Autism spectrum disorder (ASD) is a collection of development disorders characterized by difficulties in social interaction, verbal and nonverbal communication and repetitive behaviors. In addition to social deficits, ASD can also result in intellectual delays, irritability, problems with motor coordination, and additional physical ailments including gastrointestinal issues. It is estimated that there are over 4M patients worldwide with ASD and the incidence rate is rising rapidly. The estimated lifetime healthcare and societal cost of ASD is $3.2MM per patient indicating a real need for effective ASD therapies. The therapeutic market for ASD is expected to grow to over $700MM by 2017. There are currently two FDA approved therapeutics for ASD (Risperdal® and Abilify®). However, they treat only the irritability component of ASD.
While the etiology of ASD is still unknown, there is evidence that some of the core characteristics of ASD, including repetitive behaviors, are due to an imbalance in the serotonin system. Dr. Hollander and colleagues validated the use of the selective serotonin-reuptake inhibitor fluoxetine to treat ASD in a pilot double-blind placebo controlled human proof-of-concept study. The outcome indicated treatment with fluoxetine reduced ASD-associated repetitive behaviors. Over 11 clinical trials of fluoxetine in autism have been conducted.
Current Development Status
- Pivotal Phase III clinical trial designed in accordance with outcome of Type C meeting with FDA on necessary trial parameters
- Reformulated as orally-disintegrating cherry-flavored tablet in 2,4, and 9mg dosages
- Seeking partner to support conduct of pivotal Phase III clinical trial and eventual marketing
Applications
- Autism spectrum disorder
Advantages
- Fluoxetine (Prozac®); FDA approved anti-depressant for a variety of indications
- Generic (10mg and 20mg tablets and liquid formulations)
Publications
- Hollander, E. et. al. “A double-blind placebo-controlled trial of fluoxetine for repetitive behaviors and global severity in adult autism spectrum disorders. Am J Psychiatry. 2012. 169(3):292-299
Intellectual Property Status
- Received Orphan Drug Designation
Contact Information
Lisa Placanica, PhD
Business Development Director
Mount Sinai Innovation Partners | Icahn School of Medicine at Mount Sinai
Phone: 646.605.7325