HD Insights™

HD INSIGHTS TM Meet the Company, continued... KEMPLER, cont.: In the animal experiments that we've done, we've demonstrated that our drug PBT2 actually had profound effects on a memory test that we gave to old mice that were not transgenically modified to have either Alzheimer disease nor Huntington disease. These mice were able to effectively complete the task post-treatment with the drug as effectively as young healthy mice. It told us that the drug would be useful wherever there is a cognitive deficit, particularly where that cognitive deficit emphasizes executive function deficit, such as early in the disease process. INSIGHTS: So among the disorders that affect cognition, what attracted you specifically to Huntington disease? KEMPLER: I think it was the advocacy of the Huntington disease researches. We have been approached by different researchers and different diseases, and we're a little company so we can't take on everything. But the people in Huntington disease were able to instill their confidence of our own drug in this indication. From a pragmatic perspective we saw that it was a disease where we could come along, present our case, get some attention, and actually see if we can bring benefit to patients. INSIGHTS: Where there any researchers in particular who were especially strong advocates? KEMPLER: I think I would have to certainly begin with Professor Ira Shoulson from Georgetown University, who had a lot of influence in our thinking, as well as Steve Hersch from Massachusetts General Hospital in Boston. INSIGHTS: What did they do to persuade you? KEMPLER: They were able to look at our data, explain the relevance of it to Huntington disease patients, and work with our scientists to generate new data. They were able to prepare feedback on what we'd produced and what others had produced, and really helped us understand that we might have a unique position with our drug. INSIGHTS: There's only one FDA approved treatment for HD right now. You are also aware that the Dimebon study failed to show benefit on cognition relative to placebo. How did that affect your thinking about coming into with a new compound to look at cognition in Huntington disease? KEMPLER: We've spent so long on the outside of the accepted paradigms that we're quite comfortable living in that little space. We feel that we're likely to be in a pretty small group if not a bit unique amongst the drugs that actually have some promise. INSIGHTS: Looking forward, what are your hopes for Prana and for Huntington disease? KEMPLER: My big hope for Prana is that the many, many years of dedicated effort by outsiders, our staff, the investors, and by the community patient groups that have cooperated in our clinical trials and in our advocacy will be rewarded by PBT2 in the first instance. Getting approval for a drug that could bring cognitive benefit to patients with Huntington disease will parallel our ongoing important work in Alzheimer disease. We actually have an entire strategy that's probably gone for the very high bar, and that's to deal with the actual underlying disease process that's behind each of these diseases. My hope for Prana in the very short-term is that we'll be seen by the Huntington disease community as the drug in Phase II that appeared out of nowhere because it was from an Alzheimer universe. 10 Copyright © Huntington Study Group 2011. All rights reserved. HD InsightsTM, Vol. 1

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