Description:

Nastech has initiated a program for the development of a nasal dosage form of Carbetocin, a long-acting analog of the naturally produced hormone, oxytocin. This program is intended to address significant unmet medical needs related to the treatment of core symptoms of autism and possibly other psychiatric disorders.

Oxytocin receptors are expressed in brain regions associated with social behavior and emotion. Dr. Eric Hollander, Professor and Chair of Psychiatry at Mount Sinai's School of Medicine's Seaver and New York Autism Center of Excellence, reported that oxytocin may help reduce debilitating symptoms in those diagnosed with autism. Nastech's proprietary drug delivery technology has the potential to efficiently deliver Carbetocin via a patient-friendly nasal spray. An injectable form of Carbetocin is already approved in several countries for treatment of specific postpartum complications

Development Status:

Several Phase I pharmacokinetic studies are planned, and patient dosing for the first of these has been completed. Analysis of patient samples is currently in progress.

Clinical Need:

Autism is the most common condition in a group of developmental disorders known as the autism spectrum disorders (ASDs). Autism is characterized by three distinctive behaviors. Those suffering from autism have difficulties with social interaction, display problems with verbal and nonverbal communication, and exhibit repetitive behaviors or obsessive interests. These behaviors can range in impact from mild to disabling. Autism varies widely in its severity and symptoms and may go unrecognized, especially in mildly affected children or when more debilitating handicaps mask it.

According to the Centers for Disease Control, approximately 1 in 150 children have an autism spectrum disorder by the age of eight. Current treatment options include highly structured educational programs, behavioral interventions, medications and dietary modifications. Medications are often used to treat behavioral symptoms such as aggression, self-injurious behavior and severe tantrums that keep the person with ASD from functioning effectively at both home and in school. Medications in current use have been developed to treat similar symptoms in other disorders, but there is no approved drug to treat the core symptoms of autism.

Partnership Status:

Currently unpartnered

External Links:

• Autism Society of America
• National Institutes of Health
• National Institute of Neurological Disorders and Stroke