Tavalisse (fostamatinib) / Rigel Pharmaceuticals, Kissei, JW Pharma, Grifols, Knight Therap, Tanabe Pharma - LARVOL DELTA

Fostamatinib Dual Immunomodulation in Post-Haploidentical HSCT Autoimmune Cytopenia and Autoimmune Hepatitis: A Case Report and Review of Literature. (PubMed, J Clin Immunol) - No abstract available

Journal • Autoimmune Hepatitis • Bone Marrow Transplantation • Hematological Disorders • Immunology • Inflammation • Pediatrics • Transplantation

NETWORK-BASED PROTEOMIC SIGNATURES REVEAL DIAGNOSTIC BIOMARKERS AND REPURPOSABLE THERAPEUTICS IN PARKINSON'S DISEASE (ADPD 2026) - "Drug perturbation mapping identified SAL-1, fostamatinib, homoharringtonine, and 2-aminopurine as candidates capable of reversing PD-associated proteomic signatures. Our findings demonstrate that proteomics-based models outperform RNA-based and multimodal approaches in classifying PD and tracking disease severity. Our findings demonstrate that proteomics-based models outperform RNA-based and multimodal approaches in classifying PD and tracking disease severity. The identified biomarkers are functionally enriched in extracellular and immune-related processes, offering insight into PD pathophysiology. Network-informed perturbation analysis further nominates candidate therapeutics for experimental validation."

Biomarker • CNS Disorders • Movement Disorders • Parkinson's Disease

A multicentre analysis of efficacy, safety and molecular response correlates of fostamatinib in warm autoimmune haemolytic anaemia and Evans syndrome. (PubMed, Br J Haematol) - "Only one patient required drug discontinuation and one patient dose reduction. In an eight-patient subset, migration inhibitory factor (MIF) levels, naïve (CD62Lhi) T-cell and HLA-DRhi monocyte subsets each correlated with treatment response."

Journal • Anemia • Autoimmune Hemolytic Anemia • Cardiovascular • Hematological Disorders • Hypertension • Immunology • Neutropenia

Spleen tyrosine kinase as a novel target in medulloblastoma (AACR 2026) - "We used pharmacological inhibition (R406, Bay61, Fostamatinib) in MYC-amplified MB cell lines (HD-MB03 and D425) to assess cell viability and MYC protein levels via MTT assays and Western blotting. The preliminary findings support the hypothesis that SYK acts as a key regulator of MYC-driven tumorigenesis in aggressive medulloblastoma. The observed synergistic toxicity when combining SYK inhibitors with cisplatin in D425 cells highlights a prospective chemo-sensitization mechanism. Targeting the SYK-MYC axis using SYK inhibitors, especially in combination with existing chemotherapies, offers a powerful, clinically translatable strategy to overcome treatment resistance and significantly improve outcomes for patients with high-risk, MYC-driven medulloblastoma"

Brain Cancer • Medulloblastoma • Oncology • Solid Tumor • MYC • SYK

A Retrospective US Claims Database Study Assessing Treatment Patterns and Bleeding Episodes in Patients with Previously Treated Primary Immune Thrombocytopenia (THSNA 2026) - "Previously treated patients were defined as currently receiving ITP treatment, with 1 prior therapy of OCS, IVIg, IV anti-D, or platelet transfusion, and ≥1 prior therapy of TPO-RA, rituximab, fostamatinib, anti-CD38 monoclonal antibodies, immunosuppressants, danazol, dapsone, bortezomib, vinca alkaloid, or splenectomy after the initial diagnosis. According to this US-based claims study, many patients with primary ITP experience bleeding episodes requiring treatment with rescue therapies, despite receiving first- and second-line treatments, implying a clinically significant decrease in platelets, and highlighting the unmet need for more effective therapies for patients with primary ITP. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

Claims database • Retrospective data • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Rare Diseases • Thrombocytopenia • Thrombocytopenic Purpura

Targeting B-cells in Hidradenitis Suppurativa: A Systematic Review of Bruton's Tyrosine Kinase Inhibition as a Target (AAD 2026) - "Adalimumab remains the only FDA approved biologic, but many patients experience suboptimal control...While no BTKi studies in HS exist, a small proof-of-concept trial of the spleen tyrosine kinase inhibitor fostamatinib reported Hidradenitis Suppurativa Clinical Response score (HiSCR) achievement in 85% of participants by week 4...Further early-phase clinical trials are warranted to establish safety, efficacy, and durability of BTK inhibition in HS. This review underscores an urgent need to expand therapeutic options beyond TNF-α blockade for patients with refractory disease."

