IntrinsicIntrinsic
← All summaries

AGIOS PHARMACEUTICALS, INC.

AGIO
Financials & Chart →

Business Summary

Agios Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company focused on redefining rare disease treatment, with a foundation in hematology and expertise in cellular metabolism. The company generates revenue through its approved products and is advancing a pipeline of rare disease medicines. Its core business model involves developing and commercializing innovative medicines, with a mix of product sales and potential milestone and royalty income from collaborations . The primary customer segments for its approved products are specialty distributors and specialty pharmacy providers in the United States, and distribution partners in other regions .

The company's lead product candidate, mitapivat, is an activator of pyruvate kinase (PK) enzymes for the potential treatment of hemolytic anemias. Mitapivat is approved in the United States under the brand name AQVESME™ for the treatment of anemia in adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia . It is also approved under the brand name PYRUKYND® in the United States for the treatment of hemolytic anemia in adults with PK deficiency, and in the European Union (EU) and Great Britain for the treatment of PK deficiency in adults . In Saudi Arabia, PYRUKYND® is approved for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia . The company commercially launched AQVESME™ in the United States in late January 2026 .

Agios is also developing tebapivat, a novel PK activator, for the potential treatment of lower-risk myelodysplastic syndromes (LR MDS) and sickle cell disease (SCD) . AG-181, a phenylalanine hydroxylase (PAH) stabilizer, is being developed for the potential treatment of phenylketonuria (PKU) . Additionally, AG-236, an siRNA targeting the transmembrane serine protease 6 (TMPRSS6) gene, is in development for the potential treatment of polycythemia vera (PV) .

For the fiscal year ended December 31, 2025, total revenue was $54.028 million . This comprised U.S. product revenue of $49.170 million and rest of world product revenue of $4.858 million . Cost of sales for the period was $6.345 million . Research and development expenses totaled $339.535 million , and selling, general and administrative expenses were $180.280 million . The company reported a net loss of $412.781 million and a diluted EPS of $(7.12) . As of December 31, 2025, cash and cash equivalents were $89.130 million , and marketable securities totaled $1.075 billion (current marketable securities of $765.295 million plus non-current marketable securities of $310.013 million ). The company's accumulated deficit as of December 31, 2025, was $561.705 million .

Comparing 2025 to 2024, total revenue increased by $17.5 million , driven by a $12.8 million increase in U.S. product revenue due to increased PYRUKYND® volume and a $4.7 million increase in rest of world product revenue from the Avanzanite Agreement. Total operating expenses increased by $63.9 million , primarily due to a $38.2 million increase in research and development expenses and a $23.5 million increase in selling, general and administrative expenses, the latter driven by preparations for AQVESME™ approval. The net loss in 2025 of $412.781 million contrasts with a net income of $673.725 million in 2024, with the 2024 income primarily attributable to the $889.1 million gain on sale of contingent payments and the $200.0 million Vorasidenib Milestone Payment.

During 2025, the FDA approved AQVESME™ for the treatment of anemia in adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia in December 2025 , with a commercial launch in the United States in late January 2026 . The Saudi Food and Drug Authority approved PYRUKYND® for the treatment of adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia in August 2025 . The CHMP of the EMA adopted a positive opinion for a new indication for PYRUKYND® in adults for the treatment of anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia in October 2025 , with a final decision expected in early 2026 . In November 2025, the phase 3 portion of the RISE UP trial for mitapivat in SCD achieved its primary endpoint of hemoglobin response, with 40.6% of patients in the mitapivat arm achieving a response compared to 2.9% in the placebo arm . The annualized rate of SCPCs was 2.62 in the mitapivat arm and 3.05 in the placebo arm, not achieving statistical significance for that primary endpoint . The ACTIVATE-kids trial for pediatric PK deficiency met its primary endpoint of hemoglobin response, with 31.6% of patients in the mitapivat arm achieving a response compared to 0% in the placebo arm . The ACTIVATE-kidsT trial for regularly transfused pediatric PK deficiency patients did not meet its primary endpoint of transfusion reduction response using Bayesian methodology , with 28.1% of patients in the mitapivat arm achieving the primary endpoint compared to 11.8% in the placebo arm . A regulatory milestone for AG-236 triggered a $10.0 million payment to Alnylam in 2025 . The phase 1 clinical trial of AG-181 in healthy volunteers was completed in December 2025 .

Business Outlook

The European Commission is reviewing the CHMP's positive opinion for PYRUKYND® in adults for the treatment of anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia, with a final decision expected in early 2026 . The company plans to have a pre-supplemental New Drug Application (sNDA) meeting with the FDA in the first quarter of 2026 and intends to submit a U.S. marketing application for mitapivat in sickle cell disease (SCD) following that engagement .

A major growth area for the company is the expansion of its PK activator franchise, particularly mitapivat, across multiple hemolytic anemias. Mitapivat is being evaluated for the treatment of pediatric patients with PK deficiency . The company also expects to initiate a phase 1b proof of mechanism trial of AG-181 in patients with PKU in the first half of 2026 and confirm proof of mechanism in the second half of 2026 . For AG-236, a phase 1 clinical trial in healthy volunteers was initiated in July 2025 , and topline data for this trial are expected in the first half of 2026 .

