Latest news with #GAVRETO
Yahoo
06-08-2025
- Business
- Yahoo
Rigel Pharmaceuticals Inc (RIGL) Q2 2025 Earnings Call Highlights: Record Revenue and Raised ...
Net Product Sales: $58.9 million, a 76% increase year over year. Total Revenue: $101.7 million, including $42.7 million in contract revenues from collaborations. Net Income: $59.6 million, compared to a net loss of $1 million in the same period in 2024. Cash Balance: $108.4 million at the end of the quarter. TAVALISSE Sales: $40.1 million, a 52% increase year over year. GAVRETO Sales: $11.8 million, with a 32% sequential growth from Q1 2025. REZLIDHIA Sales: $7 million, a 36% increase year over year. Revenue Guidance for 2025: Raised to $270 million to $280 million from the previous range of $200 million to $210 million. Net Product Sales Guidance for 2025: Increased to $210 million to $220 million from the prior range of $185 million to $192 million. Contract Revenues from Collaborations: Approximately $60 million, including $40 million in non-cash revenue from Lilly. Warning! GuruFocus has detected 4 Warning Signs with RIGL. Release Date: August 05, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Positive Points Rigel Pharmaceuticals Inc (NASDAQ:RIGL) achieved net product sales of over $58 million in Q2 2025, marking a 76% increase year over year and the company's best quarter ever. Total revenue for the second quarter was $101.7 million, including $42.7 million in contract revenues from collaborations. The company reported a net income of $59.6 million for the second quarter, compared to a net loss of $1 million in the same period in 2024. Rigel Pharmaceuticals Inc (NASDAQ:RIGL) raised its total revenue guidance for 2025 to between $270 million and $280 million, reflecting anticipated growth of 45% to 52% compared to 2024. The company has a strong cash position, ending the quarter with cash, cash equivalents, and short-term investments of $108.4 million, up from $77.3 million at the end of 2024. Negative Points Rigel Pharmaceuticals Inc (NASDAQ:RIGL) decided not to exercise its right to share in future development expenses for ocadusertib, resulting in a $40 million non-cash revenue recognition. The company faces challenges in maintaining growth momentum, as the impact of improved patient affordability due to the Inflation Reduction Act may not be as strong in the second half of the year. There is uncertainty regarding the long-term growth trajectory beyond 2025, as the company has not provided guidance for future years. The company noted a change in status for one patient in the R289 study, previously reported as a responder, due to additional red cell transfusions not initially entered into the database. Rigel Pharmaceuticals Inc (NASDAQ:RIGL) continues to face competition and unmet needs in the lower-risk MDS market, with existing therapies only achieving around 38% to 40% transfusion independence rates. Q & A Highlights Q: Can you explain the factors contributing to the 52% growth in TAVALISSE sales? A: David Santos, Executive Vice President, Chief Commercial Officer: The growth was primarily driven by an increase in new patient starts, which was higher than ever before, and improved patient affordability in 2025 due to the elimination of the coverage gap by the Inflation Reduction Act. This allowed patients easier access to TAVALISSE, an oral agent, with a $2,000 annual out-of-pocket cap. Q: Is the 45% to 52% growth expectation for 2025 indicative of a new long-term growth trajectory for Rigel? A: Raul Rodriguez, President, Chief Executive Officer, Director: While we haven't provided guidance beyond 2025, the underlying business has been growing steadily. The dynamics that contributed to this year's growth, such as improved affordability and strong product performance, are expected to continue, but we remain focused on ensuring sustained growth. Q: What are the plans for the Phase 2 study in glioma, and how does the CONNECT study fit into your strategy? A: Lisa Rojkjaer, Executive Vice President, Chief Medical Officer: We will disclose more about the Phase 2 study design in recurrent glioma later this year. Regarding the CONNECT study, we are still discussing with CONNECT and potentially the agency about using the data to support a filing. We do not anticipate conducting an additional study in the same space. Q: How does the IRA impact your guidance for TAVALISSE and GAVRETO sales going forward? A: David Santos, Executive Vice President, Chief Commercial Officer: We expect continued growth in revenues, with improved affordability dynamics having a stronger impact in the first half of the year. The coverage gap issue was more pronounced at the beginning of the year, so we anticipate a lesser impact in the second half. Q: What is considered a clinically meaningful reduction in transfusion dependence for low-risk MDS patients? A: Lisa Rojkjaer, Executive Vice President, Chief Medical Officer: Current agents like luspatercept and imetelstat show an eight-week transfusion independence rate of around 38% to 40%. There is room for improvement, as many patients still do not benefit, and there are associated toxicities and administration inconveniences with these agents. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus.
