Illuminating the natural history of PK deficiency, as well as potential disease-specific treatment options, is central to improving care of patients with PK deficiency,1 and is a key focus at Agios.
Mitapivat (AG-348) is an investigational compound currently being developed as part of Agios' PK deficiency clinical program.
Two global pivotal (phase 3) trials for mitapivat in adults with PK deficiency are actively recruiting patients:
A 1:1 randomized, placebo-controlled trial of 80 patients who do not receive regular transfusions (NCT03548220).
The primary objective of the ACTIVATE Phase 3 study is to evaluate the efficacy of treatment with mitapivat, as assessed by a sustained increase in hemoglobin (Hb) concentrations.
A single-arm trial of up to 40 regularly transfused patients (NCT03559699).
The primary objective of the ACTIVATE-T Phase 3 study is to evaluate the efficacy of treatment with mitapivat, as assessed by the reduction in transfusion burden.
The Peak Registry, a global, longitudinal study of patients with PK deficiency, has been established to better understand the full spectrum of disease variability, including impact on quality of life.
The registry is committed to furthering the understanding of PK deficiency, including disease burden.
Your participation as an enrolling site will help increase knowledge and awareness of this rare genetic disorder.
Contact Agios Medical Affairs to learn about the Agios-sponsored studies shown here.
The safety and efficacy of mitapivat has not been established. There is no guarantee that it will receive health authority approval or become commercially available in any country for the use being investigated.