Drug intelligence / Profile preview

PBT2

Development stage
Phase 2
Lead developer
Alterity Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

PBT2 is a second-generation 8-hydroxyquinoline analog and metal protein-attenuating compound (MPAC) developed primarily for the treatment of Alzheimer's disease and Huntington's disease. It acts as an ionophore that disrupts the interaction between metals (notably copper and zinc) and amyloid-beta (Aβ) peptide in the brain. By translocating copper and zinc ions into cells, PBT2 reduces their extracellular levels, thereby decreasing metal-mediated Aβ aggregation—a process implicated in neurodegenerative diseases. This mechanism also helps restore copper and zinc ion homeostasis in neural tissue. In preclinical models, PBT2 has been shown to improve synaptic health indicators such as spine density and synaptic protein levels. Clinically, it has demonstrated safety and tolerability in Phase II trials for Alzheimer's disease with some evidence of cognitive benefit at higher doses[1][2][3][4]. Additionally, research suggests potential utility for reversing antibiotic resistance when co-administered with zinc[3][4].

Other names
8-hydroxyquinoline analogmetal protein-attenuating compoundMPAC
02

Targets

Aβ (Amyloid-beta peptides and aggregates)Zn-Aβ (Amyloid-beta zinc-binding site)SLC39A6 (Solute carrier family 39 member 6)

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