Agios publishes phase 2 clinical results of "first in class" innovative therapy

At the 25th annual meeting of the European hematology Association (EHA), Agios pharmaceuticals reported the positive phase 2 clinical data of mitapivat in the treatment of thalassemia and the proof of concept clinical data of treating sickle cell anemiaMitapivat is a "first in class" oral small molecule allosteric activator of pyruvate kinase-r (PKR)It can activate wild-type and PKR with multiple gene mutationsThese data suggest that PKR activation may be a new mechanism for the treatment of thalassemia and sickle cell anemia< br / > thalassemia and sickle cell anemia are both inherited blood diseases caused by mutations in the gene encoding hemoglobinThalassemia can be divided into two types: α and βα - thalassemia patients have mutations in the gene encoding hemoglobin α subunit, β - thalassemia patients have mutations in the gene encoding hemoglobin β subunitPatients with severe thalassemia and sickle cell anemia need blood transfusion< br / > at present, gene therapy for these two kinds of anemia has attracted many people's attentionThe development strategy of Agios is "a new way"They target the PKR enzyme in the metabolism of red blood cellsPKR is one of the key enzymes in glycolysis of red blood cellsRed blood cells rely almost entirely on glycolysis for energyGlycolysis is essential for maintaining the health and differentiation of red blood cellsIn hemolytic anemia, this metabolic pathway is often abnormal< br / > < br / > ▲ the role of PKR in the glycolysis of erythrocytes (picture source: reference [3]) < br / > mitapivat is a "first in class" PKR allosteric activatorBy activating PKR, it may improve the energy supply of red blood cells, improve the health level of red blood cells, and reduce the occurrence of sickle and hemolysis of red blood cells < br / > in the phase 2 concept validation clinical trial for the treatment of thalassemia patients who do not rely on blood transfusion, a total of 18 patients received the drug, and 13 patients could receive the efficacy evaluation on the data deadline Efficacy data showed that 12 of 13 patients (92.3%) completed 12 weeks treatment, including 8 of all 4 patients (100%) with α - thalassemia and 9 patients (88.9%) with β - thalassemia, reached the main end point of the trial (defined as the increase of hemoglobin concentration ≥ 1.0 g / dl compared with the baseline in one or more evaluations between the 4th and 12th cycles) < br / > in week 4-12, the mean hemoglobin level of all 13 patients with evaluable efficacy increased 1.34 g / dl from baseline < br / > < br / > Among the 8 patients who completed the mitapivat plan, 7 (88%) had an increase in hemoglobin level, and 5 (63%) had an increase of ≥ 1.0 g / dl (1.0-2.7 g / dl) from baseline According to < br / > Agios, these data support the development of mitapivat in the treatment of thalassemia to a critical stage Critical clinical trials are expected to start in 2021 At the same time, the company will also launch research in 2021 to support the critical development of mitapivat in the treatment of sickle cell anaemia < br / > References: < br / > [1] Agios announcements clinical proof of concept has been established in phase 1 study of mitapivat, first in class PKR activator, in wickle cell release Retrieved June 12, 2020, From < br / >