[Forward-Looking Statements](index=3&type=section&id=FORWARD-LOOKING%20STATEMENTS) This section outlines the report's forward-looking statements, which are subject to inherent risks and uncertainties, with no obligation for the company to update them except as required by law - The report contains forward-looking statements regarding the company's plans and expectations for its research and development programs, clinical trials (including for bitopertin, DISC-0974, and DISC-3405), potential for accelerated FDA approval, manufacturing capabilities, funding needs, and future financial performance[10](index=10&type=chunk) - These statements are based on current expectations and are not guarantees of future performance. The company cautions that actual results may differ materially due to various risks and uncertainties[11](index=11&type=chunk) - The company explicitly states it does not undertake any obligation to update forward-looking statements, except as required by law, and refers readers to the 'Risk Factors' section for a detailed discussion of these risks[11](index=11&type=chunk)[12](index=12&type=chunk) [Risk Factor Summary](index=5&type=section&id=RISK%20FACTOR%20SUMMARY) This section summarizes the principal risks, including limited operating history, significant losses, funding needs, clinical development uncertainties, third-party reliance, and internal control weaknesses - Financial Risks: The company has a limited operating history, has incurred significant net losses since inception, has no revenue from product sales, and may need to raise substantial additional funding[15](index=15&type=chunk) - Developmental Risks: No product candidates have progressed through a Phase 3 trial, the company's approach is novel and may not succeed, and early-stage clinical data may not predict later results. There is also a risk of significant adverse events in trials[15](index=15&type=chunk) - Operational and Competitive Risks: The company relies on third parties for clinical trials and manufacturing, faces substantial competition, and its intellectual property protection may not be sufficient[15](index=15&type=chunk)[17](index=17&type=chunk) - Governance and Market Risks: A material weakness in internal controls over financial reporting has been identified. The stock price is expected to be volatile, and the company faces increased costs associated with being a public company[17](index=17&type=chunk) [PART I. FINANCIAL INFORMATION](index=8&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Financial Statements (Unaudited)](index=8&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents the unaudited condensed consolidated financial statements for Q1 2025, reflecting increased assets and stockholders' equity from a stock offering, alongside a wider net loss due to higher operating expenses [Condensed Consolidated Balance Sheets](index=8&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets increased to **$709.3 million** by March 31, 2025, from **$496.8 million** at year-end 2024, driven by marketable securities and a **$216.8 million** rise in stockholders' equity from an equity offering Condensed Consolidated Balance Sheet Highlights (in thousands) | | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $122,675 | $192,434 | | Marketable securities | $571,987 | $297,447 | | Total current assets | $705,600 | $493,615 | | **Total assets** | **$709,266** | **$496,773** | | **Liabilities & Stockholders' Equity** | | | | Total current liabilities | $18,741 | $23,316 | | Total liabilities | $48,904 | $53,186 | | Total stockholders' equity | $660,362 | $443,587 | | **Total liabilities and stockholders' equity** | **$709,266** | **$496,773** | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Net loss for Q1 2025 widened to **$34.1 million** from **$26.9 million** in Q1 2024, primarily due to increased R&D and SG&A expenses, partially offset by higher interest income Statement of Operations Highlights (in thousands, except per share data) | | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | Research and development | $27,763 | $23,704 | | Selling, general and administrative | $12,183 | $7,758 | | **Loss from operations** | **($39,946)** | **($31,462)** | | Interest income | $6,880 | $4,519 | | **Net loss** | **($34,085)** | **($26,949)** | | **Net loss per share, basic and diluted** | **($1.02)** | **($1.09)** | [Condensed Consolidated Statements of Stockholders' Equity](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Stockholders%27%20Equity) Stockholders' equity increased to **$660.4 million** by March 31, 2025, primarily due to **$234.0 million** from common stock and **$9.4 million** from pre-funded warrants in a January 2025 offering, offset by the **$34.1 million** net loss - In Q1 2025, the company completed an underwritten offering, issuing 4,533,182 shares of