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[1]. 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.

[1] (Bartz 2008, Ishak 2010, Feifel 2010, Neumann 2008, Kosfeld 2005)