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

A Decade of Progress Towards Safer and More Broadly Applicable Allogeneic Stem Cell Transplantation (ASH 2026) - "Hany Elmariah (Stanford) presenting "New Standards in GVHD Prophylaxis: Towards a Future Without GVHD," which examines the latest advances in prophylactic strategies, including Orca-T, abatacept, and post-transplant cyclophosphamide (PTCy), and how these approaches are reshaping the standard of care...These include ruxolitinib and mesenchymal stromal cells (MSCs) for acute GVHD (with pediatric indications), as well as belumosudil, ruxolitinib, and axatilimab for chronic GVHD, and how they are improving outcomes for patients with steroid-refractory disease...Holtan's talk, "A Donor for All: Increasing Access and Improving Outcomes," which addresses expanding donor options through mismatched unrelated donor (MMURD), haploidentical, and cord blood transplantation, emphasizing the data supporting broader access and equitable outcomes across donor types. These presentations chart a bold trajectory for the field, illuminating how scientific innovation,..."

Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Transplantation

Indications for Autologous and Allogeneic Hematopoietic Stem-Cell Transplantation in Adults: State of the Art. (PubMed, J Clin Med) - "In parallel, post-transplant cyclophosphamide (PTCy) has largely equalized outcomes across matched sibling, matched unrelated, and mismatched alternative donors, and novel agents (ruxolitinib, belumosudil, axatilimab) have materially reduced graft-versus-host disease (GVHD) morbidity. This narrative review synthesizes current indications for autologous (auto-HSCT) and allogeneic (allo-HSCT) transplantations in adults, and flags areas of persistent controversy where randomized data are still maturing. Across all indications, the clinician's question is shifting from "transplant or not?" towards "which donor, which conditioning, which bridge, and which post-transplant maintenance?", each tailored to disease biology, MRD trajectory, and patient fitness."

IO biomarker • Journal • Review • Acute Myelogenous Leukemia • Aplastic Anemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Lymphoma • Multiple Myeloma • Myelodysplastic Syndrome • Myelofibrosis • Oncology • Transplantation

AXALAP: Phase Ib Study of Axatilimab in Combination With Olaparib in BRCA1/2 and PALB2- Associated Metastatic HER2-negative Breast Cancer (clinicaltrials.gov) - P1 | N=20 | Recruiting | Sponsor: Dana-Farber Cancer Institute | Trial primary completion date: Sep 2026 ➔ May 2027

Trial primary completion date • Breast Cancer • HER2 Breast Cancer • HER2 Negative Breast Cancer • Oncology • Solid Tumor • BRCA1 • BRCA2 • HER-2 • PGR

A Review of Modern Therapies and New Innovations in the Treatment of Cutaneous Graft-versus-Host Disease. (PubMed, Dermatol Ther (Heidelb)) - "Recent advances in GVHD management have led to the introduction of targeted systemic agents, such as ruxolitinib, belumosudil, axatilimab, ibrutinib, and abatacept, each addressing different pathways implicated in disease pathogenesis and leading to incorporation into disease management based on established algorithms or select clinical contexts. Additionally, local immunomodulatory therapies, such as topical ruxolitinib, phototherapies, and extracorporeal photopheresis, can be utilized as adjunctive therapies for persistent or localized cutaneous disease. Cellular therapies, specifically mesenchymal stromal cells and regulatory T cells (Treg), are emerging therapies with promising potential in modulating the immune response and improving clinical outcomes. This review summarizes the current standards of care and recent therapeutic advances in the management of cutaneous GVHD, focusing on evidence-based therapies and skin-specific outcomes."

Journal • Review • Bone Marrow Transplantation • Graft versus Host Disease • Immunology • Transplantation

Non-CE Satellite Symposium: Continuity Through Connected Care: Hear About the Impact of Niktimvo (axatilimab-csfr) From a Patient and Caregiver Incyte Corporation (JADPRO 2026) - "Sponsored by Caregiver Incyte Corporation Katie Sellers, PA, featuring recordings by patient and caregiver Don and Angie Martin"

Clinical

ABRAXAS: CSF-1R Inhibitor, Axatilimab, for Prevention of Acute and Chronic GVHD After HLA-Matched Related and Unrelated Donor Allogeneic Stem Cell Transplantation (clinicaltrials.gov) - P2 | N=72 | Not yet recruiting | Sponsor: Medical College of Wisconsin

New P2 trial • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Transplantation

Axatilimab Plus Standard of Care Therapy for the Prevention of Graft Versus Host Disease Following Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Hematologic Cancer, ABRAXAS Trial (clinicaltrials.gov) - P2 | N=0 | Withdrawn | Sponsor: Mayo Clinic | N=72 ➔ 0 | Not yet recruiting ➔ Withdrawn

Enrollment change • Trial withdrawal • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Oncology • Transplantation

Axatilimab for Chronic Graft-Versus-Host Disease After Failure of at Least Two Prior Systemic Therapies: Results of a Phase I/II Study. (PubMed, J Clin Oncol) - P1/2 | "Targeting profibrotic macrophages with axatilimab is a therapeutically promising novel strategy with a favorable safety profile for refractory cGVHD."