common stock for net proceeds of **$234.0 million** and pre-funded warrants for net proceeds of **$9.4 million**[25](index=25&type=chunk) Changes in Stockholders' Equity (in thousands) | | Amount | | :--- | :--- | | **Balance at December 31, 2024** | **$443,587** | | Sale of common stock in underwritten offering, net | $234,003 | | Sale of pre-funded warrants in underwritten offering, net | $9,386 | | Stock-based compensation expense | $6,363 | | Net loss | ($34,085) | | **Balance at March 31, 2025** | **$660,362** | [Condensed Consolidated Statements of Cash Flows](index=11&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operations increased to **$41.4 million** in Q1 2025, while investing activities used **$272.6 million**, largely offset by **$244.3 million** from financing activities, resulting in a **$69.8 million** net decrease in cash Cash Flow Summary (in thousands) | | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | ($41,376) | ($33,599) | | Net cash used in investing activities | ($272,633) | ($67) | | Net cash provided by financing activities | $244,251 | $15,900 | | **Net decrease in cash, cash equivalents and restricted cash** | **($69,758)** | **($17,766)** | [Notes to Unaudited Condensed Consolidated Financial Statements](index=12&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) These notes detail the company's focus on hematologic diseases, its **$694.7 million** capital position sufficient for 12 months, key license and loan agreements, and the **$243.4 million** net proceeds from the January 2025 equity offering - The company is a clinical-stage biopharmaceutical firm focused on hematologic diseases, with key product candidates including bitopertin, DISC-0974, DISC-3405, and DISC-0998[30](index=30&type=chunk) - As of March 31, 2025, the company had **$694.7 million** in cash, cash equivalents, and marketable securities, which is expected to fund planned operations for at least the next twelve months[32](index=32&type=chunk) - The company has a loan agreement for up to **$200.0 million**, with an initial **$30.0 million** borrowed in November 2024. The loan matures in December 2029[61](index=61&type=chunk) - In January 2025, the company raised net proceeds of **$243.4 million** from an underwritten offering of common stock and pre-funded warrants[70](index=70&type=chunk)[71](index=71&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=25&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses Q1 2025 financial results, highlighting a **$34.1 million** net loss driven by increased R&D and SG&A, significant clinical pipeline progress, and a strong **$694.7 million** liquidity position expected to fund operations into 2028 [Overview](index=25&type=section&id=Overview) This overview highlights the company's clinical pipeline progress, including planned NDA submission for bitopertin and Phase 2 trials for DISC-0974 and DISC-3405, supported by **$694.7 million** in cash expected to fund operations into 2028 - The company plans to submit a New Drug Application (NDA) for accelerated approval of bitopertin for EPP and XLP in the second half of 2025[98](index=98&type=chunk) - Key data readouts are expected in the second half of 2025 for the Phase 2 trial of DISC-0974 in anemia of MF and the MAD portion of the Phase 1b trial in non-dialysis dependent CKD[99](index=99&type=chunk)[100](index=100&type=chunk) - A Phase 2 clinical trial of DISC-3405 for polycythemia vera (PV) is planned to be initiated in the first half of 2025[101](index=101&type=chunk) - As of March 31, 2025, the company had **$694.7 million** in cash, cash equivalents, and marketable securities, which it believes is sufficient to fund operations into 2028[102](index=102&type=chunk) [Results of Operations](index=30&type=section&id=Results%20of%20Operations) Net loss increased by **$7.1 million** to **$34.1 million** in Q1 2025, driven by a **$4.1 million** rise in R&D and a **$4.4 million** increase in SG&A expenses, partially offset by higher interest income Comparison of Results of Operations (in thousands) | | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | Change | | :--- | :--- | :--- | :--- | | Research and development | $27,763 | $23,704 | $4,059 | | Selling, general and administrative | $12,183 | $7,758 | $4,425 | | **Loss from operations** | **($39,946)** | **($31,462)** | **($8,484)** | | **Net loss** | **($34,085)** | **($26,949)** | **($7,136)** | - R&D expenses increased by **$4.1 million**, primarily due to a **$2.4 million** increase in DISC-0974 development and a **$2.1 million** increase for bitopertin, related to advancing clinical trials and manufacturing. This was partially offset by a **$3.0 million** decrease in DISC-3405 expenses[114](index=114&type=chunk) - SG&A expenses increased by **$4.4 million**, mainly from a **$2.0 million** increase in personnel-related costs