Agios Pharmaceuticals
Agios Pharmaceuticals is a biopharmaceutical company developing oral pyruvate kinase activators and related therapies for rare hemolytic anemias, including pyruvate kinase deficiency, thalassemia, and sickle cell disease. It commercializes PYRUKYND and AQVESME and pursues pipeline expansion via licensing and partnerships.
- Company typePublic
- Founded2008
- HeadquartersCambridge, United States
- Headcount501–1,000
- GTM typeB2B
- OfferingHardware or Manufacturing
What Agios Pharmaceuticals does
Agios Pharmaceuticals is a publicly traded biopharmaceutical company (NASDAQ: AGIO) headquartered in Cambridge, Massachusetts, that develops oral therapies for rare hemolytic anemias and related rare diseases. After selling its oncology business to Servier for up to $2 billion in December 2020, the company pivoted to focus exclusively on cellular metabolism and rare blood disorders. Its commercial products — PYRUKYND and AQVESME, both formulations of the oral pyruvate kinase (PK) activator mitapivat — treat adults with pyruvate kinase deficiency (FDA-approved February 2022) and alpha- or beta-thalassemia (FDA-approved December 2025), respectively. The company's core scientific platform is built around PK activation, the mechanism by which mitapivat increases red blood cell ATP production, reduces 2,3-DPG levels, and improves red blood cell energy metabolism and lifespan in patients with hemolytic anemia.
The company's pipeline extends its cellular metabolism platform into adjacent indications, including tebapivat (a next-generation oral PK activator in Phase 2 for sickle cell disease and lower-risk MDS), cevidoplenib (an oral SYK inhibitor licensed from Oscotec in June 2026 for immune thrombocytopenia, with Phase 3 trials planned for H1 2028), AG-236 (an siRNA therapeutic targeting TMPRSS6 for polycythemia vera), and AG-181 (a phenylalanine hydroxylase stabilizer for phenylketonuria). Agios has also filed an sNDA seeking accelerated FDA approval for mitapivat in sickle cell disease, based on Phase 3 RISE UP data showing a 40.6% hemoglobin response rate versus 2.9% on placebo.
Agios generates revenue through direct U.S. product sales via specialty pharmacies and REMS-certified prescribers, supplemented by international royalty and milestone revenue from exclusive commercialization partners (Avanzanite Bioscience for 32 European countries and the UK/Switzerland; NewBridge Pharmaceuticals for the GCC region). AQVESME is priced at $425,000 per patient per year in the United States, with the U.S. thalassemia launch population estimated at approximately 4,000 patients and a global peak-year sales potential of $1 billion across multiple indications. Q1 2026 worldwide net product revenue was $20.7 million (up 138% year-over-year from $8.7 million), with 242 prescriptions written for AQVESME by quarter-end. The company ended Q1 2026 with $1.0 billion in cash and marketable securities against an annual operating cash burn of approximately $385 million.
Agios Pharmaceuticals firmographics
Firmographics- Name
- Agios Pharmaceuticals
- Legal name
- Agios Pharmaceuticals, Inc.
- Website
- https://agios.com
- Company type
- Public
- Founded year
- 2008
- Operating status
- Operating
- Headcount range
- 501–1,000 employees
- Short description
- Agios Pharmaceuticals is a biopharmaceutical company developing oral pyruvate kinase activators and related therapies for rare hemolytic anemias, including pyruvate kinase deficiency, thalassemia, and sickle cell disease. It commercializes PYRUKYND and AQVESME and pursues pipeline expansion via licensing and partnerships.
- Ownership category
- akta.pro rank
Agios Pharmaceuticals industry classification
Industry- Product category
- Rare Disease Pharmaceuticals
- NAICS
- Pharmaceutical and Medicine Manufacturing (3254), Medicinal and Botanical Manufacturing (325411)
- SIC
- Medicinal Chemicals & Botanical Products (2833)
- akta.pro primary industry
- Hematology Specialty Pharmaceuticals (Non-malignant) (HLAIACAI)
Keywords
Where Agios Pharmaceuticals is headquartered
LocationHeadquarters
- HQ city
- Cambridge
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
Agios Pharmaceuticals business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations, Marketing or Sales, Others
Revenue model
- Product Sales (PYRUKYND/AQVESME): Agios generates revenue from direct product sales of mitapivat under two brand names: PYRUKYND for pyruvate kinase deficiency (approved in US 2022, EU 2022, UAE, KSA) and AQVESME for thalassemia (approved in US December 2025, EU May 2022). Revenue is generated through prescription sales via specialty pharmacies and healthcare providers, with 242 prescriptions written by Q1 2026.