Yahoo
05-08-2025
- Business
- Yahoo
Rigel Reports Second Quarter 2025 Financial Results and Provides Business Update
Second quarter 2025 total revenue of approximately $101.7 million, which includes net product sales of $58.9 million and contract revenues from collaborations of $42.7 million Completed enrollment in the dose escalation part of the ongoing Phase 1b study evaluating R289, a dual IRAK1/4 inhibitor, in patients with R/R lower-risk MDS Generated $59.6 million of net income in the second quarter of 2025 Updated 2025 Outlook: Total revenue of approximately $270 to $280 million, which includes net product sales of $210 to $220 million Total revenue and net income are inclusive of $40 million in non-cash contract revenue related to Rigel's agreement with Lilly Conference call and webcast scheduled today at 4:30 p.m. Eastern Time SOUTH SAN FRANCISCO, Calif., Aug. 5, 2025 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, today reported financial results for the second quarter ended June 30, 2025, including sales of TAVALISSE® (fostamatinib disodium hexahydrate), GAVRETO® (pralsetinib) and REZLIDHIA® (olutasidenib), and recent business progress. "Our strategic and disciplined approach to building our business has generated another strong quarter for the company. In the second quarter, we grew net product sales by 76% year-over-year, generated $59.6 million in net income and increased our cash balance to $108.4 million. This strong performance has enabled us to raise our 2025 financial guidance," said Raul Rodriguez, Rigel's president and CEO. "We're also excited by the continued advancement of our hematology and oncology development pipeline. In July, we completed enrollment in the dose escalation part of our ongoing Phase 1b study evaluating R289 in patients with relapsed or refractory lower-risk MDS. Later this year, we plan to share updated data from that study and initiate the dose expansion part of the study." Second Quarter 2025 Business Update Commercial Net product sales were $58.9 million, an increase of 76% from the same period of 2024. Rigel's partner Kissei Pharmaceutical Co., Ltd. (Kissei) announced that its licensing partner, JW Pharmaceutical Corporation, commercially launched TAVALISSE in South Korea in early July. Corporate In April, Rigel notified Eli Lilly and Company (Lilly) that it would not exercise its opt-in right related to the development and commercialization of ocadusertib (previously R552) for the treatment of non-central nervous system (CNS) diseases. As a result of the notification, Rigel recognized $40.0 million in non-cash revenue resulting from the release of the remaining cost share liability in the second quarter. Per the agreement with Lilly, Rigel will continue to be entitled to receive milestone and tiered royalty payments on future net sales of ocadusertib and the companies' CNS penetrant program. Clinical Development Rigel continues to advance its Phase 1b clinical study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of R2891, a novel and selective dual interleukin receptor-associated kinases 1 and 4 (IRAK1/4) inhibitor, in patients with relapsed or refractory (R/R) lower-risk myelodysplastic syndrome (MDS). Enrollment in the dose escalation part of the study was completed in July. The company expects to share updated data from the study later this year and plans to initiate the dose expansion part of the study in the second half of this year. Rigel presented 4 posters at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and 3 posters at the European Hematology Association (EHA) 2025 Congress. Presentations included the final data from the GAVRETO (pralsetinib) Phase 1/2 ARROW study in RET fusion-positive non-small cell lung cancer (NSCLC) and other solid tumors, and supportive data for REZLIHDIA (olutasidenib) in patients with mutated isocitrate dehydrogenase-1 (mIDH1) relapsed or refractory (R/R) acute myeloid leukemia (AML). Key Publications A paper titled "Effectiveness of Olutasidenib versus Ivosidenib in Patients With Mutated Isocitrate Dehydrogenase 1 Acute Myeloid Leukemia Who Are Relapsed or Refractory to Venetoclax: The 2102-HEM-101 Trial Versus a US Electronic Health Record-Based External Control Arm," was published in June by Catherine Lai, M.D., MPH, lead author and associate professor of Clinical Medicine, Division of Hematology-Oncology, Department of Medicine at University of Pennsylvania, in Leukemia & Lymphoma. A paper titled "Olutasidenib Alone or Combined with Azacitidine in Patients with Mutant IDH1 Myelodysplastic Syndrome," was published in July by Jorge E. Cortes, M.D., lead author and director, Georgia Cancer Center, Cecil F. Whitaker Jr., GRA Eminent Scholar Chair in Cancer, in Blood Advances. This is the first full report of the safety and clinical activity of an mIDH1 inhibitor plus hypomethylating agent (HMA) combination in patients with treatment-naïve and R/R MDS. The paper showed olutasidenib, alone or in combination with azacitidine, induced a high rate of clinically meaningful complete remission and hematologic improvement responses with durable remission duration, accompanied by low rates of serious treatment-emergent-adverse events in patients with intermediate-, high- or very high-risk MDS harboring mIDH1. Second Quarter 2025 and Year-to-Date Financial UpdateFor the second quarter ended June 30, 2025, total revenues were $101.7 million, consisting of $58.9 million in net product sales and $42.7 million in contract revenues from collaborations. Net product sales grew 76% compared to $33.5 million in the same period of 2024. TAVALISSE net product sales were $40.1 million, growth of 52% compared to $26.4 million in the same period of 2024. GAVRETO net product sales were $11.8 million compared to $1.9 million in the same period of 2024. GAVRETO became commercially available from Rigel in late June 2024. REZLIDHIA net product sales were $7.0 million, growth of 36% compared to $5.2 million in the same period of 2024. Contract revenues from collaborations primarily consisted of $40.0 million in non-cash revenue resulting from the release of the remaining cost share liability related to the agreement with Lilly for the development and commercialization of ocadusertib, $2.0 million of revenue from Grifols S.A. (Grifols) related to delivery of drug supplies and earned royalties, $0.4 million of revenue from Kissei related to the delivery of drug supplies, and $0.2 million of revenue from Medison Pharma (Medison) related to delivery of drug supplies and earned royalties. Total costs and expenses