MARLBOROUGH, Mass. — Sept. 28, 2011 – Advanced Cell Technology, Inc. (“ACT”; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that the independent Data and Safety Monitoring Board (DSMB) overseeing the Company’s two ongoing stem cell clinical trials in the United States authorized ACT to move forward with enrolling and treating the next set of patients in each of the trials. In July, surgeons led by 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 treated the first patients in the Company’s clinical trials for Stargardt’s Macular Dystrophy (SMD) and Dry Age-Related Macular Degeneration (Dry AMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). Each patient received an injection of 50,000 hESC-derived RPE cells in one of their eyes. Both the Dry AMD and SMD clinical trials are dose-escalation studies involving twelve patients in each, and are designed to evaluate the safety and tolerability of the injected RPE cells. The study design involves four cohorts of three patients each being treated at a predetermined dose of RPE cells, ranging from 50,000 to 200,000 cells. Based on the results of the first patient in each study, the DSMB authorized the Company to move forward with the next two patients in the studies. Each of these additional patients will also be treated with 50,000 RPE cells.
“The unanimous recommendation of the DSMB to proceed with the next four patients represents a significant milestone for our clinical programs. We hope that the positive trend in clinical results derived from treating the first patients with our RPE cells will continue,” said Robert Lanza, M.D., chief scientific officer of ACT. “The DSMB approval is an important step towards confirming the safety of our cells. We hope that the treatment of the next patients will provide further evidence that our stem cell-derived RPE cells are a safe and effective treatment option for patients with a range of macular degenerative diseases such as Dry AMD and SMD.”
The progress of disease in both Stargardt’s Macular Dystrophy and Dry AMD includes atrophy or thinning of the layer of RPE cells in the patient’s macula at the center of the retina, the region specialized for high acuity vision. The progressive loss of RPE cells in the macula leads to the eventual loss of photoreceptors. Over time, this can cause severe central vision deterioration and even blindness as the macula becomes less functional. ACT’s therapeutic programs utilize transplanted hESC-derived RPE cells to treat these conditions by replacing the missing RPE cells in the patient’s eyes before all RPE function and photoreceptor activity is lost.
“The outcome of this in-depth safety review by an unbiased team of experts is very reassuring,” said Gary Rabin, chairman and CEO of ACT. “The DSMB, which includes independent experts in macular degeneration, has recommended that the study continue at the present dosage of cells. We hope that in the near future we will be able to share both safety and physiological data we are continuing to obtain in these trials. For now, it will have to suffice to say that we are extremely pleased and believe that these trials will validate our earlier pre-clinical findings. In the meantime, we have begun to set our sights on expanding our clinical trials to in Europe, having been recently approved to begin our SMD trial in the United Kingdom, and various countries in Asia. Our trials have captured the interest of some of the best eye hospitals and surgeons in the world, and it is our aim to bring that expertise to bear in our efforts to find treatment for these debilitating diseases.”
About hESC-RPE Cells
The retinal pigment epithelium (RPE) is a highly specialized tissue that is located between the choroids and the neural retina. RPE cells support, protect and provide nutrition for the light sensitive photoreceptors. Human embryonic stem cells (hESCs) can differentiate into any cell type, including RPE cells. hESC-RPE cells have a similar expression of RPE-specific genes compared to human RPE cells and demonstrate the full transition from the hESC state.
About SMD, Dry AMD and Degenerative Diseases of the Retina
Stargardt’s Macular Dystrophy (SMD) is one of the most common forms of macular degeneration in the world. SMD causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium or RPE cell layer.
Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world. As many as thirty million people in the United States and Europe suffer from macular degeneration, which represents a $25-30 billion worldwide market that has yet to be effectively addressed. Approximately 10% of people ages 66 to 74 will have symptoms of macular degeneration, the vast majority the “dry” form of AMD - which is currently untreatable. The prevalence increases to 30% in patients 75 to 85 years of age.
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.
About the Jules Stein Eye Institute
Established in 1966, the Jules Stein Eye Institute represents the culmination of a dream shared by ophthalmologist, businessman and philanthropist Dr. Jules Stein and his wife, Doris, of creating a world-renowned center dedicated to the preservation of vision and the prevention of blindness. The Institute’s comprehensive programs for the care of patients with eye disorders, research in the vision sciences, education in the field of ophthalmology and outreach to the community, coupled with its state-of-the art facilities, have brought national and international recognition to UCLA and the Institute, as it continues its mission to advance ophthalmology worldwide.
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, 2010. 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.
Martina Schwarzkopf, Ph.D.
Account Supervisor
Russo Partners, LLC
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martina.schwarzkopf@russopartnersllc.com
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