Loading organizations...
§ Private Profile · 88 Sidney Street, Cambridge, MA 02139, USA
Biopharmaceutical company develops small-molecule medicines targeting cellular metabolism for rare genetic and blood disorders.
Agios Pharmaceuticals is a Cambridge, Massachusetts-based biopharmaceutical company that discovers and develops small-molecule medicines targeting cellular metabolism for rare genetic and blood disorders. Operating as a publicly traded entity with a market capitalization exceeding $1 billion, the firm generates revenue through commercial drug sales, milestone payments, and royalties. Historically, the enterprise collaborated with industry partners like Celgene to advance its precision oncology medicines. In 2021, the company completed the strategic sale of its commercial, clinical, and research-stage oncology portfolio to Servier for $1.8 billion in upfront cash to pivot entirely toward rare diseases. Under the leadership of current CEO Brian Goff and Board Chair Jackie Fouse, the organization secured FDA approval for its disease-modifying therapy PYRUKYND in 2022. Agios Pharmaceuticals was founded in 2008 by scientists Lewis Cantley, Tak Mak, and Craig Thompson.
Agios Pharmaceuticals has raised $120.0M across 3 funding rounds.
Agios Pharmaceuticals has raised $120.0M in total across 3 funding rounds.
Agios Pharmaceuticals has raised $120.0M across 3 funding rounds. Most recently, it raised $78.0M Series C in November 2011.
| Date | Round | Lead Investors | Other Investors | Status |
|---|---|---|---|---|
| Nov 1, 2011 | $78M Series C | — | ARCH Venture Partners, Flagship Ventures, Samsara BioCapital, Perry Karsen, Flagship Ventures, Third Rock Ventures | Announced |
| Apr 1, 2010 | $9M Series B | — | ARCH Venture Partners, Flagship Ventures, Samsara BioCapital | Announced |
| Jul 1, 2008 | $33M Series A | — | ARCH Venture Partners, Flagship Ventures | Announced |
Agios Pharmaceuticals has raised $120.0M in total across 3 funding rounds.
Agios Pharmaceuticals's investors include ARCH Venture Partners, Flagship Ventures, Samsara BioCapital, Perry Karsen, Third Rock Ventures.
Agios Pharmaceuticals is a publicly traded biopharmaceutical company (NASDAQ: AGIO) focused on developing therapies targeting cellular metabolism for genetically defined diseases, particularly rare hemolytic anemias like pyruvate kinase (PK) deficiency, sickle cell disease, and thalassemia.[1][2][4] It builds precision medicines such as the approved PYRUKYND (mitapivat), a first-in-class PK activator that modifies disease progression in PK deficiency by addressing the underlying metabolic defect, serving patients with rare anemias who lack effective long-term treatments.[1][2][4] The company solves unmet needs in oncology and rare diseases by pioneering IDH inhibition (leading to approved cancer drugs IDHIFA and TIBSOVO) and PK activation, with a current pipeline emphasizing disease-modifying therapies for hemolytic anemias to improve patient quality of life beyond symptom relief.[1][2][4] Growth momentum includes FDA approvals for mitapivat in 2022, ongoing pivotal trials, and partnerships like the 2016 Celgene deal, alongside public offerings raising hundreds of millions.[1][2]
Agios was founded in 2007 or 2008 in Cambridge, Massachusetts, by scientists Lewis Cantley, Tak Mak, and Craig Thompson, emerging from Flagship Pioneering to pioneer cellular metabolism as a new therapeutic field.[1][2][3] The idea stemmed from a "blank sheet of paper" approach to metabolism, with early breakthroughs like the 2009 Nature publication linking mutated IDH1 gene to cancer, establishing IDH inhibition science.[1][2] David Schenkein, M.D., became CEO in 2009, guiding initial oncology focus; pivotal moments included the 2013 IPO, first FDA approval for IDHIFA in 2017, TIBSOVO in 2018, and mitapivat's 2022 approval as the first disease-modifying therapy for PK deficiency.[1][2] Early traction built through partnerships (e.g., Celgene in 2016 for $120M potential) and stock raises ($250M in 2017, $256M in 2019), despite a 2012 lawsuit over research origins involving founder Thompson.[2] Brian Goff serves as CEO as of 2022.[2]
Agios rides the trend of precision medicine in genetically defined diseases, leveraging cellular metabolism—a novel field it helped unlock—to address unmet needs in oncology and rare anemias amid rising demand for targeted, disease-modifying biologics.[1][3][4] Timing aligns with advances in genomic sequencing and orphan drug incentives, amplified by FDA priority reviews and approvals (e.g., mitapivat in 2022), favoring small biotechs like Agios over traditional pharma.[1][2] Market forces include growing rare disease funding and partnerships with giants like Celgene, enabling Agios to influence the ecosystem by validating metabolism targets, inspiring similar ventures, and expanding access via support programs.[1][2][4]
Agios is poised to expand its hemolytic anemia portfolio with mitapivat data from ongoing trials in sickle cell and thalassemia, potentially securing more approvals and building a multi-product franchise in rare diseases.[1][4] Trends like AI-driven metabolism research and gene therapy synergies will shape its path, enhancing pipeline efficiency and market reach.[1][3] Its influence may evolve from oncology pioneer to rare disease leader, driving long-term value through patient collaborations and strategic deals, reinforcing its role in transforming genetically defined unmet needs.[1][2][4] This positions Agios as a key player in biopharma's metabolism revolution, starting from that blank-sheet oncology foray.