NEWARK, Calif., Feb. 12, 2014 (GLOBE NEWSWIRE) -- StemCells, Inc. (Nasdaq:STEM) announced today that it has completed enrollment of the first of two planned patient cohorts in the Company's clinical trial of its proprietary HuCNS-SC® product candidate (purified human neural stem cells) for dry age-related macular degeneration (AMD). This cohort consisted of eight subjects, four of whom each received 200,000 cells and four of whom each received 1,000,000 cells. The last patient in this cohort was transplanted by Dr. Ted Leng, M.D. Director of Ophthalmic Diagnostics at the Byers Eye Institute at Stanford.
"Our immediate goal for the study in the next quarter is to complete enrollment of the second cohort. The eight patients in this stage of the trial will have better visual acuity than those in the first cohort," said Stephen Huhn, M.D., FACS, FAAP, Vice President, CNS Clinical Research at StemCells, Inc. "We are expanding the trial from two sites at the beginning to ultimately a total of five sites, and with additional sites actively evaluating patients, the second cohort should be completed by mid-year. We also expect to announce interim follow-up data on the first cohort later this year. Following enrollment of the second cohort, we expect to initiate a controlled phase II efficacy proof-of-concept study by year end."
About Age-Related Macular Degeneration
Age-related macular degeneration refers to a loss of photoreceptors (rods and cones) from the macula, the central part of the retina. AMD is a degenerative retinal disease that typically strikes adults in their 50s or early 60s and gradually progresses to destroy central vision. According to the Foundation Fighting Blindness website, an estimated 10 million people in the U.S. either have AMD or are at substantial risk for receiving the diagnosis. Overall, about 85% of patients with AMD suffer from the dry form of the disease, and the advanced dry form of AMD is referred to as Geographic Atrophy.
About the Trial
The Phase I/II trial will evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for dry AMD. The investigation will be divided into two sequential cohorts. Subjects will be enrolled into each cohort based on best-corrected visual acuity (BCVA) visual acuity as determined by the Electronic Early Treatment Diabetic Retinopathy Study (E-ETDRS) acuity test. BCVA of less than or equal to 20/400 in the Study Eye will be enrolled in Cohort I. Subjects with BCVA of 20/320 to 20/100 in the Study Eye will be enrolled in Cohort II. Cohort I will consist of four subjects who will undergo transplant with 200,000 cells followed by four subjects who will undergo transplant with 1 million cells. Cohort II will consist of 8 subjects who will undergo transplant with 1 million cells. The HuCNS-SC cells will be administered by a single injection into the space behind the retina in the most affected eye. Patients' vision will be evaluated using both conventional and advanced state-of-the-art methods of ophthalmological assessment. Evaluations will be performed at predetermined intervals over a one-year period to assess safety and signs of visual benefit. Patients will then be followed for an additional four years in a separate observational study. The trial is currently enrolling patients at four centers:
Retina Foundation of the Southwest, Dallas, TX; Byers Eye Institute at Stanford, Stanford Hospital and Clinics; Palo Alto, CA; New York Eye and Ear Infirmary, New York City, NY; and Retina Research Institute of Texas, Abilene, TX. Patients who are interested in participating in the study should contact the Retina Foundation of the Southwest at 214-363-3911, Byers Eye Institute at 650-498-4486, the New York Eye and Ear Infirmary at 212-979-4251, or the Retina Research Institute of Texas at 325-690-4414.
Information about the Company's Dry Age Related Macular Degeneration program can be found on the StemCells, Inc. website at:
http://www.stemcellsinc.com/Therapeutic-Programs/AMD-and-Retinal-Disorders.htm
Additional information about the clinical trial is available at:
http://www.stemcellsinc.com/Therapeutic-Programs/Clinical-Trials.htm
and at the U.S. National Institutes of Health website at:
http://www.clinicaltrials.gov/ct2/show/NCT01632527?term=stemcells+inc+amd&rank=1
About HuCNS-SC Cells
StemCells' proprietary product candidate, HuCNS-SC cells, is a highly purified composition of human neural stem cells that are expanded and stored as banks of cells. The Company's preclinical research has shown that HuCNS-SC cells can be directly transplanted in the central nervous system (CNS) with no sign of tumor formation or adverse effects. Because the transplanted HuCNS-SC cells have been shown to engraft and survive long-term, there is the possibility of a durable clinical effect following a single transplantation. StemCells believes that HuCNS-SC cells may have broad therapeutic application for many diseases and disorders of the CNS, and to date has demonstrated human safety data from completed and ongoing clinical studies.
About StemCells, Inc.
StemCells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery. The Company's lead therapeutic product candidate, HuCNS-SC® cells (purified human neural stem cells), is currently in development as a potential treatment for a broad range of central nervous system disorders. In a Phase I clinical trial in Pelizaeus-Merzbacher disease (PMD), a fatal myelination disorder in children, the Company has shown preliminary evidence of progressive and durable donor-derived myelination in all four patients transplanted with HuCNS-SC cells. The Company is conducting a Phase I/II clinical trial in chronic spinal cord injury in Switzerland, Canada and the United States, and has reported positive interim data for the first three patients. . In addition, the Company is pursuing preclinical studies in Alzheimer's disease, with support from the California Institute for Regenerative Medicine (CIRM). StemCells also markets stem cell research products, including media and reagents, under the SC Proven® brand. Further information about StemCells is available at http://www.stemcellsinc.com.
Apart from statements of historical fact, the text of this press release constitutes forward-looking statements within the meaning of the Securities Act of 1933, as amended, and the Securities Exchange Act of 1934, as amended, and is subject to the safe harbors created therein. These statements include, but are not limited to, statements regarding the prospect of the Company's HuCNS-SC cells to preserve vision; the prospect and timing of patient enrollment in the Company's clinical trial in dry AMD; and the future business operations of the Company. These forward-looking statements speak only as of the date of this news release. The Company does not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Such statements reflect management's current views and are based on certain assumptions that may or may not ultimately prove valid. The Company's actual results may vary materially from those contemplated in such forward-looking statements due to risks and uncertainties to which the Company is subject, including the fact that additional trials will be required to demonstrate the safety and efficacy of the Company's HuCNS-SC cells for the treatment of any disease or disorder; uncertainty as to whether the FDA or other applicable regulatory agencies or review boards will permit the Company to continue clinical testing in AMD; uncertainties regarding the timing and duration of any clinical trials; uncertainties regarding the Company's ability to recruit the patients required to conduct its clinical trials or to obtain meaningful results; uncertainties regarding the Company's ability to obtain the increased capital resources needed to continue its current and planned research and development operations; uncertainty as to whether HuCNS-SC cells and any products that may be generated in the future in the Company's cell-based programs will prove safe and clinically effective and not cause tumors or other adverse side effects; ; and other factors that are described under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2012, and in its subsequent reports on Forms 10-Q and 8-K.