Core Insights - Rigel Pharmaceuticals presented five-year efficacy data for REZLIDHIA® (olutasidenib) at the EHA 2024 Hybrid Congress, demonstrating its effectiveness in treating relapsed or refractory (R/R) mutated isocitrate dehydrogenase-1 (mIDH1) acute myeloid leukemia (AML) [1][5][6] Group 1: Efficacy Data - The oral presentation highlighted that 35% of 147 efficacy evaluable patients achieved complete remission (CR) or CR with partial hematologic recovery (CRh), with a median time to CR/CRh of 1.9 months and a median duration of 25.3 months [6] - In patients previously treated with venetoclax, 33% achieved CR/CRh, with a median overall survival of 16.2 months [7] - The overall response rate (ORR) was 48%, with a median duration of 15.5 months and a maximum duration ongoing at 54.6 months [6][9] Group 2: Safety and Tolerability - Olutasidenib was reported to have a well-characterized and manageable safety profile, with differentiation syndrome occurring in 16% of patients [13][14] - The treatment was generally well tolerated in elderly patients, with 31% achieving CR/CRh in a subgroup analysis of patients aged 75 and older [10] Group 3: Subgroup Analyses - Data presented included the efficacy of olutasidenib in elderly patients, those who failed prior venetoclax treatment, and as a bridge to allogeneic hematopoietic stem cell transplantation (HSCT) [4][10][11] - In patients with mIDH1 AML secondary to myeloproliferative neoplasms (MPN), 40% achieved CR, with a median duration of response of 15.6 months [8][11] Group 4: Future Implications - The data supports the potential of REZLIDHIA in treating patient populations with limited treatment options, particularly those with mIDH1 AML secondary to MPN [5][12] - The findings suggest that olutasidenib may serve as a bridge to allogeneic stem cell transplantation for previously ineligible patients, with 75% of those proceeding to transplant achieving CR/CRh prior to the procedure [11]

Core Insights - Rigel Pharmaceuticals presented data on REZLIDHIA® (olutasidenib) at the 2024 ASCO Annual Meeting, highlighting its long-term efficacy in treating relapsed or refractory (R/R) mutated isocitrate dehydrogenase-1 (mIDH1) acute myeloid leukemia (AML) patients, including those previously treated with venetoclax [1][2][4] - The data showed that olutasidenib achieved a complete remission (CR) or CR with partial hematologic recovery (CRh) in 35% of efficacy evaluable patients, with a median duration of CR/CRh of 25.3 months [4] - The ongoing Phase 1b trial of R289, a dual inhibitor of IRAK1 and IRAK4, is also being presented, targeting lower-risk myelodysplastic syndrome (LR-MDS) patients [2][8] Group 1: REZLIDHIA® Efficacy and Safety - The five-year results from the Phase 2 trial of olutasidenib demonstrated durable responses in heavily pretreated patients with mIDH1 AML, with an overall response rate of 48% and median overall survival of 11.6 months [4][5] - In a subgroup analysis of elderly patients aged 75 and older, 31% achieved CR/CRh, with a median duration of 25.9 months [5][6] - Among patients who were R/R to prior venetoclax, 33% achieved CR/CRh, with a median overall survival of 16.2 months [4] Group 2: R289 Trial Overview - R289 is being evaluated in a Phase 1b trial for patients with LR-MDS, an area identified as having high unmet medical needs [2][8] - The trial aims to assess the safety and preliminary efficacy of R289, with multiple dose levels already completed and active recruitment ongoing [11][12] - R289 is a prodrug of R835, which has shown potential in blocking inflammatory cytokine production linked to persistent cytopenias in lower-risk MDS patients [8]

Company Overview - Rigel Pharmaceuticals, Inc. is a biotechnology company focused on discovering, developing, and providing novel therapies for patients with hematologic disorders and cancer [3] - The company was founded in 1996 and is headquartered in South San Francisco, California [3] Upcoming Event - Raul Rodriguez, the president and CEO of Rigel, will present a company overview at the Jefferies Global Healthcare Conference on June 6, 2024, at 9:30 am ET in New York [1] - The presentation will be accessible via a live webcast, and interested parties are encouraged to connect to Rigel's website prior to the event for any necessary software downloads [2]

