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AN2-504661
- Development stage
- Preclinical
- Lead developer
- AN2 Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
AN2-504661 is a novel, oral, boron-based small-molecule inhibitor of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) under development by AN2 Therapeutics. It is being developed as a potential best-in-class oral treatment for hypophosphatasia (HPP), a rare inherited metabolic bone disease caused by loss-of-function mutations in the ALPL gene. Preclinical evaluations have demonstrated that AN2-504661 exhibits potent and selective ENPP1 inhibition, oral bioavailability, efficacy in animal models, and a favorable nonclinical safety profile with low potential for hERG-related cardiac effects. The compound is expected to be Phase 1-ready in the second half of 2027.
02
Targets
ENPP1
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