HSG conducts clinical trial with novel drug formulation for treating Huntington disease

Boston University Medical CenterJun 24 2013

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."