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

Repurposing of FDA-Approved Drugs to Disrupt Iron Uptake in Mycobacterium abscessus: Targeting Salicylate Synthase as a Novel Approach. (PubMed, Chem Biol Drug Des) - "Then, in vitro assays on the recombinant enzyme highlighted three competitive inhibitors, namely fostamatinib, esomeprazole, and hydroxystilbamidine. These results confirm the potential of the repurposing approach and pave the way for further experimental validation and optimization of these inhibitors as promising compounds against NTM infections."

FDA event • Journal • Infectious Disease • Nontuberculous Mycobacterial Disease • Respiratory Diseases

Developing a novel aging assessment model to uncover heterogeneity in organ aging and screening of aging-related drugs. (PubMed, Genome Med) - "The 2A model represents a significant advancement in aging assessment by integrating multi-dimensional validation strategies, enhancing its accuracy and applicability. The identification of organ-specific aging pathways and candidate pharmacological interventions provides a theoretical foundation and translational framework for precision anti-aging therapies."

Heterogeneity • Journal • Aesthetic Medicine

Novel second-line treatments for immune thrombocytopenia (PubMed, Rinsho Ketsueki) - "Thrombopoietin receptor agonists, rituximab, and splenectomy have been the main second-line options. However, the Syk inhibitor fostamatinib and the FcRn inhibitor efgartigimod, two novel agents that are expected to improve platelet counts through a new mechanism of action, have recently been added as second-line treatment options in Japan as well. Many new therapeutic agents for ITP such as BTK inhibitors, BAFF receptor inhibitors, and anti-CD38 antibody agents are also under development. It is hoped that these novel treatment options with different effects will improve the management of ITP in the future."

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

EVALUATION OF ARTERIAL THROMBOSIS FREQUENCY AND RISK FACTORS IN PATIENTS WITH IMMUNE THROMBOCYTOPENIA (EHA 2025) - "9 patients were receiving Eltrombopag, 10 patients received corticosteroids and 2 patients received combined therapy. As a result, it should not be forgotten that the arterial thrombosis risk of ITP will increase both without treatment and during the treatment period. Patients should be questioned in detail about their comorbidities, atherosclerotic and thrombotic risk factors. ITP treatment options should be determined together with these risk factors."

Clinical • Atherosclerosis • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Peripheral Arterial Disease • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis • Venous Thromboembolism

Innovative Use of Fostamatinib in Managing Refractory Immune Thrombocytopenia in an HIV-Positive Patient: A Case Report. (PubMed, Clin Case Rep) - "Managing immune thrombocytopenia (ITP) in HIV patients remains challenging due to refractory cases and treatment limitations. This report highlights fostamatinib as a safe, effective, and antiretroviral therapy-compatible option, offering durable platelet stabilization in a difficult-to-treat population."

Journal • Hematological Disorders • Human Immunodeficiency Virus • Immune Thrombocytopenic Purpura • Infectious Disease • Thrombocytopenia • Thrombocytopenic Purpura

Integrative machine learning and structure-based drug repurposing for identifying potent inhibitors of human SYK activity against cancer. (PubMed, Life Sci) - "Despite the investigation of inhibitors of SYK including fostamatinib, entospletinib, cerdulatinib, and TAK-659 for cancer therapy, their lack of specificity and potential off-target effects remain significant concerns...Rifabutin, darunavir, and sildenafil were found as promising SYK inhibitors, showing strong interactions and stable conformations...Our findings underscore the value of computational methods in drug discovery and advocate for further experimental validation of these compounds as SYK-targeted therapies. This study aims to advance the development of more effective and safer treatments for cancers associated with SYK overexpression."

Journal • Oncology • SYK

Decreased PECAM-1 May be a potential pathological factor for vascular injury in T2DM patients. (PubMed, Sci Rep) - "Abnormalities in PECAM-1 transcriptional factors are likely associated with the reduction in plasma PECAM-1 levels, which may be involved in the mechanism of vascular injury in T2DM. Fostamatinib may be a candidate drug for vascular injury in T2DMs."

