Corporate Presentation August 6, 2024 1 Forward Looking Statements Please visit www.TAVALISSE.com for Full Prescribing Information. Please visit www.REZLIDHIA.com for Full Prescribing Information, including Boxed WARNING. Please visit www.GAVRETO.com for Full Prescribing Information. 2 Growing Our Hematology and Oncology Business Commercial Execution Development & Expansion Development Programs1 In-Licensing and Product Acquisition • Identify new late-stage assets which leverage current capabilities and cap ...

Rigel Pharmaceuticals (RIGL) 2025 Conference June 04, 2025 12:50 PM ET Speaker0 This is standard presentation. Welcome, Rahul. Thank you so much, Farshan. It's nice to see you and my thanks to our colleagues at Jefferies for having invited us again. I appreciate the opportunity. I'd like to tell you about Rigel and where we are. First, some important forward looking statements here. This is also available on our website, so please feel free to read this. Let me tell you where Rigel is right now. It's a pret ...

Core Insights - Rigel Pharmaceuticals is set to present seven posters at the 2025 ASCO Annual Meeting and EHA Congress, focusing on their hematology and oncology product portfolio, particularly GAVRETO® and REZLIDHIA® [1][2][3] Group 1: GAVRETO® (pralsetinib) - GAVRETO is being highlighted for its treatment of metastatic RET fusion-positive non-small cell lung cancer (NSCLC), with final data from the Phase 1/2 ARROW study demonstrating a 70.3% overall response rate (ORR) and a median duration of response (DOR) of 19.1 months [6][8] - The study also reported a median overall survival (OS) of 44.3 months and a median progression-free survival (PFS) of 13.1 months, with notable differences in PFS across regions: 25.9 months in the U.S. compared to 12.6 months in Asia and 12.9 months in Europe [6][8] - Additional data from the ARROW study indicated promising anti-tumor activity in various RET fusion-positive solid tumors, suggesting GAVRETO's potential to address unmet medical needs [3][6] Group 2: REZLIDHIA® (olutasidenib) - REZLIDHIA is being presented for its efficacy in relapsed or refractory acute myeloid leukemia (AML), with data supporting its use in earlier lines of treatment, particularly for patients with mutated isocitrate dehydrogenase-1 (mIDH1) [3][5] - In a pivotal cohort of R/R AML patients, REZLIDHIA showed a 50% ORR and a 30% complete response (CR) rate, with a median duration of CR of 17.6 months [12] - The drug demonstrated clinically meaningful activity and a durable response in patients with primary refractory AML, indicating its potential as an effective therapeutic option for this challenging patient population [12][19] Group 3: Clinical Data and Comparisons - A matching-adjusted indirect comparison (MAIC) analysis of olutasidenib versus ivosidenib in mIDH1 R/R AML showed comparable response rates, with olutasidenib favoring longer durations of CR [11] - Final results from the Phase 2 portion of the ARROW study in RET fusion-positive solid tumors other than NSCLC indicated an ORR of 46.4%, including a 100% ORR in pancreatic cancer [11] - The data validate RET fusions as a tissue-agnostic target, supporting the promising potential of pralsetinib to address unmet medical needs across various tumor types [11][12]

