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

Fostamatinib and the risk of acute aortic dissection in immune thrombocytopenia. (PubMed, Br J Clin Pharmacol) - "While causality cannot be definitively established, clinicians should be vigilant when prescribing fostamatinib, even in patients without known risk factors for AAD. Further studies are needed to clarify the vascular safety profile of fostamatinib in ITP and other settings."

Journal • Cardiovascular • Genetic Disorders • Hematological Disorders • Hypertension • Immune Thrombocytopenic Purpura • Immunology • Pain • Thrombocytopenia • Thrombocytopenic Purpura • SYK

Beneath the surface in autoimmune hemolytic anemia: pathogenetic networks, therapeutic advancements and open questions. (PubMed, Front Immunol) - "Glucocorticoids remain the standard first-line therapy for warm AIHA; in contrast, CAD/CAS is increasingly managed with agents targeting B-cell function or complement activation, including rituximab and sutimlimab...Emerging therapeutics targeting the classical complement pathway include novel anti-C1s monoclonal antibodies such as riliprubart, which exhibits an extended half-life due to enhanced affinity for the neonatal Fc receptor. Parallel strategies aim to disrupt B-cell receptor (BCR) signaling cascades, employing Bruton tyrosine kinase (BTK) inhibitors such as ibrutinib, spleen tyrosine kinase (SYK) inhibitors such as fostamatinib and sovleplenib, and phosphoinositide 3-kinase (PI3K) inhibitors such as parsaclisib. Collectively, these advances are reshaping the therapeutic landscape of AIHA toward a precision medicine model guided by mechanistic insights into disease biology. In this review, we delineate the evolving immunopathogenesis of AIHAs and examine..."

Journal • Review • Anemia • Autoimmune Hemolytic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Immunology • SYK

Future directions in the management of warm autoimmune hemolytic anemia (PubMed, Rinsho Ketsueki) - "Rituximab and splenectomy are effective second-line treatments; however, there is no consensus on the optimal therapeutic approach for patients with wAIHA that is refractory to second-line therapy...Nipocalimab, a fully human IgG1 monoclonal antibody targeting FcRn, is expected to be effective in wAIHA by reducing the half-life of pathogenic IgG autoantibodies. Fostamatinib, an SyK inhibitor approved for the treatment of chronic immune thrombocytopenia in adults, demonstrated a significantly higher rate of sustained hemoglobin responses compared to placebo in the global, randomized, double-blind, placebo-controlled phase 3 FORWARD trial. The efficacy of Sovleplenib, another SyK inhibitor, was also demonstrated in a randomized, double-blind, placebo-controlled phase 2 trial."

Clinical • Journal • Review • Anemia • Autoimmune Hemolytic Anemia • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura • SYK

Clinical Utility Of Platelet Kinetics Studies In Patients With Idiopathic Thrombocytopenic Purpura (ITP). (EANM 2025) - "Follow-up without drug treatment was the most frequent therapeutic option (61%) followed by treatment with thrombopoietin receptor agonists (TPO-RA) (21.9%), corticosteroids (4.9%), rituximab or fostamatinib (4.9%), splenectomy (4.9%) and immunosuppressants combined with TPO-RA (2.4%). Analysis of the discriminative ability of the B/B 30max index by ROC curve showed an ABC of 0.687 (95% CI 0.501-0.873) with the cut-off point with the best sensitivity/specificity ratio being 1.67. Conclusion Patients with lower hypersplenism indices need less frequent pharmacological treatment with respect to those patients with higher B/B 30 ."

Clinical • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenic Purpura

Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease (clinicaltrials.gov) - P1 | N=25 | Recruiting | Sponsor: National Heart, Lung, and Blood Institute (NHLBI) | Trial primary completion date: Aug 2025 ➔ May 2026

Trial primary completion date • Anemia • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

RL406. Trust me, I'm a Doctor: Medical History's Impact on Contemporary Black American Health Disparities (1 CME) (ASA 2025) - "It will probe into the lasting repercussions of closing a majority of black medical schools as well as the discriminatory practice of medical associations and the persisting effect to present day. SessionLearningObjective1: Gain awareness of medical history as it pertains to the black population.SessionLearningObjective2: Distinguish the ethical impact on contemporary medical standards and practices.SessionLearningObjective3: Determine the prolonged effects of medical history's impact on black patients and physicians."

CME • Anesthesia

NEK9-mediated Wnt signalling repressor TLE3 rewires Docetaxel resistance in cancer cells by inducing pyroptosis. (PubMed, Br J Cancer) - "Our study demonstrated that NEK9 plays an important role in Docetaxel resistance. The novel combination of NEK9 inhibitor Fostamatinib and Docetaxel needs further clinical investigation in advanced OSCC."

