Core Insights - Rigel Pharmaceuticals announced updated data from its ongoing Phase 1b study of R289, an oral prodrug of R835, targeting relapsed or refractory lower-risk myelodysplastic syndrome (MDS) [1][2] - The study results were presented at the 67th American Society of Hematology Annual Meeting, highlighting the potential of R289 as a treatment option for patients with transfusion-dependent lower-risk MDS [1][2] Study Overview - The Phase 1b study is evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of R289 in patients with relapsed or refractory lower-risk MDS [2] - Enrollment in the dose escalation phase was completed in July 2025, with the dose expansion phase starting in October 2025, involving up to 40 patients [2] Key Data Highlights - As of October 28, 2025, 33 patients were enrolled, with a median age of 75 and a median of 3 prior therapies [4] - R289 was generally well tolerated, with 33% of evaluable transfusion-dependent patients achieving durable red blood cell transfusion independence (RBC-TI) [3][4] - The most common treatment-emergent adverse events included diarrhea (30%), constipation (27%), and fatigue (27%) [4] Efficacy Results - Among evaluable transfusion-dependent patients, 6 out of 18 (33%) achieved RBC-TI lasting more than 8 weeks, with a median time to onset of 1.9 months [4] - Peak hemoglobin increases ranged from 2.9 to 6.1 g/dL compared to baseline in patients achieving RBC-TI [4] Regulatory Designations - R289 has received Orphan Drug designation and Fast Track designation from the FDA for the treatment of previously-treated transfusion-dependent lower-risk MDS [5]

Financial Performance & Guidance - Q3 2025 net product sales grew to $64.1 million, a 65% increase compared to Q3 2024[10, 158] - Rigel increased its 2025 net product sales guidance to $225 million - $230 million, up from $210 million - $220 million[12] - Rigel anticipates 2025 total revenue of ~$285 million to $290 million[162] - Cash, cash equivalents & short-term investments as of September 30, 2025, was $137.1 million compared to $77.3 million as of December 31, 2024[162] Product Performance - TAVALISSE Q3 2025 net product sales reached $44.7 million, a 70% increase compared to Q3 2024[16] - GAVRETO Q3 2025 net product sales reached $11.1 million, a 56% increase compared to Q3 2024[16] - REZLIDHIA Q3 2025 net product sales reached $8.3 million, a 50% increase compared to Q3 2024[16] R289 Clinical Development - In a Phase 1b study in R/R lower-risk MDS, 33% (6/18) of evaluable transfusion-dependent patients receiving ≥500 mg QD achieved RBC-TI >8 weeks[124, 131] - The median duration of RBC-TI was 22.9 weeks (9 - 104.3)[124] Strategic Collaborations - Contract revenues from collaborations were $5.4 million in Q3 2025[158]

Summary of Rigel Pharmaceuticals Conference Call Company Overview - Rigel Pharmaceuticals is a hematology and oncology biotech company focused on expanding its commercial business and pipeline [2][3] Commercial Products - Rigel has three approved products: - **TAVALISSE**: Indicated for immune thrombocytopenia (ITP) with net sales of $44.7 million last quarter, a 70% increase year-over-year [6] - **REZLIDHIA**: Approved for mutant IDH1 relapsed and refractory acute myeloid leukemia (AML) with sales of $8.3 million, a 50% growth from the previous year [6] - **GAVRETO**: Approved for RET fusion positive non-small cell lung cancer and thyroid cancer, with sales increasing from $7 million to $11.1 million [6][12] Financial Performance - Rigel reported $64 million in net product sales last quarter, a 65% increase from the same quarter last year, and $166 million over three months, surpassing total sales for all of 2024 [5] - The company generated $60 million in cash this year and has maintained profitability [4][30] - Guidance for revenue is set at $285-$290 million, with net product sales expected to be $225-$230 million [30] Pipeline Development - Rigel is focused on developing transformational programs, particularly R289 for low-risk myelodysplastic syndromes (MDS) [4][17] - R289 has received fast-track and orphan designations from regulatory agencies, indicating its potential for significant improvement in treatment options [20] - The company plans to initiate a registration study for R289 in 2027 [24] Market Opportunities - The U.S. market for adult chronic ITP has approximately 81,000 patients, with Rigel targeting the second-line treatment population of about 24,000 patients [7] - The market for low-risk MDS is substantial, with current treatments showing limited efficacy, presenting an opportunity for R289 [18][19] Strategic Partnerships - Rigel has partnered with various companies for product distribution, including Grifols in Europe and Kissei in Asia for TAVALISSE [13] - Collaborations with MD Anderson and MyeloMatch are underway to explore additional indications for Olutasidenib and AML treatments [25][27] Future Outlook - Rigel aims to continue in-licensing differentiated assets in hematology and oncology to enhance its portfolio [14][16] - The company is committed to maintaining financial discipline while investing in its pipeline to drive growth [31][32] Key Takeaways - Rigel Pharmaceuticals is experiencing significant growth in its commercial products and is strategically positioning itself for future opportunities in hematology and oncology [5][32] - The focus on developing innovative treatments like R289 and expanding its product offerings through partnerships is central to Rigel's growth strategy [4][14][24]

