Agios Medical Booth
American Society of Hematology Meeting 2020
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.
Resources for PK Deficiency Patients
These patient-friendly resources provide more information about PK deficiency, symptoms and strategies for disease management.
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.
IDH Inhibition in Oncology
IDH Mutations in Cancer
At Agios, our lead oncology programs are focused on cancers with a mutated isocitrate dehydrogenase (IDH) protein. IDH mutations occur in a wide range of blood and solid tumor cancers, including acute myeloid leukemia (AML), cholangiocarcinoma and glioma. Normally, IDH enzymes help to break down nutrients and generate energy for cells. When mutated, IDH creates a molecule that alters the cells’ genetic programming, and instead of maturing, the cells remain primitive and proliferate quickly. Agios believes that targeting mutated IDH may have the potential to benefit the subset of patients with tumors that carry IDH mutations.
IDH Inhibition Mechanism of Action
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