– Results from pivotal Phase 2b ReNeu trial of mirdametinib in patients with NF1-PN to be presented in an oral presentation – – Additional data and analyses from Phase 3 DeFi trial of OGSIVEO® (nirogacestat) highlighting consistent safety and efficacy across subgroups of adults with desmoid tumors also being presented in oral and poster sessions – STAMFORD, Conn., May 23, 2024 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe ra ...

Start Time: 08:30 January 1, 0000 9:06 AM ET SpringWorks Therapeutics, Inc. (NASDAQ:SWTX) Q1 2024 Earnings Conference Call May 02, 2024, 08:30 AM ET Company Participants Saqib Islam - CEO Francis Perier - CFO Bhavesh Ashar - Chief Commercial Officer Jim Cassidy - CMO Samantha Sandler - Senior Director of IR Conference Call Participants Anupam Rama - JPMorgan Yaron Werber - TD Cowen Corinne Johnson - Goldman Sachs Peter Lawson - Barclays Alec Stranahan - Bank of America Michael Schmidt - Guggenheim Operator ...

Financial Performance - SpringWorks reported $21.0 million in net product revenue for OGSIVEO in the first full quarter on the market[22, 55] - The company's cash, cash equivalents, and marketable securities totaled $573.0 million as of March 31, 2024[4, 33] - Total operating expenses were $114.9 million for the quarter ended March 31, 2024[55] - Net loss for the quarter was $87.4 million[55] OGSIVEO Commercialization - There are approximately 5,500 to 7,000 U.S desmoid tumor patients actively managed annually[9] - 76% of surveyed oncologists are aware of OGSIVEO and have already used or plan to prescribe it[48] - 82% of oncologists who have used OGSIVEO prefer it to other systemic treatments[48] Mirdametinib Development - Mirdametinib rolling NDA submission for NF1-PN is underway, representing opportunity for a potential second approval by 2025[41] - The company completed a pre-NDA meeting and initiated the rolling NDA submission for mirdametinib[51] Pipeline Expansion - A Phase 1 trial of SW-682 in Hippo mutant solid tumors is planned for initiation in Q2 2024[2, 14] - Initial data from the Phase 2 OvGCT study of nirogacestat is expected in 2H 2024[54]

[PART I – FINANCIAL INFORMATION](index=6&type=section&id=PART%20I%20%E2%80%93%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements (Unaudited)](index=6&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Unaudited Q1 2024 financial statements detail $656.8 million in assets, an $87.4 million net loss, and $21.0 million in product revenue [Condensed Consolidated Balance Sheets](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of March 31, 2024, total assets were $656.8 million, decreasing from $725.8 million, with liabilities at $89.4 million and stockholders' equity at $567.4 million Condensed Consolidated Balance Sheet Highlights (in thousands) | | March 31, 2024 | December 31, 2023 | | :--- | :--- | :--- | | **Total current assets** | $471,798 | $500,912 | | **Total assets** | $656,832 | $725,788 | | **Total current liabilities** | $68,917 | $78,170 | | **Total liabilities** | $89,392 | $99,569 | | **Total stockholders' equity** | $567,440 | $626,219 | [Condensed Consolidated Statements of Operations](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Q1 2024 saw first product revenue of $21.0 million, increased operating expenses to $114.9 million, and a net loss of $87.4 million Condensed Consolidated Statements of Operations (in thousands, except per-share data) | | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :--- | :--- | :--- | | **Product revenue, net** | $21,006 | $— | | **Total operating costs and expenses** | $114,937 | $77,699 | | **Loss from operations** | $(93,931) | $(77,699) | | **Net loss** | $(87,385) | $(73,420) | | **Net loss per share, basic and diluted** | $(1.18) | $(1.18) | [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Q1 2024 net cash used in operating activities increased to $78.9 million, while investing activities provided $21.5 million, resulting in a $58.0 million net