Please visit www.TAVALISSE.com for Full Prescribing Information. Please visit www.REZLIDHIA.com for Full Prescribing Information, including Boxed WARNING. Ray Furey, J.D. Dean Schorno Commercial Execution Development & Expansion • Evaluating additional olutasidenib and fostamatinib opportunities Partnered Programs 5 rigel. Select Important Safety Information 6 Please see Important Safety Information on slide 27. Please visit www.TAVALISSE.com for Full Prescribing Information. $24.5M 500 1000 1500 2000 2500 ...

Financial Performance - For the nine months ended September 30, 2023, net product sales of TAVALISSE were $68.1 million, a 26% increase compared to the same period in 2022[105]. - Net product sales for the nine months ended September 30, 2023, rose by 39% to $74,755,000 from $53,935,000 in the prior year[186]. - Product sales for the three months ended September 30, 2023, increased by 41% to $27,129,000 compared to $19,188,000 in the same period of 2022[186]. - TAVALISSE net product sales increased by $5.3 million or 27% in Q3 2023 and $14.1 million or 26% in the nine months of 2023 compared to the same periods in 2022[186]. - REZLIDHIA generated net product sales of $2.7 million and $6.7 million for the three and nine months ended September 30, 2023, respectively[186]. - Interest income increased to $1.6 million for the nine months ended September 30, 2023, compared to $0.3 million in the same period of 2022, reflecting a rise of approximately 533%[214]. - Interest expense rose to $5.0 million for the nine months ended September 30, 2023, from $2.6 million in the same period of 2022, an increase of approximately 92%[215]. Research and Development - The Phase 1b trial of R289 is expected to enroll approximately 22 patients, with preliminary results anticipated by mid-year 2024[109]. - Lilly has initiated a Phase 2a trial for R552, which plans to enroll 100 patients globally, focusing on moderately to severely active rheumatoid arthritis[110]. - The primary endpoint of the FOCUS Phase 3 clinical trial for fostamatinib in hospitalized COVID-19 patients was met after correcting a statistical error, showing lower mean days on oxygen compared to placebo (4.8 vs. 7.6 days, p=0.0136)[112]. - The NIH/NHLBI recommended ceasing enrollment in the fostamatinib study arm of the ACTIV-4 Host Tissue Trial due to low likelihood of benefit for primary outcomes[115]. - The Phase 2 trial of olutasidenib showed an overall response rate (ORR) of 46% and a median overall survival of 10.5 months for patients with CR/CRh[143]. - The Phase 2 trial data for fostamatinib in wAIHA was published in the American Journal of Hematology, highlighting its potential to increase hemoglobin levels in nearly half of the patients[170]. - The FORWARD Phase 3 clinical trial for wAIHA did not demonstrate statistical significance in the primary efficacy endpoint of durable hemoglobin response in the overall study population[174]. Product Development and Commercialization - The company paid an upfront fee of $2.0 million for the in-licensing of olutasidenib, with potential additional payments of up to $67.5 million upon achieving specified development and regulatory milestones[107]. - The commercialization agreement with Grifols includes an upfront cash payment of $30 million and potential regulatory and commercial milestones of up to $297.5 million[131]. - The agreement with Medison for Canada and Israel includes an upfront payment of $5 million and potential milestones of approximately $35 million[135]. - REZLIDHIA demonstrated a complete remission (CR) plus complete remission with partial hematologic recovery (CRh) rate of 35% in patients with relapsed or refractory acute myeloid leukemia (R/R AML) with mIDH1 mutations[140]. - The FDA approved REZLIDHIA for R/R AML with IDH1 mutation on December 1, 2022, and commercialization began on December 22, 2022[141]. - Commercial activities for REZLIDHIA will focus on healthcare providers managing R/R AML patients, with plans for collaborations outside the US[153]. Financial Position and Cash Flow - Cash and cash equivalents as of September 30, 2023, were approximately $62.4 million, up from $58.2 million as of December 31, 2022[221]. - Net cash provided by operating activities for the nine months ended September 30, 2023, was $0.5 million, a significant recovery from a net cash used of $51.8 million in the same period of 2022[222]. - Net cash provided by investing activities for the nine months ended September 30, 2023 was $17.4 million, a decrease from $54.0 million in the same period of 2022[223]. - Net cash provided by financing activities for the nine months ended September 30, 2023 was $20.0 million from term loan financing, compared to $19.5 million in the same period of 2022[224]. - The company expects to incur significant selling, general, and administrative expenses as it expands its commercial activities[213]. - The company may need to raise additional funds through public or private offerings, which could lead to substantial dilution of shareholders' ownership interests[233]. Regulatory and Market Considerations - The competitive landscape for TAVALISSE includes existing therapies and potential generic versions that could impact revenue[127]. - The competitive landscape includes TIBSOVO® (ivosidenib), which is also approved for patients with IDH1 mutation, highlighting the need for effective differentiation in marketing strategies[152]. - The company retains global rights to fostamatinib outside of specific territories under various license agreements[129]. - The company has not sold any shares of common stock under the Open Market Sale Agreement, which allows for sales up to $250.0 million[229].