were $40.6 million compared to $36.4 million for the same period of 2024. The increase in costs and expenses was mainly due to higher cost of product sales, increased research and development costs driven by the timing of clinical activities related to olutasidenib and R289, and higher personnel-related costs and stock-based compensation expense. Rigel reported net income of $59.6 million, or $3.33 basic and $3.28 diluted per share, compared to a net loss of $1.0 million, or $0.06 basic and diluted per share, for the same period of 2024. For the six months ended June 30, 2025, total revenues were $155.0 million, consisting of $102.5 million in net product sales and $52.5 million in contract revenues from collaborations. Net product sales grew 72% compared to $59.5 million in the same period of 2024. TAVALISSE net product sales were $68.5 million, growth of 44% compared to $47.5 million in the same period of 2024. GAVRETO net product sales were $20.8 million compared to $1.9 million in the same period of 2024. GAVRETO became commercially available from Rigel in late June 2024. REZLIDHIA net product sales were $13.1 million, growth of 31% compared to $10.0 million in the same period of 2024. Contract revenues from collaborations primarily consisted of $40.0 million in non-cash revenue resulting from the release of the remaining cost share liability related to the agreement with Lilly for the development and commercialization of ocadusertib, $6.7 million of revenue from Grifols related to delivery of drug supplies and earned royalties, $5.1 million of revenue from Kissei related to a milestone payment and delivery of drug supplies and $0.6 million of revenue from Medison related to delivery of drug supplies and earned royalties. Total costs and expenses were $81.1 million compared to $72.9 million for the same period of 2024. The increase in costs and expenses was mainly due to higher cost of product sales, increased research and development costs driven by the timing of clinical activities related to olutasidenib and R289, and higher personnel-related costs. These increases were partially offset by decreased stock-based compensation expense primarily from performance-based stock awards. Rigel reported net income of $71.1 million, or $3.98 basic and $3.91 diluted per share, compared to a net loss of $9.3 million, or $0.53 basic and diluted per share, for the same period of 2024. Cash, cash equivalents and short-term investments as of June 30, 2025 was $108.4 million, compared to $77.3 million as of December 31, 2024. 2025 OutlookRigel is updating its 2025 total revenue guidance to approximately $270 to $280 million, an increase from the previous range of approximately $200 to $210 million, which includes: Net product sales of approximately $210 to $220 million, an increase from the previous range of approximately $185 to $192 million. Contract revenues from collaborations of approximately $60 million, an increase from the previous range of approximately $15 to $18 million. The company anticipates it will report positive net income for the full year 2025, while funding existing and new clinical development programs. The above total revenues and contract revenues from collaborations are inclusive of $40 million in non-cash contract revenue related to Rigel's agreement with Lilly. Conference Call and Webcast with Slides Today at 4:30pm Eastern TimeRigel will hold a live conference call and webcast today at 4:30pm Eastern Time (1:30pm Pacific Time). Participants can access the live conference call by dialing (877) 407-3088 (domestic) or (201) 389-0927 (international). The conference call will also be webcast live and can be accessed from the Investor Relations section of the company's website at The webcast will be archived and available for replay after the call via the Rigel website. About ITPIn patients with immune thrombocytopenia (ITP), the immune system attacks and destroys the body's own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. Patients suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs), and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP. About NSCLCIt is estimated that over 226,000 adults in the U.S. will be diagnosed with lung cancer in 2025. Lung cancer is the leading cause of cancer death in the U.S, with non-small cell lung cancer (NSCLC) being the most common type accounting for 85-90% of all lung cancer diagnoses.2 RET fusions are implicated in approximately 1-2% of patients with NSCLC.3 About AMLAcute myeloid leukemia (AML) is a rapidly progressing cancer of the blood and bone marrow that affects myeloid cells, which normally develop into various types of mature blood cells. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that there will be about 22,010 new cases in the United States, most in adults, in 2025.4 Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.5,6 Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.7 Quality of life declines for patients with each successive line of treatment for AML, and well-tolerated treatments in relapsed or refractory disease remain an unmet need. About TAVALISSE®TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. Please click here for Important Safety Information and Full Prescribing Information for TAVALISSE. About GAVRETO®GAVRETO is indicated for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test and 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).* *Thyroid indication is approved 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(s). Please click here for Important Safety Information and Full Prescribing Information for GAVRETO. About REZLIDHIA®REZLIDHIA is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test. Please click here for Important Safety Information and Full Prescribing Information, including Boxed WARNING, for REZLIDHIA. To report side effects of prescription drugs to the FDA, visit or call 1-800-FDA-1088 (800-332-1088). TAVALISSE, GAVRETO and REZLIDHIA are registered trademarks of Rigel Pharmaceuticals, Inc. About RigelRigel Pharmaceuticals, Inc. (Nasdaq: RIGL) is a biotechnology company dedicated to discovering, developing and providing novel therapies that significantly improve the lives of patients with hematologic disorders and cancer. Founded in 1996, Rigel is based in South San Francisco, California. For more information on Rigel, the Company's marketed products and pipeline of potential products, visit R289 is an investigational compound not approved by the FDA. The American Cancer Society. Key Statistics for Lung Cancer. Revised January 16, 2025. Accessed March 31, 2025: Kato, S. et al. RET Aberrations in Diverse Cancers: Next-Generation Sequencing of 4,871 Patients. Clin Cancer Res. 2017;23(8):1988-1997 doi: 10.1158/ The American Cancer Society. Key Statistics for Acute Myeloid Leukemia (AML). Revised March 4, 2025. Accessed March 31, 2025: Patel, A, et al. Outcomes of Patients With Acute Myeloid Leukemia Who Relapse After 5 Years of Complete Remission. 