Review • Dermatology • Hidradenitis Suppurativa • Immunology • SDC1

FPR2/ALX stimulation modulates microglia and natural killer cells to restrict autoimmune astrocytopathy. (PubMed, Acta Pharmacol Sin) - "Notably, the benefits of FPR2/ALX stimulation were attenuated in mice with autoimmune astrocytopathy after microglial depletion using the CSF1R inhibitor PLX5622 or natural killer (NK) cell depletion using an anti-NK1.1 monoclonal antibody. Additionally, the protective effects of FPR2/ALX stimulation were diminished in mice with autoimmune astrocytopathy that received the SYK inhibitor R406...FPR2/ALX stimulation suppresses autoimmune astrocytopathy: Using a mouse model of autoimmune astrocytopathy, we demonstrated that FPR2/ALX stimulation with the small molecule Quin-C1 reduces the CNS infiltration of lymphocytes and augments the anti-inflammatory activity of microglia, leading to attenuated astrocyte pathology induced by AQP4-IgG and complement-mediated attacks. Mechanistically, the benefits of FPR2/ALX stimulation using Quin-C1 involve microglia, natural killer (NK) cells, and SYK-AKT signaling."

Journal • Immunology • Inflammation • FPR2 • SYK

Efficacy of fostamatinib add-on for TPO receptor agonist-refractory immune thrombocytopenia (PubMed, Rinsho Ketsueki) - "The combination of fostamatinib and TPO-RAs may provide an additive effect due to their distinct mechanisms of action, offering a promising therapeutic option for refractory ITP. As this report includes only a small number of cases, more cases are needed to evaluate the efficacy and safety of this combination."

Journal • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura • SYK

A Clinician Perspective for a Personalized Approach to Management of Chronic Immune Thrombocytopenia with Targeted Therapies Alone or in Combination. (PubMed, J Clin Med) - "This review summarizes ITP pathogenesis and the treatment landscape and proposes a personalized treatment approach for ITP after first-line treatment (corticosteroids, intravenous immunoglobulin, anti-D therapy) based on targeting underlying disease mechanisms with immunomodulatory and bone marrow-supportive therapies (fostamatinib, rituximab, and thrombopoietin receptor agonists [TPO-RAs]) prior to proceeding to later-line therapies. Patients with inadequate response to fostamatinib or TPO-RA monotherapy may combine these therapies to address both platelet destruction and platelet production deficits. This novel framework tailors therapy to patient-specific pathophysiology by preferentially targeting both impaired platelet production and increased platelet destruction to support individualized care."

Journal • Review • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Sustained response off-treatment and thrombotic events in patients with immune thrombocytopenia treated with fostamatinib: a systematic review and meta-analysis. (PubMed, Haematologica) - "Not available."

Journal • Retrospective data • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Fostamatinib treatment for patients with antiphospholipid syndrome and low platelet count: A case series. (PubMed, Br J Haematol) - No abstract available

Journal • Genetic Disorders • Hematological Disorders • Thrombocytopenia

Fmoc-DDT@Fos hydrogel mitigates temporomandibular joint osteoarthritis through regulating macrophage reprogramming and ferroptosis. (PubMed, Mater Today Bio) - "In this study, we develop a novel peptide hydrogel drug delivery system named Fmoc-DDT@Fos, combining Fmoc-FF peptides with PLGA/TPP nanospheres loaded with Fostamatinib (Fos), functionalized for sustained release and locally targeted application of Fos...By elucidating these mechanisms, our research not only offers a novel therapeutic strategy for TMJ OA but also deepens our understanding of its pathophysiology. Moreover, this biomaterial-based strategy may provide a safer and more effective framework for managing other inflammatory and degenerative diseases."

