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

AXALAP: Phase Ib study of axatilimab in combination with olaparib in BRCA1/2 and PALB2-associated metastatic HER2-negative breast cancer (BC). (ASCO 2025) - P1/2 | "Clinical Trial Registration Number: NCT03604692 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

Combination therapy • Metastases • P1 data • Breast Cancer • HER2 Breast Cancer • HER2 Negative Breast Cancer • HER2 Positive Breast Cancer • Oncology • Solid Tumor • BRCA1 • BRCA2 • HER-2 • PALB2

THE EFFECTS OF PRIOR LINES OF THERAPY ON CLINICAL OUTCOMES FOR PATIENTS WITH CHRONIC GRAFT-VERSUS-HOST DISEASE RECEIVING AXATILIMAB: A POST HOC ANALYSIS OF AGAVE-201 (EBMT 2025) - P2 | "Patients across all doses and with 0.3 mg/kg had a median (range) of 4 (2-15) and 4 (2-12) prior LOT, respectively; 204 patients (84.6%) across all doses and 67 (83.8%) in the 0.3 mg/kg group had prior cGVHD therapy (ruxolitinib, belumosudil, or ibrutinib). In AGAVE-201, ORRs and response durability with axatilimab were consistent regardless of the number of prior LOT received. Patients who received axatilimab immediately after a regimen with ruxolitinib demonstrated rapid, durable clinical responses. Organ-specific responses to axatilimab were noted regardless of last prior therapy, and there is a trend toward higher response rates for those treated in their last LOT with ruxolitinib compared with belumosudil in some organs (joints/fascia, mouth, lungs, eyes, and skin)."

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

THE EFFECTS OF PRIOR LINES OF THERAPY ON CLINICAL OUTCOMES FOR PATIENTS WITH CHRONIC GRAFT-VERSUS-HOST DISEASE RECEIVING AXATILIMAB: A POST HOC ANALYSIS OF AGAVE-201 (EBMT 2025) - P2 | "Patients across all doses and with 0.3 mg/kg had a median (range) of 4 (2-15) and 4 (2-12) prior LOT, respectively; 204 patients (84.6%) across all doses and 67 (83.8%) in the 0.3 mg/kg group had prior cGVHD therapy (ruxolitinib, belumosudil, or ibrutinib). In AGAVE-201, ORRs and response durability with axatilimab were consistent regardless of the number of prior LOT received. Patients who received axatilimab immediately after a regimen with ruxolitinib demonstrated rapid, durable clinical responses. Organ-specific responses to axatilimab were noted regardless of last prior therapy, and there is a trend toward higher response rates for those treated in their last LOT with ruxolitinib compared with belumosudil in some organs (joints/fascia, mouth, lungs, eyes, and skin)."

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

REAL-WORLD TREATMENT PATTERNS OF PATIENTS WITH CHRONIC GRAFT-VERSUS-HOST DISEASE WHO RECEIVED 3 OR MORE LINES OF TREATMENT IN THE UNITED STATES (EBMT 2025) - "cGVHD treatment options have evolved, with multiple contemporary agents (ruxolitinib, belumosudil, ibrutinib, and axatilimab) approved by the US Food and Drug Administration...The most common 2L regimens were ruxolitinib (45.9%), rituximab (14.0%), methotrexate (13.8%), belumosudil (6.3%), and ibrutinib (4.4%)...The most common 3L regimens were ruxolitinib (19.4%), belumosudil (15.0%), and mycophenolate mofetil (12.7%)... Patients with cGVHD started 3L treatment within 1 year of cGVHD diagnosis; a significant proportion (41.3%) required additional treatment. Ruxolitinib was the most common agent used across treatment lines, suggesting its clinical benefit in early settings as well as for salvage therapy after failure of multiple lines of treatment. Combination treatment in later lines of cGVHD were common, suggesting the need to consider cGVHD mechanistic biology and tailoring therapies with nonoverlapping mechanisms of action when considering therapeutic strategy in..."

