The connection between people with rare diseases and our therapies may be the most important connection we make.
Before investigational medicines can be made broadly available, the U.S. Food and Drug Administration (FDA) and other health authorities around the world require that they are studied in clinical trials.
Clinical trials are research studies that are designed to determine if a medicine is safe and effective for patients. Throughout the process, the safety of people participating in our clinical studies is of utmost importance to us, and many safeguards are in place.
People who choose to participate in clinical trials play a critical role in developing new medicines, particularly for rare diseases and conditions that are especially difficult to treat.
Our ongoing clinical trials are listed below.
Pyruvate Kinase Deficiency
Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007
Phase 2 DRIVE PK Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency
Pyruvate Kinase Deficiency Global Longitudinal Registry (PEAK Registry)
A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non-Transfusion Dependent Alpha- or Beta-Thalassemia (ENERGIZE)
A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T)
A Phase 2, Open-label, Multicenter Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Subjects With Non-transfusion-dependent Thalassemia
Sickle Cell Disease
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease
Phase 2a/2b, Open-Label, Proof of Concept (Phase 2a) and Double-Blind, Randomized, Placebo-Controlled (Phase 2b), Multicenter, Efficacy, and Safety Study of AG-946 in Participants with Anemia Due to Lower-Risk Myelodysplastic Syndromes
The safety and efficacy of the agents and uses under investigation have not been established.
There is no guarantee that the agents will receive health authority approval or become commercially available in any country for the uses being investigated.
Clinical trials are the most appropriate and most common way for people to access medicines before they are approved by local health authorities.
At this time, Agios does not have an expanded access program in place to provide access to our investigational medicines outside of our clinical programs, as the benefit/risk profile is still being evaluated.
Meet Dr. Sarah Gheuens, Agios Chief Medical Officer
Our Chief Medical Officer, Sarah Gheuens, M.D., Ph.D., was a guest on Cheat Codes: A Sickle Cell Podcast. In this episode, she shares more about her background, the clinical development process, and Agios’ approach to partnering with patient communities.
A pipeline of possibility
Our unique understanding of cellular metabolism is foundational to our success and the connective thread that binds our people and our pipeline together.