Lineage Cell Therapeutics announced that it has restarted patient enrollment in a phase 1/2a clinical study of its lead product candidate, OpRegen, a retinal pigment epithelium (RPE) cell transplant therapy currently in development for the treatment of dry age-related macular degeneration (AMD), following a temporary pause in enrollment as a result of the COVID-19 pandemic.
The first patient dosed in the re-start was at Cincinnati Eye Institute (CEI) under the direction of principal investigator Christopher D. Riemann, MD, Vitreoretinal Surgeon and Fellowship Director, CEI and University of Cincinnati School of Medicine. CEI is a global leader in ophthalmology research and a clinical site where local health and safety regulations recently enabled and cleared clinical study recruitment and treatment. The patient enrolled at CEI was administered Lineage’s new thaw-and-inject formulation of OpRegen with Gyroscope Therapeutics’ Orbit Subretinal Delivery System (SDS). The company anticipates that it will complete enrollment of the last 3 patients in Cohort 4 of the OpRegen clinical study in the third quarter of 2020.
“OpRegen is a promising candidate for the future treatment of dry AMD and I am excited to have dosed the first patient at our institute,” Christopher D. Riemann, MD, said in a company news release. “Delivering OpRegen RPE cells to the subretinal space using the Orbit SDS was straightforward and appears to offer superior dose control and safety compared to traditional delivery methods. We have resumed our patient screening efforts and I look forward to enrolling additional patients into the study as soon as possible.”
“We are pleased to safely resume patient enrollment in our OpRegen study under the direction of Dr. Riemann at CEI,” Brian M. Culley, Lineage CEO, said in the news release. “Data collected from the OpRegen program continues to support the use of our cells in dry AMD with GA, particularly after we moved into treating patients with less advanced disease. Additionally, we recently announced the first known finding of retinal tissue regeneration alongside improvements in the progression of geographic atrophy, visual acuity, and reading speeds in some patients. With enrollment once again underway, our immediate objective is to treat and monitor the final three patients in Cohort 4 of the study and utilize these data to direct our clinical, regulatory, and partnership discussions. Our goal is to combine the best cell line, the best production process, and the best delivery system, to position OpRegen as the front-runner in the race to address the unmet need in the potential billion-dollar dry AMD market.”
Recently, Lineage reported the first known finding of retinal tissue regeneration in a patient receiving OpRegen for the treatment of atrophic dry AMD. This finding supports the view that dry AMD is not an irreversible, degenerative condition and that some portion of diseased retinal tissue may be recoverable in atrophic end-stage disease patients. The loss of RPE cells over time creates progressively larger areas of geographic atrophy (GA) in the adult retina, leading to impaired vision or complete blindness. Humans lack the innate ability to regenerate retinal tissue and replace lost retina cells, which has led to a presumption that progression of GA may someday be slowed or halted but cannot be reversed. The unique finding from the ongoing OpRegen clinical trial supports a different view, in which an RPE cell transplant can potentially replace or rescue retinal cells in patients who suffer from retinal lesions or degeneration. Lineage has reported evidence from a patient with atrophic end-stage disease who received a transplant of allogeneic RPE cells and showed substantial restoration of retinal tissue within the area of GA. Specifically, the area of GA assessed at 9 months was approximately 25% smaller than the patient’s pre-treatment baseline. These findings were initially observed by an independent external advisor using multiple imaging technologies and were subsequently confirmed by the reading center and additional experts in the field of retinal imaging.
The company has also observed evidence of benefit in some patients, including increases in best corrected visual acuity (BCVA), reduction in the growth of GA and increases in reading speed. The addition of signs of retinal tissue regeneration provides further support that OpRegen may be a viable treatment for the millions of individuals living with dry AMD, one of the leading causes of vision loss in the world.