Agios Pharmaceuticals
Biopharmaceutical company focused on cellular metabolism to develop small-molecule and modality-diverse therapies for rare diseases, with a near-term emphasis on hemolytic anemias. Activities include discovery and preclinical R&D, clinical development through late-stage trials, regulatory submissions and approved therapies, patient support and global access programs, and partnerships to advance translational science.
Industries
Nr. of Employees
large (251-1000)
Agios Pharmaceuticals
Patents
Compounds and methods useful for stabilizing phenylalanine hydroxylase mutations
US-12673961-B2
View DetailsMethods of treatment with aminolevulinic acid synthase 2 (ALAS2) modulators
US-12667087-B2
View DetailsCrystalline forms of n-(4-(4-(cyclopropylmethyl)piperazine-1-carbonyl)phenyl)quinoline-8-sulfonamide
US-12612381-B2
View Details
Compounds and methods useful for stabilizing phenylalanine hydroxylase mutations
US-12673961-B2
View DetailsMethods of treatment with aminolevulinic acid synthase 2 (ALAS2) modulators
US-12667087-B2
View DetailsCrystalline forms of n-(4-(4-(cyclopropylmethyl)piperazine-1-carbonyl)phenyl)quinoline-8-sulfonamide
US-12612381-B2
View DetailsProducts
Mitapivat (oral pyruvate kinase activator)
Orally administered small-molecule activator of pyruvate kinase developed to improve red blood cell energy metabolism and reduce hemolysis in pyruvate kinase deficiency; being evaluated for other hemolytic anemias.
Pyrukynd
Film-coated tablets containing 20 mg of mitapivat (as sulfate), indicated for the treatment of pyruvate kinase deficiency in adult patients.
Mitapivat
An oral, allosteric activator of pyruvate kinase (PK) approved for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.
AG-236
siRNA TMPRSS6 candidate for polycythemia vera.
AG-181
PAH stabilizer for phenylketonuria.
AQVESME
AQVESME (mitapivat) is an approved medicine developed by Agios for targeted rare disease indications.
Mitapivat (oral pyruvate kinase activator)
Orally administered small-molecule activator of pyruvate kinase developed to improve red blood cell energy metabolism and reduce hemolysis in pyruvate kinase deficiency; being evaluated for other hemolytic anemias.
Pyrukynd
Film-coated tablets containing 20 mg of mitapivat (as sulfate), indicated for the treatment of pyruvate kinase deficiency in adult patients.
Mitapivat
An oral, allosteric activator of pyruvate kinase (PK) approved for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.
AG-236
siRNA TMPRSS6 candidate for polycythemia vera.
AG-181
PAH stabilizer for phenylketonuria.
AQVESME
AQVESME (mitapivat) is an approved medicine developed by Agios for targeted rare disease indications.
Services
Centralized support offering disease education, access support, prescription fulfillment and financial assistance for eligible patients (U.S. residents only).
Pathway for healthcare providers to request access to approved medicines for patients in regions where commercial availability is limited.
Support for independent investigator-initiated research aligned with clinical and nonclinical areas of interest.
Grant support for independent educational activities to improve awareness and understanding in the medical community.
Review and release process for clinical trial data from completed, company‑sponsored interventional trials, subject to qualification and agreement terms.
Centralized support offering disease education, access support, prescription fulfillment and financial assistance for eligible patients (U.S. residents only).
Pathway for healthcare providers to request access to approved medicines for patients in regions where commercial availability is limited.
Support for independent investigator-initiated research aligned with clinical and nonclinical areas of interest.
Grant support for independent educational activities to improve awareness and understanding in the medical community.
Review and release process for clinical trial data from completed, company‑sponsored interventional trials, subject to qualification and agreement terms.
Expertise Areas
- Cellular metabolism research
- Rare disease therapeutics
- Hemolytic anemias (pyruvate kinase deficiency, thalassemia, sickle cell disease)
- Small-molecule drug discovery
Key Technologies
- Pyruvate kinase activation (mechanism-based therapeutics)
- Small-molecule therapeutics
- siRNA therapeutics
- PAH stabilizer modalities
News & Updates
Quarterly financial results and corporate business update.
Clinical update regarding a Phase 2 study in sickle cell disease.
Regulatory update: priority review granted for supplemental NDA in sickle cell disease.
Regulatory approval announcement for mitapivat in the EU for adults with thalassemia.
Peer-reviewed publication reporting Phase 2 study results for mitapivat in pyruvate kinase deficiency.
Quarterly financial results and corporate business update.
Clinical update regarding a Phase 2 study in sickle cell disease.
Regulatory update: priority review granted for supplemental NDA in sickle cell disease.
Regulatory approval announcement for mitapivat in the EU for adults with thalassemia.
Peer-reviewed publication reporting Phase 2 study results for mitapivat in pyruvate kinase deficiency.