ACT announces European phase I clinical trial for Stargardt's disease using retinal pigment epithelial cells

Apr 26 2013

Advanced Cell Technology, Inc. ("ACT"; OTCBB: ACTC), a leader in the field of regenerative medicine, today announced treatment of the first patient in the third dosage cohort, and seventh patient overall, in its European Phase I clinical trial for Stargardt's macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The patient was injected with 150,000 hESC-derived RPE cells, as compared with the 100,000-cell dose used in patients of the second cohort. The surgery was performed on Friday, April 19, without any complications, and the patient is recovering uneventfully.

“We are eagerly anticipating advancing toward the final cohort in all three trials.”

"Europe represents a huge potential market for us, the world's largest after the U.S., so we are particularly pleased to now be past the halfway point in all three of our clinical trials on both continents," commented Gary Rabin, chairman and CEO of ACT. "SMD affects as many as 100,000 patients in the U.S. and Europe. Moreover, as previously announced, the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) officially granted our hESC-derived RPE cells orphan medicinal product designation for the treatment of SMD. We expect this will provide a number of benefits once our SMD treatment has made the transition from the clinic to the bedside, including reduced fees and protection from competition."

The Phase 1/2 trial is designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with SMD at 12 months, the study's primary endpoint. It will involve a total of 12 patients, with cohorts of three patients each in an ascending dosage format.

"We are pleased to have now moved into the second-half of all our clinical trials for macular degeneration," said Robert Lanza, M.D., chief scientific officer. "We are eagerly anticipating advancing toward the final cohort in all three trials."