- Licensing and Partnership Revenue: Agios receives milestone payments and royalties from commercial partners including Avanzanite Bioscience (Europe) and NewBridge Pharmaceuticals (GCC), who hold exclusive rights to commercialize mitapivat in their respective territories under revenue-sharing or royalty arrangements.
Pricing tiers
| Model | Billing | Price |
|---|---|---|
| Subscription | Annual | AQVESME (mitapivat for thalassemia) - Annual wholesale pricing |
Go-to-market motion3 records
Distribution channels4 records
Marketing channels7 records
Agios Pharmaceuticals product offering
Product offeringCore offering
Agios Pharmaceuticals develops and commercializes oral small-molecule therapeutics for rare hemolytic anemias, led by its first-in-class pyruvate kinase (PK) activator mitapivat marketed as PYRUKYND for pyruvate kinase deficiency and AQVESME for alpha- and beta-thalassemia. The company supplements these approved products with a pipeline of next-generation PK activators (tebapivat), a SYK inhibitor (cevidoplenib), an siRNA candidate (AG-236), and a PAH stabilizer (AG-181), supported by patient access programs such as MyAgios and the Global Managed Access Program.
Product overview
Agios Pharmaceuticals is a biopharmaceutical company developing a portfolio of therapies for rare diseases, particularly hemolytic anemias. The company's core approved products include PYRUKYND (mitapivat) for pyruvate kinase deficiency and AQVESME (mitapivat) for thalassemia, both oral pyruvate kinase activators that improve red blood cell energy metabolism. The pipeline includes next-generation candidates: Tebapivat (next-generation PK activator) for sickle cell disease and MDS, Cevidoplenib (SYK inhibitor) for immune thrombocytopenia, AG-236 for polycythemia vera, and AG-181 for phenylketonuria. Patient support services include the MyAgios program and Global Managed Access Program. The company leverages expertise in cellular metabolism to advance these rare disease therapies.
Differentiator
Problem solved
Functional benefit
Products and services
- PYRUKYND (mitapivat) First-in-class oral pyruvate kinase (PK) activator approved for treating adults with pyruvate kinase deficiency. Increases ATP production and decreases 2,3-DPG in red blood cells to improve red blood cell energy metabolism and lifespan. Distributed in the U.S. through REMS-certified physicians and in Europe via Avanzanite Bioscience.
- AQVESME (mitapivat) Oral pyruvate kinase activator approved by the FDA in December 2025 for treating anemia in adults with alpha- or beta-thalassemia; the only FDA-approved treatment for both non-transfusion-dependent and transfusion-dependent thalassemia. U.S. annual wholesale pricing is $425,000.
- Tebapivat (AG-946) Next-generation oral pyruvate kinase activator in Phase 2 development for sickle cell disease and lower-risk myelodysplastic syndromes (MDS); designed as an improved follow-on to mitapivat.
- Cevidoplenib Next-generation oral SYK inhibitor licensed from Oscotec for treating immune thrombocytopenia (ITP); Agios plans Phase 3 development in H1 2028 with peak US sales potential estimated at up to $1 billion.
- MyAgios Patient Support Program Comprehensive patient support services program providing access support, prescription fulfillment, financial assistance, disease education, and community connections for eligible patients on Agios medicines, with dedicated Patient Support Managers.
- Global Managed Access Program (GMAP) Program providing a pathway for patients to obtain access to PYRUKYND in countries where the medicine is not yet widely available, evaluating requests in consultation with qualified treating healthcare providers.
Quantifiable outcome
- 138% year-over-year revenue growth in Q1 2026 ($20.7M vs $8.7M Q1 2025)
- +6 more outcomes
Companies that use Agios Pharmaceuticals
Customer profileNamed customers5 records
Segments2 records
Ideal customer profiles2 records
Agios Pharmaceuticals technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature5 records
Agios Pharmaceuticals partnerships and signals
Strategic signalPartnerships
Six partnerships are on record, tiered minor and core.