decrease in cash Condensed Consolidated Statements of Cash Flows Highlights (in thousands) | | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :--- | :--- | :--- | | **Net cash used in operating activities** | $(78,930) | $(61,818) | | **Net cash provided by investing activities** | $21,463 | $98,604 | | **Net cash used in financing activities** | $(552) | $(1,848) | | **Net (decrease) increase in cash and cash equivalents** | $(58,019) | $34,938 | [Notes to Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) Notes detail the company's transition to commercial stage with OGSIVEO, $21.0 million Q1 revenue, and $573.0 million liquidity sufficient for twelve months - The company is a commercial-stage biopharmaceutical firm with its first product, **OGSIVEO**, approved in November 2023 for desmoid tumors. It generated **$21.0 million** in net revenue in Q1 2024[26](index=26&type=chunk) - As of March 31, 2024, the company had **$573.0 million** in cash, cash equivalents, and marketable securities, which management estimates will fund operations for at least the next **twelve months**[27](index=27&type=chunk) - In January 2024, the company made an additional investment of **$8.2 million** in MapKure for Series C preferred units, increasing its ownership interest to **39.7%**[44](index=44&type=chunk) Equity-Based Compensation Expense (in thousands) | | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :--- | :--- | :--- | | Research and development | $12,423 | $8,918 | | Selling, general and administrative | $18,039 | $14,477 | | **Total** | **$30,462** | **$23,395** | [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations (MD&A)](index=17&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) MD&A discusses Q1 2024 financial performance, including $21.0 million OGSIVEO revenue, increased operating expenses, and sufficient liquidity for the next year [Overview](index=17&type=section&id=Overview) SpringWorks is a commercial-stage biopharma company with FDA-approved OGSIVEO, advancing mirdametinib, and developing other pipeline assets - **OGSIVEO (nirogacestat)** was approved by the FDA on November 27, 2023, for adult patients with progressing desmoid tumors and is the **first and only FDA-approved therapy** for this indication[65](index=65&type=chunk) - The company initiated a **rolling New Drug Application (NDA) submission** for mirdametinib for NF1-PN in March 2024, with completion expected in Q2 2024[70](index=70&type=chunk) - The company plans to initiate a Phase 1a trial of SW-682, a TEAD inhibitor development candidate, in Hippo mutant solid tumors in the second quarter of 2024[73](index=73&type=chunk) - In March 2024, the company **terminated its license agreement** with Dana-Farber for a portfolio of EGFR inhibitors, ceasing further investment in the program[74](index=74&type=chunk) [Results of Operations](index=21&type=section&id=Results%20of%20Operations) Q1 2024 saw $21.0 million product revenue, a 48% increase in total operating expenses to $114.9 million, and an $87.4 million net loss Comparison of Results of Operations (in thousands) | | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | $ Change | % Change | | :--- | :--- | :--- | :--- | :--- | | **Product revenue, net** | $21,006 | $— | $21,006 | —% | | **Selling, general and administrative** | $60,113 | $44,175 | $15,938 | 36% | | **Research and development** | $53,622 | $33,524 | $20,098 | 60% | | **Total operating costs and expenses** | $114,937 | $77,699 | $37,238 | 48% | | **Net loss** | $(87,385) | $(73,420) | $(13,965) | 19% | - SG&A expenses increased by **$15.9 million (36%)** in Q1 2024, primarily due to commercial activities supporting the U.S. launch of OGSIVEO, including growth of the commercial organization and sales force[87](index=87&type=chunk)[88](index=88&type=chunk) Research and Development Expenses by Program (in thousands) | Program | Q1 2024 | Q1 2023 | $ Change | | :--- | :--- | :--- | :--- | | Nirogacestat | $13,700 | $7,256 | $6,444 | | Mirdametinib | $16,155 | $6,499 | $9,656 | | Other | $9,583 | $1,987 | $7,596 | | **Total program specific costs** | **$39,438** | **$15,742** | **$23,696** | [Liquidity and Capital Resources](index=22&type=section&id=Liquidity%20and%20Capital%20Resources) The company had an accumulated deficit of $982.4 million, with $573.0 million in liquidity, estimated to fund operations for at least the next twelve months - The company had an accumulated deficit of **$982.4 million** as of March 31, 2024, and management estimates its current liquidity will fund operations for at least **twelve months** after the filing date of the report[94](index=94&type=chunk) Summary of Cash Flows (in thousands) | | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :--- | :--- | :--- | | Net cash used in operating activities | $(78,930) | $(61,818) | | Net cash provided by investing activities | $21,463 | $98,604 | | Net cash used in financing activities | $(552) | $(1,848) | [Item 3. Quantitative and Qualitative Disclosure About Market Risk](index=25&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosure%20About%20Market%20Risk) No material changes to the company's market risks from those described in its 2023 Form 10-K - There were **no material changes** to the company's market risks from those disclosed in the Annual Report on Form 10-K for the year ended December 31, 2023[108](index=108&type=chunk) [Item 4. Controls and Procedures](index=25&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls and procedures were effective, with no material changes in internal control over financial reporting - Management concluded that the company's disclosure controls and procedures were **effective as of the end of the period** covered by the report[109](index=109&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, the company's internal control[110](index=110&type=chunk) [PART II. OTHER INFORMATION](index=26&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=26&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any material legal proceedings - The company is **not currently a party to any material legal proceedings**[111](index=111&type=chunk) [Item 1A. Risk Factors](index=26&type=section&id=Item%201A.%20Risk%20Factors) No material changes in the company's risk factors from those disclosed in the 2023 Form 10-K - There have been **no material changes** in the company's risk factors from those disclosed in the 2023 Form 10-K[112](index=112&type=chunk) [Item 5. Other Information](index=26&type=section&id=Item%205.%20Other%20Information) Several directors and executive officers adopted Rule 10b5-1 trading plans during Q1 2024 - Several directors and executive officers, including the **CEO (Saqib Islam)**, COO (Badreddin Edris), and CCO (Bhavesh Ashar), **adopted Rule 10b5-1 trading plans** during the quarter ended March 31, 2024[117](index=117&type=chunk) [Item 6. Exhibits](index=27&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including required CEO and CFO certifications - The report includes an index of exhibits filed, such as **CEO and CFO certifications** pursuant to Section 302 and Section 906 of the Sarbanes-Oxley Act[119](index=119&type=chunk)

– Achieved $21.0 million in OGSIVEO (nirogacestat) net product revenue in the first quarter – ® – Received validation from the EMA for MAA of nirogacestat for the treatment of adults with desmoid tumors – Exhibit 99.1 SpringWorks Therapeutics Reports First Quarter 2024 Financial Results and Highlights Recent Business Updates – Initiated rolling submission of NDA to the FDA for mirdametinib for the treatment of children and adults with NF1-PN – – Phase 2b ReNeu trial results accepted for oral presentation at ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-K ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission file number: 001-39044 SPRINGWORKS THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 83-4066827 (State of Other Jurisdiction of incorporation o ...

