Core Insights - Agios Pharmaceuticals reported a notable revenue beat in Q2 2025, with GAAP net product revenue of $12.5 million for its lead product PYRUKYND, reflecting strong sales momentum despite increasing costs as the company prepares for major regulatory events and product launches [1][5] Financial Performance - EPS (GAAP) for Q2 2025 was $(1.93), a decline of 14.2% year-over-year from $(1.69) in Q2 2024 [2] - Revenue (GAAP) reached $12.5 million, up 45.4% from $8.6 million in Q2 2024 [2] - Net loss increased to $(112.0 million), a rise of 16.5% compared to $(96.1 million) in Q2 2024 [2][8] - R&D expenses rose to $91.9 million, an increase of 18.7% from $77.4 million in Q2 2024 [2] - SG&A expenses increased by 29.3% to $45.9 million, driven by preparations for regulatory approvals [2][6] Business Overview and Strategy - Agios specializes in therapies for rare blood disorders, focusing on high unmet needs, with PYRUKYND targeting pyruvate kinase deficiency [3] - The company aims to expand PYRUKYND's reach to other rare diseases, including thalassemia and sickle cell disease, through clinical trials and regulatory filings [3] Regulatory and Commercial Developments - The company is preparing for a key regulatory milestone with a PDUFA goal date of September 7, 2025, for PYRUKYND's approval in thalassemia [6][7] - Agios has established a European distribution partnership with Avanzanite Bioscience to launch PYRUKYND across Europe and the UK [6] - The FDA is currently reviewing the application for PYRUKYND in thalassemia, with late-stage trial results for sickle cell disease expected by year-end [7][9] Pipeline and Future Outlook - PYRUKYND product revenue grew 44.6% year-over-year, with unique patients enrolling for treatment reaching 248, a 6% increase from the prior quarter [5] - The company closed the quarter with $1.34 billion in cash and marketable securities, a decrease from year-end 2024 [8] - Management did not provide detailed financial guidance for fiscal 2025 but indicated that the current liquidity position supports plans for new launches and ongoing research investments [10]

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, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File Number: 001-36014 AGIOS PHARMACEUTICALS, INC. (Exact Name of Registrant as Specified in Its Charter) Delaware 26-0662915 (State or Other Jurisdiction of Inc ...

Financial Data and Key Metrics Changes - In Q2 2025, the company reported net revenue of $12.5 million, a 45% increase compared to $8.6 million in Q2 2024 and a 44% increase compared to $8.7 million in Q1 2025 [9][10] - The company ended Q2 2025 with approximately $1.3 billion in cash, cash equivalents, and marketable securities, indicating a strong financial position to support future growth [7][11] Business Line Data and Key Metrics Changes - The net revenue growth was attributed to strong commercial execution in Pyrokine, with an increase in the number of patients on active treatment [10][15] - The company dosed the first patient in the Phase II trial of tebapivat in sickle cell disease and received IND clearance for AG-236, targeting polycythemia vera [8][9] Market Data and Key Metrics Changes - The company anticipates a potential launch of Pyrokine for thalassemia in the U.S. pending FDA approval, with a PDUFA goal date set for September 7, 2025 [6][19] - The GCC market has an estimated 70,000 thalassemia patients, with a focus on Saudi Arabia for initial market entry [23][24] Company Strategy and Development Direction - The company aims to deliver sustainable growth and unlock long-term shareholder value through a capital-efficient commercial build-out and strategic investments in its pipeline [5][12] - The focus is on expanding the pipeline through internal efforts and business development activities, with a strong emphasis on rare disease treatments [13][32] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential to transform the treatment landscape for thalassemia and sickle cell disease with Pyrokine, highlighting the high unmet need in these patient populations [8][19] - The company is well-prepared for the potential launch of Pyrokine in thalassemia, with a robust commercial strategy and a dedicated sales force [21][56] Other Important Information - The company has entered into revenue-sharing agreements with Avanzanite Bioscience for commercialization in Europe and Newbridge Pharmaceuticals for the GCC, which are expected to favor Agios in the long term [12][22] - The company is actively engaging with healthcare professionals and patients to understand their needs better, which is crucial for the successful launch of Pyrokine [20][57] Q&A Session Summary Question: Any updates on ometipivat safety profile? - Management confirmed there are no new updates regarding the safety profile, including liver toxicity [36][37] Question: Insights on GCC approval dates? - Management indicated ongoing discussions across multiple regions and readiness for commercialization [38] Question: Are you in labeling discussions for thalassemia? - Management stated that updates to the PKD label are anticipated to reflect the new indication and dosing [43][46] Question: Expectations for SG&A spending? - Management expects some growth in SG&A expenses related to the thalassemia launch, with additional launch-related expenses anticipated upon approval [48][49] Question: Initial target patient population for thalassemia? - Management identified approximately 4,000 symptomatic patients as the initial target for the launch in the U.S. [55][56] Question: Pediatric opportunity for thalassemia? - Management plans to expand trials to the pediatric population once adult data is available [66][67] Question: Changes in sickle cell trial protocol? - Management confirmed that monitoring protocols have been aligned with previous findings to ensure patient safety [82][83] Question: Will hepatocellular injury be in the label? - Management indicated that the final label will be determined at the PDUFA date, with ongoing discussions with the FDA [88][92] Question: Prescriber base for thalassemia launch? - Management emphasized the importance of engaging both academic centers and community hematologists to address the unmet needs of thalassemia patients [96][98]

