Drug intelligence / Profile preview

OCU300

Development stage
Unknown
Lead developer
Ocugen
Modality
Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems, Small Molecules
Administration
Ophthalmic
01

Overview

OCU300 is a proprietary nanoemulsion formulation of the FDA-approved drug brimonidine tartrate, developed by Ocugen for the treatment of ocular graft versus host disease (oGVHD). The drug leverages Ocugen’s patented OcuNanoE technology to enhance ocular surface retention and efficacy. Brimonidine tartrate is an established alpha-2 adrenergic receptor agonist with anti-inflammatory properties. While traditionally used in glaucoma, this novel preservative-free nanoemulsion aims to address the significant unmet need in oGVHD—a severe immune-mediated complication affecting 40–60% of allogeneic stem cell transplant recipients. OCU300 was granted Orphan Drug Designation by the FDA for oGVHD and advanced into Phase 3 clinical trials as potentially the first approved therapy for this indication[1][2][4][5][7].

Brand names
OCU300OCU-300OCU 300
Other names
brimonidine tartrate nanoemulsionbrimonidine tartrate (nanoemulsion formulation)
02

Targets

ADRA2C (α2C)ADRA2B (α2B)ADRA2A (α2A)

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