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pentamidine (University of Massachusetts)

Development stage
Phase 1
Lead developer
UMass Chan Medical School
Modality
Small Molecules
Administration
Oral
01

Overview

VLX103 is an oral formulation of pentamidine, a diamidine compound traditionally used as an anti-infective agent for the treatment of *Pneumocystis* pneumonia and leishmaniasis. Developed as a novel therapeutic approach for liver diseases, specifically alcoholic steatohepatitis (ASH), VLX103 aims to leverage the anti-inflammatory properties of pentamidine through a more convenient oral delivery system. The drug is thought to exert its therapeutic effects by inhibiting inflammatory cytokine production (such as TNF-alpha) and potentially modulating signaling pathways involved in liver cell damage and fibrosis, including the inhibition of protein tyrosine phosphatases. Clinical evaluation has focused on its safety, pharmacokinetics, and preliminary efficacy in reducing liver inflammation in patients with moderate ASH, representing the first oral form of pentamidine to enter clinical development.

Other names
oral pentamidine
02

Targets

Protozoal DNA-associated enzymesProtozoal AT-rich DNAKCNH2 (Voltage-gated potassium channel subfamily H member 2)

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