Financial Data and Key Metrics Changes - The company achieved net sales of $21.3 million for TAVALISSE in Q2 2023, representing a 15% year-over-year increase from the same quarter last year [15][56] - The gross product sales for TAVALISSE were $30.2 million, with 2,265 bottles shipped to patients and clinics [55][56] - For REZLIDHIA, net product sales reached $2.6 million in Q2 2023, with a total launch-to-date sales of $4.9 million [30][49] Business Line Data and Key Metrics Changes - TAVALISSE saw a 10% growth in the number of bottles shipped compared to Q2 2022, marking three consecutive record highs since its launch [14][15] - REZLIDHIA shipped 200 bottles in Q2 2023, representing a 77% growth over Q1 [30][56] - The company is focusing on expanding TAVALISSE's use in earlier lines of therapy, with over 70% of new patients being treated in the second or third line [26][90] Market Data and Key Metrics Changes - The American Cancer Society estimates over 20,000 patients will be diagnosed with AML in 2023, with approximately 1,000 patients eligible for REZLIDHIA treatment each year [29][30] - The company is targeting a significant portion of the chronic ITP market, with over 80,000 patients estimated to be affected [25][26] Company Strategy and Development Direction - The company is focused on growing its hematology/oncology business through the development of existing products and exploring new indications [7][42] - There is an emphasis on building awareness for REZLIDHIA through promotional activities and scientific publications, particularly in academic institutions [18][32] - The company is evaluating potential in-licensing opportunities that align with its existing heme/onc infrastructure [38][86] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory of TAVALISSE and REZLIDHIA, with expectations for continued strength in net product sales [51][57] - The company is committed to achieving profitability while also investing in new opportunities for fostamatinib and olutasidenib [69][70] - Management highlighted the importance of addressing unmet needs in the treatment landscape for relapsed or refractory AML [17][34] Other Important Information - The company ended the quarter with cash, cash equivalents, and short-term investments of $64.4 million [61] - The gross to net adjustment for TAVALISSE and REZLIDHIA was approximately 29.5% and 20.7% of gross product sales, respectively [49][57] Q&A Session Summary Question: Path to profitability and managing expenses - Management discussed the importance of balancing growth with investments in new opportunities while managing operating expenses effectively to reach profitability [64][69] Question: Compliance and response rates among treatment lines - Management noted that while specific response rates cannot be determined, there is a positive trend in patient persistency for TAVALISSE [75][76] Question: Future opportunities for REZLIDHIA - Management indicated interest in exploring IDH positive patients in AML and other indications outside of AML for REZLIDHIA [78][79] Question: In-licensing discussions - Management emphasized the focus on late-stage heme/onc opportunities that align with their existing capabilities, but specifics could not be disclosed until contracts are finalized [84][86] Question: TAVALISSE market penetration - Management acknowledged the growth in TAVALISSE usage in earlier treatment lines and expressed optimism about continuing to expand this segment [90][91]

Forward Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1955 ("PSLRA") relating to, among other things, the potential and market opportunity of olutasidenib as therapeutics for R/R AML and other conditions; the potential and market opportunity for fostamatinib as therapeutics for chronic ITP, COVID-19 and other conditions; the regulatory approval and commercialization of fostamatinib or olutasidenib in the U.S. an ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED JUNE 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 (650) 624-1100 (Registrant's telephone number, including area code) Securities registered pursuant to Section 12(b) of the Act: | Title of each class: | Trading Symbol | Na ...