due to higher headcount and a **$2.1 million** increase in legal and professional fees for commercialization readiness[115](index=115&type=chunk)[116](index=116&type=chunk) [Liquidity and Capital Resources](index=31&type=section&id=Liquidity%20and%20Capital%20Resources) The company maintains a strong liquidity position with **$694.7 million** in cash and marketable securities as of March 31, 2025, expected to fund operations into 2028, primarily bolstered by **$244.3 million** from the January 2025 equity offering - The company has funded operations primarily through sales of preferred and common stock and debt borrowings, with **$694.7 million** in cash, cash equivalents, and marketable securities as of March 31, 2025[120](index=120&type=chunk) - Management believes current cash reserves are sufficient to fund operating plans and debt service obligations into 2028[121](index=121&type=chunk) Cash Flow Summary (in thousands) | | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | ($41,376) | ($33,599) | | Net cash used in investing activities | ($272,633) | ($67) | | Net cash provided by financing activities | $244,251 | $15,900 | [Quantitative and Qualitative Disclosures About Market Risk](index=35&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The primary market risk is interest rate sensitivity, where a 1% increase could decrease the fair value of **$694.7 million** in investments by **$3.6 million**, while foreign currency and inflation risks are minimal - The company's main market risk is interest rate sensitivity. A hypothetical 1% increase in interest rates would decrease the fair value of its marketable securities by approximately **$3.6 million**[140](index=140&type=chunk) - Exposure to foreign currency risk is considered minimal as operations are primarily in the U.S. and contracts in foreign currencies are short-term[141](index=141&type=chunk) - Inflation is not believed to have had a material effect on the business during the reported periods[142](index=142&type=chunk) [Controls and Procedures](index=35&type=section&id=Item%204.%20Controls%20and%20Procedures) Disclosure controls were deemed ineffective as of March 31, 2025, due to a material weakness in IT General Controls, though no material misstatements occurred, and remediation efforts are underway - Management concluded that as of March 31, 2025, the company's disclosure controls and procedures were not effective at a reasonable assurance level[144](index=144&type=chunk) - The ineffectiveness is due to a material weakness identified in the design and maintenance of Information Technology General Controls (ITGCs) over certain key financial IT systems[145](index=145&type=chunk) - The company has begun remediation efforts, including developing training programs, engaging third-party consultants, expanding internal resources, and enhancing reporting to the Audit Committee[147](index=147&type=chunk) [PART II. OTHER INFORMATION](index=38&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Legal Proceedings](index=38&type=section&id=Item%201.%20Legal%20Proceedings) The company reports no material legal proceedings or claims that would significantly adversely affect its business or financial condition - The company is not currently aware of any material legal proceedings or claims against it[151](index=151&type=chunk) [Risk Factors](index=38&type=section&id=Item%201A.%20Risk%20Factors) This section details numerous risks, including limited operating history, substantial funding needs, clinical development uncertainties, reliance on third parties, intellectual property protection, regulatory hurdles, and internal control weaknesses - Financial Risks: The company has a limited operating history, has incurred significant net losses (**$34.1 million** for Q1 2025), and anticipates needing substantial additional funding to continue its development and potential commercialization efforts[153](index=153&type=chunk)[155](index=155&type=chunk)[166](index=166&type=chunk) - Clinical Development Risks: The company has no products approved for sale and has not yet progressed any candidate through a Phase 3 trial. The success of its clinical trials is uncertain, and early positive results are not predictive of final outcomes[158](index=158&type=chunk)[174](index=174&type=chunk)[196](index=196&type=chunk) - Third-Party Reliance: The business is highly dependent on third parties for conducting clinical trials (CROs, NIH) and for the manufacturing of its product candidates, which increases risks related to supply, quality, and regulatory compliance[236](index=236&type=chunk)[253](index=253&type=chunk) - Intellectual Property Risks: The company's success depends on obtaining and maintaining robust patent protection for its technology and product candidates, much of which is in-licensed from partners like Roche, AbbVie, and