Drug intelligence / Profile preview

Apratoxin S4

Development stage
Preclinical
Lead developer
University of Florida
Modality
Small Molecules
Administration
Intraperitoneal, Intravenous, Local Ocular
01

Overview

**Apratoxin S4** is a synthetic small molecule derived from marine cyanobacterial natural products (apratoxins A and E), developed as a potent inhibitor of the Sec61 translocon, which blocks cotranslational translocation of secretory and membrane proteins into the endoplasmic reticulum (ER). This mechanism underlies its anticancer activity by disrupting protein secretion in tumor cells, antiangiogenic effects by inhibiting retinal endothelial cell and pericyte activation to prevent pathological neovascularization in ocular diseases, and broad-spectrum antiviral potency (subnanomolar against SARS-CoV-2 and influenza A) by impairing viral protein production, replication organelle formation (e.g., double-membrane vesicles), and secretory pathway-dependent trafficking. It shows subnanomolar potency in vitro, in vivo efficacy in HCT116 xenograft models without toxicity, stability under physiological conditions, and potential synergy with VEGF inhibitors; commercial rights to the apratoxin family are licensed to Oceanyx Pharmaceuticals.

Other names
DTXSID001334242DTXSID-001334242DTXSID 001334242DTXCID301763642DTXCID-301763642DTXCID 301763642orb1982520orb-1982520orb 1982520
02

Targets

SEC61A1 (Sec61 subunit alpha-1)

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