Financial Data and Key Metrics Changes - The company reported net sales of TAVALISSE at $22.3 million for Q1 2023, representing a 38% year-over-year increase compared to the same quarter last year [31][41] - REZLIDHIA generated net sales of $1.5 million in Q1 2023, with total product sales since launch reaching $2.3 million [15][42] - Total cost and expenses decreased to $38.8 million from $43 million in the same period for 2022, primarily due to reduced research and development costs [46] Business Line Data and Key Metrics Changes - TAVALISSE achieved a record shipment of 2,256 bottles in Q1 2023, marking a 23% growth over Q1 2022 [29] - REZLIDHIA shipped 109 bottles to patients in clinics during Q1, with a total of 111 bottles shipped since its launch [13][41] - The company shipped 113 bottles of REZLIDHIA to its distribution network, resulting in gross product sales of $1.8 million [41] Market Data and Key Metrics Changes - The American Cancer Society estimates over 20,000 patients will be diagnosed with AML in 2023, with 6% to 9% having the IDH1 mutation, representing a near-term opportunity for REZLIDHIA [11][12] - Approximately 90% of Medicare lives have confirmed published coverage for REZLIDHIA, indicating strong market access [22] Company Strategy and Development Direction - The company is focused on expanding its hematology-oncology business through internal development programs and in-licensing opportunities [8][35] - The launch of REZLIDHIA is being supported by a specialized institutional team targeting key leukemia treaters and academic accounts [76] - The company aims to continue growing TAVALISSE sales while executing the launch of REZLIDHIA [50] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong start to 2023, highlighting the continued momentum for TAVALISSE and the successful launch of REZLIDHIA [5][49] - The company is optimistic about achieving cash flow breakeven and is focused on revenue growth [57][58] Other Important Information - The company ended the quarter with cash, cash equivalents, and short-term investments of $58.7 million, having accessed an additional $20 million term loan [48] - The gross to net adjustment for TAVALISSE and REZLIDHIA was approximately 29% and 20% of gross product sales, respectively [42][44] Q&A Session Summary Question: Changes in partnered program with Eli Lilly regarding RIPK1 inhibitor - Management clarified that Eli Lilly has always been interested in multiple indications for the RIPK1 inhibitor, with a current focus on rheumatoid arthritis due to market opportunities [52][53] Question: Future cash burn expectations - The company ended Q1 with $58.7 million in cash and is focused on achieving cash flow breakeven, with a path towards that goal [56][58] Question: Progress on REZLIDHIA prescriber base and expectations - Management reported approximately 29 prescribers for REZLIDHIA and expressed optimism about increasing prescriber breadth as awareness grows [71][73] Question: Key factors for sustaining momentum in TAVALISSE - Management highlighted the importance of ongoing awareness efforts among clinicians and the potential for continued growth as patients fail other therapies [80][81]

Q1 2023 Financial Results Presentation This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1955 ("PSLRA") relating to, among other things, the potential and market opportunity of olutasidenib as therapeutics for R/R AML and other conditions; the potential and market opportunity for fostamatinib as therapeutics for chronic ITP, COVID-19 and other conditions; the regulatory approval and commercialization of fostamatinib in the U.S. and in ...

