RIGEL PHARMACEUTICALS INC 10-Q

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations This discussion and analysis should be read in conjunction with our financial statements and the accompanying notes included in this report and the audited financial statements and accompanying notes included in our Annual Report on Form 10-K for the year ended December 31, 2025 filed with the SEC on March 3, 2026. Our financial results for the three and six months ended June 30, 2026 are not necessarily indicative of results that may occur in future interim periods or for the full fiscal year. This Quarterly Report on Form 10-Q contains statements indicating expectations about future performance and other forward-looking statements within the meaning of Section 27A of the Securities Act and Section 21E of the Securities Exchange Act of 1934, as amended (Exchange Act), and the Private Securities Litigation Reform Act of 1995, that involve risks and uncertainties. We usually use words such as “may,” “will,” “would,” “should,” “could,” “expect,” “plan,” “anticipate,” “might,” “believe,” “estimate,” “predict,” “intend,” or the negative of these terms or similar expressions to identify these forward-looking statements. These statements appear throughout this Quarterly Report on Form 10-Q and are statements regarding our current expectations, beliefs or intent, primarily with respect to our operations and related industry developments. Examples of these statements include, but are not limited to: our business and scientific strategies; risks and uncertainties associated with the commercialization, distribution, marketing, and payment for our products in the US and outside the US; risks that the FDA, EMA, the Medicines and Health Products Regulatory Agency (MHRA) or other regulatory authorities may make adverse decisions regarding our products; the impact of the US federal government shutdowns or agency funding disruptions; the progress of our and our collaborators’ product development programs, including clinical testing, and the timing of results thereof; our corporate collaborations and revenues that may be received from our collaborations and the timing of those potential payments; our expectations with respect to obligations to entities party to commercial or licensing agreements with us and the timing of those obligations; our expectations with respect to timing of recognizing product sales; our expectations with respect to the volume of product sales; the anticipated commercial launch and commercialization of VEPPANU; our expectations with respect to potential patient populations; our expectations with respect to regulatory submissions and approvals; our drug discovery technologies; our research and development expense; protection of our intellectual property and our intention to vigorously enforce our intellectual property rights; the availability and sufficiency of our cash and capital resources and the need for additional capital; our ability to successfully identify and acquire or in-license products or companies; our operations and legal risks; and the effectiveness of our cybersecurity risk management process. You should not place undue reliance on these forward-looking statements. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including as a result of the risks and uncertainties discussed under the heading “Risk Factors” in Item 1A of Part II of this Quarterly Report on Form 10-Q. Any forward-looking statement speaks only as of the date on which it is made, and we undertake no obligation to update any forward-looking statement to reflect events or circumstances after the date on which the statement is made or to reflect the occurrence of unanticipated events, except as required by applicable law. New factors emerge from time to time, and it is not possible for us to predict which factors will arise. In addition, we cannot assess the impact of each factor on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements . 25 Table of Contents 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 SYK inhibitor for the treatment of adult patients with chronic ITP who have had an insufficient response to a previous treatment. The product is also commercially available in Europe and the UK (as TAVLESSE), and in Japan, Korea, Canada and Israel (as TAVALISSE) for the treatment of chronic ITP in adult patients. REZLIDHIA (olutasidenib) is our second FDA-approved product indicated for the treatment of adult patients with R/R AML with a susceptible IDH1 mutation as detected by an FDA-approved test. W e in-licensed REZLIDHIA from 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 RET and oncogenic RET fusions. GAVRETO is approved by the FDA for the treatment of adult patients with metastatic RET fusion-positive NSCLC as detected by an FDA-approved test. GAVRETO is also approved under accelerated approval based on overall response rate and duration response, 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 pursuant to an asset purchase agreement entered in February 2024. VEPPANU (vepdegestrant) is our fourth FDA-approved product which we expect to become commercially available in mid-August 2026. VEPPANU is an oral PROTAC approved by the FDA for the treatment of ER+/HER2-, ESR1- mutated advanced or metastatic breast cancer, as detected by an FDA-authorized test, with disease progression following at least one line of endocrine therapy. We in-licensed VEPPANU pursuant to a license agreement entered in May 2026 with Arvinas and Pfizer. Our development pipeline includes R289, our dual IRAK1/4 inhibitor, 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 MDS who are relapsed, refractory or resistant to prior therapies. To expand our evaluation of olutasidenib in other disease areas with IDH1 mutations, we have strategic development collaborations with MDACC and with CONNECT. Business Updates Commercialized Products TAVALISSE net product sales for the six months ended June 30, 2026 were $84.7 million, an increase of $16.1 million, or 24%, compared to $68.5 million for the same period in 2025. The increase was primarily driven by higher volumes and higher price per bottle, as well as a favorable impact from lower revenue reserves. REZLIDHIA net product sales for the six months ended June 30, 2026 were $17.0 million , an increase of $3.8 million, or 29%, compared to $13.1 million for the same period in 2025 . The increase was primarily driven by higher volumes and higher price per bottle, partially offset by higher revenue reserves. GAVRETO net product sales for the six months ended June 30, 2026 were $20.3 million , a decrease of $0.5 million, or 2%, compared to $20.8 million for the same period in 2025. The decrease was primarily driven by lower volumes and higher revenue reserves, partially offset by higher price per bottle. VEPPANU is our fourth FDA-approved product which we expect to become commercially available in mid-August 2026. We in-licensed VEPPANU pursuant to a license agreement entered in May 2026 with Arvinas and Pfizer (together, the Licensors), which agreement became effective on June 11, 2026 upon the early termination of the waiting period under the HSR Act. Pursuant to the license agreement, the Licensors granted us an exclusive, royalty-bearing license to develop, manufacture and commercialize VEPPANU (vepdegestrant) and vepdegestrant-containing products (the licensed products) worldwide. VEPPANU is approved in the US for the treatment of adults with ER+/HER2-negative, 26 Table of Contents ESR1 -mutated advanced or metastatic breast cancer, as detected by an FDA-authorized test, with disease progression following at least one line of endocrine therapy. Under the license agreement, we agreed to pay the Licensors a license fee of up to $85.0 million, including $70.0 million upfront payment which we paid in June 2026, and up to an additional $15.0 million payable upon the successful completion of certain transition activities. In addition, the Licensors are eligible to receive up to $60.0 million in regulatory milestones upon achievement of specified regulatory approvals, and up to $260.0 million in commercial milestone payments upon achievement of specified net sales thresholds. We are also obligated to pay tiered royalties on annual net sales of licensed products ranging in percentages from the mid-teens to mid-twenties, subject to certain reductions and customary adjustments, and to share a portion of sublicense revenue with the Licensors at tiered rates that decrease based on the timing of execution of the applicable sublicense. Under the license agreement, we will have the sole rights and will be primarily responsible for the development and commercialization of the licensed products worldwide, subject to certain transition activities to be performed by the Licensors. The license agreement includes customary diligence obligations for us to use commercially reasonable efforts to develop and commercialize the licensed products, including to seek regulatory approvals in specified major markets. The license agreement will remain in effect on a product-by-product and country-by-country basis until the expiration of the applicable royalty term for each licensed product in each country, after which the license becomes fully paid-up and perpetual. The license agreement may be terminated by either party under customary circumstances, including for material breach or certain insolvency events. In addition, the Licensors may terminate the license agreement if we cease all material development and commercialization activities for the licensed products for an extended period of time, subject to specified exceptions, or if we breach certain compliance-related obligations relating to anti-corruption and global trade controls. Upon termination of the license agreement prior to its expiration, the licenses granted to us will terminate and, at the Licensors’ request, the parties will negotiate in good faith an exclusive license from us to the Licensors under certain patent rights and know-how controlled by us covering the terminated licensed products. The license agreement contains customary provisions relating to, among other things, intellectual property, indemnification, confidentiality, and representations and warranties. Pursuant to the license agreement, the Licensors will continue to be responsible for specified ongoing development, regulatory, manufacturing and transition activities. We are obligated to reimburse development costs incurred by the Licensors in connection with the ongoing studies, subject to an aggregate funding cap of $40.0 million and specified cumulative annual and quarterly funding caps through 2029. The related costs are recognized as research and development expense as the related services are performed. During the three and six months ended June 30, 2026, we recognized $1.5 million of research and development expense related to these activities. We also agreed to purchase certain drug product inventories from Pfizer pursuant to a manufacturing and supply agreement. 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. This Phase 1b study is expected to enroll approximately 86 patients (up to 36 patients in the dose escalation phase, up to 40 patients in the dose expansion phase, and 10 patients in an exploratory cohort evaluating post- or ineligible erythropoiesis-stimulating agent (ESA), treatment naïve patients). The primary objective of the study is safety, with secondary and exploratory objectives to assess prel iminary efficacy and characterize the pharmacokinetic and pharmacodynamic profile of R289. Enrollment in the dose escalation part of the study was completed in July 2025. In October 2025, we announced enrollment of the first patient in the dose expansion part of the study, where up to 40 patients will be randomized to receive either 500 mg once daily or twice daily to determine the recommended Phase 2 dose for future clinical studies. Enrollment is ongoing and we expect to complete enrollment of the dose expansion phase of the Phase 1b study and select the recommended Phase 2 dose for future clinical studies in the second half of 2026. We anticipate sharing preliminary data from the dose expansion phase of the study by the end of 2026. 