Group 1 - Rigel Pharmaceuticals announced upcoming presentations at the 2024 ASCO Annual Meeting and EHA 2024 Congress, showcasing their hematology and oncology portfolio [1][2] - Presentations will include data on REZLIDHIA® (olutasidenib) for mutated IDH1 acute myeloid leukemia (AML) and TAVALISSE® (fostamatinib) for chronic immune thrombocytopenia (ITP), along with a Phase 1b trial of R2891 [2][3] - The five-year data from the Phase 2 trial of REZLIDHIA indicates rapid and durable responses, particularly in difficult-to-treat populations, including elderly patients [3][6][7] Group 2 - TAVALISSE demonstrated efficacy as a second-line therapy for ITP, allowing successful tapering of corticosteroids while maintaining platelet counts above 50,000/μL [4][10] - The ASCO and EHA presentations will include various abstracts highlighting the efficacy and safety of olutasidenib in different patient populations, including those with relapsed or refractory AML [5][8][11] - Observational studies provide real-world insights into the use of fostamatinib in ITP patients, emphasizing the need for additional treatment options [10][15] Group 3 - Rigel Pharmaceuticals focuses on developing therapies for hematologic disorders and cancer, with a commitment to improving patient outcomes [36] - The company has a robust pipeline, including investigational compounds like R289, which targets IRAK1 and IRAK4 for lower-risk myelodysplastic syndrome [16][36] - Rigel's marketed products and ongoing clinical trials reflect its dedication to addressing unmet medical needs in hematology and oncology [36][37]

Financial Data and Key Metrics Changes - For Q1 2024, Rigel Pharmaceuticals reported net product sales of TAVALISSE at $21.1 million, a 5% decrease compared to the same period in 2023, primarily due to a reduction in inventory levels at distributors [83][84] - REZLIDHIA generated $4.9 million in net sales for Q1 2024, significantly up from $1.5 million in the same period last year, reflecting strong growth since its launch in December 2022 [84] - Total cost and expenses for Q1 2024 were $36.5 million, down from $38.8 million in the same period for 2023, mainly due to decreased research and development costs [91] Business Line Data and Key Metrics Changes - TAVALISSE achieved its sixth consecutive quarterly record high for bottles shipped, with a 10% year-over-year growth in demand for Q1 2024 [18] - REZLIDHIA saw a 17% growth in bottles shipped compared to Q4 2023, nearly tripling the demand from the same period last year [23] - The acquisition of GAVRETO is expected to enhance the commercial product portfolio, with anticipated top-line growth and synergies with existing products [11][16] Market Data and Key Metrics Changes - GAVRETO generated $28 million in US net product sales in 2023, and the company expects to begin shipping GAVRETO to patients and clinics in July 2024 [15][42] - The market for RET fusion-positive non-small cell lung cancer is projected to expand, with approximately 3,000 new patients identified annually in the US [33][52] Company Strategy and Development Direction - The company aims to grow its Hematology and Oncology business, focusing on increasing sales of TAVALISSE and REZLIDHIA while integrating GAVRETO into its operations [94] - Rigel is pursuing additional in-licensing deals and acquisitions to expand its product portfolio, similar to its strategies with REZLIDHIA and GAVRETO [95] - The company is advancing its development programs, including olutasidenib for various cancers and R289 for lower-risk MDS, through collaborations with MD Anderson Cancer Center and the CONNECT Consortium [60][62] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving financial breakeven, with expectations of revenue growth from the launch of GAVRETO and continued demand for existing products [102] - The company is focused on maintaining a disciplined financial approach while investing in clinical trials and expanding its product pipeline [93][95] - Management highlighted the importance of educating clinicians about the efficacy of GAVRETO and olutasidenib, particularly in underserved patient populations [39][60] Other Important Information - The company ended Q1 2024 with cash, cash equivalents, and short-term investments of $49.6 million [93] - Rigel has entered into an amendment to its credit agreement, extending the maturity date and interest-only period by one year [93] Q&A Session Summary Question: Cash guidance and path to breakeven - The company is burning about $8 million a quarter and is focused on increasing revenues while managing operational expenses to reach breakeven [100][102] Question: Brain activity for GAVRETO and olutasidenib - Both GAVRETO and olutasidenib have shown the ability to cross the blood-brain barrier, which is critical for their therapeutic effects [104][106] Question: Balancing current pipeline and appetite for acquisitions - The company is excited about the growth of TAVALISSE and REZLIDHIA, and sees opportunities for further clinical trials and acquisitions to enhance its pipeline [110][114]

