BioTime, Inc. (NYSE American: BTX), a clinical-stage biotechnology company focused on addressing degenerative diseases, today announced that the independent Data Safety Monitoring Board (DSMB) has approved initiation of the fourth cohort for the ongoing OpRegen® clinical trial. The DSMB is an independent group of medical experts closely monitoring the Phase I/IIa OpRegen®clinical trial.
The approval was based on the continued safety observed throughout the first three cohorts. Although safety will remain the main focus of cohort 4, the fourth cohort will include earlier stage dry-AMD patients with better vision than the previous three cohorts. Cohort 4 will also include a wide range of preliminary functional assessments, such as best corrected visual acuity. These earlier stage patients, would likely be the target patient population for this therapy. BioTime expects to share initial data from cohort 4 in Q4 2018.
"This DSMB approval, which now includes enrollment of patients in earlier stages of dry-AMD, is an important milestone for the OpRegen study," said Adi Mohanty, Co-Chief Executive Officer at BioTime. "We are excited to move towards this expected target patient population and look forward to sharing data from cohort 4 later this year. We are happy with the continued progress of OpRegen, as dry-AMD is estimated to affect more than 25 million worldwide and is one of the leading causes of blindness in people over the age of 60."
The Company recently announced that interim data from the Phase I/IIa OpRegen®trial would be presented at the upcoming Association for Research in Vision and Ophthalmology meeting taking place from April 29th – May 3rd, 2018 in Honolulu, Hawaii. The poster presentation will take place on April 29th, 2018 from 8:15am – 10:00am local time at the Hawaii Convention Center.
About OpRegen®
OpRegen®, which is being studied for the treatment of the dry form of AMD, consists of a suspension of Retinal Pigment Epithelial (RPE) cells that are delivered subretinally during a simple intraocular injection. RPE cells are essential components of the back lining of the retina, and function to help nourish the retina including photoreceptors. A proprietary process that drives the differentiation of human pluripotent stem cells is used to generate high purity OpRegen® RPE cells. OpRegen® RPE cells are also "xeno-free," meaning that no animal products are used at any point in the derivation and production process. The avoidance of the use of animal products eliminates some potential safety concerns. Preclinical studies in rats have shown that following a single subretinal injection of OpRegen®, the cells can rapidly organize into its natural monolayer structure in the subretinal space and survive throughout the lifetime of the animal. OpRegen® is designed to be an "off-the-shelf" allogeneic (non-patient specific) product. Unlike treatments that require multiple, frequent injections into the eye, it is expected that OpRegen® would be administered in a single procedure. OpRegen® was granted Fast Track designation from the FDA, which allows more frequent interactions with the agency, and eligibility for accelerated approval and priority review. OpRegen® is a registered trademark of Cell Cure Neurosciences Ltd., a majority-owned subsidiary of BioTime, Inc.
About BioTime, Inc.
BioTime is a clinical-stage biotechnology company focused on degenerative diseases. Its clinical programs are based on two platform technologies: cell replacement and cell/drug delivery. With its cell replacement platform, BioTime is creating new cells and tissues with its proprietary pluripotent cell technologies. These cells and tissues are developed to replace those that are either rendered dysfunctional or lost due to degenerative diseases. BioTime’s cell/drug delivery programs are based upon its proprietary HyStem® cell and drug delivery matrix technology. HyStem® was designed to provide for the transfer, retention, engraftment and metabolic support of cellular replacement therapy. BioTime’s lead cell delivery clinical program, Renevia®, which consists of our proprietary HyStem® cell-transplantation delivery matrix combined with the patient's own adipose progenitor cells (Fat), met its primary endpoint in an EU pivotal clinical trial for the treatment of facial lipoatrophy in HIV patients in 2017. Submission for approval of Renevia® in the EU is expected to be early 2018, with possible approval in 2018. There were no device related serious adverse events reported to date. Our lead cell replacement clinical program, OpRegen®, which is a retinal pigment epithelium transplant therapy, is in a Phase I/IIa multicenter trial for the treatment of dry age-related macular degeneration, the leading cause of blindness in developing countries. There were no related serious adverse events reported to date. BioTime also has significant equity holdings in two publicly traded companies, Asterias Biotherapeutics, Inc. (NYSE American: AST) and OncoCyte Corporation (NYSE American: OCX), and a private company, AgeX Therapeutics, Inc.
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