Journal • Immunology • Inflammation • Osteoarthritis • Pain • Rheumatology

Translational immunothrombosis in autoimmune Heparin-Induced thrombocytopenia: targeting the FcγRIIa-Syk-BTK and complement pathways. (PubMed, Clin Exp Med) - "Preclinical and clinical data indicate that targeting these axes with Syk inhibitors (fostamatinib), BTK inhibitors (rilzabrutinib, zanubrutinib), and complement inhibitors (sutimlimab) can restore platelet counts and mitigate immune-driven thrombosis. These findings underscore the therapeutic potential of pathway-specific interventions in aHIT, highlighting the need for biomarker-guided, translational trials to validate their efficacy and safety. Bridging mechanistic evidence from ITP and APS provides a framework for precision immunotherapy in autoimmune HIT."

IO biomarker • Journal • Review • Cardiovascular • Genetic Disorders • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis • SYK

GARD: Genomic Data based Drug Repurposing in Head and Neck Cancer with Large Language Model Validation. (PubMed, bioRxiv) - "Drug-gene mapping revealed candidates spanning already in clinical trials for HNC (e.g. Afatinib, Cabozantinib, Dasatinib, Brigatinib, Lenvatinib, Capivasertib, Erdafitinib) and emerging or repurposing candidates (Amuvatinib, XL765 (Voxtalisib), Golotimod, Artenimol, Quercetin, and Acetylsalicylic Acid), offering opportunities for precision repurposing...These included targeted therapies such as Fostamatinib, Nintedanib, Brigatinib, Regorafenib, and Lenvatinib, as well as emerging compounds like Artenimol, Quercetin, and Acetylsalicylic Acid (Aspirin). Through a combination of genomic analysis, network expansion, and literature validation, the GARD pipeline offers a powerful way to accelerate personalized cancer treatments while reducing cost and development time."

IO biomarker • Journal • Head and Neck Cancer • Oncology • Solid Tumor • CLDN1 • EGFR • EIF4G1 • HER-2 • PIK3CA • SOX2 • TLR7 • TP53

Refractory Primary Immune Thrombocytopenia With Bleeding and Thrombosis: A Case Report. (PubMed, Am J Case Rep) - "He was nonresponsive to initial prednisone treatment and had petechial gastritis...Still, the patient was refractory to other therapeutic options, including vincristine and anti-D immunoglobulins, showing only transient platelet count response while also showing cerebral hemorrhage. Hence, he was treated with splenectomy and rituximab...A course of immunosuppressant therapy with azathioprine and mycophenolate mofetil was started with no response. Alternative therapies including eltrombopag, bortezomib, and dapsone resulted in no response, and romiplostim was re-started, causing acute coronary syndrome and unstable angina pectoris. Therefore, fostamatinib was started, but then was stopped due to melena. Finally, avatrombopag was started at a personalized dose of 20 mg daily in association with dual antiplatelet therapy, and this achieved good platelet count response. CONCLUSIONS This report shows that ITP is characterized by complexity, requiring management of..."

Journal • Acute Coronary Syndrome • Cardiovascular • Cerebral Hemorrhage • Gastrointestinal Disorder • Hematological Disorders • Immune Thrombocytopenic Purpura • Myocardial Ischemia • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

Update on diagnostic and therapeutic strategies for antibody-mediated rejection in kidney transplantation. (PubMed, World J Transplant) - "Ongoing clinical trials evaluating high-dose intravenous immunoglobulin, efgartigimod, fostamatinib, and other novel therapies aim to expand treatment options. These developments highlight the need for well-designed clinical trials to validate biomarkers and therapies and to improve long-term outcomes for kidney transplant recipients."

IO biomarker • Journal • Review • Antibody-mediated Rejection • Inflammation • Transplant Rejection • Transplantation

Porphyromonas gingivalis-derived extracellular vesicles aggravate bone destruction in rheumatoid arthritis by promoting Syk-dependent osteoclastogenesis. (PubMed, Int J Oral Sci) - "R406, a Syk inhibitor, significantly attenuated Pg EV-induced RA osteoclastogenesis and bone destruction...Our findings reveal a new mechanism by which Pg EVs can exacerbate RA via transport through the circulation and the promote Syk-dependent osteoclastogenesis. This study deepens our understanding of the significant pathogenic role of EVs derived from oral bacterial in RA and explores targeted therapeutic strategies by inhibiting the activation of Syk."

