ACT Secures Approval from Data Safety Monitoring Board to Complete Third Patient Cohort in All Three Clinical Trials

MARLBOROUGH, Mass.--(BUSINESS WIRE)--

Advanced Cell Technology, Inc. (ACT) (ACTC), a leader in the field of regenerative medicine, announced today that the Data and Safety Monitoring Board (DSMB), an independent group of medical experts closely monitoring the companys three ongoing clinical trials, has authorized the company to move forward with enrollment and treatment of remaining two patients in the third cohort of each of the three clinical trials. The decision follows an interim review by the DSMB six weeks after the first patient was treated in the third cohort of each trial. ACT will proceed with screening and enrollment for the patients who, in keeping with trial protocol, will be injected with 150,000 retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs).

We are pleased that the first patients treated with the higher dose of RPE cells are doing well and the DSMB sees fit for enrollment to continue in all three clinical trials, commented Gary Rabin, chairman and CEO of ACT. Even with several patients failing laboratory evaluations late in the inclusion/exclusion review process, which is not atypical in clinical trials, we have four of the six patient surgeries for the third cohorts scheduled, with two remaining to complete screening. We are confident that all surgeries will be performed in ample time for a complete data review at the companys Ophthalmic Advisory Board meeting in early October, where Phase 2 trial designs will be discussed.

ACTs three clinical trials in the U.S. and Europe use hESC-derived RPE cells to treat forms of macular degeneration, dry AMD and Stargardts Macular Dystrophy (SMD). Each trial will enroll four cohorts, with three patients each, in an ascending dosage format from 50,000 to 200,000 hESC-derived RPE cells. The U.S.-based clinical trials in AMD and SMD are currently enrolling a cohort of four patients with better vision receiving 100,000 hESC-derived RPE cells, referred to as Cohort 2a. To date, the company has treated four patients in Cohort 2a, with two more surgeries scheduled. These trials are prospective, open-label studies, designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with dry AMD or SMD at 12 months, the studys primary endpoint.

Robert Lanza, M.D., chief scientific officer of ACT, added, The DSMB approval is an important milestone, especially as we continue to significantly increase the RPE dosage in all three of our clinical trials. This independent review of safety comes on the two year anniversary of the treatment of our first two patients in July 2011. Those original patients continue to do well, and the gains in visual acuity we first reported in The Lancet in January 2012, have persisted. We are excited to enroll the next patients in these cohorts, as well as the better vision patients in Cohort 2a of the U.S. trials.

Preliminary results from the U.S. Stargardts and dry AMD trials were reported in The Lancet last year.

Further information about patient eligibility for ACTs dry AMD study and the companys concurrent SMD studies in the U.S. and E.U. is available at http://www.clinicaltrials.gov, with the following Identifiers: NCT01344993 (dry AMD), NCT01345006 (U.S. SMD), and NCT01469832 (E.U. SMD).

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.

Forward-Looking Statements

Excerpt from:
ACT Secures Approval from Data Safety Monitoring Board to Complete Third Patient Cohort in All Three Clinical Trials