Report Overview and Business Highlights [Executive Summary](index=1&type=section&id=Executive%20Summary) In 2023, SpringWorks Therapeutics achieved its first FDA approval for OGSIVEO™ for desmoid tumors, generating $5.4 million in its initial partial quarter, and reported positive Phase 2b data for mirdametinib in NF1-PN, planning an NDA submission in H1 2024 - OGSIVEO™ (nirogacestat) generated net product revenue of **$5.4 million** in its first partial quarter after FDA approval on November 27, 2023[1](index=1&type=chunk) - Submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for nirogacestat for the treatment of desmoid tumors[1](index=1&type=chunk) - Positive topline data from the Phase 2b ReNeu trial of mirdametinib in patients with NF1-PN was presented, with a New Drug Application (NDA) submission planned for the first half of 2024[1](index=1&type=chunk) - The company ended 2023 with **$662.6 million** in cash, cash equivalents, and marketable securities[2](index=2&type=chunk) - 2024 focus includes the U.S. launch of OGSIVEO, filing the NDA for mirdametinib, expanding OGSIVEO's geographic reach, and advancing the emerging portfolio[3](index=3&type=chunk) [Pipeline and Corporate Updates](index=1&type=section&id=Recent%20Business%20Highlights%20and%20Upcoming%20Milestones) The company achieved FDA approval for OGSIVEO™, submitted it for EMA approval, and reported positive Phase 2b results for mirdametinib, planning an NDA submission, while advancing its emerging pipeline and strengthening its balance sheet [OGSIVEO™ (Nirogacestat)](index=1&type=section&id=OGSIVEO%E2%84%A2%20(Nirogacestat)) OGSIVEO™ received FDA approval on November 27, 2023, for adult patients with progressing desmoid tumors, generating $5.4 million in its first partial quarter, with an MAA submitted to the EMA in February 2024 - Received U.S. FDA approval on November 27, 2023, for adult patients with progressing desmoid tumors[4](index=4&type=chunk) - Achieved **$5.4 million** in net product revenue in the first partial quarter of its U.S. launch, with payers representing over **98%** of commercial lives providing reimbursement[4](index=4&type=chunk) - Submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in February 2024[4](index=4&type=chunk) - Initial data from a Phase 2 trial in patients with recurrent ovarian granulosa cell tumors is expected in the second half of 2024[9](index=9&type=chunk) [Mirdametinib](index=2&type=section&id=Mirdametinib) The Phase 2b ReNeu trial of mirdametinib in patients with NF1-PN demonstrated positive results, with confirmed objective response rates of 52% in pediatric patients and 41% in adult patients, leading to an NDA submission planned for H1 2024 Phase 2b ReNeu Trial Topline Data | Patient Cohort | Confirmed Objective Response Rate (ORR) | | :--- | :--- | | Pediatric | 52% | | Adult | 41% | - Mirdametinib was generally well tolerated, with most adverse events being Grade 1 or 2, primarily rash, diarrhea, and nausea/vomiting[9](index=9&type=chunk) - An NDA submission to the FDA for the treatment of children and adults with NF1-PN is on track for the first half of 2024[9](index=9&type=chunk) [Emerging Pipeline & Corporate Updates](index=2&type=section&id=Emerging%20Pipeline) The company is advancing its emerging pipeline, with plans to present additional data for brimarafenib in H2 2024 and initiate a Phase 1a trial for SW-682 in H1 2024, supported by a public offering and new patent issuances - The FDA cleared the IND application for SW-682, a TEA Domain inhibitor, with a Phase 1a trial in Hippo mutant solid tumors planned for H1 2024[9](index=9&type=chunk) - Strengthened the balance sheet with a public offering in December 2023, raising gross proceeds of approximately **$316.2 million**[9](index=9&type=chunk) - New patents were issued for OGSIVEO and mirdametinib, extending intellectual property protection for both products into **2043**[9](index=9&type=chunk) Financial Performance [Fourth Quarter and Full Year 2023 Financial Results