Financial Performance - In Q4 2025, Agios reported worldwide net revenues of $20.0 million for PYRUKYND® (mitapivat) and $54.0 million for the full year [5]. - U.S. net revenue from PYRUKYND in Q4 2025 was $16.0 million, a 49% increase from $10.7 million in Q4 2024 and a 24% increase from $12.9 million in Q3 2025 [6]. - The company had a net loss of $108.0 million in Q4 2025, compared to a net loss of $96.5 million in Q4 2024 [10]. - Total revenue for 2025 was $54.0 million, an increase from $36.5 million in 2024 [21]. Cash and Expenses - As of December 31, 2025, Agios had $1.2 billion in cash, cash equivalents, and marketable securities, down from $1.5 billion a year earlier [16]. - Research and Development (R&D) expenses for Q4 2025 were $88.1 million, up from $82.8 million in Q4 2024 [16]. - Selling, General and Administrative (SG&A) expenses were $51.6 million in Q4 2025, nearly flat compared to $51.7 million in Q4 2024 [16]. Product Development and Approvals - The FDA approved AQVESME™ (mitapivat) in December 2025, making it the only medicine for treating anemia in adults with alpha- or beta-thalassemia [11]. - Agios plans to expand its PK activation franchise into additional indications such as sickle cell disease and lower-risk myelodysplastic syndromes in 2026 [3]. - The Phase 2 trial of tebapivat in sickle cell disease is fully enrolled, with topline results expected in the second half of 2026 [9].

Core Insights - Agios Pharmaceuticals reported strong execution in 2025, highlighted by the U.S. approval and launch of AQVESME, the first treatment for anemia in adults with thalassemia, which has received positive feedback from the community [2][5] - The company aims to expand its product portfolio and pipeline, focusing on high-value indications such as sickle cell disease and myelodysplastic syndromes, while maintaining disciplined capital allocation [2] Financial Performance - For Q4 2025, Agios reported net revenues of $20.0 million from PYRUKYND, with a full-year total of $54.0 million, marking a 49% increase from Q4 2024 [5][12] - The net loss for Q4 2025 was $108.0 million, compared to a net loss of $96.5 million in Q4 2024 [8] - Total revenues for the year 2025 reached $54.0 million, up from $36.5 million in 2024 [15] Research and Development Highlights - AQVESME received FDA approval in December 2025 and is now available in the U.S. [6][12] - A pre-sNDA meeting with the FDA for mitapivat in sickle cell disease is scheduled for Q1 2026, with plans to submit a marketing application thereafter [5][6] - The Phase 2 trial of tebapivat in sickle cell disease has been fully enrolled, with topline results expected in the second half of 2026 [7] Cash Position - As of December 31, 2025, Agios had $1.2 billion in cash, cash equivalents, and marketable securities, down from $1.5 billion a year earlier [12][14] - The company anticipates that its financial resources will support the U.S. launch of AQVESME and the advancement of its clinical programs [12]

Wall Street expects a year-over-year decline in earnings on lower revenues when Agios Pharmaceuticals (AGIO) reports results for the quarter ended December 2025. While this widely-known consensus outlook is important in gauging the company's earnings picture, a powerful factor that could impact its near-term stock price is how the actual results compare to these estimates.The stock might move higher if these key numbers top expectations in the upcoming earnings report, which is expected to be released on Fe ...

Core Viewpoint - Agios Pharmaceuticals, Inc. will host a conference call and live webcast on February 12, 2026, to report its fourth quarter and full year 2025 financial results and business highlights [1]. Company Overview - Agios Pharmaceuticals is a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases [1]. - The company aims to redefine the future of rare disease treatment by building trusted partnerships and collaborating to develop transformative medicines [3]. - Agios is headquartered in Cambridge, Massachusetts, and has a foundation in hematology, combining biological expertise with real-world insights to advance its pipeline of rare disease medicines [3]. Event Details - The live webcast will be accessible on the Investors section of Agios' website under the "Events & Presentations" tab [2]. - A replay of the webcast will be available approximately two hours after the event on the company's website [2]. Contacts - Investor Contact: Morgan Sanford, Vice President, Investor Relations, Agios Pharmaceuticals [4]. - Media Contact: Eamonn Nolan, Senior Director, Corporate Communications, Agios Pharmaceuticals [4].

Core Thesis - Agios Pharmaceuticals, Inc. is positioned as a compelling investment opportunity due to its innovative therapies and strong financial standing, particularly with the recent FDA approval of its lead product, mitapivat, for thalassemia [1][4][5] Company Overview - Agios Pharmaceuticals is a clinical-stage biotech company focused on cellular metabolism through oral, small-molecule allosteric activators of the pyruvate kinase (PK) enzyme [2] - The lead product, mitapivat, is marketed as PYRUKYND for PK deficiency and received FDA approval in December 2025 under the brand name AQVESME for alpha- and beta-thalassemia [2] Clinical Trials and Efficacy - Phase 3 trials in thalassemia demonstrated strong efficacy, with ENERGIZE showing a 42.3% hemoglobin response in non-transfusion-dependent patients and ENERGIZE-T achieving a 30.4% transfusion reduction in dependent patients [3] - In sickle cell disease, the RISE UP trial met hemoglobin endpoints but showed only favorable trends in pain crisis reduction [3] - Agios is advancing tebapivat, which achieved proof-of-concept in lower-risk myelodysplastic syndromes, with 40% of patients reaching transfusion independence [3] Financial Position - As of Q3 2025, Agios holds approximately $1.3 billion in cash and marketable securities, sufficient to fund launches and clinical programs without dilutive financing [4] - PYRUKYND revenues grew 44% year-over-year, although net losses persist due to high R&D and SG&A expenses [4] Market Position and Competitive Advantage - The thalassemia approval provides a first-mover advantage in a larger patient population, while the upcoming sickle cell opportunity could further expand its market [4] - Agios differentiates from competitors like Reblozyl and gene therapies by offering a twice-daily pill with broad accessibility [4] Future Catalysts - Led by CEO Brian Goff, the company is positioned to scale globally, with upcoming catalysts in early 2026 including the AQVESME U.S. launch, FDA discussions for sickle cell indications, and Phase 2b tebapivat data [5] - Multiple approved indications and a strong balance sheet enhance Agios's potential for successful commercial execution and global expansion [5]

