Ruxolitinib

Current understanding

JAK1/2 inhibitor approved for myelofibrosis (COMFORT-I/II) and PV (RESPONSE). Reduces splenomegaly and constitutional symptoms; modest impact on allele burden.

Evidence log

• 2026-10-04 — Navtemadlin Shows Biomarker Response in Patients with R/R Myelofibrosis: Phase 3 POIESIS (trial-late): ruxolitinib serving as backbone with navtemadlin (MDM2 inhibitor) add-on in JAK inhibitor-naïve MF patients with suboptimal Jakafi response — adds an MDM2/p53-axis mechanism to the ruxolitinib add-on portfolio and formalizes ‘suboptimal responder’ as a distinct enrollment phenotype. (novelty: 3)
• 2026-09-30 — Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of Mutant Calreticulin Targeted Antibodies in CALR-mutant Myeloproliferative Neoplasms: Phase 1/1b (trial-early): ruxolitinib serving as combination backbone with DMR-001 (first-in-class mutant CALR-targeted monoclonal antibody) in CALR-mutant MF — adds a second CALR-neoantigen-directed antibody partner (alongside INCA033989 and JNJ-88549968) to the ruxolitinib add-on portfolio. (novelty: 4)
• February 3 — Prelude Therapeutics Receives FDA Clearance of Investigational New Drug Application for PRT12396: cross-connection with prt12396-mutant-selective-jak2-inhibitor: PRT12396’s mutant-selective JH2-pocket mechanism is designed to spare wild-type JAK2, potentially avoiding the on-target cytopenias seen with pan-JAK inhibitors like ruxolitinib. (novelty: 3)
• 2026-09-16 — RVU120 in Patients With Intermediate or High-risk, Primary or Secondary Myelofibrosis: Trial-early: ruxolitinib serving as combination backbone with RVU120 (investigational agent) in intermediate/high-risk primary or secondary myelofibrosis — adds another candidate to the growing ruxolitinib add-on portfolio. (novelty: 3)
• 2026-08-13 — Study of Flonoltinib Maleate Tablets in the Treatment of Proliferative Bone Marrow Tumors: Comparator context (preclinical → trial-early): flonoltinib maleate is being positioned as a higher-JAK2-selectivity, lower-toxicity alternative to ruxolitinib in preclinical models, with early clinical evaluation now underway in MF, PV, and ET. (novelty: 3)
• 2026-07-20 — Reduced Intensity Haploidentical Transplantation for the Treatment of Primary or Secondary Myelofibrosis: Phase II trial (trial-early) of reduced-intensity haploidentical HCT in primary/secondary MF: pre-transplant ruxolitinib achieved universal engraftment with no graft failure but high GVHD, motivating a new cohort with peri-transplant (pre/during/post) ruxolitinib to lower GVHD without raising graft-failure risk — reinforces the peri-transplant JAKi paradigm in the specific haploidentical RIC setting. (novelty: 3)
• 2026-07-15 — Phase I/II Clinical Trial of Axatilimab, a CSF1R Monoclonal Antibody, in Combination With Ruxolitinib as Therapy for Patients With Myelofibrosis (MF) and Chronic Myelomonocytic Leukemia (CMML): Trial-early (Phase I/II): ruxolitinib serving as combination backbone with axatilimab (CSF1R monoclonal antibody) in MF and CMML — adds a macrophage/monocyte-directed (CSF1R) mechanism to the ruxolitinib add-on portfolio and extends the ruxolitinib combination framework into CMML. (novelty: 3)
• 2026-08-10 — Flonoltinib Maleate Oral Regimens in Patients With Myelofibrosis: cross-connection with flonoltinib-maleate: Trial enrolls patients whose MF was not adequately controlled by the most common approved therapy (ruxolitinib), positioning flonoltinib maleate as a post-JAK1/2-inhibitor option. (novelty: 3)
• 2026-07-16 — An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell Transfusions: cross-connection with luspatercept: Phase 3 trial specifically enrolls MF patients on concomitant JAK2 inhibitor therapy, positioning luspatercept as an add-on to ruxolitinib (and other JAKi) for transfusion-dependent anemia. (novelty: 3)