Financial Performance - Total revenue for Q1 2024 was $29.5 million, including $21.1 million from TAVALISSE and $4.9 million from REZLIDHIA[5]. - Total revenues for Q1 2024 increased to $29,534,000, up 13.5% from $26,070,000 in Q1 2023[51]. - Product sales, net rose to $26,003,000, compared to $23,745,000 in the same period last year, reflecting an increase of 9.5%[51]. - Contract revenues from collaborations grew significantly to $3,531,000, up 51.8% from $2,325,000 in Q1 2023[51]. - Total costs and expenses for Q1 2024 were $36.5 million, a decrease from $38.8 million in the same period of 2023[10]. - Total costs and expenses decreased to $36,500,000, down 5.7% from $38,795,000 in Q1 2023[51]. - Rigel's net loss for Q1 2024 was $8.2 million, or $0.05 per share, an improvement from a net loss of $13.5 million, or $0.08 per share, in Q1 2023[11]. - Net loss for Q1 2024 was $8,247,000, an improvement of 39.5% compared to a net loss of $13,536,000 in Q1 2023[51]. - Net loss per share improved to $0.05, compared to $0.08 in the prior year[51]. - Rigel's cash, cash equivalents, and short-term investments totaled $49.6 million as of March 31, 2024, down from $56.9 million at the end of 2023[12]. - Cash, cash equivalents, and short-term investments decreased to $49,550,000 from $56,933,000 as of December 31, 2023[53]. - Total assets increased to $126,519,000, up from $117,225,000 at the end of 2023[53]. - Stockholders' deficit widened to $31,671,000 from $28,644,000 as of December 31, 2023[53]. - Stock-based compensation expense included in selling, general and administrative expenses was $4,484,000, significantly higher than $1,735,000 in Q1 2023[51]. Product Sales and Market Performance - TAVALISSE sales volume reached 2,193 bottles, the highest quarterly sales since launch, while REZLIDHIA sales grew significantly with 390 bottles sold[6]. - The company expanded its portfolio with the acquisition of GAVRETO, expected to start recognizing product sales in July 2024[5]. - Contract revenue from collaborations amounted to $3.5 million, with contributions from Kissei Pharmaceutical, Grifols, and Medison Pharma[9]. Clinical Development and Trials - Rigel is advancing its Phase 1b clinical trial for R289, a novel IRAK1/4 inhibitor, with preliminary data expected by the end of 2024[8]. - The company announced a strategic collaboration with CONNECT to evaluate REZLIDHIA in combination with temozolomide for high-grade glioma patients[7]. - Rigel Pharmaceuticals is focused on the commercialization of olutasidenib and pralsetinib for various cancers, with ongoing clinical trials[48]. Adverse Reactions and Safety - TAVALISSE patients experienced serious adverse reactions at a rate of 1%, with severe reactions including dyspnea and hypertension at 2%[31]. - Hepatotoxicity occurred in 23% of patients treated with REZLIDHIA, with 13% experiencing grade 3 or 4 hepatotoxicity[32]. - GAVRETO showed a 12% incidence of pneumonitis, with 3.3% classified as Grade 3-4 and 0.2% resulting in fatal reactions[43]. - 35% of GAVRETO patients experienced hypertension, with 18% having Grade 3 hypertension[43]. - Increased AST levels were observed in 49% of GAVRETO patients, with 7% experiencing Grade 3 or 4 increases[43]. - The median time to onset for increased AST in GAVRETO patients was 15 days, with a range from 5 days to 2.5 years[43]. - Common adverse reactions for GAVRETO included musculoskeletal pain, constipation, and fatigue, occurring in ≥25% of patients[46]. - The company advises against the use of REZLIDHIA with strong or moderate CYP3A inducers due to potential drug interactions[40]. - Rigel Pharmaceuticals emphasizes the importance of monitoring liver function tests for patients on REZLIDHIA, especially during the first two months of treatment[33]. Leadership and Management - Rigel appointed Lisa Rojkjaer, M.D. as Chief Medical Officer, bringing over 20 years of experience in clinical development and oncology[6].

