Lineage Cell Therapeutics, Inc, announced today that Genentech, its partner, has launched a phase 2a, multicenter, open-label, single arm clinical study of RG6501 (OpRegen), a retinal pigment epithelial cell therapy.
The study intends to optimize subretinal surgical delivery and evaluate the safety and activity of OpRegen in approximately 30, and up to 60 patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
The study’s primary objectives include evaluating: (i) the proportion of patients with subretinal surgical delivery of OpRegen to target regions under the retina, and (ii) to evaluate the safety of subretinal surgical delivery of OpRegen as measured by the incidence and severity of procedure-related adverse events at 3 months following surgery.
A key secondary objective is to evaluate the proportion of patients with qualitative improvement in retinal structure, as determined by optical coherence tomography (SD-OCT) imaging, within 3 months following surgery.
RG6501 (OpRegen) is currently being developed under an exclusive worldwide collaboration between Lineage, Roche, and Genentech.
“In our Phase 1/2a clinical trial, RG6501 (OpRegen) demonstrated the potential to slow, stop, or reverse the progression of GA in patients with GA. These results can be maintained beyond one year following a one-time, approximately 30-minute outpatient procedure”
According to Lineage CEO Brian M. Culley, the phase 1/2a clinical trial of RG6501 demonstrated the potential to slow, stop, or reverse the progression of GA in patients with AMD.
“These results can be maintained beyond one year following a one-time, approximately 30-minute outpatient procedure,” Culley stated in a news release.
The OpRegen program’s larger clinical study will seek to optimize and potentially improve its delivery, as well as confirm the safety and activity of RG6501’s use in GA patients, Culley added. “We expect the findings from this phase 2a study will be highly informative and may increase the probability of success in any future larger, comparative trials,” he stated.
About the phase 2a study
The planned phase 2a clinical study is a multicenter, open-label, single arm study to optimize subretinal surgical delivery and to evaluate the safety and activity of RG6501 in patients with GA secondary to AMD. Approximately 30 and up to 60 patients may be enrolled across multiple sites and will receive RG6501 administered as a single subretinal injection to one eye with impaired vision. Study treatment will consist of a single subretinal injection of RG6501 at a dose of up to approximately 200,000 cells delivered to target areas of GA in the study eye.
The primary objectives of the study are to evaluate (i) the proportion of patients with subretinal surgical delivery of RG6501 to target regions under the retina, and (ii) to evaluate the safety of subretinal surgical delivery of RG6501 as measured by the incidence and severity of procedure-related adverse events at 3 months following surgery. A key secondary objective is to evaluate the proportion of patients with qualitative improvement in retinal structure, as determined by SD-OCT imaging within 3 months following surgery.