Financial Data and Key Metrics Changes - The company reported total revenue of $53.3 million for Q1 2025, with a year-over-year net product sales growth of 68% [4][40] - Net income for the quarter was $11.4 million, a significant improvement from a net loss of $8.2 million in the same period of 2024 [4][43] - The company anticipates total revenue for 2025 to be in the range of $200 million to $210 million, with net product sales expected to be approximately $185 million to $192 million [6][45] Business Line Data and Key Metrics Changes - TAVALISSE generated net product sales of $28.5 million, a 35% increase year-over-year [40] - GAVRETO, which became available in June 2024, reported net product sales of $9 million, reflecting a 15% year-over-year growth [10][40] - Reslidia achieved net product sales of $6.1 million, marking a 25% increase compared to the prior year [40] Market Data and Key Metrics Changes - The company is expanding access to its products in international markets, with TAVALISSE now available in Japan, Europe, Canada, and Israel [16] - Regulatory approvals for TAVALISSE have been received in Mexico and South Korea, indicating growth opportunities in these regions [16] Company Strategy and Development Direction - The company aims to continue focusing on commercial growth while maintaining financial discipline, with plans to expand its hematology and oncology portfolio through business development [6][19] - The company is committed to advancing its development pipeline, particularly with R289 and elutacitinib, and plans to initiate a Phase II study in recurrent glioma later this year [18][39] - The company has a strategic alliance with MD Anderson Cancer Center to broaden the development of elutacitinib into AML and MDS [36] Management's Comments on Operating Environment and Future Outlook - Management highlighted a strong start to the year despite a challenging business environment for the biotech market, emphasizing the robustness of the corporate strategy [4][6] - The company expects to report positive net income for the full year of 2025 while funding existing and new clinical development programs [45][47] Other Important Information - The company notified Lilly that it will not exercise its right to share in future development expenses for ogadusertib, which may impact future royalty rates [7] - The company reported a sequential decrease in net product sales from Q4 2024 due to inventory drawdowns across distribution channels [41] Q&A Session Summary Question: Clarification on the Target D program and its objectives - Management explained that the Target D program is an umbrella study for patients post-surgery and radiotherapy, while the company's own clinical study will target a different patient population [50][51] Question: Commentary on product sales and first-quarter resets - Management expressed satisfaction with the growth in demand for all three brands, indicating that they were prepared for changes related to the Inflation Reduction Act [60][63] Question: Expectations for R289 and dose escalation - Management confirmed that they are exploring split doses and will seek regulatory feedback on dose escalation data before advancing to the recommended Phase II dose [68][71] Question: Inquiry about ANDA filers and market entry - Management clarified that while there are no current ANDA filers, it is technically possible for second filers to enter the market if they can defeat existing patents [72][74]

Core Viewpoint - Rigel Pharmaceuticals reported strong financial results for Q1 2025, with significant revenue growth and net income, positioning the company for continued investment in its pipeline and commercial growth [2][5][6]. Financial Performance - Total revenues for Q1 2025 were $53.3 million, comprising $43.6 million in net product sales and $9.8 million in contract revenues, marking a 68% increase in net product sales compared to $26.0 million in Q1 2024 [5][9]. - Net income for the quarter was $11.4 million, or $0.64 per share, a turnaround from a net loss of $8.2 million in the same period of 2024 [9][26]. - The company anticipates total revenue for 2025 to be approximately $200 to $210 million, excluding $40 million in non-cash revenue expected in Q2 2025 [6][11]. Product Sales - TAVALISSE net product sales reached $28.5 million, a 35% increase from $21.1 million in Q1 2024 [5][7]. - GAVRETO, which became commercially available in June 2024, generated $9.0 million in net product sales [5][7]. - REZLIDHIA net product sales were $6.1 million, reflecting a 25% increase from $4.9 million in the same period of 2024 [5][7]. Clinical Development - The company is advancing its Phase 1b clinical study of R289 for patients with relapsed or refractory lower-risk myelodysplastic syndromes (MDS) [2][7]. - R289 has received Orphan Drug and Fast Track designations from the FDA for the treatment of MDS [6][7]. Corporate Developments - Rigel appointed Dr. Mark Frohlich to its Board of Directors as an independent director [7]. - The company entered a settlement agreement with Annora Pharma regarding patent litigation related to TAVALISSE, allowing Annora to sell a generic version by Q2 2032 under certain conditions [7][8]. - Rigel notified Eli Lilly that it will not exercise its opt-in right for the development of ocadusertib, expecting to recognize approximately $40 million in non-cash revenue in Q2 2025 as a result [7][8]. Cost Structure - Total costs and expenses for Q1 2025 were $40.6 million, up from $36.5 million in Q1 2024, primarily due to increased personnel and research and development costs [8][26].

Blueprint Medicines Corporation (BPMC) reported first-quarter 2025 adjusted loss of 74 cents per share, which was wider than the Zacks Consensus Estimate of a loss of 42 cents. The adjusted figure excludes the one-time equity investment gain of $50 million recorded in the reported quarter, following the closing of GSK's acquisition of IDRx. The company incurred a loss of $1.32 per share in the year-ago quarter.Quarterly revenues of $149.4 million, generated entirely from the net product sales of Ayvakit, mi ...