Journal • Diabetes • Metabolic Disorders • Type 2 Diabetes Mellitus • CD31 • CUX1 • GATA1 • PECAM1 • RELA • TEAD3

REAL-WORLD EFFICACY AND SAFETY OF FOSTAMATINIB FOR ADULT PATIENTS WITH IMMUNE THROMBOCYTOPENIA: ITALIAN MULTICENTER EXPERIENCE. FINAL RESULTS OF THE GIMEMA ITP1122 STUDY (EHA 2025) - "54% of pts had already received more than one TPO-RA and 23% had undergone splenectomy.41 pts (=43%) suffered from cardiovascular comorbidities or other cardiovascular/thrombotic risk factors before starting F, and 19 pts (20%) experienced a total of 23 previous thrombotic events.Twenty-nine (30%) pts received F concomitantly with other anti-ITP medications: corticosteroids (19%), IVIg (4%), romiplostim (3%), eltrombopag (1%), cyclosporin-A (2%), azathioprine (1%).Complete response (CR) and overall response (CR+R) to F, according to IWG criteria, were achieved in 30 (32%) and 69 (73%) pts, respectively. This Italian experience confirms the efficacy and safety of F in a heavily pretreated population of ITP pts. F was used in most cases as fourth or further line of therapy, after failure of TPO-RAs. Our findings indicate that a significant proportion of pts (45% of the overall population) benefit from F after 6 months, suggesting that this treatment may be considered as a..."

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Cardiovascular • Hematological Disorders • Hypertension • Immune Thrombocytopenic Purpura • Neutropenia • Thrombocytopenia • Thrombocytopenic Purpura

REAL WORLD EXPERIENCE OF DISCONTINUATION OF THROMBOPOIETIN RECEPTOR AGONIST (TPO-RA) IN PRIMARY IMMUNE THROMBOCYTOPENIA (ITP) (EHA 2025) - "The combinations were avatrombopag + fostamatinib; eltrombopag + mycophenolate; romiplostim + mycophenolate; romiplostim + mycophenolate + fostamatinib.29 patients were eligible to stop their TPO-RA. TPO-RA therapy can be safely discontinued in selected patients with primary ITP. The majority of patients stopping TPO-RA sustained adequate platelet counts to maintain haemostasis (≥20-30 x 109/L). A trial of TPO discontinuation could be considered in more patients in our centre, but shared-decision making is important in patients where there is clear reason for continued TPO-RA therapy."

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

New Launch of TAVALISSE for Chronic Idiopathic Thrombocytopenic Purpura Treatment in South Korea (Kissei Press Release) - "Kissei Pharmaceutical...announced that its licensing partner, JW Pharmaceutical Corporation...has launched the spleen tyrosine kinase (SYK) inhibitor TAVALISSE (fostamatinib disodium hexahydrate) for the treatment of thrombocytopenia in adult patients with chronic idiopathic thrombocytopenic purpura (ITP) who have had an insufficient response to a previous treatment in South Korea under the brand name 'TAVALISSE' on July 1, 2025."

Launch non-US • Immune Thrombocytopenic Purpura

Proteomics and Phosphoproteomics Characteristics of the Rhesus Macaque Lung Infected With Original SARS-CoV-2, Delta, and Omicron Variants. (PubMed, J Med Virol) - "Kinase prediction analysis suggested that six kinases (DAPK1, DAPK2, DAPK3, PRACK, TTK, and MAP2K2), potentially inhibited by Fostamatinib, were activated across all three variants, and might be potential drug targets, pending further verification. Omicron infection, compared to other mutants, significantly disrupted proteins related to pulmonary structural support, like integrin and collagens, and inhibited efferocytosis, reducing the host's ability to eliminate the pathogen. These findings suggest that innate immune activation and structural disruption may contribute to Omicron-related pathology, potentially being useful for research into the molecular mechanisms underlying lung injury from SARS-CoV-2 variants."

Journal • Infectious Disease • Novel Coronavirus Disease • Respiratory Diseases • DAPK1 • DAPK3 • DDX58 • IFIT2 • MAP2K2 • MX1 • STAT1

RETROSPECTIVE APPLICATION OF THE TH2 SCORE IN A MULTICENTER COHORT OF ITP PATIENTS: A RELIABLE PREDICTIVE TOOL OR A SCORE IN NEED OF REFINEMENT? (EHA 2025) - "SYKi (e.g., fostamatinib, FST) may help by mitigating inflammation... In one case, an increased TH2 predicted a CV event, but the score remained 1 post-event despite TPOra use. In other cases, low platelet counts likely limited score changes during therapy shifts. Except for one, no thrombotic events occurred despite prothrombotic score."