• 2026-09-03 — A Study of Ruxolitinib in Combination With Abemaciclib for the Treatment of Myelofibrosis: Trial-early: ruxolitinib serving as combination backbone with abemaciclib (CDK4/6 inhibitor) in primary and post-PV/post-ET myelofibrosis — adds a cell-cycle inhibitor mechanism to the growing ruxolitinib add-on portfolio. (novelty: 3)
• 2026-08-01 — Type II JAK2 Inhibitor Gets Off to a Strong Start.: Trial-early comparator context: early Phase 1 responses to AJ1-11095 (type II JAK2 inhibitor) are being framed as potentially outperforming currently approved type I JAK2 inhibitors including ruxolitinib, sharpening the case that inactive-conformation JAK2 binding may overcome type I inhibitor limitations. (novelty: 4)
• 2026-06-16 — Final analysis of phase 2 clinical trial of ruxolitinib and azacitidine combination therapy in patients with myelodysplastic syndrome/myeloproliferative neoplasms.: Final Phase 2 analysis (trial-early) of ruxolitinib + azacitidine in MDS/MPN overlap: 58% ORR and median OS 26.7 months, with encouraging post-alloHCT survival — positions ruxolitinib as a viable HMA-combination backbone in the MDS/MPN overlap setting distinct from its MF/PV indications. (novelty: 3)
• 2026-06-30 — A systematic review of 47 published cases of extramedullary hematopoiesis in myelofibrosis: clinical patterns, treatment responses, and prognostic implications.: Systematic review of 47 published cases of atypical extramedullary hematopoiesis in myelofibrosis identifies ruxolitinib as a frequently effective systemic therapy for EMH lesions, though the syndrome marks advanced disease with high short-term mortality warranting early transplant referral. (novelty: 3)
• 2026-09-01 — Matching-Adjusted Indirect Comparison of Gecacitinib, Fedratinib, Pacritinib, and Momelotinib in Second-Line Myelofibrosis Therapy.: MAIC (observational) of second-line JAK inhibitors in ruxolitinib-pretreated MF adds gecacitinib to the post-ruxolitinib salvage comparator set alongside fedratinib, pacritinib, and momelotinib, with gecacitinib ranking highest on spleen/symptom response and GI tolerability. (novelty: 3)
• 2026-08-23 — Sequential High-Dose Ruxolitinib and Low-Dose Splenic Irradiation for Pretransplant Management of Splenomegaly Before Allogeneic Hematopoietic Cell Transplantation With Fludarabine/Busulfan-Based Conditioning in Myelofibrosis.: Retrospective series (observational): high-dose ruxolitinib as first step of a sequential pretransplant debulking protocol (followed by low-dose splenic irradiation) shrank spleen size before Flu/Bu allo-HCT with acceptable engraftment and early survival — reinforces high-dose/intensified ruxolitinib dosing as a pretransplant strategy. (novelty: 3)
• 2026-08-27 — Final results of a phase II study of the combination of azacitidine and ruxolitinib in patients with myelofibrosis.: Final phase II results (trial-early) of azacitidine + ruxolitinib combination in MF reported high IWG-MRT objective response rates, positioning HMA add-on as one route to address ruxolitinib monotherapy’s limited disease-modifying effect. (novelty: 3)
• 2026-06-02 — Selinexor Plus Ruxolitinib in Janus Kinase Inhibitor-Naïve Myelofibrosis: Phase III SENTRY Trial.: Phase III SENTRY (trial-late): ruxolitinib as backbone vs selinexor + ruxolitinib in JAKi-naïve MF — combination arm improved SVR35 and showed early OS signal, but symptom scores were not improved and additive hematologic toxicity was observed. (novelty: 3)
