Core Insights - Rigel Pharmaceuticals announced the publication of five-year data from a Phase 2 trial of REZLIDHIA (olutasidenib) for treating relapsed or refractory mutant isocitrate dehydrogenase-1 acute myeloid leukemia (mIDH1 AML) [1][2] Efficacy and Safety Data - The five-year follow-up analysis supports the durable responses and manageable safety profile of olutasidenib, including efficacy in patients previously treated with venetoclax [2][5] - Among 147 efficacy evaluable patients, the complete remission (CR) or CR with partial hematologic recovery (CRh) rate was 35%, with a median duration of 25.3 months [5] - The overall response rate (ORR) was 48%, with a median duration of 15.5 months, and median overall survival (OS) was 11.5 months [5] - Transfusion independence was achieved in 39% of patients dependent on red blood cells and 41% of those dependent on platelets at baseline [5] Patient Response Insights - 66% of patients who achieved an overall response did so within 2 months, while 10% required at least 4.6 months of therapy [5] - Patients with one to two prior regimens had a higher CR/CRh rate of 41% and a median OS of 13 months compared to those with three prior regimens [5] - In patients R/R to prior venetoclax, 33% achieved a CR/CRh, with a median OS of 16.2 months [5] Ongoing Research and Collaborations - Rigel is evaluating olutasidenib in other disease areas and has strategic collaborations with The University of Texas MD Anderson Cancer Center [4][12] - A fifth study under the strategic alliance with MD Anderson has opened for enrollment, focusing on olutasidenib in combination with co-targeted therapies [12] Industry Context - Acute myeloid leukemia (AML) is a rapidly progressing cancer affecting myeloid cells, with an estimated 22,010 new cases in the U.S. in 2025 [6] - Relapsed AML affects about half of all patients post-treatment, while refractory AML occurs in 10-40% of newly diagnosed patients [7]

Q3 2025 Financial Results Presentation November 4, 2025 1 Forward Looking Statements Raul Rodriguez President & Chief Executive Officer Ray Furey, J.D. Executive Vice President, General Counsel & Corporate Secretary This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 ("PSLRA") relating to, among other things, continued development and expansion of our business, projected financial performance including sales and revenue growth, con ...

Core Insights - Rigel Pharmaceuticals is set to present data from its ongoing Phase 1b study of R289, a dual IRAK1/4 inhibitor, at the 67th American Society of Hematology (ASH) Annual Meeting on December 7, 2025, focusing on patients with relapsed or refractory lower-risk myelodysplastic syndrome (MDS) [1][2][5] - The ASH Annual Meeting will also feature four poster presentations regarding REZLIDHIA (olutasidenib) for treating relapsed or refractory mutated isocitrate dehydrogenase-1 (mIDH1) acute myeloid leukemia (AML) [1][5] R289 Study Details - The Phase 1b study of R289 has enrolled 33 patients as of July 15, 2025, with a median age of 75 and a median of 3 prior therapies; 61% of these patients had a high transfusion burden at baseline [6] - R289 was administered at doses ranging from 250 mg once daily to 500 mg twice daily, with the most common treatment-emergent adverse events being diarrhea (28.1%), constipation (25%), and increased creatinine/ALT (21.9%) [6] - Among evaluable transfusion-dependent patients receiving doses of at least 500 mg, 31% achieved durable red blood cell transfusion independence for over 8 weeks, with a median time to onset of 2.2 months and a median duration of 24.3 weeks [6] REZLIDHIA Data Highlights - In a pivotal cohort of the Phase 2 registrational study involving 147 patients with R/R mIDH1 AML, 24% maintained stable disease after two treatment cycles, with a subsequent response rate of 33% [9] - The median overall survival for late responders was reported at 23.9 months, indicating that patients with stable disease after two cycles may benefit from continued treatment [9][10] - The overall response rate in a real-world cohort of olutasidenib-treated patients was 50%, supporting its use as a viable therapeutic option in post-venetoclax treatment settings [9][10] Company Overview - Rigel Pharmaceuticals, Inc. is a biotechnology company focused on developing therapies for hematologic disorders and cancer, founded in 1996 and based in South San Francisco, California [26]

Financial Performance & Guidance - Q2 2025 net product sales reached $58.9 million[81] - Q2 2025 contract revenues from collaborations totaled $42.7 million[81] - Rigel increased its 2025 net product sales guidance to a range of $210 million to $220 million, up from the previous guidance of $185 million to $192 million[14, 86] - The company anticipates total revenue for 2025 to be approximately $270 million to $280 million, revised from the initial estimate of $200 million to $210 million[85] - Rigel reported net product sales of $102.5 million YTD[15] Product Performance - Q2 2025 net product sales for TAVALISSE were $40.1 million[26, 83] - Q2 2025 net product sales for GAVRETO were $11.8 million[29, 83] - Q2 2025 net product sales for REZLIDHIA were $7.0 million[29, 83] R&D and Clinical Development - Rigel is evaluating R289, a dual IRAK1/4 inhibitor, in a Phase 1b study for relapsed/refractory lower-risk MDS[13] - The company plans to initiate a Phase 2 study of olutasidenib in recurrent glioma[13] Strategic Collaborations - Collaboration revenues included $40.0 million from Eli Lilly[83] - Grifols contributed $2.0 million in collaboration revenue[35, 83] - Kissei contributed $0.4 million in collaboration revenue[35, 83] - Medison contributed $0.2 million in collaboration revenue[35, 83]

Financial Performance - Total net product sales for Q2 2024 were $33.5 million[148] - TAVALISSE net product sales for Q2 2024 were $26.4 million[34, 148], representing a 25% growth compared to Q2 2023[34] and 8% growth compared to Q1 2024[34] - REZLIDHIA net product sales for Q2 2024 were $5.2 million[65, 148], a 102% increase compared to Q2 2023[65] and a 5% increase compared to Q1 2024[65] - GAVRETO net product sales for Q2 2024 were $1.9 million[96, 148] Product Sales and Growth - TAVALISSE bottles shipped to patients and clinics in Q2 2024 totaled 2,672[34], an 18% increase compared to Q2 2023[33] and a 24% increase compared to Q1 2024[34] - REZLIDHIA bottles shipped to patients and clinics in Q2 2024 totaled 424[64], a 127% increase compared to Q2 2023[64] and a 30% increase compared to Q1 2024[64] - 228 GAVRETO 60 ct eq bottles were sold in Q2 2024[96] Clinical Development and Collaborations - Rigel and The University of Texas MD Anderson Cancer Center will evaluate olutasidenib in combination with other agents to treat newly-diagnosed and relapsed/refractory patients with IDH1-mutated AML[106] - Rigel will provide funding up to $3 million and study material over the 4-year collaboration with CONNECT to Advance Olutasidenib in Glioma[115]