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    Company profile for Agios Pharmaceuticals

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    ChemWerth works in generic API development & supply, non-infringement patent strategy development and regulatory support.

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    Cohance Lifesciences, offers full range of CDMO services for small molecule APIs, intermediates, ADCs, Pellets and Formulations.

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    LEBSA- Your strategic API source for niche molecules.

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    Mission CDMO team brings reliability, resourcefulness, & responsiveness in executing your development project.

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    About

    At Agios, our culture and values – which form the foundation of our company and differentiate us – are embodied in the Greek word Arete, the ancient ideal of excellence and fulfillment of one’s potential. Arete has several meanings, and each is applicable to our company. It is often used to describe virtue, to characterize someone as a “good person.” Our company comprises good people who are striving to achieve a vir...
    At Agios, our culture and values – which form the foundation of our company and differentiate us – are embodied in the Greek word Arete, the ancient ideal of excellence and fulfillment of one’s potential. Arete has several meanings, and each is applicable to our company. It is often used to describe virtue, to characterize someone as a “good person.” Our company comprises good people who are striving to achieve a virtuous goal. It also connotes a sense of driving for excellence, which speaks to how far we can push ourselves and reflects our focus on achieving “the other side of possible.

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    CONTACT DETAILS

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    Agios PharmaceuticalsUnited_States_of_America Flag
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    Country
    U.S.A
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    88 Sidney Street Cambridge, MA 02139-4169
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    617-649-8600
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    enquiries@pharmacompass.com
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    https://www.agios.com/
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    https://x.com/agiospharma

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    NEWS #PharmaBuzz

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    Agios Details 2026 Strategy To Boost Rare Disease Portfolio
    Agios Details 2026 Strategy To Boost Rare Disease Portfolio

    12 Jan 2026

    // GLOBENEWSWIRE

    https://www.globenewswire.com/news-release/2026/01/12/3216709/31990/en/Agios-Outlines-2026-Strategic-Priorities-and-Key-Milestones-to-Accelerate-Rare-Disease-Portfolio-Growth.html

    GLOBENEWSWIRE
    12 Jan 2026
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    Agios To Present At J.P. Morgan Healthcare Conference
    Agios To Present At J.P. Morgan Healthcare Conference

    05 Jan 2026

    // GLOBENEWSWIRE

    https://www.globenewswire.com/news-release/2026/01/05/3212595/31990/en/Agios-to-Present-at-the-44th-Annual-J-P-Morgan-Healthcare-Conference-on-January-14-2026.html

    GLOBENEWSWIRE
    05 Jan 2026
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    Agios shares jump as FDA expands approval for Blood Disorder
    Agios shares jump as FDA expands approval for Blood Disorder

    27 Dec 2025

    // REUTERS

    https://www.reuters.com/sustainability/boards-policy-regulation/agios-pharma-shares-jump-us-fda-expands-approval-its-blood-disorder-drug-2025-12-24/

    REUTERS
    27 Dec 2025
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    FDA Approves Agios' AQVESME For Anemia In Adult Thalassemia
    FDA Approves Agios' AQVESME For Anemia In Adult Thalassemia

    23 Dec 2025

    // GLOBENEWSWIRE

    https://www.globenewswire.com/news-release/2025/12/24/3210205/31990/en/U-S-FDA-Approves-Agios-AQVESME-mitapivat-for-the-Treatment-of-Anemia-in-Adults-with-Alpha-or-Beta-Thalassemia.html

    GLOBENEWSWIRE
    23 Dec 2025
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    Agios Provides Update on U.S. sNDA for Mitapivat in Thalassemia
    Agios Provides Update on U.S. sNDA for Mitapivat in Thalassemia

    08 Dec 2025

    // GLOBENEWSWIRE

    https://www.globenewswire.com/news-release/2025/12/08/3201402/31990/en/Agios-Provides-Update-on-U-S-sNDA-for-Mitapivat-in-Thalassemia.html

    GLOBENEWSWIRE
    08 Dec 2025
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    Agios’ Pyrukynd turns in mixed results in sickle cell ph. 3
    Agios’ Pyrukynd turns in mixed results in sickle cell ph. 3

