Niktimvo (axatilimab-csfr) / Knight Therap, Syndax Pharma, UCB, Incyte, Royalty - LARVOL DELTA

Skin Responses in Patients With Chronic Graft-Versus-Host Disease Treated With Axatilimab in the AGAVE-201 Trial Using Current and Modified NIH Criteria (HOPA 2026) - P2 | "Application of the modified NIH criteria identified improvements in sclerotic cutaneous cGVHD not captured by the 2014 NIH criteria, highlighting the importance of multidimensional outcome measures for cGVHD."

Clinical • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • CSF1R

Infection Characteristics in Patients Treated With Axatilimab for Chronic Graft-Versus-Host Disease in the AGAVE-201 Trial (HOPA 2026) - P2 | "Among 7 patients receiving cyclosporine and 11 receiving tacrolimus, 1 (14.3%) and 3 (27.3%), respectively, completed tapering within 6 cycles. Among 241 randomized patients, 192 (79.7%) had severe cGVHD with a median of 4 prior cGVHD therapies; 130 (53.9%) had ≥4 organs involved. Concomitant medications included antivirals, most commonly acyclovir (60.6%); antibacterials, including sulfamethoxazole/trimethoprim (63.9%) and azithromycin (32.4%); and antifungals, including posaconazole (24.1%) and fluconazole (11.6%). Concomitant corticosteroid use at baseline was reported in 156/239 (65.3%) patients; mean daily dose was 19.4 mg."

Clinical • Chronic Graft versus Host Disease • Cytomegalovirus Infection • Epstein-Barr Virus Infections • Graft versus Host Disease • Immunology • Infectious Disease • Novel Coronavirus Disease • Pneumonia • Respiratory Diseases

Long-Term Safety of Axatilimab and Feasibility of Switching to 0.6 mg/kg Every 4 Weeks Dosing in Patients Treated in the AGAVE-201 Trial: Considerations for Pharmacists (HOPA 2026) - P2 | "Among the 80 patients randomized to axatilimab 0.3 mg/kg Q2W, 59 (73.8%) achieved a response by Cycle 7, and 19 transitioned from 0.3 mg/kg Q2W to 0.6 mg/kg Q4W. Median (range) treatment duration before and after the switch were 7.4 (6–14) and 20.9 (2–32) months, respectively. Q4W dosing was maintained in 16 patients (84.2%)."

Clinical • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology

Infusion-Related Reactions in Patients Treated With Axatilimab for Chronic Graft-Versus-Host Disease in the AGAVE-201 Study (HOPA 2026) - P2 | "At the time of the IRR, 13 patients (56.5%) were receiving concomitant medications, mainly prednisone (26.1%), prednisolone (21.7%), topical hydrocortisone (13.0%), famotidine (13.0%), hydrocortisone (8.7%), and diphenhydramine (8.7%). Of the 239 patients who received axatilimab during AGAVE-201, 23 patients (9.6%) experienced 38 IRRs. IRR incidence by group was 11/79 patients (13.9%) for 0.3 mg/kg Q2W, 5/81 patients (6.2%) for 1 mg/kg Q2W, and 7/79 patients (8.9%) for 3 mg/kg Q4W. Among these 23 patients, cGVHD most commonly involved the eyes (69.6%), skin (69.6%), and lungs (56.5%)."

Clinical • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Urticaria

SNDX-6352-0506: MAXPIRe: Study to Evaluate Axatilimab in Participants With Idiopathic Pulmonary Fibrosis (IPF) (clinicaltrials.gov) - P2 | N=145 | Active, not recruiting | Sponsor: Syndax Pharmaceuticals | Recruiting ➔ Active, not recruiting

Enrollment closed • Idiopathic Pulmonary Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases

CLINICAL OUTCOMES AMONG PATIENTS WITH CHRONIC GRAFT-VERSUS-HOST DISEASE TREATED WITH AXATILIMAB: ANALYSIS OF AN EXPANDED ACCESS PROGRAM (EBMT 2026) - P | "The most common concomitant cGVHD therapies were belumosudil (76.0%), prednisone (63.5%), and ruxolitinib (54.8%); fewer patients received concomitant calcineurin inhibitors (21.2%) and sirolimus (16.3%). The US EAP represents the largest real-world experience of axatilimab treatment at the FDA-approved dose of 0.3 mg/kg Q2W in patients with cGVHD. Most patients (86.5%) experienced clinical benefit from axatilimab treatment per clinician assessment, and the majority (67.3%) transitioned to commercial product upon EAP closure, suggesting ongoing clinical benefit. Axatilimab safety was consistent with that observed in clinical trials, with no new or unexpected toxicities."

