Sensory gains demonstrate the clinical effect of
HuCNS–SC® transplantation
Results hold promise for Phase II PathwayTM
cervical spinal cord injury study
StemCells, Inc. (Nasdaq:STEM), a leading stem cell company
developing novel cell-based therapeutics for treating diseases of
the central nervous system, announced today that Dr. Armin Curt,
principal investigator, will present a summary of the safety and
preliminary efficacy data from the Phase I/II study investigating
Human Central Nervous System Stem Cell (HuCNS-SC) intramedullary
transplantation in thoracic spinal cord injury. The summary will be
presented today at 10:15 a.m. EDT at the 4th Joint International
Spinal Cord Society (ISCoS) and American Spinal Injury Association
(ASIA) meeting being held in Montreal, Canada. The abstract was
selected as one of the top six platform submissions for the
conference.
The international, open-label, Phase I/II trial evaluated both
safety and preliminary efficacy of StemCells, Inc.'s proprietary
HuCNS-SC human neural stem cells as a treatment for chronic spinal
cord injury. The trial enrolled twelve patients who had suffered
injury to the thoracic cord and were in the early chronic stage of
recovery. The severity of spinal cord trauma is classified by the
ASIA Impairment Scale (AIS) according to the degree of sensory and
motor loss. Patients classified as AIS A, the most severe injury,
have lost both sensory and motor function below the level of
injury. AIS B patients are considered less severely injured
because, although they also have no motor function below the level
of injury, some limited sensory function is maintained. The
protocol was specifically designed to test safety and preliminary
efficacy across this spectrum of injury severity, and ultimately
the trial transplanted seven AIS A and five AIS B patients.
The analysis of the study demonstrated that the surgical
transplantation technique and cell dose were safe and well
tolerated by all patients. HuCNS-SC cells were injected directly
into the cord both above and below the level of injury and
sequential examinations of the patients over the course of twelve
months showed no abnormal changes in spinal cord function
associated with the transplantation technique. There were no
adverse events attributed to the HuCNS-SC cells.
In addition to safety, analysis of the twelve-month data
revealed sustained improvements in sensory function that emerged
consistently around three months after transplantation and
persisted until the end of the study. The patterns of sensory gains
were confirmed to involve multiple sensory pathways and were
observed more frequently in the patients with less severe injury;
three of the seven AIS A patients and four of the five AIS B
patients showed signs of positive sensory gains confirming the
previously released interim results. In addition, two patients
progressed during the study from the most severe classification,
AIS A, to the lesser degree of injury grade, AIS B.
"It has been a privilege to be a part of the first study to test
the potential of neural stem cell transplantation in thoracic
spinal cord injury," said Dr. Armin Curt, Professor and Chairman of
the Spinal Cord Injury Center at Balgrist University Hospital,
University of Zurich. "The gains we have detected indicate that
areas of sensory function have returned in more than half the
patients. Such gains are unlikely to have occurred spontaneously
given the average time from injury. This patient population
represents a form of spinal cord injury that has historically
defied responses to experimental therapies, and the measurable
gains we have found strongly argue for a biological result of the
transplanted cells. These gains are exciting evidence that we are
on the right track for developing this approach for spinal cord
injury. This early outcome in thoracic injury firmly supports
testing in cervical spinal cord injury."
Stephen Huhn, M.D., FACS, FAAP, Vice President, Clinical
Research and CMO at StemCells, Inc., said, "This research program
has the potential to revolutionize the therapeutic paradigm for
spinal cord injury patients. The clinical gains observed in this
first study are a great beginning. We found evidence of sensory
gains in multiple segments of the injured thoracic spinal cord
across multiple patients. Our primary focus in this study for
spinal cord injury was to evaluate safety and also to look for even
small signs of an effect that went beyond the possibility of
spontaneous recovery. We are obviously very pleased that the
pattern of sensory gains observed in this study are both durable
and meaningful, and indicate that the transplantation has impacted
the function of damaged neural pathways in the cord. The Company's
development program has now advanced to a Phase II controlled study
in cervical spinal cord injury where the corollary of sensory
improvements in thoracic spinal cord injury could well be improved
motor function in the upper extremities of patients with cervical
spinal cord injuries."
