Drug Pipeline

Methotrexate enters tissues and is converted to a methotrexate polyglutamate by folylpolyglutamate. Methotrexate's mechanism of action is due to its inhibition of enzymes responsible for nucleotide synthesis including dihydrofolate reductase, thymidylate synthase, aminoimidazole caboxamide ribonucleotide transformylase (AICART), and amido phosphoribosyltransferase. Inhibtion of nucleotide synthesis prevents cell division. In rheumatoid arthritis, methotrexate polyglutamates inhibit AICART mor

Tissue factor pathway inhibitor inhibitor

Phenylalanine hydrolytic enzyme

Phenylketonuria (PKU) is an inherited metabolic disorder characterized by reduced functionality of phenylalanine hydroxylase (PAH), an enzyme responsible for converting phenylalanine (Phe) to tyrosine. This deficiency leads to elevated blood Phe levels, which can result in neurocognitive deficits. Sepiapterin is a natural precursor of the enzymatic co-factor tetrahydrobiopterin (BH4), which is critical for PAH function. Sepiapterin acts as a dual pharmacological chaperone, binding to variant PA

Sapropterin dihydrochloride is a synthetic form of BH4, the cofactor for the enzyme phenylalanine hydroxylase (PAH). PAH hydroxylates Phe through an oxidative reaction to form tyrosine. In patients with PKU, PAH activity is absent or deficient. Treatment with BH4 can activate residual PAH enzyme activity, improve the normal oxidative metabolism of Phe, and decrease Phe levels in some patients.