Clinical • Clinical data • Chronic Graft versus Host Disease • Fibrosis • Graft versus Host Disease • Immunology • Inflammation • CSF1R

SKIN RESPONSES USING MODIFIED CRITERIA AMONG PATIENTS WITH SCLEROTIC CHRONIC GRAFT-VERSUS-HOST DISEASE TREATED WITH AXATILIMAB IN THE AGAVE-201 TRIAL (EBMT 2026) - P2 | "These data demonstrate that axatilimab was associated with sBSA and skin/joint tightening improvement at C7D1 in a subset of patients with sclerotic cGVHD who had a skin feature score of 3 and would not have met the 2014 NIH criteria for clinical response. These data highlight the need for additional, sensitive assessments of treatment benefit in clinical trials for patients with sclerotic cutaneous involvement."

Clinical • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • CSF1R

ASSESSMENTS OF BONE HEALTH AMONG PATIENTS WITH CHRONIC GRAFT-VERSUS-HOST DISEASE RECEIVING AXATILIMAB IN THE AGAVE-201 TRIAL (EBMT 2026) - P2 | "In AGAVE-201, axatilimab was associated with minimal changes in bone turnover markers in patients with cGVHD. Bone-related TEAEs were rare, with only a small number of patients having multiple fractures; this is notable given that 65.3% of patients received concomitant corticosteroids. These findings may indicate that axatilimab had minimal effects on bone metabolic markers, although additional analyses, including bone density measurements in this population with refractory cGVHD, are needed."

Clinical • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Fibrosis • Gastrointestinal Disorder • Graft versus Host Disease • Immunology • Inflammation • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Osteoarthritis • Osteoporosis

SAFETY AND FEASIBILITY OF 0.6 MG/KG EVERY 4 WEEKS DOSING OF AXATILIMAB IN PATIENTS TREATED IN THE AGAVE-201 STUDY (EBMT 2026) - P2 | "Transition to 0.6 mg/kg Q4W (without dose capping) is feasible and well tolerated in patients treated with axatilimab 0.3 mg/kg Q2W in AGAVE-201. The Q4W dosing strategy at the 0.6 mg/kg dose identified no new safety signals. Patients had a prolonged treatment duration (median, 20.9 months [1.7 y]), and 84% of patients continued treatment, suggesting ongoing clinical benefit."

Clinical • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Infectious Disease • Pneumonia • Respiratory Diseases • CSF1R

CHARACTERISTICS OF CLINICAL TRIALS IN GRAFT-VERSUS-HOST DISEASE: LANDSCAPE OF STUDY DRUGS AND ASSESSMENT OF OUTCOME MEASURES (EBMT 2026) - "The most frequent treatments studied were corticosteroids (n=64), followed by mesenchymal stem cells (n=52), ruxolitinib (n=31), extracorporeal photopheresis (n=26), fecal microbiota transplantation (n=17), rituximab (n=15), belumosudil and cyclosporin (n=13 each), itacitinib (n=11), axatilimab and donor regulatory T-cells (n=10 each), and ibrutinib (n=9). Acute and chronic GvHD remain major areas of clinical research, with their distinct clinical courses reflected in differences in trial design and outcome measures. Recent shifts toward multicenter designs, shorter study durations, and more frequent evaluation of targeted therapies suggest evolving priorities and growing methodological maturity in GvHD trial planning."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology

SYNERGISTIC EFFECTS OF AXATILIMAB AND RUXOLITINIB IN AN INFLAMMATORY IN VITRO MODEL OF GVHD (EBMT 2026) - "Our in vitro findings demonstrate that CSF-1R– and JAK-dependent cytokine networks function as interconnected drivers of cell survival and inflammatory activity relevant to cGVHD. Combined CSF-1R and JAK inhibition produced coordinated effects on monocyte viability and cytokine output not observed with either agent alone, suggesting that dual targeting disrupts reinforcing circuits between myeloid and lymphoid pathways. These results support the mechanistic rationale for therapeutic combinations of axatilimab and ruxolitinib to interrupt the immunologic programs sustaining cGVHD more effectively."

