GERMANTOWN, Md., Sept. 29,
2015 /PRNewswire/ -- Neuralstem, Inc. (Nasdaq: CUR), a
biopharmaceutical company using neural stem cell technology to
develop small molecule and cell therapy treatments for central
nervous system diseases, announced that nine-month Phase II and
combined Phase I and Phase II data on the NSI-566 trial in
amyotrophic lateral sclerosis (ALS) was presented at the American
Neurological Association Annual Meeting by principal investigator,
Eva Feldman, MD, PhD, Director of
the A. Alfred Taubman Medical Research Institute and Director of
Research of the ALS Clinic at the University
of Michigan Health. The data showed that the
intraspinal transplantation of the cells was safe and
well-tolerated throughout the escalating doses, reaching a maximum
tolerated dose of 16 million cells via 20 bilateral
injections. There appeared to be no acceleration in disease
progression due to the therapeutic intervention.
Researchers calculated a 95% confidence limit around the slopes
of decline of ALSFRSr scores, forced vital capacity (FVC) and grip
strength of the ProAct historical database subjects, and evaluated
if trial subjects fell within or outside those limits. 73% of Phase
II patients, and 79% of combined Phase I and II patients, fell
above the upper confidence limit of the ALSFRSr score. 50% of Phase
I and II combined, and 40% of Phase II patients' forced vital
capacity percent predicted fell above the upper confidence limit,
compared to the ProAct database. ALSFRSr scores correlated
most strongly with FVC preservation, which was the target of the
cervical injections. For grip strength control,
researchers used the Ceftriaxone (CEF) study database, since grip
strength data was not available in the ProAct database. 67%
of Phase I and II combined, and 60% of Phase II patients, all at
nine months post-intervention, fell above the 95% upper confidence
limit.
The most common adverse event (AE) was transient post-operative
pain due to surgery. One serious adverse event due to the
surgical procedure was observed, but was not attributed to the
cells themselves. The patient's motor function was initially
weakened and then recovered to the patient's ALS
baseline.
"Based on this encouraging safety and clinical effect, we
look forward to moving to a registration-directed trial in
2016," said Karl Johe, PhD, Chief
Scientific Officer.
About the Trial
The Phase II open-label, dose-escalating trial of NSI-566
evaluated 15 ambulatory patients with ALS, averaging a mean
duration of disease of 15.5 months. Participants were divided into
five dosing cohorts with three patients in each, who received
increasing quantities of cells in the cervical region of the spinal
cord via bilateral intraspinal injections ranging from two million
to eight million cells. The fifth cohort received an additional
eight million cells in the lumbar region. There was no control or
placebo group included in the trial. The primary endpoint of the
study was the safety of the maximum tolerated dose of stem cell
transplantation. Secondary efficacy endpoints included
stabilization of ALS Functional Rating Scale-revised (ALSFRSr)
scores, and assessment of respiratory functioning, grip strength
and muscle strength. 9 of the 15 participants in Phase I and all 15
participants in Phase II were included in the 9-month data. Dr.
Eva Feldman, principal investigator,
is an unpaid consultant to Neuralstem.
About Amyotrophic Lateral Sclerosis (ALS)
ALS is a progressive disease that affects nerve cells, or
neurons, in the brain and the spinal cord, leading to degeneration
and eventual atrophy of the surrounding muscles. As the condition
worsens, motor neurons die, and the brain can no longer control the
affected muscles. In time, this causes the loss of patients'
ability to speak, eat, move and breathe, eventually resulting in
death. It is estimated that more than 5,600 people in the U.S. are
diagnosed with ALS each year, amounting to 15 new cases per day,
totaling approximately 30,000 Americans living with the disease at
any given time. There is currently no cure or treatment that that
halts or reverses the progression of the disease.1
About Neuralstem
Neuralstem's patented technology enables the commercial-scale
production of multiple types of central nervous system stem cells,
which are under development for the potential treatment of central
nervous system diseases and conditions.
Neuralstem's ability to generate human neural stem cell lines
for chemical screening has led to the discovery and patenting of
compounds that Neuralstem believes may stimulate the brain's
capacity to generate neurons, potentially reversing pathologies
associated with certain central nervous system (CNS) conditions.
The company has completed Phase Ia and Ib trials evaluating
NSI-189, its first neurogenic small molecule product candidate, for
the treatment of major depressive disorder (MDD), and is expecting
to initiate a Phase II study for MDD and a Phase Ib study for
cognitive deficit in schizophrenia in 2015.
Neuralstem's first stem cell product candidate, NSI-566, a
spinal cord-derived neural stem cell line, is under development for
treatment of amyotrophic lateral sclerosis (ALS). Neuralstem has
completed two clinical studies, in a total of thirty patients,
which met primary safety endpoints. In addition to ALS, NSI-566 is
also in a Phase I trial in chronic spinal cord injury at UC San
Diego School of Medicine, as well as in clinical development to
treat ischemic stroke.
Neuralstem's next generation stem cell product, NSI-532.IGF,
consists of human cortex-derived neural stem cells that have been
engineered to secrete human insulin-like growth factor 1 (IGF-1).
In animal data presented at the Congress of Neurological Surgeons
2014 Annual Meeting, the cells rescued spatial learning and memory
deficits in an animal model of Alzheimer's disease.
For more information, please visit www.neuralstem.com or connect
with us on Twitter, Facebook and LinkedIn.
Cautionary Statement Regarding Forward Looking Information:
This news release contains "forward-looking statements" made
pursuant to the "safe harbor" provisions of the Private Securities
Litigation Reform Act of 1995. Such forward-looking statements
relate to future, not past, events and may often be identified by
words such as "expect," "anticipate," "intend," "plan," "believe,"
"seek" or "will." Forward-looking statements by their nature
address matters that are, to different degrees, uncertain. Specific
risks and uncertainties that could cause our actual results to
differ materially from those expressed in our forward-looking
statements include risks inherent in the development and
commercialization of potential products, uncertainty of clinical
trial results or regulatory approvals or clearances, need for
future capital, dependence upon collaborators and maintenance of
our intellectual property rights. Actual results may differ
materially from the results anticipated in these forward-looking
statements. Additional information on potential factors that could
affect our results and other risks and uncertainties are detailed
from time to time in Neuralstem's periodic reports, including the
Annual Report on Form 10-K for the year ended December 31, 2014, and Form 10-Q for the three
and six months ended June 30, 2015,
filed with the Securities and Exchange Commission (SEC), and in
other reports filed with the SEC.
References:
1. ALS Association. About ALS. Available at:
http://www.alsa.org/about-als/. September
28, 2015.
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