• 2026-07-01 — Selinexor in combination with azacitidine or ruxolitinib in myelodysplastic/myeloproliferative neoplasm overlap syndromes: A multicenter prospective study.: Multicenter prospective study (trial-early): ruxolitinib serving as one of two backbone partners (with azacitidine as the alternative) for selinexor combination therapy in MDS/MPN overlap syndromes, achieving 60% ORR — extends the ruxolitinib add-on portfolio into the MDS/MPN overlap population. (novelty: 3)
• 2026-08-01 — A dynamic prognostic model for polycythemia vera long-term outcomes in patients treated with ruxolitinib.: Observational (dynamic prognostic model): PV-RR6, a 6-month on-treatment score combining ruxolitinib dose intensity, spleen reduction, and ongoing phlebotomy need, stratifies ruxolitinib-treated PV patients into low/intermediate/high-risk groups with 5-year event-free survival of ~89%/71%/39% — enables early identification of ruxolitinib underperformers in PV. (novelty: 3)
• 2026-07-22 — Disease Burden, Inadequate Hematocrit Control, and Thromboembolic Events in Patients with Polycythemia Vera Despite Current Standard of Care Treatment: A Retrospective Claims Study.: US retrospective claims analysis (n=11,311, observational): ruxolitinib is captured within the PV SoC cytoreductive treatment mix, where despite therapy most patients fail to maintain HCT <45% and continue to experience thromboembolic events and high post-TE costs — flags real-world efficacy gap even within cytoreductive-treated PV subpopulations. (novelty: 3)
• 2026-07-14 — Targeting nuclear export and Janus Kinase/Signal Transducer and Activator of Transcription (JAK/STAT) signalling in myelofibrosis: A novel combinatorial strategy that impacts intrinsic and microenvironment-related pathways.: Preclinical mechanistic study identifies NF-κB-driven inflammatory signaling and microenvironment cytokine crosstalk as ruxolitinib-resistance pathways that are re-suppressed when selinexor (XPO1 inhibitor) is added, providing a molecular rationale for the SENTRY combination and for combinations targeting nuclear export in JAKi-resistant MF. (novelty: 3)
• 2026-08-14 — Janus kinase inhibitors after ruxolitinib failure in myelofibrosis: A systematic review and pooled analysis of phase 3 efficacy and integrated safety across clinical trials and real-world evidence.: Meta-analysis of second-line JAK inhibitors after ruxolitinib failure formalizes the post-ruxolitinib salvage landscape, showing fedratinib, momelotinib, and pacritinib offer non-overlapping spleen/symptom/safety profiles that can be matched to the ruxolitinib-failure phenotype (cytopenias, anemia, GI intolerance). (novelty: 3)
• 2026-07-01 — Ruxolitinib for the Treatment of Chronic Myelomonocytic Leukemia (CMML): A Phase 2 Expansion: Phase 2 expansion trial (trial-early) evaluating ruxolitinib in chronic myelomonocytic leukemia (CMML) — extends the JAK1/2 inhibitor into an MDS/MPN overlap disorder distinct from its approved MF/PV indications. (novelty: 3)
• 2025-11-20 — Splenic Artery Aneurysm Associated with Myelofibrosis Managed by Coil Embolization.: Case report: Ruxolitinib-induced splenomegaly reduction in a JAK2 V617F-positive secondary myelofibrosis patient enabled safe spleen-preserving coil embolization of a splenic artery aneurysm, suggesting a novel role for JAK inhibition as a hemodynamic bridge to endovascular intervention in MF-associated vascular complications. (novelty: 3)
• 2026-04-27 — JAK Inhibitor-Induced Improvement of IgA Nephropathy in a Patient With Polycythemia Vera: A Case Report.: Case report: Ruxolitinib in a PV patient with concurrent IgA nephropathy produced reduction in proteinuria and stabilization of renal function, proposing JAK-STAT-mediated suppression of IL-6/IL-11-driven mesangial proliferation and aberrant hematopoietic IgA production as the mechanism — first reported off-target benefit of JAK inhibition in IgAN. (novelty: 4)