2021 Sep 7;28(7):811-814. doi: Thol F, Ganser, A. Treatment of Relapsed Acute Myeloid Leukemia. Curr. Treat. Options on Oncol. (2020) 21: 66. doi: Thol F, Schlenk RF, Heuser M, Ganser A. How I treat refractory and early relapsed acute myeloid leukemia. Blood (2015) 126 (3): 319-27. doi: Forward Looking StatementsThis press release contains forward-looking statements relating to, among other things, expected commercial and financial results, projected financial performance and outlook for 2025, expectations for growing our commercial business, potential investment in our pipeline, continued advancement and updated results of the dose escalation portion of our R289 study, continued ability for developing and commercializing TAVALISSE, GAVRETO, and REZLIDHIA domestically and in certain international markets, and expectations for Rigel's partnering and collaboration efforts. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Forward-looking statements can be identified by words such as "anticipates", "plan", "outlook", "potential", "may", "look to", "expects", "will", "initial", "promising", and similar expressions in reference to future periods. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Rigel's current beliefs, expectations, and assumptions and hence they inherently involve significant risks, uncertainties and changes in circumstances that are difficult to predict and many of which are outside of our control. Therefore, you should not rely on any of these forward-looking statements. Actual results and the timing of events could differ materially from those anticipated in such forward looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with the commercialization and marketing of fostamatinib, olutasidenib and pralsetinib; risks that the FDA, European Medicines Agency, PMDA or other regulatory authorities may make adverse decisions regarding fostamatinib, pralsetinib or olutasidenib; risks that clinical trials may not be predictive of real-world results or of results in subsequent clinical trials; risks that fostamatinib, pralsetinib or olutasidenib may have unintended side effects, adverse reactions or incidents of misuses; the availability of resources to develop Rigel's product candidates; market competition; as well as other risks detailed from time to time in Rigel's reports filed with the Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended March 31, 2025 and subsequent filings. Any forward-looking statement made by us in this press release is based only on information currently available to us and speaks only as of the date on which it is made. Rigel does not undertake any obligation to update forward-looking statements, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise, and expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein, except as required by law. Contact for Investors & Media: Investors:Rigel Pharmaceuticals, Inc.650.624.1232ir@ Media:David RosenArgot RIGEL PHARMACEUTICALS, INC STATEMENTS OF OPERATIONS (in thousands, except per share amounts) Three Months Ended June 30,Six Months Ended June 30, 2025202420252024 (unaudited) Revenues: Product sales, net $ 58,948$ 33,450$ 102,498$ 59,453Contract revenues from collaborations 42,7373,39152,5206,922Total revenues 101,68536,841155,01866,375 Costs and expenses: Cost of product sales 4,5042,8078,9134,832Research and development (see Note A) 6,8215,54015,25711,566Selling, general and administrative (see Note A) 29,25728,04756,97256,496 Total costs and expenses 40,58236,39481,14272,894 Income (loss) from operations 61,10344773,876(6,519)Interest income 7535521,3441,145Interest expense (1,874)(2,029)(3,727)(3,903) Income (loss) before income taxes 59,982(1,030)71,493(9,277) Provision for income taxes 369—434— Net income (loss) $ 59,613$ (1,030)$ 71,059$ (9,277)Net income (loss) per share Basic $ 3.33$ (0.06)$ 3.98$ (0.53)Diluted $ 3.28$ (0.06)$ 3.91$ (0.53) Weighted average shares used in computing net income (loss) per share Basic 17,88517,54917,84817,534Diluted 18,16217,54918,16817,534Note AStock-based compensation expense included in: Selling, general and administrative $ 2,759$ 2,223$ 5,211$ 6,707Research and development 5173051,389955 $ 3,276$ 2,528$ 6,600$ 7,662 SUMMARY BALANCE SHEET DATA (in thousands)As of June 30,As of December 31, 20252024 (1) (unaudited) Cash, cash equivalents and short-term investments $ 108,378$ 77,321 Total assets 206,736163,976 Stockholders' equity 81,9343,288 (1) Derived from audited financial statements View original content to download multimedia: SOURCE Rigel Pharmaceuticals, Inc.
Yahoo
10-07-2025
- Business
- Yahoo
H.C. Wainwright Reiterates a Buy Rating on Rigel Pharmaceuticals (RIGL)
Rigel Pharmaceuticals, Inc. (NASDAQ:RIGL) is one of the 13 Cheap Healthcare Stocks with Huge Upside Potential. On June 3, H.C. Wainwright analyst Joseph Pantginis reiterated a Buy rating on Rigel Pharmaceuticals, Inc. (NASDAQ:RIGL) and set a price target of $57.00. The optimistic rating was based on the company's prospects, including promising results from the ARROW trial for GAVRETO. A researcher in a lab coat monitoring a biotechnological experiment. The trial demonstrated safety and strong efficacy for the treatment of RET fusion-positive NSCLC and other solid tumors. It also exhibited a significant median duration of response and a high overall response rate, pointing towards the drug's meaningful and durable outcomes. The analyst further reasoned that the trial data shows GAVRETO's optimistic anti-tumor activity across a number of RET fusion-positive solid tumors, which shows its market potential and paints an optimistic picture for Rigel Pharmaceuticals, Inc. (NASDAQ:RIGL). Rigel Pharmaceuticals, Inc. (NASDAQ:RIGL) is a clinical-stage biotechnology company that discovers and develops targeted, novel drugs in oncology, immunology, and immune oncology. Its product portfolio includes Tavalisse, Fostamatinib, and R835. While we acknowledge the potential of RIGL as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 30 Stocks That Should Double in 3 Years and 11 Hidden AI Stocks to Buy Right Now. Disclosure: None. This article is originally published at Insider Monkey.