Financial Data and Key Metrics Changes - In Q2 2025, Agios Pharmaceuticals reported net revenue of $12.5 million, a 45% increase compared to $8.6 million in Q2 2024 and a 44% increase compared to $8.7 million in Q1 2025 [9][10] - The company ended the quarter with approximately $1.3 billion in cash, cash equivalents, and marketable securities, indicating a strong financial position to support future growth [6][11] Business Line Data and Key Metrics Changes - The net revenue growth in the second quarter was attributed to strong commercial execution in pyruvate kinase deficiency (PKD) and an increase in the number of units processed directly by specialty pharmacies [10][14] - The company anticipates continued variability in net revenues due to ordering patterns, especially with the upcoming focus on thalassemia treatment [10][11] Market Data and Key Metrics Changes - As of Q2 2025, 248 patients completed prescription enrollment forms, a 6% increase from the previous year, with 142 patients actively receiving treatment, reflecting a 4% sequential increase [15] - The company is preparing for a potential launch in thalassemia, with a focus on the 4,000 patients actively managed due to their symptoms [19][53] Company Strategy and Development Direction - Agios aims to deliver sustainable growth and unlock long-term shareholder value through the commercialization of Pyrokine and the advancement of its pipeline [4][30] - The company has entered into revenue-sharing agreements with Avanzanite Bioscience for Europe and Newbridge Pharmaceuticals for the GCC, allowing for capital-efficient commercialization strategies [12][20] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential to transform the treatment landscape for thalassemia and sickle cell disease with Pyrokine, pending FDA approval [5][8] - The company is focused on building a diversified rare disease portfolio and advancing its PK activator franchise across multiple indications [31][30] Other Important Information - The company has received IND clearance for AG-236, an siRNA targeting TMPRSS6 for polycythemia vera, and has dosed the first patient in the Phase II trial of tebapivat in sickle cell disease [7][29] - Agios is actively engaging with healthcare professionals and patients to understand their needs and prepare for the upcoming product launches [18][54] Q&A Session Summary Question: Any updates on ometipivat safety profile? - Management confirmed there are no new updates regarding the safety profile [36] Question: Insights on GCC approval dates? - Management indicated ongoing discussions across multiple regions and readiness for commercialization [37] Question: Are you in labeling discussions for thalassemia? - Management stated that updates to the PKD label are anticipated to reflect the new indication and dosing [44] Question: Expectations for SG&A spending? - Management expects some growth in SG&A expenses related to the thalassemia launch, with additional launch-related expenses anticipated upon approval [46] Question: Initial target patient population for thalassemia? - The initial focus will be on approximately 4,000 patients who are actively managed due to their symptoms [52] Question: Pediatric opportunity for thalassemia? - Management confirmed a high unmet need in pediatric patients and plans to run trials once adult data is available [64] Question: Changes in sickle cell trial protocol? - Management confirmed that monitoring protocols have been updated to align with safety concerns identified in other trials [81] Question: Will hepatocellular injury be in the label? - Management indicated that the final label will be determined at the PDUFA date, with ongoing discussions with the FDA [90]

Agios Pharmaceuticals shares have added about 14.2% since the beginning of the year versus the S&P 500's gain of 8.2%. What's Next for Agios Pharmaceuticals? While Agios Pharmaceuticals has outperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock? There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expect ...