Clinical • HEOR • Real-world • Real-world evidence • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology

Cytokines in hematopoietic cell transplantation and related cellular therapies. (PubMed, Best Pract Res Clin Haematol) - "We focus on current and potential uses of cytokines to enhance engraftment and potentiate IEC therapies, as well as strategies to mitigate cytokine-mediated complications using cytokine blockers (e.g., tocilizumab, anakinra) and JAK inhibitors (e.g., ruxolitinib). We discuss new insights into GVHD physiology that have led to novel treatments, such as CSF1R blockade, which is effective in refractory chronic GVHD."

Journal • Review • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Inflammation • Transplantation • CSF1R

REAL-WORLD TREATMENT PATTERNS OF PATIENTS WITH CHRONIC GRAFT-VERSUS-HOST DISEASE WHO RECEIVED 3 OR MORE LINES OF TREATMENT IN THE UNITED STATES (EBMT 2025) - "cGVHD treatment options have evolved, with multiple contemporary agents (ruxolitinib, belumosudil, ibrutinib, and axatilimab) approved by the US Food and Drug Administration...The most common 2L regimens were ruxolitinib (45.9%), rituximab (14.0%), methotrexate (13.8%), belumosudil (6.3%), and ibrutinib (4.4%)...The most common 3L regimens were ruxolitinib (19.4%), belumosudil (15.0%), and mycophenolate mofetil (12.7%)... Patients with cGVHD started 3L treatment within 1 year of cGVHD diagnosis; a significant proportion (41.3%) required additional treatment. Ruxolitinib was the most common agent used across treatment lines, suggesting its clinical benefit in early settings as well as for salvage therapy after failure of multiple lines of treatment. Combination treatment in later lines of cGVHD were common, suggesting the need to consider cGVHD mechanistic biology and tailoring therapies with nonoverlapping mechanisms of action when considering therapeutic strategy in..."

Clinical • HEOR • Real-world • Real-world evidence • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology

PHENOTYPIC CHANGES IN SYSTEMIC IMMUNE CELLS IN RESPONSE TO AXATILIMAB IN HEALTHY MALE JAPANESE VOLUNTEERS (EBMT 2025) - "Following a single dose of axatilimab, changes in NCMs and CSF-1R ligands, IL-34 and CSF-1, were similar between healthy Japanese volunteers and non-Japanese volunteers, supporting a future clinical trial in Japanese patients with cGVHD. Changes in cell surface markers expressed on NCMs suggest that subpopulations of NCMs may represent newly generated monocyte progenitors and/or may represent existing NCMs with differential responses to axatilimab treatment. Further investigation regarding the effects of axatilimab on NCMs in patients with cGVHD is warranted."

Clinical • Immune cell • Chronic Graft versus Host Disease • Fibrosis • Graft versus Host Disease • Immunology • Inflammation • CSF1R • IL34 • ITGAM • PTPRC

PHENOTYPIC CHANGES IN SYSTEMIC IMMUNE CELLS IN RESPONSE TO AXATILIMAB IN HEALTHY MALE JAPANESE VOLUNTEERS (EBMT 2025) - "Following a single dose of axatilimab, changes in NCMs and CSF-1R ligands, IL-34 and CSF-1, were similar between healthy Japanese volunteers and non-Japanese volunteers, supporting a future clinical trial in Japanese patients with cGVHD. Changes in cell surface markers expressed on NCMs suggest that subpopulations of NCMs may represent newly generated monocyte progenitors and/or may represent existing NCMs with differential responses to axatilimab treatment. Further investigation regarding the effects of axatilimab on NCMs in patients with cGVHD is warranted."

Clinical • Immune cell • Chronic Graft versus Host Disease • Fibrosis • Graft versus Host Disease • Immunology • Inflammation • CSF1R • IL34 • ITGAM • PTPRC

DYNAMICS OF OVERALL AND ORGAN-SPECIFIC RESPONSES TO AXATILIMAB IN CHRONIC GRAFT-VERSUS-HOST DISEASE: ANALYSIS FROM THE AGAVE-201 STUDY (EBMT 2025) - P2 | "Most patients who achieved an overall clinical response or ≥7-point mLSS improvement within the first 6 cycles of AGAVE-201 did so by D84. By D56, more than half of responders had clinical responses and reported symptom improvement. Lower and upper gastrointestinal tracts, esophagus, liver, and joints/fascia were fastest to respond, whereas lung, mouth, eye, and skin responses were slower."

