Oxytocin delivered with the novel OptiNose nose-to-brain powder device - a novel therapy in Autism

PROJECT SUMMARY The project is pursuing the development of a treatment for Autism Spectrum Disorders (ASD as a CNS therapy for the application of the OptiNose technology for nose-to-brain delivery. known compound with proven pro-social effects will be tested with an OptiNose device that will be optimized for N2B delivery . High doses typically used today in studies with the compound represent a logistical challenge and increase the risk of adverse-events in long term use. These issues are major obstacles for the clinical development of this very promising compound into a commercial product. OptiNose in collaboration with SINTEF, will develop a specific device customized for nose-to-brain delivery. SINTEF will also be the partner for the optimization of the formulation Since June 2013 we have, in collaboration with Sintef, finalized the design and manufacture of the N2B nosepieces to be used in the clinical trial. We have successfully completed the pilot study and the phase 1 clinical study - including PK testing, fMRI and cognitive testing of subjects, in collaboration with Smerud ( CRO)and the Oslo University Hospital- (Prof Andreassen and assoc. Prof Westbye). We have analyzed the results that are very promising and the first article was published at a leading psychiatry scientific journal. A poster with the data has been presented at a prestigious psychiatry conference in Toronto. The fMRI results have been accepted for publication at a leading journal. We have completed a phase 2a/pilot study in patients to confirm the phase 1 findings prior to starting a full phase 2 study. The analysis of the data showed positive results regarding certain parameters of the cognitive function of ASD patients. A scientific publication is in the works.

The project is pursuing the development of a treatment for Autism Spectrum Disorders (ASD) as a CNS therapy for the application of the OptiNose technology for nose-to-brain delivery. A known compound with proven pro-social effects will be tested with an OptiNose device that will be optimized for N2B delivery . High doses typically used today in studies with the compound represent a logistical challenge and increase the risk of adverse-events in long term use. These issues are major obstacles for the cli nical development of this very promising compound into a commercial product. OptiNose in collaboration with SINTEF, will develop a specific device customized for nose-to-brain delivery. SINTEF will also be the partner for the optimization of the formulati on. In the clinical program OptiNose will collaborate with Oslo University Hospital and Smerud Clinical Research. The Phase 1 trial we l assess pharmacokinetics and explore the effects on brain activity and cognition by applying advanced fMRI in conjuncti on with validated cognitive tests. The Phase 2a trial will test the effect on cognitive and social symptoms in ASD patients. Positive outcome of these trials would make it possible to attract the funds for further development required for marketing appro val and commercialization, a major step towards a much needed therapy for ASD patients. The market potential of such product exceeds $1 Billion per year in sales. In addition, demonstration of N2B delivery in this project will represent a major breakthro ugh and will offer a new treatment modality in many other brain disorders.