Mabwell[268](index=268&type=chunk)[270](index=270&type=chunk) - Regulatory and Commercialization Risks: The company may seek accelerated approval for bitopertin, but this pathway is not guaranteed and carries post-marketing requirements. The company also faces risks from healthcare reform, pricing pressures, and competition from potential generics or biosimilars[335](index=335&type=chunk)[343](index=343&type=chunk)[352](index=352&type=chunk) - Internal Control Risks: The company has identified a material weakness in its internal controls over financial reporting related to Information Technology General Controls (ITGCs), which could impact the accuracy of financial reporting[414](index=414&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=122&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) There were no unregistered sales of the company's equity securities during the quarter ended March 31, 2025 - There were no unregistered sales of equity securities during the reported quarter[430](index=430&type=chunk) [Defaults Upon Senior Securities](index=122&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reports no defaults upon its senior securities - No defaults upon senior securities occurred[433](index=433&type=chunk) [Mine Safety Disclosures](index=122&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company's business - This section is not applicable[434](index=434&type=chunk) [Other Information](index=122&type=section&id=Item%205.%20Other%20Information) This section discloses that three executive officers adopted Rule 10b5-1 trading plans during the first quarter of 2025 Adoption of Rule 10b5-1 Trading Plans (Q1 2025) | Name and Title | Date of Adoption | Aggregate Securities to be Sold | | :--- | :--- | :--- | | William Savage, M.D., Ph.D. (CMO) | January 23, 2025 | Up to 2,996 | | John Quisel, J.D., Ph.D. (CEO) | February 13, 2025 | Up to 80,000 | | Jean Franchi (CFO) | March 10, 2025 | Up to 6,031 | [Exhibits](index=123&type=section&id=Item%206.%20Exhibits) This section provides an index of exhibits filed with the Quarterly Report on Form 10-Q, including required officer certifications and an employment agreement - The exhibit index lists all documents filed with the report, including required officer certifications under Sarbanes-Oxley Sections 302 and 906[438](index=438&type=chunk)

Exhibit 99.1 Disc Medicine Reports First Quarter 2025 Financial Results and Provides Business Update WATERTOWN, Mass., May 7, 2025 – Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today reported financial results for the first quarter ended March 31, 2025, and provided a recap of recent program and corporate developments. "2025 is off to a ...

Core Insights - Disc Medicine, Inc. reported strong operational progress in Q1 2025, with a focus on advancing its lead program, bitopertin, for erythropoietic protoporphyria (EPP) [2][5] - The company has a solid financial foundation, with cash reserves expected to fund operations into 2028, following a public offering that raised $259 million [3][9] Financial Performance - Cash, cash equivalents, and marketable securities totaled $694.7 million as of March 31, 2025, compared to $489.9 million at the end of 2024 [20] - Research and Development (R&D) expenses increased to $27.8 million in Q1 2025 from $23.7 million in Q1 2024, driven by advancements in clinical studies and increased headcount [14] - Selling, General and Administrative (SG&A) expenses rose to $12.2 million in Q1 2025 from $7.8 million in Q1 2024, reflecting the establishment of infrastructure for potential commercialization [14] - The net loss for Q1 2025 was $34.1 million, compared to a net loss of $26.9 million in Q1 2024 [14] Clinical Development Updates - The APOLLO trial for bitopertin has been initiated, aimed at supporting the conversion of a potential accelerated approval to full approval [6] - The company plans to submit a New Drug Application (NDA) for bitopertin in EPP in the second half of 2025 [5][6] - Data readouts for the DISC-0974 program, including results from the Phase 2 study for myelofibrosis (MF) anemia and the Phase 1b study for non-dialysis dependent chronic kidney disease (NDD-CKD) anemia, are expected in the second half of 2025 [5][7] - A Phase 2 study of DISC-3405 in polycythemia vera (PV) is set to be initiated in the first half of 2025 [8]

About DISC-0974 DISC-0974 is an investigational monoclonal antibody (mAb) targeting a BMP-signaling co-receptor called hemojuvelin (HJV) and is designed to suppress hepcidin production and increase serum iron levels in patients suffering from anemia of inflammation. DISC-0974 was in-licensed by Disc from AbbVie in 2019. Anemia of inflammation arises from abnormally elevated hepcidin and is the second most common form of anemia, affecting millions of patients in the US across numerous diseases, such as chron ...