Regarding operational outlook, the company expects to continue to incur significant expenses and net losses until it is able to report profitable results . Research and development costs related to its portfolio are expected to increase as product candidate development programs progress . Selling, general and administrative expenses are also anticipated to increase to support continued research and development activities and ongoing and future commercialization activities, including the hiring of additional personnel . The company has updated its mitapivat clinical trial protocols across all indications to incorporate monthly monitoring of liver tests for the first six months of treatment, following observations of hepatocellular injury in the ENERGIZE and ENERGIZE-T trials .

Planned capital allocation includes funding future development and commercialization costs related to PYRUKYND® and AQVESME™ . The company expects its existing cash, cash equivalents, and marketable securities as of December 31, 2025, together with anticipated product revenue and interest income, to provide the financial independence to commercially launch AQVESME™ in the United States, prepare for the potential U.S. commercial launch of mitapivat in SCD, advance existing clinical programs, and opportunistically expand its pipeline through both internally and externally discovered assets . The company may also pursue opportunistic debt offerings, and equity or equity-linked offerings . Under the license agreement with Alnylam, the company may be required to pay up to an additional $120.0 million in potential development and regulatory milestones, in addition to sales milestones and tiered royalties on annual net sales, if any, of licensed products .

The company explicitly flagged several structural headwinds and execution risks. These include the possibility that PYRUKYND® or AQVESME™ may be less effective than previously believed or cause undesirable side effects not previously identified, which could compromise marketability . The USPI for AQVESME™ contains a boxed warning regarding the potential for hepatocellular injury, and it is available only through a REMS program . Failure to comply with REMS requirements could result in regulatory action or decreased sales . The company also faces substantial competition from major pharmaceutical and biotechnology companies, academic institutions, and governmental agencies . Competitors may develop more effective, safer, more convenient, or less costly products, or obtain regulatory approval more rapidly . The company is singularly focused on rare diseases, making it more susceptible to changing market conditions specific to these markets . Furthermore, the company relies on third parties for manufacturing and supply, and any performance failure could delay development or commercialization . Geopolitical events, such as war, could negatively impact the ability to initiate, enroll, and retain patients in clinical trials . Changes in U.S. trade policy, including tariffs, could negatively impact costs of materials, production processes, and supply chain .

Risk Factors

The company faces material risks including the potential for PYRUKYND® or AQVESME™ to be less effective or cause undesirable side effects not previously identified, which could compromise marketability, as evidenced by the boxed warning for hepatocellular injury in AQVESME™'s USPI and the requirement for a REMS program . The company is singularly focused on rare diseases, making it more susceptible to market fluctuations specific to these patient populations . Competition is substantial, with numerous large pharmaceutical and biotechnology companies developing products for the same indications, potentially leading to more effective, safer, or cheaper alternatives, or faster regulatory approvals . Geopolitical events and trade policy changes, such as U.S. tariffs on imports, could disrupt supply chains, increase costs, and delay product development or commercialization . Non-compliance with U.S. and foreign export control, import, sanctions, anti-corruption, and anti-money laundering laws could lead to criminal and/or civil liability, including significant fines and penalties . The company's ability to use its net operating loss carryforwards and other tax attributes may be limited by ownership changes or regulatory changes .

Management Priorities

Management's message to shareholders emphasizes building a sustainable, value-creating company grounded in expertise in cellular metabolism and classical hematology, with a focus on developing and delivering innovative medicines for patients. They believe their proven PK activator franchise has the potential to become the standard of care across multiple hemolytic anemias, supported by approved indications in PK deficiency and thalassemia. Management also sees significant opportunity to unlock further value through a robust early- and mid-stage pipeline and believes the company has a clear path to profitability supported by its existing commercial portfolio. The company's strategic priorities include the successful commercialization of PYRUKYND® and AQVESME™ for approved indications, advancing existing clinical programs for mitapivat, tebapivat, AG-181, and AG-236, and opportunistically expanding the pipeline through both internally and externally discovered assets. Management expects to have a pre-sNDA meeting with the FDA in the first quarter of 2026 and intends to submit a U.S. marketing application for mitapivat in sickle cell disease following that engagement . The European Commission's final decision on the new indication for PYRUKYND® in thalassemia is expected in early 2026 .

