during week 1 of Cycle 7. Optional ECP therapy will be administered at a frequency of two treatment sessions during weeks 2 and 4 of Cycles 4 through 6, when mandatory ECP is not administered. Optional ECP therapy will also be administered as two treatment sessions during week 3 of Cycle 7. After Cycle 7, participants may receive ECP therapy only at the Investigator's discretion for a maximum Treatment Period of 12 months.
RuxolitinibDRUGRuxolitinib will be administered at protocol defined dose.
CorticosteroidsDRUGCorticosteroids will be administered at protocol defined dose.
NivolumabDRUGNivolumab is a programmed death receptor-1 (PD-1)-blocking antibody
OlaparibDRUGInhibitor of poly ADP ribose polymerase (PARP)1-3, 100 or 150 mg tablet, taken orally per standard of care.
AzacitidineDRUGGiven IV or SC
Biospecimen CollectionPROCEDUREUndergo blood sample collection
Bone Marrow Aspiration and BiopsyPROCEDUREUndergo bone marrow biopsy and aspiration
Survey AdministrationOTHERAncillary study
BiopsyPROCEDUREUndergo tumor biopsy
Computed TomographyPROCEDUREUndergo CT scan
Magnetic Resonance ImagingPROCEDUREUndergo MRI
PaclitaxelDRUGGiven IV
Positron Emission TomographyPROCEDUREUndergo PET scan
RetifanlimabBIOLOGICALGiven IV
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Eligibility Criteria

Age Range12 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: 1. Recipient of allogeneic hematopoietic cell transplantation (HCT). 2. Age greater or equal to 12. 3. Chronic GVHD per 2014 National Institutes of Health Consensus Criteria (NCC) (Jagasia et al. 2015) or overlap syndrome requiring new therapy in patients with at least 2 prior l...

Countries:United StatesBelgiumCanadaGermanyItalySpainUnited Kingdom
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Recent Changes (Last 90 Days)

MEDIUMSep 2, 2026NCT06488378primaryCompletionDate: changed
MEDIUMSep 2, 2026NCT06488378primaryCompletionDate: changed
MEDIUMSep 2, 2026NCT06488378primaryCompletionDate: changed
LOWAug 27, 2026NCT06523556Enrollment: 52 → 49
LOWAug 27, 2026NCT06523556Enrollment: 52 → 49
LOWJul 8, 2026NCT06320405lastUpdatePostDate: changed
LOWJul 8, 2026NCT06320405lastUpdatePostDate: changed
LOWJun 22, 2026NCT06663722lastUpdatePostDate: changed
LOWJun 22, 2026NCT06663722lastUpdatePostDate: changed

Frequently asked questions about Axatilimab

What is Axatilimab used for?

Axatilimab is an investigational monoclonal antibody being developed for oncology and immunology indications, including advanced malignant solid neoplasms, Hodgkin lymphoma, breast cancer, chronic graft-versus-host disease, and atypical chronic myeloid leukemia. It is also being studied in combination with other agents for various advanced or metastatic solid tumors and myeloproliferative neoplasms.

What does Axatilimab target?

Axatilimab is a monoclonal antibody of the -mab class. Its specific molecular target has not been disclosed in the available clinical trial information. The drug is being investigated for its potential role in treating conditions such as chronic graft-versus-host disease and certain hematologic malignancies.

Who is developing Axatilimab?

Axatilimab is being developed by Incyte Corporation, a biopharmaceutical company traded on the NASDAQ under the ticker symbol INCY. Incyte is conducting multiple clinical trials to evaluate the safety and efficacy of Axatilimab across various oncology and immunology indications.

What phase is Axatilimab in?

Axatilimab is in early-stage clinical development. It is currently being evaluated in Phase 1 and Phase 2 clinical trials. The drug is investigational and has not been approved by regulatory authorities. Ongoing studies are assessing its use alone or in combination with other therapies for conditions like chronic graft-versus-host disease and advanced solid tumors.

What clinical trials is Axatilimab in?

Axatilimab is being studied in several clinical trials, including NCT06320405 (Phase 1, Axatilimab with retifanlimab and paclitaxel for advanced solid tumors), NCT06523556 (Phase 1, Axatilimab with or without azacitidine for myeloproliferative neoplasms), NCT06663722 (Phase 2, Axatilimab with extracorporeal photopheresis for chronic graft-versus-host disease), and NCT06713590 (Phase 1, single-dose subcutaneous versus intravenous Axatilimab in healthy participants).

Is Axatilimab the same as other drugs?

Axatilimab is a distinct investigational monoclonal antibody developed by Incyte Corporation. No alternative names or brand names have been disclosed in the available clinical trial information. It is being studied as a monotherapy and in combination with other agents, but it is not known to be identical to any other approved or investigational drug.