27 Table of Contents Olutasidenib in AML, Other Hematologic Cancers and HGG We have a Strategic Collaboration Agreement with MDACC, a comprehensive cancer research, treatment, and prevention center. The collaboration expanded our evaluation of olutasidenib in AML and other hematologic cancers with IDH1 mutations. Under the Strategic Collaboration Agreement, we jointly lead the clinical development efforts with MDACC to evaluate the potential of olutasidenib to treat newly diagnosed and R/R patients with AML and advanced myeloproliferative neoplasms, in combination with other agents. The collaboration also supports the evaluation of olutasidenib as monotherapy in patients with IDH1 mutated clonal cytopenia of undetermined significance (CCUS) and lower-risk MDS, as well as maintenance therapy following hematopoietic stem cell transplant. Further, this collaboration also supports the evaluation of olutasidenib in combination with co-targeted therapies in patients with R/R IDH1 -mutated myeloid malignancies harboring activated signaling pathway mutations. The multi-year strategic development alliance continues to support multiple ongoing clinical studies that are open for enrollment. Under the Strategic Collaboration Agreement, we are obligated to provide study materials and up to $15.0 million in time-based milestone payments as compensation for services to be provided for the studies, over the five-year collaboration term, unless terminated earlier as provided for in the agreement. Through June 30, 2026 , we provided $5.3 million funding to MDACC. We also have a collaboration with CONNECT, an international collaborative network of pediatric cancer centers, to conduct a Phase 2 clinical trial to evaluate olutasidenib in combination with temozolomide in patients with HGG harboring an IDH1 mutation. Under the collaboration, CONNECT will include the olutasidenib treatment arm within CONNECT’s TarGet study, a molecularly guided Phase 2 umbrella clinical trial for HGG. In our sponsored arm, TarGet-D, adolescents and young adult patients (ages 12 to 39 years old) with newly-diagnosed IDH1 -mutation positive HGG will receive maintenance therapy with olutasidenib in combination with temozolomide for the first year after radiotherapy, followed by olutasidenib monotherapy for the second year. Under the collaboration, we will provide CONNECT with funding up to $3.0 million and study material over the four-year collaboration. Enrollment in the Phase 2 TarGet-D study is ongoing. Global Strategic Partnership with Lilly We entered into a global exclusive and strategic collaboration with Lilly in February 2021 to develop and commercialize ocadusertib (previously R552), an investigational, potent and selective RIPK1 inhibitor, for the treatment of non-CNS diseases, and additional RIPK1 inhibitors for the treatment of CNS diseases. RIPK1 is implicated in a broad range of key inflammatory cellular processes and plays a key role in tumor necrosis factor signaling, especially in the induction of pro-inflammatory necroptosis. On April 16, 2026, we received a written notice from Lilly of its decision to terminate the Lilly Agreement, which became effective June 15, 2026. Following termination, the rights previously licensed to Lilly under the Lilly Agreement reverted to us in accordance with the terms of the Lilly Agreement, and we do not expect to receive any future milestone payments or royalties thereunder. Credit Agreement with MidCap On May 5, 2026, we terminated our Credit Agreement with MidCap, which provided for a $60.0 million term loan facility and repaid all outstanding borrowings thereunder, including applicable fees and expenses. Concurrently, we entered into a new Credit Agreement with MidCap, which provides for a revolving credit facility with an initial borrowing capacity of $40.0 million and an option to increase to $60.0 million, subject to customary conditions. Availability under the revolving credit facility is subject to a borrowing base based primarily on eligible accounts receivable and inventory. The revolving credit facility under the new Credit Agreement has a five-year term and bears interest at a rate equal to one-month SOFR, subject to a 2.00% floor, plus an applicable margin of 4.00%. The obligations under the revolving credit facility are secured by a first-priority security interest in substantially all of our assets, including our intellectual property. The revolving credit facility includes customary fees, including an unused commitment fee, administrative fee and prepayment premiums during the initial period. At June 30, 2026, we had an outstanding borrowing of $40.0 million under the revolving credit facility, consisting of an initial draw of $8.0 million following the execution of the new Credit Agreement in May 2026 and an additional draw of $32.0 million in June 2026. In July 2026, we repaid $32.0 million of the outstanding borrowings under the revolving credit facility. Following the repayment, $8.0 million remained outstanding under the facility. 28 Table of Contents Our Product Portfolio The following table summarizes our portfolio: Commercialized Products TAVALISSE/Fostamatinib in ITP TAVALISSE overview Chronic ITP affects an estimated 81,300 adult patients in the US. In patients with ITP, the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. ITP patients can suffer extraordinary bruising, bleeding and fatigue as a result of low platelet counts. Current therapies for ITP include steroids, blood platelet production boosters that imitate thrombopoietin (TPO) and splenectomy. Taken in tablet form, fostamatinib blocks the activation of SYK inside immune cells. ITP is typically characterized by the body producing antibodies that attach to healthy platelets in the blood stream. Immune cells recognize these antibodies and affix to them, which activates the SYK enzyme inside the immune cell, and triggers the destruction of the antibody and the attached platelet. When SYK is inhibited by fostamatinib, it interrupts this immune cell function and allows the platelets to escape destruction. The results of our Phase 2 clinical trial, in which fostamatinib was orally administered to 16 adults with chronic ITP, published in Blood , showed that fostamatinib significantly increased the platelet counts of certain ITP patients, including those who had failed other currently available agents. Our Fostamatinib for Immune Thrombocytopenia (FIT) Phase 3 clinical program had a total of 150 ITP patients who were randomized into two identical multicenter, double-blind, placebo-controlled clinical trials. The patients were diagnosed with persistent or chronic ITP, and had blood platelet counts consistently below 30,000 per microliter of blood. Two-thirds of the subjects received fostamatinib orally at 100 mg twice daily (bid) and the other third received placebo on the same schedule. Subjects were expected to remain on treatment for up to 24 weeks. At week four of treatment, subjects who failed to meet certain platelet counts and met certain tolerability thresholds could have their dosage of fostamatinib (or corresponding placebo) increased to 150 mg bid. The primary efficacy endpoint of this program was a stable platelet response by week 24 with platelet counts at or above 50,000 per microliter of blood for at least four of the final six qualifying blood draws. In August 2016, we announced the results of the first FIT study, reporting that fostamatinib met the study’s primary efficacy endpoint. The study showed that 18% of patients receiving fostamatinib achieved a stable platelet response compared to none receiving a placebo control. In October 2016, we announced the results of the second FIT study, reporting that the response rate (16% in the treatment group, versus 4% in the placebo group) was consistent with the first study, although the difference was not statistically significant. In the ITP double-blind studies, the most 29 Table of Contents commonly reported adverse reactions occurring in at least 5% of patients treated with TAVALISSE were diarrhea, hypertension, nausea, dizziness, increased alanine aminotransferase, increased aspartate aminotransferase, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia. Serious adverse drug reactions occurring in at least 1% of patients treated with TAVALISSE in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis. A post-hoc analysis from our Phase 3 clinical program in adult patients with chronic ITP, highlighting the potential benefit of using TAVALISSE in earlier lines of therapy, was published in the British Journal of Haematology in July 2020. In addition, a report describing the long-term safety and durable efficacy of TAVALISSE with up to five years of treatment was published in Therapeutic Advances in Hematology in 2021. The FDA granted orphan drug designation for fostamatinib for the treatment of ITP in August 2015. TAVALISSE was approved by the FDA in April 2018 for the treatment of ITP in adult patients who have had an insufficient response to a previous treatment, and successfully launched in the US in May 2018. Competitive landscape for TAVALISSE Our industry is intensely competitive and subject to rapid and significant technological change. TAVALISSE is competing with other existing therapies. In addition, a number of companies are pursuing the development of pharmaceuticals that target the same diseases and conditions that we are targeting. For example, there are existing therapies and drug candidates in development for the treatment of ITP that may be alternative therapies to TAVALISSE. Currently, corticosteroids remain the most common first line therapy for ITP, occasionally in conjunction with intravenous immunoglobulin (IVIg) or anti-Rh(D) to help further augment platelet count recovery, particularly in emergency situations. However, it has been estimated that frontline agents lead to durable remissions in only a small percentage of newly diagnosed adults with ITP. Moreover, concerns with steroid-related side effects often restrict therapy to approximately four weeks. As such, many patients progress to persistent or chronic ITP, requiring other forms of therapeutic intervention. In long-term treatment of chronic ITP, patients are often cycled through several therapies over time in order to maintain a sufficient response to the disease. Other approaches to treat ITP are varied in their mechanism of action, and there is no consensus about the sequence of their use. Options include splenectomy, thrombopoietin receptor agonists (TPO-Ras) and various immunosuppressants (such as rituximab). The response rate criteria of the above-mentioned options vary, precluding a comparison of response rates for individual therapies. Even with the above treatment options, a significant number of patients remain severely thrombocytopenic for long durations and are subject to risk of spontaneous or trauma-induced hemorrhage. The addition of fostamatinib to the currently available treatment options could be beneficial because it has a different mechanism of action than any of the therapies that are currently available. Fostamatinib is a potent and relatively selective SYK inhibitor, and its inhibition of Fc receptors and B-cell receptors of signaling pathways make it a potentially broad immunomodulatory agent. The FDA recently approved the product WAYRILZ TM (Sanofi SA) for the treatment of adults with persistent or chronic ITP. Other products in the US that are approved by the FDA to increase platelet production through binding to TPO receptors on megakaryocyte precursors include PROMACTA ® (Novartis International AG), Nplate ® (Amgen, Inc.), DOPTELET ® (Swedish Orphan Biovitrum AB) and ALVAIZ TM ( Teva Pharmaceutical Industries Ltd .). In addition, the availability of generic versions of TPO receptor agonists may further intensify competition and adversely affect the market. In the longer term, we may eventually face competition from potential manufacturers of generic versions of our marketed products, including the proposed generic version of TAVALISSE, which, if approved and allowed to enter the market, could result in significant decreases in the revenue derived from the sale of TAVALISSE and thereby materially harm our business and financial condition. TAVALISSE Commercial activities, including sales and marketing Our marketing and sales efforts are focused on hematologists and hematologist-oncologists in the US who manage chronic adult ITP patients. We have a fully integrated commercial team consisting of sales, marketing, market access, and commercial operations functions. Our sales team promotes our products in the US using customary pharmaceutical company practices. Our products are sold initially through third-party wholesale distribution and specialty pharmacy channels and group purchasing organizations before being ultimately prescribed to patients. To facilitate our commercial activities in the US, we also enter into arrangements with various third parties, including advertising agencies, market research firms and other sales-support-related services as needed. We believe that our commercial team and distribution 30 Table