BUSINESS WIRE
Represents A Large Market Opportunity, With No Treatments
Currently Available
MARLBOROUGH, Mass.--(BUSINESS WIRE)--Advanced Cell Technology, Inc. (“ACT”; OTCBB:ACTC),
a leader in the field of regenerative medicine, announced today that the
US Food and Drug Administration (FDA) has cleared the Company’s
Investigational New Drug (IND) application to treat Dry Age-Related
Macular Degeneration (AMD) using retinal pigment epithelial (RPE) cells
derived from human embryonic stem cells (hESCs). ACT is now permitted to
initiate a Phase I/II multicenter clinical trial to treat patients with
Dry AMD, the most common form of macular degeneration in the world.
There are currently no treatments available for this prevalent disease
of an aging global population. Dry AMD, representing a substantial
global market opportunity and afflicts between 10-15 million Americans.
Age-Related Macular Degeneration has two predominant forms, wet and dry.
Dry AMD is the most common form, accounting for almost 90% of all cases.
The progress of Dry AMD includes a breakdown or thinning of the layer of
RPE cells in the patient’s macula, the region at the center of the
retina responsible for high acuity vision. Over time, the progressive
loss of RPE cells and accompanying loss of photoreceptors can cause
severe vision loss and even blindness.
“ACT is now the first company to receive FDA clearance for two hESC
trials, and is now a true translational leader in the field of
regenerative medicine,” said Gary Rabin, Interim Chairman and CEO of
ACT. “It marks a major step forward, not just within the stem cell
sector, but, potentially for modern healthcare techniques. We plan to
proceed into the clinic with both of our hESC-based programs as quickly
as possible. .”
The Phase I/II trial will be a prospective, open-label study that is
designed to determine the safety and tolerability of the RPE cells
following sub-retinal transplantation into patients with Dry AMD. Twelve
patients will be enrolled in the study at multiple clinical sites. Sites
currently under consideration are the Jules Stein Eye Institute at UCLA,
and the Ophthalmology Department at Stanford University School of
Medicine. Additional sites may be considered.
“Dry AMD is the leading cause of blindness in individuals over the age
of 55,” stated Robert Lanza, MD, ACT’s Chief Scientific Officer. “As the
population ages, the incidence of AMD is expected to double over the
next 20 years, further exacerbating this unmet medical need. Using our
clinical-grade hESC lines, we are able to generate a virtually unlimited
and reproducible supply of healthy RPE cells. Because only a small
number of cells (50-200K) are needed to treat each patient,
manufacturing and distribution of the therapeutic product is scalable
with many similarities to the drug businesses that pharmaceutical
companies understand well. Based on our animal model studies, we are
very excited about the opportunity to treat patients. In a rat model of
macular degeneration, we have seen a remarkable improvement in visual
performance over untreated animals, without any adverse effects. We have
also maintained near-normal function in a mouse model of Stargardt’s
Disease, a form of juvenile macular degeneration. In addition to this
trial, we plan to concurrently use our RPE cells in our Phase I/II
Clinical Trial for Stargardt’s Disease, which received the green light
from the FDA in November. We hope to see a similar benefit in both
Stargardt’s Disease and Dry AMD patients.”
ACT’s Dry AMD therapeutic program uses RPE cells derived from hESCs to
replace the lost RPE cells in the patient’s eyes. ACT’s proprietary RPE
cell manufacturing process is protected by a number of broad patents, as
is the use of hESC-derived RPE cells for treating macular degeneration.
While the initial portion of the clinical trial will focus on safety, in
subsequent clinical trials the Company hopes to demonstrate that the RPE
cells injected into the retinal space will be capable of slowing or
halting progression of the disease, and potentially even restoring some
visual acuity to patients.
“It is estimated that over ten million Europeans suffer from Age-Related
Macular Degeneration, representing a vast unmet need and a significant
market opportunity,” commented Edmund Mickunas, ACT’s Vice President of
Regulatory Affairs. “We are moving ahead aggressively to seek regulatory
clearance from the European Medicines Agency to conduct clinical trials
in Europe.”
Specific inclusion and exclusion requirements and Investigator contact
information will be posted shortly at http://clinicaltrials.gov.
Patients and their caregivers should refer to this source rather than
contacting ACT or its representatives.
About Advanced Cell Technology, Inc.
Advanced Cell Technology, Inc. is a biotechnology company applying
cellular technology in the field of regenerative medicine. For more
information, visit http://www.advancedcell.com.
Forward-Looking Statements
Statements in this news release regarding future financial and
operating results, future growth in research and development programs,
potential applications of our technology, opportunities for the company
and any other statements about the future expectations, beliefs, goals,
plans, 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: limited operating history, need for future
capital, risks inherent in the development and commercialization of
potential products, protection of our intellectual property, 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 the company’s periodic reports,
including the report on Form 10-K for the year ended December 31, 2009.
Forward-looking statements are based on the beliefs, opinions, and
expectations of the company’s management at the time they are made, and
the company 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 the company’s management at the
time they are made, and the company 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 the Company’s clinical trials will be successful.