Yahoo
22-05-2025
- Business
- Yahoo
Rigel Announces Poster Presentations at the 2025 ASCO Annual Meeting and EHA2025 Congress
Final data from the GAVRETO® (pralsetinib) Phase 1/2 ARROW study in RET fusion-positive NSCLC and other solid tumors Supportive data for REZLIDHIA® (olutasidenib) utilization in patients with mIDH1 R/R AML SOUTH SAN FRANCISCO, Calif., May 22, 2025 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, today announced seven upcoming poster presentations at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and European Hematology Association (EHA) 2025 Congress. The ASCO Annual Meeting is being held in Chicago, Illinois and virtually from May 30 to June 3, 2025. The EHA2025 Congress is being held in Milan, Italy and virtually from June 12 to June 15, 2025. Rigel's poster presentations will include data for GAVRETO® (pralsetinib) for the treatment of metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), including final data from the Phase 1/2 ARROW study, and REZLIDHIA® (olutasidenib) for the treatment of relapsed or refractory (R/R) mutated isocitrate dehydrogenase-1 (mIDH1) acute myeloid leukemia (AML). "We look forward to several posters highlighting the strength of our hematology and oncology product portfolio at ASCO and EHA, including the final efficacy and safety data from the Phase 1/2 ARROW study of GAVRETO. The study data continue to demonstrate GAVRETO's clinically meaningful and durable responses in patients with RET fusion-positive NSCLC, regardless of prior therapies, with a manageable safety profile. The study also showed promising anti-tumor activity in patients with various RET fusion-positive solid tumors, suggesting the potential for GAVRETO to address these unmet needs," said Raul Rodriguez, Rigel's president and CEO. "In addition, the collective data being presented on REZLIDHIA support its duration of response and potential clinical benefit when used in earlier lines of treatment for R/R AML patients and in primary refractory patients who are traditionally difficult to treat." ASCO Annual Meeting abstracts may be accessed online via Details of the poster presentations are as follows: ASCO Poster Presentations Saturday, May 31, 2025, 1:30pm to 4:30pm CTAbstract #: 8644Title: Efficacy and Safety of Pralsetinib in Patients with Advanced RET-fusion-positive NSCLC: Final Data from the Phase 1/2 ARROW StudyPresenter: Gilberto de Lima Lopes, M.D., MBA Final top-line efficacy and safety in RET fusion-positive NSCLC patients produced clinically meaningful and durable responses regardless of prior therapies, with a manageable safety profile. These results support the findings in previously published results, with a longer follow up period. Overall response rate (ORR) was 70.3% and median duration of response (DOR) was 19.1 months in the measurable disease population (n=259). In the efficacy population (n=281), median overall survival (OS) was 44.3 months with median follow up of 47.6 months. Median progression-free survival (PFS) was 13.1 months overall but was longer in the United States (25.9 months) vs. Asia (12.6 months) or Europe (12.9 months). Sunday, June 1, 2025, 9:00am to 12:00pm CTAbstract #: 6545Title: A Phase 2 Study of Olutasidenib in Relapsed/Refractory AML: Outcomes by Number of Prior Treatment RegimensPresenter: Eunice S. Wang, M.D. A post-hoc analysis of the pivotal cohort from the Phase 1/2 study evaluated outcomes in patients with R/R mIDH1 AML who received olutasidenib after either 1-2 or ≥3 prior lines of therapy. Patients in the 1-2 prior regimens group showed higher ORR and complete response (CR) / complete response with hematologic improvement (CRh) rates and longer median OS than those with ≥3 prior lines of therapy, providing a rationale for initiating olutasidenib earlier in the R/R treatment paradigm. Abstract #: 6546Title: Matching-adjusted Indirect Comparison (MAIC) of Olutasidenib and Ivosidenib in IDH1-mutated Relapsed/Refractory AMLPresenter: Justin M. Watts, M.D. In the absence of a head-to-head trial, a matching-adjusted indirect comparison (MAIC) analysis compared relative treatment effects of olutasidenib vs. ivosidenib in mIDH1 R/R AML, leveraging registrational data for olutasidenib and ivosidenib to match patients using key baseline clinical variables. Naïve and adjusted rates of response for olutasidenib vs. ivosidenib were comparable (adjusted point estimate favored olutasidenib for CR and ivosidenib for CR+CRh), while a longer duration of CR+CRh was observed with olutasidenib. Adjusted OS was similar between the two groups, although the hazard ratio (HR) favored olutasidenib. Results rely on the assumption of no unmeasured confounders, which reflects a limitation of the methodology. Monday, June 2, 2025, 1:30pm to 4:30pm CTAbstract #: 3116Title: Efficacy and Safety of Pralsetinib in RET Fusion-positive Solid Tumors: Final Data from the ARROW TrialPresenter: Vivek Subbiah, M.D. Final results from the Phase 2 portion of ARROW in patients with RET fusion-positive solid tumors other than NSCLC and thyroid cancer showed an ORR of 46.4% (13/28), including an ORR for pancreatic cancer of 100% (5/5). Overall, 10.7% (3/28) achieved complete response (pancreatic cancer, n=2; cancer of unknown primary, n=1) and 35.7% (10/28) achieved partial response. Median PFS was 7 months and median DOR was 11.1 months. Median OS was 10.3 months. Pralsetinib demonstrated robust and durable anti-tumor activity, with responses observed in many tumor types. These data validate RET fusions as a tissue-agnostic target with sensitivity to RET inhibition and activity beyond NSCLC and thyroid cancer, further supporting the promising potential of pralsetinib to address the unmet medical need in these patients. EHA2025 Congress abstracts may be accessed online via the EHA Library. Details of the poster presentations and publications are as follows: EHA Poster Presentations Friday, June 13, 2025, Time 18:30 to 19:30 CESTAbstract #: PF511Title: Efficacy and Safety of Olutasidenib Monotherapy in Primary Refractory AML: A Post Hoc Analysis of a Phase 2 StudyPresenter: Antonio Curti, M.D., Ph.D. In the pivotal cohort of R/R AML patients, 46 were primary refractory to first line treatment or subsequent induction