Ocugen announced that it has initiated the first of two pivotal, phase 3 clinical trials of OCU300 for the treatment of ocular discomfort and ocular redness in patients with ocular graft versus host disease (oGVHD) following recent acceptance of Ocugen’s investigational new drug (IND) application by the FDA.
OCU300 consists of FDA-approved brimonidine tartrate formulated in a proprietary nanoemulsion based on Ocugen’s patented OcuNanoE technology. Ocugen is the first and only company to advance a therapy into a phase 3 clinical trial for treating patients with oGVHD and OCU300 is the only product candidate to be granted Orphan Drug Designation for oGVHD by the FDA.
oGVHD is a severe ophthalmic comorbidity that affects about 60% of patients who undergo allogeneic bone marrow transplant, yet there is currently no approved treatment. It is a chronic and debilitating autoimmune condition characterized by painful, dry, tearless eyes, vision issues and potential ocular scarring. The proof of concept for using brimonidine to treat oGVHD was established by two previous investigator-led clinical studies. In an exploratory observational study, approximately 90% of oGVHD patients treated for 6 months with brimonidine eye drops reported an improvement in their symptoms without any significant side effects. In a randomized, placebo-controlled, double-masked phase 1/2 study, oGVHD patients using brimonidine eye drops had a significant reduction in ocular redness scores (P<0.05) compared to patients using placebo, as well as a clinically meaningful reduction in ocular discomfort after only three months of treatment.
“We are excited to advance OCU300 into pivotal-stage clinical development and to potentially deliver to oGVHD patients the first FDA-approved therapy to address this condition,” Daniel Jorgensen, MD, MPH, Chief Medical Officer of Ocugen, said in a company news release. “The preclinical efficacy data we presented last month at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting further highlight the ability of OCU300, in particular its novel nanoemulsion formulation, to inhibit the underlying pathophysiological processes associated with oGVHD. We believe OCU300 has significant potential to improve the quality of life of patients living with this debilitating disease.”
Shankar Musunuri, PhD, MBA, Chairman, CEO and Co-Founder of Ocugen, commented, “This is a significant milestone for Ocugen and I would like to thank our dedicated team and our participating clinical sites for their work, which has enabled us to reach this stage and commence Phase 3 development in a highly efficient manner. As we begin enrolling oGVHD patients in this study, we look forward to continuing that efficiency and reporting top-line data as soon as it is available, which we currently expect to be in the second half of 2019. We also expect to launch pivotal studies for our second lead candidate, OCU310 for the treatment of dry eye disease, later this year.”