View Source Annual Report on SEC.gov ↗

References

  1. [1] Item 7, MD&A — Financial Operations Overview
  2. [2] Item 7, MD&A — Product Revenue
  3. [3] Item 1, Business — Business Overview
  4. [4] Item 1, Business — Business Overview
  5. [5] Item 1, Business — Business Overview
  6. [6] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  7. [7] Item 1, Business — Business Overview
  8. [8] Item 1, Business — Business Overview
  9. [9] Item 1, Business — Business Overview
  10. [10] Item 7, MD&A — Revenues
  11. [11] Item 7, MD&A — Revenues
  12. [12] Item 7, MD&A — Revenues
  13. [13] Item 7, MD&A — Total Operating Expenses
  14. [14] Item 7, MD&A — Total Operating Expenses
  15. [15] Item 7, MD&A — Total Operating Expenses
  16. [16] Item 7, MD&A — Net (Loss) Income
  17. [17] Item 7, MD&A — Net (Loss) Income
  18. [18] Item 7, MD&A — Sources of Liquidity
  19. [19] Item 7, MD&A — Sources of Liquidity
  20. [20] Item 7, MD&A — Sources of Liquidity
  21. [21] Item 7, MD&A — Sources of Liquidity
  22. [22] Item 7, MD&A — Financial Operations Overview
  23. [23] Item 7, MD&A — Total Revenue – 2025 vs. 2024
  24. [24] Item 7, MD&A — Total Revenue – 2025 vs. 2024
  25. [25] Item 7, MD&A — Total Revenue – 2025 vs. 2024
  26. [26] Item 7, MD&A — Total Operating Expenses – 2025 vs. 2024
  27. [27] Item 7, MD&A — Total Operating Expenses – 2025 vs. 2024
  28. [28] Item 7, MD&A — Total Operating Expenses – 2025 vs. 2024
  29. [29] Item 7, MD&A — Net (Loss) Income
  30. [30] Item 7, MD&A — Net (Loss) Income
  31. [31] Item 7, MD&A — Other Income and Expense – 2025 vs. 2024
  32. [32] Item 7, MD&A — Other Income and Expense – 2025 vs. 2024
  33. [33] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  34. [34] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  35. [35] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  36. [36] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  37. [37] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  38. [38] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  39. [39] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  40. [40] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  41. [41] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  42. [42] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  43. [43] Item 1, Business — Alnylam License Agreement
  44. [44] Item 1, Business — Alnylam License Agreement
  45. [45] Item 1, Business — Other Programs
  46. [46] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  47. [47] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  48. [48] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  49. [49] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  50. [50] Item 1, Business — Other Programs
  51. [51] Item 1, Business — Other Programs
  52. [52] Item 1, Business — Other Programs
  53. [53] Item 1, Business — Other Programs
  54. [54] Item 7, MD&A — Financial Operations Overview
  55. [55] Item 7, MD&A — Research and Development Expenses
  56. [56] Item 7, MD&A — Selling, General and Administrative Expenses
  57. [57] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  58. [58] Item 1, Business — PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
  59. [59] Item 7, MD&A — Funding Requirements
  60. [60] Item 7, MD&A — Funding Requirements
  61. [61] Item 7, MD&A — Contractual Obligations
  62. [62] Item 7, MD&A — Contractual Obligations
  63. [63] Item 1A, Risk Factors — Risks Related to the Discovery, Development, and Commercialization of our Products and Product Candidates
  64. [64] Item 1A, Risk Factors — Risks Related to the Discovery, Development, and Commercialization of our Products and Product Candidates
  65. [65] Item 1A, Risk Factors — Risks Related to the Discovery, Development, and Commercialization of our Products and Product Candidates
  66. [66] Item 1A, Risk Factors — Risks Related to the Discovery, Development, and Commercialization of our Products and Product Candidates
  67. [67] Item 1A, Risk Factors — Risks Related to the Discovery, Development, and Commercialization of our Products and Product Candidates
  68. [68] Item 1A, Risk Factors — Risks Related to Our Financial Position
  69. [69] Item 1A, Risk Factors — Risks Related to Our Dependence on Third Parties
  70. [70] Item 1A, Risk Factors — Risks Related to the Discovery, Development, and Commercialization of our Products and Product Candidates
  71. [71] Item 1A, Risk Factors — Risks Related to Regulatory Approval of Our Product Candidates and Other Legal Compliance Matters
  72. [72] Item 1A, Risk Factors — PYRUKYND®, AQVESME™ or any of our product candidates that may receive marketing approval in the future, may fail to achieve the degree of market acceptance by physicians, patients, healthcare payors and others in the medical community necessary for commercial success.
  73. [73] Item 1A, Risk Factors — We face challenges as a less diversified company.
  74. [74] Item 1A, Risk Factors — We face substantial competition, which may result in others discovering, developing or commercializing products before or more successfully than we do.
  75. [75] Item 1A, Risk Factors — Changes in and uncertainty surrounding U.S. trade policy could have a material adverse impact on our business, financial condition and results of operations.
  76. [76] Item 1A, Risk Factors — We are subject to U.S. and foreign export control, import, sanctions, anti-corruption and anti-money laundering laws with respect to our operations, and non-compliance with such laws can subject us to criminal and/or civil liability and harm our business.
  77. [77] Item 1A, Risk Factors — Our ability to use our net operating loss carryforwards and certain other tax attributes may be limited.
  78. [78] Item 7, MD&A — Overview
  79. [79] Item 7, MD&A — Overview
  80. [80] Item 7, MD&A — Overview

Analysis on 5/22/2026