LA JOLLA, Calif., Jan. 14, 2015 (GLOBE NEWSWIRE) -- Auspex Pharmaceuticals, Inc. (Nasdaq:ASPX), a biopharmaceutical company dedicated to developing innovative medicines for people with movement disorders and other rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Auspex's investigational compound SD-809 for the treatment of Tourette syndrome in the pediatric population (defined as zero through 16 years of age). Auspex is currently evaluating the preliminary efficacy, pharmacokinetic and safety profile of SD-809 in a Phase 1b clinical trial for the treatment of tics associated with Tourette syndrome. Topline data from this trial are expected by mid-2015. In November 2014, SD-809 was granted orphan drug designation by the FDA for the treatment of Huntington's disease.
"Receiving orphan drug designation of SD-809 for the treatment of Tourette syndrome, in addition to Huntington's disease, is an important step forward in our goal of bringing this novel treatment approach to the patients and families affected by these serious disorders," said Pratik Shah, president and CEO of Auspex. "There have been no new drugs developed for Tourette syndrome in over 30 years. The orphan drug designation recognizes the significant need that exists among children living with this debilitating disease, and we look forward to assessing the potential role that SD-809 may have in addressing that."
The FDA's Orphan Drug program offers orphan status to drugs and biologics that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the U.S. The designation provides sponsors with development and commercial incentives for designated compounds and medicines, including eligibility for a seven-year period of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design and an exemption from FDA user fees.
About Tourette Syndrome
Tourette syndrome is a hyperkinetic movement disorder manifested by motor and phonic tics, which are often accompanied by neurobehavioral disorders such as attention-deficit hyperactivity disorder and obsessive-compulsive disorder. Tics can be simple, such as blinking, eye rolling, nose twitching, head nodding and mouth pouting, or more complex, such as touching, squatting, jumping or hopping. Tics can result in significant long-term social, legal and developmental consequences for patients, as well as injury and physical disability including pain and secondary neurological deficits. In the U.S., an estimated 100,000 children have tics associated with Tourette syndrome. According to the U.S. Centers for Disease Control and Prevention, 37% of these children have moderate to severe forms of Tourette syndrome. The mean age of onset is at four to six years, with peak severity around 12 years of age, with an estimated 13% to 22% of affected children continuing to take medications for tics as adults. There have been no new drugs introduced for treating tics associated with Tourette syndrome in more than 30 years and we believe that physicians consider the two approved neuroleptics to be inadequate. These treatments carry, among other adverse events, the risk of causing permanent neurologic deficits, such as tardive dyskinesia.
About Auspex Pharmaceuticals
Auspex Pharmaceuticals is a biopharmaceutical company dedicated to developing innovative medicines for hyperkinetic movement disorders and other rare diseases. Auspex employs its proprietary technology to create patent-protected, new chemical entities from known, clinically proven pharmacologics. The company's lead product SD-809 is in the final stages of development for the treatment of chorea associated with Huntington's disease, a neurodegenerative movement disorder that impacts cognition, behavior and movements. In addition, Auspex is investing in the broad potential of SD-809 for the treatment of other movement disorders, including tardive dyskinesia and tics associated with Tourette syndrome. The company's pipeline also includes SD-560, which is being developed for potentially treating fibrotic conditions, and SD 1077 for the potential treatment of Parkinson's disease. For further information, please visit the company's website www.auspexpharma.com.
Forward-Looking Statements
Statements made in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding Auspex's ability to successfully complete its ongoing clinical trials and development programs, Auspex's ability to obtain regulatory approval for its product candidates and market penetration and acceptance of its product candidates. Risks that contribute to the uncertain nature of the forward-looking statements include: Auspex's future preclinical studies and clinical trials may not be successful; changes in regulatory requirements in the United States and foreign countries may prevent or significantly delay regulatory approval of Auspex's product candidates; Auspex may change its plans to develop and commercialize its product candidates; the U.S. Food and Drug Administration (FDA) may not agree with Auspex's interpretation of the data from clinical trials of its product candidates; Auspex may decide, or the FDA may require Auspex, to conduct additional clinical trials or to modify Auspex's ongoing clinical trials; Auspex may experience delays in the commencement, enrollment, completion or analysis of clinical testing for its product candidates, or significant issues regarding the adequacy of its clinical trial designs or the execution of its clinical trials, which could result in increased costs and delays, or limit Auspex's ability to obtain regulatory approval; the third parties with whom Auspex has partnered for the development of its product candidates and upon whom Auspex relies to conduct its clinical trials and manufacture its product candidates may not perform as expected; Auspex's product candidates may not receive regulatory approval or be successfully commercialized; unexpected adverse side effects or inadequate therapeutic efficacy of Auspex's product candidates could delay or prevent regulatory approval or commercialization; Auspex may be unable to obtain and maintain intellectual property protection for its product candidates; the loss of key scientific or management personnel; Auspex's ability to obtain additional financing; and the accuracy of Auspex's estimates regarding expenses, future revenues and capital requirements. All forward-looking statements contained in this press release speak only as of the date on which they were made. Other risks and uncertainties affecting Auspex are described more fully in Auspex's filings with the Securities and Exchange Commission. Auspex undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.