therapy. With olutasidenib therapy, the ORR was 50% (23/46) and 30% (14/46) achieved a CR/CRh, with a mediation duration of CR/CRh of 17.6 months. The most commonly occurring treatment-emergent adverse events (AEs) were nausea (41%), vomiting (30%), and an increase in white blood cells (30%), with no new safety signals observed. Olutasidenib as a single agent demonstrated clinically meaningful activity and a durable response in patients with primary refractory AML, with an acceptable tolerability profile, suggesting it may be an effective therapeutic option for this patient population with a traditionally poor prognosis. Abstract #: PF516Title: Effect of Mutation Type and Co-mutations on Response to Olutasidenib in Patients With R/R Mutated IDH1 AMLPresenter: Stéphane de Botton, M.D., Ph.D. In the final five-year analysis of the registrational trial, mutational analysis showed durable responses across IDH1-R132 mutation types, particularly in the absence of RTK pathway co-mutations. Response rates were lower in patients with ≥4 co-mutations. Results show that it is possible for patients with deleterious co-mutations (i.e., FLT3, TP53, etc.) to respond favorably to olutasidenib, although the sample sizes are small for some co-mutations. Abstract #: PF530Title: Olutasidenib as Maintenance Therapy after Treatment Response in Mutated IDH1 Acute Myeloid LeukemiaPresenter: Andrew H. Wei, MBBS, Ph.D. In a separate cohort (n=18) of the Phase 2 trial, olutasidenib was evaluated as maintenance therapy in patients who achieved minimal residual disease (MRD)-positive CR or CR with incomplete blood count recovery (CRi) with prior treatment. The 4-month relapse-free survival (RFS) was 83% with a median RFS of 18.4 months. At 12 months, RFS was 71% and OS was 89%. RFS at 2 years was 48%. Two patients who had received prior venetoclax therapy entered with a CRi, improved to a CRh and ultimately a CR during the study. Most common treatment-emergent AEs were fatigue (33%), headache (33%) and nausea (28%), with no new safety signals. Olutasidenib as a single agent demonstrated clinically meaningful activity as a maintenance strategy in a subset of AML patients with CR/CRi and persistent MRD ≥0.01% after prior therapy. The analysis supports the potential benefit of switching to olutasidenib upon response to therapy. EHA Publications Abstract #: PB2499Title: Comparative Effectiveness of Olutasidenib and Ivosidenib in mIDH1 Relapsed or Refractory Acute Myeloid Leukemia Patients Post-Venetoclax: Insights From 2102-HEM-101 and a Real-World External ControlAuthors: Catherine Lai, M.D., MPH, Thomas Leahy, Ph.D., CStat, Alex Turner, Ph.D., Amber Thomassen, AGNP-BC, AOCNP, Lixia Wang, Ph.D., Aaron D. Sheppard, Ph.D., Jorge E. Cortes, M.D. Abstract #: PB2492Title: A Phase 2 Study of Olutasidenib in Relapsed/Refractory AML: Outcomes by Number of Prior Treatment RegimensAuthors: Eunice S. Wang, M.D., Jorge E. Cortes, M.D., Andrew H. Wei, M.D., Stéphane de Botton, M.D., Ph.D., Antonio Curti, M.D., Ph.D., Pau Montesinos, M.D., Ph.D., Karen W.L. Yee, M.D., Joseph G. Jurcic, M.D., William B. Donnellan, M.D., Jay Yang, M.D., Brian A. Jonas, M.D., Ph.D., Aaron D. Sheppard, Ph.D., Hua Tian, M.D., Justin M. Watts, M.D. Abstract #: PB2528Title: Matching-adjusted Indirect Comparison (MAIC) of Olutasidenib (OLU) and Ivosidenib (IVO) in isocitrate dehydrogenase-1 (IDH1)-mutated Relapsed/Refractory (R/R) AMLAuthors: Brian A. Jonas, M.D., Ph.D., Justin M. Watts, M.D., Eunice S. Wang, M.D., Florence R. Wilson, MSc, Julie Park, MMath, Shannon Cope, MSc, Aaron D. Sheppard, Ph.D., Jorge E. Cortes, M.D., Stéphane de Botton, M.D., Ph.D. About NSCLCIt is estimated that over 226,000 adults in the U.S. will be diagnosed with lung cancer in 2025. Lung cancer is the leading cause of cancer death in the U.S, with non-small cell lung cancer (NSCLC) being the most common type accounting for 85-90% of all lung cancer diagnoses.1 RET fusions are implicated in approximately 1-2% of patients with NSCLC.2 About AMLAcute myeloid leukemia (AML) is a rapidly progressing cancer of the blood and bone marrow that affects myeloid cells, which normally develop into various types of mature blood cells. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that there will be about 22,010 new cases in the United States, most in adults, in 2025.3 Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.4,5 Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.6 Quality of life declines for patients with each successive line of treatment for AML, and well-tolerated treatments in relapsed or refractory disease remain an unmet need. About GAVRETO® (pralsetinib) INDICATIONS GAVRETO (pralsetinib) is indicated for the treatment of: Adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test 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 is approved 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(s). IMPORTANT SAFETY INFORMATION Interstitial Lung Disease (ILD)/Pneumonitis: Severe, life-threatening, and fatal ILD/pneumonitis can occur in patients treated with GAVRETO. Pneumonitis occurred in 12% of patients who received GAVRETO, including 3.3% with Grade 3-4, and 0.2% with fatal reactions. Monitor for pulmonary symptoms indicative of ILD/pneumonitis. Withhold GAVRETO and promptly investigate for ILD in any patient who presents with acute or worsening of respiratory symptoms (e.g., dyspnea, cough, and fever). Withhold, reduce dose or permanently discontinue GAVRETO based on severity of confirmed ILD. Hypertension: Occurred in 35% of patients, including Grade 3 hypertension in 18% of patients. Overall, 8% had their dose interrupted and 4.8% had their dose reduced for hypertension. Treatment-emergent hypertension was most commonly managed with anti-hypertension medications. Do not initiate GAVRETO in patients with uncontrolled hypertension. Optimize blood pressure prior to initiating GAVRETO. Monitor blood pressure after 1 week, at least monthly thereafter and as clinically indicated. Initiate or adjust anti-hypertensive therapy as appropriate. Withhold, reduce dose, or permanently discontinue GAVRETO based on the severity. Hepatotoxicity: Serious hepatic adverse reactions occurred in 1.5% of patients treated with GAVRETO. Increased