– Safety Confirmed with No Concerns Reported in Cohort 1 –
– Approval to Immediately Proceed to Cohort 2 –
ALAMEDA, Calif. & JERUSALEM–(BUSINESS WIRE)–Jun. 13, 2016– BioTime, Inc. (NYSE MKT and TASE: BTX) and Cell Cure Neurosciences Ltd. (Cell Cure), a clinical-stage regenerative medicine company with a focus on pluripotent stem cell technology, today announced that the Data Safety Monitoring Board (DSMB), an independent group of medical experts closely monitoring the clinical trial, has reviewed the initial safety data from the first cohort in the Phase I/IIa clinical trial of OpRegen®. OpRegen® is comprised of retinal pigment epithelial cells designed for the treatment of the advanced form of dry age-related macular degeneration (dry-AMD). The DSMB has authorized the OpRegen® clinical trial to move forward with enrollment and dose escalation to the second cohort. Recruitment for the second cohort will begin immediately, with patients receiving a higher, more clinically significant dose of 200,000 cells of OpRegen®.
“The positive DSMB recommendation confirms the acceptable safety profile of OpRegen® in the initial low-dose cohort, and allows us to proceed with the second cohort,” said Adi Mohanty, Co-Chief Executive Officer of BioTime. “The escalation of dosing in this Phase I/IIa trial is a significant achievement for our OpRegen® cell therapy program, and we will now begin to evaluate the higher cell doses that we believe will have the most likely potential and favorable outcomes to benefit patients.”
The Phase I/IIa clinical trial is evaluating the safety and efficacy of three different dose regimens of OpRegen® in the advanced form of dry-AMD, the leading cause of blindness in an aging population over the age of 60, and a condition for which there is currently no FDA-approved therapy. The first cohort was successfully dosed earlier this year. Enrollment in the second cohort is expected to be completed in 2016 and, if the data are positive, it is anticipated that DSMB approval to proceed to the third cohort will be received by the end of 2016. OpRegen® has received Fast Track designation from theU.S. Food and Drug Administration (FDA) for the treatment of dry-AMD, which occurs in approximately 90% of those afflicted with AMD. Preclinical data demonstrated that OpRegen® preserved vision and retinal structure when it was transplanted into the leading animal model of retinal disease, and was presented at ARVO last year.
“The successful completion of the DSMB’s review of the first dose escalation cohort represents an important milestone in the clinical development of OpRegen®,” said Dr. Eyal Banin, Director of the Hadassah Center for Retinal and Macular Degenerations, Department of Ophthalmology, Hadassah-Hebrew University Medical Center, a key developer of the technology and a leading investigator of the OpRegen® trial.
Details of the trial and about a patient’s eligibility are available at https://clinicaltrials.gov/, with the following Identifier: NCT02286089 (dry AMD).
About Dry Age-Related Macular Degeneration (Dry–AMD)
Dry-AMD is the most common type of macular degeneration and affects approximately 90% of people with the disorder. 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 blindness. Generally, the damage caused by the “dry” form is not as severe or rapid as that of the “wet” form. However, over time, it can cause profound vision loss. The more advanced stage of dry macular degeneration is called geographic atrophy. While there are therapeutics available to treat the wet form of AMD, there are currently no FDA-approved therapies for dry-AMD.
OpRegen® consists of retinal pigment epithelial (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 mice 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.
About Cell Cure Neurosciences Ltd.
Established in 2005, Cell Cure is located in Jerusalem, Israel on the campus of Hadassah Medical Center. Cell Cure’s mission is to become a leading supplier of human cell-based therapies for the treatment of retinal and neural degenerative diseases. Its technology platform is based on the manufacture of diverse cell products sourced from clinical-grade (GMP-compatible) human embryonic stem cells. Its current focus is the development of retinal pigment epithelial (RPE) cells for the treatment of age-related macular degeneration. Cell Cure’s major shareholders include BioTime, Inc.(NYSE MKT and TASE: BTX), HBL Hadasit Bio-Holdings Ltd., and Teva Pharmaceuticals Industries Ltd. Additional information about Cell Cure can be found on the web at www.cellcureneurosciences.com.
BioTime, Inc. is a clinical-stage biotechnology company focused on developing and commercializing novel therapies developed from what we believe to be the world’s premier collection of pluripotent cell assets. The foundation of our 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 visitwww.biotimeinc.com or connect with the company on Twitter, LinkedIn, Facebook, YouTube, and Google+.
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. BioTimedisclaims any intent or obligation to update these forward-looking statements.
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Source: BioTime, Inc.
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