Full Press Release Details
| Deanne Eagle - Media Relations | 917.837.5866 |
| Susan Roush - Investor Relations | 818.222.8330 |
NEURALSTEM ANNOUNCES 2012 FINANCIAL RESULTS,
PROVIDES CLINICAL TRIALS UPDATE
ROCKVILLE, MD, March 15, 2013 --
Neuralstem, Inc. (NYSE MKT: CUR) today provided an update on its clinical trial programs and reported its 2012 financial results
for the year ended December 31, 2012.
"2012 saw the company achieve success
in all of its Phase I clinical trial objectives," said Karl Johe, Ph.D., Neuralstem's Chairman of the Board and Chief
Scientific Officer. "We have been able to demonstrate the safety and tolerability of Neuralstem's novel core technologies,
from intraspinal transplantation procedures, to the cells themselves in ALS patients, as well as our NSI-189 neurogenic small molecule
drug in healthy volunteers. Additionally, we believe we have seen evidence of a treatment effect in some NSI-566 cell therapy patients
over a sustained period of time, as measured by levels of functional recovery and a slowdown in the progression of ALS. In spinal
cord injury, a leading peer-reviewed scientific journal, CELL,' published compelling evidence that NSI-566 cells can
bridge the gap' in a severed spinal cord animal model and return functionality. We have recently been approved by
the FDA to commence a trial treating chronic spinal cord injury patients.
A highlight of 2012 was the completion
of our ground-breaking ALS Phase I trial. Following the last treatment of the final cervical cohort of return patients in August,
our collaborators, University of Michigan's Dr. Eva Feldman and Emory University's Dr. Jonathan Glass, presented trial
data that showed promise of a treatment effect for ambulatory ALS patients as well as definitive DNA-fingerprint evidence of long-term
NSI-566 cell survival," continued Dr. Johe. "These positive data support our plan to accelerate the ALS trial in Phase
II, by increasing both the number of NSI-566 cells and number of injections delivered to the cervical spinal cord, where we believe
we can most positively affect patients' lives by sustaining their breathing capacity. Our proposed Phase II trial protocol, which
would be simultaneously conducted at Emory University Hospital and University of Michigan, is currently in review at the FDA. The
National Institutes of Health and ALSA have committed to generous grants totaling nearly $3,000,000 in funding for this next phase
of the study, pending FDA approval. We join with our esteemed collaborators in being eager to move forward to future trial phases
to examine therapeutic efficacy of NSI-566. We hope to commence the ALS Phase II trial in the second quarter."
Dr. Johe continued, "2013 promises
to be a transformative year for the company, with five NSI-566 cell therapy trials planned. The two new U.S. trials will be the
ALS Phase II, and the recently FDA-approved Phase I in chronic spinal cord injury. We hope to have agreements with multiple sites
for the Phase I chronic spinal cord injury trial in place by the end of the 2nd Quarter and then begin the transplantations. Internationally,
our ischemic stroke trial is expected to commence in Beijing at world-class BaYi Brain Hospital in the coming weeks, through our
subsidiary, Neuralstem China. A planned ALS combined Phase I/II is expected to take place in Mexico City, pending finalization
of a partnership agreement. Later this month, we expect to file an IND for an acute spinal cord injury trial in Seoul, South Korea,
which we anticipate conducting with our partner, CJ CheilJedang.
"2012 also saw advances through the
clinic for NSI-189, the company's lead compound in our first-in-class neurogenic small molecule drug," Dr. Johe commented
further. "The FDA approved dosing the second cohort of eight depression patients in our ongoing NSI-189/major depressive
disorder (MDD) Phase Ib trial. Dosing has now increased from 40 mg. q.d.(once/day) for 28 days, which had been shown to be well-tolerated
and safe in the first cohort, to 40 mg. b.i.d.(twice/day) for 28 days. Dosing of all patients in the second cohort will be completed
this month and, pending FDA approval, we will commence dosing the final cohort."
Neuralstem's President and CEO Richard
Garr added, "Neuralstem continued to strengthen its patent estate in 2012. Among the highlights, U.S. Patent number 8,236,299
includes claims covering processes for dissociating our neural stem cells from CNS 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, chronic and acute spinal cord injury, and stroke. Both the substance and life of this patent (into 2030) are reflective
of the Intellectual Property value we are creating in our cell therapy programs. Additionally, during the past 12 months we have
executed the first two out-licenses of our spinal platform cell therapy technology."
