Neuralstem Receives FDA Approval To Commence Spinal Cord Injury Trial

    Neuralstem Receives FDA Approval To Commence Spinal Cord Injury Trial

NSI-566 to be tested in patients with chronic spinal cord injury

PR Newswire

ROCKVILLE, Md., Jan. 14, 2013

ROCKVILLE, Md., Jan. 14, 2013 /PRNewswire/ --Neuralstem, Inc. (NYSE MKT: CUR)
announced that it received approval from the United States Food and Drug
Administration (FDA) to commence a Phase I safety trial of its lead cell
therapy candidate, NSI-566, in chronic spinal cord injury patients. This
open-label, multi-site study, will enroll up to eight patients with thoracic
spinal cord injuries (T2-T12), who have an American Spinal Injury Association
(AIS) A level of impairment, between one and two years after injury. AIS A
impairment refers to a patient with no motor or sensory function in the
relevant segments at and below the injury, and is considered to be complete
paralysis.

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The primary objective of the study is to determine the safety and toxicity of
human spinal stem cell transplantation for the treatment of paralysis and
related symptoms due to chronic spinal cord injury (SCI). The secondary
objectives of the study are to evaluate graft survival in the transplant site
by MRI, as well as the effectiveness of transient immunosuppression.
Additionally, the study will look at exploratory objectives to evaluate the
ability of human spinal cord stem cell (HSSC) transplantation to positively
affect AIS level, ISNC SCI motor and sensory index scores, bowel and bladder
function, pain, UAB IMR scores, SCIM scores, evoked sensory and motor
potentials, and electromyogram (EMG).

All patients in the study will receive six injections in, or around, the
injury site. The first four patients will receive 100,000 cells per injection,
the second four patients will receive 200,000 cells per injection. All
patients will also receive physical therapy post surgery, as well as
immunosuppressive therapy, which will be for three months, as tolerated. The
trial study period will end six months post-surgery for each patient.

"The company is both pleased and excited to begin this long-awaited trial to
treat spinal cord injury patients with our spinal cord cells, NSI-566," said
Karl Johe, Ph.D., Neuralstem Chairman of the Board and Chief Scientific
Officer. "In August, in the peer-reviewed journal 'Cell,' compelling evidence
was presented that the cells can 'bridge the gap' in a severed spinal cord
model and return functionality.

This will be the first stem cell trial to attack the problem that way, as well
as the first to treat chronic spinal cord injury patients," Dr. Johe went on
to say. "In addition to the pre-clinical animal data, we have conducted 18
successful surgeries using the same cells and surgical device in our ALS
trial. That trial has demonstrated that the surgical route of administration
and the cells are safe and, well-tolerated and that the cells survive
long-term in the patients. The successes of our human clinical experience,
combined with the compelling data from the preclinical spinal cord injury
animal studies gives us confidence that we are prepared to move into this
additional indication for NSI-566."

"This is another critical milestone for the company," said Richard Garr,
Neuralstem President and CEO. "We believe the FDA approval of a trial for a
second indication for our NSI-566 cells demonstrates an increasing level of
comfort at the FDA with our technology and acknowledges the positive strides
being made in the ALS trial. Our goal for this SCI trial is to transplant and
collect the 6 month data for all patients within a one-year timeframe.

The approval of this trial is also a demonstration of our product development
strategy," Mr. Garr continued. "While we can create cell products from various
regions of the CNS that might be applicable to all manner of indications, we
have chosen to address multiple indications where the same cell product is
applicable. This allows us to leverage the product manufacturing and safety
data created in each trial with the next. Along those lines, we will begin
dosing patients with NSI-566 to treat paralysis from stroke in the next month,
in China, and we are preparing for a trial with NSI-566 to treat acute spinal
cord injury patients in Korea this summer. We are also currently in
discussions with the FDA over the design and scope of our Phase II trial for
ALS with NSI-566. As we begin to create proof-of-principle data in
multiple indications, we are also creating additional NSI-556 safety data
across indications and borders."

In a September study published in the journal CELL
(http://www.cell.com/abstract/S0092-8674%2812%2901018-5), paralyzed rats
transplanted with NSI-566 stem cells recovered significant locomotor function,
regaining movement in all lower extremity joints. Additionally, the
transplanted neural stem cells turned into neurons which grew multiple axons.
These axons extended over 17 spinal segments above and below the point of
severance, where made reciprocal synaptic connectivity with the host spinal
cord neurons, improving electrophysiological and functional outcome

About Chronic Spinal Cord Injury

There are more than 10,000 new spinal cord injuries (SCI) in the US each year.
At the time of a recent survey done by the Christopher & Dana Reeve
Foundation, there were approximately 840,000 people living with chronic SCI,
which are patients in whom paralysis persists and becomes permanent. According
to the National Spinal Cord Injury Association, 85% of SCI patients who
survive the first 24 hours are still alive 10 years later.

About Neuralstem

Neuralstem's patented technology enables the ability to produce neural stem
cells of the human brain and spinal cord in commercial quantities, and the
ability to control the differentiation of these cells constitutively into
mature, physiologically relevant human neurons and glia. Neuralstem completed
dosing of the last patient in an FDA-approved Phase I safety clinical trial
for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's
disease, in August 2012; the trial ends six months after that last surgery.
Neuralstem has been awarded orphan status designation by the FDA for its ALS
cell therapy.

In addition to ALS, the company is also targeting major central nervous system
conditions with its NSI-566 cell therapy platform, including spinal cord
injury, ischemic stroke and glioblastoma (brain cancer). The company received
approval to commence a Phase I safety trial in chronic spinal cord injury in
January, 2013.

Neuralstem also has the ability to generate stable human neural stem cell
lines suitable for the systematic screening of large chemical libraries.
Through this proprietary screening technology, Neuralstem has discovered and
patented compounds that may stimulate the brain's capacity to generate new
neurons, possibly reversing the pathologies of some central nervous system
conditions. The company is in a Phase Ib safety trial evaluating NSI-189, its
first neurogenic small molecule compound, for the treatment of major
depressive disorder (MDD).Additional indications could include chronic
traumatic encephalopathy (CTE), Alzheimer's disease, and post-traumatic stress
disorder (PTSD).

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 may contain forward-looking statements made pursuant to the
"safe harbor" provisions of the Private Securities Litigation Reform Act of
1995. Investors are cautioned that such forward-looking statements in this
press release regarding potential applications of Neuralstem's technologies
constitute forward-looking statements that involve risks and uncertainties,
including, without limitation, 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, 2011 and the quarterly report on
Form 10-Q for the period ended September 30, 2012.

SOURCE Neuralstem, Inc.

Website: http://www.neuralstem.com
Contact: Deanne Eagle - Media Relations, +1-917-837-5866, Susan Roush -
Investor Relations, +1-818-222-8330
 
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