Neuralstem Announces NSI-189 Granted Orphan Drug Designation from the U.S. FDA for the Treatment of Angelman Syndrome
09 August 2018 - 9:00PM
Neuralstem, Inc. (Nasdaq:CUR), a biopharmaceutical company focused
on the development of nervous system therapies based on its neural
stem cell technology, today announced that the U.S. Food and
Drug Administration has granted orphan drug designation to NSI-189
for the treatment of Angelman syndrome.
“Angelman Syndrome is a rare disease with significant unmet
medical need, and for which there are no FDA-approved therapies,”
said Jim Scully, Chief Executive Officer of Neuralstem. “Orphan
drug designation is an important regulatory milestone in the
development of NSI-189 and we are committed to evaluating NSI-189’s
role as a treatment to improve the lives of patients with Angelman
syndrome.”
In pre-clinical models, NSI-189 has demonstrated the ability to
restore long term potentiation (LTP), a measure of synaptic
plasticity and an in vitro biomarker of memory.
The FDA's orphan-drug designation program provides special
status and incentives to encourage the development of drugs for
diseases affecting fewer than 200,000 people in the U.S. Orphan
drug designation confers seven years of market exclusivity upon FDA
approval, as well as other development incentives, such as tax
credits related to clinical trial expenses, an exemption from the
FDA-user fee and FDA assistance in clinical trial design.
About Angelman Syndrome
Angelman Syndrome (AS) is a rare congenital genetic disorder
caused by a lack of function in the UBE3A gene on the
maternal 15th chromosome. It affects approximately one
in 15,000 people - about 500,000 individuals globally.
Symptoms of AS include developmental delay, lack of speech,
seizures, and walking and balance disorders. Patients with AS may
never walk or speak and require life-long care. Life expectancy is
normal which places a significant burden on patients and
caregivers. There are currently no FDA-approved therapies for
the treatment of Angelman syndrome.
About Neuralstem
Neuralstem is a clinical-stage biopharmaceutical company
developing novel treatments for nervous system diseases of high
unmet medical need. The Company has two lead development
candidates:
- NSI-189 is a small molecule in clinical development for major
depressive disorder and in preclinical development for Angelman
syndrome, irradiation-induced cognitive impairment, Type 1 and Type
2 diabetes, and stroke.
- NSI-566 is a stem cell therapy being tested for treatment of
paralysis in stroke, Amyotrophic Lateral Sclerosis (ALS), chronic
spinal cord injury (cSCI), and traumatic brain injury (TBI).
NSI-566 was shown to have restorative function in a primate
paralysis model. The study was published in the peer reviewed
journal Nature Medicine, and the full manuscript can be found
here.
Neuralstem’s diversified portfolio of product candidates is
based on its proprietary neural stem cell technology.
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, 2017 filed with the Securities and Exchange
Commission (SEC), and in other reports filed with the SEC. We do
not assume any obligation to update any forward-looking
statements.
Contact:Kimberly MinarovichArgot Partners (Investor
Relations)212-600-1902neuralstem@argotpartners.com
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