Neuralstem Announces NSI-189 Granted Orphan Drug Designation from the U.S. FDA for the Treatment of Angelman Syndrome

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 powder 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.

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