Drug intelligence / Profile preview

port-77

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

Overview

PORT-77 is an orally bioavailable small molecule inhibitor of the ATP-binding cassette transporter subfamily G member 2 (ABCG2, also known as BCRP). It is under development for the treatment of erythropoietic protoporphyria (EPP), a rare genetic disorder characterized by phototoxic skin reactions and progressive liver damage due to accumulation of protoporphyrin IX. Preclinical studies demonstrated that PORT-77 potently inhibits ABCG2 activity *in vitro* and reduces skin phototoxicity and liver damage in mouse models of EPP. The drug has shown favorable safety and pharmacokinetic profiles in early studies, with ongoing phase 1 clinical trials evaluating its safety in healthy volunteers. The originator is Portal Therapeutics, with BridgeBio Pharma also involved in development[1][3][4].

02

Targets

ABCG2 (ATP-binding cassette sub-family G member 2)

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