Date: 2015-02-16
Type of information: Initiation of the trial
phase: 1-2a
Announcement: initiation of the trial
Company: Cell Cure Neurosciences (Israel) Biotime (USA - CA)
Product: OpRegen®
Action
mechanism: cell therapy. OpRegen® consists of retinal pigment epithelial cells (RPE) derived from human embryonic stem cells using a proprietary directed differentiation method. OpRegen® is formulated as a suspension of RPE cells. Preclinical studies in mice have shown that OpRegen® transplanted subretinally as a suspension of cells can rapidly organize into their natural monolayer structure and survive throughout the lifetime of the animal. OpRegen® will be an \"off-the-shelf\" allogeneic product provided to retinal surgeons in a final formulation ready for transplantation. Unlike treatments that require multiple injections into the eye, such as currently-marketed products like Lucentis and Eylea for wet-AMD, it is expected that OpRegen® will be administered in a single procedure.
Disease: geographic atrophy
Therapeutic area: Ophtalmological diseases
Country: Israel
Trial
details: Cell Cure's Phase I/IIa clinical trial is a dose escalation safety and preliminary efficacy study of hESC-derived Retinal Pigment Epithelial (RPE) cells transplanted subretinally in patients with geographic atrophy. The open-label, single center, nonrandomized trial will evaluate three different dose regimens of 50,000 to 500,000 cells. A total of 15 patients will be enrolled. The patients will be 55 years of age and older, with non-neovascular (dry-AMD) who have funduscopic findings of GA in the macula with absence of additional concomitant ocular disorders. The eye most affected by the disease will be treated with the contralateral eye being the control. Following transplantation, the patients will be followed for 12 months at specified intervals, to evaluate the safety and tolerability of OpRegen®. A secondary objective of the clinical trial will be to examine the ability of transplanted OpRegen® to engraft, survive, and induce changes in visual acuity. In addition to thorough characterization of visual function, a battery of defined ophthalmic imaging modalities will be used to quantify structural changes and rate of GA expansion. The study will be performed at Hadassah Ein Kerem Medical Center in Jerusalem, Israel. (NCT02286089)
Latest
news: * On February 17, 2015, BioTime and its subsidiary Cell Cure Neurosciences provided an update on Cell Cure's product development and partnering activities. On February 16, 2015 , Cell Cure opened the clinical trial of OpRegen® titled "Phase I/IIa Dose Escalation Safety and Efficacy Study of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelium Cells Transplanted Subretinally in Patients with Advanced Dry-Form Age-Related Macular Degeneration with Geographic Atrophy" at Hadassah University Medical Center in Jerusalem, Israel . Patient enrollment is expected to begin shortly. The Phase I/IIa clinical trial, will evaluate three different dose regimens of OpRegen®. Following transplantation, the patients will be followed for 12 months at specified intervals, to evaluate the safety and tolerability of the product. Following the initial 12 month period, patients will continue to be monitored at longer intervals for an additional period of time. A secondary objective of the clinical trial will be to examine the ability of transplanted OpRegen to engraft, survive, and moderate disease progression in the patients. In addition to thorough characterization of visual function, a battery of ophthalmic imaging modalities will be used to quantify structural changes and rate of GA expansion. Cell Cure also announced that the option granted to Teva Pharmaceutical Industries Ltd. ("Teva") under a Research and Exclusive Option Agreement of October 7, 2010 to license-in rights to its OpRegen® product has expired without having been exercised by Teva. Cell Cure will therefore be continuing the clinical development of OpRegen on its own and pursuing discussions with other potential strategic partners, including those that have already indicated interest in participating in development and commercialization of the product. * On November 3, 2014, BioTime and Cell Cure Neurosciences announced that the FDA has cleared Cell Cure's Investigational New Drug (IND) application to initiate a Phase I/IIa clinical trial of OpRegen® in patients with the severe form of age-related macular degeneration (AMD) called geographic atrophy (GA). Cell Cure will conduct the trial in Israel where OpRegen® will be transplanted as a single dose into the subretinal space of the eye to test the safety and efficacy of the product. Patient enrollment is expected to begin in 2014 following approval of the trial by the Israel Ministry of Health . * On October 6, 2014, BioTime, HBL Hadasit Bio-Holdings and Cell Cure Neurosciences announced that Cell Cure has filed an Investigational New Drug (IND) application with the FDA seeking to initiate a Phase I/IIa clinical trial of OpRegen® in patients with geographic atrophy. The design of the proposed clinical trial, \"Phase I/IIa Dose Escalation Safety and Efficacy Study of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelium Cells Transplanted Subretinally in Patients with Advanced Dry-Form Age-Related Macular Degeneration with Geographic Atrophy,\" is based on a pre-IND meeting with the FDA and a series of earlier interactions with the agency. Patients will undergo a single transplantation and the study will explore three different doses of OpRegen®. Following transplantation, the patients will be followed over 12 months at specified intervals and then at longer time periods, to evaluate the safety and tolerability of the product. A secondary objective of the clinical trial will be to explore the ability of transplanted OpRegen® to engraft, survive, and moderate the disease progression.