    20 Nov 2025

    // GLOBENEWSWIRE

    https://www.globenewswire.com/news-release/2025/11/19/3190791/31990/en/Agios-Announces-Topline-Results-from-RISE-UP-Phase-3-Trial-of-Mitapivat-in-Sickle-Cell-Disease.html

    GLOBENEWSWIRE
    20 Nov 2025
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    Drugs in Development

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    Fda Approves Agios' AQVESME™ for Adult Thalassemia Anemia

    Details:

    Mitapivat, a miscellaneous product targeting pyruvate kinase, shows promising results in treating anemia.


    Lead Product(s): Mitapivat

    Therapeutic Area: Hematology Brand Name: Aqvesme

    Study Phase: Approved FDFProduct Type: Miscellaneous

    Sponsor: Undisclosed

    Deal Size: Inapplicable Upfront Cash: Inapplicable

    Deal Type: Inapplicable December 23, 2025

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    Lead Product(s) : Mitapivat

    Therapeutic Area : Hematology

    Highest Development Status : Approved FDF

    Partner/Sponsor/Collaborator : Undisclosed

    Deal Size : Inapplicable

    Deal Type : Inapplicable

    DEALS_DEV arrow-down

    Fda Approves Agios' AQVESME™ for Adult Thalassemia Anemia

    Details : Mitapivat, a miscellaneous product targeting pyruvate kinase, shows promising results in treating anemia.

    Product Name : Aqvesme

    Product Type : Miscellaneous

    Upfront Cash : Inapplicable

    December 23, 2025

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    A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Participants With Phenylketonuria

    Details:

    AG-181 is a small molecule drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of Phenylketonurias.


    Lead Product(s): AG-181

    Therapeutic Area: Genetic Disease Brand Name: Undisclosed

    Study Phase: Phase IProduct Type: Miscellaneous

    Sponsor: Undisclosed

    Deal Size: Inapplicable Upfront Cash: Inapplicable

    Deal Type: Inapplicable November 21, 2025

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    Lead Product(s) : AG-181

    Therapeutic Area : Genetic Disease

    Highest Development Status : Phase I

    Partner/Sponsor/Collaborator : Undisclosed

    Deal Size : Inapplicable

    Deal Type : Inapplicable

    DEALS_DEV arrow-down

    A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Part...

    Details : AG-181 is a small molecule drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of Phenylketonurias.

    Product Name : Undisclosed

    Product Type : Miscellaneous

    Upfront Cash : Inapplicable

    November 21, 2025

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    AG-236 Safety, Tolerability and ADME In Healthy Participants

    Details:

    AG-236 is a Oligonucleotide drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of unspecified medical condition.


    Lead Product(s): AG-236

    Therapeutic Area: Undisclosed Brand Name: Undisclosed

    Study Phase: Phase IProduct Type: Oligonucleotide

    Sponsor: Undisclosed

    Deal Size: Inapplicable Upfront Cash: Inapplicable

    Deal Type: Inapplicable July 20, 2025

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    Lead Product(s) : AG-236

    Therapeutic Area : Undisclosed

    Highest Development Status : Phase I

    Partner/Sponsor/Collaborator : Undisclosed

    Deal Size : Inapplicable

    Deal Type : Inapplicable

    DEALS_DEV arrow-down

    AG-236 Safety, Tolerability and ADME In Healthy Participants

    Details : AG-236 is a Oligonucleotide drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of unspecified medical condition.

    Product Name : Undisclosed

    Product Type : Oligonucleotide

    Upfront Cash : Inapplicable

    July 20, 2025

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    Mitapivat Safety and Proof of Concept In RBC Membranopathies/CDAII

    Details:

    Mitapivat is a Other Small Molecule drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Anemia.


