Public Release:  The Huntington Study Group announces first-HD study

Novel drug for the treatment of Huntington Disease

Boston University Medical Center

The Huntington Study Group (HSG), under the leadership of Samuel Frank, MD, Principal Investigator, (Boston University School of Medicine) and Claudia Testa, MD, PhD, co-Principal Investigator (Virginia Commonwealth University), is conducting a clinical trial with a formulation of the novel drug SD-809 in the treatment of HD the United States and Canada. The trial is sponsored by Auspex Pharmaceuticals, Inc. who is developing the drug for the treatment of Huntington disease, as well as Tourette syndrome and tardive dyskinesia.

First-HD is a randomized, double blind, placebo controlled study that examines the efficacy, safety and tolerability of a formulation of the investigational drug known as SD-809 for the treatment of chorea associated with Huntington Disease. The drug has the same mechanism of action as the FDA approved drug tetrabenazine that is used to lessen chorea in people with HD, an inherited disease that affects over 30,000 people in both the United States and Canada. HD is characterized by brain cell death that usually begins between the ages of 30 to 50. This cell loss results in motor, cognitive and behavioral signs and symptoms. Chorea, which is characterized by involuntary movements, is a hallmark of the disease.

Because SD-809 is more slowly metabolized than tetrabenazine, it provides more consistent and predictable drug levels and can be given less often than tetrabenazine. With these changes, a lower overall dose of SD-809 can be administered and it may be more convenient for persons with HD. This study will test the efficacy, safety and tolerability of a formulation of SD-809 in the management of chorea symptoms in HD patients who have not previously taken tetrabenazine. Participants will be involved in this trial for approximately 4 months.

Dr. Samuel Frank, the Principal Investigator of First-HD notes that "we are excited to work with Auspex to investigate the efficacy, safety and tolerability of this interesting and innovative new treatment for Huntington disease. We have few treatment options for Huntington disease, and only one for chorea. We hope this is a step forward in addressing this large unmet need for our patients."

There is no cost to participate in First-HD. Individuals with a diagnosis of HD who are interested in participating in First-HD should visit the HSG website: http://www.huntington-study-group.org or call toll free: 1(800) 487-7671 (North America). More information regarding this trial can also be found at http://clinicaltrials.gov

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About the Huntington Study Group (HSG):

HSG is an independent not-for-profit network of more than 200 clinical investigators, coordinators, and scientists from 100 participating universities and clinics in the United States, Canada, Europe, Australia, New Zealand and South America who provide comprehensive care for Huntington disease (HD) patients and families and carry out multi-center clinical research including observational studies and controlled clinical trials. The mission of the HSG is to relieve the burden of illness and develop treatments that make a difference for HD patients and families.

http://www.huntington-study-group.org

About Auspex Pharmaceuticals:

Auspex Pharmaceuticals is a privately held biopharmaceutical company located in La Jolla, California. The company is a pioneer in the use of deuterium in medicinal chemistry, and is focused on the treatment of hyperkinetic movement disorders including Huntington disease, Tourette syndrome and tardive dyskinesia. Its lead compound, SD-809, is a novel inhibitor of the vesicular monoamine transporter 2 (VMAT-2). This drug offers the potential for significant advantages over existing therapies including improved safety, reduced drug-drug interactions, and less frequent dosing. Auspex is also exploiting its deuterium chemistry approach to optimize drugs in several additional therapeutic areas. Included in the Auspex portfolio are SD-900, a JAK kinase inhibitor for the treatment of autoimmune diseases, and SD-560, for the treatment of fibrotic diseases, as well as other compounds.

http://www.auspexpharma.com

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