Ocugen's OCU100 gets FDA orphan drug designation for treatment of retinitis pigmentosa

Ocugen, Inc. and the University of Colorado today announced exclusive license agreements that allow for Ocugen to continue developing two drug candidates for the treatment for ophthalmology indications, and that one of the assets, OCU100, recombinant lens epithelium derived growth factor 1-326 (LEDGF1-326), received orphan-drug status from the U.S. Food and Drug Administration for treatment of retinitis pigmentosa (RP), a rare eye disease.

"Orphan drug designation from the FDA's Office of Orphan Products Development is a significant milestone that will allow Ocugen to accelerate the clinical development of OCU100, which has the potential to be the first approved therapeutic for retinitis pigmentosa," said Shankar Musunuri, PhD, MBA, founder and chairman of the Ocugen Board of Directors.

Ocugen scientific founder and board member Uday Kompella, PhD, a professor of Pharmaceutical Sciences, Ophthalmology and Bioengineering at CU's Anschutz Medical Campus, is the inventor of OCU100. Ocugen licensed all assets related to LEDGF, including LEDGF1-326 and OCU200, an anti-angiogenic tumstatin fusion protein, to be developed for treatment of wet age-related macular degeneration (AMD) from the University of Colorado in March.

Retinitis pigmentosa is a rare eye disease caused by inherited gene mutations that lead to retinal degeneration affecting approximately 100,000 people in the United States, according to the Foundation Fighting Blindness. People with RP experience a gradual decline in their vision because photoreceptor cells in the retina die. It is a progressive disorder, and most people with RP are legally blind by age 40. There is no FDA approved therapeutic for RP.

"OCU100 has shown the potential as a promising therapeutic agent for treating retinitis pigmentosa by reducing protein aggregation and associated cellular stresses, which are known to contribute to this condition," said Dr. Kompella. "With impressive preclinical data, we look forward to progressing with a phase 1 study for safety and tolerability in patients sometime in 2015."

Dr. Kompella said a variety of mutations, including P23H mutation in rhodopsin, a critical protein in the retina that is responsible for vision, have been linked to the development of RP. P23H rhodopsin is known to form large clusters or aggregates within retinal cells, leading to cellular stress and ultimately cell death. OCU100 with its ability to rescue retinal cells from protein aggregation and associated stresses, has the potential to revolutionize the treatment of RP.0

SOURCE Ocugen, Inc.