aspartate aminotransferase (AST) occurred in 49% of patients, including Grade 3 or 4 in 7% and increased alanine aminotransferase (ALT) occurred in 37% of patients, including Grade 3 or 4 in 4.8%. The median time to first onset for increased AST was 15 days (range: 5 days to 2.5 years) and increased ALT was 24 days (range: 7 days to 3.7 years). Monitor AST and ALT prior to initiating GAVRETO, every 2 weeks during the first 3 months, then monthly thereafter and as clinically indicated. Withhold, reduce dose or permanently discontinue GAVRETO based on severity. Hemorrhagic Events: Serious, including fatal, hemorrhagic events can occur with GAVRETO. Grade ≥3 events occurred in 4.1% of patients treated with GAVRETO including one patient with a fatal hemorrhagic event. Permanently discontinue GAVRETO in patients with severe or life-threatening hemorrhage. Tumor Lysis Syndrome (TLS): Cases of TLS have been reported in patients with medullary thyroid carcinoma receiving GAVRETO. Patients may be at risk of TLS if they have rapidly growing tumors, a high tumor burden, renal dysfunction, or dehydration. Closely monitor patients at risk, consider appropriate prophylaxis including hydration, and treat as clinically indicated. Risk of Impaired Wound Healing: Impaired wound healing can occur in patients who receive drugs that inhibit the vascular endothelial growth factor (VEGF) signaling pathway. Therefore, GAVRETO has the potential to adversely affect wound healing. Withhold GAVRETO for at least 5 days prior to elective surgery. Do not administer for at least 2 weeks following major surgery and until adequate wound healing. The safety of resumption of GAVRETO after resolution of wound healing complications has not been established. Embryo-Fetal Toxicity: Based on findings from animal studies and its mechanism of action, GAVRETO can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective non-hormonal contraception during treatment with GAVRETO and for 2 weeks after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with GAVRETO and for 1 week after the last dose. Common adverse reactions (≥25%) were musculoskeletal pain, constipation, hypertension, diarrhea, fatigue, edema, pyrexia, and cough. Common Grade 3/4 laboratory abnormalities (≥2%) were decreased lymphocytes, decreased neutrophils, decreased hemoglobin, decreased phosphate, decreased leukocytes, decreased sodium, increased aspartate aminotransferase (AST), increased alanine aminotransferase (ALT), decreased calcium (corrected), decreased platelets, increased alkaline phosphatase, increased potassium, decreased potassium, and increased bilirubin. Avoid coadministration of GAVRETO with strong or moderate CYP3A inhibitors, P-gp inhibitors, or combined P-gp and strong or moderate CYP3A inhibitors. If coadministration cannot be avoided, reduce the GAVRETO dose. Avoid coadministration of GAVRETO with strong or moderate CYP3A inducers. If coadministration cannot be avoided, increase the GAVRETO dose. Lactation: Advise women not to breastfeed during treatment with GAVRETO and for 1 week after the last dose. Pediatric Use: Monitor open growth plates in adolescent patients. Consider interrupting or discontinuing GAVRETO if abnormalities occur. Click here for Important Safety Information and Full Prescribing Information. To report side effects of prescription drugs to the FDA, visit or call 1-800-FDA-1088 (800-332-1088). About REZLIDHIA® INDICATIONREZLIDHIA is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test. IMPORTANT SAFETY INFORMATION WARNING: DIFFERENTIATION SYNDROME Differentiation syndrome, which can be fatal, can occur with REZLIDHIA treatment. Symptoms may include dyspnea, pulmonary infiltrates/pleuropericardial effusion, kidney injury, hypotension, fever, and weight gain. If differentiation syndrome is suspected, withhold REZLIDHIA and initiate treatment with corticosteroids and hemodynamic monitoring until symptom resolution. WARNINGS AND PRECAUTIONS Differentiation SyndromeREZLIDHIA can cause differentiation syndrome. In the clinical trial of REZLIDHIA in patients with relapsed or refractory AML, differentiation syndrome occurred in 16% of patients, with grade 3 or 4 differentiation syndrome occurring in 8% of patients treated, and fatalities in 1% of patients. Differentiation syndrome is associated with rapid proliferation and differentiation of myeloid cells and may be life-threatening or fatal. Symptoms of differentiation syndrome in patients treated with REZLIDHIA included leukocytosis, dyspnea, pulmonary infiltrates/pleuropericardial effusion, kidney injury, fever, edema, pyrexia, and weight gain. Of the 25 patients who experienced differentiation syndrome, 19 (76%) recovered after treatment or after dose interruption of REZLIDHIA. Differentiation syndrome occurred as early as 1 day and up to 18 months after REZLIDHIA initiation and has been observed with or without concomitant leukocytosis. If differentiation syndrome is suspected, temporarily withhold REZLIDHIA and initiate systemic corticosteroids (e.g., dexamethasone 10 mg IV every 12 hours) for a minimum of 3 days and until resolution of signs and symptoms. If concomitant leukocytosis is observed, initiate treatment with hydroxyurea, as clinically indicated. Taper corticosteroids and hydroxyurea after resolution of symptoms. Differentiation syndrome may recur with premature discontinuation of corticosteroids and/or hydroxyurea treatment. Institute supportive measures and hemodynamic monitoring until improvement; withhold dose of REZLIDHIA and consider dose reduction based on recurrence. HepatotoxicityREZLIDHIA can cause hepatotoxicity, presenting as increased alanine aminotransferase (ALT), increased aspartate aminotransferase (AST), increased blood alkaline phosphatase, and/or elevated bilirubin. Of 153 patients with relapsed or refractory AML who received REZLIDHIA, hepatotoxicity occurred in 23% of patients; 13% experienced grade 3 or 4 hepatotoxicity. One patient treated with REZLIDHIA in combination with azacitidine in the clinical trial, a combination for which REZLIDHIA is not indicated, died from complications of drug-induced liver injury. The median time to onset of hepatotoxicity in patients with relapsed or refractory