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 ...

Here's the structured report outline based on your provided content and requirements: PART I [Item 1. Business](index=7&type=section&id=Item%201.%20Business) Rigel Pharmaceuticals is a biotechnology company focused on developing novel therapies for hematologic disorders and cancer, with commercial products TAVALISSE and REZLIDHIA, and a pipeline of clinical programs - **Rigel Pharmaceuticals** is a biotechnology company focused on hematologic disorders and cancer, with a strategy to expand its commercial business in the US and globally through partnerships[16](index=16&type=chunk)[42](index=42&type=chunk) - The company's commercial products include **TAVALISSE (fostamatinib)** for chronic ITP and **REZLIDHIA (olutasidenib)** for relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible IDH1 mutation[17](index=17&type=chunk)[18](index=18&type=chunk)[43](index=43&type=chunk) - Key development programs include an **IRAK1/4 inhibitor (R289)** for low-risk MDS, fostamatinib in **COVID-19** (Phase 3 did not meet statistical significance), and partnered **RIPK1 inhibitors** with **Eli Lilly and Company**[19](index=19&type=chunk)[27](index=27&type=chunk)[30](index=30&type=chunk)[33](index=33&type=chunk) [Overview](index=7&type=section&id=Overview) Rigel Pharmaceuticals is a biotechnology company dedicated to discovering, developing, and providing novel therapies for hematologic disorders and cancer, focusing on critical signaling pathways - **Rigel Pharmaceuticals** is a biotechnology company dedicated to discovering, developing, and providing novel therapies for hematologic disorders and cancer, focusing on critical signaling pathways[16](index=16&type=chunk) - **TAVALISSE (fostamatinib)** is the only approved oral spleen tyrosine kinase (SYK) inhibitor for **chronic immune thrombocytopenia (ITP)** in adults, commercially available in the US, Europe, UK, Canada, and Israel[17](index=17&type=chunk) - **REZLIDHIA (olutasidenib)** was **FDA**-approved in **December 2022** for adult patients with **relapsed or refractory (R/R) acute myeloid leukemia (AML)** with a susceptible **IDH1 mutation**, with commercialization beginning in **December 2022**[18](index=18&type=chunk) - **fostamatinib's** Phase 3 trial for **warm autoimmune hemolytic anemia (wAIHA)** did not lead to an sNDA filing due to top-line data and **FDA** guidance, and its FOCUS Phase 3 trial for hospitalized **COVID-19** patients was completed but did not meet statistical significance in the primary endpoint[18](index=18&type=chunk) - Other clinical programs include an **IRAK inhibitor (R289)** and a **RIPK1 inhibitor** program partnered with **Eli Lilly and Company**, along with product candidates with **BerGenBio ASA** and **Daiichi Sankyo**[19](index=19&type=chunk) [Business Updates](index=7&type=section&id=Business%20Updates) Rigel's business updates include **TAVALISSE** sales growth, **REZLIDHIA** FDA approval and launch, **R289** Phase 1b initiation, and **fostamatinib** trial results for **COVID-19** and **wAIHA** TAVALISSE Net Product Sales (2021-2022) | Year | Net Product Sales (Millions USD) | Change YoY | Primary Drivers | | :--- | :------------------------------- | :--------- | :-------------- | | **2022** | **$75.8 million** | +20% | Increased quantities sold, price per bottle. Offset by higher revenue reserves (rebates). | | **2021** | **$63.0 million** | - | - | - **REZLIDHIA (olutasidenib)** received **FDA** approval on **December 1, 2022**, for **R/R AML** with a susceptible **IDH1 mutation**, with commercialization starting on **December 22, 2022**, generating **$0.9 million** in net product sales in **2022**[22](index=22&type=chunk)[24](index=24&type=chunk) - **Rigel** in-licensed **olutasidenib** from **Forma Therapeutics, Inc.** for an upfront fee of **$2.0 million**, with potential milestone payments up to **$67.5 million** (development/regulatory) and **$165.5 million** (commercial), plus tiered royalties[25](index=25&type=chunk)[26](index=26&type=chunk) - **Rigel** dosed the first patient in a Phase 1b trial of **R289**, an **IRAK1/4 inhibitor**, for low-risk **Myelodysplastic Syndromes (MDS)** in **December 2022**, aiming to assess tolerability and preliminary efficacy[27](index=27&type=chunk) - The FOCUS Phase 3 clinical trial of **fostamatinib** for hospitalized **COVID-19** patients completed enrollment in **July 2022**, but top-line results in **November 2022** did not meet statistical significance (**p=0.0603**) for the primary efficacy endpoint[30](index=30&type=chunk) - The **FORWARD study**, a Phase 3 trial of **fostamatinib** in **warm autoimmune hemolytic anemia (wAIHA)**, did not demonstrate statistical significance in its primary efficacy endpoint, leading **Rigel** not