Full Press Release Details
| Deanne Eagle - Media Relations | 917.837.5866 |
| Susan Roush - Investor Relations | 818.222.8330 |
NEURALSTEM REPORTS THIRD QUARTER FINANCIAL
RESULTS AND PROVIDES UPDATE ON CLINICAL PROGRAMS AND BUSINESS HIGHLIGHTS
Maryland, November9, 2012-Neuralstem, Inc. (NYSE MKT: CUR) reported its financial results for the three months and nine
months ended September 30, 2012 and provided an update on clinical programs and business highlights.
the third quarter, the company achieved three significant clinical milestones. The first was the completion of the eighteenth and
final surgery in our ALS Phase I trial. All ALS trial assessments show the NSI-566 neural stem cells and intraspinal transplantation
procedure, the first in the world, to be safe. The trial's principal investigator, Dr. Eva Feldman, saidshe believes we are
seeing evidence of a treatment effect in some Phase I patients over a sustained period of time. We look forward to advancing tothe
next ALS trials, to increase both number of patients treated and NSI-566 dosage, and effectively evaluate efficacy. To that end,
we are working diligently with our investigators to file an IND with the FDA for a Phase IIALS trial by year-end," said Karl
Johe, PhD, Chairman of Neuralstem's Board of Directors and Chief Scientific Officer.
"The second significant clinical
milestone was approval to begin our ischemic stroke trial in China. Our collaborators in Beijing, at the world-class BaYi Brain
Hospital are on schedule to start enrollmentearly in the first quarter for a combined Phase I/II clinical trial to treat motor
deficits due to ischemic stroke with NSI-566. The first part of the study, Phase I, will be open-label and enroll up to 18 patients
who will be assigned to one ofthree cohorts. Each cohort will receive ascending doses of NSI-566 directly into the brain at the
stroke area (another first for Neuralstem), to define the maximal safe dose, before proceeding tothe Phase II/proof-of-concept
study to evaluate efficacy," said Dr. Johe. "One of several independent Neuralstem studiespresented at The Society
for Neuroscience's annual meeting demonstrated significant recovery of motor and neurological functions in ischemic stroke
rats with NSI-566; a second ischemic stroke study demonstrated safety and feasibility in a chronic model in mini-pigs. Together,
with the extensive human safety data established in our ALS trial, these two studies demonstrate strong proof-of-principle data
that our NSI-566 cells are ready to test in humans to treat paralysis in stroke patients. A rat spinal cord injury study, reported
in the peer-reviewed scientific journal CELL,demonstrated that NSI-566 cells induce regeneration of injured spinal cord
axons into the graft and serve as a bridge to reconnect to gray matter motor neurons for many spinal cord segments below an injury,
inducing significant recovery of motor functions."
Dr. Johe concluded: "The third significant
clinical milestone was the FDA approval for dosing the second cohort of major depressive disorder patients in the Phase Ib trial
of NSI-189. Our lead neurogenic drug, NSI-189 is a first-in-class orally active drug that stimulates new neuron growth and increased
volume in the hippocampus, which we believe can help patients with MDD and other psychological and cognitive conditions. We are
on track to complete the Ib, an ascending-dose, 28-day-cycle safety trial designed in collaboration with Dr. Maurizio Fava of Harvard
University and Massachusetts General, during the first quarter of 2013."
Neuralstem's President and CEO Richard
Garr added: "The third quarter of 2012 marked business milestones as well.We saw a major addition to our patent estate with
U.S. patent number 8,236,299, with a patent life into 2030, titled: Transplantation of Human Neural Cells for Treatment
of Neurodegenerative Conditions.' This is significant in that it covers processes involved in our first potential transplantation
product, including claims covering processes for dissociating our neural stem cells from central nervous system tissue; culturing
the cells; expanding the cells in vitro and transplanting the cells into the spinal cord of a patient to treat a wide array of
neurodegenerative conditions, including ALS."
Mr. Garr continued: "The company
granted its first licenses for use of our proprietary Spinal Cord Delivery Platform and Floating Cannula. As the industry standard
for delivering therapeutic agents to the spinal cord, we expect to continue to announce licenses of the surgical devices to both
industry and academia as they now begin to explore the possibilities our technology has enabled. We are also continuing discussions
for licensing and partnership opportunities for our neurogenic small molecule program, for which we are seeing strong interest.
Our goal is to move NSI-189 and our preclinical library of additional novel neurogenic compounds forward in multiple indications,
which range from Alzheimer's disease and anti-aging (nootropic) to chronic traumatic encephalopathy and post-traumatic stress
disorder. We believe this can best be accomplished through partnering with industry."
and Business Highlights
NSI-566 Phase I Clinical Trial in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease) at Emory University Hospital
Neuralstem completed the final transplantation of its ground-breaking Phase I safety trial of NSI-566 spinal cord neural stem cells
for the treatment of ALS. Phase I is scheduled to conclude six months after this final surgery of returning patient #18.
patient 17 was the second ALS patient to return to the trial, receiving five injections in the cervical region in addition to the
ten previously received in the lumbar region of the spinal cord, for a total of 15 injections of neural stem cells.
