Drug intelligence / Profile preview

PXS-4728A

Development stage
Phase 2
Lead developer
Boehringer Ingelheim
Modality
Small Molecules
Administration
Oral
01

Overview

PXS-4728A (also known as BI 1467335) is a first-in-class, highly selective and potent small molecule inhibitor of semicarbazide-sensitive amine oxidase (SSAO), also referred to as vascular adhesion protein-1 (VAP-1) or amine oxidase copper containing 3 (AOC3)[6][7][1]. By inhibiting SSAO/AOC3, the drug reduces leukocyte adhesion and migration, thereby decreasing inflammation and oxidative stress[6][8]. It has demonstrated efficacy in preclinical models of chronic obstructive pulmonary disease (COPD), where it suppressed airway inflammation and fibrosis and improved lung function[3]. In addition to its anti-inflammatory effects via AOC3 inhibition, PXS-4728A also inhibits monoamine oxidase B (MAO-B), increasing dopamine availability in the brain[4]. The drug was originally developed by Pharmaxis and later acquired by Boehringer Ingelheim for development in cardiometabolic diseases such as non-alcoholic steatohepatitis (NASH)[5], but is currently being investigated primarily for REM sleep behavior disorder by Syntara Limited[4][7].

Other names
4-[(E)-2-(aminomethyl)-3-fluoroprop-2-enoxy]-N-tert-butylbenzamide
02

Targets

AOC3 (Amine oxidase copper-containing 3)

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