BioTime’s Subsidiary Cell Cure Neurosciences Receives DSMB Approval to Start Second Patient Cohort in Clinical Trial for Dry-AMD
– Safety Confirmed with No Concerns Reported in Cohort 1 –
– Approval to Immediately Proceed to Cohort 2 –
ALAMEDA, Calif. & JERUSALEM–(BUSINESS WIRE)–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 the U.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.
About OpRegen®
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.
About BioTime
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 visit www.biotimeinc.com
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