HD Insights™

Vol. 10 v.9 FINAL

Issue link: http://www.e-digitaleditions.com/i/464617

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HD Insights, Vol. 10 1 HD Insights A H u n t i n g t o n d i s e a s e r e s e a r c h Issue N o 10-- Winter 2015 p e r i o d i c a l TM Copyright © Huntington Study Group 2015. All rights reserved. Meet the Compound Ryan Korn, BA, discusses the evaluation of laquinimod for treatment of HD. Page 6 Research Round-up Lise Munsie, PhD, summarizes new HD basic science and pharmaceutical research. Page 5 Highlights of HSG 2014 The 2014 Annual Meeting explored "Ideas for the Future" in Minneapolis, MN. Page 11 Clinical Trials Update We bring you the latest in HD clinical trials. Pages 9-10 (continued on Page 2...) First-HD Results Released
 Meet the Investigator Francesca Cicchetti, PhD, shares her groundbreaking neuroscience research.
 Pages 3-4, 12-13 Auspex announces promising results from First-HD trial of deuterated tetrabenazine (SD-809) By: Meredith A. Achey, BM On December 16, 2014, Auspex Pharmaceuticals announced positive top-line results for the "First Time Use of SD-809 in Huntington Disease" (First-HD) registration trial of SD-809, a deuterated formulation of tetrabenazine (see HD Insights, Vol. 7). The company also released preliminary results of the ongoing open-label "Alternatives for Reducing Chorea in HD" (ARC-HD) study showing that individuals who switched from tetrabenazine to SD-809 maintained chorea control while taking SD-809 (see Table 1 for a summary of the trials). The complete press release is available here. The primary efficacy endpoint for First-HD was improvement in the total maximal chorea score of the Unified Huntington's Disease Rating Scale. Trial participants who took SD-809 showed an average improvement of 2.5 points (p<0.0001) from baseline to maintenance therapy. In addition, secondary endpoints assessing clinical relevance, including patient global impression of change, clinical global impression of change, and change in the SF-36 physical functioning score (a quality of life measure) showed improvement over placebo (p<0.05), while the Berg Balance test did not show significant improvement (p=0.14). More than 90% of First-HD participants enrolled in the open-label, long-term followup ARC-HD study. ARC-HD "Switch" was completed in parallel to First-HD, as patients currently taking tetrabenazine for chorea control were switched overnight to SD-809 at approximately half their usual dose of tetrabenazine (see Table 1). After four weeks, the 35 individuals for whom data was available showed an average improvement of 0.8 points (standard error 0.5) in total maximal chorea score. After eight weeks, data for 21 individuals showed an average improvement of 1.9 points (standard error 0.8). The safety profile was similar to that in the First-HD study. These preliminary results have given the HD community hope for improvement in the treatment of HD. Dr. Samuel A. Frank, principal investigator for First-HD, told HD Insights, "Combined with the excellent adverse event profile seen with the analysis to date, we are far in the process of developing another potential treatment option for patients with HD."

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