of Contents practices are adequate to ensure that our marketing efforts reach relevant customers and deliver our products to patients in a timely and compliant fashion. Also, to help ensure that all eligible patients in the US have appropriate access to our products, we have established a reimbursement and patient support program called Rigel OneCare ® (ROC). Through ROC, we provide co-pay assistance to qualified, commercially insured patients to help minimize out-of-pocket costs and provide free products to uninsured or under-insured patients who meet certain established clinical and financial eligibility criteria. In addition, ROC is designed to provide reimbursement support, such as information related to prior authorizations, benefits investigations and appeals. We have entered into various license and commercial agreements to commercialize fostamatinib globally as discussed below, but we retain the global rights to fostamatinib outside of the respective territories under such license and commercial agreements. Fostamatinib outside of the US We have a commercialization license agreement with Grifols for exclusive rights to commercialize fostamatinib for human diseases, and non-exclusive rights to develop fostamatinib in their territory. Grifols territory includes European Union (EU), the UK, Turkey, the Middle East, North Africa and Russia (including CIS). In January 2020, the European Commission (EC) granted a centralized MA for fostamatinib (TAVLESSE) valid throughout the EU and which has been grandfathered in the UK, after the departure of the UK from the EU, for the treatment of chronic ITP in adult patients who are refractory to other treatments. Grifols has launched TAVLESSE in the UK and certain countries in EU including Germany, France, Italy and Spain, and continues a phased rollout across the rest of EU. We have an exclusive license and supply agreement with Kissei to develop and commercialize fostamatinib in all current and potential indications in Japan, China, Taiwan and Korea. Kissei is a Japan-based pharmaceutical company addressing patients’ unmet medical needs through its research, development and commercialization efforts, as well as through collaborations with partners. Japan has the third highest prevalence of chronic ITP in the world behind the US and Europe. Kissei was granted orphan drug designation from the Japanese Ministry of Health, Labor and Welfare for R788 (fostamatinib) in chronic ITP in February 2020. In December 2022, Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) approved TAVALISSE for the treatment of persistent and chronic ITP, and in April 2023, Kissei launched TAVALISSE for chronic ITP in Japan. In January 2025, Kissei announced the Korean Ministry of Food and Drug Safety approved TAVALISSE for the treatment of thrombocytopenia in adult patients with chronic ITP who have had an insufficient response to a previous treatment. In July 2025, Kissei announced that its licensing partner, JW Pharmaceutical Corporation, commercially launched TAVALISSE in Korea. We have exclusive commercial and license agreements with Medison to commercialize fostamatinib in all potential indications in Canada and Israel. In November 2020, Health Canada approved the New Drug Submission for TAVALISSE for the treatment of thrombocytopenia in adult patients with chronic ITP who have had an insufficient response to other treatments. In August 2021, Medison Israel received the licenses for registrational approval from the Ministry of Health. TAVALISSE is commercially available in Canada and Israel. We have a commercial license agreement with Knight under which Knight has exclusivity rights to commercialize fostamatinib for approved indications in Latin America, consisting of Mexico, Central and South America, and the Caribbean, and we are responsible for the exclusive manufacture and supply of fostamatinib for all development and commercialization activities under a related supply agreement. Knight submitted MAAs in Mexico, Colombia, Brazil, Argentina and Paraguay for fostamatinib for the treatment of adult patients with chronic ITP who had insufficient response to a previous treatment. In Decemb er 2024, Knight announced that TAVALISSE was approved in Mexico for this indication, and Knight commercially launched TAVALISSE in Mexico in May 2026. In May 2026, Knight announced that Brazil's Agência Nacional de Vigilância Sanitária (ANVISA) approved TAVALISSE for the same indication. REZLIDHIA/Olutasidenib in R/R AML with mIDH1 REZLIDHIA overview m IDH1 alterations are seen in AML, MDS, glioma, chondrosarcoma, and intrahepatic cholangiocarcinoma. It is estimated that there are approximately 1,000 adult patients, a well-identified patient population, with m IDH1 R/R AML, part of an AML market estimated to have an incidence of approximately 22,720 cases in the US in 2026, and an estimated 120,000 cases globally. Despite having approved treatment options for R/R AML patients who are m IDH1 positive, an unmet need remains. 31 Table of Contents Olutasidenib, an oral, small molecule drug designed to selectively bind to and inhibit mIDH1, is a treatment option with durable remissions, reduced QTc potential (referring to a lower observed impact on the heart rate–corrected QT interval on electrocardiogram), and a stable pharmacokinetics profile that enables a consistent drug exposure over time. This targeted agent has the potential to provide therapeutic benefit by reducing 2-hydroxyglutarate levels and restoring normal cellular differentiation. IDH1 is a natural enzyme that is part of the normal metabolism of all cells. When mutated, IDH1 activity can promote blood malignancies and solid tumors. Olutasidenib was granted orphan drug designation by the FDA for the treatment of AML, which provides orphan drug market exclusivity from the time of marketing approval on December 1, 2022. REZLIDHIA is designed to bind to and inhibit mIDH1 to reduce 2-hydroxyglutarate levels and restore normal cellular differentiation of myeloid cells. REZLIDHIA is a novel, non-intensive monotherapy treatment in the R/R AML setting demonstrating a CR+CRh rate of 35% in patients with over 90% of those responders in complete remission. W e in-licensed REZLIDHIA from Forma pursuant to a license and transition services agreement entered in July 2022, with exclusive, worldwide rights for development, manufacturing and commercialization of REZLIDHIA for any uses, including for the treatment of AML and other malignancies. In accordance with the terms of the license and transition services agreement, we paid an upfront fee of $2.0 million, with the potential to pay up to $67.5 million additional payments upon achievement of specified development and regulatory milestones and up to $165.5 million additional payments upon achievement of certain commercial milestones. In 2022, certain milestones were met which entitled Forma to receive a $17.5 million milestone payments. In addition, subject to the terms and conditions of the license and transition services agreement, Forma would be entitled to tiered royalty payments on net sales of licensed products at percentages ranging from low-teens to mid-thirties, as well as certain portions of our sublicensing revenue, subject to certain standard reductions and offsets. In December 2022, the FDA approved REZLIDHIA capsules for the treatment of adult patients with R/R AML with IDH1 mutation as detected by an FDA-approved test, and we began the commercialization of REZLIDHIA and made it available to patients. The recommended dosage of REZLIDHIA is 150 mg taken orally twice daily until disease progression or unacceptable toxicity. The FDA approval was based on the New Drug Application ( NDA) for olutasidenib for the treatment of m IDH1 R/R AML submitted by Forma , that had a PDUFA action date for the application of February 15, 2023. The NDA was supported with a Phase 2 registrational trial for olutasidenib in m IDH1 R/R AML. Interim results from the Phase 2 registrational trial were reported at the American Society of Clinical Oncology (ASCO) annual meeting in June 2021. The interim results of this trial of 153 patients showed that olutasidenib demonstrated a favorable tolerability profile as a monotherapy in patients with R/R AML who have a susceptible m IDH1 , and achieved a complete remission (CR) plus CR with partial hematologic recovery (CRh) rate of 33.3% (30% CR and 3% CRh), the primary efficacy endpoint. While a median duration of CR/CRh was not yet reached, a sensitivity analysis (with a hematopoietic stem cell transplant, as the end of a response) indicated the median duration of CR/CRh was 13.8 months . The overall response rate, comprised CR, CRh, Cri, partial response, and morphologic leukemia-free state (MLFS), was 46% and the median duration of overall response rate (ORR) was 11.7 months. The median overall survival was 10.5 months. For patients with CR/CRh, the median overall survival was not reached, but the estimated 18-month survival was 87%. The most frequently reported treatment emergent adverse events were nausea, constipation, increased white blood cell count, decreased red blood cell count, pyrexia, febrile neutropenia, and fatigue. In January 2023, we announced that REZLIDHIA has been added by the National Comprehensive Cancer Network (NCCN) to the latest NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for AML. REZLIDHIA is now included as a recommended targeted therapy for adult patients with R/R AML with IDH1 mutation. In February 2023, we announced peer-reviewed publication data in Blood Advances , which summarize clinical results from the Phase 2 registrational trial of REZLIDHIA in patients with m IDH1 R/R AML. The published data demonstrate that REZLIDHIA induced durable remissions and transfusion independence with a well-characterized safety profile. The observed efficacy is clinically meaningful and represents a therapeutic advance in this poor prognosis patient population with limited treatment options. REZLIDHIA demonstrated both a high rate of response and an extended median duration of complete response of 28.1 months, which is more than a year longer than what is reported with the standard of care. In June 2023, we announced the second REZLIDHIA publication in Blood Advances , a review article examining the preclinical and clinical development, and the positioning of REZLIDHIA in the m IDH1 AML treatment landscape. The review concluded that the approval of REZLIDHIA is a critical addition to the m IDH1 AML treatment landscape. Further, the available data supports the use of REZLIDHIA as monotherapy in R/R AML patients who have failed intensive chemotherapy or venetoclax plus hypomethylating agents combination therapy. In April 2024, we announced a peer-reviewed publication in Leukemia & Lymphoma on data from an analysis of the Phase 2 study evaluating REZLIDHIA in patients with m IDH1 AML who are R/R to prior venetoclax-based regimens. 