UCLA’s Investigator IND Application for Embryonic Stem Cell-Based Clinical Trial to Evaluate Safety and Tolerability in Patients with Severe Myopia, or Nearsightedness, Approved by FDA
MARLBOROUGH, Mass. — February 11, 2013 – Advanced Cell Technology, Inc.’s (“ACT”; OTCBB: ACTC or the “Company”), clinical partner, the University of California, Los Angeles (UCLA), has received approval of its Investigator Investigational New Drug (IND) Application with the US Food and Drug Administration (FDA), 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, to initiate a Phase I/II study using ACT’s retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs) to treat myopic macular degeneration (MMD, or myopia), commonly known as nearsightedness. The primary focus of the study will be to evaluate the safety in patients with severe myopia of the type that causes fissures in the RPE layer of the eye. Dr. Schwartz is the principal investigator in each of ACT’s two Phase I/II clinical trials for Stargardt’s macular dystrophy and dry age-related macular degeneration (dry AMD) using RPE cells derived from hESCs. The approval was announced by Dr. Schwartz in his presentation at Bascom Palmer Eye Institute’s tenth annual angiogenesis meeting, “Angiogenesis, Exudation, and Degeneration 2013,” on Saturday, February 9 in Miami, Fla.
“We are encouraged by Dr. Schwartz’s keen interest in the evaluation of ACT’s RPE cells in potentially treating myopia,” commented Gary Rabin, chairman and CEO of ACT. “We are pleased to be on track to broaden the scope of our RPE program with the initiation of the new Investigator IND. Nearsightedness is a very common condition, worldwide, and it is estimated that roughly 30% of cases are high, or severe, myopia.”
The myopia clinical trial will follow a similar protocol as the company’s three other human clinical trials in the U.S. and Europe using hESC-derived RPE cells to treat forms of macular degeneration. The trial will enroll a total of 12 patients, with cohorts of three patients in an ascending dosage format. The trial is a prospective, open-label study designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with myopia at 12 months, the study’s primary endpoint.
Preliminary results from the U.S. Stargardt’s and Dry Age Related Macular Degeneration trials were reported in The Lancet earlier this year.
“We look forward to initiating the clinical trial,” commented Dr. Schwartz. “Myopic degeneration is an increasingly important global cause of permanent central vision loss for which there is no accepted treatment. Applying our key learnings from the ongoing ACT-sponsored stem cell trials allows the research promise of regenerative medicine to include myopic vision loss.”
Robert Lanza, M.D., ACT’s chief scientific officer, commented, “Myopia is one of the most common medical ailments in the world and myopic patients have a higher risk of permanent vision loss due to complications such as fissures in the RPE layer of the eye. We are anticipating Dr. Schwartz’s evaluations, potentially leading to a treatment for those patients at high risk of vision loss from this condition.”
Advanced Cell Technology, Inc., is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit www.advancedcell.com.
Established in 1966, the Jules Stein Eye Institute at UCLA 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.
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, 2011. 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.
Investors:
CEOcast, Inc., James Young, 212-732-4300
Press:
ACT Corporate Communications, Bill Douglass, 646-450-3615
or:
Russo Partners, Martina Schwarzkopf, Ph.D., 212-845-4292
UCLA:
Jules Stein Eye Institute:
Elaine Schmidt, [email protected], 310-794-2272
Bill Douglass
646-450-3615
actandrsquo-s-clinical-partner-receives-fda-approval-to-initiate-clinical-trial-using-the-companyandrsquo-s-hesc-derived-cells-to-treat-severe-myopia.pdf
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