Kyalin Biosciences Inc. Intranasal Carbetocin Fact Sheet
Kyalin Biosciences, Inc. lead asset, a highly optimized, intranasal delivery form of carbetocin, represents a potential breakthrough treatment for the core deficits that characterize the autistic spectrum disorders. Intranasal carbetocin leverages the natural biology of oxytocin, the 'trust hormone' shown to promote social behavior in hundreds of studies. Intranasal carbetocin is a long-acting, synthetic oxytocin analog, optimized for intranasal delivery thanks to years of work and investment in formulation development. It is a clinic-ready asset, needing only $1M to finish a key proof-of-concept study that wil focus on comparing the biological and functional activities of intranasal carbetocin to those of intranasal oxytocin.
Unmet Need
Autistic spectrum disorders (ASD) are characterized by the presence of three core symptom clusters: social deficits, repetitive behaviors, and abnormalities in language. While autism affects 1 in 110 children in the U.S. (CDC, 2009), there are no approved therapies for the core social and repetitive behavior deficits associated with autism. Recent studies estimate that the lifetime societal cost of an individual with autism is $3.2 mil ion, with lost productivity and adult care comprising the largest components of costs.
Well-characterized
Carbetocin is a synthetic version of the endogenous hormone, oxytocin, structural y modified to obtain a longer serum half-life, improved receptor selectivity, and improved potency. This compound was original y developed by Ferring Pharmaceuticals and is sold in multiple countries outside of the U.S., solely in a parenteral form for the treatment of postpartum hemorrhage. Marina Biotech—with its expertise in intranasal peptide delivery originating from its predecessor company, Nastech Pharmaceuticals—developed an intranasal formulation of this agent with the goal of assessing its efficacy for the treatment of autism.
Near-term milestone
We currently anticipate initiating a proof-of-concept trial (involving normal healthy volunteers) in 2011 (funding permitting) and expect results from such studies to become available in 2012.
Market Opportunity
The atypical antipsychotics presently dominate this market, both in prescription share and sales. However, these agents only address the irritability associated with autism, not its core symptoms. Two drugs, Risperdal™ and Abilify™, have been specifical y approved for the treatment of the irritability associated with autism in children. Given the limited treatment options available, the current market for drug therapies to treat autism is small—reaching just $194 mil ion in 2008 in the United States, France, Germany, Italy, Spain, United Kingdom, and Japan combined—with Risperdal representing the market leader.
The United States is the most significant market for ASD, claiming a 74% market share. This discrepancy between the United States and other geographic regions does not reflect any significant difference in patient population or prescribing patterns, but rather the substantial y lower price of Risperdal in these other markets.1 Global y, the US remains the largest market for autism therapeutics, and was valued at $189m in 2009. It is forecast to grow over the next seven years to reach $442m by 2016.2
Selected Publications
Insel,Thomas. The Chal enge of Translation in Social Neuroscience: A review of Oxytocin, Vasopressin, and Affiliative Behavior. Neuron. 2010 vol. 65 (6) pp. 768-779
MacDonald, K, MacDonald T. The Peptide That Binds: A Sytematic Review of Oxytocin and it Prosocial Effects in Humans. Harv Rev Psychiatry. 2010 vol. 18 (1) pp. 1-21
CDC: Prevalence of autism spectrum disorders – autism and developmental disabilities monitoring network, United States, 2006. MMWR Surveil ance Summaries, December 18, 2009; 58(SS10): 1-20.
1 SPECTRUM Therapeutics Markets – Autism. Decision Resources. August 2009.
2 www.articlebase.com Contact for More Information: Srinivas Rao, Kyalin Biosciences, Inc. Phone: 858-779-4253, [email protected]
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