About Mitapivat
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
2014
Agios initiates clinical study of mitapivat3
2020
Establishes clinical proof-of-concept for mitapivat in sickle cell disease and reports phase 1 clinical trial findings3,4
2021
Reports phase 2 open-label clinical trial findings of mitapivat in patients with sickle cell disease5
2022
Launches phase 2/3 RISE UP clinical trial for mitapivat in patients with sickle cell disease
Get ready for the next phase of rise up
Learn more about RISE UP, including full enrollment details, all study objectives, and key inclusion and exclusion details. Then talk to your eligible patients about the study.
Call Agios Pharmaceuticals Medical Affairs at 1-833-228-8474, Mon-Fri, 9 am to 7 pm ET, or email medinfo@agios.com.
References:
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.