BioTime announced that it has dosed its first patient with the Orbit Subretinal Delivery System (Orbit SDS) as well as with a new thaw-and-inject formulation of OpRegen, the company’s retinal pigment epithelium (RPE) transplant therapy, in its ongoing phase 1/2a clinical study for the treatment of dry age-related macular degeneration (AMD).
“We are excited to be evaluating the FDA-cleared Orbit SDS in the next six patients of our phase I/IIa study,” Brian M. Culley, Chief Executive Officer of BioTime, said in a company news release. “We believe that precise administration of OpRegen cells to the back of the eye utilizing the Orbit SDS will lead to better dose control and an overall safer procedure, with an increased likelihood of positive clinical outcomes. In preparation for potential future commercialization, we also are introducing our new thaw-and-inject formulation, enabling rapid off-the-shelf administration of RPE cells upon thawing, which we believe will streamline the use of OpRegen by retinal surgeons.”
The primary objective of the hase 1/2a study is to evaluate the safety and tolerability of OpRegen as assessed by the incidence and frequency of treatment emergent adverse events (AEs). Secondary objectives are to evaluate the preliminary efficacy of OpRegen treatment by assessing the changes in ophthalmological parameters measured by various methods of primary clinical relevance. Additionally, for the patients in Cohort 4 that receive subretinal delivery of OpRegen utilizing the Orbit SDS, objectives will include the evaluation of the safety of delivery of OpRegen using the Orbit SDS.
About the Phase 1/2a Clinical Study
This is a Phase 1/2a open-label, dose escalation safety and efficacy study of human embryonic stem cell-derived retinal pigment epithelium cells transplanted subretinally in patients with advanced dry age-related macular degeneration with geographic atrophy. The study will enroll approximately 24 subjects, divided into 4 cohorts. The first 2 cohorts, each consisting of 3 legally blind subjects with best corrected visual acuity (BCVA) of 20/200 or less, received a single subretinal implantation of OpRegen. The third cohort included 6 subjects with BCVA of 20/200 or less, who received a single subretinal implantation of OpRegen. Staggered intervals within and between cohorts are applied to ensure subject safety and welfare. The fourth cohort will include approximately 12 subjects with BCVA between 20/64 and 20/250, who will receive a single subretinal implantation of OpRegen. Cohort 4 includes two formulations of OpRegen; the first 3 subjects were treated with an ophthalmic Balanced Salt Solution Plus (BSS Plus) version of OpRegen. Remaining subjects will be treated with an “off-the-shelf” or “thaw and inject” (TAI) formulation. OpRegen TAI can be shipped directly to sites and used upon thawing, which removes the complications and logistics of having to use a dose preparation facility. Staggered intervals will be applied between at least the first two subjects of each delivery modality to ensure subject safety and welfare.