The Study Drug
PK Activation supports red blood cell health
Mitapivat — the study drug in RISE UP — is an investigational, oral, allosteric activator of the pyruvate kinase enzyme1,2
PK enzyme activation may improve the health, energy, and lifespan of red blood cells (RBCs) for patients with hemolytic anemias1
- Increasing ATP production, helping to match red blood cell energy needs
- Decreasing 2,3-DPG which reversibly increases oxygen affinity for hemoglobin, potentially reducing sickling
- Maintaining antioxidants, thereby reducing cellular damage
Agios is a pioneer in PK activation
Agios initiates clinical study of mitapivat3
Establishes clinical proof-of-concept for mitapivat in sickle cell disease and reports phase 1 clinical trial findings3,4
Reports phase 2 open-label clinical trial findings of mitapivat in patients with sickle cell disease5
Launches phase 2/3 RISE UP clinical trial for mitapivat in patients with sickle cell disease
1. Data on file. Agios Pharmaceuticals, Inc.
2. Howard J, Kuo KHM, Oluyadi A, et al. A phase 2/3, randomized, double-blind, placebo-controlled study of mitapivat in patients with sickle cell disease. Blood. 2021;138(suppl 1):3109.
3. A timeline of innovation and patient impact. Agios Pharmaceuticals, Inc. Accessed March 21, 2022. https://www.agios.com/about-us/history/
4. Xu JZ, Conrey A, Frey I, et al. Phase 1 multiple ascending dose study of safety, tolerability, and pharmacokinetics/pharmacodynamics of mitapivat (AG-348) in subjects with sickle cell disease. Presented at: American Society of Hematology Annual Meeting; December 5-8, 2020.
5. van Dijk MJ, Rab MAE, Rijneveld AW, et al. Safety and efficacy of mitapivat (AG-348), an oral activator of pyruvate kinase R, in subjects with sickle cell disease: A phase 2, open-label study (ESTIMATE). Presented at: American Society of Hematology Annual Meeting; December 11-14, 2021; Atlanta, GA.