Ocugen announced that it presented additional preclinical findings in a poster highlighting the potential efficacy of its unique, patented nanoemulsion formulation of brimonidine tartrate (OCU300) at the Association for Research in Vision and Ophthalmology (ARVO) 2018 Annual Meeting, held April 29 – May 3, 2018, in Honolulu, Hawaii. The results highlight superior (statistically significant) efficacy of OCU300 in a surrogate mouse model of ocular graft versus host disease (oGVHD), as compared to placebo, untreated control, and marketed brimonidine. OCU300 is in phase 3 clinical development for treating oGVHD and is the only product to be granted Orphan Drug Designation for this indication from the U.S. FDA.
“The data we presented yesterday clearly demonstrate the efficacy of OCU300 in inhibiting the underlying pathophysiological processes associated with oGVHD. The data also showed that OCU300 provides efficacy that was statistically significant compared to untreated and placebo controls, as well as to the current commercial formulation of brimonidine tartrate, which is approved for treating glaucoma as a standard ophthalmic solution,” Rasappa Arumugham, PhD, Chief Scientific Officer of Ocugen, said in a company news release.
“These encouraging results add to a growing body of evidence that our proprietary nanoemulsion formulation technology may confer enhanced drug properties and lead to improved outcomes for ophthalmic diseases. Therefore, we believe OCU300 is a strong candidate for the treatment of oGVHD that could become the first approved therapy for this significantly underserved ocular disease, which affects 40%-60% of allogeneic stem cell transplant patients. We look forward to initiating our phase 3 trial of OCU300 for the treatment of oGVHD later this quarter,” Dr. Arumugham added.
Daniel Jorgensen, MD, MPH, Chief Medical Officer of Ocugen, said, “Of all the drugs tested in this study, OCU300 was the only treatment that showed a favorable response in reducing corneal surface inflammation, improvement of lacrimal gland pathology and increased mucin producing goblet cells. We hope to demonstrate similar improvements in our upcoming clinical trials on patients with oGVHD.”