Financial Performance - PYRUKYND net revenues reached $12.5 million in Q2 2025, a 44% increase compared to $8.7 million in Q1 2025 and a 45% increase compared to $8.6 million in Q2 2024[10] - The company maintains a strong financial position with $1.3 billion in cash on hand[9, 10, 14] - Net loss for Q2 2025 was $112.0 million, compared to a net loss of $96.1 million for Q2 2024[14] Pipeline Development - First patient dosed in Phase 2 Sickle Cell Disease trial for AG-236 after receiving IND clearance[11] - RISE UP Phase 3 trial in sickle cell disease is expected to have topline data by the end of 2025[37] - Tebapivat Phase 2b trial for LR-MDS is ongoing, with topline results expected in early 2026[9, 45] Commercialization - 248 unique PK deficiency patients completed prescription enrollment forms since launch in the U S [21] - 142 patients are on treatment in the U S , including new prescriptions and treatment continuations, with 215 unique prescribers[22] - The company anticipates a potential U S launch of PYRUKYND for thalassemia in Q3 2025, with a PDUFA goal date of September 7, 2025[11, 23, 24] Strategic Partnerships - Announced a commercialization and distribution partnership with Avanzanite Bioscience in Europe[10] - A commercialization and distribution agreement is in place with NewBridge for the GCC region, with anticipated first regulatory approval in the coming months[30]

[Business and Operational Highlights](index=1&type=section&id=Business%20and%20Operational%20Highlights) Agios reported strong PYRUKYND® commercial performance, advanced its R&D pipeline, and prepared for potential U.S. approval for thalassemia - The company is focused on the upcoming PDUFA goal date of September 7, 2025, for the potential U.S. approval of PYRUKYND for thalassemia[3](index=3&type=chunk) - Topline results from the RISE UP Phase 3 trial of mitapivat in sickle cell disease are expected by the end of the year, with a potential U.S. commercial launch in 2026[3](index=3&type=chunk)[5](index=5&type=chunk)[10](index=10&type=chunk) - Entered into a distribution agreement with Avanzanite Bioscience B.V. to commercialize PYRUKYND in the European Economic Area, the UK, and Switzerland[6](index=6&type=chunk) [Commercial Performance – PYRUKYND® (mitapivat)](index=1&type=section&id=Commercial%20Performance%20%E2%80%93%20PYRUKYND%C2%AE%20%28mitapivat%29) PYRUKYND® net revenue reached **$12.5 million** in Q2 2025, driven by steady growth in patient enrollment and therapy PYRUKYND® Q2 2025 Performance | Metric | Q2 2025 | Q2 2024 | QoQ Growth (vs Q1 2025) | | :--- | :--- | :--- | :--- | | Net Revenue | $12.5 million | $8.6 million | N/A | | Unique Patient Enrollments | 248 | N/A | 6% | | Patients on Therapy | 142 | N/A | 4% | [R&D Highlights](index=2&type=section&id=R%26D%20Highlights) R&D pipeline advanced with PYRUKYND's sNDA for thalassemia, progress in sickle cell and LR-MDS trials, and AG-236 IND clearance - **PYRUKYND (mitapivat):** - **Thalassemia:** sNDA under active FDA review with a PDUFA goal date of September 7, 2025 - **Sickle Cell Disease:** Topline Phase 3 (RISE UP) results on track for year-end 2025[10](index=10&type=chunk) - **Tebapivat:** - **Sickle Cell Disease:** Dosed the first patient in a Phase 2 trial - **Lower-risk Myelodysplastic Syndromes (LR-MDS):** Phase 2b trial enrollment is progressing, with completion targeted by the end of 2025[8](index=8&type=chunk)[10](index=10&type=chunk) - **Early Pipeline:** - Received Investigational New Drug (IND) clearance for AG-236, an siRNA targeting TMPRSS6 for treating polycythemia vera (PV)[10](index=10&type=chunk) [Second Quarter 2025 Financial Results](index=2&type=section&id=Second%20Quarter%202025%20Financial%20Results) Agios reported a **net loss of $112.0 million** in Q2 2025, driven by increased R&D and SG&A expenses, with **$1.3 billion** in cash Q2 2025 Key Financial Metrics (in millions, vs. Q2 2024) | Metric | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | Net Product Revenue | $12.5 million | $8.6 million | | R&D Expenses | $91.9 million | $77.4 million | | SG&A Expenses | $45.9 million | $35.5 million | | Net Loss | $112.0 million | $96.1 million | - The increase in R&D expenses was primarily due to a **$10.0 million** regulatory milestone payment to Alnylam for AG-236[13](index=13&type=chunk) - The increase in SG&A expenses was mainly attributable to increased commercial activities in preparation for the potential approval of PYRUKYND in thalassemia[13](index=13&type=chunk) - The company ended the quarter with **$1.3 billion** in cash, cash equivalents, and marketable securities, expected to fund operations, potential launches, and pipeline expansion[5](index=5&type=chunk)[13](index=13&type=chunk) [Financial Statements](index=5&type=section&id=Financial%20Statements) Consolidated financial statements show **$1.47 billion** in total assets and a **net loss of $112.0 million** for Q2 2025, or **($1.93)** per share [Consolidated Balance Sheet Data](index=5&type=section&id=Consolidated%20Balance%20Sheet%20Data) As of June 30, 2025, Agios reported **$1.34 billion** in cash, **$1.47 billion** in total assets, and **$1.37 billion** in stockholders' equity Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents, and marketable securities | $1,339,404 | $1,532,031 | | Total assets | $1,471,237 | $1,663,199 | | Stockholders' equity | $1,369,555 | $1,540,956 | [Consolidated Statements of Operations Data](index=5&type=section&id=Consolidated%20Statements%20of%20Operations%20Data) For Q2 2025, net product revenue was **$12.5 million**, with loss from operations at **$127.1 million** and a **net loss of $112.0 million**, or **($1.93)** per share Statement of Operations Summary (in thousands, except per share data) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | Product revenue, net | $12,455 | $8,615 | | Total operating expenses | $139,511 | $114,432 | | Loss from operations | $(127,056) | $(105,817) | | Net loss | $(112,020) | $(96,118) | | Net loss per share | $(1.93) | $(1.69) |