• 2026-06-23 — A Study of LY2784544 in Participants With Myeloproliferative Neoplasms: Trial-early: LY2784544 (selective JAK2 inhibitor) enrolling PV/ET/MF patients including ruxolitinib-intolerant and ruxolitinib-refractory cases — adds another JAK2-selective candidate to the post-ruxolitinib salvage landscape alongside AJ1-11095, flonoltinib, and PRT12396. (novelty: 3)
• December 7 — Early Data Show Mutant CALR-Specific Monoclonal Antibody Well Tolerated in Myelofibrosis: ASH 2025 (trial-early): ruxolitinib was well tolerated as a combination backbone with the mutant CALR-specific monoclonal antibody INCA033989 in myelofibrosis, adding a CALR-neoantigen-directed antibody to the growing ruxolitinib add-on portfolio. (novelty: 4)
• May 1, 202 — Myelofibrosis (MF) in 2026: Matching Therapy to the Dominant Clinical Problem: Narrative review (2026, review-level) frames ruxolitinib as the combination backbone in MANIFEST-2 (pelabresib add-on) within a broader ‘match therapy to dominant clinical problem’ paradigm in MF. (novelty: 3)
• April 28, — Biomed Valley Discoveries Phase 1/2 Trial: Ulixertinib + Ruxolitinib for Myelofibrosis: Biomed Valley Discoveries Phase 1/2 trial (trial-early): ruxolitinib serving as backbone with ulixertinib (selective ERK1/2 inhibitor) add-on in myelofibrosis, targeting MAPK pathway reactivation as a downstream JAK-inhibition escape mechanism. (novelty: 4)
• June 12-15 — Advancing Frontiers in Myeloproliferative Neoplasms - EHA 2025 Congress Review: EHA 2025 congress review (review-level) features ruxolitinib as the combination backbone in headline MF programs — pelabresib + ruxolitinib (MANIFEST-2 Phase III) and elritercept add-on for anemia — alongside CALR-directed immunotherapy (INCA035784) as a complementary mutation-selective approach. (novelty: 3)
• 2026-06-17 — A Study Comparing Imetelstat Versus Best Available Therapy for the Treatment of Intermediate-2 or High-risk Myelofibrosis (MF) Who Have Not Responded to Janus Kinase (JAK)-Inhibitor Treatment: IMpactMF (Phase III, trial-late): imetelstat vs best available therapy in intermediate-2/high-risk MF patients who have relapsed or are refractory to JAK inhibitors (predominantly ruxolitinib) — defines the post-ruxolitinib salvage population as the trial entry criterion and tests OS benefit in this high-unmet-need setting. (novelty: 4)
• 2025-10-30 — Effective Treatment with Ruxolitinib and Ropeginterferon Alfa-2b for Refractory TAFRO-like Syndrome.: Case report: ruxolitinib combined with ropeginterferon alfa-2b resolved refractory TAFRO-like syndrome in a JAK2 V617F-positive PV patient, suggesting a novel application of the JAKi + pegylated IFN combination in a rare inflammatory complication of PV. (novelty: 3)
• 2026-06-18 — Disseminated Tuberculosis Mimicking Metastatic Cancer During Ruxolitinib Therapy for Polycythemia Vera: A Case Report and Literature Review.: Case report + literature review of 35 cases: ruxolitinib-induced immunosuppression in PV can precipitate disseminated tuberculosis mimicking metastatic cancer, with false-negative IGRA and spuriously elevated tumor markers — reinforces a clinically meaningful infection-safety signal and a diagnostic pitfall during ruxolitinib therapy. (novelty: 3)
• 2026-06-08 — Selinexor in Myelofibrosis Refractory or Intolerant to JAK1/2 Inhibitors: Trial-early: A phase II single-arm trial of selinexor monotherapy in patients refractory or intolerant to ruxolitinib and/or other JAK1/2 inhibitors highlights the unmet need in the post-ruxolitinib salvage setting and positions XPO1 inhibition as a non-JAK mechanistic alternative for ruxolitinib failures. (novelty: 3)