Summary](index=2&type=section&id=Financial%20Results%20Summary) For Q4 2023, SpringWorks reported its first product revenue of $5.4 million from OGSIVEO, with increased operating expenses leading to a net loss of $94.3 million, and a full-year net loss of $325.1 million, while maintaining a strong cash position of $662.6 million Q4 & Full Year 2023 Financial Highlights (in millions) | Metric | Q4 2023 | Q4 2022 | Full Year 2023 | Full Year 2022 | | :--- | :--- | :--- | :--- | :--- | | Net Product Revenues | $5.4M | $0 | $5.4M | $0 | | R&D Expenses | $43.7M | $37.9M | $150.5M | $146.1M | | SG&A Expenses | $59.8M | $40.5M | $197.6M | $134.6M | | Net Loss | $94.3M | $74.2M | $325.1M | $277.4M | | Net Loss Per Share | $1.44 | $1.19 | $5.15 | $5.21 | - The increase in SG&A expenses was largely attributable to commercial readiness activities to support the U.S. launch of OGSIVEO[14](index=14&type=chunk) - Cash, cash equivalents and marketable securities were **$662.6 million** as of December 31, 2023[14](index=14&type=chunk) [Condensed Consolidated Financial Statements](index=5&type=section&id=Condensed%20Consolidated%20Financial%20Statements) This section presents the unaudited condensed consolidated statements of operations for 2021-2023 and selected balance sheet data for 2022-2023, detailing revenue, operating expenses, net loss, and key financial positions Condensed Consolidated Statements of Operations (in thousands) | | Year Ended December 31, | | | | :--- | :--- | :--- | :--- | | | **2023** | **2022** | **2021** | | **Product revenue, net** | **$5,447** | **$—** | **$—** | | Total operating expenses | $348,460 | $280,674 | $173,468 | | Loss from operations | ($343,013) | ($280,674) | ($173,468) | | **Net loss** | **($325,104)** | **($277,417)** | **($173,910)** | | Net loss per share | ($5.15) | ($5.21) | ($3.59) | Selected Balance Sheet Data (in thousands) | | As of December 31, | | | :--- | :--- | :--- | | | **2023** | **2022** | | Cash, cash equivalents and marketable securities | $662,588 | $597,006 | | Total assets | $725,788 | $630,242 | | Total liabilities | $99,569 | $72,050 | | **Total stockholders' equity** | **$626,219** | **$558,192** | Company Information and Disclosures [About SpringWorks Therapeutics](index=3&type=section&id=About%20SpringWorks%20Therapeutics) SpringWorks Therapeutics is a commercial-stage biopharmaceutical company focused on precision medicine for severe rare diseases and cancer, with OGSIVEO™ as its first FDA-approved therapy - SpringWorks is a commercial-stage biopharmaceutical company focused on precision medicine for severe rare diseases and cancer[10](index=10&type=chunk) - The company's first FDA-approved therapy is OGSIVEO™ (nirogacestat), for adult patients with progressing desmoid tumors[10](index=10&type=chunk) [Forward-Looking Statements](index=3&type=section&id=Forward-Looking%20Statements) This press release contains forward-looking statements regarding the company's business, development, and commercialization plans, which are subject to risks and uncertainties, advising readers to consult SEC filings for detailed risk discussions - The press release includes forward-looking statements concerning business plans, clinical results, regulatory submissions (e.g., mirdametinib NDA), and commercialization efforts for OGSIVEO[13](index=13&type=chunk)[15](index=15&type=chunk) - Actual results may differ materially from expectations due to various risks and uncertainties[15](index=15&type=chunk) - For a detailed discussion of risks, investors should review the "Risk Factors" section in the company's Annual Report on Form 10-K for the year ended December 31, 2023[17](index=17&type=chunk)

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 ____________________________________________________ FORM 10-Q ____________________________________________________ x QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED SEPTEMBER 30, 2023 OR ¨ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM _ TO _ COMMISSION FILE NUMBER 001-39044 _ ...

