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

Surfactant Protein D and Club Cell Secretory Protein as Biomarkers of Disease Severity and Fostamatinib Efficacy in Severe COVID-19. (PubMed, Crit Care Explor) - "SPD and CC16 are valuable biomarkers for assessing lung epithelial injury in severe COVID-19. These biomarkers can serve as prognostic indicators of disease severity and predictive markers of response to fostamatinib, guiding therapeutic strategies to improve outcomes in patients with severe respiratory complications."

Biomarker • Clinical • Journal • Retrospective data • Infectious Disease • Novel Coronavirus Disease

Taurine and a-ketoglutarate induce innate immune memory in macrophages and facilitate the reactivation of latently infected HIV by ß-glucan and MDP. (IMMUNOLOGY 2025) - "Our studies further showed that taurine and AKG enhanced the training effect of previously identified stimuli such as Syk kinase inhibitors (SYKi IV or R406), β-glucan, and Muramyl dipeptide (MDP), the active component of the BCG vaccine...Our studies also showed that combined application of AKG or taurine with either MDP, β-glucan or SYKi IV, can reverse HIV latency in cell line models more efficiently than training with MDP, β-glucan or SYKi IV alone. We also show that combined application of taurine or AKG with MDP, β-glucan, or SYKi IV can efficiently reactivate HIV from PBMCs of people living with HIV."

Late-breaking abstract • Human Immunodeficiency Virus • Immunology • Infectious Disease • Oncology • IL6 • SYK

Molecular alterations identified in a phase 1 study of fostamatinib plus paclitaxel in platinum-resistant ovarian cancer. (ASCO 2025) - P1 | "Clinical Trial Registration Number: NCT03246074 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

P1 data • Platinum resistant • Oncology • Ovarian Cancer • Solid Tumor

Fostamatinib for the Treatment of Refractory ITP: Data from the UK Adult ITP Registry. (BSH 2025) - No abstract available

Clinical • Hematological Disorders

Repurposing of the Syk inhibitor fostamatinib using a machine learning algorithm. (PubMed, Exp Ther Med) - "Taken together, these findings demonstrated that this artificial intelligence model could be effectively used for drug repurposing and repositioning. Furthermore, by identifying its novel mechanism of action, this study confirmed the potential for fostamatinib to expand its indications as a TAM inhibitor."

Journal • Oncology • AXL • MERTK • 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 International, S.A."

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 International, S.A."

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

In Silico Identification of ANKRD22 as a Theragnostic Target for Pancreatic Cancer and Fostamatinib's Therapeutic Potential. (PubMed, Int J Med Sci) - "ANKRD22 exhibited strong binding affinity (ΔG = -7.0 kcal/mol in molecular docking and ∆Gbind = -38.66 ± 6.09 kcal/mol in MDs). Taken together, ANKRD22 could be a promising theragnostic target that might be inhibited by fostamatinib, thereby suppressing PC growth."

Biomarker • Journal • Hepatology • Oncology • Pancreatic Cancer • Solid Tumor • ANKRD22 • E2F1 • KRAS • TP53

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

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 International, S.A."

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

Rilzabrutinib for the Treatment of Immune Thrombocytopenia. (PubMed, Eur J Haematol) - "We also offer a comparative assessment of other BTK inhibitors investigated for ITP and discuss rilzabrutinib's potential positioning relative to existing therapies, including thrombopoietin receptor agonists (TPO-RAs), rituximab, fostamatinib, and immunosuppressants. Preliminary data presented at ASH 2024 from the ongoing Phase 3 LUNA 3 trial, a randomized, double-blind study, further support rilzabrutinib's efficacy and long-term safety. If confirmed, these findings suggest that rilzabrutinib could represent a valuable therapeutic option for patients with refractory ITP, addressing a critical unmet need and potentially redefining treatment paradigms."

Clinical • Journal • Review • Cardiovascular • Hematological Disorders • Hepatology • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

R406 and its structural analogs reduce SNCA/α-synuclein levels via autophagic degradation. (PubMed, Autophagy) - "We further validated the proposed mechanism, in which knockdown of essential gene ATG5 for autophagy formation and using the autophagy inhibitor chloroquine (CQ) blocked the effect of R406. Additionally, R406 also reduced the levels of phosphorylated serine 129 of SNCA (p-S129-SNCA) in SNCA preformed fibrils (PFFs)-induced cellular models and rescued neuron degeneration."