• 2026-06-03 — A Study to Compare Elritercept to Placebo in Adults With Myelofibrosis and Anemia Who Are Taking Ruxolitinib: Trial-late (randomized placebo-controlled): ruxolitinib serving as the ongoing background therapy in a placebo-controlled trial of elritercept add-on for MF-associated anemia — advances the anemia-targeted ligand-trap add-on portfolio (alongside luspatercept, sapablursen, DISC-0974) from early-phase into late-phase randomized evidence. (novelty: 3)
• 2026-06-04 — A Phase 1/2 Study of CPI-0610 With and Without Ruxolitinib in Patients With Hematologic and Myeloproliferative Malignancies: Phase 1/2 CPI-0610 study (trial-early): ruxolitinib served as the combination backbone with pelabresib (BET bromodomain inhibitor) in MF and ET — the foundational early-phase trial establishing the BET + JAK combination rationale later advanced into MANIFEST-2/3. (novelty: 3)
• 2026-06-05 — The impact of novel bet inhibitors on the treatment armamentarium of Myelofibrosis.: Review-level synthesis argues that BET inhibitors mechanistically complement ruxolitinib by targeting transcriptional programs (NF-κB-driven inflammation, TGF-β-driven fibrosis, MYC-driven proliferation) that persist under JAK inhibition, supporting the rationale for BET + JAK combination therapy in frontline MF. (novelty: 3)
• May 21, 20 — CALR Mutation–Directed Agents and Targeted Therapies Represent Key Research Avenue in MPN Management: Review-level synthesis (May 2026) reinforces ruxolitinib’s role as the combination backbone across the emerging MF pipeline — SENTRY (selinexor add-on, first co-primary endpoint met), MANIFEST-3 (pelabresib add-on), IMpactMF (imetelstat), and CALR-directed agents (INCA033989) — anchoring the precision-medicine combination paradigm in frontline MF. (novelty: 3)
• December 6 — Prelude Therapeutics Presents Data at 2025 ASH Annual Meeting from its Myeloproliferative Neoplasm Programs: ASH 2025 (preclinical, comparator context): PRT12396 (mutant-selective JAK2V617F JH2 inhibitor) reported preclinical superiority to ruxolitinib while sparing WT JAK2 cytokine signaling — positioning mutant-selective inhibition as a mechanism to overcome ruxolitinib’s WT-JAK2-driven cytopenias and immunosuppression. (novelty: 4)
• May 22, 20 — All Eyes Are on Novel Combination Therapies and Molecular Targets in MPN at ASH 2025: ASH 2025 review: 96-week MANIFEST-2 Phase 3 data confirm durable spleen volume reduction, symptom improvement, and anemia benefit with pelabresib + ruxolitinib in JAK-inhibitor-naive myelofibrosis, supporting the combination’s long-term benefit over ruxolitinib monotherapy. (novelty: 3)
• 2026-05-19 — TGR-1202 + Ruxolitinib PMF PPV-MF PET-MF MDS/MPN Polycythemia Vera Resistant to Hydroxyurea: Trial-early (Phase 1, open-label): ruxolitinib serving as combination backbone with TGR-1202 (umbralisib, PI3Kδ inhibitor) across PMF, PPV-MF, PET-MF, MDS/MPN, and HU-resistant PV — adds a third PI3Kδ inhibitor partner (alongside roginolisib and IOA-244) to the ruxolitinib add-on portfolio. (novelty: 3)
• January 21 — Zavabresib Earns FDA Orphan Drug Designation in Myelofibrosis: 2026 — Zavabresib (OPN-2853, selective BET inhibitor) reported durable spleen reduction when added to ruxolitinib in ruxolitinib-inadequate responders, joining pelabresib and INCB057643 as BET-inhibitor add-ons to the ruxolitinib salvage portfolio; FDA Orphan Drug Designation granted. (novelty: 3)
