Phase 1/2 Clinical Trials of RPE Cells for the
Treatment of Dry Age-Related Macular Degeneration and Stargardt’s
Macular Degeneration Show Positive Long Term Safety Results and
Signs of Visual Improvement
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Advanced Cell Technology, Inc. ("ACT"; OTCBB: ACTC), a leader in
the field of regenerative ophthalmology, announced today that Phase
1/2 clinical data published online in The Lancet demonstrate
positive long-term safety results using ACT’s proprietary Retinal
Pigment Epithelium (RPE) cells for the treatment of Stargardt’s
macular degeneration (SMD) and dry age-related macular degeneration
(AMD). The publication features data from 18 U.S.-based patients
with at least six months of post-transplant follow-up.
“These study results represent an important milestone and
strengthen our leadership position in regenerative ophthalmology,”
said Paul K. Wotton, Ph.D., President and Chief Executive Officer.
“We would like to thank the patients for their willingness to
participate in these studies. Our findings underscore the potential
to repair or replace tissues damaged from diseases. We plan to
initiate comprehensive Phase 2 clinical trials for the treatment of
both AMD and SMD, two disease states where there is currently no
effective treatment.”
These two studies provide the first evidence of the mid- to
long-term safety, survival, and potential biologic activity of
pluripotent stem cell progeny into humans with any disease. In
addition to showing no adverse safety issues related to the
transplanted tissue, anatomic evidence confirmed successful
engraftment of the RPE cells, which included increased pigmentation
at the level of the RPE layer after transplantation in 13 of 18
patients.
Robert Lanza, M.D., Chief Scientific Officer of ACT and
co-senior author of the paper, commented, “Diseases affecting the
eye are attractive first-in-man applications for this type of
investigational therapy due to the immune-privileged nature of the
eye. Despite the degenerative nature of these diseases, the vision
of 10 of 18 patients showed measurable improvement at the six month
follow up, after transplantation of the RPE cells. Furthermore, the
cells have been well tolerated for a median period of 22 months
with two of the patients treated more than three years ago. We are
pleased that there have been no serious safety issues attributable
to the cells observed in any of the patients.”
Vision was measured using the widely accepted standard for
visual acuity testing, the Early Treatment Diabetic Retinopathy
Study (ETDRS) visual acuity exam.
Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at
the David Geffen School of Medicine at UCLA and retina division
chief at UCLA’s Jules Stein Eye Institute, principal investigator
and lead author of the publication said “The data published in The
Lancet support the potential safety and biological activity of stem
cell-derived retinal tissue. Once again, surgical access to the
subretinal space has proven safe. However, for the first time in
humans, terminally differentiated stem cell progeny seem to
survive, and do so without safety signals. Combined with the
functional signals observed, these data suggest that this
regenerative strategy should move forward. This is a hopeful and
exciting time for ophthalmology and regenerative medicine.”
About the Trials
The SMD and dry AMD trials are prospective, open-label studies
designed to evaluate the safety and tolerability of human embryonic
stem cell (hESC)-derived RPE cells following sub-retinal
transplantation into patients at 12 months, the studies’ primary
endpoint. Three dose cohorts were treated for each condition in an
ascending dosage format (50,000 cells, 100,000 cells, and 150,000
cells). Both the SMD and dry AMD patients had subretinal
transplantation of fully-differentiated RPE cells derived from
hESCs. In addition to the two clinical trials in the U.S., ACT is
carrying out a Phase 1/2 clinical trial of hESC-derived stem cells
for the treatment of SMD in the United Kingdom.
About Age-related Macular Degeneration
Age-related macular degeneration is the leading cause of vision
loss in people over the age of 50, with late stage AMD affecting
about 30 million people worldwide. Dry AMD, accounts for 90 percent
of all AMD and occurs when light-sensitive cells in the macula,
located in the center of the retina, slowly break down, causing
vision loss. As the disease progresses, patients may have
difficulty reading and recognizing faces. There is currently no
proven medical therapy for dry AMD. Including the earlier stages of
disease, the projected number of people worldwide with age-related
macular degeneration in 2020 is 196 million, increasing to 288
million in 2040 underscoring the urgent need for new
treatments.
