Jakafi (ruxolitinib) / Novartis, Incyte - LARVOL DELTA

Pacritinib is a potent ACVR1 inhibitor with significant anemia benefit in patients with myelofibrosis. (PubMed, Blood Adv) - "Pacritinib inhibited ACVR1 with greater potency (IC50 = 16.7 nM; Cmax:IC50 = 12.7) than momelotinib (IC50 = 52.5 nM; Cmax:IC50 = 3.2), fedratinib (IC50 = 273 nM; Cmax:IC50 = 1.0), or ruxolitinib (IC50 >1000; Cmax:IC50 <0.01). Among patients on PERSIST-2 who were not TI at baseline based on Gale criteria, a significantly greater proportion became TI on pacritinib compared to best available therapy (37% vs. 7%, P=0.001), and significantly more had a ≥50% reduction in transfusion burden (49% vs. 9%, P<0.0001). These data indicate that the anemia benefit of the JAK2/IRAK1 inhibitor pacritinib may be a function of potent ACVR1 inhibition."

Journal • Anemia • Hematological Disorders • Myelofibrosis • ACVR1 • IRAK1

Pelabresib plus ruxolitinib for JAK inhibitor-naive myelofibrosis: a randomized phase 3 trial. (PubMed, Nat Med) - P3 | "Pelabresib in combination with ruxolitinib is well tolerated, improves signs of underlying myelofibrosis pathobiology and provides substantial clinical benefit over standard-of-care JAK inhibitor monotherapy. ClinicalTrials.gov identifier: NCT04603495 ."

Journal • P3 data • Myelofibrosis

PELABRESIB IN COMBINATION WITH RUXOLITINIB FOR JANUS KINASE INHIBITOR-NAIVE PATIENTS WITH MYELOFIBROSIS: 72-WEEK FOLLOW-UP WITH LONG-TERM EFFICACY OUTCOMES OF THE PHASE III MANIFEST-2 STUDY (EHA 2025) - P3 | "PELA+RUX showed sustained improvements over 72 weeks in splenic response, symptoms, SVR35/TSS50 dual response, BMF, and anemia vs PBO+RUX. The safety profile for Grade ≥3 TEAEs was similar and consistent across treatment arms, with the imbalance in leukemic transformation cases decreasing over time. Survival outcomes show a trend in favor of the PELA+RUX arm."

Clinical • Combination therapy • P3 data • Anemia • Fibrosis • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Myelofibrosis • Oncology • Thrombocytopenia

A Prognostic Model to Predict Survival After 6 Months of Ruxolitinib in Patients with Myelofibrosis. (PubMed, Blood Adv) - P | "The RR6 model was validated and confirmed in an external cohort comprised of 40 MF patients. In conclusion, the RR6 prognostic model allows for the early identification of RUX-treated MF patients with impaired survival who might benefit from a prompt treatment shift."

Journal • Myelofibrosis

INCA33989 is a novel, first in class, mutant calreticulin-specific monoclonal antibody that demonstrates safety and efficacy in patients with essential thrombocythemia (ET) (EHA 2025) - P1 | "INCA33989-101 (NCT05936359) and -102 (NCT06034002) are phase 1, first-in-human, multicenter, open-label studies evaluating INCA33989 in patients with ET or myelofibrosis (monotherapy or in combination with ruxolitinib). INCA33989 monotherapy in ET patients who were resistant/intolerant to prior therapy was well-tolerated with no DLTs, and 98% of patients remain on treatment. Rapid and durable normalization of blood counts and reduction of mutCALR VAF was observed in most patients, and the potential for disease modification was evidenced by 25% of patients achieving an early PMR."

Clinical • Late-breaking abstract • Anemia • Cardiovascular • Essential Thrombocythemia • Fatigue • Hematological Disorders • Infectious Disease • Myelofibrosis • Myeloproliferative Neoplasm • Neutropenia • Pancreatitis • Respiratory Diseases • Thrombocytopenia • Thrombocytosis • Thrombosis • CALR

Ruxolitinib versus allogeneic stem cell transplantation for patients with myelofibrosis according to donor availability: A prospective multicenter phase II trial (ASH 2025) - P2 | "The RuxoAllo prospective study demonstrates that SCT following ruxolitinib induction significantlyimproves EFS and disease control compared to continued ruxolitinib in patients with myelofibrosis,particularly in those with intermediate- or high-risk disease. Despite a relatively low non-relapse mortalityof 7%, SCT remains associated with substantial risks, including graft-versus-host disease and othertoxicities, underscoring the need for careful patient selection. Ruxolitinib remains valuable for symptommanagement and can serve as an effective bridge to transplant."