Preclinical • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • CCL2 • IL10

SINGLE-AGENT CSF-1R OR JAK INHIBITION REDUCES FIBROSIS IN A MURINE MODEL OF CHRONIC GRAFT-VERSUS-HOST DISEASE (EBMT 2026) - "While CSF-1 receptor (CSF-1R) inhibition (axatilimab) and JAK1/JAK2 inhibition (ruxolitinib) have each shown clinical benefit, the potential for combined targeting of these pathways warrants further exploration. Both CSF-1R and JAK pathway inhibition individually attenuated in vivo tissue fibrosis and improved clinical outcomes in a murine cGVHD model. RNA-seq analysis of skin identified transcriptional changes associated with each treatment. These findings, derived from single-agent therapeutic approaches currently used in cGVHD, support the therapeutic potential of targeting CSF-1R and JAK pathways in this setting."

Preclinical • Chronic Graft versus Host Disease • Fibrosis • Graft versus Host Disease • Immunology • JAK1

A COMPREHENSIVE ANALYSIS OF AXATILIMAB IN PATIENTS WITH CHRONIC GRAFT-VERSUS-HOST DISEASE AND RELATED BRONCHIOLITIS OBLITERANS SYNDROME: INTEGRATED ANALYSIS FROM 2 CLINICAL STUDIES (EBMT 2026) - P1/2, P2 | " Of 117 patients with cGVHD-BOS at baseline, 84.6% had prior treatment with belumosudil, ibrutinib, and/or ruxolitinib, and the median (range) number of organs involved at baseline was 4 (1–9). Axatilimab demonstrated clinical and symptom responses in patients with cGVHD-BOS across a spectrum of lung involvement, including those with FEV1 ≤39% and NIH lung scores of 3. Symptom responses were observed in some patients who were NIH nonresponders. There were no statistically significant differences in NIH lung response among patients with different baseline characteristics, suggesting that a broad range of patients with BOS may respond to axatilimab."

Clinical • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Infectious Disease • Novel Coronavirus Disease • Pneumonia • Pulmonary Disease • Respiratory Diseases

AXATILIMAB IN COMBINATION WITH RUXOLITINIB IN PATIENTS WITH NEWLY DIAGNOSED CHRONIC GRAFT-VERSUS-HOST DISEASE: UPDATED SAFETY ANALYSIS OF A RANDOMIZED, PHASE 2 STUDY (EBMT 2026) - P2 | "In the updated safety analysis of this phase 2 trial, the combination of AXA+RUX was well tolerated and had a similar safety profile to RUX alone, with no evidence of additive toxicity."

Clinical • Combination therapy • P2 data • Bone Marrow Transplantation • Chronic Graft versus Host Disease • CNS Disorders • Constipation • Fibrosis • Gastroenterology • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Inflammation • Insomnia • Mood Disorders • Respiratory Diseases • Sleep Disorder • CSF1R • JAK1 • JAK2

EFFICACY AND SAFETY OF AXATILIMAB IN JAPANESE PATIENTS WITH RECURRENT OR REFRACTORY CHRONIC GRAFT-VERSUS-HOST DISEASE AFTER AT LEAST 2 PRIOR LINES OF SYSTEMIC THERAPY (EBMT 2026) - P2, P3 | "Axatilimab 0.3 mg/kg Q2W demonstrated clinical benefit and was well tolerated in Japanese patients with recurrent or refractory cGVHD."

Clinical • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Dermatology • Fibrosis • Graft versus Host Disease • Immunology • Infectious Disease • Inflammation • Influenza • Pruritus • Respiratory Diseases

