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NEURALSTEM ANNOUNCES 2013 FINANCIAL RESULTS
ROCKVILLE, MD, March 10, 2014 -
Neuralstem, Inc. (NYSE MKT: CUR) today provided an update on its clinical trials program and reported its 2013 financial results
for the year ended December 31, 2013.
"2013 was an exciting year for advancement
in our NSI-566 cell therapy clinical programs," said Karl Johe, PhD, Neuralstem's Chairman of the Board and Chief Scientific
Officer. "Our Phase II ALS trial commenced in September, with increased dosing levels of our spinal cord cells towards what
we expect to be the maximum safe therapeutic dose for the final cohort in this trial. At the close of 2013, we expanded our NSI-566
programs globally with the dosing of the first patient in our ischemic stroke trial in China. We also filed our IND to treat acute
spinal cord injury in South Korea."
Dr. Johe continued, "We expect to
receive IRB approval and to transplant the first patient at UC San Diego School of Medicine, in our FDA-approved NSI-566 chronic
spinal cord injury (cSCI) Phase I trial this Spring.
"2013 also brought significant progress
in our neurogenic small molecule program. The company completed dosing of Phase Ib patients for NSI-189, in major depressive disorder
(MDD), in August. A comprehensive review of the data is currently underway. Measurements include tolerability, pharmacokinetics,
and pharmacodynamic effects including hippocampal volume by MRI, BDNF/CRH/other factors in blood, urine biomarkers, cortisol in
saliva, qEEG, MADRS, NGH Depression Questionnaire, and Columbia-Suicidal Severity Rating Scale. While this data analysis will take
us through the second quarter of 2014, the early review is encouraging. We expect to be commencing a Phase II trial in NSI-189
MDD in the second half of this year," concluded Dr. Johe.
to new clinical levels in both our cell therapy and neurogenic small molecule programs in multiple CNS indications around the world,
we are encouraged by the enthusiasm and support of leading institutional investors, including dedicated healthcare investors. Our
recent financings have strengthened our cash balance, establishing a strong foundation for all of our programs going forward,"
said Richard Garr, Neuralstem's President and CEO. "My fellow Directors and I are also
very pleased to have recently welcomed Dr. Catherine Sohn to our Board. Dr. Sohn's extensive experience directing global
commercialization for one of the world's largest pharmaceutical companies will prove invaluable as our clinical progress moves
our company to the next level of development.
many highlights of 2013, Dr. Johe and I were pleased to grant licenses to IP surrounding our proprietary spinal cord delivery platform,
floating cannula, and method for delivering therapeutic agents to the spinal cord to Cedars-Sinai Medical Center, a nonprofit academic
medical center in Los Angeles, for academic research," continued Garr. "We further strengthened our global IP portfolio
to 49 issued and 60 pending patents, with the addition of 37 patents and applications both in the U.S. and internationally during
and I continue to applaud the tremendous dedication, skill and tireless commitment of the great minds and skilled surgical hands
that are helping us achieve our aim of effectively treating CNS diseases and conditions that have doggedly remained huge unmet
medical needs for so long. We are all very encouraged by the data and excited about the clinical progress planned in the months
ahead. We praise and thank the brave patients and their families and caregivers who are helping us develop treatments that have
the promise of hope for so many," added Garr.
Business Highlights for 2013
NSI-566: Cell Therapy Lead Candidate
- ALS Phase I and II Clinical Trials (amyotrophic lateral sclerosis, or Lou Gehrig's disease)
In February, the NSI-566/ALS Phase I trial
officially concluded, six months after the final surgery at Emory University Hospital in Atlanta.
In April, Neuralstem
received FDA approval to commence the NSI-566/ALS Phase II trial, following the excellent safety and tolerability demonstrated
in Phase I. The Phase II dose escalation and safety trial expands to two centers: Emory University Hospital, and the ALS Clinic
at the University of Michigan Health System, in Ann Arbor, subject to IRB approvals.
data on the intraspinal delivery method employed in the NSI-566/ALS Phase I trial was presented at the American Association of
Neurological Surgeons' Annual Meeting. "Intraspinal Stem Cell Transplantation in ALS, A Phase I Trial: Cervical Microinjection
Safety Outcomes," was presented by Jonathan Patrick Riley, MD, of the Department of Neurological Surgery at Emory University.