Core Viewpoint - Agios Pharmaceuticals reported its financial results for Q2 2025, highlighting progress in its drug pipeline, particularly with PYRUKYND for thalassemia and ongoing trials for sickle cell disease [1][2]. Financial Performance - The company generated $12.5 million in net revenue for Q2 2025, an increase from $8.6 million in Q2 2024 [6][11]. - The net loss for Q2 2025 was $112.0 million, compared to a net loss of $96.1 million in Q2 2024 [7][17]. - As of June 30, 2025, Agios had $1.3 billion in cash, cash equivalents, and marketable securities, down from $1.5 billion at the end of 2024 [11][14]. Product Development and Pipeline - PYRUKYND's supplemental New Drug Application (sNDA) for thalassemia is under active review by the FDA, with a PDUFA goal date of September 7, 2025 [5][6]. - The topline results from the RISE UP Phase 3 trial for PYRUKYND in sickle cell disease are expected by year-end 2025, with a potential U.S. commercial launch in 2026 [5][6]. - The first patient has been dosed in the Phase 2 trial for tebapivat in sickle cell disease, which is currently enrolling patients [11]. Operational Highlights - There was a 6% increase in unique patients completing prescription enrollment forms compared to Q1 2025, totaling 248 patients [6]. - The number of patients on PYRUKYND therapy increased by 4% over Q1 2025, reaching 142 patients [6]. - Agios entered into a distribution agreement with Avanzanite Bioscience B.V. to commercialize PYRUKYND across the European Economic Area, the UK, and Switzerland [6]. Research and Development Expenses - R&D expenses for Q2 2025 were $91.9 million, up from $77.4 million in Q2 2024, primarily due to a $10 million regulatory milestone payment [11][17]. - Selling, General and Administrative (SG&A) expenses increased to $45.9 million in Q2 2025 from $35.5 million in Q2 2024, attributed to increased commercial activities [11][17]. Strategic Vision - Agios aims to redefine the future of rare disease treatment by developing innovative medicines that can transform patients' lives [9].

Core Insights - Agios Pharmaceuticals will host a conference call and live webcast on July 31, 2025, at 8:00 a.m. ET to report its Q2 2025 financial results and business highlights [1] - The live webcast will be accessible on the company's website, with a replay available approximately two hours after the event [2] Company Overview - Agios Pharmaceuticals is a leader in pyruvate kinase (PK) activation, focusing on developing therapies for rare diseases [3] - The company markets a first-in-class PK activator for adults with PK deficiency, which is the first disease-modifying therapy for this condition [3] - Agios is advancing a clinical pipeline that includes investigational medicines for alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndrome (MDS)-associated anemia, and phenylketonuria (PKU) [3] - Additionally, Agios is developing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera [3]

Core Insights - Agios Pharmaceuticals has appointed Dr. Jay Backstrom to its Board of Directors, which is expected to enhance the company's strategic direction and clinical program advancements [1][2][3] Company Overview - Agios Pharmaceuticals is a leader in cellular metabolism and pyruvate kinase (PK) activation, focusing on therapies for rare diseases [1][4] - The company markets a first-in-class PK activator for adults with PK deficiency, representing the first disease-modifying therapy for this condition [4] Leadership and Expertise - Dr. Backstrom brings extensive experience from his previous roles, including President and CEO of Scholar Rock and Executive Vice President of Research and Development at Acceleron Pharma [2][3] - His leadership has been instrumental in advancing clinical programs to regulatory approval, particularly in rare diseases [2][3] Strategic Focus - Agios is focused on maximizing the potential of its PK activator, PYRUKYND (mitapivat), and advancing its pipeline of rare disease medicines [2][3] - The company is developing investigational medicines for conditions such as alpha- and beta-thalassemia, sickle cell disease, and myelodysplastic syndromes [4]