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

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

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

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. 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

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

104: Immune Thrombocytopenia: Management Strategies for the Medical Enigma (HOPA 2026) - "In addition to thrombopoietin receptor agonists (TPO-RAs) and the Syk inhibitor, fostamatinib, is the novel BTK inhibitor, rilzabrutinib, which was recently FDA approved for management of chronic ITP using a unique mechanism of action for ITP yet intimately familiar mechanism for most malignant hematology pharmacists.Management of ITP is tailored considering a multitude of patient-specific factors, but also Knowledge or Application Based: Coming soon! Learning Objectives: Coming Soon!"

Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura • SYK

Real-World Effectiveness and Safety of Fostamatinib in Difficult-to-Treat Immune Thrombocytopenia Patients. A Prospective, Multicenter Registry in France. (PubMed, Am J Hematol) - "In conclusion, fostamatinib in combination with TPO-RA should be considered in difficult-to-treat ITP patients. No new safety signal was observed."

Journal • Real-world evidence • Cardiovascular • Hematological Disorders • Hypertension • Immune Thrombocytopenic Purpura • Infectious Disease • Pulmonary Arterial Hypertension • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

The small-molecule Syk inhibitor R788 inhibits hematopoiesis and worsens anemia in sickle cell disease mice. (PubMed, Blood Vessel Thromb Hemost) - "Severe anemia and neutropenia induced by R788 in the sickle mouse model suggests that concomitant use of Syk inhibitors with hydroxyurea in patients with SCD should be approached cautiously. Further research is required to clarify the benefits and risks of selective Syk inhibition in SCD and other hemolytic conditions exhibiting stress hematopoiesis."

Journal • Preclinical • Anemia • Cardiovascular • Genetic Disorders • Hematological Disorders • Neutropenia • Oncology • Sickle Cell Disease • Thrombosis • SYK • TNFA

Combining an immunomodulatory drug with a TPO-RA to treat multirefractory ITP patients: The Spanish ITP Group experience. (PubMed, Br J Haematol) - "The IS/IMs were steroids, purine synthesis inhibitors and fostamatinib. The TPO-RAs were romiplostim, eltrombopag and avatrombopag...Three thromboembolic events and 14 infections that required hospitalization were reported, none fatal. The combination of IS/IMs and TPO-RAs arises as an alternative option of treatment in multirefractory ITP."

Journal • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Thrombocytopenia • Thrombocytopenic Purpura

Feasibility assessment of indirect treatment comparison between off-label rituximab and novel treatments in patients with warm autoimmune hemolytic anemia (ASH 2025) - "Among the clinical trials included, five studied rituximab, in combination with prednisone, prednisolone, ibrutinib, or bortezomib. Three trials studied fostamatinib, while other studied treatments included pegcetacoplan, sovleplenib, parsaclisib, and rilzabrutinib...Future work should consider de novo sources of real-world evidence for rituximab that more closely align with registrational trial characteristics and endpoint definitions. However, aligning timing of endpoint measurements between registration trials and real-world data to match definitions remains challenging."

Clinical • Anemia • Autoimmune Hemolytic Anemia • Hematological Disorders • Immunology

Disease burden and treatment patterns in patients with previously treated primary immune thrombocytopenia (ITP): A retrospective study using administrative claims data in the United States (ASH 2025) - "Following the initial ITP diagnosis, previously treated patients were identified as those who were currently on ITP treatment and who had received one prior therapy comprising an oral corticosteroid, IVIG, IV anti-D, or platelet transfusion, as well as one or more prior therapies comprising TPO-RA, rituximab, fostamatinib, anti-CD-38 monoclonal antibodies, immunosuppressants, danazol, dapsone, bortezomib, vinca alkaloid, or splenectomy. Conclusions Despite receiving at least one currently available first-line and second-line therapy, in this US-based claims study of patients with primary ITP, a significant proportion continued to experience bleeding events and required rescue therapy. This analysis highlights significant unmet needs for novel therapies for the primary ITP patient population."

Retrospective data • Gynecology • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

A case for CD38 targeted therapy in multi-refractory immune thrombocytopenia (ASH 2025) - "Initial treatment with high-dose dexamethasone and IVIG was ineffective. He subsequently received rituximab, eltrombopag and avatrombopag...During weeks 8–14, he was treated with romiplostim, a second steroid pulse, mycophenolate, cyclophosphamide, and cyclosporine without response...While this study is ongoing, we present this case to highlight that daratumumab can be associated with a rapid platelet recovery in multi-refractory ITP, was safe and well tolerated with fostamatinib and danazol, and produced a durable response after a finite treatment course (12 weekly doses). In conclusion, for patients with prolonged, severe ITP unresponsive to conventional treatments, daratumumab offers a rational, mechanism-based intervention. Our case contributes to growing evidence supporting anti-CD38 immunotherapy in ITP and underscores the need for clinical trials to more broadly evaluate other plasma cell directed therapies for the treatment of ITP."

Clinical • Autoimmune Hemolytic Anemia • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Hodgkin Lymphoma • Immune Thrombocytopenic Purpura • Immunology • Lymphoma • Non-Hodgkin’s Lymphoma • Thrombocytopenia • Thrombocytopenic Purpura

Comparative effectiveness of second-line therapies for chronic immune thrombocytopenia: A network meta-analysis of RCTs (ASH 2025) - "The treatment groups were: Avatrombopag (96), Eltrombopag (283), Fostamatinib (101), Rilzabrutinib (133), Romiplostim (2116), and Rozanolixizumab (41)...Rituximab was analyzed indirectly and showed moderate efficacy... This NMA provides a comparative analysis of second-line therapies for chronic ITP, with Rozanolixizumab identified as the most effective treatment. Despite variability in safety profiles, all therapies demonstrated significant clinical benefits, supporting the adoption of personalized treatment strategies."