About the StemCells, Inc. Thoracic Spinal Cord Injury
Clinical Trial
The Company's Phase I/II clinical trial was designed to assess
both safety and preliminary efficacy of HuCNS-SC cells as a
treatment for chronic spinal cord injury. The Company enrolled 12
subjects with thoracic T2-T11 level (chest-level) neurological
injuries classified as either AIS A or B according to the ASIA
Impairment Scale (AIS) who were a minimum of 3 months from injury.
Both AIS A and B are considered to have complete loss of motor
function below the level of injury, but AIS B subjects have some
degree of preserved sensory function below the level of injury.
The trial involved three world-leading medical centers for
spinal cord injury and rehabilitation, and associated principal
investigators; Dr. Armin Curt at the University of Zurich and
Balgrist University Hospital, Dr. Steve Casha at the University of
Calgary, and Dr. Michael Fehlings at the University of Toronto.
All subjects who enrolled in the trial received HuCNS-SC cells
through direct transplantation into the spinal cord and underwent
temporary treatment with immunosuppressive drugs. Evaluations
were performed regularly throughout the post-transplant period in
order to monitor and assess the safety of the HuCNS-SC cells, the
surgery and the immunosuppression, as well as to measure any change
in neurological function. Preliminary efficacy was evaluated
based on defined clinical endpoints using established spinal cord
injury scales assessing changes in sensory and motor function.
Patients were enrolled in a separate observational study after
completing the twelve-month Phase I/II trial.
Enrollment included seven AIS A and 5 AIS B patients. The study
showed a favorable safety, feasibility and tolerability profile
with no adverse events associated to the HuCNS-SC cells. Twelve
months after transplantation of the HuCNS-SC cells, data showed
gains in sensory function in three of the seven AIS A patients and
four of the five AIS B patients. In addition, two patients
progressed from the most severe classification, AIS A, to a lesser
degree of injury grade, AIS B.
In follow-up to the thoracic study, the Company has initiated a
Phase II trial designed to measure efficacy in cervical spinal cord
injury. The Phase II trial, also called the Pathway study, is a
controlled study and was initiated in late 2014.
Information about the Company's Spinal Cord Injury program can
be found on the StemCells, Inc. website at:
http://www.stemcellsinc.com/Clinical-Programs/SCI
About the Pathway Cervical Spinal Cord Injury Clinical
Trial
The Pathway Phase II 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 in the
cervical region of the spinal cord. Conducted as a randomized,
controlled, single-blind study, the trial 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 approximately 52 subjects and
follow the patients for 12 months post-transplant. The first cohort
of six patients completed enrollment in April. This is a dose
confirmation cohort. The results from this cohort will determine
the cell dosage to be used for the remainder of the study. The
six-month interim data from this cohort will be disclosed later
this year.
Information about the Company's spinal cord injury program can
be found on the StemCells, Inc. website at:
http://www.stemcellsinc.com/Clinical-Programs/SCI
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:
http://www.stemcellsinc.com/Science/Pre-Clinical-Studies/About-SCI
Information for patients interested in participating in the
study is available at the Pathway website at:
http://www.sciresearchstudy.com
Additional information about the clinical trial is available
at:
http://clinicaltrials.gov/ct2/show/NCT02163876?term=stem+cells+cervical+spinal+cord+injury&rank=1
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 in
humans, 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 disorders of the central
nervous system (CNS). 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 2 of 7 patients enrolled in
the study with complete injuries (AIS A) converting to incomplete
injuries (AIS B), post-transplant. In addition to the completion of
the Phase I/II thoracic SCI study and the ongoing Pathway study,
StemCells, Inc. has completed enrollment in its Phase I/II clinical
trial in geographic atrophy of age-related macular degeneration
(GA-AMD), the most severe form of dry AMD and the leading cause of
blindness in the elderly. Based upon interim results for patients
who had completed twelve months of follow-up, the Company reported
reductions in the rate of progression in GA as compared to the
untreated eye and the expected natural history of the disease. 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 restore sensory
function in patients with chronic spinal cord injury; the prospect
for continued clinical development of the Company's HuCNS-SC cells
in CNS disorders; 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 spinal cord injury, AMD, PMD, or any other condition;
uncertainties about whether data in any Phase I 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 whether results in preclinical research in animals will
be indicative of future clinical results in humans; uncertainties
regarding the Company's manufacturing capabilities given its
increasing preclinical and 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.
CONTACT: Greg Schiffman
StemCells, Inc.
Chief Financial Officer
(510) 456-4128
Lena Evans
Russo Partners
(212) 845-4262
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