Journal • CNS Disorders • Movement Disorders • Parkinson's Disease • ATG5 • SNCA

Rigel Announces Settlement Agreement Resolving TAVALISSE (fostamatinib disodium hexahydrate) Patent Litigation (PRNewswire) - "Rigel Pharmaceuticals, Inc...announced that it has entered into a settlement agreement with Annora Pharma Private Ltd., Hetero Labs Ltd., and Hetero USA, Inc. (collectively 'Annora') resolving patent litigation related to Rigel's product TAVALISSE (fostamatinib disodium hexahydrate). The litigation resulted from submission by Annora of an Abbreviated New Drug Application to the U.S. Food and Drug Administration (FDA) seeking approval to market a generic version of TAVALISSE in the United States. Under the terms of the settlement agreement, Annora will have a license to sell its generic product in Q2 2032 or earlier under certain circumstances. In accordance with the agreement, the parties terminated all ongoing litigation between Rigel and Annora regarding TAVALISSE patents pending in New Jersey."

Patent • Thrombocytopenia

Identification of m6A-associated autophagy genes in non-alcoholic fatty liver (APASL 2025) - "Ultimately, we found that the TF-mRNA network included FOXP1-GOPC, ATF1- RAB1A and other relationship pairs, and eight therapeutic agents such as R-406 and adavosertib were predicted based on the TBK1. The study investigated the potential molecular mechanisms of m6A related autophagy feature genes (TBK1, RAB1A, and GOPC) in NAFLD through bioinformatic analyses and animal model validation. However, it is critical to note that these findings, although consequential, demonstrate correlations rather than cause- and-effect relationships. As such, more research is required to fully elucidate the underlying mechanisms and validate the clinical relevance of these feature genes."

Hepatology • Metabolic Dysfunction-Associated Steatotic Liver Disease • ATF1 • FOXP1 • TBK1

Physiologically Based Pharmacokinetic Modeling of Phosphate Prodrugs─Case Studies: Fostemsavir and Fostamatinib. (PubMed, Mol Pharm) - "All predictions were within 2-fold of the observed Cmax, AUC, and Tmax, with 81% being within 1.25-fold. The developed modeling strategy can be effectively adopted to increase the confidence of using PBPK modeling to prospectively assess the in vivo performance of phosphate prodrugs and support the development of optimal oral extended-release formulations for this class of drugs."

Journal • PK/PD data

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 2025 ➔ Oct 2026 | Trial primary completion date: Aug 2025 ➔ May 2026

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

Treatment of autoimmune hemolytic anemia: novel and investigational approaches. (PubMed, Minerva Med) - "Exact diagnostic workup is critical for selection of optimal therapy in AIHA. While therapy is becoming increasingly evidence-based, several unmet needs remain. The ideal therapy has not been found for wAIHA or CAD, and evidence-based options are largely lacking for the still rarer subtypes. Patients with AIHA should be considered for clinical trials."

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

Safety and efficacy of rilzabrutinib vs placebo in adults with immune thrombocytopenia: the phase 3 LUNA3 study. (PubMed, Blood) - P3 | "Rilzabrutinib-- in ITP patients who failed multiple previous therapies (splenectomy, TPO-RA, rituximab and/or fostamatinib) resulted in rapid and durable platelet response, reduced rescue medication use and bleeding, significantly improved physical fatigue, and showed favorable safety. NCT04562766; EudraCT 2020-002063-60."

Journal • P3 data • Fatigue • Hematological Disorders • Immune Thrombocytopenic Purpura • Infectious Disease • Pneumonia • Respiratory Diseases • Thrombocytopenia • Thrombocytopenic Purpura

Gene Expression Profiling in Acute Myeloid Leukemia Patient Subgroups With High and Low Sensitivity Toward SYK Inhibitors. (PubMed, Hematol Oncol) - "This study investigates in vitro antiproliferative effects of SYK inhibitors on leukemia cells by analyzing 48 primary AML samples treated with five SYK inhibitors: fostamatinib, entospletinib, cerdulatinib, TAK-659, and RO9021. Gene set enrichment analysis further highlighted that high-sensitivity patient samples were upregulated in pathways associated with oxidative phosphorylation and MYC targets, whereas low-sensitivity patient samples showed enrichment in TGF beta signaling and IL6 JAK STAT3 signaling. These results identify gene expression profile signatures that may predict sensitivity to SYK inhibition and underscore the potential for personalized therapeutic strategies in AML."

Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IL6 • SYK • TGFB1

First Oral PNH Treatment 'Fabhalta' Surpasses 8th Ridge of Reimbursement [Google translation] (Medipana) - "Paroxysmal nocturnal hemoglobinuria (PNH) treatment 'Fabhalta (iptacopan)' by Novartis Korea is one step closer to being listed for health insurance benefits...Thrombocytopenia treatment drugs 'Tavalis (fostamatinib)' by JW Pharmaceutical and 'Doptelet (avatrombopag)' by Handok have also received conditional benefits...On the 6th, the Health Insurance Review & Assessment Service held the 3rd Pharmaceutical Benefit Evaluation Committee in 2025 and disclosed the results of the review of the treatment benefits of the drugs for which decisions were submitted...Accordingly, after the drug price negotiation with the National Health Insurance Service (within 60 days), the upper limit (insurance drug price) will be set, and after the Ministry of Health and Welfare’s Health Policy Deliberation Committee announces the drug price announcement, it will be listed for reimbursement."

Reimbursement • Paroxysmal Nocturnal Hemoglobinuria • Thrombocytopenia

Combination therapy with efgartigimod, fostamatinib, eltrombopag and prednisolone for a patient with refractory immune thrombocytopenia (PubMed, Rinsho Ketsueki) - "Additional treatment with rituximab or TPO-RAs also failed, and thus efgartigimod was added to fostamatinib. Although new agents for ITP (fostamatinib and efgartigimod) have recently been introduced in Japan, there is little clinical experience with combination therapies incorporating these agents. This case suggests that four-drug combination might be beneficial."

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

Fourth Quarter and Full Year 2024 Financial Update (PRNewswire) - "For the fourth quarter ended December 31, 2024, total revenues were $57.6 million, consisting of $31.0 million in TAVALISSE net product sales....TAVALISSE net product sales grew 21% compared to $25.7 million in the same period of 2023....For the full year 2024, total revenues were $179.3 million, consisting of $104.8 million in TAVALISSE net product sales....TAVALISSE net product sales grew 12% compared to $93.7 million in the same period of 2023."

Sales • Immune Thrombocytopenic Purpura • Thrombocytopenia

Novel drug targets for monkeypox: From viral to host proteins. (PubMed, Infect Med (Beijing)) - "In total, we identified 112 drugs targeting MPXV proteins and 371 drugs targeting MMIPs, with fostamatinib, trilostane, and raloxifene being able to inhibit both viral and host proteins. This work provides critical insights into MPXV-human interactions and supports the development of targeted antiviral therapies."

Journal • Infectious Disease

Sustained response off treatment after fostamatinib in refractory immune thrombocytopenia: A series of four case reports. (PubMed, Hematology) - "He was pre-treated with prednisone and rituximab before being in the FIT-2 clinical trial (placebo)...Before fostamatinib, he was unresponsive to high-dose dexamethasone, IVIG, eltrombopag and romiplostim...These cases emphasize that SROT is achievable with fostamatinib in complex ITP cases unresponsive to multiple previous therapies. Additional research is needed to identify the magnitude of the underlying mechanisms, and the clinical factors associated with, and potentially predictive of, SROT."

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

Integrating State-Space Modeling, Parameter Estimation, Deep Learning, and Docking Techniques in Drug Repurposing: A Case Study on COVID-19 Cytokine Storm. (PubMed, J Am Med Inform Assoc) - "This framework provides an efficient method for identifying critical regulatory proteins and advancing drug repurposing, contributing to the rapid development of therapeutic strategies for COVID-19 and future global pandemics."

Journal • Infectious Disease • Inflammation • Novel Coronavirus Disease • Respiratory Diseases