Drug intelligence / Profile preview

BT01001

Development stage
Phase 1
Lead developer
Beyang Therapeutics
Modality
Small Molecules
Administration
Ophthalmic, Topical
01

Overview

BT01001 is a first-in-class small molecule inhibitor being developed by Beyang Therapeutics for the treatment of diabetic retinopathy (DR), specifically severe non-proliferative diabetic retinopathy (sNPDR). Formulated as a topical ophthalmic solution (eye drop), the drug targets both the vascular endothelial growth factor receptor (VEGFR) and fibroblast growth factor receptor (FGFR) kinase families. It represents a novel non-invasive alternative to traditional intravitreal injections, designed to shift the treatment paradigm from standard observation to a proactive strategy aimed at slowing disease progression and preventing vision-threatening complications. The U.S. FDA cleared the Investigational New Drug (IND) application for BT01001 in March 2025, and the company is conducting Phase I clinical trials to evaluate its safety, tolerability, and pharmacokinetics.

Other names
BT01001 Ophthalmic SolutionBT-01001 Ophthalmic SolutionBT 01001 Ophthalmic Solution
02

Targets

FGFR (FGFR family)

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