Event: In October, principal investigator and president of the American Neurological Association (ANA) Eva Feldman, MD, PhD, updated
data on the trial at the ANA annual meeting. She called the transplantations "a paradigm shift in the treatment of ALS"
and presented interim results on all 18 procedures in 15 patients.
NSI-566 Phase I/II Clinical Trial in Ischemic Stroke at BaYi Brain Hospital
Neuralstem was approved to commence a clinical trial to treat motor deficits due to ischemic stroke with NSI-566 at BaYi Brain
Hospital, in Beijing, China, through its subsidiary, Neuralstem China. The trial approval includes a combined phase I/II design
of up to 118 patients, and will test direct injections into the brain of NSI-566. It is scheduled to commence in the first quarter
NSI-566 Pre-clinical Programs
a peer-reviewed article in the leading scientific journal CELL reported that Neuralstem NSI-566 cells induced significant functional
improvement in permanent rat spinal cord injury, with paralyzed rats regaining use of lower limbs. The study, entitled "Long-Distance
Growth and Connectivity of Neural Stem Cells After Severe Spinal Cord Injury: Cell-Intrinsic Mechanisms Overcome Spinal Inhibition,"
reports that the animals recovered significant locomotor function, regaining movement in all lower extremity joints, and that the
transplanted neural stem cells turned into neurons which grew a "remarkable" number of axons that extended for "very
long distances" over 17 spinal segments, making connections both above and below the point of severance.
Event: In October, NSI-566/animal ischemic stroke data was presented in two independent studies at The Society for Neuroscience's
Annual Meeting. "Histopathological Assessment of Adult Ischemic Rat Brains after 4 Weeks of Intracerebral Transplantation
of NSI-566RSC Cell Line," conducted in the laboratory of Dr. Cesar Borlongan at the Center of Excellence for Aging and Brain
Repair at the University of South Florida College of Medicine, showed significant recovery of motor and neurological functions
in ischemic stroke rats with NSI-566. The other ischemic stroke study presented was conducted by Dr. Martin Marsalaat the Neuroregeneration
Laboratory at University of California San Diego, and entitled "Survival and Differentiation of Human Neural Stem Cells (NSI-566RSC)
After Grafting into Ischemia-Injured Porcine Brain." Neuralstem presented three additional posters of preclinical data at
Small Molecule NSI-189: Phase I Clinical Trial in Major Depressive Disorder (MDD)
-Subsequent Event: In October, Neuralstem
began dosing the second cohort of eight patients, upon receiving FDA approval, in the Phase Ib trial. The trial is testing the
safety of escalated dosing for 28 daily administrations of the company's lead neurogenic small molecule drug, NSI-189, in
the treatment of major depressive disorder.
- In September, Neuralstem granted
the first licenses for use of its Spinal Cord Delivery Platform and Floating Cannula, used in the ALS Phase I trial for delivering
therapeutic agents to the spinal cord.
- In September, Chairman and Chief
Scientific Officer Dr. Karl Johe presented "Novel Neurogenic Drugs Targeting Hippocampal Degeneration" at the 6th
Neurodegenerative Conditions Research and Development conference, and President and CEO Richard Garr presented corporate and clinical
updates at the 2012 Aegis Healthcare Conference and the 2012 Rodman and Renshaw Annual Healthcare Conference.
-In July, Neuralstem received a notice
of issuance for patent applicationnumber 12/710,097 (subsequently issued patent number 8,236,299) titled: "Transplantation
of Human Neural Cells for Treatment of Neurodegenerative Conditions." This patent, which will expire in the first quarter
of 2030, covers both the culturing of central nervous system (CNS) cells as well as their transplantation into spinal cord tissue
to treat neurodegenerative conditions, including ALS.
- During the quarter, the company
raised $9.76 million through offerings of its common stock to fund its ongoing clinical trials, research and development, and working
- Subsequent Events: In October, Neuralstem
engaged Locust Walk Partners, LLC, a life sciences' commercial licensing and partnering specialist advisory firm, to further
explore partnership opportunities for a clinical-stage neurogenic small molecule program targeting a broad range of psychiatric
and cognitive disorders.
- Subsequent Events: In October, Neuralstem
received a notice of issuance for Vietnam patent number 10524 titled: "Transplantation of Human Neural Cells for Treatment
of Neurodegenerative Conditions." This issuance bringsNeuralstem's worldwide patent count to 28 issued and 43 pending.