    Lead Product(s): Mitapivat

    Therapeutic Area: Hematology Brand Name: Undisclosed

    Study Phase: Phase IIProduct Type: Miscellaneous

    Recipient: University Health Network, Toronto

    Deal Size: Inapplicable Upfront Cash: Inapplicable

    Deal Type: Inapplicable July 08, 2025

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    Lead Product(s) : Mitapivat

    Therapeutic Area : Hematology

    Highest Development Status : Phase II

    Recipient : University Health Network, Toronto

    Deal Size : Inapplicable

    Deal Type : Inapplicable

    DEALS_DEV arrow-down

    Mitapivat Safety and Proof of Concept In RBC Membranopathies/CDAII

    Details : Mitapivat is a Other Small Molecule drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Anemia.

    Product Name : Undisclosed

    Product Type : Miscellaneous

    Upfront Cash : Inapplicable

    July 08, 2025

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    A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)

    Details:

    Tebapivat is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Anemia, Sickle Cell.


    Lead Product(s): Tebapivat

    Therapeutic Area: Genetic Disease Brand Name: Undisclosed

    Study Phase: Phase IIProduct Type: Miscellaneous

    Sponsor: Undisclosed

    Deal Size: Inapplicable Upfront Cash: Inapplicable

    Deal Type: Inapplicable April 13, 2025

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    05

    Agios Pharmaceuticals

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    Lead Product(s) : Tebapivat

    Therapeutic Area : Genetic Disease

    Highest Development Status : Phase II

    Partner/Sponsor/Collaborator : Undisclosed

    Deal Size : Inapplicable

    Deal Type : Inapplicable

    DEALS_DEV arrow-down

    A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell ...

    Details : Tebapivat is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Anemia, Sickle Cell.

    Product Name : Undisclosed

    Product Type : Miscellaneous

    Upfront Cash : Inapplicable

    April 13, 2025

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    Agios' Mitapivat Meets Primary Goal in Phase 3 ACTIVATE-Kids PK Deficiency Study

    Details:

    Pyrukynd (mitapivat sulfate) is a pyruvate kinase activator. It is under phase 3 development for the treatment of children with PK deficiency who are regularly transfused.


    Lead Product(s): Mitapivat

    Therapeutic Area: Genetic Disease Brand Name: Pyrukynd

    Study Phase: Phase IIIProduct Type: Miscellaneous

    Sponsor: Undisclosed

    Deal Size: Inapplicable Upfront Cash: Inapplicable

    Deal Type: Inapplicable February 13, 2025

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    Agios Pharmaceuticals

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    Lead Product(s) : Mitapivat

    Therapeutic Area : Genetic Disease

    Highest Development Status : Phase III

    Partner/Sponsor/Collaborator : Undisclosed

    Deal Size : Inapplicable

    Deal Type : Inapplicable

    DEALS_DEV arrow-down

    Agios' Mitapivat Meets Primary Goal in Phase 3 ACTIVATE-Kids PK Deficiency Study

    Details : Pyrukynd (mitapivat sulfate) is a pyruvate kinase activator. It is under phase 3 development for the treatment of children with PK deficiency who are regularly transfused.

    Product Name : Pyrukynd

    Product Type : Miscellaneous

    Upfront Cash : Inapplicable

    February 13, 2025

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    Agios Receives FDA Acceptance for PYRUKYND® in Thalassemia

    Details:

    Pyrukynd (mitapivat sulfate) is a pyruvate kinase activator. It is being developed for adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.


    Lead Product(s): Mitapivat

    Therapeutic Area: Hematology Brand Name: Pyrukynd

    Study Phase: Phase IIIProduct Type: Miscellaneous

    Sponsor: Undisclosed

    Deal Size: Inapplicable Upfront Cash: Inapplicable

    Deal Type: Inapplicable January 08, 2025

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    Agios Pharmaceuticals

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    Lead Product(s) : Mitapivat

    Therapeutic Area : Hematology

    Highest Development Status : Phase III

    Partner/Sponsor/Collaborator : Undisclosed

    Deal Size : Inapplicable

    Deal Type : Inapplicable

    DEALS_DEV arrow-down

    Agios Receives FDA Acceptance for PYRUKYND® in Thalassemia

    Details : Pyrukynd (mitapivat sulfate) is a pyruvate kinase activator. It is being developed for adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.