Core Insights - Agios is at a pivotal growth inflection point, driven by three key components [3] - The PK activator franchise is advancing as the standard of care for various hemolytic anemias, establishing Agios's leadership in rare hematology [3] - The company is progressing its early and mid-stage pipeline, which has the potential to unlock further value [4] - Recent approval for thalassemia provides a clear path to profitability with existing products [4]

Summary of Agios Pharmaceuticals FY Conference Call (January 14, 2026) Company Overview - **Company**: Agios Pharmaceuticals (NasdaqGS:AGIO) - **Event**: 44th Annual J.P. Morgan Healthcare Conference - **Speaker**: CEO Brian Goff Key Industry and Company Insights Growth and Strategic Focus - Agios is at a pivotal growth inflection point driven by three key components: 1. Advancement of the pyruvate kinase (PK) activator franchise as the standard of care for hemolytic anemias [2] 2. Progress in early and mid-stage pipeline, unlocking further value [3] 3. Clear path to profitability with the existing commercial portfolio, especially following the recent approval for thalassemia [3] Pipeline and Market Potential - The PK activation franchise is anchored on mitapivat, approved for pyruvate kinase deficiency and thalassemia, with ongoing pursuits in sickle cell disease [4] - The total market potential for Agios's pipeline by 2030 is projected to exceed $10 billion, with thalassemia alone representing over $1 billion [4][26] - The company is also advancing tevapivat for sickle cell disease and low-risk myelodysplastic syndromes (MDS) [4][16] Recent Approvals and Launch Strategy - Acvezmi, approved for thalassemia on December 23, 2025, is positioned as a historic approval for the thalassemia community [7][8] - The launch strategy includes a highly experienced field force, a patient support program (myAgios), and a focus on global expansion [5][6] - The pricing for Acvezmi is set at $425,000 per patient per year in the U.S., reflecting its clinical value [11] Market Dynamics and Patient Population - Approximately 4,000 adult patients in the U.S. are deemed addressable at launch, with a total adult population of about 6,000 [10] - The launch will initially focus on transfusion-dependent patients, who are in regular contact with healthcare systems [39] Financial Discipline and Future Outlook - Agios aims to maintain flat operating expenses in 2026 compared to 2025 while maximizing the Acvezmi launch opportunity [21][23] - The company is committed to financial discipline to ensure long-term sustainability and growth [21] Pipeline Developments and Future Catalysts - Upcoming milestones include: - Pre-SNDA meeting with the FDA regarding sickle cell disease data [24][45] - Phase 2b readout for tevapivat in low-risk MDS [24] - Phase 1 healthy volunteer data for the TMPRSS6 inhibitor for polycythemia vera [24] - Phase 2 data for tevapivat in sickle cell disease and proof of mechanism data for AG181 for phenylketonuria [25] Conclusion - Agios Pharmaceuticals is positioned for significant growth in 2026, focusing on the successful launch of Acvezmi and advancing its pipeline while maintaining financial discipline [24][30]

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Business Overview - Agios is positioned for growth and aims for profitability with its existing commercial portfolio[3, 4] - The company has a strong foundation in hematology and aspires to become a sustainable rare disease company[5] - Agios estimates a total global market size of over $10 billion by 2030 for its current pipeline indications[11] Product Pipeline and Approvals - AQVESME (mitapivat) is now approved in the U S for anemia in adults with alpha- or beta-thalassemia, addressing approximately 4,000 addressable patients at launch[7, 17, 23] - AQVESME has a potential to deliver $1 billion in global peak-year-sales across PKD and thalassemia indications[24] - Pyrukynd is approved in the U S , EU, and UK for Pyruvate Kinase Deficiency[7] Clinical Development - In Sickle Cell Disease, 40.6% of patients achieved Hemoglobin response (≥1 g/dL) in the mitapivat trial[37] - Phase 1 tebapivat data demonstrates a 1.2 g/dL increase (2mg) and 1.9 g/dL increase (5mg) in mean change from baseline in Hb across dosing cohorts at Day 28 in SCD patients[44] - In LR-MDS, 40% of low transfusion burden cohort achieved transfusion independence with tebapivat[51]

Core Insights - Agios Pharmaceuticals (AGIO) has outlined its strategic priorities for 2026, focusing on key milestones and commercial activities in the coming year [1] Group 1: Strategic Priorities - The U.S. commercial launch of AQVESME for thalassemia is underway following FDA approval in December 2025 [1] - A pre-sNDA meeting with the FDA for mitapivat in sickle cell disease is anticipated in the first quarter of 2026, with a planned U.S. regulatory submission to follow [1] Group 2: Pipeline Development - The company is advancing its early- and mid-stage pipeline across multiple high-value indications [1] Group 3: Financial Outlook - Agios Pharmaceuticals aims for a path to profitability through its existing commercial presence in thalassemia and PK deficiency, with the potential to achieve over $1 billion in peak global sales [1]