Chronic Graft versus Host Disease • Fibrosis • Gastrointestinal Disorder • Graft versus Host Disease • Immunology • Inflammation • CSF1R

DYNAMICS OF OVERALL AND ORGAN-SPECIFIC RESPONSES TO AXATILIMAB IN CHRONIC GRAFT-VERSUS-HOST DISEASE: ANALYSIS FROM THE AGAVE-201 STUDY (EBMT 2025) - P2 | "Most patients who achieved an overall clinical response or ≥7-point mLSS improvement within the first 6 cycles of AGAVE-201 did so by D84. By D56, more than half of responders had clinical responses and reported symptom improvement. Lower and upper gastrointestinal tracts, esophagus, liver, and joints/fascia were fastest to respond, whereas lung, mouth, eye, and skin responses were slower."

Chronic Graft versus Host Disease • Fibrosis • Gastrointestinal Disorder • Graft versus Host Disease • Immunology • Inflammation • CSF1R

CORRELATIONS OF CLINICIAN-REPORTED RESPONSES WITH OTHER RESPONSE MEASURES IN PATIENTS WITH CHRONIC GRAFT-VERSUS-HOST DISEASE: AN ANALYSIS FROM THE AGAVE-201 TRIAL (EBMT 2025) - P2 | "These results suggest there is limited agreement between NIH-ORR as a clinical trial endpoint and symptom-based clinical improvement or worsening perceived by clinicians and patients. These data provide insights into the clinical benefit achieved with axatilimab and suggest that additional exploration of secondary endpoints is warranted for cGVHD therapies. Clinical Trial Registry: NCT04710576"

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

CORRELATIONS OF CLINICIAN-REPORTED RESPONSES WITH OTHER RESPONSE MEASURES IN PATIENTS WITH CHRONIC GRAFT-VERSUS-HOST DISEASE: AN ANALYSIS FROM THE AGAVE-201 TRIAL (EBMT 2025) - P2 | "These results suggest there is limited agreement between NIH-ORR as a clinical trial endpoint and symptom-based clinical improvement or worsening perceived by clinicians and patients. These data provide insights into the clinical benefit achieved with axatilimab and suggest that additional exploration of secondary endpoints is warranted for cGVHD therapies. Clinical Trial Registry: NCT04710576"

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

AXATILIMAB ABROGATES INFLAMMATORY CYTOKINES AND CHEMOKINES AND INTERRUPTS THE DIFFERENTIATION OF MONOCYTES TO MACROPHAGES, A PATHOGENIC DRIVER OF INFLAMMATION AND FIBROSIS IN CGVHD (EBMT 2025) - P2 | "Together, these data suggest that blocking CSF-1/CSF-1R signaling by axatilimab reduces proinflammatory signaling in monocytes and monocyte-derived macrophages and specifically reduces the number of monocytes in the peripheral blood of human CSF-1R knock-in mice."

Chronic Graft versus Host Disease • Fibrosis • Graft versus Host Disease • Immunology • Inflammation • Oncology • CCL4 • CCL8 • CD14 • CXCL5 • CXCL6 • CXCL8 • ITGAM • PTPRC • TGFB1