- Cayenne Wellness CenterminorCo-hosting partnership for the 7th Annual 24-Hour World Sickle Cell Day Marathon on June 19, 2026, themed 'Closing the Survival Gap: Equity in Sickle Cell Care.' Agios serves as Advocate Sponsor alongside Community Sponsor Fulcrum Therapeutics. The virtual event brings together patients, caregivers, advocates, healthcare professionals, researchers, and policymakers to address disparities in diagnosis, treatment, and mental health services for sickle cell disease.
- OscoteccoreExclusive global license agreement with South Korea-based Oscotec to develop and commercialize cevidoplenib, a next-generation oral SYK inhibitor for treating immune thrombocytopenia (ITP). Agios paid $25 million upfront with potential additional payments of up to $140 million in development and regulatory milestones plus tiered royalties on future net sales. Agios assumes full responsibility for future development and commercialization costs, with plans to advance cevidoplenib into Phase 3 development for ITP in H1 2028. Peak US sales potential estimated at up to $1 billion.
- Avanzanite Bioscience B.V.coreExclusive partnership agreement with Amsterdam-based Avanzanite Bioscience B.V. for commercialization and distribution of PYRUKYND (mitapivat) across the European Economic Area, United Kingdom, and Switzerland. Avanzanite commercialized the drug following EU approval for thalassemia in May 2026. This is Avanzanite's fourth rare disease launch in Europe. The partnership was originally signed in June 2025.
- NewBridge PharmaceuticalscorePartnership with NewBridge Pharmaceuticals for commercialization of PYRUKYND in the Gulf Cooperation Council (GCC) region, covering Saudi Arabia and other GCC countries. The partnership was established to expand access to mitapivat in the Middle East market, pending regulatory approvals in those territories.
- Global Alliance of Sickle Cell Disease Organizations (GASCDO)minorCo-hosting partnership with GASCDO for the 7th Annual 24-Hour World Sickle Cell Day Marathon, a free virtual event streamed live for 24 hours to address disparities in sickle cell disease care globally.
- Thalassaemia International Federation (TIF)coreLong-standing partnership with Thalassaemia International Federation to support TIF's coalition building efforts and patient advocacy. Agios has assisted TIF in establishing the first patient advocacy group for all hemolytic anemias. The partnership includes both hands-on collaboration and financial donations to advance TIF's mission of ensuring equal access to quality healthcare for every patient with thalassemia and other hemoglobin disorders worldwide.
Scale indicators12 records
Recent moves9 records
Expansion highlights7 records
Agios Pharmaceuticals competitors and assessment
Company assessmentDirect peers
- BioMarin Pharmaceutical: BioMarin is a direct peer as a rare disease-focused biopharmaceutical company with a portfolio of high-priced specialty enzyme replacement and gene therapies for ultra-rare conditions. Both companies operate with similar commercial models targeting small patient populations through specialty distribution, with comparable revenue scale and operating profiles.
- Alnylam Pharmaceuticals: Alnylam is a direct peer developing RNA interference (RNAi) therapeutics for rare diseases, with commercial products targeting conditions like hereditary ATTR amyloidosis. Both Agios and Alnylam operate as platform-based rare disease companies with premium-priced specialty therapies requiring specialized prescriber engagement.
- bluebird bio: bluebird bio is a direct peer developing gene therapies for sickle cell disease (Lyfgenia) and beta-thalassemia, competing in the same hematology rare disease space. Both companies pursue disease-modifying therapies for inherited blood disorders through specialty channels with comparable patient identification and access challenges.
- Vertex Pharmaceuticals: Vertex Pharmaceuticals is a direct peer focused on serious diseases with multiple approved therapies including Casgevy (exa-cel) gene therapy for sickle cell disease and beta-thalassemia. Both companies pursue rare hematology indications with high-cost specialty therapies and rely on specialized prescriber networks, making them highly comparable on business model and target market.
- Alexion (AstraZeneca Rare Disease): Alexion, acquired by AstraZeneca, pioneered rare disease commercialization with Soliris/Ultomiris for PNH and other complement-mediated conditions, including hematology indications. Agios CEO Brian Goff was recruited from Alexion, and the companies share highly comparable commercial models for ultra-rare hematology and specialty distribution.
