Animal studies demonstrate safety profile of ACT's RPE cell therapy for SMD

Apr 24 2010

Advanced Cell Technology, Inc. ("ACT"; OTCBB: ACTC) announced today that it has completed key animal studies in connection with its Phase I multicenter study using embryonic stem cell derived Retinal Pigment Epithelium (RPE) cells to treat patients with Stargardt's Macular Dystrophy (SMD), for which it filed an Investigational New Drug Application (IND) with the US Food and Drug Administration (FDA) in November. The studies demonstrated an excellent safety profile with no safety signals such as tumors or ectopic tissues. The studies were designed to address the FDA's request for additional data on tumorigenicity and biodistribution. The Company believes that the data will support the FDA granting clearance for the Company to commence the SMD study in humans later this year. The studies were completed in conjunction with Sinclair Research, based in Columbia, Missouri, Charles River Labs, MPI and Althea Labs, which have been the company's outside independent collaborators on both studies.

"We believe these animal studies demonstrate an excellent safety profile for our RPE cell therapy," said William M. Caldwell IV, ACT's Chairman & CEO . "We look forward to concluding our discussions with the FDA so that we can commence our study later this year."

The Phase I trial will be a prospective, open-label study that is designed to determine the safety and tolerability of the retinal pigment epithelium (RPE) cells following sub-retinal transplantation to advanced patients with SMD. A total of twelve patients will be enrolled into the study at three clinical sites, to include the Casey Eye Institute, Portland, Oregon (headed by Dr. Peter Francis of the Oregon Health & Sciences University); the University of Massachusetts Memorial Medical Center, Worcester, Massachusetts (headed by Dr. Shalesh Kaushal, Chair of the Department of Ophthalmology); and the UMDNJ - New Jersey Medical School, Newark, New Jersey (headed by Dr. Marco Zarbin, Chair, Institute of Ophthalmology and Visual Science).

Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world. As many as ten million people in the United States have photoreceptor degenerative disease. While most of these patients have Age-Related Macular Degeneration (AMD), a smaller number of patients have Stargardt's Macular Dystrophy, a disease indication for which ACT was granted "Orphan Status" for its proposed clinical trial by the US Department of Health and Human Services earlier this year. ACT's treatment for eye disease is sourced with human Pluripotent stem cells that are differentiated into RPE cells used to augment the patient's existing, deteriorating RPE layer. The RPE cells are often the first to die off in SMD and AMD, which is a contributor to the patient's loss of vision.

Several years ago ACT scientists successfully derived RPE cells from human embryonic stem cells. Later, they were able to derive them from cells developed from the Company's proprietary "blastomere" technology that does not destroy the embryo. Subsequent studies found that the cells could restore vision in animal models of macular degeneration. In the Royal College of Surgeon (RCS) rat model, implantation of RPE cells resulted in 100% improvement in visual performance over untreated controls, without any adverse effects. The cells survived for more than 220 days and sustained extensive photoreceptor rescue. Functional rescue was also achieved in the "Stargardt's" mouse with near-normal functional measurements recorded at more than 70 days.