In May, Neuralstem's
NSI-566/ALS principal investigator, Eva Feldman, MD, PhD presented updated Phase I data results from all 15 patients at the Romanian
Neurological Society Congress. Dr. Feldman is Director of the A. Alfred Taubman Medical Research Institute and Director of Research
of the ALS Clinic at the University of Michigan Health System, and an unpaid consultant to Neuralstem.
In June, Dr. Feldman
gave the grand plenary address at the Canadian Neurological Sciences Federation Annual Congress, which included a presentation
of the final NSI-566/ALS Phase I results, which included new cervical cohort data.
In September, Neuralstem's NSI-566/ALS
Phase II dose escalation and safety trial commenced. The multicenter trial is designed to treat up to 15 ambulatory ALS patients
in five different dosing cohorts. The first 12 patients will receive injections in the cervical region of the spinal cord only,
and the final three patients will receive both cervical and lumbar injections.
In October, University of Michigan Health
System treated its second patient, which represented the completion of the first of the NSI-566/ALS Phase II trial's five
cohorts in less than a month's time.
In October, Dr. Feldman gave an update
on the NSI-566/ALS trial at the annual American Neurological Association Meeting.
NSI-566: cSCI Phase I Clinical Trial
(chronic spinal cord injury)
In January, the FDA approved NSI-566 for
a Phase I trial to treat cSCI. The open-label, multi-site, ascending-dose study will enroll up to eight patients with thoracic
spinal cord injuries who have an American Spinal Injury Association AIS-A level of impairment (patients who are considered to be
in complete paralysis) between nine months and two years post injury.. NSI-566/cSCI patients will receive post-surgery physical
therapy, 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.
NSI-189: Neurogenic Small Molecule Lead
Compound: Major Depressive Disorder Phase I Trial
FDA approved treatment of the third and final cohort in the ongoing NSI-189/major depressive disorder (MDD) Phase Ib. Phase Ib
tested the safety of escalating doses of NSI-189 for 28 daily administrations in 24 depressed patients in three cohorts. NSI-189
is a proprietary new chemical entity that stimulates new neuron growth in the hippocampus, a region of the brain believed to be
implicated in MDD, as well as other diseases and conditions such as: traumatic brain injury, Alzheimer's disease, and post-traumatic
stress disorder (PTSD).
In April, Neuralstem
announced an initiative to investigate feasibility of an NSI-189 trial to treat cognitive and psychiatric impairment of former
NFL players from traumatic brain injury.
NSI-566 Research Papers
In May, a University of California, San
Diego study reported in STEM CELL RESEARCH AND THERAPY, showed that rats transplanted with NSI-566 cells three days after a spinal
cord injury showed improvement along several measures of motor function and a reduction of spasticity. The study, "Amelioration
of Motor/Sensory Dysfunction and Spasticity in a Rat Model of Acute Lumbar Spinal Cord Injury by Human Neural Stem Cell Transplantation,"
was led by principal investigator, Martin Marsala, MD, of the UCSD School of Medicine. The study demonstrated that intraspinal
grafting of NSI-566 cells during the acute phase of a spinal cord injury could represent a safe and effective treatment that ameliorates
post-injury motor and sensory deficits.
Licenses and Intellectual Property
In January, Neuralstem received issuance
of U.S. Patent #8,362,262 (Small Molecule: Divisional Compositions and Methods of Use), and issuance of PCT Patent #2337517 (Licensed:
Floating Cannula and Method of Use) and in four European countries: Germany, France, Italy and United Kingdom, for a total of five
company granted licenses to IP surrounding its spinal cord delivery platform, floating cannula, and method for delivering therapeutic
agents to the spinal cord to Cedars-Sinai Medical Center, a nonprofit academic medical center in Los Angeles, for academic research.
In March, Neuralstem received issuance
of EP Patent # 1576134 (Small Molecule: Method for Discovering Neurogenic Agents) and in ten European countries: Belgium, Switzerland,
Germany, France, United Kingdom, Ireland, Luxembourg, Netherlands and Sweden, for a total of 11 European patents.