Oxytocin is a small, naturally occurring peptide currently used to stimulate lactation in breastfeeding women. It has recently attracted attention as a potential novel treatment alternative in several brain disorders, including autism. Because oxytocin is a peptide and has very poor oral bioavailability, nasal delivery has been investigated as a means of improved delivery. With standard liquid spray delivery only roughly 3 percent of the drug reaches the systemic circulation. It is estimated that only a tiny fraction (less than 0.01 percent) of oxytocin in the blood enters the brain across the blood brain barrier.
OptiNose undertook a Phase I trial in late 2013 to investigate “nose-to-brain” transport of oxytocin via the patented OptiNose Bi-Directional™ Breath Powered delivery technology. The OptiNose device offers the potential for a more efficient and consistent direct transport of oxytocin into the brain itself, using relatively low doses — which will significantly reduce drug levels in the rest of the body, reducing the risk of side-effects.
Effects of Intranasal Administration of a Single Dose of Oxytocin Using a Novel Device in Healthy Adults: This placebo-controlled study of 19 adults was designed to identify any differences between single dose 8 or 24 international units (IU) oxytocin delivered via the OptiNose device and 1 IU oxytocin administered as slow intravenous infusion. Results will be measured in terms of brain activity as measured with functional magnetic resonance imaging (fMRI), performance on cognitive tests, and physiological markers.
 (Bartz 2008, Ishak 2010, Feifel 2010, Neumann 2008, Kosfeld 2005)