    Product Name : Pyrukynd

    Product Type : Miscellaneous

    Upfront Cash : Inapplicable

    January 08, 2025

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    A Study to Determine How Tebapivat is Absorbed, Broken Down, and Removed From the Body and the Extent to Which Tebapivat is Made Available in the Body i...

    Details:

    14-C Tebapivat is a Radiolabeled Compounds drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of undefined medical condition.


    Lead Product(s): 14-C Tebapivat,Tebapivat

    Therapeutic Area: Undisclosed Brand Name: Undisclosed

    Study Phase: Phase IProduct Type: Radiolabeled Compound

    Sponsor: Undisclosed

    Deal Size: Inapplicable Upfront Cash: Inapplicable

    Deal Type: Inapplicable December 20, 2024

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    Agios Pharmaceuticals

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    Lead Product(s) : 14-C Tebapivat,Tebapivat

    Therapeutic Area : Undisclosed

    Highest Development Status : Phase I

    Partner/Sponsor/Collaborator : Undisclosed

    Deal Size : Inapplicable

    Deal Type : Inapplicable

    DEALS_DEV arrow-down

    A Study to Determine How Tebapivat is Absorbed, Broken Down, and Removed From the Body and the Ext...

    Details : 14-C Tebapivat is a Radiolabeled Compounds drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of undefined medical condition.

    Product Name : Undisclosed

    Product Type : Radiolabeled Compound

    Upfront Cash : Inapplicable

    December 20, 2024

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    EU Commission Grants Orphan Drug Designation To Agios' Mitapivat for Sickle Cell Disease

    Details:

    Pyrukynd (mitapivat sulfate) is a pyruvate kinase activator. It is under phase 3 clinical development for the treatment of pateints with sickle cell disease.


    Lead Product(s): Mitapivat

    Therapeutic Area: Genetic Disease Brand Name: Pyrukynd

    Study Phase: Phase IIIProduct Type: Miscellaneous

    Sponsor: Undisclosed

    Deal Size: Inapplicable Upfront Cash: Inapplicable

    Deal Type: Inapplicable December 18, 2024

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    Agios Pharmaceuticals

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    Lead Product(s) : Mitapivat

    Therapeutic Area : Genetic Disease

    Highest Development Status : Phase III

    Partner/Sponsor/Collaborator : Undisclosed

    Deal Size : Inapplicable

    Deal Type : Inapplicable

    DEALS_DEV arrow-down

    EU Commission Grants Orphan Drug Designation To Agios' Mitapivat for Sickle Cell Disease

    Details : Pyrukynd (mitapivat sulfate) is a pyruvate kinase activator. It is under phase 3 clinical development for the treatment of pateints with sickle cell disease.

    Product Name : Pyrukynd

    Product Type : Miscellaneous

    Upfront Cash : Inapplicable

    December 18, 2024

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    Agios Completes Enrollment of Phase 3 RISE UP Study of Mitapivat in Sickle Cell Disease

    Details:

    Pyrukynd (mitapivat sulfate) is a pyruvate kinase activator. It is under phase 3 clinical development for the treatment of pateints with sickle cell disease.


    Lead Product(s): Mitapivat

    Therapeutic Area: Genetic Disease Brand Name: Pyrukynd

    Study Phase: Phase IIIProduct Type: Miscellaneous

    Sponsor: Undisclosed

    Deal Size: Inapplicable Upfront Cash: Inapplicable

    Deal Type: Inapplicable October 23, 2024

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    Agios Pharmaceuticals

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    Lead Product(s) : Mitapivat

    Therapeutic Area : Genetic Disease

    Highest Development Status : Phase III

    Partner/Sponsor/Collaborator : Undisclosed

    Deal Size : Inapplicable

    Deal Type : Inapplicable

    DEALS_DEV arrow-down

    Agios Completes Enrollment of Phase 3 RISE UP Study of Mitapivat in Sickle Cell Disease

    Details : Pyrukynd (mitapivat sulfate) is a pyruvate kinase activator. It is under phase 3 clinical development for the treatment of pateints with sickle cell disease.

    Product Name : Pyrukynd

    Product Type : Miscellaneous

    Upfront Cash : Inapplicable

    October 23, 2024

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