mitapivat

an oral, small-molecule allosteric activator of pyruvate kinase

Also Known As:

8-Quinolinesulfonamide, N-(4-((4-(cyclopropylmethyl)-1-piperazinyl)carbonyl)phenyl)-; AG-348; N-(4-((4-(Cyclopropylmethyl)-1-piperazinyl)carbonyl)phenyl)-8-quinolinesulfonamide; N-(4-(4-(cyclopropylmethyl)piperazine-1-carbonyl)phenyl)quinoline-8-sulfonamide; PYRUKYND; mitapivat hemisulfate sesquihydrate; mitapivat sulfate

Networked: 33 relevant articles (11 outcomes, 18 trials/studies)

Relationship Network

Bio-Agent Context: Research Results

Heterocyclic Compounds: 198
- 1-Ring Heterocyclic Compounds
  - Piperazines: 26
    - mitapivat: 33
- Fused-Ring Heterocyclic Compounds
  - 2-Ring Heterocyclic Compounds
    - Quinolines: 146
      - mitapivat: 33

Experts

1.	Kung, Charles: 8 articles (11/2022 - 01/2017)
2.	Kosinski, Penelope A: 7 articles (11/2022 - 01/2017)
3.	Dang, Lenny: 6 articles (11/2022 - 01/2017)
4.	van Beers, Eduard J: 6 articles (01/2022 - 01/2019)
5.	Hawkins, Peter: 3 articles (11/2022 - 01/2019)
6.	Al-Samkari, Hanny: 3 articles (01/2022 - 01/2021)
7.	Rab, Minke A E: 3 articles (01/2022 - 01/2021)
8.	Eaton, William A: 2 articles (11/2022 - 01/2022)
9.	Li, Quan: 2 articles (11/2022 - 01/2022)
10.	Mangus, Heidi: 2 articles (11/2022 - 01/2021)

Related Diseases

1.	Pyruvate Kinase Deficiency of Red Cells 01/01/2019 - "In this uncontrolled, phase 2 study, we evaluated the safety and efficacy of mitapivat in 52 adults with pyruvate kinase deficiency who were not receiving red-cell transfusions. " 01/01/2019 - "Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency." 11/09/2023 - "This study aimed to validate two new PRO instruments in adult patients enrolled in the ACTIVATE clinical trial (NCT03548220), where patients with PK deficiency received the drug mitapivat or a placebo. " 11/09/2023 - "This study aimed to psychometrically validate the first disease-specific patient-reported outcome (PRO) instruments: the 7-item PK Deficiency Diary (PKDD) and 12-item PK Deficiency Impact Assessment (PKDIA), designed to assess signs, symptoms, and impacts of PK deficiency in patients enrolled in the ACTIVATE global phase 3 study of mitapivat versus placebo (NCT03548220). " 11/01/2023 - "Based on findings from two phase III clinical trials, twice-daily oral mitapivat provided clinical benefit in adults with PK deficiency, both in patients not requiring, and requiring, regular RBC transfusions. "
2.	Sickle Cell Anemia (Hemoglobin S Disease) 11/07/2023 - "One-year safety and efficacy of mitapivat in sickle cell disease: follow-up results of a phase 2, open-label study." 09/01/2023 - "Focusing on another indication, the ongoing RISE trial investigates the optimal dosage of mitapivat in sickle cell disease. " 01/01/2021 - "Following these successful early-phase trials, two phase III trials of mitapivat in thalassemia and a phase II/III trial of mitapivat in sickle cell disease are beginning worldwide. " 03/01/2023 - "Mitapivat for sickle cell disease and thalassemia." 01/01/2022 - "Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in sickle cell disease: A phase 2, open-label study."
3.	Thalassemia 01/01/2021 - "In ex vivo studies on erythroid precursors from patients with β-thalassemia, mitapivat enhanced erythropoiesis, promoted erythroid maturation, and decreased apoptosis. " 03/01/2023 - "Together, these efficacy and safety results provide rationale for continuing investigation of mitapivat for the treatment of thalassemia and SCD, developing other PK activators and starting investigational studies in other acquired diseases characterized by dyserythropoiesis and hemolytic anemia." 03/01/2023 - "The efficacy and safety of mitapivat were confirmed in an open-label, multicenter, phase II study of patients with non-transfusion-dependent α-thalassemia or β-thalassemia, where activation of PKR improves anemia, and the drug showed a tolerable safety profile comparable to that in previous studies in other hemolytic anemias. " 01/01/2021 - "Following these successful early-phase trials, two phase III trials of mitapivat in thalassemia and a phase II/III trial of mitapivat in sickle cell disease are beginning worldwide. " 03/01/2023 - "Mitapivat for sickle cell disease and thalassemia."
4.	Hemolysis 11/01/2023 - "In both trials, mitapivat improved other clinical parameters of haemolysis and patient-reported health-related quality of life. " 10/01/2022 - "Given the considerable genetic heterogeneity of PKD and the rapid effect on improving hemoglobin and reducing hemolysis, a therapeutic trial of mitapivat should be considered in all patients with PKD who are not homozygous for the PKLR R479H mutation. " 01/01/2023 - "Both mitapivat and etavopivat have been evaluated in mouse models for SCD, showing an increased 2-3DPG/ATP ratio and a reduction in haemolysis as well as in sickling. " 09/01/2023 - "Mitapivat is an appropriate treatment for adults with PK deficiency requiring transfusions and may be considered for patients with symptomatic anemia who do not require transfusions and/or PK deficiency with compensated hemolysis without overt anemia." 01/01/2022 - "In patients with pyruvate kinase deficiency, mitapivat significantly increased the hemoglobin level, decreased hemolysis, and improved patient-reported outcomes. "
5.	Hemolytic Anemia 10/01/2022 - "We discuss preclinical data and phase 1, 2, and 3 clinical studies evaluating mitapivat for adult patients with hemolytic anemia secondary to PKD. " 01/01/2024 - "Mitapivat: A Novel Treatment of Hemolytic Anemia in Adults with Pyruvate Kinase Deficiency." 03/01/2023 - "Together, these efficacy and safety results provide rationale for continuing investigation of mitapivat for the treatment of thalassemia and SCD, developing other PK activators and starting investigational studies in other acquired diseases characterized by dyserythropoiesis and hemolytic anemia." 10/01/2022 - "An evaluation of mitapivat for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency." 11/01/2023 - "Mitapivat (Pyrukynd®), an oral, allosteric activator of pyruvate kinase (PK), is approved in the USA for the treatment of haemolytic anaemia in adults with PK deficiency and in the EU and UK for the treatment of PK deficiency in adults. "

Related Drugs and Biologics

1.	Pyruvate Kinase
2.	Hemoglobins (Hemoglobin)
3.	Adenosine Triphosphate (ATP)
4.	Reactive Oxygen Species (Oxygen Radicals)
5.	Iron
6.	2,3-Diphosphoglycerate (2,3 Bisphosphoglycerate)
7.	Quinolones
8.	Sulfonamides
9.	Enzymes
10.	voxelotor

Related Therapies and Procedures

1.	Drug Therapy (Chemotherapy)
2.	Blood Transfusion (Blood Transfusions)
3.	Therapeutics
4.	Splenectomy