Journal • Oncology • Oral Cancer • Pancreatic Cancer • Solid Tumor • Squamous Cell Carcinoma • TLE3

Role of Daratumumab in Refractory Immune Thrombocytopenia: A Systematic Review (SOHO 2025) - "Concomitant therapies included steroids (57%), IVIG (38%), thrombopoietin receptor agonists (21%), fostamatinib (14%), and mycophenolate mofetil (7%). Daratumumab use was associated with platelet recovery, minimal toxicity, and potential for durable response in patients with refractory ITP. However, the available data remain limited and heterogeneous. Further prospective trials are needed to define optimal patient selection, dosing strategies, and long-term safety."

Review • Oncology

SIK2 Drives Pulmonary Fibrosis by Enhancing Fibroblast Glycolysis and Activation. (PubMed, Biomedicines) - "Fibroblast-specific Sik2 KO mice evaluated effects on bleomycin-induced fibrosis. Furthermore, we identified a novel therapeutic application for the clinically approved drug fostamatinib, demonstrating it inhibits fibroblast activation via SIK2 targeting and alleviates PF in mice. Our findings highlight SIK2 as a promising therapeutic target and provide compelling preclinical evidence for two distinct anti-fibrotic strategies with significant potential for future PF treatment."

Journal • Fibrosis • Immunology • Interstitial Lung Disease • Oncology • Pulmonary Disease • Respiratory Diseases

Real-world efficacy and safety of fostamatinib in ITP patients: Italian multicentre experience. GIMEMA ITP1122 study. (PubMed, Br J Haematol) - "Fifty-nine adverse events were reported in 38 patients, mostly grade 1-2, leading to fostamatinib discontinuation in 8% of patients. This real-world data confirm fostamatinib as an effective and safe option in relapsed/refractory ITP."

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

PLK3-Activated Mitochondrial Apoptosis Pathway in the Pathogenesis of Sepsis-Associated Acute Kidney Injury. (PubMed, J Biochem Mol Toxicol) - "Cecal ligation and puncture (CLP) was used to induce sepsis in mice, followed by treatment with the PLK3 antagonist R406 to assess the effects of PLK3 inhibition on inflammatory responses and renal damage...PLK3 is a key driver of sepsis-associated AKI (S-AKI), transcriptionally activated by MAFF, and mediates its effects through the mitochondrial apoptotic pathway. Targeting PLK3 could be an effective strategy to reduce the impact of S-AKI."

Journal • Acute Kidney Injury • Infectious Disease • Inflammation • Nephrology • Renal Disease • Septic Shock • KIM1

Multilevel Analysis Reveals the Critical Role of Cell Death-Related Molecules and Drugs in Temporomandibular Disorders. (PubMed, Int Dent J) - "Based on multilevel evidence of the blood, tissue and cell, we found that cell death-related genes TIE1, IFI16 and GATM were associated with TMD risk and predict potential target drugs such as fostamatinib. This study further elucidates the critical role of cell death-related molecules and drugs in TMD."

Journal • Immunology • Musculoskeletal Diseases • Osteoarthritis • Pain • Rheumatology • IFI16 • TIE1

Rigel Reports Second Quarter 2025 Financial Results and Provides Business Update (Rigel Press Release) - "For the second quarter ended June 30, 2025, total revenues were $101.7 million, consisting of $58.9 million in net product sales and $42.7 million in contract revenues from collaborations. Net product sales grew 76% compared to $33.5 million in the same period of 2024. TAVALISSE net product sales were $40.1 million, growth of 52% compared to $26.4 million in the same period of 2024....For the six months ended June 30, 2025, total revenues were $155.0 million, consisting of $102.5 million in net product sales and $52.5 million in contract revenues from collaborations. Net product sales grew 72% compared to $59.5 million in the same period of 2024. TAVALISSE net product sales were $68.5 million, growth of 44% compared to $47.5 million in the same period of 2024."

Sales • Immune Thrombocytopenic Purpura

TREM-1 as a potential gatekeeper of neuroinflammatory responses: therapeutic validation and mechanistic insights in experimental traumatic brain injury. (PubMed, Front Immunol) - "Spleen tyrosine kinase (SYK) inhibition was achieved using R406, and TREM-1 small interfering RNA (siRNA) and overexpressing plasmids were performed to validate its role in NF-κB signaling and pyroptosis quantified using WB and enzyme-linked immunosorbent assay (ELISA)...These effects are mediated through suppression of TREM-1-dependent NF-κB signaling and pyroptosis. These results highlight TREM-1 as a promising therapeutic target for TBI."

Journal • CNS Disorders • Inflammation • Vascular Neurology • SYK

Transmission of fosD-carrying Methicillin-Resistant Staphylococcus aureus ST764 in East Asia: evidence of human and livestock transmission. (PubMed, J Glob Antimicrob Resist) - "fosD-carrying ST764 MRSA isolates are spreading across borders in East Asia, with clinical isolates from China, Japan, and Thailand, posing an increasing threat to public health. Fosfomycin should be prudently used under supervision in livestock. Furthermore, the cross-border spread of MRSA should be carefully monitored."

Journal • Infectious Disease • FOS

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