[PART I FINANCIAL INFORMATION](index=3&type=section&id=PART%20I%20FINANCIAL%20INFORMATION) [Financial Statements](index=3&type=section&id=Item%201.%20Financial%20Statements) Rigel Pharmaceuticals reported increased Q1 2023 revenues and a narrowed net loss, with total assets of $123.6 million and a stockholders' deficit Condensed Statements of Operations (Unaudited) | | Three Months Ended March 31, | | :--- | :--- | :--- | | | **2023** | **2022** | | **Total revenues** | $26,070 | $16,735 | | Total costs and expenses | $38,795 | $42,996 | | **Loss from operations** | $(12,725) | $(26,261) | | **Net loss** | $(13,536) | $(27,445) | | **Net loss per share, basic and diluted** | $(0.08) | $(0.16) | Condensed Balance Sheet Highlights (Unaudited) | | March 31, 2023 | December 31, 2022 | | :--- | :--- | :--- | | Cash and cash equivalents | $40,285 | $24,459 | | Short-term investments | $18,377 | $33,747 | | **Total current assets** | $106,972 | $115,903 | | **Total assets** | $123,612 | $134,279 | | **Total current liabilities** | $45,518 | $65,211 | | Loans payable, net of discount | $59,484 | $39,448 | | **Total liabilities** | $147,869 | $147,895 | | **Total stockholders' deficit** | $(24,257) | $(13,616) | Condensed Statements of Cash Flows (Unaudited) | | Three Months Ended March 31, | | :--- | :--- | :--- | | | **2023** | **2022** | | **Net cash used in operating activities** | $(4,074) | $(25,637) | | Net cash provided by investing activities | $777 | $22,629 | | Net cash provided by financing activities | $19,123 | $8,797 | | **Net increase in cash and cash equivalents** | $15,826 | $5,789 | [Notes to Condensed Financial Statements](index=8&type=page&id=Notes%20to%20Condensed%20Financial%20Statements) The notes detail accounting policies, revenue recognition, and collaboration agreements, confirming **$58.7 million** in cash and investments sufficient for 12 months of operations - The company's two approved products are TAVALISSE® (fostamatinib) for chronic immune thrombocytopenia (ITP) and REZLIDHIA® (olutasidenib) for relapsed or refractory acute myeloid leukemia (AML), which was commercialized in December 2022[19](index=19&type=chunk)[20](index=20&type=chunk) Disaggregated Revenues (in thousands) | | Three Months Ended March 31, | | :--- | :--- | :--- | | | **2023** | **2022** | | **Product sales, net** | **$23,745** | **$16,197** | | Gross product sales | $33,198 | $22,618 | | Discounts and allowances | $(9,453) | $(6,421) | | **Revenues from collaborations** | **$2,325** | **$538** | | **Total revenues** | **$26,070** | **$16,735** | - The company has a credit agreement with MidCap for a **$60.0 million** term loan facility, fully funded as of March 31, 2023, with an outstanding principal balance of **$60.0 million**[84](index=84&type=chunk) - Based on its current operating plan, management believes existing cash, cash equivalents, and short-term investments of **$58.7 million** are sufficient to fund operations for at least the next 12 months[26](index=26&type=chunk)[27](index=27&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=23&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses a **47%** increase in Q1 2023 net product sales, reduced R&D expenses, and an improved liquidity position expected to fund operations for at least 12 months [Business Updates](index=24&type=page&id=Business%20Updates) Q1 2023 saw TAVALISSE sales grow **38%**, REZLIDHIA launch, and pipeline advancement with R289 entering Phase 1b and R552 progressing to Phase 2a - Net product sales of TAVALISSE were **$22.3 million** for Q1 2023, a **38%** increase compared to the same period in 2022, driven by increased sales volume and price[102](index=102&type=chunk) - REZLIDHIA, launched in December 2022 for R/R AML, generated **$1.5 million** in net product sales in Q1 2023[103](index=103&type=chunk) - The company dosed the first patient in its Phase 1b trial of R289, an oral IRAK1/4 inhibitor, for low-risk myelodysplastic syndrome (MDS)[105](index=105&type=chunk) - Partner Eli Lilly is advancing R552 (RIPK1 inhibitor) into a Phase 2a trial for rheumatoid arthritis, expected to begin in Q2 2023[106](index=106&type=chunk) [Results of Operations](index=36&type=page&id=Results%20of%20Operations) Q1 2023 total revenues increased by **$9.3 million** to **$26.1 million**, driven by product sales, while R&D expenses decreased due to trial winding down Comparison of Results of Operations (in thousands) | | Three Months Ended March 31, | Change | | :--- | :--- | :--- | :--- | | | **2023** | **2022** | **($)** | | **Total revenues** | $26,070 | $16,735 | $9,335 | | Cost of product sales | $977 | $121 | $856 | | Research and development expense | $10,089 | $15,474 | $(5,385) | | Selling, general and administrative expense | $27,729 | $27,401 | $328 | - The decrease in R&D expense was mainly due to winding down activities for the Phase 3 COVID-19 trial (**$2.5 million**) and the Phase 3 wAIHA trial (**$1.3 million**)[193](index=193&type=chunk) - The increase in cost of product sales was partly due to **$0.3 million** in amortization of intangible assets and **$0.2 million** in royalty expenses related to REZLIDHIA, which were not incurred in the prior year[191](index=191&type=chunk) [Liquidity and