Axatilimab After Second-Line Therapy Generates ORR Benefit in Steroid-Refractory cGVHD (OncLive) - P2 | N=241 | AGAVE-201 (NCT04710576) | Sponsor: Syndax Pharmaceuticals | "Treatment with axatilimab-csfr (Niktimvo) following second-line therapy yielded consistent overall response rates (ORRs) regardless of number and type of prior lines of therapy in patients with hematologic malignancies and steroid-refractory chronic graft-vs-host disease (cGVHD), according to data from a post hoc analysis of the phase 2 AGAVE-201 trial (NCT04710576) that were presented at the 51st Annual EBMT Meeting. Across all studied dose levels of axatilimab, the agent produced similar ORRs between patients who had received 2 prior lines of therapy (n = 35; 51.4%; 95% CI, 34.0%-6.6%), 3 prior lines of therapy (n = 49; 61.2%; 95% CI, 46.2%-74.8%), 4 prior lines of therapy (n = 49; 63.3%; 95% CI, 48.3%-76.6%), and 4 or more prior lines of therapy (n = 157; 66.9%; 95% CI, 58.9%-74.2%)."

P2 data • Chronic Graft versus Host Disease

Axatilimab in Patients With Lung Chronic Graft-versus-Host Disease and Related Bronchiolitis Obliterans Syndrome: Results From the AGAVE-201 Study (ATS 2025) - No abstract available

Clinical • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Pulmonary Disease • Respiratory Diseases

Axatilimab for Patients With Dermatologic Manifestations of Chronic Graft-Versus-Host Disease: A Post Hoc Analysis (AAD 2025) - P1/2, P2 | "These results support the clinical activity and symptom improvement with axatilimab in the skin, mouth, and joints/fascia among patients with long-standing cGVHD, including difficult-to-treat, severe sclerotic skin disease. These findings corroborate results from a prior phase 1/2 study (NCT03604692)."

Clinical • Retrospective data • Chronic Graft versus Host Disease • Dermatology • Graft versus Host Disease • Immunology • Pain • CSF1R

Incyte to Unveil New Data from its Dermatology Portfolio at 2025 American Academy of Dermatology (AAD) Annual Meeting (Businesswire) - "Featured abstracts for ruxolitinib cream (Opzelura) include multiple presentations in atopic dermatitis and late-breaking data in prurigo nodularis (PN); Pipeline data presented includes data for axatilimab (Niktimvo) in patients with dermatologic manifestations of chronic graft-versus-host disease (GVHD)."

Clinical data • Atopic Dermatitis • Chronic Graft versus Host Disease • Prurigo Nodularis

A Ph1b/2 Study of Axatilimab Monotherapy in Chinese Participants With Recurrent or Refractory cGVHD (clinicaltrials.gov) - P1/2 | N=30 | Not yet recruiting | Sponsor: Incyte Corporation

Monotherapy • New P1/2 trial • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology

Treatment Response in Individual Organs Affected by Chronic Graft-Versus-Host Disease. (PubMed, Cells) - "For cutaneous sclerosis, we observed large discrepancies in organ response rates according to the current NIH criteria and patient-reported improvement, highlighting the need for better measurement tools. High response rates for lung involvement with certain novel drugs deserve further investigation."

Journal • Review • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Scleroderma • Systemic Sclerosis • Transplantation

Axatilimab with or Without Azacitidine for the Treatment of Patients with Advanced Phase Myeloproliferative Neoplasms, Myeloproliferative Neoplasm/Myelodysplastic Syndrome Overlap or High Risk Chronic Myelomonocytic Leukemia (clinicaltrials.gov) - P1/2 | N=52 | Suspended | Sponsor: Uma Borate | Recruiting ➔ Suspended

Trial suspension • Atypical Chronic Myeloid Leukemia • Chronic Myeloid Leukemia • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology

Reinvigorating TNBC Response to Immunotherapy with Combination Myeloid Inhibition and Radiation (clinicaltrials.gov) - P2 | N=35 | Active, not recruiting | Sponsor: Stephen Shiao | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2025 ➔ Dec 2026 | Trial primary completion date: Dec 2025 ➔ Dec 2026

Enrollment closed • Trial completion date • Trial primary completion date • Breast Cancer • HER2 Breast Cancer • Hormone Receptor Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer • HER-2 • PD-L1

The Effects of Prior Lines of Therapy on Clinical Outcomes for Patients with Chronic Graft-Versus-Host Disease Receiving Axatilimab: A Post Hoc Analysis of Agave-201 (TCT-ASTCT-CIBMTR 2025) - P2 | "Overall response rate (ORR), time to first response (TTR), sustained response rate (SRR; proportion of patients with responses lasting ≥20 wk based on a landmark analysis of duration of response), and organ-specific responses were assessed post hoc by number of prior LOT and last therapy (ruxolitinib [RUX], belumosudil [BEL], and other therapies [all therapies other than RUX, including BEL]). Small patient numbers and mixed treatment regimens may limit interpretation. These data demonstrate the efficacy of AXA for steroid-refractory cGVHD after 2 LOT, including patients treated with RUX as last LOT."

