Drug intelligence / Profile preview

doxycycline + tauroursodeoxycholic acid

Development stage
Unknown
Lead developer
Pfizer
Modality
Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
Administration
Oral
01

Overview

Doxycycline + tauroursodeoxycholic acid is an oral combination therapy investigated primarily for the treatment of transthyretin (TTR) amyloidosis, including both hereditary and wild-type forms. Doxycycline is a tetracycline antibiotic known to disrupt existing amyloid fibrils and inhibit their formation, while tauroursodeoxycholic acid (TUDCA), a hydrophilic bile acid, acts as a chemical chaperone that stabilizes protein folding and prevents apoptosis by interfering with mitochondrial pathways. The combination has shown synergistic effects in preclinical models, leading to reduced TTR deposition and stabilization of disease progression in clinical studies. This regimen has been evaluated in phase II trials for its ability to slow or stabilize neuropathy and cardiomyopathy associated with TTR amyloidosis[1][3][4][5].

Other names
Doxy-TUDCA
02

Targets

BAX (Apoptosis regulator BAX)Bacterial RibosomeTTR (Transthyretin)

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