32 Table of Contents The findings from these analyses suggest that REZLIDHIA alone or in combination with azacitidine demonstrated potential efficacy in patients with AML following failure of venetoclax combination therapy. In May 2024, we announced the presentation of the registrational Phase 2 trial of REZLIDHIA in R/R m IDH1 AML patients at the 2024 ASCO Annual Meeting and EHA 2024 Hybrid Congress. The data presented reinforces REZLIDHIA’s efficacy in heavily pretreated patients with m IDH1 AML, including those R/R to prior venetoclax. The safety profile was consistent with what was previously reported. Further, REZLIDHIA was generally well tolerated in elderly patients with R/R m IDH1 AML and induced remissions. Despite the challenges of treating elderly patients who had already failed prior AML treatment, the results suggest that elderly patients can benefit from therapy with REZLIDHIA. REZLIDHIA was also effective in achieving remission in patients with m IDH1 R/R AML and served as a bridging strategy towards potentially curative allogeneic transplantation in a substantial subset of these previously ineligible patients. Additionally, REZLIDHIA was well tolerated in a subset of patients with myeloproliferative neoplasms m IDH1 AML, a patient population often associated with poor responses to available therapies. In October 2025, we announced the publication of the final five-year data for REZLIDHIA in patients with R/R m IDH1 AML in the Journal of Hematology and Oncology . The publication reports the final follow-up analysis of the registrational Phase 2 trial, with an additional two years of efficacy and safety data. These five-year data further support the durable responses and manageable safety profile observed with olutasidenib in patients with R/R m IDH1 AML, including those R/R to prior venetoclax. The safety profile remained consistent with what was previously reported, with no new safety signals identified. Competitive landscape for REZLIDHIA There is currently one other product approved in the US for patients with IDH1 mutation. The FDA granted approval to TIBSOVO ® (ivosidenib), an oral targeted IDH1 mutation inhibitor, (i) in July 2018, for adult patients with R/R AML with a susceptible IDH1 mutation, (ii) in May 2019, for newly diagnosed AML with a susceptible IDH1 mutation who are at least 75 years old or who have comorbidities that preclude use of intensive induction chemotherapy, (iii) in August 2021, for adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with an IDH1 mutation as detected by an FDA-approved test, (iv) in May 2022, in combination with azacitidine (azacitidine for injection) for newly diagnosed AML with a susceptible IDH1 mutation, as detected by an FDA-approved test in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy, and (v) in October 2023, for adult patients with R/R MDS with a susceptible IDH1 mutation, as detected by an FDA-approved test. In addition, some clinicians may utilize non-targeted treatments for patients with m IDH1 R/R AML, including use of venetoclax combinations, hypomethylating agents, other chemotherapy regimens, or investigational agents that may be available to them. REZLIDHIA commercial activities, including sales and marketing We believe REZLIDHIA is highly synergistic with our existing hematology-oncology focused commercial and medical affairs infrastructure. Our commercial effort focuses on growing awareness of REZLIDHIA within key institutions, and among targeted HCPs who manage patients with R/R AML with m IDH1 . We retain the global rights, excluding certain geographies as discussed below, to develop and commercialize olutasidenib for all indications, and we are currently exploring other ex-US partnership opportunities. Olutasidenib outside of the US We have a collaboration and license agreement with Kissei for an exclusive right to develop and commercialize olutasidenib in all human diseases in Japan, Korea and Taiwan. Kissei will initially seek approval for REZLIDHIA in Japan for R/R mIDH1 AML and will be responsible for conducting clinical studies as required by the Japanese PMDA. We remain responsible for the manufacture and supply of olutasidenib for all development and commercialization activities and will supply Kissei with bulk drug product for use under the license and supply agreements. We also have a commercial license agreement with Dr. Reddy’s for an exclusive license to develop and commercialize olutasidenib in Dr. Reddy’s territory which includes Latin America, South Africa, India, certain countries in the CIS, Southeast Asia region and North Africa, Australia, and New Zealand. We are responsible for the exclusive manufacture and supply of olutasidenib for all future development and commercialization activities under a supply agreement. 33 Table of Contents Under the license and services agreement with Forma, Forma is entitled to a certain portion of sublicensing revenue, which include, but are not limited to upfront payment, milestone payments and royalties, that we receive from a third party sublicensee. Following the license agreements with Kissei and Dr. Reddy’s as discussed above, Forma is entitled to a portion of the sublicensing revenue we receive from Kissei and Dr. Reddy’s. GAVRETO/Pralsetinib in metastatic RET fusion-positive NSCLC and advanced thyroid cancers GAVRETO overview RET is a receptor tyrosine kinase that activates multiple downstream pathways involved in cell proliferation and survival. RET can be activated by mutation or when a portion of the RET gene that encodes the kinase domain is joined to part of another gene creating a fusion gene that encodes an aberrantly activated RET fusion protein. RET alterations, such as fusions or mutations, drive the growth of multiple tumor types. It is estimated that over 229,000 adult patients in the US will be diagnosed with lung cancer in 2026. NSCLC is the most common type of lung cancer in the US accounting for 77% of all lung cancer diagnoses. RET activating fusions are key disease drivers in NSCLC. RET fusions are implicated in approximately 1-2% of patients with NSCLC. We acquired the rights to research, develop, manufacture and commercialize GAVRETO from Blueprint, pursuant to an Asset Purchase Agreement entered in February 2024. GAVRETO is a once daily, small molecule, oral, kinase inhibitor of wild-type RET and oncogenic RET fusions. Currently, GAVRETO is one of only two approved RET inhibitors on the market for patients. GAVRETO is approved by the FDA for the treatment of adult patients with metastatic RET fusion-positive NSCLC as detected by an FDA-approved test. GAVRETO is also approved 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). This indication was approved by the FDA under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial. Discussions with the FDA regarding confirmatory requirements are ongoing. In June 2024, we announced the completion of the transfer to us of the NDA for GAVRETO, and GAVRETO became commercially available from us in the US by prescription. GAVRETO was co-marketed by Blueprint and Genentech, a member of Roche Group (Roche), to patients in the US since September 2020 pursuant to a collaboration agreement between Blueprint and Roche, which agreement was terminated effective in February 2024. On December 22, 2025, the FDA notified us of the approval of a Prior Approval supplemental NDA for GAVRETO, which updated the US Prescribing Information to add a boxed warning regarding serious infections, including opportunistic infections. We previously communicated this risk information to healthcare providers via a Dear Healthcare Provider letter in October 2024. The FDA also notified us that we have met our postmarketing commitment for GAVRETO from our September 2020 accelerated approval to submit the final report for the AcceleRET-Lung study. In January 2026, initial data from the TAPISTRY study was presented in a poster presentation at the ASCO Gastrointestinal Cancers Symposium. TAPISTRY is a Phase 2, global, open-label, multicohort study evaluating the efficacy and safety of pralsetinib in patients with RET fusion-positive solid tumors, including pancreatic, colorectal, and hepatobiliary cancers. The reported analysis included results from an efficacy-evaluable population of 39 patients, in which pralsetinib demonstrated an overall response rate of 67%. These results support continued evaluation of pralsetinib in RET fusion-positive tumors beyond lung cancer; however, further clinical investigation is ongoing, and there can be no assurance that these results will be confirmed in future studies or lead to regulatory approval for additional indications. In March 2026, we announced the publication of the final data from the registrational trial evaluating pralsetinib for the treatment of patients with metastatic RET fusion-positive NSCLC in the Journal of Clinical Oncology . The final data, which includes an additional 42 months of follow-up from data previously published, further supports the robust, durable responses with a manageable safety profile and no new safety signals identified in treatment-naïve and previously treated patients with RET fusion-positive NSCLC and advanced or metastatic thyroid carcinoma. The NCCN Guidelines for NSCLC recommends pralsetinib as a preferred first-line treatment option for RET + patients, including for patients identified during first-line treatment with systemic therapy. 34 Table of Contents The FDA granted GAVRE TO new chemical entity exclusivity until September 2025 and orphan drug exclusivity until September 2027 with respect to the approval for treatment of adult patients with metastatic RET fusion-positive NSCLC as detected by an FDA-approved test. The FDA also granted GAVRETO two orphan drug exclusivities until December 2027 with respect to FDA approval 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), and for the treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET -mutant medullary thyroid carcinoma who require systemic therapy. Competitive landscape for GAVRETO GAVRETO faces competition for RET fusion-positive NSCLC and advanced thyroid cancers from Lilly’s selpercatinib (Retevmo®) . In addition, other commercially available therapies used to treat RET fusion-positive NSCLC include cabozantanib and platinum-based chemotherapy regimens with or without pembrolizumab, atezolizumab, nivolumab/ipilumumab, cemiplimab or tremelimumab-durvalumab. GAVRETO may also face competition from other drug candidates in development for RET -altered cancers, as well as multi-kinase inhibitors with RET activity being evaluated in clinical trials. GAVRETO commercial activities, including sales and marketing We began our commercialization and started recognizing revenue from product sales of GAVRETO in June 2024. We distribute and market GAVRETO for approved indications in RET fusion-positive NSCLC and advanced thyroid cancers. VEPPANU/Vepdegestrant in ER+/HER2-, ESR1-mutated advanced or metastatic breast cancer VEPPANU overview Breast cancer is the most commonly diagnosed cancer among women in the US. Approximately 70% of breast cancers are ER+. Patients with ER+/HER2- locally advanced or metastatic breast cancer are commonly treated with endocrine-based therapies, often in combination with targeted agents such as CDK4/6 inhibitors. Despite available therapies, disease progression is common, and treatment options following progression remain limited. Published data indicate that up to approximately 50% of patients with ER+/HER2- metastatic breast cancer may develop an ESR1 mutation following exposure to endocrine therapy. As a result, we believe there is a meaningful unmet medical need for patients whose disease progresses after prior endocrine-based treatment. Based on published epidemiology and our assessment of the treatment landscape, we estimate that approximately 20,000 patients in the US with second line- or third-line ER+/HER2-, ESR1 -mutated advanced or metastatic breast cancer may be candidates for therapies targeting this patient population. Our estimates of the addressable patient population are based on published literature, epidemiological data, market research and internal analyses, and actual patient numbers may differ from these estimates. Given the significant prevalence of ER+/HER2- breast cancer and the limited treatment options available following progression on prior endocrine therapies, we believe VEPPANU has the potential to address a substantial patient population and represents a significant commercial opportunity in the US. On May 1, 2026, VEPPANU (vepdegestrant) was approved by the FDA for the treatment of adults with ER+/HER2-, ESR1 -mutated advanced or metastatic breast cancer, as detected by an FDA-authorized test, with disease progression following at least one line of endocrine therapy. FDA approval was granted based on data from VERITAC-2 clinical trial that evaluated vepdegestrant versus fulvestrant in patients with ER+/HER2-, ESR1 -mutated advanced or metastatic breast cancer. In the trial, among patients with an ESR1 mutation (n=270), vepdegestrant demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS), reducing the risk of disease progression or death by 43% compared to fulvestrant. Median PFS was 5.0 months (95% CI: 3.7, 7.4) in the vepdegestrant arm and 2.1 months (95% CI: 1.9, 3.5) in the fulvestrant arm (hazard ratio 0.57 (95% CI: 0.42, 0.77); p-value 0.0001). Overall survival was immature with 16% of deaths in this population at the time of the PFS analysis. The majority of adverse events with vepdegestrant were low grade (Grade 1-2) and the most common (≥10%) adverse reactions, including laboratory abnormalities, were decreased white blood cells, increased AST, musculoskeletal pain, fatigue, decreased hemoglobin, decreased neutrophils, increased ALT, increased alkaline phosphatase, nausea, decreased blood potassium, increased bilirubin, decreased appetite, electrocardiogram QT prolonged, decreased platelets, and constipation. 