Neuralstem has announced that their investigational therapy NSI-189 has been granted Orphan Drug designation for the treatment of Angelman syndrome (AS).

NSI-189 is a small molecule that is also currently in clinical development for major depressive disorder. For AS, the treatment is in pre-clinical stages; the Company states that NSI-189 has demonstrated the ability to restore long term potentiation, a measure of synaptic plasticity and an in vitro biomarker of memory. 

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AS is a rare genetic disorder that, according to the Angelman Syndrome Foundation, affects as many as 5,000 people in the US and Canada. Delay or lack of speech, seizures, and walking/balance disorders are some of the symptoms associated with AS. Orphan Drug status provides incentives to companies to develop treatments affecting ≤200,000 individuals. 

“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,” said Jim Scully, CEO of Neuralstem.

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