Mitapivat

SKU: orb1223357

Description

Mitapivat, also known as PKM2 activator 1020 is a PKM2 activator (pyruvate kinase activator) for the treatment of pyruvate kinase deficiency.

Key Properties

• CAS Number: 1260075-17-9
• MW: 450.56
• Purity: >98% (HPLC)
• Formula: C₂₄H₂₆N₄O₃S
• SMILES: C1CC1CN1CCN(CC1)C(=O)c1ccc(cc1)NS(=O)(=O)c1cccc2c1nccc2
• Target: Syk
• Solubility: DMSO : 50 mg/mL. 110.98 mM

Bioactivity

• In Vivo:
  Animal model: WT C57B6 and Hbbth3/+ mice (both are 2-month-old female mice; β-thalassemia model). Dosage: 50 mg/kg. Administration: In animal feedings; single daily for 3 weeks. Result: Increased the expression of pyruvate kinase isoforms in both red cells and erythroid precursors from Hbbth3/+mice. Elevated pyruvate kinase activity in cells from Hbbth3/+mice, and markedly increased ROS level in erythrocytes. Increased the expression of PKM2 in polychromatic and orthochromatic erythroblasts of Hbbth3/+mice. Animal model: WT C57B6 and Hbbth3/+ mice (both are 2-month-old female mice; β-thalassemia model). Dosage: 50 mg/kg. Administration: Oral gavage, twice daily for 21 days. Result: Ameliorated ineffective erythropoiesis and anemia in Hbbth3/+ mice and increased ATP, reduced ROS production, as well as reduced markers of mitochondrial dysfunction associated with improved mitochondrial clearance.
• In Vitro:
  Cell Viability Assay Cell line: RBC cells. Concentration: 0.1 nM-100 μM Incubation time: 16 h (incubate overnight). Result: Increased PK-R activity in a dose-dependent manner to ~2.5-fold of DMSO control with an AC50 of 62 nM. Cell Viability Assay Cell line: RBC cells. Concentration: 0.01 nM-10 μM Incubation time: 16 h (incubate overnight). Result: Consistently increased ATP levels in a dose-dependent manner by an average of 60% over DMSO control with an AC50 of 10.9 nM.

Storage & Handling

• Storage: Storage temperature: -20°C. Stability: ≥ 2 years
• Expiration Date: 12 months from date of receipt.
• Disclaimer: For research use only

Alternative Names

PKR-IN-1 | PKM2 activator 1020 | Mitapivat

Quick Database Links

Gene Symbol

Syk