Advanced Cell Technology’s Studies To Support Phase I Multicenter Trial Of Patients With Stargardt’s Macular Dystrophy Demonstrate Excellent Safety Profile
Advanced Cell Technology, Inc. (“ACT”; OTCBB:
announced today that it has completed key animal studies in connection
with its Phase I multicenter study using embryonic stem cell derived
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).