Auspex Pharmaceuticals Completes Enrollment in a Pivotal Clinical Trial of SD-809 in Tardive Dyskinesia

LA JOLLA, Calif., Feb. 3, 2015 (GLOBE NEWSWIRE) -- Auspex Pharmaceuticals, Inc. ASPX, a biopharmaceutical company dedicated to developing innovative medicines for people with movement disorders and other rare diseases, today announced that it has completed target enrollment in its pivotal clinical trial of SD-809 for the potential treatment of tardive dyskinesia (TD), called ARM-TD (Aim to Reduce Movement in TD). The company expects to announce topline data from this study in mid-2015.

TD is a hyperkinetic movement disorder affecting about 500,000 people in the United States and is caused by widely used medications to treat psychiatric conditions such as schizophrenia and bipolar disease, as well as by certain drugs used for treating various gastrointestinal disorders.

"Tardive dyskinesia is a devastating and severely debilitating condition for many patients. There are no FDA-approved treatments for tardive dyskinesia, making the need to find a treatment all the more urgent," said Michael Huang, M.D., vice president, clinical development, Auspex Pharmaceuticals. "Completing enrollment in this trial marks an important step forward in our understanding of the potential impact SD-809 may have on the lives of the approximately half-a-million people in the U.S. living with this disease."

In ARM-TD, approximately 90 patients with moderate to severe TD were targeted for enrollment. Enrolled patients were randomized 1:1 to receive either SD-809 or placebo, which was titrated to optimal dosage over six weeks, and then administered that dose for another six weeks, for a total of 12 weeks of treatment. The primary efficacy endpoint is the change in the Abnormal Involuntary Movement Scale (AIMS) from baseline to end of therapy, as assessed by centralized video rating. The safety endpoints include adverse events, vital signs, physical/neurological/laboratory examinations and ECGs. Based on feedback obtained at a meeting with the U.S. Food and Drug Administration, the ARM-TD trial may qualify as one of the two pivotal trials needed for a 505(b)(2) New Drug Application (NDA) filing. Eligible subjects who complete the Phase 2/3 clinical trial are offered an option to rollover into a long-term safety study (RIM-TD).

"We are extremely pleased to have reached this critical milestone in the development of SD-809," said Pratik Shah, Ph.D., president and CEO, Auspex Pharmaceuticals. "We look forward to reporting on the trial results and continuing to advance our entire SD-809 clinical program. We believe strongly in the potential of SD-809 to become the treatment of choice for this serious debilitating movement disorder."

Auspex is also conducting an additional randomized, double-blind, placebo-controlled, parallel group pivotal clinical trial of SD-809 in approximately 200 people with tardive dyskinesia (AIM-TD; Addressing Involuntary Movements in Tardive Dyskinesia). Topline results from the AIM-TD trial are expected in 2016. Contingent upon successful completion of both the ARM-TD and AIM-TD studies, Auspex plans to file an NDA for SD-809 for the treatment of tardive dyskinesia in 2016.

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.