StemCells, Inc. Transplants First Patient in Phase II Clinical Trial for Dry Age-Related Macular Degeneration

Radiant(TM) Study is the First Controlled Phase II Trial to Investigate the Efficacy of Human Neural Stem Cells for the Treatment of Dry AMD


NEWARK, Calif., July 20, 2015 (GLOBE NEWSWIRE) -- StemCells, Inc. (Nasdaq:STEM), a world leader in the research and development of cell-based therapeutics for the treatment of disorders of the brain, eye and spine, announced today that it has completed transplantation of the first subject in its Radiant Study at the Retina Foundation of the Southwest. The Phase II trial is designed to evaluate both the safety and efficacy of the Company's proprietary HuCNS-SC® platform technology (purified human neural stem cells) for the treatment of geographic atrophy (GA), the most advanced form of dry age-related macular degeneration (AMD).

"Initiation of this fellow eye controlled Phase II clinical trial re-affirms our leadership in the study of stem cells to treat disorders of the eye," said Joel Naor, M.D., MSc, MBA, Vice President of Ophthalmology Clinical Development, at StemCells, Inc. "AMD is a debilitating condition that severely impacts quality of life for millions of people and for which there is currently no treatment. HuCNS-SC cells may have the potential to preserve vision in those affected with dry AMD and possibly other degenerative retinal disorders."

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 typically manifests in adults in their 50s or early 60s and gradually progresses to compromise central vision. With an estimated 25-30 million cases worldwide, AMD is the number one cause of severe vision loss and legal blindness in adults over the age of 55. According to the Retina Foundation of the Southwest approximately 18 million Americans age 40 years and older have some form of AMD, and the disease continues to be the number one cause of irreversible vision loss among older adults. There are currently no approved treatments for the dry form of the disease, which afflicts 80-90 percent of AMD patients. The advanced form of dry AMD is referred to as geographic atrophy (GA-AMD).

About the Radiant™ Study

The StemCells, Inc. Radiant Study is a Phase II clinical trial designed to evaluate the safety and efficacy of HuCNS-SC human neural stem cells as a treatment for geographic atrophy. The study is enrolling 63 patients between 50-90 years of age with bi-lateral GA-AMD (geographic atrophy associated with age related macular degeneration in both eyes). Designed as a "fellow eye" controlled study, all subjects will receive subretinal transplantation of HuCNS-SC cells via a single injection into the eye with the inferior best-corrected visual acuity; the untreated eye will serve as a control. All patients will be followed for 12 months, with evaluations performed at predetermined intervals to assess safety, anatomic and functional changes. The objective of the trial is to demonstrate a reduction in the rate of disease progression in the treated eye versus the control eye.

More information about the Company's clinical programs in AMD can be found on the StemCells, Inc. website at:

http://www.stemcellsinc.com/Clinical-Programs/AMD

Additional information about the RadiantTM Study clinical trial is available at the U.S. National Institutes of Health website at:

https://clinicaltrials.gov/show/NCT02467634

About HuCNS-SC Cells

StemCells, Inc. has demonstrated human safety data from completed and ongoing clinical studies in which its proprietary HuCNS-SC® cells have been transplanted directly into the brain, the spinal cord and the eye. StemCells, Inc. clinicians and scientists believe that HuCNS-SC cells may have broad therapeutic application for many diseases and disorders of the CNS. 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. The HuCNS-SC platform technology is a highly purified composition of human neural stem cells (tissue-derived or "adult" stem cells). Manufactured under cGMP standards, the Company's HuCNS-SC cells are purified, expanded in culture, cryopreserved and then stored as banks of cells, ready to be made into individual patient doses when needed.

About StemCells, Inc.

StemCells, Inc. is currently engaged in clinical development of its HuCNS-SC® platform technology (purified human neural stem cells) as a potential treatment for both neurological and retinal disorders. Top-line data from the Company's Phase I/II clinical trial in thoracic spinal cord injury (SCI) showed measurable gains involving multiple sensory modalities and segments, including the conversion of two of seven patients enrolled in the study with complete injuries to incomplete injuries, post-transplant. The Company's Pathway™ Study, a Phase II proof-of-concept trial in cervical SCI is actively enrolling at eight sites and interim data from the first cohort of six patients is anticipated to be forthcoming in Q4 2015. StemCells, Inc. has also completed its Phase I/II clinical trial in GA-AMD. Top-line results from this study show a positive safety profile and favorable preliminary efficacy data related to visual acuity and contrast sensitivity. The Company's Radiant™ Study, a Phase II proof-of-concept trial in GA-AMD is now enrolling at the first of approximately twenty U.S. sites expected to participate. In a Phase I clinical trial in Pelizaeus-Merzbacher disease (PMD), a fatal myelination disorder in children, the Company showed preliminary evidence of progressive and durable donor-derived myelination by MRI. Further information about StemCells, Inc. 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 U.S. securities laws, and is subject to the safe harbors created therein. These statements include, but are not limited to, statements regarding the future business operations of StemCells, Inc. (the "Company"); the timing and prospects associated with detecting potential clinical benefit from the use of the Company's HuCNS-SC cells; the ability of HuCNS-SC cells to affect the rate of geographic atrophy; the prospect for continued clinical development of the Company's HuCNS-SC cells in CNS disorders; the prospect that the Company can initiate the necessary clinical trial sites and enroll study subjects in a timely fashion, if at all, in the Radiant Phase II Study; and the likelihood that early signs of clinical efficacy can be replicated in future clinical studies. 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 uncertainties with respect to the fact that additional trials will be required to confirm the safety and demonstrate the efficacy of the Company's HuCNS-SC cells for the treatment of AMD or any other condition; uncertainties about whether data in any Phase I or Phase I/II clinical study will prove to be reproducible or biologically meaningful in any future clinical study; risks whether the FDA or other applicable regulatory agencies will permit the Company to continue clinical testing or conduct future clinical trials; 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; uncertainties regarding the Company's manufacturing capabilities given its increasing clinical commitments; uncertainties regarding the validity and enforceability of the Company's patents; uncertainties as to whether the Company will become profitable; and other factors that are described under the heading "Risk Factors" disclosed in Part I, Item 1A in the Company's Annual Report on Form 10-K for the year ended December 31, 2014 and in its subsequent reports on Form 10-Q and Form 8-K.



            

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