Core Insights - Disc Medicine (NASDAQ: IRON) presents an intriguing investment opportunity for those interested in clinical-stage biotech, particularly in the niche of rare hematologic diseases [1] - The company is entering a critical phase, indicating potential for growth and development in its sector [1] Company Analysis - The focus on high-growth companies aligns with sectors expected to experience exponential expansion, highlighting the strategic positioning of Disc Medicine [1] - The analysis emphasizes the importance of innovation and disruptive technologies in generating substantial returns, which is relevant to the company's future prospects [1]

Disc Medicine, Inc. (IRON) appears an attractive pick, as it has been recently upgraded to a Zacks Rank #2 (Buy). This upgrade is essentially a reflection of an upward trend in earnings estimates -- one of the most powerful forces impacting stock prices.The sole determinant of the Zacks rating is a company's changing earnings picture. The Zacks Consensus Estimate -- the consensus of EPS estimates from the sell-side analysts covering the stock -- for the current and following years is tracked by the system.T ...

Shares of Disc Medicine, Inc. (IRON) have gained 0.7% over the past four weeks to close the last trading session at $56.18, but there could still be a solid upside left in the stock if short-term price targets of Wall Street analysts are any indication. Going by the price targets, the mean estimate of $94.82 indicates a potential upside of 68.8%.The average comprises 11 short-term price targets ranging from a low of $73 to a high of $118, with a standard deviation of $13.84. While the lowest estimate indica ...

Financial Performance - The company reported total operating expenses of $129.72 million for the year ended December 31, 2024, an increase of $38.60 million from $91.13 million in 2023[577]. - The net loss for the year ended December 31, 2024, was $109.36 million, compared to a net loss of $76.43 million in 2023, representing an increase in loss of $32.93 million[577]. - The company reported a net cash used in operating activities of $93.9 million for 2024, compared to $73.5 million in 2023, primarily due to a net loss of $109.4 million[589]. - The company has an accumulated deficit of $298.0 million as of December 31, 2024, and anticipates ongoing significant expenses[586]. - The company expects to continue incurring significant operating losses and may require additional capital to fund operations, potentially through equity or debt financing[583]. Expenses - Research and development expenses rose to $96.67 million in 2024, up from $69.26 million in 2023, reflecting an increase of $27.41 million[577]. - Selling, general and administrative expenses increased to $33.05 million in 2024, compared to $21.86 million in 2023, marking an increase of $11.19 million[577]. - Research and development expenses increased to $96.7 million in 2024 from $69.3 million in 2023, a rise of $27.4 million, primarily due to higher personnel-related costs and external development expenses[578]. - Selling, general and administrative expenses rose to $33.0 million in 2024 from $21.9 million in 2023, an increase of $11.2 million, mainly driven by higher personnel-related costs[580]. Cash and Securities - The company had cash, cash equivalents, and marketable securities of $489.9 million as of December 31, 2024, and raised approximately $243.3 million from a public offering in January 2025[567]. - As of December 31, 2024, the company had cash, cash equivalents, and marketable securities totaling $489.9 million, an increase from $360.4 million as of December 31, 2023[617]. - The company completed an underwritten offering in January 2025, expected to generate net proceeds of approximately $243.3 million[585]. - Total contractual obligations as of December 31, 2024, amounted to $3.98 million, with payments due over various periods[597]. Product Development - Bitopertin, the lead product candidate, showed significant reduction in the toxic metabolite protoporphyrin IX (PPIX) in clinical trials, with plans to submit a New Drug Application (NDA) for accelerated approval in the second half of 2025[562]. - DISC-0974, another key product candidate, completed a Phase 1 clinical trial with positive results and is currently in Phase 1b/2 trials for anemia of myelofibrosis and chronic kidney disease[563]. - The company plans to initiate a Phase 2 clinical trial for DISC-3405 in polycythemia vera in the first half of 2025, following the completion of initial trials[566]. - The company has not generated any revenue since inception and does not expect to do so in the near future, pending successful commercialization