Ocugen said today that it launched the first of two pivotal Phase III trials for its OCU300 nanoemulsion designed to treat the symptoms of ocular graft versus host disease.
The Malvern, Penn.-based company’s drug is a nanoemulsion formulation of brimonidine tartrate made using Ocugen’s OcuNanoE technology.
Ocugen touted that it’s the first company to bring an oGVHD therapy into Phase III clinical development. oGVHD is an ophthalmic comorbidity that impacts 60% of people who have an allogeneic bone marrow transplant, according to Ocugen. The autoimmune condition is characterized by dry, tearless eyes, vision problems and ocular scarring.
In an observational study, nearly 90% of people with oGVHD who used Ocugen’s eye drops for six months reported an improvement in their symptoms without serious side effects.
The company also reported that in a Phase I/II trial, people using the brimonidine eye drops experienced a significant reduction in ocular redness compared to people using a placebo. Those in the OCU300 group also reported a clinically meaningful reduction in ocular discomfort after three months.
“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. The preclinical efficacy data we presented last month at the Association for Research in Vision and Ophthalmology 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,” CMO Dr. Daniel Jorgensen said in prepared remarks.
“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,” Ocugen CEO and co-founder, Shankar Musunuri, added. “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.”