AML treated with REZLIDHIA was 1.2 months (range: 1 day to 17.5 months) after REZLIDHIA initiation, and the median time to resolution was 12 days (range: 1 day to 17 months). The most common hepatotoxicities were elevations of ALT, AST, blood alkaline phosphatase, and blood bilirubin. Monitor patients frequently for clinical symptoms of hepatic dysfunction such as fatigue, anorexia, right upper abdominal discomfort, dark urine, or jaundice. Obtain baseline liver function tests prior to initiation of REZLIDHIA, at least once weekly for the first two months, once every other week for the third month, once in the fourth month, and once every other month for the duration of therapy. If hepatic dysfunction occurs, withhold, reduce, or permanently discontinue REZLIDHIA based on recurrence/severity. ADVERSE REACTIONSThe most common (≥20%) adverse reactions, including laboratory abnormalities, were aspartate aminotransferase increased, alanine aminotransferase increased, potassium decreased, sodium decreased, alkaline phosphatase increased, nausea, creatinine increased, fatigue/malaise, arthralgia, constipation, lymphocytes increased, bilirubin increased, leukocytosis, uric acid increased, dyspnea, pyrexia, rash, lipase increased, mucositis, diarrhea and transaminitis. DRUG INTERACTIONS Avoid concomitant use of REZLIDHIA with strong or moderate CYP3A inducers. Avoid concomitant use of REZLIDHIA with sensitive CYP3A substrates unless otherwise instructed in the substrates prescribing information. If concomitant use is unavoidable, monitor patients for loss of therapeutic effect of these drugs. LACTATIONAdvise women not to breastfeed during treatment with REZLIDHIA and for 2 weeks after the last dose. GERIATRIC USENo overall differences in effectiveness were observed between patients 65 years and older and younger patients. Compared to patients younger than 65 years of age, an increase in incidence of hepatotoxicity and hypertension was observed in patients ≥65 years of age. HEPATIC IMPAIRMENTIn patients with mild or moderate hepatic impairment, closely monitor for increased probability of differentiation syndrome. Click here for Important Safety Information and Full Prescribing Information, including Boxed WARNING. To report side effects of prescription drugs to the FDA, visit or call 1-800-FDA-1088 (800-332-1088). GAVRETO and REZLIDHIA are registered trademarks of Rigel Pharmaceuticals, Inc. About RigelRigel Pharmaceuticals, Inc. (Nasdaq: RIGL) is a biotechnology company dedicated to discovering, developing and providing novel therapies that significantly improve the lives of patients with hematologic disorders and cancer. Founded in 1996, Rigel is based in South San Francisco, California. For more information on Rigel, the Company's marketed products and pipeline of potential products, visit The American Cancer Society. Key Statistics for Lung Cancer. Revised January 16, 2025. Accessed March 31, 2025: Kato, S. et al. RET Aberrations in Diverse Cancers: Next-Generation Sequencing of 4,871 Patients. Clin Cancer Res. 2017;23(8):1988-1997 doi: 10.1158/ The American Cancer Society. Key Statistics for Acute Myeloid Leukemia (AML). Revised March 4, 2025. Accessed March 31, 2025: Patel, A, et al. Outcomes of Patients With Acute Myeloid Leukemia Who Relapse After 5 Years of Complete Remission. 2021 Sep 7;28(7):811-814. doi: Thol F, Ganser, A. Treatment of Relapsed Acute Myeloid Leukemia. Curr. Treat. Options on Oncol. (2020) 21: 66. doi: Thol F, Schlenk RF, Heuser M, Ganser A. How I treat refractory and early relapsed acute myeloid leukemia. Blood (2015) 126 (3): 319-27. doi: Forward-Looking StatementsThis press release contains forward-looking statements relating to, among other things, the potential for the referenced clinical trials or trial results to strengthen our commercial portfolio, GAVRETO's success in treating both RET fusion-positive NSCLC and RET fusion-positive solid tumors, and the benefit of REZLIDHIA as an earlier line treatment for R/R AML. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Forward-looking statements can be identified by words such as "anticipates", "plan", "outlook", "potential", "may", "look to", "expects", "will", "initial", "promising", and similar expressions in reference to future periods. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Rigel's current beliefs, expectations, and assumptions and hence they inherently involve significant risks, uncertainties and changes in circumstances that are difficult to predict and many of which are outside of our control. Therefore, you should not rely on any of these forward-looking statements. Actual results and the timing of events could differ materially from those anticipated in such forward looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with the commercialization and marketing of fostamatinib, olutasidenib and pralsetinib; risks that the FDA, European Medicines Agency, PMDA or other regulatory authorities may make adverse decisions regarding fostamatinib, pralsetinib or olutasidenib; risks that clinical trials may not be predictive of real-world results or of results in subsequent clinical trials; risks that fostamatinib, pralsetinib or olutasidenib may have unintended side effects, adverse reactions or incidents of misuses; the availability of resources to develop Rigel's product candidates; market competition; as well as other risks detailed from time to time in Rigel's reports filed with the Securities and Exchange Commission, including its most recent Annual Report on Form 10-K, and subsequent filings, including Quarterly Reports on Form 10-Q. Any forward-looking statement made by us in this press release is based only on information currently available to us and speaks only as of the date on which it is made. Rigel does not undertake any obligation to update forward-looking statements, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise, and expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein, except as required by law. Contact for Investors & Media: Investors:Rigel Pharmaceuticals, Inc.650.624.1232ir@ Media:David RosenArgot View original content to download multimedia: SOURCE Rigel Pharmaceuticals, Inc. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
