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.
Cyclopropane is the smallest cyclic compound with unique structural features and physicochemical properties, which is widely used in the design of small molecule drugs. In drug design, it is often used to increase activity, fix conformation and improve PK and water solubility. The introduction of cyclopropyl groups into drugs can change various properties of molecules, such as improving metabolic stability; increasing biological activity; enhancing drug efficacy; limiting polypeptide conformation and slowing down its hydrolysis; reducing plasma clearance; improving drug dissociation and many more. Cyclopropane rings are widely found in marketed drugs, including cardiovascular drugs, central nervous system (CNS) drugs, anticancer drugs, autoimmune and anti-inflammatory drugs.
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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