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

Axatilimab Displays Durable Responses in cGVHD Irrespective of Number of Prior Lines of Treatment (OncLive) - P2 | N=241 | AGAVE-201 (NCT04710576) | Sponsor: Syndax Pharmaceuticals | "Treatment with...axatilimab (Niktimvo) was effective in terms of overall response rate (ORR) in patients with chronic graft-versus-host disease (cGVHD) regardless of the number of prior lines of therapy (LOT) received, according to findings from a post-hoc analysis of...AGAVE-201 trial (NCT04710576) presented during the 2025 Transplantation and Cellular Therapy Meetings....For all doses of axatilimab (n = 241), ORRs were similar for patients whose best response to their last prior treatment was a complete response (CR) or partial response (PR; n = 82), no change (n = 116), and disease progression (n = 16), at respective rates of 64.6% (95% CI, 53.3%-74.9%), 62.1% (95% CI, 52.6%-70.9%), and 62.5% (95% CI, 35.4%-84.8%)....Among patients who received 2 LOT (n = 35), the ORR was 51.4% (95% CI, 34.0%-68.6%); for 3 LOT (n = 49), the ORR was 61.2% (95% CI, 46.2%-74.8%), and for 4 LOT (n = 49), the ORR was..."

P2 data • Chronic Graft versus Host Disease

Incyte Reports 2024 Fourth Quarter and Year-End Financial Results, Provides 2025 Financial Guidance and Highlights 2025 R&D Milestones (Businesswire) - "Opzelura (ruxolitinib) cream net revenues of $162 million (+48% Y/Y) in the fourth quarter 2024 and $508 million (+50% Y/Y) for the full year 2024; Opzelura net revenues guidance range of $630 - $670 million for the full year 2025...Incyte expects to deliver...key milestones in 2025. These include...new product launches: Niktimvo in 3L+ chronic graft-versus-host disease (GVHD), ruxolitinib cream in pediatric atopic dermatitis (AD)...Two Phase 3 trials (TRuE-PN1 and TRuE-PN2) evaluating ruxolitinib cream in prurigo nodularis (PN) are fully enrolled. Data from the Phase 3 studies are anticipated in the first half of 2025. The Phase 3 trial for ruxolitinib cream in mild to moderate hidradenitis suppurativa (HS) is on track to initiate in the first half of 2025. following achieving alignment on the study design with FDA."

Commercial • Launch US • New P3 trial • P3 data • Atopic Dermatitis • Graft versus Host Disease • Hidradenitis Suppurativa • Prurigo Nodularis

Axatilimab, Ruxolitinib, and Belumosudil Combo Elicits Responses in Chronic GVHD (OncLive) - "A treatment regimen of axatilimab-csfr (Niktimvo) in combination with ruxolitinib (Jakafi) with or without belumosudil (Rezurock) led to responses in heavily pretreated patients with chronic graft-vs-host disease (cGVHD), according to data from a retrospective study presented at the 2025 Transplantation and Cellular Therapy Meetings...In the single-center, retrospective study of 8 heavily pretreated patients, the treatment led to an overall response rate (ORR) of 25% by National Institutes of Health (NIH) Consensus Criteria and 75% by Clinically Significant Symptomatic Improvement (CSSI) criteria, demonstrating the clinical benefit of combination therapy in heavily pretreated patients with cGVHD....There were no patients who discontinued axatilimab because of intolerance and no fatal AEs reported."

Retrospective data • Chronic Graft versus Host Disease