Clinical • P2 data • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Eosinophilic Leukemia • Chronic Graft versus Host Disease • Fibrosis • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Myelofibrosis • Myeloproliferative Neoplasm • Thrombocytopenia • Transplantation

IMproveMF update: Phase 1/1B trial of imetelstat (IME)+ruxolitinib (RUX) in patients (pts) with intermediate (INT)-1, INT-2, or high-risk (HR) myelofibrosis (MF). (ASCO 2025) - P1, P2 | "In part 1 of IMproveMF, no DLTs were observed and the RP2D dose of 9.4 mg/kg IME was determined. AEs were consistent with those observed in other IME clinical trials, and preliminary efficacy was positive, demonstrating the potential of IME+RUX in this pt population with high unmet needs. Part 2 of this trial is ongoing across the US at 6 sites."

Clinical • P1 data • Anemia • Fatigue • Hematological Disorders • Infectious Disease • Leukopenia • Myelofibrosis • Neutropenia • Pain • Pneumonia • Respiratory Diseases

Long-term outcomes of vedolizumab targeted therapy in patients with very early onset Inflammatory Bowel Disease (ECCO-IBD 2026) - "63% of patients were biologic-naive, and VEDO was used as dual therapy in 16 patients, including canakinumab (n=3), tacrolimus (n=2), ruxolitinib (n=1), methotrexate (n=4), adalimumab (n=1), sirolimus (n=3), ustekinumab (n=1)...Three patients discontinued VEDO after 6 months due to worsening symptoms, transitioning to ustekinumab (n=1) and infliximab (n=2)...Conclusion In our experience, vedolizumab showed 12 months therapy durability in 95% of the patients with VEO-IBD in this cohort. Quiescent disease and clinical remission were achieved in > 70% of patients with few adverse events."

Clinical • Crohn's disease • Immunology • Inflammation • Inflammatory Bowel Disease • Ulcerative Colitis

BALDER: Trial of Efficacy and Safety of MC0518 Versus Best Available Therapy in Participants With Steroid-Refractory Acute Graft Versus Host Disease (clinicaltrials.gov) - P2 | N=48 | Active, not recruiting | Sponsor: medac GmbH | Recruiting ➔ Active, not recruiting | Trial primary completion date: Aug 2026 ➔ Mar 2026

Enrollment closed • Trial primary completion date • Acute Graft versus Host Disease • Graft versus Host Disease • Immunology • Pediatrics • Transplantation

Immune-Effector Cell-Associated Enteritis/Colitis (IEC-EC) after Ciltacabtagene Autoleucel (cilta-cel) in Multiple Myeloma (MM): Updated Clinicopathologic Data and Management (TCT-ASTCT-CIBMTR 2026) - "Treatments for mild cases included supportive care, mesalamine, and corticosteroids; treatments for severe cases included infliximab, vedolizumab, cyclosporine, high-dose cyclophosphamide, and ruxolitinib. IEC-EC is a heterogenous condition with a wide variety of clinical presentations. Severe cases are associated with a high risk of infection and mortality; however, we report for the first time that milder cases responsive to brief corticosteroid courses may also occur. Optimal diagnostic strategies include prompt endoscopic evaluation evaluating both small and large bowel, targeted biopsies regardless of endoscopic appearance, and rule-out of infectious or lymphoproliferative processes."

Clinical • Gastroenterology • Gastrointestinal Disorder • Hematological Malignancies • Immunology • Infectious Disease • Multiple Myeloma • CD4 • CD8

Utilization of a Microdevice for Psoriasis and Atopic Dermatitis (clinicaltrials.gov) - P4 | N=10 | Not yet recruiting | Sponsor: University of California, San Francisco

New P4 trial • Atopic Dermatitis • Dermatitis • Dermatology • Immunology • Psoriasis

Multiarm multistage randomised controlled trial of inflammatory signal inhibitors (MATIS) for patients hospitalised with COVID-19 pneumonia during the UK pandemic. (PubMed, BMJ Open) - P1/2 | "We found no evidence that FOS was superior to SOC for the treatment of COVID-19 pneumonia in patients requiring hospital admission. Due to early stopping, the trial was underpowered to establish RUX's effect in this population. Further study is needed."

