BioTime announced new data from the phase 1/2a clinical trial of OpRegen in the advanced form of dry age-related macular degeneration (AMD). The interim data were presented on May 8, 2017 at the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Baltimore by Eyal Banin, MD, PhD.
“We are encouraged by the results and look forward to the next steps in moving this therapeutic through the clinic and addressing the needs of the millions of patients with dry-AMD,” Mohanty, Co-Chief Executive Officer, said in a company news release.
The presentation at ARVO reported new clinical trial data with two patients that were treated in cohort 2, where they received a dose of 200,000 cells. Imaging analysis suggests the transplanted OpRegen cells remained in place (engrafted) in an area of the scar that was completely depleted of retinal pigment epithelium (RPE) because of the advanced stages of the disease. Cell engraftment occurred in four of the five patients treated thus far. There was also possible evidence of a biological response with some areas appearing to show structural improvement (a thickening of the thinned area of retina above the scar) without any signs of retinal edema, a fluid build-up that can further compromise vision.
“I am excited to see that the second cohort is showing evidence of cell survival and early signs suggesting a biological response,” David Boyer, MD, senior partner at Retina-Vitreous Associates Medical Group in Los Angeles and a principal investigator for the OpRegen trial, said in the news release. “I am looking forward to treating patients in the US under this trial protocol.”
Best corrected visual acuity (BCVA) remained stable in all treated eyes and importantly showed no signs of deterioration, which could be expected if no treatment was given. The data also continues to show that the procedure is well tolerated in all patients, including those patients with follow-up for more than 1 year after treatment.
“We continue to be encouraged by the lack of serious adverse events in the trial during both the surgical procedure required for cell transplantation and long-term post-operative monitoring,”Professor Eyal Banin, MD, PhD, Center for Hereditary Retinal and Macular Degeneration (CRMD) Department of Ophthalmology Hadassah-Hebrew University Medical Center, Jerusalem, Israel, said in the news release. “Thus far in the trial, we have not encountered any serious systemic side effects or unexpected severe ocular adverse events.”
OpRegen is the lead product of BioTime’s ophthalmology subsidiary Cell Cure Neurosciences Ltd., which has been conducting the clinical trial in Israel and will soon be opening additional trial sites in the United States. BioTime anticipates DSMB review of cohort 2 by the end of the second quarter of 2017 and if approved will then begin cohort 3 immediately after. Cohort 3 is expected to enroll more quickly due to the reduced stagger requirements between patients and the ability of new clinical trial sites in the U.S. to enroll patients. The Company also expects to share additional data from all patients before the end of the year.
OpRegen is an investigational therapy in which retinal pigment epithelial (RPE) cells are transplanted into the subretinal space, where they are intended to replace missing RPE cells. The ongoing trial is a Phase I/IIa dose-escalation study evaluating the safety and efficacy of three different dose regimens.
More information about the data presented at ARVO is available here.