Lineage Cell Therapeutics a clinical-stage biotechnology company developing novel cellular therapies for unmet medical needs, today provided an update from its ongoing phase 1/2a clinical study of OpRegen, the company’s retinal pigment epithelium (RPE) transplant therapy, for the treatment of dry age-related macular degeneration (dry AMD). Dry AMD is a leading cause of adult blindness in the developed world with no FDA-approved treatment options.
Based on promising preliminary results from the ongoing clinical study, including using Lineage’s new thaw-and-inject formulation of OpRegen with Gyroscope Therapeutics’ Orbit Subretinal Delivery System (SDS), the Company has been informed by the study’s independent data safety monitoring board (DSMB) that the protocol-mandated treatment stagger can be removed. In addition, Lineage is opening two new clinical sites to accelerate patient enrollment and broaden surgical experience among dry AMD experts.
All five patients in Cohort 4, those with better baseline vision and which represent the company’s intended patient population, have shown increases in BCVA. Notably, the first Cohort 4 patient dosed with both the Company’s new thaw-and-inject formulation and Orbit subretinal delivery system, gained 25 readable letters (or 5 lines) at 6 months following administration of OpRegen RPE cells as assessed by the ETDRS. This positive outcome represents an improvement in visual acuity from 20/250 to 20/100 in the patient’s treated eye. There was no change in BCVA in the patient’s untreated eye. To date, improvements have become most evident approximately 3 to 6 months after treatment with improvements in BCVA lasting at least 15 months with longer timepoints still to be collected. Safety data and evidence of successful engraftment of transplanted RPE cells is available for as long as 3 years in some patients. Because no unexpected complications have been observed with the new formulation and delivery system, the study’s DSMB has removed the protocol-mandated enrollment stagger, which will permit Lineage to accelerate the rate of patient enrollment by opening additional clinical sites, as well as to treat patients with less advanced disease and smaller areas of geographic atrophy than patients in Cohort 1 through Cohort 3, which were legally blind due to more progressive disease.
The Company is opening two new clinical sites, Cincinnati Eye Institute and Wills Eye Hospital, both global leaders in ophthalmology research. Principal investigators at the new clinical sites are Christopher D. Riemann, MD, Vitreoretinal Surgeon and Fellowship Director, Cincinnati Eye Institute and University of Cincinnati School of Medicine; Clinical Governance Board, Cincinnati Eye Institute and Allen C. Ho, MD, FACS, Wills Eye Hospital Attending Surgeon and Director of Retina Research, Professor of Ophthalmology, Thomas Jefferson University. Both Drs. Riemann and Ho are renowned therapeutic area experts and have direct experience with the Orbit SDS as they helped to develop and refine the device.
“Having been part of the team that originally designed the subretinal delivery system, I am looking forward to delivering Lineage’s RPE cells to the subretinal space of patients suffering from dry AMD,” stated Christopher D. Riemann, MD. “I believe that this combination will provide superior dose control and safety and may lead to superior outcomes when compared to alternate approaches. I am encouraged by the results observed in patients treated to date with OpRegen and I look forward to participating in this study.”
“We continue to be excited about the data we are collecting in dry AMD, which includes safety and engraftment data persisting as long as 3 years from time of transplantation of our RPE cells. Additionally, we are honored by the interest our approach is garnering from retinal surgeons at leading ophthalmology research centers such as Cincinnati Eye and Wills Eye,” stated Brian M. Culley, CEO of Lineage. “With the removal of the enrollment stagger and clearance to dose patients with vision as good as 20/65, we believe we can significantly accelerate our rate of enrollment on the OpRegen study. Our objective is to complete patient enrollment in the Orbit SDS portion of our clinical study as soon as this quarter. The sooner we treat the next four patients, the more mature and informative our clinical update will be at the Association for Research in Vision and Ophthalmology (ARVO) medical conference, which we plan to attend in Baltimore this May. We believe that by combining the best cell line, the best production process, and the best delivery system, we are positioning OpRegen as the front-runner in the race to address the unmet opportunity in the potential billion-dollar dry AMD market.”
The ETDRS eye chart consists of a set of letters of diminishing size on each line. The more letters a patient can read, the better his or her vision. The Company also is collecting data on rate of geographic atrophy growth, BCVA, low-light visual acuity, reading speed, quality of life questionnaires, microperimetry, and assessing structural changes using optical coherence tomography, fundus autofluorescence, and color fundus photography.