35 Table of Contents VEPPANU is the first and only FDA-approved oral PROTAC. PROTACs are part of a new class of heterobifunctional protein degraders designed to harness the body's natural machinery to selectively degrade, rather than inhibit, disease-causing proteins. Vepdegestrant was discovered by Arvinas using its PROTAC protein degradation platform and co-developed by Arvinas and Pfizer under a global collaboration. We obtained rights to vepdegestrant pursuant to the license agreement with Arvinas and Pfizer entered in May 2026. Vepdegestrant is covered by issued composition of matter patents listed in the FDA’s Approved Drug Products with Therapeutic Equivalence Evaluations (referred to as the “Orange Book”), with expiration dates beginning in 2037 (2038 for one patent including patent term adjustment), in each case subject to any applicable patent term extensions. We have filed applications for patent term extension for the Orange Book-listed patents, one of which, if granted, is expected to extend patent protection until May 2040. In addition to the composition of matter patents, issued patents and pending patent applications in the US and other jurisdictions cover, among other things, formulations, methods of use, dosing, polymorphs and manufacturing. Additional patent families, if issued, are expected to provide patent protection extending from 2040 through 2046. On May 8, 2026, the NCCN added vepdegestrant to the latest NCCN Guidelines for Breast Cancer. Vepdegestrant was added as a Category 2A treatment option for patients with hormone receptor (HR)-positive/HER2-negative, ESR1 -mutated advanced or metastatic breast cancer after at least one line of endocrine therapy + CDK4/6 inhibitor. Competitive landscape for vepdegestrant VEPPANU competes with currently approved therapies for ER+/HER2- metastatic breast cancer, including endocrine therapies and selective estrogen receptor degraders (SERDs) such as fulvestrant, elacestrant (Menarini Group/Stemline Therapeutics, Inc.), imlunestrant (Lilly), and investigational agents like camizestrant (AstraZeneca) and giredestrant (Roche/Genentech). In addition, several investigational agents, including oral SERDs such as camizestrant and giredestrant, are in late-stage clinical development and may represent future competition. The commercial success of vepdegestrant will depend on a number of factors, including efficacy, safety profile, physician and patient acceptance, pricing, reimbursement, competitive products and market access. VEPPANU commercial activities, including sales and marketing We expect VEPPANU to become commercially available in mid-August 2026. We plan to leverage our existing commercial infrastructure to support its commercialization and will distribute and market VEPPANU for its FDA-approved indication in adults with ER+/HER2-, ESR1 -mutated advanced or metastatic breast cancer. Clinical Stage Programs R289, an Oral IRAK1/4 Inhibitor for Hematology-Oncology, Autoimmune, and Inflammatory Diseases During the second quarter of 2018, we selected R835, a proprietary molecule from our dual IRAK1/4 inhibitor program, for human clinical trials. This investigational candidate is an orally administered, potent and selective inhibitor of IRAK1 and IRAK4 that blocks inflammatory cytokine production in response to toll-like receptor (TLR) and the interleukin-1 receptor (IL-1R) family signaling. TLRs and IL-1Rs play a critical role in the innate immune response and dysregulation of these pathways can lead to a variety of inflammatory conditions. R835 prevents cytokine release in response to TLR and IL-1R activation in vitro, and is active in multiple rodent models of inflammatory disease including psoriasis, arthritis, lupus, multiple sclerosis and gout. Preclinical studies show that R835 inhibits both the IRAK1 and IRAK4 signaling pathways, which play a key role in inflammation and immune responses to tissue damage. Dual inhibition of IRAK1 and IRAK4 allows for more complete suppression of pro-inflammatory cytokine release than inhibition of either one individually. In October 2019, we announced results from a Phase 1 randomized, placebo-controlled, double-blind clinical study evaluating the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of R835 in 91 healthy adult subjects. The Phase 1 study showed that R835 had a favorable safety, tolerability, and PK profile and established proof-of-mechanism by demonstrating the inhibition of inflammatory cytokine production in response to a lipopolysaccharide (LPS) challenge. 36 Table of Contents We advanced the development of our IRAK1/4 inhibitor program, following evaluation of single and multiple ascending doses of R289, a new pro-drug formulation of R835, in healthy subjects. In January 2022, we initiated a Phase 1b open-label, multicenter study to evaluate the safety, tolerability and preliminary efficacy of R289 in patients with R/R lower-risk MDS. In December 2022, we announced the dosing of the first patient. This Phase 1b study is expected to enroll approximately 86 patients (up to 36 patients in the dose escalation phase, up to 40 patients in the dose expansion phase, and 10 patients in an exploratory cohort evaluating post- or ineligible ESA treatment naïve patients). The primary objective of the study is safety, with secondary and exploratory objectives to assess preliminary efficacy and characterize the pharmacokinetic and pharmacodynamic profile of R289. Enrollment in the dose escalation part of the study was completed in July 2025. In October 2025, we announced enrollment of the first patient in the dose expansion part of the study, where up to 40 patients will be randomized to receive either 500 mg once daily or twice daily to determine the recommended Phase 2 dose for future clinical studies. Enrollment is ongoing and we expect to complete enrollment of the dose expansion phase of the Phase 1b study and select the recommended Phase 2 dose for future clinical studies in the second half of 2026. We anticipate sharing preliminary data from the dose expansion phase of the study by the end of 2026. In December 2024, initial data from the dose escalation part of the Phase 1b study was presented at the 66 th ASH Annual Meeting and Exposition. In summary, R289 was generally well tolerated with preliminary signs of efficacy in a heavily pretreated lower-risk MDS patient population, the majority of whom were HTB at baseline. RBC-TI ≥8 weeks was achieved by three patients (1 at 500 mg once daily and 2 at 750 mg once daily); two HTB patients achieved RBC-TI >24 weeks. The median duration of RBC-TI was 29 weeks (range 12.7-51.9 weeks). The three patients that achieved RBC-TI had peak hemoglobin increases exceeding 2.0 g/dL compared to baseline. We also reported that one HTB patient receiving 500 mg once daily achieved a minor HI-E response, with a 64% reduction in RBC transfusions compared to baseline; however, in the July 15, 2025 data cut, we determined that this patient had received blood transfusions that were not captured in the database at the time of the initial data analysis. Accordingly, this patient was subsequently determined to be a non-responder. In December 2025, we presented the updated data from the dose escalation phase of our ongoing Phase 1b study evaluating R289 in patients with R/R lower-risk MDS, at the 67th ASH Annual Meeting and Exposition. R289 continued to be generally well tolerated in a heavily pretreated R/R lower-risk MDS patient population, the majority of whom were HTB at baseline. As of the October 28, 2025 data cutoff, 33 patients were enrolled in the dose escalation part of the study. Patients had a median age of 75. The median number of prior therapies was 3 (range: 1-8); 76% (25) of patients had received luspatercept, 73% (24) had received an ESA, 67% (22) had received an HMA and 6% (2) had received imetelstat. 61% (20) of patients were HTB at baseline. 67% (22) of patients were ring sideroblast negative. Median duration of treatment was 5.5 months (range: 0.9 - 27.7 months). R289 was generally well tolerated across all dose groups in this heavily pre-treated lower-risk MDS patient population, the majority of whom were HTB at baseline. The most common Grade 1/2 treatment-emergent adverse events were diarrhea, constipation and fatigue, increased creatinine, and cough. The most frequent Grade 3/4 treatment-emergent adverse events were anemia, decreased neutrophil count and pneumonia, and increased ALT and AST. One dose limiting toxicity (Grade 4 AST increased/Grade 3 ALT increased) was reported in the 750 mg dose group. For evaluable transfusion dependent patients (≥16 weeks follow up) at dose levels of at least 500 mg once daily and higher, 6/18 (33%) patients achieved durable RBC-TI of >8 weeks (500 mg once daily (1/3), 750 mg once daily (2/5), 500/250 mg once daily (1/5), 500 mg twice daily (2/5)). Duration of RBC-TI was >16 weeks in 4 patients and >24 weeks in 3 patients. The median time to onset of RBC-TI was 1.9 months and the median duration of RBC-TI was 22.9 weeks. Peak hemoglobin increases of 2.9 to 6.1 g/dL compared to baseline occurred in patients achieving RBC-TI. Of the 6 patients achieving RBC-TI, 5 had received an HMA. The FDA granted R289 Orphan Drug designation for the treatment of myelodysplastic syndromes in January 2025 and Fast Track designation for the treatment of previously-treated transfusion dependent lower-risk myelodysplastic syndrome in November 2024. Olutasidenib for mIDH1 AML , Other Hematologic Cancers and HGG We have a strategic collaboration agreement with MDACC entered in December 2023, to expand our evaluation of olutasidenib in AML and other hematologic cancers with IDH1 mutations. Under such collaboration agreement, we will jointly lead the clinical development efforts with MDACC to evaluate the potential of olutasidenib to treat newly diagnosed and R/R patients with AML and advanced myeloproliferative neoplasms, in combination with other agents. The collaboration will also support the evaluation of olutasidenib as monotherapy in patients with IDH1 mutated CCUS and lower-risk MDS, as well as maintenance therapy following hematopoietic stem cell transplant. The multi-year strategic development alliance continues to support multiple ongoing clinical studies that are open for enrollment. The studies that are currently open for enrollment include: (i) a Phase 1b/2 triplet therapy trial of decitabine and venetoclax in combination with olutasidenib in patients with m IDH1 AML; (ii) a Phase 2 study in patients with IDH1 -mutated CCUS, lower-risk MDS and chronic myelomonocytic leukemia (CMML); (iii) a Phase 1/2 study of olutasidenib maintenance therapy following an allogeneic stem cell transplant for patients with IDH1 -mutated myeloid malignancies; and (iv) a P hase 2 37 Table of Contents multi-arm, multi-center, open-label, non-randomized clinical study will evaluate olutasidenib in combination with co-targeted therapies in patients with R/R IDH1 -mutated myeloid malignancies harboring activated signaling pathway mutations. In January 2024, we announced our collaboration with CONNECT to conduct a Phase 2 clinical trial to evaluate olutasidenib in combination with temozolomide in patients with HGG harboring an IDH1 mutation. Under the collaboration, CONNECT will include the olutasidenib treatment arm within CONNECT’s TarGet study, a molecularly guided Phase 2 umbrella clinical trial for HGG. In our sponsored arm, TarGet-D, adolescents and young adult patients (ages 12 to 39 years old) with newly-diagnosed IDH1 -mutation positive HGG will receive maintenance therapy with olutasidenib in combination with temozolomide for the first year after radiotherapy, followed by olutasidenib monotherapy for the second year. The first patient was enrolled in the Phase 2 TarGet-D study in October 2025. Partnered Clinical Programs We have product candidates in clinical development with BerGenBio for the development and commercialization of AXL receptor tyrosine kinase inhibitor, R428 (now referred to as bemcentinib (BGB324)), and with Daiichi to pursue research related to MDM2 inhibitor, DS-3032 (now referred as milademetan). The worldwide rights to milademetan were out-licensed from Daiichi to Rain Oncology Inc., now Pathos AI, Inc. Research, Preclinical and Clinical Development Programs We maintain expertise in drug development to leverage our existing proprietary collection of inhibitors, small-molecule compound libraries and large database of associated phenotypic and biochemical assay results of therapeutic interest. We also maintain leading expertise on specific areas of operation such as inhibition of SYK, IRAK1/4 and RIPK1 kinases and m IDH1 to assist clinical development and commercial affairs, as well as to expand and explore additional opportunities for such inhibitors in the clinical space. Our preclinical operations involve collaborations with clinical research organizations, leading investigators from universities and research organizations around the world, and strategic collaborations with other pharmaceutical companies. We have experts in clinical development to design and implement clinical trials and to analyze the