Capital Resources](index=40&type=page&id=Liquidity%20and%20Capital%20Resources) Rigel had **$58.7 million** in cash and investments as of March 31, 2023, sufficient for 12 months, with reduced operating cash burn and a fully drawn term loan - The company had approximately **$58.7 million** in cash, cash equivalents, and short-term investments as of March 31, 2023[214](index=214&type=chunk) - Net cash used in operating activities was **$4.1 million** for Q1 2023, a significant improvement from **$25.6 million** used in Q1 2022[217](index=217&type=chunk) - In March 2023, the company drew down the final **$20.0 million** (Tranche 5) from its term loan financing with MidCap[219](index=219&type=chunk) - The company has a commitment to co-fund development costs with Lilly for R552 up to a maximum of **$65.0 million** through April 1, 2024, with **$15.9 million** billed and paid as of March 31, 2023[235](index=235&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=44&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risks are interest rate sensitivities on investments and loans, with no material changes reported for the quarter - The company's primary market risks are interest rate sensitivities related to its short-term investments and outstanding loans, with no material changes reported for the quarter[240](index=240&type=chunk) [Controls and Procedures](index=44&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of March 31, 2023, with no material changes in internal control over financial reporting - Management concluded that as of March 31, 2023, the company's disclosure controls and procedures were effective[241](index=241&type=chunk) - No changes in internal control over financial reporting occurred during the quarter that have materially affected, or are reasonably likely to materially affect, internal controls[242](index=242&type=chunk) [PART II OTHER INFORMATION](index=45&type=section&id=PART%20II%20OTHER%20INFORMATION) [Legal Proceedings](index=45&type=section&id=Item%201.%20Legal%20Proceedings) The company is involved in an ongoing patent infringement lawsuit against Annora Pharma regarding a generic version of TAVALISSE - In July 2022, Rigel filed a lawsuit against Annora Pharma for patent infringement following Annora's submission of an ANDA for a generic version of TAVALISSE[247](index=247&type=chunk) - Annora has counterclaimed for a declaratory judgment of non-infringement and invalidity of certain patents, and Rigel intends to vigorously enforce and defend its intellectual property[247](index=247&type=chunk) [Risk Factors](index=46&type=section&id=Item%201A.%20Risk%20Factors) The company's prospects depend on product commercial success, facing risks from generic competition, clinical development challenges, and a history of operating losses - The company's prospects are highly dependent on the commercial success of TAVALISSE® and the recently launched REZLIDHIA™[250](index=250&type=chunk) - A significant risk is potential competition from generic versions of its products, highlighted by the ANDA submitted by Annora for a generic version of TAVALISSE[298](index=298&type=chunk)[300](index=300&type=chunk) - The company faces high risk in drug development, as demonstrated by the Phase 3 trial of fostamatinib in wAIHA, which did not meet its primary efficacy endpoint, leading to the decision not to file an sNDA for this indication[262](index=262&type=chunk) - The company has a history of operating losses, with an accumulated deficit of approximately **$1.4 billion** as of March 31, 2023, and expects to continue incurring losses for at least the next 12 months[326](index=326&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=92&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) None [Defaults Upon Senior Securities](index=92&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) None [Mine Safety Disclosures](index=93&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) Not applicable [Other Information](index=93&type=section&id=Item%205.%20Other%20Information) None [Exhibits](index=94&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed as part of the Quarterly Report on Form 10-Q, including certifications and Inline XBRL data files

Rigel Pharmaceuticals, Inc. (NASDAQ:RIGL) Q4 2022 Earnings Conference Call March 7, 2023 4:30 PM ET Company Participants Ray Furey - EVP, General Counsel and Corporate Secretary. Raul Rodriguez - President and CEO Dave Santos - EVP and Chief Commercial Officer Wolfgang Dummer - EVP and Chief Medical Officer Dean Schorno - EVP and Chief Financial Officer Conference Call Participants Carly Kenselaar - Citi Eun Yang - Jefferies Do Kim - Piper Sandler Kristen Kluska - Cantor Fitzgerald Gary Nachman - BMO Capita ...

| --- | --- | --- | |--------------------------------|-------|-------| | | | | | | | | | Q4 & YE 2022 Financial Results | | | | Presentation March 7, 2023 | | | | | | | Forward-Looking Statements Any statements contained in this presentation that are not statements of historical fact may be deemed to be forward-looking statements and as such are intended to be covered by the safe harbor for "forward-looking statements" provided by the PSLRA. Forward-looking statements can be identified by words such as "pla ...