Journal • P1/2 data • Chronic Graft versus Host Disease • Cytomegalovirus Infection • Fibrosis • Graft versus Host Disease • Immunology • Transplantation

A Ph1b/2 Study of Axatilimab Monotherapy in Chinese Participants With Recurrent or Refractory cGVHD (clinicaltrials.gov) - P1/2 | N=32 | Active, not recruiting | Sponsor: Incyte Corporation | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2027 ➔ Jul 2028 | Trial primary completion date: Jul 2026 ➔ Jan 2027

Enrollment closed • Monotherapy • Trial completion date • Trial primary completion date • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • CSF1

Management of chronic pulmonary GVH in 2025, indication for lung transplantation (SFGM-TC) (PubMed, Bull Cancer) - "New treatments have been developed for GvHD: Janus kinase inhibitors (ruxolitinib), rhokinase inhibitors (belumosudil) and anti-CSF1R antibody (axatilimab). Allogeneic transplant recipients represent a small proportion of lung transplant patients, even though the results are the same as for other indications. We reviewed the conditions under which a patient with pulmonary GVHD should be referred to a center with expertise in lung transplantation."

Journal • Review • Fibrosis • Graft versus Host Disease • Immunology • Interstitial Lung Disease • Pulmonary Disease • Respiratory Diseases • Transplantation

Two trials evaluating axatilimab in combination with standard of care therapies in newly diagnosed chronic graft-versus-host disease (cGVHD) patients are ongoing, including (Syndax Press Release) - "A Phase 2, open-label, randomized, multicenter trial of axatilimab in combination with ruxolitinib in patients ≥ 12 years of age with newly diagnosed cGVHD. Topline data are anticipated in the fourth quarter of 2026; A pivotal Phase 3, randomized, double-blind, placebo-controlled, multicenter trial of axatilimab in combination with corticosteroids in patients ≥ 12 years of age with newly diagnosed cGVHD. Topline data are anticipated in early 2028."

P2 data • P3 data: top line • Chronic Graft versus Host Disease

The Company anticipates topline data from MAXPIRe, a Phase 2, 26-week randomized, double-blinded, placebo-controlled trial of axatilimab on top of standard of care in patients with idiopathic pulmonary fibrosis in the fourth quarter of 2026. (Syndax Press Release)

P2 data • Idiopathic Pulmonary Fibrosis • Immunology

Achieved $60.3 million in Niktimvo net revenue in the second quarter of 2026, a 67% increase over the second quarter of 2025 and a 9% increase over the first quarter of 2026. (Syndax Press Release) - "Syndax and Incyte are co-commercializing Niktimvo. Syndax records 50% of the Niktimvo net commercial profit, defined as net product revenue minus the cost of sales and commercial expenses. Syndax’s share of the Niktimvo product contribution, reported as collaboration revenue, was $18.1 million in the second quarter of 2026."

Sales • Chronic Graft versus Host Disease

Doris Ponce, MD, discusses bone health findings with axatilimab in chronic GVHD (YouTube) - "In this SOHO Insider interview from EHA 2026, Doris Ponce, MD...discusses a secondary analysis of the phase 2 AGAVE-201 trial evaluating axatilimab in patients with chronic graft-versus-host disease. Dr. Ponce reviews bone health outcomes associated with axatilimab, a high-affinity anti-CSF-1R monoclonal antibody, including changes in bone turnover markers, treatment-emergent bone-related adverse events, and fracture rates in a population in which many patients were receiving concomitant corticosteroids."

Interview • Video

Chronic graft-versus-host disease. (PubMed, Nat Rev Dis Primers) - "Four second-line treatments have been approved by the FDA - ibrutinib, ruxolitinib, belumosudil and axatilimab - targeting B cell signalling, JAK-STAT, ROCK2 and CSF1R pathways, respectively, and emerging therapies such as rovadicitinib show promise. Multidisciplinary care, infection control and psychosocial support are critical to improving quality of life in patients with cGVHD. Future research should prioritize mechanistic insights, antifibrotic therapies and integrating organ-specific interventions to enhance outcomes in patients with cGVHD."