HARNESSING GRAFT-VERSUS-LEUKEMIA ALLOREACTIVITY TO TREAT MULTI-REFRACTORY ACUTE LYMPHOBLASTIC LEUKEMIA AFTER ALLOGENEIC HSCT (EBMT 2026) - "Particular attention was given to the rationale, timing, and outcomes of combining inotuzumab ozogamicin with haplo-DLI and IFN-α...Marrow relapse after CD19 CAR-T and another relapse after CD22 CAR-T prompted a second HSCT—a maternal haploidentical transplant—on 4/21/2020...The patient subsequently developed severe chronic SR-GvHD requiring prolonged therapy with MSCs, ruxolitinib, belumosudil, and axatilimab... This case demonstrates that intentionally harnessed GvL—via haploidentical DLI augmented with IFN-α—can produce deep, durable remission in profoundly multi-refractory pediatric ALL. Although associated with significant acute and chronic GvHD, coordinated multidisciplinary management enabled control of both leukemia and immune dysregulation. These findings support further evaluation of cytokine-enhanced DLI strategies as a salvage modality for high-risk pediatric patients who relapse despite HSCT and advanced cellular therapies."

Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • CD22 • IFNA1

CHARACTERISTICS OF CLINICAL TRIALS IN GRAFT-VERSUS-HOST DISEASE: LANDSCAPE OF STUDY DRUGS AND ASSESSMENT OF OUTCOME MEASURES (EBMT 2026) - "The most frequent treatments studied were corticosteroids (n=64), followed by mesenchymal stem cells (n=52), ruxolitinib (n=31), extracorporeal photopheresis (n=26), fecal microbiota transplantation (n=17), rituximab (n=15), belumosudil and cyclosporin (n=13 each), itacitinib (n=11), axatilimab and donor regulatory T-cells (n=10 each), and ibrutinib (n=9). Acute and chronic GvHD remain major areas of clinical research, with their distinct clinical courses reflected in differences in trial design and outcome measures. Recent shifts toward multicenter designs, shorter study durations, and more frequent evaluation of targeted therapies suggest evolving priorities and growing methodological maturity in GvHD trial planning."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology

ASSESSMENTS OF BONE HEALTH AMONG PATIENTS WITH CHRONIC GRAFT-VERSUS-HOST DISEASE RECEIVING AXATILIMAB IN THE AGAVE-201 TRIAL (EBMT 2026) - P2 | "In AGAVE-201, axatilimab was associated with minimal changes in bone turnover markers in patients with cGVHD. Bone-related TEAEs were rare, with only a small number of patients having multiple fractures; this is notable given that 65.3% of patients received concomitant corticosteroids. These findings may indicate that axatilimab had minimal effects on bone metabolic markers, although additional analyses, including bone density measurements in this population with refractory cGVHD, are needed."

Clinical • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Fibrosis • Gastrointestinal Disorder • Graft versus Host Disease • Immunology • Inflammation • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Osteoarthritis • Osteoporosis • Rheumatology

SAFETY AND FEASIBILITY OF 0.6 MG/KG EVERY 4 WEEKS DOSING OF AXATILIMAB IN PATIENTS TREATED IN THE AGAVE-201 STUDY (EBMT 2026) - P2 | "Transition to 0.6 mg/kg Q4W (without dose capping) is feasible and well tolerated in patients treated with axatilimab 0.3 mg/kg Q2W in AGAVE-201. The Q4W dosing strategy at the 0.6 mg/kg dose identified no new safety signals. Patients had a prolonged treatment duration (median, 20.9 months [1.7 y]), and 84% of patients continued treatment, suggesting ongoing clinical benefit."

Clinical • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Infectious Disease • Pneumonia • Respiratory Diseases • CSF1R

SKIN RESPONSES USING MODIFIED CRITERIA AMONG PATIENTS WITH SCLEROTIC CHRONIC GRAFT-VERSUS-HOST DISEASE TREATED WITH AXATILIMAB IN THE AGAVE-201 TRIAL (EBMT 2026) - P2 | "These data demonstrate that axatilimab was associated with sBSA and skin/joint tightening improvement at C7D1 in a subset of patients with sclerotic cGVHD who had a skin feature score of 3 and would not have met the 2014 NIH criteria for clinical response. These data highlight the need for additional, sensitive assessments of treatment benefit in clinical trials for patients with sclerotic cutaneous involvement."