About Stargardt’s Disease
Stargardt’s macular degeneration is a form of juvenile macular
degeneration that affects vision in children and young adults
between the ages of six and 20, with a prevalence of approximately
one in 10,000 people. Loss of vision is an inevitable aspect of
SMD, with more than half of patients experiencing vision loss in
the range of 20/200-20/400. Like dry AMD, there are no treatments
currently approved to prevent or slow the vision loss associated
with SMD.
About Advanced Cell Technology, Inc.
Advanced Cell Technology, Inc., (ACT) is a clinical stage
biotechnology company focused on the development and
commercialization of regenerative medicine and cell therapy
technology. ACT’s most advanced products are in clinical trials for
the treatment of dry age-related macular degeneration, Stargardt’s
macular degeneration and myopic macular degeneration. ACT’s
preclinical programs involve cell therapies for the treatment of
other ocular disorders and for diseases outside the field of
ophthalmology, including autoimmune, inflammatory and wound
healing-related disorders. ACT’s intellectual property portfolio
includes pluripotent stem cell platforms – hESC and induced
pluripotent stem cell (iPSC) – and other cell therapy research
programs. For more information, visit www.advancedcell.com
Forward-Looking Statements
All statements, other than historical facts, contained in this
news release, including statements regarding the relevance and
applicability of clinical trials, potential new applications of and
expanded indications covering ACT’s technology, the effect of ACT’s
products on the medical needs and quality of life of study subjects
or other patients, ACT’s potential product pipeline and development
efforts, and any other statements about ACT’s future expectations,
beliefs, goals, plans, results or prospects expressed by management
constitute forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995. Any statements
that are not statements of historical fact (including statements
containing the words “will,” “believes,” “plans,” “anticipates,”
“expects,” “estimates,” and similar expressions) should also be
considered to be forward-looking statements. There are a number of
important factors that could cause actual results or events to
differ materially from those indicated by such forward-looking
statements, including: the fact that ACT has no product revenue and
no products approved for marketing; ACT’s limited operating
history;, the need for and limited sources of future capital;
potential failures or delays in obtaining regulatory approval of
products; risks inherent in the development and commercialization
of potential products; reliance on new and unproven technology in
the development of products; the need to protect ACT’s intellectual
property; the challenges associated with conducting and enrolling
clinical trials; the risk that the results of clinical trials may
not support the Company’s drug candidate claims; even if approved,
the risk that physicians and patients may not accept or use ACT’s
products; ACT’s reliance on third parties to conduct its clinical
trials and to formulate and manufacture its product candidates; and
economic conditions generally. Additional information on potential
factors that could affect our results and other risks and
uncertainties are detailed from time to time in ACT’s periodic
reports, including the Quarterly Report on Form 10-Q for the three
and six months ended June 30, 2014. Forward-looking statements are
based on the beliefs, opinions, and expectations of ACT’s
management at the time they are made, and ACT does not assume any
obligation to update its forward-looking statements if those
beliefs, opinions, expectations, or other circumstances should
change. Forward-looking statements are based on the beliefs,
opinions, and expectations of ACT’s management at the time they are
made, and ACT does not assume any obligation to update its
forward-looking statements if those beliefs, opinions,
expectations, or other circumstances should change. There can be no
assurance that ACT’s future clinical trials will be successful or
that the results of previous clinical studies will lead to
commercialization or products or therapies .
Editor’s Note: Photos and b-roll are available on
request. Please contact David.Schull@russopartnersllc.com
Investors:Westwicke PartnersJohn Woolford,
443-213-0506john.woolford@westwicke.comorPress:Russo PartnersDavid
Schull, 858-717-2310david.schull@russopartnersllc.comorTony Russo,
Ph.D., 212-845-4251tony.russo@russopartnersllc.com