Neuralstem, Inc. (Nasdaq:CUR) announced on 8/9/18 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.

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. 

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