• 2026-05-14 — INCB000928 Administered as a Monotherapy or in Combination With Ruxolitinib in Participants With Anemia Due to Myeloproliferative Disorders: Trial-early (Phase 1/2): ruxolitinib serving as combination backbone with INCB000928 (putative ACVR1/ALK2 inhibitor targeting hepcidin axis) in MF patients with transfusion-dependent or symptomatic anemia — adds an ACVR1-directed anemia-targeted mechanism to the ruxolitinib add-on portfolio (alongside luspatercept, elritercept, sapablursen, DISC-0974, nelfinavir). (novelty: 4)
• 2026-04-14 — A Study of JNJ-88549968 for the Treatment of Calreticulin (CALR)-Mutated Myeloproliferative Neoplasms: Trial-early (Phase 1/2): ruxolitinib serving as combination backbone with JNJ-88549968, a novel CALR-mutation-directed agent, in CALR-mutated ET and MF — adds a mutation-specific (rather than pathway-specific) add-on strategy to the ruxolitinib combination portfolio. (novelty: 4)
• 2026-04-17 — Tolerability of Ropeginterferon Alfa-2b Add-on to Ongoing Ruxolitinib Therapy in Myelofibrosis (RopeRux in Myelofibrosis): Trial-early (RopeRux in Myelofibrosis): ruxolitinib serving as ongoing backbone with ropeginterferon alfa-2b add-on in MF — extends the ruxolitinib add-on portfolio to include long-acting pegylated interferon as a disease-modifying partner. (novelty: 4)
• 2026-02-05 — A Study of Elritercept Alone or Together With Ruxolitinib in Adults With Myelofibrosis: Trial-early (Phase 1/2): ruxolitinib serving as combination backbone with elritercept (TGF-β superfamily ligand trap / erythroid maturation agent) in adult MF, with anemia as a co-endpoint alongside spleen/symptom — adds an anemia-targeted ligand-trap mechanism to the ruxolitinib add-on portfolio (alongside luspatercept, sapablursen, DISC-0974). (novelty: 4)
• 2026-03-30 — A Study to Evaluate Safety , Efficacy and Pharmacokinetics of WJ01024 Tablets Combined With Ruxolitinib in Patients With Myelofibrosis: Trial-early (Phase Ib/II): ruxolitinib serving as combination backbone with WJ01024 (novel investigational agent) in JAK-inhibitor-refractory/intolerant medium-to-high-risk MF with splenomegaly — adds another candidate to the growing ruxolitinib salvage/augmentation portfolio. (novelty: 3)
• 2026-03-23 — Study of Oral Navitoclax Tablet In Combination With Oral Ruxolitinib Tablet When Compared With Oral Ruxolitinib Tablet To Assess Change In Spleen Volume In Adult Participants With Myelofibrosis: Phase 3 RCT (trial-late): ruxolitinib serving as both backbone and active comparator in a randomized study of navitoclax (BCL-2/BCL-XL inhibitor) + ruxolitinib vs ruxolitinib monotherapy in adult MF, with spleen volume reduction as primary endpoint — first Phase 3 readout of a BCL-2/BCL-XL add-on to the ruxolitinib backbone. (novelty: 3)
• 2026-04-14 — Peritransplant Ruxolitinib for Patients With Primary and Secondary Myelofibrosis: Phase II trial (trial-early): ruxolitinib being administered across the entire peri-transplant window (pre-, during, and post-allo-HSCT) in primary and secondary myelofibrosis to leverage its anti-inflammatory/immunomodulatory effect for GVHD prevention — extends ruxolitinib’s transplant role from bridging into a continuous peri-transplant protocol. (novelty: 3)
• 2026-03-11 — High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly: Trial-early: high-dose ruxolitinib combined with busulfan as pre-transplant conditioning being evaluated to reduce splenomegaly and improve allo-SCT success in myelofibrosis — extends ruxolitinib’s role from bridging into the conditioning regimen itself. (novelty: 3)
• 2026-04-03 — A Study of Ruxolitinib in Combination With Ulixertinib in People With Myelofibrosis: Trial-early (Phase I/II): ruxolitinib being combined with ulixertinib (ERK1/2 inhibitor) in myelofibrosis to target MAPK pathway reactivation as a downstream escape mechanism from JAK inhibition — adds an ERK1/2-targeted strategy to the expanding ruxolitinib add-on portfolio. (novelty: 4)
