Re-Imagining The Other Side of Possible

A singular focus on genetically defined diseases.


European Hematology Association Virtual Congress 2021

Agios Medical Booth

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Agios EHA Presentations

View a list of all our EHA presentations and posters.

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Interactive Pipeline

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Cheat Codes: A Sickle Cell Podcast

“Cheat Codes: A Sickle Cell Podcast” is a new podcast series focused on explaining emerging research from major medical and scientific congresses.

The inaugural episode is focused on the 2020 American Society of Hematology (ASH) Annual Meeting.

Find Cheat Codes online at bloodstreammedia.com/shows/cheat-codes, or search for “Cheat Codes: A Sickle Cell Podcast” wherever you stream your podcasts.

• The podcast is intended for HCPs.
• Agios is a founding sponsor of this new series; the content is developed independently.
• This podcast mentions mitapivat, an investigational compound. The safety and efficacy of mitapivat has not been established. There is no guarantee that mitapivat will receive health authority approval or become commercially available in any country for the uses being investigated.

PKR Activation in Hemolytic Anemias

Agios is developing mitapivat, an investigational, first-in-class, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase R (PKR) enzymes, in three distinct hemolytic anemias: pyruvate kinase (PK) deficiency, thalassemia and sickle cell disease. We believe PKR activation has the potential to transform the course of hemolytic anemias by improving red blood cell energy, health and longevity.

Voice of the Patient Report on PK Deficiency

This ground-breaking report was the direct result of PK deficiency patients, caregivers and medical communities coming together at an externally-led Patient-Focused Drug Development Meeting in September 2019. The report features perspectives from patients and caregivers who provided personal testimony of their experiences living with the disease: their challenges, their hopes for treatments and their perspectives on the risks and benefits of potential new treatments.

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Resources for PK Deficiency Patients

These patient-friendly resources provide more information about PK deficiency, symptoms and strategies for disease management.

PEAK Registry

Due to the rarity of PK deficiency, its prevalence, clinical burden, and long-term clinical course are not well defined. PEAK is an ongoing, longitudinal, observational registry study designed to address this gap. Its primary objective is to record the natural history, treatment, outcomes, variability in clinical manifestations and disease burden of patients with PK deficiency. Approximately 500 patients are expected to be enrolled over 7 years at an estimated 60 study centers in up to 20 countries in the 9-year study. All enrolled patients will be followed prospectively for at least 2 years and up to 9 years. Site and patient recruitment began in 2018.

Download Trial Card for the PEAK Registry

Ongoing Clinical Trials

Download Trial Card for ENERGIZE/ENERGIZE-T phase 3 trials