Financial Data and Key Metrics Changes - The company reported total revenue of $69.5 million for Q3 2025, including record net product sales of $64.1 million, a 65% year-over-year increase [4][27] - Net income for the third quarter was $27.9 million, compared to $12.4 million for the same period in 2024 [29] - Cash balance increased to $137.1 million, up from $77.3 million at the end of 2024 [29] Business Line Data and Key Metrics Changes - TAVALISSE generated $44.7 million in net product sales, a 70% increase year-over-year [11][27] - GAVRETO achieved $11.1 million in net product sales, reflecting a 56% increase compared to Q3 2024 [12][27] - REZLIDHIA reported $8.3 million in net product sales, a 50% increase year-over-year [12][27] Market Data and Key Metrics Changes - The company has expanded access to TAVALISSE in Japan, Europe, Canada, Israel, and South Korea, with ongoing regulatory approvals in new markets [13] - The commercial portfolio has shown strong year-over-year growth, with net product sales exceeding $200 million on a trailing 12-month basis [11] Company Strategy and Development Direction - The strategic objectives focus on growing the hematology and oncology business through commercial performance and pipeline expansion [4] - The company is pursuing additional in-licensing deals or asset acquisitions to achieve significant growth [7] - The 2025 revenue guidance has been raised to $285-$290 million, reflecting anticipated growth of 55%-59% compared to 2024 [9][30] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to achieve significant long-term growth through strong commercial execution and financial discipline [9][32] - The company aims to continue driving sales of commercial products while funding key development programs in its internal pipeline [31][32] Other Important Information - The company is advancing its development pipeline, including the ongoing phase 1b study of R289 and collaborations for olutasidenib in various cancer studies [5][16] - The company has received fast-track designation and orphan drug designation from the FDA for R289, indicating a promising regulatory pathway [19] Q&A Session Summary Question: Competitive positioning of R289 in lower-risk MDS versus Rytelo - Management indicated it is too early to speculate on competitive positioning and plans for a registrational study [34] Question: Data needed for potential priority review of R289 - Management noted that the fast-track designation opens potential for priority review, but further data from the dose expansion study is needed [38] Question: Expectations for updated data at ASH for R289 - Management confirmed that updated data will include 16 weeks of follow-up on all patients, providing a robust data set for presentation [40]

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 reported strong third-quarter performance, leading to an increase in full-year 2025 revenue guidance due to outstanding commercial execution and pipeline development [2][10]. Financial Performance - Total revenues for Q3 2025 were $69.5 million, comprising $64.1 million in net product sales (up 65% from $38.9 million in Q3 2024) and $5.4 million in contract revenues [4][10]. - Net product sales for TAVALISSE reached $44.7 million (70% growth), GAVRETO at $11.1 million (56% growth), and REZLIDHIA at $8.3 million (50% growth) compared to the same period in 2024 [4][11]. - For the nine months ended September 30, 2025, total revenues were $224.5 million, with net product sales growing 69% year-over-year [7]. Costs and Expenses - Total costs and expenses for Q3 2025 were $41.0 million, slightly down from $41.3 million in Q3 2024, primarily due to lower costs of product sales [5][10]. - Research and development costs increased due to clinical activities related to olutasidenib and R289, along with higher personnel-related costs [5][8]. Net Income - Rigel reported a net income of $27.9 million for Q3 2025, compared to $12.4 million in Q3 2024, translating to $1.55 basic and $1.46 diluted earnings per share [6][10]. Cash Position - As of September 30, 2025, cash, cash equivalents, and short-term investments totaled $137.1 million, up from $77.3 million at the end of 2024 [9][10]. Clinical Development - Rigel is advancing its Phase 1b study of R289 for lower-risk MDS, with enrollment completed in the dose escalation phase and the first patient enrolled in the dose expansion phase [2][11]. - Updated data from the R289 study will be presented at the ASH Annual Meeting in December [10][11]. 2025 Outlook - The company updated its 2025 revenue guidance to approximately $285 to $290 million, an increase from the previous estimate of $270 to $280 million [12][24]. - Rigel anticipates reporting positive net income for the full year 2025 while funding existing and new clinical development programs [12].