• 2026-03-30 — A Study to Assess a PI3Kδ Inhibitor (IOA-244) in Patients With Metastatic Cancers: Trial-early: ruxolitinib being combined with IOA-244 (selective PI3Kδ inhibitor) in primary myelofibrosis as part of a multi-arm metastatic-cancers Phase I — adds a second PI3Kδ inhibitor (alongside roginolisib) to the ruxolitinib add-on portfolio targeting PI3K-AKT signalling. (novelty: 4)
• 2026-04-08 — A Clinical Trial of Flonoltinib Maleate Tablets in the Treatment of JAK Inhibitor Refractory/Relapsed/Intolerant Patients With Medium to High Risk Myelofibrosis: cross-connection with flonoltinib-maleate-mpn: Trial population is defined by failure/intolerance to prior JAK inhibitors (predominantly ruxolitinib), positioning flonoltinib as a salvage option. (novelty: 4)
• 2026-04-13 — A Clinical Study to Test the Effects of Ruxolitinib And Thalidomide Combination for Patients With Myelofibrosis: Trial-early: ruxolitinib being evaluated in combination with thalidomide (immunomodulatory/anti-angiogenic agent) in myelofibrosis — adds an IMiD-class add-on to the expanding ruxolitinib combination portfolio, potentially targeting cytopenias and bone marrow stromal/angiogenic remodeling. (novelty: 3)
• 2026-04-09 — Study of Selinexor in Combination With Ruxolitinib in Myelofibrosis: Trial-late: Ruxolitinib serving as the backbone (with placebo control arm) in a global Phase 1/3 trial of selinexor add-on in JAKi-naïve MF. (novelty: 3)
• 2026-04-09 — Comparing Momelotinib and Ruxolitinib in People With Untreated Myelofibrosis and Low Blood Cell Counts: Trial-early: ruxolitinib serving as the active comparator (not placebo) in a frontline head-to-head trial against momelotinib in untreated MF patients with low blood counts — tests whether ruxolitinib’s established spleen response holds up against momelotinib in the cytopenic subpopulation where ruxolitinib dosing is typically constrained. (novelty: 3)
• 2026-03-24 — A Study to Evaluate INCA033989 Administered as a Monotherapy or in Combination With Ruxolitinib in Participants With Myeloproliferative Neoplasms: Phase 1 trial (trial-early): ruxolitinib serving as combination backbone with INCA033989 (novel investigational agent, undisclosed mechanism) in MPN patients — adds another candidate to the growing ruxolitinib add-on combination portfolio. (novelty: 4)
• 2026-03-31 — Ruxolitinib Phosphate and Azacytidine in Treating Patients With Myelofibrosis or Myelodysplastic Syndrome/Myeloproliferative Neoplasm: Phase II trial (trial-early): ruxolitinib being combined with azacytidine in MF and MDS/MPN overlap neoplasms — extends ruxolitinib backbone into hypomethylating-agent combinations for overlap syndromes. (novelty: 3)
• 2026-03-16 — Decitabine With Ruxolitinib, Fedratinib or Pacritinib for the Treatment of Accelerated/Blast Phase Myeloproliferative Neoplasms: Phase II trial (trial-early): ruxolitinib being evaluated as one of three JAK inhibitor options combined with decitabine as pre-transplant bridging therapy in accelerated/blast phase MPN, vs multi-agent chemotherapy or no bridging. (novelty: 3)
• 2026-04-16 — A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF): Phase 3 trial (trial-late): ruxolitinib serving as backbone in randomized comparison of pelabresib + ruxolitinib vs ruxolitinib monotherapy in JAK-inhibitor-naive MF — first Phase 3 readout of a BET inhibitor add-on to the ruxolitinib backbone. (novelty: 4)