Clinical • Journal • Cardiovascular • Infectious Disease • Novel Coronavirus Disease • Pneumonia • Respiratory Diseases • Venous Thromboembolism

Exploring new directions: Remestemcel-L-rknd added to ruxolitinib as initial treatment for steroid refractory acute GVHD (CTN2303): A phase III placebo-controlled study developed by Mesoblast and the BMT Clinical Trials Network. (TCT-ASTCT-CIBMTR 2026) - No abstract available

Clinical • P3 data • Acute Graft versus Host Disease • Graft versus Host Disease • Immunology

Case Report: A rare case of synchronous ovarian mixed germ cell tumor and mast cell leukemia in a pediatric patient. (PubMed, Front Oncol) - "Targeted therapy with avapritinib and ruxolitinib was initiated but yielded limited response. Given the consistent co-occurrence of KIT mutations in previously reported similar cases, we propose the recognition of a distinct disease entity: ovarian germ cell tumor/mastocytosis with KIT mutations. This report emphasizes the importance of early genetic profiling and multidisciplinary collaboration in diagnosing and managing rare, genetically unified malignancies in pediatric oncology."

Journal • Cognitive Disorders • Germ Cell Tumors • Hematological Disorders • Hematological Malignancies • Leukemia • Mast Cell Leukemia • Oncology • Ovarian Cancer • Pediatrics • Solid Tumor • Thrombocytopenia • AFP • KIT • NRAS • TP53

Remestemcel-L-Rknd (Ryoncil) Improves Survival after Failure of Second-Line Treatment for SR-aGVHD (TCT-ASTCT-CIBMTR 2026) - P3 | "Amongst heavily pretreated patients with third line or greater SR-GVHD, 89 % with grades III/IV disease, remestemcel-L-rknd had significant activity with overall day 100 OS of 68%, and similar survival outcomes across both pediatric and adult age groups. This was significantly higher than has been reported with other treatments used in third-line patients and very similar to OS seen with remestemcel- L-rknd in second line children with SR-aGVHD (Figure 1B). Given the high early mortality risk of SR-aGVHD after failure of second line agents, these results highlight that earlier use of remestemcel-L-rknd may increase OS."

Clinical • Acute Graft versus Host Disease • Graft versus Host Disease

Advances in Janus Kinase Inhibitors for Vitiligo Treatment. (PubMed, Drug Res (Stuttg)) - "The first generation Janus kinase inhibitors, i.e., tofacitinib and ruxolitinib, inhibit a variety of Janus kinases, whereas the new generation Janus kinase inhibitors, such as ritlecitinib and upadacitinib, exhibit inhibitory effects only on specific Janus kinases; they are therefore selective as well as safer and more effective. In this review, we aim to provide an up-to-date view of vitiligo pathogenesis at the cellular, molecular, and genetic levels and further to elucidate the relationship between Janus kinase/signal transducer and activator of transcription signaling pathway components and vitiligo. Finally, we summarize currently market-approved and preclinical Janus kinase inhibitors, highlighting the latest advances in their clinical applications."

Journal • Dermatology • Immunology • Vitiligo

Safety Analysis of Axatilimab in Patients with Chronic Graft-Versus-Host Disease in an Expanded Access Program (TCT-ASTCT-CIBMTR 2026) - P | "Introduction: Axatilimab (AXA) is a monoclonal antibody targeting colony-stimulating factor 1 receptor (CSF-1R), which depletes CSF-1R–dependent monocytes and macrophages that drive inflammation and fibrosis in chronic graft-versus-host disease (cGVHD). Of the 112 enrolled patients, 104 received AXA ( Table ). The most common prior systemic cGVHD treatments were belumosudil (80.8%), ruxolitinib (76.0%), and prednisone (74.0%). The most common concomitant cGVHD medications were belumosudil (65.4%), prednisone (60.6%), and ruxolitinib (51.0%)."

Clinical • Cardiovascular • Chronic Graft versus Host Disease • Cough • Fibrosis • Graft versus Host Disease • Heart Failure • Immunology • Infectious Disease • Pneumonia • Respiratory Diseases • Septic Shock • CSF1R

Optimal Treatment of Steroid-refractory Chronic Graft-versus-host Disease (cGvHD) in the Era of Novel drugs - a Systematic Review and Meta-analysis. (PubMed, Stem Cell Rev Rep) - "The analyzed treatment options included: axatilimab, belumosudil, extracorporeal photopheresis (ECP), ibrutinib, imatinib, rovadicitinib, and ruxolitinib. The analysis of imatinib yielded inconsistent results. As cGvHD is a disease with a heterogeneous clinical image, clinical experience remains an important factor that affects treatment choice for patients with certain disease manifestations."