Clinical • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • CSF1R

CLINICAL OUTCOMES AMONG PATIENTS WITH CHRONIC GRAFT-VERSUS-HOST DISEASE TREATED WITH AXATILIMAB: ANALYSIS OF AN EXPANDED ACCESS PROGRAM (EBMT 2026) - P | "The most common concomitant cGVHD therapies were belumosudil (76.0%), prednisone (63.5%), and ruxolitinib (54.8%); fewer patients received concomitant calcineurin inhibitors (21.2%) and sirolimus (16.3%). The US EAP represents the largest real-world experience of axatilimab treatment at the FDA-approved dose of 0.3 mg/kg Q2W in patients with cGVHD. Most patients (86.5%) experienced clinical benefit from axatilimab treatment per clinician assessment, and the majority (67.3%) transitioned to commercial product upon EAP closure, suggesting ongoing clinical benefit. Axatilimab safety was consistent with that observed in clinical trials, with no new or unexpected toxicities."

Clinical • Clinical data • Chronic Graft versus Host Disease • Fibrosis • Graft versus Host Disease • Immunology • Inflammation • CSF1R

SINGLE-AGENT CSF-1R OR JAK INHIBITION REDUCES FIBROSIS IN A MURINE MODEL OF CHRONIC GRAFT-VERSUS-HOST DISEASE (EBMT 2026) - "While CSF-1 receptor (CSF-1R) inhibition (axatilimab) and JAK1/JAK2 inhibition (ruxolitinib) have each shown clinical benefit, the potential for combined targeting of these pathways warrants further exploration. Both CSF-1R and JAK pathway inhibition individually attenuated in vivo tissue fibrosis and improved clinical outcomes in a murine cGVHD model. RNA-seq analysis of skin identified transcriptional changes associated with each treatment. These findings, derived from single-agent therapeutic approaches currently used in cGVHD, support the therapeutic potential of targeting CSF-1R and JAK pathways in this setting."

Preclinical • Chronic Graft versus Host Disease • Fibrosis • Graft versus Host Disease • Immunology • JAK1

SYNERGISTIC EFFECTS OF AXATILIMAB AND RUXOLITINIB IN AN INFLAMMATORY IN VITRO MODEL OF GVHD (EBMT 2026) - "Our in vitro findings demonstrate that CSF-1R– and JAK-dependent cytokine networks function as interconnected drivers of cell survival and inflammatory activity relevant to cGVHD. Combined CSF-1R and JAK inhibition produced coordinated effects on monocyte viability and cytokine output not observed with either agent alone, suggesting that dual targeting disrupts reinforcing circuits between myeloid and lymphoid pathways. These results support the mechanistic rationale for therapeutic combinations of axatilimab and ruxolitinib to interrupt the immunologic programs sustaining cGVHD more effectively."

Preclinical • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • CCL2 • IL10

Axatilimab Plus Ruxolitinib Shows Manageable Safety in Untreated cGVHD (Targeted Oncology) - "As of the October 3, 2025, data cutoff, 66 patients had been randomly assigned across 3 arms, with 56 patients receiving at least one dose of treatment. Anemia was the only grade 3 or higher TEAE occurring in 2 or more patients overall, with 1 case each in the combination and ruxolitinib arms. The prespecified stopping criterion for unacceptable toxicity was not met, and no treatment arm approached prespecified stopping thresholds....Median treatment duration was 4.3 months for the combination, 4.7 months for ruxolitinib, and 2.0 months for corticosteroids. Treatment discontinuation was highest in the corticosteroid arm (47.4%), followed by ruxolitinib (25.0%) and axatilimab/ruxolitinib (17.4%), with approximately one-third of patients in the corticosteroid group discontinuing to initiate a new systemic therapy for cGVHD."

P2 data • Chronic Graft versus Host Disease

Results from the largest real-world experience of axatilimab (Niktimvo) in chronic graft-vs-host disease (cGVHD) demonstrated that 86.5% of patients experienced clinical benefit from the therapy, according to findings reported in a poster presented at the 52nd Annual Meeting of the European Society for Blood and Marrow Transplantation in Madrid, Spain. (Targeted Oncology) - "At data cutoff of October 24, 2025, the median duration of treatment was 123 days (range, 14-358). Of the 104 treated patients, 90 (86.5%) were reported to have experienced clinical benefit from axatilimab during the EAP. Although most patients (73.1%) maintained stable cGVHD severity based on NIH 2014 criteria, the high rate of clinician-reported benefit reflected meaningful real-world impact across a diverse and heavily pretreated group."

Real-world evidence • Chronic Graft versus Host Disease