• 2026-04-24 — Testing the Effect of Taking Ruxolitinib and CPX-351 in Combination for the Treatment of Advanced Phase Myeloproliferative Neoplasms: Phase I/II trial (trial-early): ruxolitinib being combined with CPX-351 (liposomal daunorubicin/cytarabine) in accelerated/blast phase MPN to determine optimal dose and efficacy — extends ruxolitinib’s role into AML-like induction chemotherapy backbones for advanced-phase disease. (novelty: 3)
• 2026-03-27 — A Phase 1 Study of AJ1-11095 in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (PPV-MF), or Post-Essential Thrombocythemia Myelofibrosis (PET-MF) Who Have Been Failed by a Type I JAK2 Inhibitor (JAK2i): AJX-101 (Phase 1, trial-early): AJ1-11095, a type II JAK2 inhibitor, entering first-in-human testing specifically in patients who have failed type I JAK2 inhibitors including ruxolitinib — directly addresses the ruxolitinib-refractory population. (novelty: 4)
• 2026-04-02 — Study of Pelabresib add-on to Ruxolitinib in Japanese Adult Patients With Myelofibrosis: Trial-early (Phase 1b): ruxolitinib backbone with pelabresib add-on being evaluated in Japanese MF patients for safety, PK, and preliminary efficacy — bridging study extending the combination to an East Asian population. (novelty: 3)
• 2026-02-27 — Open Label Phase 1/2 Study of Tasquinimod in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), or Post-Essential Thrombocytosis Myelofibrosis (Post-ET MF): Trial-early (Phase 1/2): Being evaluated in combination with tasquinimod (first-in-class S100A9/HDAC4 inhibitor) in PMF, post-PV MF, and post-ET MF — adds a novel immunomodulatory/epigenetic mechanism to the expanding ruxolitinib add-on portfolio. (novelty: 4)
• 2026-04-01 — Safety and Tolerability Study of INCB057643 in Participants With Myelofibrosis and Other Advanced Myeloid Neoplasms: Trial-early: Being evaluated as combination backbone with INCB057643 (BET bromodomain inhibitor) in myelofibrosis and other advanced myeloid neoplasms — adds BET inhibition to the growing list of ruxolitinib add-on strategies (alongside selinexor, roginolisib, pelabresib). (novelty: 3)
• 2026-04-28 — MITHRIDATE: Ruxolitinib Versus Hydroxycarbamide or Interferon as First Line Therapy in High Risk Polycythemia Vera: MITHRIDATE (phase III RCT, trial-late): ruxolitinib being directly compared head-to-head against hydroxycarbamide and interferon alpha as first-line therapy in high-risk PV — first large-scale three-arm comparison of its kind. (novelty: 4)
• April 27, — Lilly to Acquire Ajax Therapeutics for Next-Generation JAK2 Inhibitors in MPNs: News (other): Lilly’s $2.3B acquisition of Ajax Therapeutics highlights development of AJ1-11095, a Type II JAK2 inhibitor explicitly targeting ruxolitinib-refractory MF — underscores the clinical gap of Type I JAK2 inhibitor resistance. (novelty: 4)
• 2026-03-24 — Karyopharm Phase 3 SENTRY Trial in Myelofibrosis Met First Co-Primary Endpoint: Phase 3 SENTRY (trial-late): adding selinexor (XPO1 inhibitor) to ruxolitinib backbone improved spleen volume response and modestly increased ≥20% VAF reduction rate (32% vs 24%) over ruxolitinib monotherapy in MF. (novelty: 4)
• April 2, 2 — Peri-Transplant Ruxolitinib Leads to Low GVHD Incidence in Myelofibrosis - OncLive: Trial-early (OncLive): Peri-transplant ruxolitinib use is associated with low GVHD incidence in myelofibrosis patients undergoing allogeneic SCT, supporting its role as a peri-transplant adjunct beyond symptom control. (novelty: 3)
• 2026-04-27 — A Study of Roginolisib in Combination With Ruxolitinib in Patients With Myelofibrosis (MF) Who Are Unresponsive to JAK Inhibitors: Trial-early: Being evaluated in combination with roginolisib (PI3K-delta inhibitor) for patients who are unresponsive to JAK inhibitor monotherapy, as a salvage/augmentation strategy in MF. (novelty: 4)

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