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]

Core Insights - Rigel Pharmaceuticals (RIGL) is set to report its third-quarter 2025 results on November 4, with revenue expectations at $61.6 million and earnings at 93 cents per share [1][6] Group 1: Revenue Drivers - The primary revenue sources for Rigel include sales from marketed products and contract revenues from collaborations [2] - The significant revenue growth in Q3 is anticipated to be driven by strong demand for Tavalisse, which treats chronic immune thrombocytopenia (ITP) [2][6] - Sales from other marketed products, Rezlidhia (olutasidenib) and Gavreto (pralsetinib), are also expected to contribute positively to Rigel's revenue in the upcoming quarter [3][6] Group 2: Stock Performance - Year-to-date, Rigel's shares have increased by 88%, significantly outperforming the industry average rise of 7.3% [3] Group 3: Pipeline Developments - Rigel is advancing its pipeline with R289, a dual IRAK1 and IRAK4 inhibitor, currently in early-stage studies for lower-risk myelodysplastic syndrome (MDS) [4] - The company is also exploring additional uses for Rezlidhia beyond its current approval for relapsed or refractory IDH1-mutated acute myeloid leukemia (AML) [7] Group 4: Earnings Surprise History - Rigel has a strong history of earnings surprises, having exceeded earnings expectations in the last four quarters with an average surprise of 1,840.49% [8] Group 5: Earnings Prediction Model - The current model does not predict a definitive earnings beat for Rigel, as the Earnings ESP stands at 0.00% with both the Most Accurate Estimate and the Zacks Consensus Estimate at 93 cents per share [9][10]

Core Insights - Rigel Pharmaceuticals has initiated the dose expansion phase of the Phase 1b study for R289, targeting patients with transfusion-dependent relapsed or refractory lower-risk myelodysplastic syndrome (MDS) [1][2] - The study aims to determine the recommended Phase 2 dose (RP2D) of R289, a dual inhibitor of IRAK1/4, which has shown potential in addressing unmet medical needs in lower-risk MDS patients [2][3] 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 [5] - The company is publicly traded on Nasdaq under the ticker RIGL [5] Product Details - R289 is a prodrug of R835, designed to inhibit IRAK1/4, and has demonstrated the ability to block inflammatory cytokine production in preclinical studies [4] - The FDA has granted R289 Orphan Drug and Fast Track designations for the treatment of myelodysplastic syndromes and previously-treated transfusion-dependent lower-risk MDS, respectively [3] Study Design - The ongoing Phase 1b study includes a dose expansion phase where up to 40 patients will be randomized to receive either 500 mg of R289 once or twice daily [2] - The study's primary focus is on evaluating safety, tolerability, pharmacokinetics, and preliminary activity of R289 in the specified patient population [2]

Summary of Rigel Pharmaceuticals (RIGL) 2025 Conference Call Company Overview - Rigel Pharmaceuticals is a hematology and oncology company focused on expanding its product portfolio and profitability [2][4] Commercial Products - **Current Products**: - TAVALISSE: For immune thrombocytopenia (ITP) - Rezlidia: For IDH1-mutant relapsed/refractory acute myeloid leukemia (AML) - Gavreto: For RET fusion-positive non-small cell lung cancer and thyroid cancer [3][4] - **Financial Performance**: - Achieved $102 million in revenue for the first half of 2025, with Q2 showing a 76% growth compared to Q2 2024 [4] - TAVALISSE grew by 52% year-over-year, Rezlidia by 36% [16][17] Market Insights - **TAVALISSE**: - Target market includes approximately 81,000 patients in the U.S. with chronic ITP, with 24,000 patients eligible for treatment beyond the first line [5][6] - Early use of TAVALISSE shows significantly better patient outcomes (80-90% benefit in second and third lines) compared to later lines [8][9] - **Rezlidia**: - Addresses a significant unmet need in AML, with 22,000 new cases annually and a high mortality rate [10] - The product has a 35% response rate and a duration of response of nearly 26 months [11] - **Gavreto**: - Positioned to capture market share from existing treatments, with a recent guideline shift favoring RET fusion inhibitors [14] - Achieved $11.8 million in net sales in Q2, showing strong growth potential [15] Clinical Development - **R289**: A dual IRAK1/4 inhibitor targeting lower-risk myelodysplastic syndromes, with fast track and orphan drug designations from the FDA [20][23] - **Oludacitinib (Rezlidia)**: Ongoing studies in glioma and AML, with collaborations established to enhance research and development [28][31] Financial Position - The company reported a profit of $71 million in the first half of 2025, with a cash position increasing from $77 million to $108 million [36] - Raised revenue guidance for total revenue to $270-$280 million and net product sales to $210-$220 million [36][37] Growth Strategy - Rigel aims to continue growing its commercial products while advancing its development programs, particularly R289 and oludacitinib [39] - The company has maintained a 32% CAGR since 2021 and expects approximately 50% growth in 2025 [38] Partnerships and Collaborations - Rigel has established partnerships for international commercialization of its products, enhancing revenue through royalties and product sales [17][18] Conclusion - Rigel Pharmaceuticals is positioned for significant growth with a strong product pipeline, effective commercialization strategies, and a focus on addressing unmet medical needs in hematology and oncology [39]