Piperidine is an azacycloalkane that is cyclohexane in which one of the carbons is replaced by a nitrogen. Although piperidine is a common organic compound, it is an immensely important class of compounds medicinally: the piperidine ring is the most common heterocyclic subunit among FDA approved drugs.
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Piperazine is an organic compound consisting of a six-membered ring containing two nitrogen atoms in opposite positions in the ring. The chemical formula of piperazine is C4H10N2, and it is an important pharmaceutical intermediate. Pyrimidines and piperazines are known to be the backbone of many bulk compounds and important core structures for approved drugs; studies have shown that combining a pyridine ring with a piperazine moiety within a single structural framework enhances biological activity.
Jan. 03 2024, Agios announces Phase 3 ENERGIZE study of PYRUKYND (Mitapivat) met primary endpoint and both key secondary endpoints in adults with non-transfusion-dependent(NTD) alpha- or beta-thalassemia. The positive data showed Mitapivat’s potential to become the first oral therapy approved for NTD thalassemia.
Mitapivat is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the US, and for the treatment of PK deficiency in adult patients in the EU. By activating and stabilizing mutant pyruvate kinase, Mitapivat targets the underlying enzymatic defect that causes chronic hemolysis and ineffective erythropoiesis in order to restore enzyme activity.
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