PART I – FINANCIAL INFORMATION This section provides the unaudited condensed consolidated financial statements and related management discussion for the period ended June 30, 2023 [Item 1. Financial Statements](index=6&type=section&id=Item%201.%20Financial%20Statements) This section presents the unaudited condensed consolidated financial statements for the period ended June 30, 2023, highlighting a decrease in total assets to **$517.3 million** and a net loss of **$151.3 million** [Condensed Consolidated Balance Sheets](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) This table presents the company's financial position, including assets, liabilities, and equity, as of June 30, 2023, and December 31, 2022 Condensed Consolidated Balance Sheet Highlights (in thousands) | Metric | June 30, 2023 | December 31, 2022 | | :--- | :--- | :--- | | Cash and cash equivalents | $115,001 | $67,490 | | Marketable securities | $361,706 | $524,722 | | **Total Assets** | **$517,329** | **$630,242** | | **Total Liabilities** | **$65,888** | **$72,050** | | **Total Stockholders' Equity** | **$451,441** | **$558,192** | [Condensed Consolidated Statements of Operations](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) This table summarizes the company's revenues, expenses, and net loss for the three and six months ended June 30, 2023 and 2022 Statement of Operations Summary (in thousands, except per-share data) | Metric | Three Months Ended June 30, 2023 | Three Months Ended June 30, 2022 | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | Research and development | $35,858 | $38,024 | $69,382 | $72,127 | | General and administrative | $46,994 | $30,987 | $91,169 | $58,353 | | **Total operating expenses** | **$82,852** | **$69,011** | **$160,551** | **$130,480** | | **Net loss** | **$(77,925)** | **$(69,050)** | **$(151,345)** | **$(130,851)** | | Net loss per share | $(1.25) | $(1.41) | $(2.43) | $(2.67) | [Condensed Consolidated Statements of Cash Flows](index=11&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) This table details the cash inflows and outflows from operating, investing, and financing activities for the six months ended June 30, 2023 and 2022 Cash Flow Summary for Six Months Ended June 30 (in thousands) | Cash Flow Activity | 2023 | 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | $(109,629) | $(87,759) | | Net cash provided by investing activities | $159,360 | $53,181 | | Net cash (used in) provided by financing activities | $(2,190) | $173 | | **Net increase (decrease) in cash** | **$47,541** | **$(34,405)** | [Notes to Condensed Consolidated Financial Statements](index=12&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) This section provides additional details and context for the financial statements, including business overview, significant accounting policies, and financial position - The company is a clinical-stage biopharmaceutical firm with two late-stage product candidates: nirogacestat and mirdametinib. An NDA for nirogacestat for desmoid tumors was submitted and accepted for **priority review** by the FDA[30](index=30&type=chunk) - The PDUFA target action date for the nirogacestat NDA was **extended by three months** to **November 27, 2023**, to allow the FDA more time to review additional data analyses[30](index=30&type=chunk) - The company has a history of losses, with an accumulated deficit of **$721.3 million** as of June 30, 2023. However, management believes its cash, cash equivalents, and marketable securities of **$476.7 million** are **sufficient to fund operations for at least the next twelve months**[31](index=31&type=chunk)[32](index=32&type=chunk) - Stock-based compensation expense for the six months ended June 30, 2023, was **$46.4 million**, an **increase** from **$35.5 million** in the same period of 2022[70](index=70&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=19&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's business overview, financial results, and liquidity, focusing on clinical progress, increased General & Administrative expenses, and sufficient capital for the next year [Overview](index=19&type=section&id=Overview) This section provides a strategic overview of the company's clinical development programs and commercialization plans for its lead product candidates - The company's most advanced product candidate, nirogacestat, is under **priority review** by the FDA for the treatment of desmoid tumors, with an updated PDUFA target action date of **November 27, 2023**[75](index=75&type=chunk) - The second lead product candidate, mirdametinib, is in a potentially registrational Phase 2b trial for NF1-PN. Topline data is expected in **Q4 2023**, with a planned NDA submission in the **first half of 2024** if data is positive[78](index=78&type=chunk) - The company is actively preparing for the **commercial launch** of nirogacestat in the U.S., if approved, and plans to file for marketing authorization in the European Union in **2024**[76](index=76&type=chunk) [Results of Operations](index=22&type=section&id=Results%20of%20Operations) This section analyzes the company's operating expenses, highlighting changes in research and development and general and administrative costs Comparison of Operating Expenses (in thousands) | Expense Category | Three Months