Retrospective data • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Inflammation • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

INDIRECT COMPARISON IN REAL LIFE BETWEEN 2 ORAL TREATMENTS IN ITP USING ARTIFICIAL INTELLIGENCE (EHA 2025) - "The potential of ChatGPT4o, in medicine, not only in hematology (https://codigorojo.tech/home/) is many and varied, as Van de Wyngaert (Haemophilia (2023) doi10.1111/hae.14858) reminds us, stating that "ChatGPT4o can be a valuable tool for patients and healthcare professionals, but it should not replace medical consultations or the experience of a multidisciplinary hemophilia team" Aims: Make an indirect comparison in terms of safety and efficacy between fostamatinib and avatrombopag base don results of two national studies in real life (Table 1). Obviously, this analyses have limitations; in general, statistical analysis performed is simplified and lacks adequate adjustments. Although the simplified analysis can provide a global view of the efficiency of both drugs, we must consider the mentioned limitations for an adequate interpretation of results. This analysis could be indicative for choosing between one or the other drug, considering the patient's..."

Clinical • Cardiovascular • Hematological Disorders • Hemophilia • Rare Diseases • Thrombosis

A PHASE I OPEN LABEL STUDY OF FOSTAMATINIB, A SYK INHIBITOR, IN PATIENTS WITH LOWER-RISK MYELODYSPLASTIC SYNDROME AND CHRONIC MYELOMONOCYTIC LEUKEMIA (EHA 2025) - P1 | "Background: Erythropoiesis-stimulating agents (ESA) and luspatercept are standard of care agents for lower-risk (LR) myelodysplastic syndromes (MDS). Fostamatinib is safe and tolerable in pts with lower-risk MDS and CMML. However, there was no observed clinical benefit in this heavily pretreated population. Further studies are needed to evaluate the role of SYK signaling in earlier stages of MDS and CMML or in clonal hematopoiesis."

Clinical • P1 data • Chronic Myelomonocytic Leukemia • Fatigue • Gastroenterology • Hematological Disorders • Hematological Malignancies • Immune Thrombocytopenic Purpura • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Oncology • Pneumonia • Pulmonary Disease • Respiratory Diseases • Thrombocytopenia • Thrombocytopenic Purpura • SYK

EFFICACY AND SAFETY OF TPO-RA-BASED COMBINATION THERAPIES IN REFRACTORY IMMUNE THROMBOCYTOPENIA: A SINGLE-CENTRE COMPARISON OF 3 TPO-RA BASED TREATMENT STRATEGIES. (EHA 2025) - "All TPO-RA-based combination strategies demonstrated good efficacy in achieving platelet response in refractory ITP, indicating that combining immunomodulatory therapy with TPO-RAs appear to be a promising strategy in patients unresponsive to multiple monotherapies.Further studies with larger sample sizes and longer follow-up are needed to validate these findings, establish clear safety profiles and to identify optimal combination strategies for refractory ITP.Importantly, there remains a critical need to define the specific patient characteristics or biomarkers that can reliably predict the response to guide selection of combination therapy."

Clinical • Combination therapy • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Thrombocytopenia • Thrombocytopenic Purpura

PLATELET REACTIVITY IS VARIABLE IN PATIENTS WITH IMMUNE THROMBOCYTOPENIA AND CAN BE CORRECTED BY TREATMENT (EHA 2025) - "Current treatments, such as corticosteroids, intravenous immunoglobulin (IVIg), rituximab, and thrombopoietin receptor agonists (TPO-RAs) aim to increase platelet number. More recently, inhibitors of the Spleen Tyrosine Kinase (SYKi) and Bruton's Tyrosine Kinase (BTKi) have been either recently licensed (Fostamatinib) or are in clinical trial (Rilzabrutinib)... Some cohorts of ITP patients display either more or less reactive platelets compared to controls, irrespective of platelet count. This signifies a role for aberrant platelet function in the pathogenesis of ITP that may contribute to the heterogeneity of this disease. This study also found more fluctuation in platelet sensitivities in patients, with some moving between reactivity groups over an average of 3-4 months."

