SoCal's Auspex Pharmaceuticals Soars 90% On Huntington's Disease Drug Trial Data

Published: Dec 18, 2014

SoCal's Auspex Pharmaceuticals Soars 90% On Huntington's Disease Drug

December 17, 2014

By Krystle Vermes, BioSpace.com Breaking News Staff

California-based biopharmaceutical company Auspex Pharmaceuticals Inc. saw its share price shoot up 90 percent in Wednesday trading after it released positive results from its Phase III registration trial evaluating SD-809 for the treatment of chorea associated with Huntington’s disease on Dec. 16. The drug showed positive top line efficacy and safety results, in addition to meeting its primary endpoint.

Patients who took the drug as a part of the trial showed significant improvements in quality of life. The study also reflected an overall favorable safety and tolerability profile.

First-HD, as the study was titled, was a randomized, double-blind, placebo-controlled parallel group trial designed to evaluate the safety and tolerability of SD-809. The primary endpoint for the study was the change from baseline to maintenance therapy in the Total Maximal Chorea score of the Unified Huntington’s Disease Rating Scale.

"For many individuals with Huntington's disease, chorea is a key symptom impacting safety, function and quality of life,” said Samuel Frank, principal investigator of the study. “New, safe and tolerable therapies for chorea treatment are clearly needed to make this disease an increasingly treatable condition. The primary and secondary efficacy results from this study were confirmed by the Huntington Study Group independent analysis. These clear and unequivocal results are clinically meaningful and suggest that SD-809 may play an important role in the treatment of Huntington's disease symptoms."

SD-809 continues to be evaluated in other clinical studies. Auspex expects more data to come from a variety of studies in 2015, including one that looks at the drug for the treatment of Tourette syndrome in mid-2015.

Orphan Drug Designation for SD-809
In November, Auspex announced that the U.S. Food and Drug Administration had granted SD-809 orphan drug status for the treatment of chorea in association with Huntington’s disease. The FDA gives this designation to drugs that are intended for the treatment of rare diseases impacting less than 200,000 people in the U.S.

"The orphan drug designation of SD-809 marks a major regulatory milestone for Auspex and a significant step forward in our goal to bring this novel treatment approach to patients as quickly as possible," said Pratik Shah, president and CEO of Auspex, at the time of the announcement. "Huntington's disease is a rare and devastating genetic disorder with very limited treatment options. The orphan drug designation recognizes the significant need that exists among individuals living with this disease."

More than 30,000 people in the U.S. and Canada are symptomatic for Huntington’s disease, while a greater number are at risk of developing the condition.

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