Financial Performance - For the three months ended March 31, 2024, net product sales of TAVALISSE were $21.1 million, a decrease of $1.2 million or 5% compared to $22.3 million in the same period in 2023[123]. - Net product sales of REZLIDHIA for the same period were $4.9 million, an increase of $3.4 million compared to $1.5 million in the same period in 2023, primarily due to increased quantities sold[124]. - Total revenues for the three months ended March 31, 2024, were $29.534 million, an increase of $3.464 million or 13.3% compared to $26.070 million in the same period in 2023[199]. - Cost of product sales for the three months ended March 31, 2024, was $2.025 million, an increase of $1.048 million compared to $977,000 in the same period in 2023[202]. - Revenue from collaborations for the three months ended March 31, 2024, included $2.3 million from Kissei, $1.1 million from Grifols, and $0.1 million from Medison[200]. Product Development and Trials - The company has a Phase 1b trial for its IRAK1/4 inhibitor program, which is expected to enroll approximately 40 patients, with preliminary data anticipated by the end of 2024[137]. - The company has strategic collaborations to expand the evaluation of REZLIDHIA in AML and other hematologic cancers[122]. - A collaboration with CONNECT was announced to conduct a Phase 2 clinical trial for REZLIDHIA in HGG, with funding of up to $3.0 million over four years[139]. - The Phase 2 registrational trial of REZLIDHIA demonstrated a CR+CRh rate of 35% and a median duration of overall response of 11.7 months[173]. - The major portion of total research and development expense in Q1 2024 was associated with the IRAK 1/4 inhibitor program[214]. Strategic Agreements and Collaborations - The company entered into an Asset Purchase Agreement to acquire GAVRETO (pralsetinib) for $15.0 million, with $10.0 million payable upon the first commercial sale and an additional $5.0 million on the first anniversary of the agreement[127]. - The company entered into a Strategic Collaboration Agreement with MDACC to evaluate REZLIDHIA (olutasidenib) in AML and other hematologic cancers, providing $15.0 million in milestone payments over five years[138]. - The company has established a reimbursement and patient support program called Rigel OneCare (ROC) to assist eligible patients with access to TAVALISSE[158]. - The commercialization license agreement with Grifols for fostamatinib includes exclusive rights in Europe, the UK, and other regions[160]. - The company entered into a commercial license agreement with Knight Therapeutics for fostamatinib in Latin America, receiving a $2.0 million upfront payment and potential for up to $20.0 million in milestones[169]. Financial Position and Cash Flow - As of March 31, 2024, the company had approximately $49.6 million in cash, cash equivalents, and short-term investments, down from $56.9 million at the end of 2023[222]. - Net cash used in operating activities was $(5,013,000) in Q1 2024 compared to $(4,074,000) in Q1 2023, reflecting an increase in cash outflow of $939,000[223]. - The company anticipates significant additional funding requirements in the foreseeable future, potentially impacting its ability to operate as a going concern[231]. - The company has a fully funded $60.0 million term loan credit facility with MidCap as of March 31, 2024[230]. - The outstanding principal amount of the loan under the credit facility with MidCap was $60.0 million[242]. Market and Competitive Landscape - GAVRETO is one of only two approved RET inhibitors on the market, with FDA approvals for metastatic RET fusion-positive NSCLC and advanced thyroid cancer[130]. - The competitive landscape includes TIBSOVO® (ivosidenib) as the only other approved product in the US for patients with IDH1 mutation[182]. - TAVALISSE is competing with existing therapies for ITP, including corticosteroids and thrombopoietin receptor agonists[151]. - REZLIDHIA was added to the NCCN Clinical Practice Guidelines for AML as a recommended targeted therapy in January 2023[177]. Sales and Marketing - TAVALISSE's sales decrease was partly due to a reduction in distributor inventory and increased revenue reserves from higher government and private payor rebates[123]. - The company plans to commercialize GAVRETO later in 2024, which is expected to contribute to future revenues[201]. - The company expects to incur significant selling, general and administrative expenses as it expands commercial activities for existing products and prepares for the commercialization of GAVRETO later this year[216]. Research and Development Expenses - Research and development expense decreased to $6,026,000 in Q1 2024 from $10,089,000 in Q1 2023, a reduction of $4,063,000 or 40.3%[204]. - The decrease in research and development expense was partly due to a $1.1 million reduction in clinical trial-related expenses and a $1.0 million decrease in personnel-related costs[205]. - Selling, general and administrative expense increased to $28,449,000 in Q1 2024 from $27,729,000 in Q1 2023, an increase of $720,000 or 2.6%[215].

