Agios Innovative Other Structure Activator reaches Phase 3 Clinical End and is expected to file a regulatory application in the second quarter

Acetone kinase deficiency is a rare genetic disorder manifested in chronic hemolytic anemia caused by the accelerated destruction of red blood cells.
mutations in the
PKR gene lead to insufficient cellular energy in red blood cells, which can be manifested in reduced PK enzyme activity, decreased levels of adenosine triphosphate (ATP) and accumulation of upstream metabolites, including 2,3-DPG.
serious complications associated with acetone kinase deficiency include gallstones, pulmonary hypertension, extra-myelin hematosis, osteoporosis and iron overload, and their sequelae.
symptoms can occur regardless of the level of anemia or the burden of blood transfusions.
there is currently no approved treatment for acetone kinase deficiency.
Mitapivat is a potential "first-in-class" PKR-constructed activator.
can activate wild PKR and a range of PKR mutants.
it can improve the energy supply of red blood cells and improve the health of red blood cells.
-label Phase 3 clinical trial, called ACTIVE-T, was conducted in adult patients with acetone kinase deficiency who required regular blood transfusions, with a total of 27 patients in the group.
they were all treated with mitapivat.
after 24 cycles of fixed-dose therapy, the blood transfusion load decreased by ≥33% (one-sided p-0.0002) compared to the patient's historical blood transfusion load.
addition, 22% of patients (n-6) do not need a blood transfusion.
Agios announced in December that mitapivat had also reached its main endpoint in phase 3 clinical trials of ACTATE in adult patients with acetone kinase deficiency who did not require regular blood transfusions.
hemoglobin levels in patients treated with mitapivat were statistically significant and sustained compared to placebos.
based on the results of two Phase 3 clinical trials, ACTIVATE and ACTATE-T, we believe mitapivat may provide meaningful benefits for patients with acetone kinase deficiency.
Chris Bowden, chief medical officer of Agios, said, "We look forward to working with regulators in the United States and the European Union to bring mitapivat to patients quickly."
: This article is intended to introduce medical and health research, not treatment options recommended.
if you need guidance on treatment options, visit a regular hospital.
: Agios Announces Phase 3 ACTIVATE-T Trial of Mitapivat Achieved Primary Endpoint in Adults with Pyruvate Kinase Health Who Are RegularLy Transfused. Retrieved January 26, 2021, from