Genentech starts phase 2 trial to optimize delivery of eye disease cell therapy

Genentech has kicked off a phase 2a clinical trial to optimize subretinal surgical delivery of OpRegen, a cell therapy that is in development as a treatment for the eye disease geographic atrophy.

Roche’s Genentech paid Lineage Cell Therapeutics $50 million upfront for worldwide rights to the retinal pigment epithelial cell therapy late last year. A phase 1/2a study recently provided preliminary evidence of the efficacy of the candidate, linking it to improved vision in people with geographic atrophy. The data led Genentech to move the program into phase 2a.

Participants in the new clinical trial will receive up to around 200,000 cells into their subretinal spaces. By testing the therapy in between 30 and 60 people with geographic atrophy secondary to age-related macular degeneration, Genentech plans to optimize subretinal surgical delivery.

In a statement, Lineage CEO Brian Culley said that by advancing OpRegen into a larger clinical study Genentech “will seek not only to optimize and potentially improve its delivery, but also confirm safety and activity of OpRegen in this patient population.” Culley expects the phase 2a data to “be highly informative and may increase the probability of success in any future larger, comparative trials.” 

Genentech has established co-primary endpoints for the study. Specifically, the researchers are focusing on the proportion of participants with subretinal surgical delivery of OpRegen to target regions and the incidence and severity of procedure-related adverse events at three months following surgery. 

The study is the first Genentech-sponsored clinical trial of OpRegen. As the candidate advances, Lineage could receive up to $620 million in development, approval and sales milestones tied to the deal.