Retrophin, Inc. (OTCQB:RTRX) today announced that it has signed an agreement with Novartis Pharma AG for an exclusive U.S. license for Syntocinon™ Nasal Spray, the intranasal formulation of a synthetic version of the naturally occurring peptide hormone oxytocin, for an upfront payment of $5.0 million plus milestone payments and royalties.
Syntocinon™ Nasal Spray was approved in the U.S. in 1960 to assist initial postpartum milk ejection, but was discontinued by Novartis in 1997 for commercial reasons. Retrophin plans to reintroduce the product for prescription use in the U.S. in Q2 2014. Additionally, the company intends to pursue a clinical trial program with Syntocinon™ for its potential use as a treatment for schizophrenia and autism.
Difficulty breastfeeding newborn infants is an unmet medical need. There is no FDA-approved drug currently available in the U.S. for this patient population. In a Retrophin-conducted survey of 50 obstetricians, respondents indicated that 16% of new mothers in their care have problems with milk let-down, and 79% of these physicians said they would use Syntocinon™ if it became available for this indication.
One percent of Americans suffer from schizophrenia according to National Institute of Mental Health estimates. Over the past four years, three randomized, double-blind, placebo-controlled, independent proof-of-concept schizophrenia trials were conducted in patients whose symptoms were inadequately controlled despite receiving therapeutic doses of an atypical antipsychotic. All three studies suggest that intranasal oxytocin administered as an adjunct to subjects' antipsychotic drugs improves positive and negative symptoms, as assessed by the Positive and Negative Syndrome Scale (PANSS), significantly more than placebo.
Preclinical evidence indicates that oxytocin has a critical role in the regulation of brain-mediated processes that are strongly relevant to many neuropsychiatric disorders. Small studies suggest that even a single dose of intranasally delivered oxytocin can have significant, pro-social effects on human behavior. Finally, there have been several positive findings with oxytocin in animal models of schizophrenia.