Journal • Dental Disorders • Immunology • Inflammatory Arthritis • Periodontitis • Rheumatoid Arthritis • Rheumatology • IL1B • IL6 • SYK • TNFA

JW Pharmaceutical shares real-world prescribing data for immune thrombocytopenia drug Tavalisse (Korea Biomedical Review) - "This information was presented at the 'Asia Bone Marrow Failure Syndrome Symposium (ABFS 2026)'...According to phase 3 clinical trial results involving 34 Japanese patients, the 'stable platelet response rate' was 0 percent in the placebo group and 36 percent in the fostamatinib treatment group. Furthermore, among patients who showed a platelet response, platelet counts tended to rise to 50,000/μL or higher within the initial treatment period (within two weeks), and cases maintaining stable counts for over a year were also reported....An interim analysis of Japan's large-scale post-marketing surveillance (PMS) showed that, among over 600 patients, no serious adverse events, such as thromboembolism, occurred, even among elderly or comorbid patients."

Clinical • Trial status • Immune Thrombocytopenic Purpura

TREM2 regulates neuroinflammation by SYK-dependent inhibition of BTK activation to improve perioperative neurocognitive dysfunction. (PubMed, Int Immunopharmacol) - "Further mechanistic analysis revealed that TREM2 regulates microglial inflammatory balance by enhancing SYK phosphorylation to inhibit BTK activity, an effect antagonized by the SYK inhibitor R406. Subsequent studies indicate this pathway modulates neuroinflammation by regulating NF-κB signaling and NLRP3 inflammasome activation. These findings illustrate that TREM2-SYK-BTK signaling pathway is the key internal mechanism to regulate microglial polarization and neuroinflammation, which provides a new theoretical basis and potential therapeutic strategy for PND."

Journal • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Developmental Disorders • Inflammation • Mood Disorders • IL10 • IL1B • IL4 • IL6 • NLRP3 • SYK

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

FOSTA-ARDS: Fostamatinib for Treating Acute Respiratory Distress Syndrome (ARDS) in Hospitalized Adults (clinicaltrials.gov) - P2 | N=40 | Not yet recruiting | Sponsor: Inova Health Care Services | Trial completion date: Oct 2026 ➔ Oct 2027 | Trial primary completion date: May 2026 ➔ May 2027

Trial completion date • Trial primary completion date • Acute Respiratory Distress Syndrome • Pulmonary Disease • Respiratory Diseases

Thrombopoietin agonists in monotherapy or associated with fostamatinib in severe or refractory thrombocytopenias to immunosuppressants in patients with systemic lupus erythematosus. (PubMed, Med Clin (Barc)) - No abstract available

Journal • Monotherapy • Hematological Disorders • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus • Thrombocytopenia

Rigel expects to report fourth quarter of 2025 gross product sales of $84.5 million. Net product sales are expected to be $65.4 million, compared to $46.5 million for the same period of 2024, including (Rigel Press Release) - "TAVALISSE (fostamatinib disodium hexahydrate) net product sales of $45.6 million...GAVRETO (pralsetinib) net product sales of $10.2 million....REZLIDHIA (olutasidenib) net product sales of $9.6 million compared to $7.4 million for the same period of 2024."

Sales projection • Acute Myelogenous Leukemia • Immune Thrombocytopenic Purpura • Non Small Cell Lung Cancer

Onco360, the nation’s leading independent specialty pharmacy, has been chosen by Rigel Pharmaceuticals as limited distribution specialty pharmacy partner for TAVALISSE (fostamatinib disodium hexahydrate), GAVRETO (pralsetinib), and REZLIDHIA (olutasidenib) (GlobeNewswire)

Licensing / partnership • Acute Myelogenous Leukemia • Immune Thrombocytopenic Purpura • Non Small Cell Lung Cancer • Thyroid Gland Medullary Carcinoma

MiR-31 suppresses lung adenocarcinoma cell proliferation through CDK1 and E2F2-mediated cell cycle arrest. (PubMed, Discov Oncol) - "Collectively, our study establishes miR-31 as a novel biomarker for LUAD proliferative potential and implicates the miR-31/CDK1-E2F2 network as a promising target for disrupting LUAD progression. These findings establish a miRNA-centric precision therapeutic paradigm for effectively suppressing oncogenic proliferation in LUAD."

Journal • Lung Adenocarcinoma • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • CDK1 • E2F2 • EGF • MIR31