Business Description

Overview We are a biotechnology company dedicated to developing and providing novel therapies that significantly improve the lives of patients with hematologic disorders and cancer. We focus on products that address signaling pathways that are critical to disease mechanisms. TAVALISSE ® (fostamatinib disodium hexahydrate) is our first FDA-approved product and is the only approved oral spleen tyrosine kinase (SYK) inhibitor for the treatment of adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. The product is also commercially available in Europe and the United Kingdom (UK) (as TAVLESSE), and in Japan, the Republic of Korea (Korea), Canada and Israel (as TAVALISSE) for the treatment of chronic ITP in adult patients. REZLIDHIA ® (olutasidenib) is our second FDA-approved product and indicated for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test. W e in-licensed REZLIDHIA from Forma Therapeutics, Inc., now Novo Nordisk (Forma), with exclusive, worldwide rights for its development, manufacturing and commercialization, pursuant to a license and transition services agreement entered in July 2022. GAVRETO ® (pralsetinib) is our third FDA-approved product which we began commercializing in June 2024. GAVRETO is a once daily, small molecule, oral, kinase inhibitor of wild-type rearranged during transfection (RET) and oncogenic RET fusions. GAVRETO is approved by the FDA for the treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test. GAVRETO is also approved under accelerated approval based on overall response rate and duration response rate, for the treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). We acquired the rights to research, develop, manufacture and commercialize GAVRETO in the US from Blueprint Medicines Corporation, now a Sanofi SA company (Blueprint), pursuant to an asset purchase agreement entered in February 2024. Our development pipeline includes R289, our dual interleukin receptor-associated kinases 1 and 4 (IRAK1/4) inhibitor program, which is being advanced in an open-label, Phase 1b study to determine the safety, tolerability and preliminary efficacy of the drug in patients with lower-risk myelodysplastic syndrome (MDS) who are relapsed, refractory or resistant to prior therapies. 4 Table of Contents To expand our evaluation of olutasidenib in other disease areas with IDH1 mutations, we have strategic development collaborations with the University of Texas MD Anderson Cancer Center (MDACC) and with Collaborative Network for Neuro-Oncology Clinical Trials (CONNECT). We also have a receptor-interacting serine/threonine-protein kinase 1 (RIPK1) inhibitor program in clinical development that is being led by our partner Eli Lilly and Company (Lilly). Business Updates TAVALISSE in ITP In 2025, we recognized $158.8 million of TAVALISSE net product sales, a 51.6% increase compared to $104.8 million in 2024. The increase was primarily due to increased quantities sold, lower revenue reserves rate and higher price per bottle. REZLIDHIA in R/R AML with mIDH1 In 2025 , we recognized $31.0 million of REZLIDHIA net product sales, a 34.8% increase compared to $23.0 million in 2024 . The increase was primarily due to increased quantities sold and higher price per bottle, which were partially offset by higher revenue reserves rate. GAVRETO in metastatic RET fusion-positive NSCLC and advanced thyroid cancers In 2025, we recognized $42.1 million of GAVRETO net product sales, compared to $17.1 million in 2024. We began our commercialization of GAVRETO in late June 2024. R289, an Oral IRAK1/4 Inhibitor for Lower-Risk MDS We advanced the development of our dual IRAK1/4 inhibitor program, following evaluation of single and multiple ascending doses of R289 in healthy subjects. The ongoing Phase 1b open-label, multicenter study evaluates the safety, tolerability and preliminary efficacy of R289 in patients with R/R lower-risk MDS.…