Broad incumbents
- Global Blood Therapeutics (Pfizer): Global Blood Therapeutics, acquired by Pfizer, developed Oxbryta for sickle cell disease before its voluntary withdrawal. Now operating within Pfizer, it represents a broad incumbent positioning in the sickle cell disease space where Agios' mitapivat is seeking accelerated approval.
- Bristol Myers Squibb: Bristol Myers Squibb is a broad incumbent in hematology with Reblozyl (luspatercept) approved for beta-thalassemia and MDS-related anemia, directly competing with Agios in the thalassemia market. As a large pharmaceutical company, BMS offers a wider portfolio but overlaps with Agios in the same hematology specialty channel and prescriber base.
- Swedish Orphan Biovitrum (Sobi): Sobi is a broad incumbent rare disease company with a strong hematology portfolio including treatments for hemophilia, ITP, and other rare blood disorders. Sobi's established European rare disease commercial infrastructure and overlap in ITP (a key indication for Agios' cevidoplenib) makes it a comparable peer in the rare hematology space.
Emerging players
- Disc Medicine: Disc Medicine is an emerging clinical-stage hematology company developing therapies for serious hematologic diseases including polycythemia vera and sickle cell disease. Both companies target rare blood disorders with novel mechanisms and operate in overlapping prescriber networks, though Disc Medicine is at an earlier commercial stage than Agios.
- Fulcrum Therapeutics: Fulcrum Therapeutics is an emerging player developing therapies for sickle cell disease and other genetically defined diseases, with pociredil in late-stage development. Agios and Fulcrum co-sponsor the World Sickle Cell Day Marathon, indicating overlap in patient community engagement and competitive positioning in sickle cell disease.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat5 records
Key risks1 record
Key highlights7 records
Customer concentration
Agios Pharmaceuticals social profiles
Digital presenceAgios Pharmaceuticals compliance and trust
Trust signalCompliance2 records
Agios Pharmaceuticals financial estimates
Financial estimateRevenue estimate
Valuation estimate
Agios Pharmaceuticals leadership team
Management profileNumber of profiles
Profiles18 records
Agios Pharmaceuticals funding detail
Funding detailFunding overview
Funding rounds7 records
Investors4 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Agios Pharmaceuticals M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Agios Pharmaceuticals
What does Agios Pharmaceuticals do?
Agios Pharmaceuticals develops and commercializes oral small-molecule therapeutics for rare hemolytic anemias, led by its first-in-class pyruvate kinase (PK) activator mitapivat marketed as PYRUKYND for pyruvate kinase deficiency and AQVESME for alpha- and beta-thalassemia. The company supplements these approved products with a pipeline of next-generation PK activators (tebapivat), a SYK inhibitor (cevidoplenib), an siRNA candidate (AG-236), and a PAH stabilizer (AG-181), supported by patient access programs such as MyAgios and the Global Managed Access Program.
Is Agios Pharmaceuticals a public or private company?
Agios Pharmaceuticals is a public company. It is classified as public and is currently operating.
When was Agios Pharmaceuticals founded?
Agios Pharmaceuticals was founded in 2008. It employs 501 to 1,000 people.
Where is Agios Pharmaceuticals based?
Agios Pharmaceuticals is headquartered in Cambridge, United States, in the North America region.
How does Agios Pharmaceuticals make money?
Two revenue lines are on record. Product Sales (PYRUKYND/AQVESME) is the primary driver. The others are licensing and Partnership Revenue.
Who are Agios Pharmaceuticals's main competitors?
Direct peers on record are BioMarin Pharmaceutical, Alnylam Pharmaceuticals, bluebird bio, Vertex Pharmaceuticals and Alexion (AstraZeneca Rare Disease). Broad incumbents are Global Blood Therapeutics (Pfizer), Bristol Myers Squibb and Swedish Orphan Biovitrum (Sobi). Emerging players are Disc Medicine and Fulcrum Therapeutics.
Does Agios Pharmaceuticals have an API?
No public API is recorded for Agios Pharmaceuticals.
What industry is Agios Pharmaceuticals in?
Agios Pharmaceuticals's product category is Rare Disease Pharmaceuticals. Its primary akta.pro industry code is HLAIACAI, Hematology Specialty Pharmaceuticals (Non-malignant). Its NAICS code is 3254 and its SIC code is 2833.