data derived from these trials. The clinical development group possesses expertise in project management and regulatory affairs. We work with external clinical research organizations with expertise in managing clinical trials, drug formulation, and the manufacture of clinical trial supplies to support our clinical development efforts. We also have strategic development collaborations with MDACC and CONNECT to conduct evaluation of olutasidenib in other diseases areas with IDH1 mutations. Commercialization and Sponsored Research and License Agreements See “Note 4 – Sponsored Research and License Agreements” and “Note 5 – In-licensing and Acquisition” to our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q for related discussions. Results of Operations Revenues The following table summarizes revenues for the periods presented (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2026 2025 Change 2026 2025 Change Product sales, net $ 67,015 $ 58,948 $ 8,067 $ 121,938 $ 102,498 $ 19,440 Contract revenues from collaborations and other 11,688 42,737 $ (31,049) 15,583 52,520 $ (36,937) Total revenues $ 78,703 $ 101,685 $ (22,982) $ 137,521 $ 155,018 $ (17,497) 38 Table of Contents The following table summarizes the percentages of revenues from each of our customers who individually accounted for 10% or more of the total net product sales and revenues from collaborations: Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 McKesson Corporation 46 % 31 % 47 % 35 % Cencora, Inc. 21 % 12 % 22 % 15 % Cardinal Health, Inc. * * 10 % * Lilly — % 39% — % 26% ______________________________________________________________________ * Denotes less than 10% Revenue from product sales is related to our sale of our products in the US, net of chargebacks, discounts and fees, government and other rebates and returns. Typically, our first quarter net sales are impacted by the first quarter reimbursement issues such as the resetting of deductibles, co-pays, and other access delays for Medicare patients with plan changes that take effect in January. Consistent with this pattern, our first quarter 2026 net product sales reflected the impact of these seasonal factors, which primarily affected volumes earlier in the year. During the second quarter of 2026, we observed improved trends across our product portfolio compared to the first quarter, as the impact of these seasonal access-related factors moderated. TAVALISSE net product sales for the three and six months ended June 30, 2026 were $47.4 million and $84.7 million, respectively, an increase of 18% and 24%, respectively, compared to $40.1 million and $68.5 million for the three and six months ended June 30, 2025, respectively. The increase was primarily driven by higher volumes and higher price per bottle, as well as a favorable impact from lower revenue reserves. REZLIDHIA net product sales for the three and six months ended June 30, 2026 were $8.9 million and $17.0 million, respectively, an increase of 27% and 29%, respectively, compared to $7.0 million and $13.1 million for the three and six months ended June 30, 2025, respectively. The increase was primarily driven by higher volumes and higher price per bottle, partially offset by higher revenue reserves. GAVRETO net product sales for the three and six months ended June 30, 2026 were $10.7 million and $20.3 million, respectively, a decrease of 10% and 2%, respectively, compared to $11.8 million and $20.8 million for the three and six months ended June 30, 2025, respectively. The decrease was primarily driven by lower volumes and higher revenue reserves, partially offset by higher price per bottle. Contract revenues from collaborations and other for the three and six months ended June 30, 2026 consisted primarily of revenue from Kissei of $5.8 million and $7.6 million, respectively, related to a milestone payment and sublicense revenue recognized in the second quarter of 2026, and the delivery of drug supplies; revenue from Grifols of $5.0 million and $6.8 million, respectively, related to royalties and delivery of drug supplies; and revenue from Medison of $0.3 million and $0.5 million, respectively, related to royalties and delivery of drug supplies. Contract revenues from collaborations and other for the three and six months ended June 30, 2025 primarily consisted of $40.0 million of non-cash revenue related to the release of cost share liability from our collaboration with Lilly. In addition, for the three and six months ended June 30, 2025, contract revenues from collaborations and other includes revenue from Grifols of $2.0 million and $6.7 million, respectively, related to royalties and delivery of drug supplies; revenue from Kissei of $0.4 million and $5.1 million, respectively, related to delivery of drug supplies and a milestone payment in the first quarter of 2025; and revenue from Medison of $0.2 million and $0.6 million, respectively, related to royalties and delivery of drug supplies. We expect that revenue from product sales to increase in the coming quarters due to moving past the seasonal reimbursement issues, continued execution of our commercial strategy, as well as the anticipated commercial launch of VEPPANU in August 2026. However, net product sales may be impacted by the demand from our customers, changes to government and private payor rebate programs, chargeback and discount programs, co-payment assistance programs, and any other rebate and discount programs we may enter in the future. In addition, our future revenues may include payments from our existing and new collaboration partners and government grants. 39 Table of Contents Cost of Product Sales The following table summarizes cost of product sales for the periods presented (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2026 2025 Change 2026 2025 Change Cost of product sales $ 8,525 $ 4,504 $ 4,021 $ 13,131 $ 8,913 $ 4,218 The cost of product sales includes the cost of inventories sold to our customers and to our collaborative partners. Certain inventories sold in prior periods were acquired or produced before FDA approval and therefore did not reflect full production costs, as pre-approval manufacturing costs were previously expensed to research and development. Specifically, we utilized zero-cost API inventory for TAVALISSE, which reduced cost of product sales in those periods. As post-approval inventory is acquired or produced, inventory and cost of product sales reflect the full manufacturing cost. We rely and will continue to rely on certain third parties, including those located outside the US to manufacture our products. The imposition or threat of imposition of trade policies, tariffs (including retaliatory tariffs), taxes and other cross-border operations could result in higher cost of product sales. Cost of product sales may also include reserves for potential excess, dated or obsolete inventories, estimated based upon assumptions about future demand and market conditions as well as product shelf lives. Cost of product sales also includes amortization of intangible assets and royalties. The increase in cost of product sales for the three and six months ended June 30, 2026, compared to the same period in 2025, was primarily driven by increase in product costs of $2.6 million and $2.4 million, respectively, primarily due to the timing of drug supply deliveries to collaboration partners and higher product costs associated with increased product sales, as well as higher royalties of $1.1 million, and $1.6 million, respectively. Research and Development Expense The following table summarizes research and development expense for the periods presented (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2026 2025 Change 2026 2025 Change Research and development expense $ 13,961 $ 6,821 $ 7,140 $ 25,637 $ 15,257 $ 10,380 Stock-based compensation expense included in research and development expense $ 1,908 $ 517 $ 1,391 $ 2,349 $ 1,389 $ 960 The increase in research and development expense in the three and six months ended June 30, 2026 compared to the same period in 2025, was primarily due to increased clinical trial related expenses of $2.7 million and $5.2 million, respectively, driven by the timing of clinical development programs, including the progress of activities on our ongoing IRAK1/4 inhibitor program; increased personnel related costs of $2.4 million and $2.2 million, respectively, primarily due to higher stock-based compensation expense and other employee-related costs; $1.5 million for both the three and six months ended June 30, 2026 of research and development expense related to reimbursable development costs under our license agreement with Arvinas and Pfizer; and increases of $0.6 million and $1.4 million, respectively, in other various research and development expenses. Our research and development expenditures include costs related to preclinical and clinical trials, scientific personnel, supplies, equipment, consultants, sponsored research, stock-based compensation, and allocated facility costs. W e expect to continue to incur significant research and development expense as we continue our activities in our clinical studies including IRAK1/4 inhibitor program; our collaborative partnerships with MDACC and CONNECT to conduct evaluation of olutasidenib in other disease areas with IDH1 mutations; and any other clinical programs we may pursue in the future. We do not track fully burdened research and development costs separately for each of our drug candidates. Our research team is focused on identifying and evaluating product candidates in our focused range of therapeutic indications that can be developed into small molecule therapeutics in our own proprietary programs or with potential collaborative partners. “Research” expenses relate primarily to personnel expenses, lab supplies, fees to third-party research consultants and compounds. Our development group leads the implementation of our clinical and regulatory strategies and prioritizes disease indications in which our compounds may be studied in clinical trials. “Development” expenses relate primarily to 40 Table of Contents clinical trials, personnel expenses, costs related to our regulatory filings, lab supplies and fees to third-party research consultants. “Other” expenses primarily consist of allocated facilities costs and allocated stock-based compensation expense relating to personnel in research and development groups. In addition to reviewing the three categories of research and development expense described in the preceding paragraph, we principally consider qualitative factors in making decisions regarding our research and development programs, which include enrollment in clinical trials and the results thereof, the clinical and commercial potential for our drug candidates and competitive dynamics. We also make our research and development decisions in the context of our overall business strategy, which includes the evaluation of potential collaborations for the development of our drug candidates. Preclinical testing and clinical development are long, expensive and uncertain processes, and we cannot reliably predict the timing of such clinical trial activities. In general, biopharmaceutical development involves a series of steps, beginning with identification of a potential target and including, among others, proof of concept in animals and Phase 1, 2 and 3 clinical trials in humans. Significant delays in clinical testing could materially impact our product development costs and timing of completion of the clinical trials. We do not know whether planned clinical trials will begin on time, will need to be halted or revamped or will be completed on schedule, or at all. Clinical trials can be delayed for a variety of reasons, including delays in obtaining regulatory approval to commence a trial, delays from scale up, delays in reaching agreement on acceptable clinical trial agreement terms with prospective clinical sites, delays in obtaining institutional review board approval to conduct a clinical trial at a prospective clinical site or delays in recruiting subjects to participate in a clinical trial. We currently do not have reliable estimates of total costs for a particular drug candidate to reach the market. Our potential products are subject to a lengthy and uncertain regulatory process that may involve unanticipated additional clinical trials and may not result in receipt of the necessary regulatory approvals. Failure to receive the necessary regulatory approvals would prevent us from commercializing the product candidates affected. In addition, clinical trials of our potential products may fail to demonstrate safety and efficacy, which could prevent or significantly delay regulatory approval. The following table presents our total research and development expense by category (in thousands). Three Months Ended June 30, Six Months Ended June 30, From January 1, 2007* to 2026 2025 2026 2025 June 30, 2026 Categories: Research $ — $ 48 $ 30 $ 781 $ 271,148 Development 11,986 6,162 23,095 12,877 635,637 Other 1,975 611 2,512 1,599 284,308 $ 13,961 $ 6,821 $ 25,637 $ 15,257 $ 1,191,093 ______________________________________________________________ * We started tracking research and development expense by category on January 1, 2007. “Other” expenses in the three and six months ended June 30, 2026 consisted of allocated facilities costs of $0.1 million and $0.2 million, respectively, and stock-based compensation expense of $1.9 million and $2.3 million , respectively. “Other” expenses in the three and six months ended June 30, 2025 consisted of allocated facilities costs of $0.1 million and $0.2 million, respectively, and stock-based compensation expense of $0.5 million and $1.4 million , respectively. 