A Huge Step Forward in the Treatment of Huntington Chorea

A Drug, SD-809, Developed By Auspex Pharmaceuticals Could Be A Huge Step Forward In the Treatment Of Chorea Associated WithHuntington’s Disease, Called First-HD Auspex Pharmaceuticals, Inc. (ASPX) announced positive topline efficacy and safety results from its Phase 3 registration trial evaluating SD-809 for the treatment of chorea associated with Huntington's disease (HD), called First-HD. Huntington's disease is a genetic disorder that causes involuntary movements, emotional problems, behavioral problems and problems thinking and processing information, Ultimately the disease leads to death. Approximately 90 percent of patients with Huntington's disease will develop chorea, which is characterized by involuntary, excessive movements that can impact all parts of the body and interferes with motor functions. Chorea associated with Huntington's disease can be severely debilitating. SD-809 met the primary efficacy endpoint with significant improvement being observed in both the patients and clinical global impressions of change and quality of life. The study demonstrated a favorable safety and tolerability profile, including low rates of depression, somnolence, akathisia/restlessness and anxiety. Data from the ARC-HD study demonstrate that patients who switched from the current standard of care, tetrabenazine, to SD-809 maintained chorea control at both week one and week four. Samuel A. Frank, M.D., associate professor of neurology, Boston University School of Medicine and principal investigator for First-HD said, among others, "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 SD-809 is a VMAT-2 Inhibitor. Auspex designed, produced and developed the drug for the treatment of Hyperkinetic Movement Disorders. The only currently approved drug is Tetrabenazine (marketed as Xenazine® in the United States). Using its deuterium technology, Auspex made chemical modifications at specific positions in the tetrabenazine molecule to create the novel drug candidate SD-809 (dutetrabenazine). Deuterium is a non-toxic, naturally occurring form of hydrogen. The substitution of deuterium (2H) for hydrogen (1H) at specific positions, in the case of SD-809, attenuates the breakdown of the drug’s active metabolites resulting in a differentiated pharmacokinetic profile compared to tetrabenazine. This profile should enable less frequent dosing, improved tolerability, reduced interpatient variability in drug metabolism, as well as reduced drug interactions and reduced need for genotyping for drug-metabolizing enzymes. Prohost Observations: This is a huge step forward we always wished taken by small biotech firms. This is why we invest in this sector in the first place. The breakthrough here is in chemistry, where the change made in the chemical structure of the drug has made a great difference. Patients taking SD-809 achieved a meaningful improvement of 2.5 points on the TMC score from baseline to maintenance therapy compared to placebo (p < 0.0001). These are good results. We believe that SD-809 will address many other unmet needs in a variety of hyperkinetic movement disorders. It is, indeed, a broad-spectrum drug. The news instigated a monster rally in ASPX For First-HD Topline Results and other details Click HERE FORWARD-LOOKING: Material presented here is for informational purposes only. Nothing in this article should be taken as a solicitation to purchase or sell securities. Before buying or selling any stock you should do your own research and reach your own conclusion. Further, these are our 'opinions' and we may be wrong. We may have positions in securities mentioned in this article. You should take this into consideration before acting on any advice given in this article. If this makes you uncomfortable, then do not listen to our thoughts and opinions. The contents of this article do not take into consideration your individual investment objectives so consult with your own financial adviser before making an investment decision. Investing includes certain risks including loss of principal.