to file a supplemental New Drug Application (sNDA) for this indication[32](index=32&type=chunk) - **Rigel** has a global exclusive license and collaboration agreement with **Eli Lilly and Company** for **RIPK1 inhibitors** (**R552** for non-CNS diseases and other **RIPK1 inhibitors** for CNS diseases), with **Lilly** advancing **R552** into a Phase 2a trial for rheumatoid arthritis in **H1 2023**[33](index=33&type=chunk)[35](index=35&type=chunk)[37](index=37&type=chunk) - **Rigel** filed a patent infringement lawsuit against **Annora Pharma Private Limited** in **July 2022** regarding a generic version of **TAVALISSE**, following **Annora's Abbreviated New Drug Application (ANDA)** submission[38](index=38&type=chunk) - The **COVID-19** pandemic has adversely impacted **Rigel's** business, including reduced patient-doctor interactions and sales force visits, but the company did not experience significant supply chain disruptions[39](index=39&type=chunk)[40](index=40&type=chunk) [Strategy](index=14&type=section&id=Strategy) Rigel's strategy focuses on expanding its commercial presence in hematology-oncology and growing its development pipeline through internal efforts and partnerships - **Rigel's** goal is to be a successful commercial-stage biopharmaceutical company by expanding its commercial business in the US and globally through partnerships, focusing on hematology-oncology[42](index=42&type=chunk) - Key value drivers include growing **TAVALISSE** sales in the global **ITP** market (estimated over **$2.0 billion**), expanding the hematology-oncology portfolio with **REZLIDHIA** for **R/R AML** (targeting ~**1,000 patients** in a US market of ~**20,000 cases**), and expanding the development pipeline[43](index=43&type=chunk) [Commercial Products](index=15&type=section&id=Commercial%20Products) Rigel's commercial products include **TAVALISSE (fostamatinib)** for **chronic ITP** and **REZLIDHIA (olutasidenib)** for **R/R AML** with **mIDH1** [TAVALISSE/Fostamatinib in ITP](index=15&type=section&id=TAVALISSE%2FFostamatinib%20in%20ITP) **TAVALISSE (fostamatinib)** is an oral **SYK inhibitor** approved for **chronic ITP**, demonstrating stable platelet responses and facing competition from existing and generic therapies - **Chronic ITP** affects an estimated **81,300 adult patients** in the US, characterized by immune system attack on blood platelets, leading to bruising, bleeding, and fatigue[44](index=44&type=chunk) - **fostamatinib**, an oral **SYK inhibitor**, blocks the activation of **SYK** inside immune cells, interrupting platelet destruction in **ITP**[45](index=45&type=chunk) - **TAVALISSE** was **FDA**-approved in **April 2018** for **chronic ITP**, and subsequently received marketing authorization in Europe (**January 2020**) and approval in Japan (**December 2022**)[48](index=48&type=chunk)[67](index=67&type=chunk) - A post-hoc analysis of Phase 3 data showed a **78% response rate** when **TAVALISSE** was used as a second-line therapy in **chronic ITP**[49](index=49&type=chunk)[50](index=50&type=chunk) - **Rigel's** commercial efforts for **TAVALISSE** in the US target hematologists and hematologist-oncologists, supported by a fully integrated commercial team and patient support programs like **Rigel OneCare (ROC)**[52](index=52&type=chunk) - **TAVALISSE** faces competition from existing **ITP** therapies like corticosteroids, **TPO-RAs (PROMACTA, Nplate, DOPTELET)**, and immunosuppressants, with potential future competition from generic versions, including an **ANDA** filed by **Annora Pharma**[54](index=54&type=chunk)[55](index=55&type=chunk)[56](index=56&type=chunk)[58](index=58&type=chunk)[59](index=59&type=chunk) [Fostamatinib in Global Markets](index=19&type=section&id=Fostamatinib%20in%20Global%20Markets) Rigel has established global partnerships for **fostamatinib** commercialization, involving upfront payments, milestones, and royalties, with key achievements in Japan and Israel - **Rigel** partnered with **Grifols** for exclusive commercialization rights of **fostamatinib** in Europe, UK, Turkey, Middle East, North Africa, and Russia, receiving a **$30.0 million** upfront payment and potential milestones up to **$297.5 million**, plus tiered royalties[62](index=62&type=chunk)[63](index=63

! The first patients have been dosed in an open-label, Phase 1b clinical trial of study in lowrisk MDS4. The primary endpoint for this trial is safety with key secondary endpoints including preliminary efficacy Dual Inhibition of IRAK1 and IRAK4 Provides Stronger Suppression of Inflammatory Cytokines Compared to IRAK4-selective Inhibitor2 Proof-of-Mechanism First-In-Human study enrolled 82 adults to characterize the safety, PK, PD of R835 IL6 25 1R835 is an investigational compound not approved by the FDA. ...