Final Results for the First Six Patients to be Released Later this Quarter
NEWARK, Calif., April 18, 2016 (GLOBE NEWSWIRE) -- StemCells, Inc. (NASDAQ:STEM), a world leader in the research and development of cell-based therapeutics for the treatment of central nervous system disorders, announced today that Dr. Stephen Huhn, the Company’s Chief Medical Officer and VP of Clinical Research, presented additional details on its ongoing Phase II Pathway™ Study of HuCNS-SC cells for the treatment of chronic cervical spinal cord injuries. The presentation, which took place at the 2016 American Spinal Injury Association (ASIA) annual meeting in Philadelphia on Friday, April 15, included a top line update for the six patients enrolled in open label Cohort I from the Pathway Study. The 6-month results from Cohort I showed that muscle strength had improved in five of the six patients with four of these five patients also demonstrating improved performance on functional tasks assessing dexterity and fine motor skills. In addition, four of the six patients had improvement in the level of cord injury as measured by ISNCSCI (International Standards for Neurological Classification of Spinal Cord Injury) assessment. The Company expects to release detailed final 12-month results on this first open-label cohort later this quarter.
"The emerging data continue to be very encouraging," said Dr. Huhn. "We believe that these types of motor changes will improve the independence and quality of life of patients and are the first demonstration that a cellular therapy has the ability to impact recovery in chronic spinal cord injury. We currently have thirteen sites in the United States and Canada that are actively recruiting patients. We have enrolled and randomized 19 of the 40 total patients in the statistically powered, single-blind, randomized controlled, Cohort II. We are projecting to complete enrollment by the end of September so that we can have final results in 2017.”
About the Pathway Spinal Cord Injury Clinical Trial
The Phase II Pathway Study, titled “Study of Human Central Nervous System (CNS) Stem Cell Transplantation in Cervical Spinal Cord Injury,” will evaluate the safety and efficacy of transplanting the Company’s proprietary human neural stem cells (HuCNS-SC® cells) into patients with traumatic injury of the cervical region of the spinal cord. Cohort I is an open label dose-ranging cohort in six AIS-A or AIS-B subjects. Cohort II is a randomized, controlled, single-blinded cohort in forty AIS-B subjects. Cohort III, at the discretion of the sponsor, is an open-label arm involving six AIS-C subjects. The study will measure efficacy by assessing motor function according to the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI). The primary efficacy outcome will focus on change in upper extremity strength as measured in the hands, arms and shoulders. The trial will enroll up to 52 subjects. Enrollment of Cohort I has been completed and the Company is now enrolling patients into Cohort II.
Information about the Company’s spinal cord injury program can be found on the Company’s website at:
Information on the Company’s pre-clinical spinal cord injury research, along with an animation on the science of HuCNS-SC cells for the treatment of spinal cord injuries, can be found at:
Information for patients interested in participating in the study is available at the Pathway website at:
Additional information about the clinical trial is available at:
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 all three components of the central nervous system: 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 chronic spinal cord injury (SCI). The Company's Pathway Study, a Phase II proof-of-concept trial in chronic cervical SCI is actively enrolling at thirteen sites in the U.S. and Canada. Six-month interim data for the first cohort of the Pathway Study showed the first-ever clinical evidence of a treatment effect improving both upper muscle strength and motor function following cellular transplant in spinal cord injury. Top-line data from the Company's earlier Phase I/II clinical trial in chronic thoracic SCI showed measurable gains involving multiple sensory modalities and segments in seven of twelve patients enrolled in the study, including the conversion of two patients from the complete AIS-A spinal cord injury to the incomplete AIS-B spinal cord injury. The Company has also completed its Phase I/II clinical trial in geographic atrophy, the most advanced form of dry age related macular degeneration. Top-line results from this study show a positive safety profile and favorable preliminary efficacy data. 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. A Phase I study in children with Batten’s disease showed that transplantation of the cells into the brain was safe and resulted in long term survival of the cells.
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 prospect for continued clinical development of the Company's HuCNS‑SC cells in CNS disorders, the timing of final data release in the Company’s Pathway Study in cervical spinal cord injury, and the belief that the improvements observed in the Pathway Study will have a meaningful impact on the quality of life and independence of patients in the study. 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 risks whether the FDA or other applicable regulatory agencies, including applicable institutional review boards at one or more clinical trial sites, will permit the Company to continue clinical testing or conduct future clinical trials; uncertainties regarding the timing of patient enrollment in the Company’s Pathway Study; 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 preclinical and clinical commitments; uncertainties regarding the Company’s plans to increase its authorized share capital; 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" in the Company's Annual Report on Form 10-K for the year ended December 31, 2015.
Greg Schiffman, Chief Financial Officer StemCells, Inc.