Financial Data and Key Metrics Changes - The company reported a net income of $737,000 for Q4 2023, attributed to increased net product sales and disciplined operating expense management [8][12][84] - Total net product sales reached nearly $104 million for the full year 2023, representing a 36% growth over 2022 [19][68] - The company ended Q4 2023 with cash, cash equivalents, and short-term investments of $56.9 million [11] Business Line Data and Key Metrics Changes - TAVALISSE generated approximately $94 million in net sales for 2023, a 24% increase over the prior year [68] - REZLIDHIA contributed $10.6 million in net product sales for 2023, with expectations for continued strength in 2024 [84] - GAVRETO generated $28 million in U.S. net product sales in 2023, with a 21% increase from 2022 [30] Market Data and Key Metrics Changes - The company is focusing on expanding its presence in the non-small cell lung cancer market, with approximately 235,000 lung cancer patients expected to be diagnosed in 2024 [35] - The incidence of RET fusion-positive non-small cell lung cancer is estimated at around 3,000 patients annually in the U.S. [35][71] Company Strategy and Development Direction - The company aims to leverage its existing commercial and medical affairs infrastructure to drive growth, particularly with the acquisition of GAVRETO [20][21] - Strategic alliances with MD Anderson and CONNECT are expected to enhance the evaluation of REZLIDHIA in various cancers [22][87] - The company is actively pursuing additional in-licensed deals and acquisitions to expand its product portfolio [90] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory of TAVALISSE and REZLIDHIA, anticipating continued momentum in 2024 [88] - The company is focused on transitioning GAVRETO into its commercial operations and expects to recognize revenue from it in Q3 2024 [82][84] - Management highlighted the importance of maintaining a disciplined financial approach to achieve financial breakeven [90] Other Important Information - The company plans to complete the transition of the GAVRETO asset to Rigel and expects to incur certain costs associated with this new product [11] - The company reported a decrease in total costs and expenses to $33.8 million in Q4 2023, down from $49.2 million in the same period in 2022 [12][83] Q&A Session Summary Question: Update on GAVRETO's post-marketing requirements and studies - Management confirmed that the AcceleRET study was not necessary for U.S. approval, and discussions with the FDA regarding the thyroid cancer indication are ongoing [100] Question: Synergies with existing sales infrastructure for GAVRETO - Management noted that over half of the sales team has lung cancer experience, which will be leveraged to efficiently promote GAVRETO alongside existing products [102][106] Question: Barriers to adoption and strategies for switching patients to GAVRETO - Management acknowledged the challenge of familiarity with other drugs but expressed confidence in raising awareness and promoting GAVRETO's efficacy to drive adoption [114]

Revenue Performance - Total revenues for 2023 were $116.9 million, a decrease of $3.4 million or 2.8% from $120.2 million in 2022[584]. - Total revenues decreased to $116.882 million in 2023 from $120.242 million in 2022, primarily due to a decline in contract revenues from collaborations[657]. - The company incurred a net loss of $25.091 million in 2023, a significant improvement from a net loss of $58.573 million in 2022[657]. - The company reported a net loss of $25,091,000 for the year ended December 31, 2023, compared to a net loss of $58,573,000 in 2022, indicating a 57.3% improvement in losses year-over-year[666]. Product Sales - Product sales, net for 2023 were $104.3 million, an increase of $27.6 million or 36% compared to $76.7 million in 2022[584]. - TAVALISSE net product sales in 2023 were $93.7 million, up by $17.9 million or 24% from $75.8 million in 2022[584]. - REZLIDHIA net product sales in 2023 were $10.6 million, an increase of $9.7 million compared to $0.9 million in 2022[584]. - The company reported net product sales of $104.294 million for the year ended December 31, 2023, an increase of 36% compared to $76.718 million in 2022[657]. Collaboration and Contract Revenues - Contract revenues from collaborations in 2023 were $11.5 million, a decrease of $27.5 million or 70.6% from $39.0 million in 2022[585]. - Revenue from Grifols in 2023 was $8.8 million, primarily related to drug supplies and earned royalty[585]. - The company has potential future contingent payments exceeding $1.3 billion under existing collaboration agreements, with $279.5 million related to development events and $796.0 million to commercial events[743]. - The global exclusive license agreement with Lilly includes up to $330 million in milestone payments for non-CNS disease products and up to $255 million for CNS disease products[746]. Expenses and Costs - Cost of product sales increased to $7.1 million in 2023 from $1.7 million in 2022, primarily due to higher amortization and royalty expenses related to REZLIDHIA[590][591]. - Research and development expense decreased to $24.5 million in 2023 from $60.3 million in 2022, driven by reduced clinical trial expenses and personnel-related costs[592][593]. - Selling, general and administrative expenses were $105.7 million in 2023, a decrease from $112.5 million in 2022, attributed to lower stock-based compensation and various operational cost reductions[603][604]. - Stock-based compensation expense for 2023 was $8,806,000, a decrease from $12,385,000 in 2022, reflecting a 28.5% reduction[666]. Cash Flow and Investments - As of December 31, 2023, the company had approximately $56.9 million in cash, cash equivalents, and short-term investments, a slight decrease from $58.2 million in 2022[610]. - Net cash used in operating activities for 2023 was $(5,743) thousand, a significant improvement from $(73,758) thousand in 2022[611]. - The company received a $20.0 million regulatory milestone payment from Kissei in January 2023, contributing to cash flow from operating activities[612]. - Cash and cash equivalents increased to $32.786 million as of December 31, 2023, compared to $24.459 million in 2022[655]. Liabilities and Financial Commitments - The total liabilities decreased slightly to $145.869 million in 2023 from $147.895 million in 2022[655]. - The company has a credit facility with MidCap, with an outstanding principal amount of $60.0 million as of December 31, 2023, maturing on September 1, 2026[620]. - The company is responsible for funding development costs for R552 up to $22.6 million through April 1, 2024, with $18.6 million billed by Lilly as of December 31, 2023[628]. - Future interest payments associated with the credit facility are estimated at $9.4 million, with approximately $4.4 million payable within 12 months[634]. Clinical Development and Strategic Initiatives - The company is advancing its IRAK1/4 inhibitor program in a Phase 1b trial for patients with lower-risk MDS[566]. - Strategic collaborations with MD Anderson and CONNECT are ongoing to evaluate REZLIDHIA in AML and pediatric patients with HGG[568]. - The company has a RIPK1 inhibitor program in clinical development with partner Lilly[569]. - The company entered into an Asset Purchase Agreement with Blueprint in February 2024 to acquire rights to GAVRETO for development and commercialization in the US[567]. Revenue Recognition and Accounting Policies - Revenue from product sales is recognized when specialty distributors obtain control of the product, with provisions for government-mandated rebates and chargebacks[681]. - The company recognizes revenues from upfront license fees when the license is transferred to the licensee and the licensee can use and benefit from the license[691]. - Future variable consideration related to regulatory and commercial milestones is fully constrained due to uncertainty of success, impacting revenue recognition[759]. - The company will recognize revenue from sales-based milestones and royalties when related sales occur or when performance obligations are satisfied[760].

Rigel Pharmaceuticals, Inc. (NASDAQ:RIGL) Q3 2023 Earnings Conference Call November 7, 2023 4:30 PM ET Company Participants Ray Furey - Executive Vice President, General Counsel & Corporate Secretary Raul Rodriguez - President & Chief Executive Officer Dean Schorno - Chief Financial Officer Dave Santos - EVP & Chief Commercial Officer Conference Call Participants Joe Pantginis - H.C. Wainwright Operator Greetings, and welcome to Rigel Pharmaceuticals Financial Conference Call for the Third Quarter of 2023. ...