Drug intelligence / Profile preview

benserazide hydrochloride

Development stage
Unknown
Lead developer
Phoenicia BioSciences
Modality
Small Molecules
Administration
Oral
01

Overview

Benserazide hydrochloride (benserazide HCl) is a peripherally-acting aromatic L-amino acid decarboxylase (AADC) or DOPA decarboxylase (DDC) inhibitor. It is unable to cross the blood-brain barrier, allowing it to selectively inhibit the conversion of levodopa to dopamine in the peripheral circulation. This increases the amount of levodopa that can cross into the central nervous system and reduces peripheral side effects such as nausea and arrhythmias. Benserazide HCl is widely used in combination with levodopa (as co-beneldopa) under brand names like Madopar and Prolopa for the treatment of Parkinson's disease and restless legs syndrome. Additionally, benserazide HCl is being investigated as a single agent in Phase 1/2 clinical trials for the treatment of beta-thalassemia and sickle cell disease due to its ability to induce fetal hemoglobin (HbF) expression. Preclinical research also suggests potential applications in oncology, particularly melanoma, through the inhibition of pyruvate kinase M2 (PKM2) and cystathionine beta-synthase (CBS).

Other names
2-amino-3-hydroxy-N'-(2,3,4-trihydroxybenzyl)propanehydrazide hydrochloridebenserazidebenserazide HClbenserazide hydrochlorideDL-serine 2-(2,3,4-trihydroxybenzyl)hydrazine hydrochlorideDL-serine, 2-(2,3,4-trihydroxybenzyl)hydrazide hydrochlorideDL-serine, 2-(2,3,4-trihydroxybenzyl)hydrazide monohydrochlorideserazide
02

Targets

DDC (Aromatic L-amino acid decarboxylase)HK1 (Hexokinase type I (mitochondrial))HK2 (Hexokinase Type II)GCK (Glucokinase)KYNU (Kynureninase)AOC3 (Amine oxidase copper-containing 3)TDO2 (Tryptophan 2,3-dioxygenase)CBS (Cystathionine beta-synthase)PKM2 (Pyruvate kinase M2 isoform)

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