41 Table of Contents Selling, General and Administrative Expense The following table summarizes selling, general and administrative expense for the periods presented (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2026 2025 Change 2026 2025 Change Selling, general and administrative expense $ 32,642 $ 29,257 $ 3,385 $ 63,293 $ 56,972 $ 6,321 Stock-based compensation expense included in selling, general and administrative expense $ 4,936 $ 2,759 $ 2,177 $ 7,951 $ 5,211 $ 2,740 The increase in selling, general and administrative expense in the three and six months ended June 30, 2026 compared to the same period in 2025 was primarily due to increased personnel-related costs of $2.6 million and $4.2 million, respectively, primarily driven by higher stock-based compensation expense and other employee-related costs; increases in third-party costs and other selling , general and administrative expenses of $0.8 million and $0.6 million, respectively, primarily related to timing of spending on professional services and other general corporate support activities; and higher commercial-related expenses of $1.5 million for the six months ended June 30, 2026, primarily due to the timing of commercial activities. Commercial-related expenses were relatively flat for the three months ended June 30, 2026 compared to the same period in 2025. We expect to incur significant selling, general and administrative expenses, and expect our commercial related expenses to increase as we continue to expand our commercial activities. Interest Income and Interest Expense The following table summarizes interest income and expense for the periods presented (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2026 2025 Change 2026 2025 Change Interest income $ 789 $ 753 $ 36 $ 1,994 $ 1,344 $ 650 Interest expense and other $ (779) $ (1,874) $ (1,095) $ (2,212) $ (3,727) $ (1,515) Interest income reflects returns earned on our cash and investment holdings, while interest expense relates to borrowing costs on our outstanding loans with MidCap. The increase in interest income for the three and six months ended June 30, 2026, compared to the same period in 2025, was primarily driven by higher average investment balances, partially offset by lower interest rates. The decrease in interest expense for the three and six months ended June 30, 2026, compared to the same period in 2025, was primarily due to reduced outstanding debt balance and impact of lower interest associated with the new revolving credit facility, partially offset by a loss on extinguishment attributable to the extinguished portion of the prior term loan. Income Taxes The following table summarizes income tax for the periods presented (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2026 2025 Change 2026 2025 Change Provision for income taxes $ 6,295 $ 369 $ 5,926 $ 9,298 $ 434 $ 8,864 The quarterly provision for income taxes is determined by applying the estimated annual effective tax rate to the year-to-date pre-tax income, adjusted for any discrete items. The estimated annual effective tax rate is updated at the end of each reporting period. The provision for income taxes for the three and six months ended June 30, 2026 primarily consisted of federal income tax expense of $5.3 million and $7.7 million, respectively, and estimated state income taxes of $1.0 million and $1.6 million, respectively . Prior to the fourth quarter of 2025, we maintained a full valuation allowance against our deferred tax assets. Although we do not expect to incur federal cash income taxes due to sufficient NOL and research and 42 Table of Contents development credit carryforwards, we recognized federal income tax expense based on the estimated impact of utilizing the deferred tax assets associated with such carryforwards. The total tax expense differs from the amount computed at the federal statutory rate primarily due to certain non-deductible expenses and state income taxes. For the three and six months ended June 30, 2025, the provision for income taxes primarily consisted of estimated state income taxes. The tax expense differs from the amount computed at the federal statutory rate primarily due to the impact of the valuation allowance and state taxes. Critical Accounting Policies and Use of Estimates Our discussion and analysis of our financial condition and results of operations is based upon our financial statements, which have been prepared in accordance with US GAAP. The preparation of these financial statements requires us to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. We base our estimates on historical experience and on various other assumptions that we believe to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. Our critical accounting estimates and significant accounting policies are described in “Note 1 – Description of Business and Summary of Significant Accounting Policies” to our “Notes to Financial Statements” contained in Part II, Item 8, “Financial Statements and Supplementary Data” of our Annual Report on Form 10-K for the year ended December 31, 2025. There have been no material changes to the accounting policies described in our Annual Report on Form 10-K for the year ended December 31, 2025. Recent Accounting Pronouncements See related discussions of recently issued accounting standards in “Note 1 – Organization and Summary of Significant Accounting Policies” to our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q. We continue to evaluate accounting standards that were recently issued but not yet adopted, as applicable. Liquidity and Capital Resources Liquidity At June 30, 2026 and December 31, 2025, we had approximately $95.3 million and $155.0 million, respectively, in cash, cash equivalents and short-term investments. We continue to maintain investment portfolios primarily in money market funds, US treasury bills, government-sponsored enterprise securities, corporate bonds and commercial paper. Cash in excess of immediate requirements is invested with a focus on liquidity and capital preservation. We view our investments portfolio as available-for-sale and are available for use in current operations. Wherever possible, we seek to minimize the potential effects of concentration and degrees of risk. We continue to monitor the impact of the changes in the conditions of the credit and financial markets on our investment portfolio and assess if future changes in our investment strategy are necessary. The following table summarizes our cash flow activity for the periods presented (in thousands): Six Months Ended June 30, 2026 2025 Net cash provided by (used in): Operating activities $ 33,378 $ 29,644 Investing activities 9,478 (33,828) Financing activities (22,620) 902 Net increase (decrease) in cash, cash equivalents and restricted cash $ 20,236 $ (3,282) 43 Table of Contents Net cash provided by operating activities for the six months ended June 30, 2026 reflected net income, adjusted for non-cash items, partially offset by net cash outflows from changes in working capital. These outflows were primarily driven by increases in accounts receivable due to the timing of collections, higher inventory levels resulting from production build-up, and decreases in liabilities due to the timing of payments, partially offset by decreases in prepaid and other current assets resulting from the transfer of advance payments to contract manufacturers to inventory. In comparison, net cash provided by operating activities for the six months ended June 30, 2025 reflected net income, adjusted for non-cash items, partially offset by net cash outflows from changes in working capital. These outflows were primarily driven by increases in inventory due to production build-up and increases in prepaid and other current assets due to advance payments to contract manufacturers, partially offset by increases in liabilities due to the timing of payments. Net cash provided by investing activities for the six months ended June 30, 2026 consisted of net maturities and sales of short-term investments of $79.8 million, partially offset by payments for acquisition of intangible assets of $70.3 million. In comparison, net cash used in investing activities for the six months ended June 30, 2025 comprised net purchases of short-term investments of $33.8 million . Net cash used in financing activities for the six months ended June 30, 2026 consisted primarily of the repayment of term loan and related fees of $55.3 million, payments of $4.3 million for repurchases of common stock in connection with employee tax withholding on RSU vesting, and $5.0 million payment of closing purchase price related to asset acquisition with Blueprint. These outflows were partially offset by the $39.7 million net proceeds from revolving facility and $2.2 million of net proceeds from the issuance of common stock under equity plans. In comparison, n et cash provided by financing activities for the six months ended June 30, 2025 consisted of $0.9 million of net proceeds from issuance of common stock under equity plans . We believe that our existing capital resources will be sufficient to support our current and projected funding requirements, including the continued commercialization of our products, through at least the next 12 months from this Form 10-Q filing date. This assessment includes our anticipated funding requirements related to VEPPANU, including planned commercialization and launch activities, transition activities under our license agreement with Arvinas and Pfizer, expected inventory purchases and our obligation to contribute toward certain ongoing development activities. We have based this estimate on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we currently expect. Because of the numerous risks and uncertainties associated with commercializing a product, the development of our product candidates and other research and development activities, we are unable to estimate with certainty our future product revenues, our revenues from our current and future collaborative partners, the amounts of increased capital outlays and operating expenditures associated with our current and anticipated clinical trials and other research and development activities. Capital Resources We finance our operations primarily through sales of our products, and contract payments under our collaboration agreements, as well as through equity securities and debt financing. Under our existing collaboration agreements entered in the ordinary course of business, we have received, and may in the future receive upfront cash payments, payments contingent upon the achievement of specified events by our partners, and royalties on net sales of products sold by such partners under the agreements. The total potential future contingent payments due to us under these agreements are approximately $652.1 million. This amount excludes terminated programs including the termination of the Lilly Agreement, and assumes achievement of all applicable milestones under the existing agreements. The estimate excludes any potential royalties that may be payable to us if our partners successfully commercialize licensed products. Potential future milestone payments under these agreements are contingent solely upon our partners’ future efforts and the achievement of specified development, regulatory, and commercial milestones. See further discussion in “Note 4 – Sponsored Research and License Agreements” to our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q. We have an Open Market Sale Agreement with Jefferies LLC (Jefferies), as a sole agent, entered on August 4, 2020, and amended and restated on August 2, 2024. Pursuant to such Open