Drug intelligence / Profile preview

PBT-4

Development stage
Preclinical
Lead developer
Alterity Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

PBT-4 is a research-stage small molecule drug candidate developed by Alterity Therapeutics (formerly Prana Biotechnology). It belongs to the Metallo-Protein Attenuating Compound (MPAC) class, a proprietary platform of compounds designed to cross the blood-brain barrier and modulate the distribution of transition metals in the central nervous system. PBT-4 is specifically engineered to redistribute labile iron and copper, which are known to play a critical role in the misfolding and aggregation of proteins such as alpha-synuclein and beta-amyloid. By restoring metal homeostasis and preventing the formation of toxic protein aggregates and associated oxidative stress, PBT-4 aims to treat various neurodegenerative nervous system diseases. While its sibling compound ATH434 (formerly PBT434) is the company's lead clinical candidate for Multiple System Atrophy, PBT-4 remains a distinct asset within the PBT series portfolio.

02

Targets

MAPT (Microtubule-associated protein tau)SNCA (Alpha-synuclein)Cu (Copper ion)

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