New Patent Complements STX209 Orphan Drug Designation for Fragile X Syndrome
CAMBRIDGE, Mass., Mar 27, 2012 (BUSINESS WIRE) — Seaside Therapeutics, Inc. announced today that the United States Patent and Trademark Office (USPTO) has granted US patent No: 8,143,311, titled “Methods of Treating Fragile X Syndrome and Autism,” which covers the use of the Company’s lead product, STX209, for the improvement of social and communication functions in autism. Seaside’s lead clinical candidate, STX209, is an oral selective gamma-aminobutyric acid-B (GABA-B) receptor agonist and is currently being studied in a Phase 2b study in autism spectrum disorders and two Phase 3 studies in Fragile X syndrome.
“We believe STX209 has the potential to improve social function, which represents a new paradigm in the treatment of autism spectrum disorders and Fragile X syndrome, and are truly excited about the prospect of helping patients and their families achieve an improved quality of life,” said Randy Carpenter, MD, President and Chief Executive Officer of Seaside Therapeutics. “We continue to make excellent progress advancing the STX209 clinical program and securing this key piece of intellectual property is an important achievement as we drive these late stage clinical trials to completion.”
Seaside Therapeutics was the first to identify GABA-B as an important drug target in treating autism and Fragile X syndrome. Recent studies in the literature have demonstrated that there is deficient inhibitory neurotransmission in autism and Fragile X syndrome, and that STX209, a GABA-B agonist, has the potential to normalize this deficiency. In September of 2010, Seaside announced results from an open-label Phase 2a study of STX209 in autism spectrum disorders that demonstrated statistically significant improvements across a number of global and specific neurobehavioral outcomes, including significant improvements in social impairment–a core symptom of autism spectrum disorders. Social impairment includes symptoms such as preference to be alone, being withdrawn or isolated and lack of social reactivity. In July of 2010, Seaside announced results from a randomized, blinded, placebo controlled trial of STX209 program in Fragile X syndrome. Clinically meaningful improvements on global and specific neurobehavioral outcomes were observed in the general study population and these improvements were statistically significant in pediatric patients with more severe impairments in sociability. Additionally, in both studies STX209 was found to be well-tolerated. The most common adverse events were sedation and headache, which were generally mild and resolved spontaneously.
The claims of the US Patent No: 8,143,311 relate to methods to improve social and communication functions in autistic patients by administering a GABA agonist. This patent is currently set to expire in 2028. In addition, STX209 received Orphan Drug Designation from the U.S. Food and Drug Administration in 2008 and from the European Medicines Agency in 2011 for the treatment of Fragile X syndrome.
STX209 is an oral selective gamma-amino butyric acid type B (GABA-B) receptor agonist. Pathologies observed in certain neurodevelopmental disorders, including autism spectrum disorders and Fragile X syndrome are believed to be caused by excessive activation of glutamate receptors and abnormally high ratios of excitatory to inhibitory neurotransmission in the brain. GABA-B receptors play an important role in modulating the release of glutamate and optimizing the ratio of excitatory to inhibitory neurotransmission. STX209 has demonstrated efficacy in preclinical models, suggesting that it may improve function in individuals with autism spectrum disorders and Fragile X syndrome. With STX209, Seaside has successfully completed the largest, randomized, blinded, placebo-controlled trial (Phase 2) in patients with Fragile X syndrome and an open-label Phase 2a exploratory trial in patients with autism spectrum disorders. A Phase 3 study in adolescents and adults (ages 12 to 50) and children (ages 5 to 11) with Fragile X syndrome and a Phase 2b study in children, adolescents and adults (ages 5 to 21) with autism spectrum disorders are currently ongoing and open to enrollment.
About Autism Spectrum Disorders:
Autism Spectrum Disorders are characterized by three hallmark symptoms that can range from mild to disabling, including difficulties with social interaction, problems with verbal and nonverbal communication and repetitive behaviors or narrow, obsessive interests. Experts estimate that as many as 1 in 110 children are diagnosed with an autism spectrum disorder, with boys being four times more likely than girls to be diagnosed with the disorder. There is no cure for autism and there are currently no FDA approved therapeutics to treat the core symptoms of autism spectrum disorders.
About Fragile X Syndrome:
Fragile X syndrome is a neurodevelopmental disorder characterized by impaired social function, cognition and speech, as well as attention deficits and low functional independence. It is the most common inherited form of intellectual disability and affects roughly 100,000 individuals in the U.S. It is also the largest known cause of autism. Fragile X syndrome is caused by a mutation of a single gene, the Fragile X mental retardation 1 (FMR1) gene, on the X chromosome. The FMR1 gene produces a protein needed for normal brain development. Individuals with Fragile X syndrome lack this protein and, as a result, the majority of affected individuals will have significant intellectual disabilities and require life-time care. To date, there are no approved treatments for this disorder. The FDA and EMA have designated Fragile X syndrome as an orphan disease.
About Seaside Therapeutics:
Seaside Therapeutics, Inc. is creating novel drug treatments to correct or improve the course of autism, Fragile X syndrome and other neurodevelopmental disorders. The Company is dedicated to translating breakthrough discoveries in neurobiology into therapeutics that improve the lives of patients and their families. For more information please visit www.seasidetherapeutics.com.