Market Sale Agreement, we may sell from time to time, through Jefferies, shares of our common stock in sales deemed to be “at-the-market offerings” as defined in Rule 415 under the Securities Act, subject to conditions specified in the Open Market Sale Agreement, including maintaining an effective registration statement covering the sale of shares under the Open Market Sale Agreement. We have an active Registration Statement filed with the SEC, which registered, among other securities, a base prospectus which covers the offering, issuance, and sale by us of up to $250.0 million in the aggregate of the securities identified from time to time in 44 Table of Contents one or more offerings, which include the $100.0 million of shares of our common stock that may be offered, issued and sold under the Open Market Sale Agreement. At June 30, 2026 , we have not sold any shares of common stock under such Open Market Sale Agreement. We had a Credit Agreement with MidCap that provided for $60.0 million term loan credit facility. On May 5, 2026, we terminated the term loan credit facility and repaid all outstanding borrowings therein, including the applicable prepayment premiums, accrued interest and final payment fees, using cash on hand. Concurrently, we entered into a new Credit Agreement with MidCap, which provides for a revolving credit facility with a maximum borrowing capacity of $40.0 million, with an option to increase to $60.0 million, subject to customary conditions. Availability under the revolving credit facility is subject to a borrowing base based primarily on eligible accounts receivable and inventory. While the revolving credit facility enhances our financial flexibility to support operations and working capital needs, our liquidity is dependent on the level of borrowing base availability. At June 30, 2026, we had an outstanding borrowing of $40.0 million under the revolving credit facility, consisting of an initial draw of $8.0 million following the execution of the new Credit Agreement in May 2026 and an additional draw of $32.0 million in June 2026. In July, 2026, we repaid $32.0 million of the outstanding borrowings under the revolving credit facility. Following the repayment, $8.0 million remained outstanding under the facility. We may borrow or repay amounts under the facility from time to time based on our operating needs, working capital requirements, cash management objectives and overall liquidity planning. Accordingly, our outstanding borrowings and related cash balances may vary during a reporting period, and period-end balances may not be indicative of balances at other times during the period. We may from time to time consider raising additional funds through public and/or private offerings of equity securities, debt financings, or from other sources, in order to fund ongoing operations, to strengthen our long-term financial profile or to pursue opportunistic corporate development activities. However, certain external factors such as global geopolitical tensions, political and economic legislations, lingering economic effects of the global pandemic, and other factors may continue to rapidly evolve which could significantly disrupt the global financial markets. Our ability to raise additional funds may be adversely impacted by potential worsening of global economic conditions and volatility in the credit and financial markets in the US and worldwide. We could experience an inability to access additional funds, which could in the future negatively affect our capacity for certain corporate development transactions or our ability to make important, opportunistic investments. To the extent that we raise additional funds through the sale of equity, our shareholders’ ownership interest may experience substantial dilution . Our current credit facility with MidCap and any debt financing that we can obtain in the future may involve operating covenants that may restrict our business. To the extent that we raise additional funds through collaboration and licensing arrangements, we may be required to relinquish some of our rights to our technologies or product candidates or grant licenses on terms that are not favorable to us. Our future funding requirements will depend upon many factors, including, but not limited to: • the ongoing costs to commercialize our products, or any other future product candidates, if any such candidate receives regulatory approval for commercial sale; • our ability to generate expected revenue from our commercialization efforts; • the progress and success of our clinical trials and preclinical activities (including studies and manufacture of materials) of our product candidates conducted by us; • our ability to secure and maintain our patent protection and regulatory rights; • our ability to meet operating covenants under our current and future credit facilities, if any; • our ability to enter into partnering opportunities across our pipeline within and outside the US; • the costs and timing of regulatory filings and approvals by us and our collaborators; • the progress of research and development programs carried out by us and our collaborative partners; • any changes in the breadth of our research and development programs; • the ability to achieve the events identified in our collaborative agreements that may trigger payments to us from our collaboration partners; • our ability to acquire or license other technologies or compounds that we may seek to pursue; • our ability to manage our growth; • competing technological and market developments; 45 Table of Contents • the costs and timing of obtaining, enforcing and defending our patent and other intellectual property rights, including regulatory rights such as regulatory data exclusivities; • expenses associated with any unforeseen litigation, including any arbitration and securities class action lawsuits; and • pressures on and uncertainty surrounding the US federal government policies, and potential changes in budgetary priorities. Insufficient funds may require us to delay, scale back or eliminate some or all of our commercial efforts and/or research or development programs, to lose rights under existing licenses or to relinquish greater or all rights to product candidates at an earlier stage of development or on less favorable terms than we would otherwise choose or may adversely affect our ability to operate as a going concern. Material Cash Requirements We conduct our commercial activities and research and development programs internally and with third parties that include, among others, arrangements with vendors, consultants, contract research organizations (CROs) and universities. Our contract arrangements with these third parties are generally cancellable on reasonable notice, and our obligations under such arrangements are generally based on services performed. We have agreements with certain clinical research organizations to conduct our clinical trials including our strategic development collaborations with MDACC and CONNECT. The timing of payments for any amounts owed under the respective agreements depends on various factors including, but not limited to, patient enrollment and other progress of the clinical trials. We can terminate these agreements at any time, and if terminated, we would not be liable for the full amount of the respective agreements. Instead, we will be liable for services provided through the termination date plus certain cancellation charges, if any, as defined in each of the respective agreements. In addition, these agreements may, from time to time, be subjected to amendments as a result of any change orders executed by the parties. We expect to continue entering into contracts in the normal course of business with various third parties to support our commercial activities and research and development programs. In the ordinary course of business, we enter into agreements with contract manufacturers to manufacture our inventory products. These agreements generally include termination provisions that may require us to pay cancellation fees, which vary depending on the timing of termination and may equal up to the full value of the work order. In October 2024, we entered into an agreement with a third-party contract manufacturer to manufacture TAVALISSE, with deliveries expected from 2026 through 2029. At June 30, 2026, the contractual obligation not included in our financial statements related to an agreement that may potentially be subjected to cancellation fees were approximately $16.3 million, of which, $2.5 million is expected to be due in the remainder of 2026, and $9.5 million is expected to be due in 2027 and 2028. At June 30, 2026, we have not incurred any cancellation fees under our agreements with contract manufacturers. In addition, as contemplated by the license agreement with Arvinas and Pfizer, in July 2026, we entered into a manufacturing and supply agreement with Pfizer for the commercial manufacture and supply of VEPPANU. The agreement includes certain minimum purchase obligations on a take-or-pay basis, with expected purchases from 2026 through 2030. If we do not satisfy the applicable minimum purchase commitments within the required time periods, we may be required to make payments to Pfizer with respect to those commitments, as provided in the agreement. The estimated contractual obligation not included in our financial statements related to this agreement was approximately $26.8 million. Of this amount, approximately $4.6 million is expected to be due in the remainder of 2026 and $11.0 million is expected to be due in 2027 and 2028, with the remaining amount expected to be due thereafter through 2030. As discussed in “Note 5 – In-licensing and Acquisition” to our “Notes to Condensed Financial Statements” included in Part I, Item 1 of this Quarterly Report on Form 10-Q, pursuant to our license agreement with Arvinas and Pfizer, we may be required to make certain additional payments, including license fees payable upon the successful completion of certain transition activities, regulatory and commercial milestone payments, royalties on net sales, as well as payments related to sublicensing arrangements. Also, as discussed in detail in “Note 5 – In-licensing and Acquisition” of our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q, pursuant to our license and transition services agreement with Forma, Forma is entitled to potential development and regulatory milestone payments and tiered royalty payments on net sales as well as certain portion of sublicensing revenue. In connection with our sublicensing agreements with Kissei and Dr. Reddy’s, Forma is entitled to a portion of the sublicensing revenue we receive under those agreements. 46 Table of Contents Additionally, as discussed in detail in “Note 5 – In-licensing and Acquisition” of our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q, pursuant to an Asset Purchase Agreement with Blueprint, Blueprint is entitled to potential commercial and regulatory milestone payments, as well as tiered royalty payments. As discussed above, we have the revolving credit facility with MidCap under our new Credit Agreement. The revolving credit facility has a five-year term and bears interest at a rate equal to one-month SOFR, subject to a 2.00% floor, plus an applicable margin of 4.00%. The obligations under the revolving credit facility are secured by a first-priority security interest in substantially all of our assets, including our intellectual property. The new Credit Agreement also requires us to pay customary fees, including an unused commitment fee, administrative fees and, during an initial period, prepayment premiums. Accordingly, our material cash requirements include interest payments on any outstanding borrowings under the revolving credit facility, as well as related fees and potential prepayment premiums, which may vary based on utilization levels and prevailing interest rates. At June 30, 2026, we have a contractual commitment related to our leased facility, which lease will expire in July 2027. Our remaining lease commitment was approximately $0.8 million, of which $0.7 million is due within the next 12 months. We are also subject to claims related to the patent protection of certain of our technologies, other litigations, and other contractual agreements. We are required to assess the likelihood of any adverse judgments or outcomes to these matters as well as potential ranges of probable losses. A determination of the amount of reserves required, if any, for these contingencies is made after careful analysis of each individual matter. We do not have other material contractual commitments with respect to matters discussed above.