Ocugen, Inc., (NASDAQ: OCGN) today announced that it has completed 50% of enrollment of its Phase 3 clinical trial for OCU300 for patients with ocular Graft Versus Host Disease (oGVHD). Ocugen is the first and only company to receive Orphan Drug Designation for a product candidate designed to treat oGVHD and is the first company to conduct a Phase 3 clinical trial in patients with this disease.

The ongoing Phase 3 trial is a double-masked, placebo-controlled 84-day trial, in which 60 patients will be randomized in a 2:1 ratio to receive either OCU300 (brimonidine 0.18% nanoemulsion) or a placebo. The purpose of the trial is to evaluate the safety and efficacy of brimonidine tartrate nanoemulsion eye drop solution in the treatment of ocular redness and ocular discomfort in patients with oGVHD. The trial has co-primary endpoints of ocular discomfort based on a 10-point visual analog scale and ocular redness based on a 100-point validated bulbar redness score.

Daniel Jorgensen, M.D., M.P.H., M.B.A., Chief Medical Officer of Ocugen, stated, “The achievement of 50% enrollment is a key milestone towards the completion of this important Phase 3 trial. oGVHD is a severe ocular autoimmune disease that occurs in up to 60% of allogeneic bone marrow transplant patients. There is no approved therapy for this debilitating condition, and it is our belief that OCU300 will provide needed relief for patients suffering from this disease.” Dr. Jorgensen continued, “We remain on track to report topline results from this trial in the second half of 2020.”

About OCU300 OCU300 is in pivotal stage clinical development for treating ocular discomfort and ocular redness in patients with the debilitating autoimmune condition called ocular Graft Versus Host Disease (oGVHD), which develops in many patients following an allogeneic bone marrow transplant. It is the only product candidate to be granted Orphan Drug Designation for this indication from the U.S. Food and Drug Administration (FDA), and it consists of an improved 0.18% ophthalmic nanoemulsion of brimonidine tartrate, an FDA-approved drug with established safety for ocular use, enabling Ocugen to develop OCU300 under the accelerated 505(b)(2) regulatory pathway. Ocugen’s patented OcuNanoE™ technology is designed to enhance efficacy by prolonging retention of this potent anti-inflammatory drug on the eye surface. In addition, it allows OCU300 to be sterile filtered into single-use vials as preservative-free nanoemulsion, thereby eliminating potentially irritating effects of preservatives.