"Dr. Johe and I would like to extend
our continued deep appreciation to our patients, their families and caregivers, our world-class clinical collaborators, and the
Neuralstem team for enabling positive, ground-breaking clinical work which has now positioned us to move forward," added
NSI-566 Phase I Clinical Trial in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease) at Emory University Hospital
Neuralstem completed the final surgery of its Phase I NSI-566/ALS trial in August. The trial proved the safety of the company's
proprietary technology, the cells and proprietary intraspinal surgical procedure. Data presented at the International Symposium
on ALS/MND in December (2012) showed evidence of long-term NSI-566 cell survival in patients, through DNA fingerprinting. It was
further announced that the study team has received a grant from the National Institutes of Health (NIH) to cover a majority of
the cost of an upcoming NSI-566/ALS Phase II trial. In October, Eva Feldman, M.D., Ph.D., the trial's principal investigator
and Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of
Michigan Health System, presented updated data at the American Neurological Association (ANA) annual meeting. Dr. Feldman, who
is president of the ANA, presented interim results on all 18 procedures in 15 patients, and said, " we are exploring
a paradigm shift in the treatment of ALS. Although this phase of the trial was not powered to demonstrate efficacy, we
appear to have interrupted the progression of the disease in one subgroup of patients." The Phase I NSI-566/ALS safety trial
consisted of 18 treatments in 15 patients, at Emory University Hospital. All injections delivered 100,000 cells, for a dosing range
of up to 1.5 million cells. In February, Patient 14 was the second patient to be dosed in the cervical (upper back) region where
the cells could support breathing, the key function that is lost as ALS progresses. In May, the
FDA approved additional NSI-566 dosing of patients who had previously received lumbar injections, to return for cervical injections
as the trial's final cohort; the FDA-approved trial amendment was subsequently approved by the Emory
University Institutional Review Board in June. Additionally, Neuralstem had submitted
a trial amendment to the FDA to increase both the number of patients treated as well as the dose in future cohorts, which was then
withdrawn and modified to become the Phase II protocol. The entire Phase I ALS trial officially concluded in February 2013, six
months after the final surgery.
Small Molecule NSI-189: Phase I Clinical Trial in Major Depressive Disorder (MDD)
- The company's lead neurogenic
small molecule compound, NSI-189, advanced with FDA approval to treat the second cohort of eight depression patients in the ongoing
NSI-189/major depressive disorder (MDD) Phase Ib clinical trial, in October. NSI-189 was shown to be well-tolerated and safe during
the 28-day dosing of the first cohort, in the randomized, double-blind, placebo-controlled, multiple-dose escalating trial. The
24-patient Phase Ib commencement was announced in June, with efficacy data blinded for all cohorts until the end of the trial.
Neuralstem is expected to complete the entire NSI-189/MDD Phase I trial in the third quarter of 2013. This proprietary new chemical
entity increases hippocampal volume and stimulates new neuron growth in the hippocampus, a region of the brain believed to be implicated
in MDD, as well as other cognitive and psychological diseases and conditions such as chronic traumatic encephalopathy (CTE), Alzheimer's
disease, and post-traumatic stress disorder (PTSD). Data collection includes post-dosing MRIs at both four and eight weeks after
completion of the 28-day dosing.
- In February, the company raised
approximately $5.2 million in gross proceeds through an offering of its common stock to fund its ongoing clinical trials and working
capital for general corporate purposes.
In March, the science journal, "STEM CELLS," published early Neuralstem NSI-566/ALS Phase I interim results, entitled,
"Lumbar Intraspinal Injection of Neural Stem Cells in Patients with ALS: Results of a Phase I Trial in 12 Patients."
"STEM CELLS" reported that one patient had shown dramatic improvement in his clinical status, and that there was no
evidence of accelerated disease progression due to the intervention in any of the 12 patients (who were followed from 6-18 months
after injections at this point), nor any long-term complications related to either the surgery or the cells.
- In July, Neuralstem received a notice
of issuance for patent application number 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, ischemic stroke, and chronic and acute spinal cord injury.
- Also in July, the company extended the employment contracts
of Dr. Karl Johe, Richard Garr, and Dr. Thomas Hazel for an additional 60 months.
- In September, Neuralstem received
approval to commence a combined Phase I/II clinical trial to treat motor deficits due to ischemic stroke with direct injections
into the brain of NSI-566 cells, at BaYi Brain Hospital, in Beijing, through its subsidiary, Neuralstem China
(Suzhou Neuralstem Biopharmaceutical Company, Ltd.). The trial is designed to enroll up to 118 patients, and involve one-time intracerebral