BioTime, Inc. (NYSE MKT and TASE: BTX), a clinical stage biotechnology company with a focus on pluripotent stem cell technologies, today reported that data from the first patient cohort of the Phase I/IIa clinical trial of OpRegen® in the advanced form of dry age-related macular degeneration (dry-AMD) will be presented at the International Symposium on Ocular Pharmacology and Therapeutics (ISOPT) on Friday, December 2, 2016, in Rome, Italy.
The Phase I/IIa clinical trial, which is being conducted by BioTime’s subsidiary, Cell Cure Neurosciences Ltd., is evaluating the safety of three different dose regimens of OpRegen in the advanced form of dry-AMD that is accompanied by geographic atrophy. The first Phase I/IIa patient cohort received an initial targeted dose of 50,000 cells.
“The primary focus of this cohort is safety. OpRegen was successfully administered with no serious adverse events,” commented Prof. Eyal Banin, Director of the Center for Retinal and Macular Degenerations at Hadassah Medical Center, where the trial is being conducted. “Importantly, retinal imaging suggests that OpRegen RPE cells are able to engraft. Imaging from the first patient, who just completed one-year of post-treatment clinical assessment, may indicate that the graft can survive for at least 12 months. We are encouraged by these data and our continued progress in this important trial.”
“OpRegen RPE cells were created without the use of animal-derived products through a proprietary directed differentiation process to produce a highly purified population of RPE cells,” said Prof. Benjamin Reubinoff, Chief Scientific Officer of Cell Cure Neurosciences Ltd. and director of Hadassah's Stem Cell Research Center. “We are pleased that OpRegen did not cause serious adverse events in the first cohort of patients.”
The safety profile of the first cohort of subjects was thoroughly assessed by the Data Safety Monitoring Board (DSMB), an independent group of physicians and medical experts closely monitoring the clinical trial, before it gave its recommendation that the company continue the trial with the second cohort at a higher dose of 200,000 cells. The company expects a similar review by the DSMB at the end of the second cohort.
“We are looking forward to the opportunity to present data from the first patient cohort at ISOPT,” said Adi Mohanty, Co-Chief Executive Officer of BioTime. “OpRegen’s progress in the clinic is gaining momentum and we have already treated patients with 200,000 cell doses. It is in these higher cell dose cohorts where we believe OpRegen has the potential to demonstrate more meaningful clinical outcomes and we expect to start reporting on these data in early 2017. Our goal is to develop a treatment that can serve the millions of dry-AMD patients for whom there are currently no FDA-approved therapies.”
Enrollment in the second cohort is expected to be completed in 2016. Depending on the outcome of the DSMB’s review of the second patient cohort, approval to begin administering the 500,000 cell dosage to the third patient cohort could be provided by the end of the current year. The first OpRegen clinical trial site in the United States is expected to be selected in the near future.
OpRegen has received Fast Track designation from the FDA for treatment of the advanced form of dry-AMD. Details of the trial and about a patient’s eligibility are available at https://clinicaltrials.gov/ with the following Identifier: NCT02286089 (dry-AMD).
About the ISOPT Clinical Symposium
The ISOPT Clinical Symposium is an annual symposium focusing on clinical drug treatments in ophthalmology via a pragmatic clinical angle. Symposium participants are clinicians, clinical investigators, academy based researchers and members of the industry. The ISOPT Clinical board has two major missions: 1) To provide updates on current paradigms of therapy for common ophthalmic diseases with profound risk / benefit coverage, and 2) To assess expected therapeutic paradigm shifts in the near future as reflected in current clinical research. The ISOPT Clinical meeting will focus on case presentations as they demonstrate current treatment options. This year’s symposium will take place December 1-3, 2016, in Rome, Italy. For more information please go to http://www.isoptclinical.com/home.ehtml.
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.
About OpRegen ®
OpRegen consists of RPE cells that are produced using a proprietary process that drives the differentiation of human embryonic stem cells into high purity RPE cells. OpRegen is also “xeno-free," meaning that no animal products were used either in the derivation and expansion of the human embryonic stem cells or in the directed differentiation process. The avoidance of the use of animal products eliminates some safety concerns. OpRegen is formulated as a suspension of RPE cells. Preclinical studies in rats have shown that following a single subretinal injection of OpRegen, as a suspension of cells, the cells can rapidly organize into its natural monolayer structure 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® is a registered trademark of Cell Cure Neurosciences Ltd., a majority-owned subsidiary of BioTime, Inc.
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.
In addition to the development of therapeutics, BioTime’s research and other activities have resulted, over time, in the creation of other subsidiaries that address other non-therapeutic market opportunities such as cancer diagnostics, drug development and cell research products, and mobile health software applications.
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+.
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Statements pertaining to future financial and/or operating results, future growth in research, technology, clinical development, and potential opportunities for BioTime 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 and its subsidiaries, particularly those mentioned in the cautionary statements found in BioTime's Securities and Exchange Commission filings. BioTime disclaims any intent or obligation to update these forward-looking statements.
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Last updated on: 20/10/2016
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