of product candidates[568]. Income and Interest - Interest income increased to $21.29 million in 2024 from $14.80 million in 2023, while interest expense was $572, reflecting a new debt issuance[577]. - Other income increased to $20.7 million in 2024 compared to $14.8 million in 2023, reflecting a $5.9 million rise in interest income[581]. Market and Economic Factors - If U.S. market interest rates were to increase by one percentage point, the net fair value of the company’s marketable securities would decrease by approximately $1.4 million[617]. - The company has minimal exposure to foreign currency exchange rate fluctuations due to the short duration of transactions[618]. - Inflation has not materially affected the company’s business, financial condition, or results of operations during the years ended December 31, 2024, and 2023[619]. Accounting and Valuation - The company’s stock-based compensation expense is based on fair value estimates using the Black-Scholes option-pricing model, which involves subjective assumptions[606]. - Prior to the merger closing, the estimated fair value of the company’s common stock was determined based on third-party valuations and management assessments[609]. - After the merger, the fair value of the company’s common stock is now determined based on the quoted market price[613]. - The company was classified as an "emerging growth company" until December 31, 2024, allowing it to benefit from certain reporting exemptions[615]. - The company’s accounting policies and estimates are critical to the preparation of its consolidated financial statements, affecting reported amounts of assets and liabilities[602].

Financial Performance - Disc Medicine reported a net loss of $109.4 million for the full year 2024, compared to a net loss of $76.4 million in 2023, reflecting an increase of 43%[13] - Total operating expenses for 2024 were $129.7 million, compared to $91.1 million in 2023, marking a 42% increase[17] - Total stockholders' equity increased to $443.6 million as of December 31, 2024, compared to $345.1 million in 2023[19] Research and Development - Research and development expenses increased to $96.7 million in 2024 from $69.3 million in 2023, driven by advancements in clinical studies and increased headcount[13] - Positive data from the Phase 1b trial of DISC-0974 in myelofibrosis anemia was presented, with initial data from the ongoing Phase 2 expected in H2 2025[4] - The company presented positive preclinical data for DISC-3405 in sickle cell disease, highlighting its potential therapeutic benefits[4] Cash Position and Funding - The company ended 2024 with $490 million in cash, cash equivalents, and marketable securities, up from $360.4 million at the end of 2023, providing a cash runway into 2028[4] - The company completed a public offering in January 2025, raising approximately $259 million, which will support ongoing operations[4] Future Plans - The company plans to submit a New Drug Application (NDA) for bitopertin in the second half of 2025, targeting accelerated approval based on clinical data[4] - The company is on track to initiate the APOLLO trial for bitopertin by mid-2025, following alignment with the FDA on trial design[4]

Core Insights - Disc Medicine, Inc. reported significant progress in 2024, particularly with the potential accelerated approval for bitopertin based on Phase 2 results in erythropoietic protoporphyria (EPP) patients [2][5] - The company plans to initiate the APOLLO trial in mid-2025 and submit a New Drug Application (NDA) in the second half of 2025 [2][5] - Positive clinical data was reported across the company's portfolio, including for DISC-0974 in anemias of myelofibrosis (MF) and non-dialysis-dependent chronic kidney disease (NDD-CKD) [2][5] Recent Highlights and Anticipated Milestones - Bitopertin is positioned for NDA submission in EPP through an accelerated approval pathway, with the APOLLO trial set to begin by mid-2025 [5][6] - Positive updates from Phase 1b trials of DISC-0974 in MF and NDD-CKD were presented, with initial data from ongoing studies expected in H2 2025 [5][6] - The company also plans to initiate a Phase 2 study for DISC-3405 in polycythemia vera (PV) in H1 2025 [5][6] Financial Overview - As of December 31, 2024, the company reported cash, cash equivalents, and marketable securities totaling $489.9 million, an increase from $360.4 million in 2023 [9][18] - Research and development expenses rose to $96.7 million in 2024 from $69.3 million in 2023, driven by advancements in clinical studies and increased headcount [9][15] - The net loss for 2024 was $109.4 million, compared to $76.4 million in 2023, attributed to higher operating costs [9][16]