Lineage Cell Therapeutics (NASDAQ, TASE:LCTX) reported that restoration of retinal tissue was observed in two additional patients enrolled in the company’s Phase 1/2a study of its lead product candidate, OpRegen.
OpRegen is an allogeneic retinal pigment epithelium cell transplant therapy in development for the treatment of age-related macular degeneration (AMD) with geographic atrophy, or dry atrophic AMD.
These new findings occurred in two of the three Cohort 4 patients treated in November 2020, where surgeons successfully covered the majority of the area of atrophy with a suspension of OpRegen cells.
In a statement, the company said outer retinal layer restoration, which was observed using clinical high-resolution optical coherence tomography, was evidenced by the presence of new areas of retinal pigment epithelium monolayer with overlying ellipsoid zone, external limiting membrane, and outer nuclear layer, “which were not present at the time of baseline assessment.”
The totality of these findings supports the view that “atrophic AMD is not an irreversible, degenerative condition and that some portion of diseased retinal tissue may be recoverable,” Lineage said.
In connection with these findings, Lineage has filed a patent cooperation treaty application, which describes restoration of the anatomy and/or functionality of diseased retinas. Allowed claims of a national patent application based on a patent cooperation treaty application would have an estimated expiration date of at least May 24, 2041.
Jordi Mores, M.D., principal investigator and founder of the Barcelona Macula Foundation, said that after reporting the first case of retinal restoration last year, “it is extremely exciting for me to see retinal restoration replicated in additional OpRegen-treated patients.”
Brian Culley, CEO of Lineage, said that “to our knowledge, these three patients represent the only examples of an experimental treatment for dry AMD demonstrating a reduction, rather than attenuating further expansion, of an area of atrophy in humans.”
Now that these findings of retinal restoration have been confirmed in multiple patients over clinically meaningful time periods, he said Lineage is in a “position to pioneer a new paradigm for how we and others evaluate and treat atrophic AMD.”