PALO ALTO, Calif., Apr 8, 2011 (GlobeNewswire via COMTEX) --
StemCells, Inc. (Nasdaq:STEM) today announced that it has discontinued its Phase Ib clinical trial in neuronal ceroid lipofuscinosis (NCL, also referred to as Batten disease), a rare and fatal neurodegenerative disorder in children, due to lack of patient accrual. In 2009, the Company completed a Phase I safety trial of its HuCNS-SC(R) human neural stem cells in six patients with advanced stages of NCL at Oregon Health & Science University (OHSU) Doernbecher Children's Hospital. In October 2010, the Company initiated a Phase Ib trial at OHSU to evaluate the cells in an additional six patients in earlier stages of the disease, and therefore most likely to benefit from a timely neural stem cell transplant. However, no eligible patients have been identified or enrolled despite diligent efforts by the clinical investigators over the past six months.
Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc., stated, "In clinical trials for rare diseases, timely patient enrollment is always of concern to study sponsors. Unfortunately, this concern has materialized in our Phase Ib NCL study. Out of 22 initial prospects, not one patient has met the entry criteria. This experience has also highlighted the significant challenges the Company would face in completing, within a reasonable period of time, the much larger studies in the target patient population that would ultimately be required for marketing approval. These factors, combined with our limited corporate resources, have prompted the decision to shelve our clinical development program in NCL at this time."
Martin McGlynn, President and CEO of StemCells, Inc., commented, "We wish to thank the clinicians and staff at OHSU for their dedication and contributions to this pioneering stem cell research. We are also deeply grateful to the families of the six patients involved in our Phase I trial. No doubt, this decision will come as a great disappointment to the NCL community, but I would like to point out that their support has greatly contributed to the prospect that this groundbreaking technology will one day result in a treatment for a broad range of debilitating neurodegenerative disorders. As a result of this clinical research, we have established that we can safety transplant meaningful doses of HuCNS-SC cells directly into the brain, and that patients can tolerate both the surgical intervention and the immunosuppression regimen. We also now have evidence that, once transplanted, the cells are capable of engrafting and surviving long-term, suggesting the possibility of durable clinical benefit. And, importantly, we have not observed any evidence of aberrant cell behavior or tumor formation in patients who are now more than three years post-transplant, and whom we continue to follow as part of a long-term observational study."
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(R) cells (purified human neural stem cells), is currently in development as a potential treatment for a broad range of central nervous system disorders. Clinical trials are currently underway in spinal cord injury and in Pelizaeus-Merzbacher disease (PMD), a fatal myelination disorder in children. The Company's HuCNS-SC cells are also in preclinical development for retinal disorders, such as age-related macular degeneration, and for Alzheimer's disease and Stroke. StemCells also markets stem cell research products, including media and reagents, under the SC Proven(R)brand, and is developing stem cell-based assay platforms for use in pharmaceutical research, drug discovery and drug development. Further information about StemCells is available at http://www.stemcellsinc.com.
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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 conducting future clinical research in NCL; the potential of the Company's HuCNS-SC cells to treat a broad range of central nervous system disorders; and the future business operations of the Company, including its ability to conduct clinical trials as well as its other research and product development efforts. 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 will permit the Company to continue clinical testing in spinal cord injury, PMD or in future clinical trials of proposed therapies for other diseases or conditions given the novel and unproven nature of the Company's technologies; 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 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; uncertainties regarding the Company's ability to commercialize a therapeutic product and its ability to successfully compete with other products on the market; 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, 2010, and in its subsequent reports on Form 8-K.
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SOURCE: StemCells, Inc.
CONTACT: Megan Meloni
Media Relations & Corporate Communications