Auspex Pharmaceuticals, Inc. (Nasdaq:ASPX), a late clinical stage biopharmaceutical company focused on developing and commercializing novel medicines for the treatment of orphan diseases, today announced results from a pre-specified interim subgroup analysis of the ongoing open-label ARC-HD Switch study in which the clinical experience of switching subjects’ treatment from tetrabenazine to SD-809 was evaluated.
The interim analysis was conducted to determine whether chorea control was maintained after converting subjects with Huntington’s disease (HD) from stable doses of tetrabenazine to SD-809, which is administered in lower doses and with a simplified dosing regimen. In accordance with the pre-specified interim analysis plan, 12 subjects were included in the subgroup analysis, all of which had chorea assessed one week after switching from tetrabenazine to SD-809 and eight of which had chorea assessed four weeks after switching (the remaining four subjects had not yet reached the four-week point in the study at the time of the pre-specified interim analysis). At one and four weeks after switching to SD-809, the mean total chorea score decreased by approximately one point from baseline (week one change for 12 subjects was -0.83 +/-0.51 and week 4 change for eight subjects was -0.75 +/-0.75, mean and standard error), indicating that SD-809 maintained chorea control in these subjects. The conversion to SD-809 was generally well tolerated as no subject prematurely discontinued from the study, there were no adverse events of loss of chorea control and no serious adverse events were reported.
“These data suggest that SD-809 is biologically active in these Huntington’s disease patients and that switching to SD-809 was generally well tolerated,” said David Stamler, M.D., Chief Medical Officer at Auspex. “The data give us confidence to complete the ongoing trials in HD as planned and to accelerate the development of SD-809 in other hyperkinetic movement disorders such as tardive dyskinesia and Tourette syndrome.”
The Principal Investigator of the SD-809 First-HD and ARC-HD Phase 3 clinical studies, Dr. Samuel Frank, M.D., Associate Professor of Neurology at the Boston University School of Medicine, said, “The interim analysis data from this small number of patients are very encouraging and provide the first indication that subjects can safely switch to SD-809 with at least equal control of chorea and other motor signs of Huntington’s disease. These interim data support the continued evaluation of patients in the ARC-HD and the First-HD pivotal studies of SD-809.”
The adverse events reported to date in these interim analysis subjects during treatment with study drug were mild or moderate, with dry eye and dry mouth (one subject), fall (two subjects) and back pain (one subject), none of which were deemed related to study drug; and one subject with mood altered, deemed possibly related to study drug. The ARC-HD Switch study is ongoing and further assessments of safety, tolerability and chorea control in these and additional subjects will be made to confirm these interim findings.
About ARC-HD Switch
ARC-HD Switch is an open-label clinical trial in approximately 36 patients with chorea associated with Huntington’s disease in which safety, tolerability and chorea control are assessed in subjects switching from stable doses of tetrabenazine to SD-809, which is administered in lower doses and with a simplified dosing regimen. The study aims to provide guidance to physicians on how to switch subjects’ treatment from tetrabenazine to SD-809. The interim analysis was not designed to compare the safety and tolerability of SD-809 to tetrabenazine. Participating subjects are eligible to receive open-label SD-809 treatment in a one-year long-term safety study. Top-line data from all enrolled subjects in the ARC-HD Switch study are expected to be reported in the fourth quarter of 2014. This study is being conducted with the Huntington Study Group. More information about clinical trials evaluating SD-809 can be found at www.huntington-study-group.org or by calling 800-487-7671.
About Auspex Pharmaceuticals
Auspex Pharmaceuticals is a late clinical stage biopharmaceutical company focused on the development and commercialization of novel medicines for the treatment of orphan diseases. Auspex’s pipeline includes product candidates to address unmet medical needs in hyperkinetic movement disorders, such as chorea associated with Huntington’s disease, tardive dyskinesia and Tourette syndrome, as well as fibrotic indications, including idiopathic pulmonary fibrosis (IPF) and other orphan conditions. Auspex’s lead product candidate, SD-809 (deutetrabenazine), is in a Phase 3 registration clinical trial for the treatment of chorea (abnormal involuntary movements) associated with Huntington’s disease. Auspex has initiated a Phase 2/3 clinical trial of SD-809 in tardive dyskinesia and a Phase 1b study in Tourette syndrome. Auspex also plans to evaluate SD-560 (deuterium-containing form of pirfenidone) in a Phase 1 clinical trial. Auspex has employed its deuterium chemistry approach to optimize other compounds in its portfolio that are at various stages of development. For further information, please visit the company’s websitewww.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 and Auspex’s ability to obtain regulatory approval for 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 with 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.
Source: Auspex Pharmaceuticals