Ocata Therapeutics Inc. (NASDAQ:OCAT), based in Marlborough, Massachusetts, and formerly known as Advanced Cell Technology Inc., or ACT, have announced the enrollment of their first patient in a Phase II clinical trial of proprietary RPE cells for the treatment of dry age-related macular degeneration (dry AMD). The results of the first cohort are expected to be released within the second quarter of FY2016. The company have stated that their main objective is to assess safety and efficacy compared to a parallel control group.
The US-based company, with a market capitalization of approximately $180M, is one of the key commercial entities at the coalface of developing stem cell technologies for the treatment of human diseases. The company’s primary focus on ophthalmic conditions includes Stargardt’s, dry AMD, myopic macular degeneration, retinitis pigmentosa, glaucoma and corneal blindness. The application of stem cell treatments to human disorders is a highly active field with both commercial and research scientist / clinician led trials seeking to demonstrate robust proof-of-concept.
Formally entitled, “A Phase I/II, Open-Label, Multi-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (MA09-hRPE) Cells in Patients With Advanced Dry AMD”, the study is recruiting participants at four US sites in Los Angeles, Miami, Boston and Philadelphia. The trial consists of 5 cohorts, comprising 16 patients in total. Four low vision cohorts will contain 3 patients each while a single “better vision” cohort will contain 4 patients. Each patient will receive between 50,000 and 200,000 MA09-hRPE cells and each cohort will be enrolled sequentially with each patient under-going independent review through-out the course of the trial. The cells used for treatment, MA09-hRPE cells, are human embryonic stem cell-derived retinal pigment epithelium (RPE) cells and have been used in a number of clinical studies in both the US and UK, including testing of the treatment in patients of Stargardt’s Macular Dystrophy. The MA09-hRPE cells are understood to have has Orphan Drug designation in the EU and US for Stargardt’s.
Commenting on the clinical milestone, Prof. Carl D. Regillo, MD, FACS, Director, Retina Service at Wills Eye Hospital and Professor of Ophthalmology, Jefferson Medical College stated, “We expect this Phase 2 clinical study will provide important information about the ability of Ocata’s fully differentiated RPE cells to treat dry age-related macular degeneration, when transplanted into the retina. We are pleased to be a participating site in this clinical trial, as we believe that Ocata’s RPE program has the potential to be an effective treatment option for dry AMD, as well as other ophthalmological indications, such as Stargardt’s Disease.”