Ended June 30, 2023 | Three Months Ended June 30, 2022 | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | Research and development | $35,858 | $38,024 | $69,382 | $72,127 | | General and administrative | $46,994 | $30,987 | $91,169 | $58,353 | - General and administrative expenses **increased by 52%** for Q2 2023 and **56%** for H1 2023 compared to the same periods in 2022. This **increase** was primarily driven by commercial readiness activities for the potential U.S. launch of nirogacestat, including hiring for sales, marketing, and commercial functions[94](index=94&type=chunk)[95](index=95&type=chunk)[101](index=101&type=chunk) - Research and development expenses **decreased by 6%** for Q2 2023 and **4%** for H1 2023 compared to the prior year periods, mainly due to lower external costs for drug manufacturing and clinical trials[92](index=92&type=chunk)[93](index=93&type=chunk)[99](index=99&type=chunk) [Liquidity and Capital Resources](index=24&type=section&id=Liquidity%20and%20Capital%20Resources) This section discusses the company's financial position, cash flows, and ability to fund future operations - The company had an accumulated deficit of **$721.3 million** as of June 30, 2023. Management estimates that its current cash, cash equivalents, and marketable securities are **sufficient to fund operating expenses for at least the next twelve months**[104](index=104&type=chunk) - Net cash used in operating activities **increased to $109.6 million** for the first six months of 2023, compared to **$87.8 million** for the same period in 2022, driven by a higher net loss and changes in operating assets and liabilities[105](index=105&type=chunk)[106](index=106&type=chunk) - Net cash provided by investing activities was **$159.4 million** for the first six months of 2023, primarily from the net sale and maturity of marketable securities[107](index=107&type=chunk) [Item 3. Quantitative and Qualitative Disclosure About Market Risk](index=26&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosure%20About%20Market%20Risk) The company reports that there have been **no material changes** to its market risks from those disclosed in its Annual Report on Form 10-K for the year ended December 31, 2022 - There were **no material changes** to the company's market risks from those described in the 2022 Form 10-K[122](index=122&type=chunk) [Item 4. Controls and Procedures](index=26&type=section&id=Item%204.%20Controls%20and%20Procedures) Management, including the CEO and CFO, concluded that the company's disclosure controls and procedures were **effective** as of June 30, 2023, with **no changes** to internal control over financial reporting during the quarter - Management evaluated disclosure controls and procedures and found them to be **effective** as of the end of the reporting period[123](index=123&type=chunk)[124](index=124&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, these controls[125](index=125&type=chunk) PART II – OTHER INFORMATION This section covers legal proceedings, updated risk factors, and other standard disclosures [Item 1. Legal Proceedings](index=28&type=section&id=Item%201.%20Legal%20Proceedings) As of the report date, the company is **not currently involved in any material legal proceedings** - The company is **not currently involved in any material legal proceedings**[127](index=127&type=chunk) [Item 1A. Risk Factors](index=28&type=section&id=Item%201A.%20Risk%20Factors) This section updates the company's risk factors, highlighting a **new risk factor** related to the financial services industry and its potential adverse impacts - A **new risk factor** has been added concerning adverse developments in the financial services industry, prompted by recent events like the failure of Silicon Valley Bank (SVB)[129](index=129&type=chunk) - Potential impacts from financial institution failures include **delayed or lost access** to deposits, **termination of cash management services**, and **impaired ability to secure financing**[131](index=131&type=chunk)[132](index=132&type=chunk) - The instability could also affect customers and suppliers, potentially leading to **payment defaults or disruptions** that would **materially harm** the company's business[133](index=133&type=chunk) [Other Items (Items 2-6)](index=29&type=section&id=Other%20Items%20(Items%202-6)) This section covers several standard disclosure items, reporting **no activity** for most, and listing the **index of exhibits** filed with the Form 10-Q - The company reported **no activity** under Item 2 (Unregistered Sales of Equity Securities), Item 3 (Defaults Upon Senior Securities), Item 4 (Mine Safety Disclosures), or Item 5 (Other Information)[134](index=134&type=chunk)[135](index=135&type=chunk)[136](index=136&type=chunk)[137](index=137&type=chunk) - Item 6 provides an **index of exhibits** filed with the report, including **required certifications** from the CEO and CFO[139](index=139&type=chunk)

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 ____________________________________________________ FORM 10-Q ____________________________________________________ x QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED MARCH 31, 2023 OR ¨ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM _ TO _ COMMISSION FILE NUMBER 001-39044 _____ ...