Clinical • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura • BTK • SYK

THE SYK INHIBITRO BAY 61-3606 DIHYDROCHLORIDE INDUCES APOPTOSIS OF ACUTE MYELOID LEUKEMIA CELLS BY MODULATING PI3K/AKT?NF/κB PATHWAY (EHA 2025) - "Current SYK inhibitors like Entospletinib, Mivavotinib, and Fostamatinib face limitations due to RAS/RAF/MEK/ERK pathway activation and off-target effects. BAY 61-3606 dihydrochloride significantly inhibits the proliferation of acute myeloid leukemia (AML) cells and induces apoptosis. Mechanistically, BAY 61-3606 dihydrochloride downregulates the expression of anti-apoptotic proteins MCL-1 and BCL-XL, thereby activating the apoptotic pathway in AML cells. Additionally, it inhibits the activation of several key signaling pathways, including P-SYK, PI3K/Akt, ERK, NF/κB, and JAK/STAT."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • BCL2L1 • MCL1 • SYK

How does real-life treatment with fostamatinib compare with the FIT1 and FIT2 studies? Results from the Italian GIMEMA study (EHA 2025) - "Sponsored by Grifols"

Clinical • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Fostamatinib in difficult-to-treat ITP: real-world evidence from the French CARMEN Registry (EHA 2025) - "Sponsored by Grifols"

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

Spanish experience with fostamatinib in ITP: efficacy, safety, and tapering insights from FOSTASUR and FOSTAMES studies (EHA 2025) - "Sponsored by Grifols"

Clinical • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

TSS9009: Exploring real-world evidence of fostamatinib in immune thrombocytopenia (ITP): a comprehensive review of European data (EHA 2025) - "Sponsored by Grifols"

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

A singular case of refractory paraneoplastic heparin independent antiPF4-related thrombotic disorder (ISTH 2025) - "After prednisone withdrawal, in December 2024, Fostamatinib 200 mg daily was started...As hyperfibrinolysis signs appeared, a tranexamic acid course per os was also started. Results After starting pembrolizumab in November 2024, results on coagulopathy and platelet count were not appreciable. In December 2024, under Danaparoid and Fostamatinib (acetylsalicylic acid withdrawn after further platelet count fall in November 2024), he was admitted to Cardiology Unit (different hospital) after pulmonary embolism and myocardial infarction and died. At admission platelet count raise was finally appreciable, but coagulopathy was still present. Table or Figure Upload"

Clinical • Immune Thrombocytopenic Purpura • Immunology • Lung Cancer • Myocardial Infarction • Obesity • Oncology • Pulmonary Embolism • Respiratory Diseases • Solid Tumor • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

Long-term high-dose IVIG for monoclonal gammopathy of thrombotic/thrombocytopenic significance (ISTH 2025) - "Fostamatinib was attempted but discontinued due to side effects. Daratumumab, a CD38-targeting antibody, showed minimal impact on anti-PF4 titers, indicating a potential need for intensified immunosuppression."

Cardiovascular • Hematological Disorders • Ischemic stroke • Monoclonal Gammopathy • Myocardial Infarction • Pulmonary Embolism • Respiratory Diseases • Thrombocytopenia

Platelet antibodies induce distinct platelet and megakaryocyte phenotypes in ITP (ISTH 2025) - "Methods We conducted a comprehensive investigation using multiple approaches: (1) Megakaryocytes differentiated from CD34+ cells were treated with monoclonal antibodies (mAbs) targeting CD41 (GPIIb), CD61 (GPIIIa), CD42a (GPIX), and CD42b (GPIbα); (2) Washed platelets were treated with these mAbs and assessed for various phenotypes including activation, aggregation, procoagulant activity, and apoptosis using flow cytometry; (3) Signaling pathway analysis was performed using specific inhibitors including IV.3 (FcγRIIA-blocking antibody) and Syk inhibitors (Lanraplenib and R406). Notably, the signaling pathways differed between antibody specificities. In the clinical context, 16% of ITP patient sera induced similar patterns of platelet activation, apoptosis, and procoagulant phenotype."

Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura • ANXA5 • CD34 • ITGA2B • ITGB3 • SYK

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 | Initiation date: May 2025 ➔ Jul 2025

Trial initiation date • Acute Respiratory Distress Syndrome • Pulmonary Disease • Respiratory Diseases