HEOR • Retrospective data • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura • SYK

Efficacy and safety of spleen tyrosine kinase inhibitors in chronic immune thrombocytopenic purpura: A meta-analysis of randomized controlled trials (ASH 2025) - "SYK inhibitors significantly improve platelet response in chronic ITP without increased thrombotic risk. Fostamatinib and the investigational agent sovleplenib both demonstrated efficacy compared to placebo. These agents may represent promising options for patients with refractory ITP, particularly those at risk of thrombosis from TPO-RAs."

Retrospective data • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis • SYK

Real-world treatment patterns and clinical outcomes of therapies in primary immune thrombocytopenia (ASH 2025) - "Patients received oral CS, IVIG, anti-D, or rituximab as 1L and TPO-RA,rituximab, fostamatinib, splenectomy, or immunosuppressants as 2L treatments. Among 3287 eligible patients diagnosed with ITP who initiated a 1L treatment, median (IQR) ageat diagnosis was 64.6 (49.8-75.4) years, 59.4% were female, and median (IQR) time from ITP diagnosis toindex date was 0.1 (0-6.9) months. This contemporary study from US EHRs confirmed that oral CS were the main 1L therapy forITP and revealed that a substantial proportion of patients either discontinued or recycled oral CS withoutinitiating guideline-recommended 2L treatment. During 1L follow-up, approximately one-thirdexperienced ≥1 BRE and nearly one-quarter used ≥1 rescue therapy. Substantial variations in baselineand follow-up platelet counts were observed."

Clinical • Clinical data • HEOR • Real-world • Real-world evidence • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

Daratumumab can lead to long-lasting remissions in patients with refractory immune thrombocytopenia but with a high incidence of severe infections (ASH 2025) - "In addition, 2 patients hadantibodies against GPIIb-IIIa (acquired Glanzmann syndrome, n=1) and GPVI (n=1) responsible for chronicbleeding symptoms.Median ITP duration was 78 months [range 4-594], and patients had previously received a mediannumber of 8 [range, 3-12] treatment lines for ITP, including corticosteroids (100%), rituximab (100%),intravenous immunoglobulin (95%), thrombopoietin receptor agonists (95%; including eltrombopag[90%] and romiplostim [86%]), mycophenolate mofetil (81%), splenectomy (71%), fostamatinib (48%), andone or more other immunosuppressive drug (43%). However, thiscame at the cost of a high rate of severe infections in this particular group of heavily treated, frequentlysplenectomized, immunocompromised and fragile patients. Careful assessment of benefit/risk balance istherefore warranted before daratumumab administration."

Clinical • Congestive Heart Failure • Genetic Disorders • Heart Failure • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Inflammatory Arthritis • Multiple Myeloma • Neutropenia • Otorhinolaryngology • Pneumonia • Respiratory Diseases • Rheumatoid Arthritis • Rheumatology • Septic Shock • Thrombocytopenia • Thrombocytopenic Purpura

Real-world effectiveness and safety of fostamatinib in difficult-to-treat patients: Results of a three-year registry in France. (ASH 2025) - "Seventy-height percent ofpatients had been exposed to eltrombopag, 74.4% to romiplostim, 86.0% to rituximab, 48.8% tomycopenolate or azathioprine, and 28.0% were splenectomized. Fostamatinib was used in previously very heavily treated patients, with response rates of44.0% at M3 and of 20.0% at M24. Combination therapy with TPO-RA is an option to consider in thispopulation. No new safety signal was observed."

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Hematological Malignancies • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Lymphoma • Musculoskeletal Diseases • Myocardial Infarction • Neutropenia • Pulmonary Arterial Hypertension • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

Efficacy of daratumumab in treatment of relapsed and/or refractory warm autoimmune hemolytic anemia in children (ASH 2025) - "Treatment options for steroiddependent, refractory/relapsed (r/r) wAIHA include rituximab, mycophenolate mofetil (MMF), and otherimmunosuppressants with splenectomy reserved for select cases...She was treated with steroids, rituximab, cyclosporine A (CSA), MMF, bortezomib,abatacept, sirolimus, and fostamatinib with multiple relpases...He was started on MMF anddaratumumab with remarkable recovery of hemolysis within 1 week; remains transfusion independent at1 month follow-up.Patient 4: A Caucasian girl diagnosed at 5 months of age with immunoglobulin G & complementmediated wAIHA initially responsive to prednisone and rituximab; she developed nephrotic syndromeand lupus nephritis by 1 year of age, complicated by renal failure. Multiple relapses of wAIHA weretreated with rituximab, cyclophosphamide, CSA, MMF, sirolimus, eculizumab, raviluzimab, andpegcetacoplan... In our experience daratumumab showed remarkable efficacy in treating r/r wAIHA inchildren. Responses were..."

Clinical • Anemia • Autoimmune Hemolytic Anemia • Glomerulonephritis • Hematological Disorders • Hematological Malignancies • Immune Thrombocytopenic Purpura • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Multiple Myeloma • Nephrology • Renal Disease • Thrombocytopenia • Thrombocytopenic Purpura