Journal • Retrospective data • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology

A Comprehensive Analysis of Axatilimab in Patients with Chronic Graft-Versus-Host Disease and Related Bronchiolitis Obliterans Syndrome: Integrated Analysis from 2 Clinical Studies (TCT-ASTCT-CIBMTR 2026) - P1/2, P2 | "Results : : 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; Table 1 ). 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. To evaluate the efficacy of axatilimab in patients with chronic graft-versus-host disease–related bronchiolitis obliterans syndrome (BOS) To assess clinical responses in patients with severe BOS and symptom responses in nonclinical responders when treated with axatilimab To assess clinical predictors of lung responses in patients treated with 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: Interim Safety Analysis of a Randomized, Phase 2 study (TCT-ASTCT-CIBMTR 2026) - P2 | "As of the interim analysis, 44 patients have enrolled (AXA+RUX, n=15; RUX, n=15; corticosteroids, n=14), and 43 patients have received study treatment ( Table ). Eight patients discontinued treatment (AXA+RUX, n=1 [relapse or progression of underlying hematologic disease, considered unrelated to AXA+RUX]; RUX, n=1 [death due to bronchospastic crisis, considered unrelated to RUX]; corticosteroids, n=6 [received a new systemic therapy due to insufficient response to treatment, n=5; patient withdrawal, n=1]). Among all treated patients, 32 experienced TEAEs (AXA+RUX, n=11 [73.3%]; RUX, n=12 [80.0%]; corticosteroids, n=9 [69.2%])."

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

A Phase I/II Trial of Ruxolitinib with Chemotherapy for Patients with Relapsed and/or Refractory Philadelphia-like Acute Lymphoblastic Leukemia. (PubMed, Clin Lymphoma Myeloma Leuk) - P2 | "Continued efforts should focus on identifying optimal treatment strategies for this high-risk group of patients."

Journal • P1/2 data • Acute Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • ABL1 • CRLF2 • EPOR • HMBOX1 • JAK2 • NUP214

Ruxolitinib Therapy in Pansclerotic Morphea refractory to Autologous Stem-Cell Transplantation (SSWC 2026) - No abstract available

Transplantation

POSTER OF DISTINCTION: A HUMAN IPSC-DERIVED SCREENING MODEL FOR CROHN'S DISEASE DEMONSTRATES THE POTENTIAL ROLE OF JAK INHIBITORS IN MITIGATING INTESTINAL FIBROSIS (CCCongress 2026) - " Our fibrosis model of iPSC-MES found a robust antifibrotic effect from the JAK1/2 inhibitor ruxolitinib (RX z =-4.91), in all 6 iPSC-MES lines...We then evaluated other JAK inhibitors currently prescribed for the treatment of IBD: filgotinib (FG), tofacitinib (TF) and upadacitinib (UPA)... We demonstrate the feasibility of utilizing iPSC-MES cells for HTS to identify potential antifibrotic therapies. This approach overcomes the limitations of biopsy-derived myofibroblasts, illustrates the potential utility of JAK inhibitors and thus opens up a new more feasible approach for studying precision-based treatments."

Crohn's disease • Fibrosis • Gastroenterology • Immunology • Inflammatory Bowel Disease • TGFB1 • TNFA

Preserving thrombosis and life years in polycythemia vera: start by reading the biology of the disease. (PubMed, Haematologica) - "Interferon, particularly ropeginterferon alfa-2b, and ruxolitinib reduces JAK2V617F allele burden, systemic inflammation, as reflected by the neutrophil-to-lymphocyte ratio (NLR), and thrombosis rates, demonstrating long-term disease-modifying potential. Biological markers such as persistent leukocytosis, elevated NLR, rising JAK2V617F variant allele frequency, or high phlebotomy burden can guide treatment decisions more precisely than age alone. Tailoring therapy in younger PV patients according to disease biology and individual tolerance may prevent irreversible complications, improve quality of life, and ultimately reduce the years of life lost."

JAK2V617F • Journal • Cardiovascular • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombosis

Differential effects of GVHD therapies on intestinal epithelium. (PubMed, Blood Adv) - "Ruxolitinib prevented inflammatory apoptosis in both human and murine organoids and preserved ISC function and proliferation, while corticosteroids offered no protection and in fact suppressed proliferation. This study highlights the importance of GVHD therapies that facilitate epithelial repair and regeneration, protect target tissues and suppress alloreactivity of donor T cells."

Journal • Bone Marrow Transplantation • Gastrointestinal Disorder • Graft versus Host Disease • Immunology • Transplantation