ALAMEDA, Calif.--(BUSINESS WIRE)--BioTime, Inc. (NYSE MKT: BTX), a clinical-stage biotechnology company developing and commercializing products addressing degenerative diseases, today announced that the Data Safety Monitoring Board (DSMB) has authorized the Company to move forward with enrollment for cohort 3. The DSMB is an independent group of medical experts closely monitoring the Phase I/IIa OpRegen® clinical trial. In cohort 3 BioTime plans to treat patients at its current sites in Israel and at two US sites, with leading ophthalmologists: Dr. David S. Boyer, Retina-Vitreous Associates Medical Group and Dr. H. Richard McDonald, West Coast Retina Medical Group.
The administration of the implant in cohort 3 is being optimized for cell concentration and volume prior to Cohort 4. In cohort 4, the Company plans to treat patients in earlier stages of the disease that are likely be the target patient population for the therapy.
“Our team is enthusiastic about the early data showing a positive response from the implanted cells,” commented Dr. McDonald. “The DSMB’s approval is an important milestone. We look forward to enrolling our first patient in the trial as this is a debilitating disease."
“We are seeing encouraging signals of biological activity in the early stages of the OpRegen clinical program and we look forward to the continued progression of the trial,” commented Adi Mohanty, co-Chief Executive Officer. “We will start enrolling patients in cohort 3 in the coming weeks in both Israel and the U.S. Our goal is to complete this cohort during the fourth quarter of 2017.”
The Phase I/IIa clinical trial is evaluating the safety and efficacy of implantation regimens of OpRegen in the advanced form of dry-AMD. Dry-AMD is the leading cause of blindness in people over 60, and a condition for which there is currently no FDA-approved therapy. OpRegen has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the treatment of dry-AMD, which comprises approximately 90% of those afflicted with AMD.
The Company presented data from the Phase I/IIa clinical trial of OpRegen at the Association for Research in Vision and Ophthalmology (ARVO) annual meeting in May. The presentation reported clinical trial data from patients in the first two cohorts. Imaging analysis suggests the transplanted OpRegen cells remained in place (engrafted) even at the one year follow up and particularly uniquely in an area of the scar that was completely depleted of retinal pigment epithelium (RPE) because of the advanced stages of the disease. There was also possible evidence of a biological response with some areas appearing to show structural improvement (a thickening of the area of the neural retina above the scar) without any signs of retinal edema, a fluid build-up that can further compromise vision.
OpRegen®, which is being studied for the treatment of the dry form of age-related macular degeneration (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 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 Dry Age-Related Macular Degeneration (Dry-AMD)
Macular degeneration affects approximately 11 million people in the U.S. and is the leading cause of blindness in people over the age of 60. Approximately 90 percent of these patients suffer from the dry form, for which there are no FDA-approved therapies. In dry-AMD, there is a loss or dysfunction of the layer of retinal pigment epithelial (RPE) cells generally in the region of the eye called the macula, which is the part of the retina responsible for sharp, central vision that is important for facial recognition, reading and driving. These RPE cells support the light detecting photoreceptor cells that are so critical to vision. When we look at something, the photoreceptors (rods and cones) detect the light and send the information to the brain allowing us to perceive our surroundings. The age-dependent loss of the RPE cells therefore leads to degeneration of nearby photoreceptors and this can lead to severe vision loss or even legal blindness. Generally, the damage caused by the “dry” form is not as severe or rapid as that of the “wet” form. However, in the advanced stage of dry macular degeneration widespread loss of RPE and photoreceptors in the macular area, called geographic atrophy, leads to severe vision loss. While therapeutics are available to treat the wet form of AMD, there are currently no FDA-approved therapies for dry-AMD.
BioTime, Inc. is a clinical-stage biotechnology company focused on developing and commercializing novel therapies developed from what the company believes to be the world’s premier collection of pluripotent cell assets. The foundation of BioTime’s core therapeutic technology platform is pluripotent cells that are capable of becoming any of the cell types in the human body. Pluripotent cells have potential application in many areas of medicine with large unmet patient needs, including various age-related degenerative diseases and degenerative conditions for which there presently are no cures. Unlike pharmaceuticals that require a molecular target, therapeutic strategies based on the use of pluripotent cells are generally aimed at regenerating or replacing affected cells and tissues, and therefore may have broader applicability than pharmaceutical products. BioTime also has significant equity holdings in two publicly traded companies, Asterias Biotherapeutics, Inc. and OncoCyte Corporation, which BioTime founded and which, until recently, were majority-owned consolidated subsidiaries of BioTime.
BioTime common stock is traded on the NYSE MKT and TASE under the symbol BTX. For more information, please visit www.biotimeinc.com or connect with the company on Twitter, LinkedIn, Facebook, YouTube, and Google+.
Certain statements contained in this release are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Statements pertaining to future financial and/or operating results, future growth in research, technology, clinical development, and potential opportunities for BioTime, Inc. and its subsidiaries, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as “will,” “believes,” “plans,” “anticipates,” “expects,” “estimates” should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of BioTime, Inc. and its subsidiaries, particularly those mentioned in the cautionary statements found in more detail in the “Risk Factors” section of its Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q filed with the SEC (copies of which may be obtained at www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. BioTime specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.
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