Journal • Review • Chronic Graft versus Host Disease • Fibrosis • Graft versus Host Disease • Immunology • Infectious Disease • Transplantation • CSF1R

Chronic graft-versus-host disease: Update on pathobiology, biomarkers, and evolving treatment algorithms. (PubMed, Cell Transplant) - "Recognizing that these phases are conceptual and frequently overlap in clinical practice, we explore the alignment of contemporary biomarkers with each phase-ST2/CXCL9 (Phase 1), BAFF/autoantibodies (Phase 2), and MMP3/TGF-β (Phase 3)-and discuss how FDA-approved agents (ibrutinib, ruxolitinib, belumosudil, axatilimab) may target phase-specific pathways. A conceptual, risk-stratified treatment algorithm is proposed to link pathobiology to clinical decision-making, with the important caveat that biomarker-guided selection remains investigational and currently complements, rather than replaces, comprehensive clinical assessment. Notably, the adoption of prophylaxis and upfront strategies to prevent cGVHD has substantially reduced cGVHD incidence in modern cohorts."

Biomarker • Journal • Review • Chronic Graft versus Host Disease • Fibrosis • Graft versus Host Disease • Immunology • Inflammation • Transplantation • CXCL9 • MMP3 • TGFB1

Topline data from the Phase 2 trial evaluating axatilimab in combination with ruxolitinib in patients with newly diagnosed chronic GVHD are anticipated in the second half of 2026. (Businesswire)

P2 data • Chronic Graft versus Host Disease

Hematology and Oncology portfolio net sales of $222 million, an increase of 69%, compared to the second quarter of 2025. (Businesswire) - "The increase was driven by increased demand for Niktimvo, Monjuvi/Minjuvi and Zynyz."

Commercial • Anal Squamous Cell Carcinoma • Chronic Graft versus Host Disease • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Marginal Zone Lymphoma • Merkel Cell Carcinoma

Effects of Axatilimab on Skin in Chronic Graft-versus-Host Disease (clinicaltrials.gov) - P=N/A | N=15 | Not yet recruiting | Sponsor: Northwestern University

New trial • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • CSF1R

Highlighted R&D Day Topics (Syndax Press Release) - "During the R&D event, the Company will highlight its late-stage...axatilimab programs in newly diagnosed acute leukemia and chronic GVHD, respectively, as well as the Phase 2 trial of axatilimab in IPF that is expected to readout in the fourth quarter of 2026. During the R&D event, Dr. Toby M. Maher will discuss the unmet patient needs, evolving treatment landscape, and the potential for axatilimab’s mechanism in IPF."

Clinical • P2 data • Chronic Graft versus Host Disease • Idiopathic Pulmonary Fibrosis • Leukemia

Recent Business Highlights and Anticipated Milestones:…Niktimvo (axatilimab-csfr) (GlobeNewswire) - "Niktimvo net revenue for the full year 2025 totaled $151.6 million....Syndax’s share of the Niktimvo product contribution, reported as collaboration revenue, was $19.4 million and $42.4 million in the fourth quarter and full year 2025, respectively....Completed enrollment in MAXPIRe, a Phase 2, 26-week randomized, double-blinded, placebo-controlled trial of axatilimab on top of standard of care in patients with idiopathic pulmonary fibrosis (IPF) in the first quarter of 2026. The Company expects to report topline data in the fourth quarter of 2026."

Enrollment closed • P2 data • Sales • Chronic Graft versus Host Disease • Idiopathic Pulmonary Fibrosis

AXATILIMAB IN COMBINATION WITH RUXOLITINIB IN PATIENTS WITH NEWLY DIAGNOSED CHRONIC GRAFT-VERSUS-HOST DISEASE: UPDATED SAFETY ANALYSIS OF A RANDOMIZED PHASE 2 STUDY (EHA 2026) - P2 | "Anemia was the only grade ≥3 TEAE that occurred in 2 patients (n=1 each in AXA+RUX and RUX). Summary/Conclusion 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

AGAVE-201: A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD) (clinicaltrials.gov) - P2 | N=296 | Recruiting | Sponsor: Syndax Pharmaceuticals | Active, not recruiting ➔ Recruiting

Enrollment open • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • CSF1

ASSESSMENTS OF BONE HEALTH AMONG PATIENTS WITH CHRONIC GRAFT-VERSUS-HOST DISEASE RECEIVING AXATILIMAB IN THE AGAVE-201 TRIAL (EHA 2026) - P2 | "Bone-related TEAEs were rare, with few patients having multiple fractures; this is notable since 65.3% of patients received concomitant corticosteroids. These results may indicate that AXA had minimal effects on bone metabolic markers, although additional analyses 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 • CSF1R

Adult Cutaneous Chronic Graft-Versus-Host Disease: An In-Depth Review. (PubMed, J Cutan Med Surg) - "Recently, therapeutic agents such as ibrutinib, ruxolitinib, belumosudil, and axatilimab, which modulate distinct immune and fibrotic pathways, improved management of this condition...Advances in scoring systems, imaging, and patient-reported outcomes are improving disease assessment. Given its heterogeneity, individualized, mechanism-based treatments are needed to improve long-term outcomes."

Journal • Review • Chronic Graft versus Host Disease • Dermatology • Fibrosis • Graft versus Host Disease • Immunology • Pain • Transplantation