NAME: Prana Biotechnology
HEADQUARTERS: Parkville, Victoria, Australia
STOCK PRICE AS OF 10/05/11: $1.59
52 WEEK RANGE: $1.13-4.50
MARKET CAPITALIZATION AS OF 10/05/11: $42.4 million
EMPLOYEES: Approximately 10
Prana Biotechnology’s research is driven by the hypothesis that metal protein attenuating compounds bind transition metals and prevent oligomerization of amyloid proteins in Alzheimer disease and of the huntingtin protein in Huntington disease. Prana Biotechnology CEO Geoffrey Kempler sat down with HD InsightsTM at the World Congress on Huntington’s Disease to discuss Prana’s research in neurological disease modification. Excerpts from the discussion are below.
INSIGHTS: Could you tell us a little bit about Prana?
KEMPLER: Prana began in 1997 as a private company through some seed capital financing that came in from myself and a friend of mine, Barry Lieberman. We became interested in some work that was happening at Harvard Mass General. The work was on the amyloid protein and the role of metals in facilitating the aggregation of this protein into oligomers and plaques. What we were doing was considered heretical because we were really the first group to start to understand the metal binding sites on these proteins, and that these metals were driving the aggregation of the proteins. We supported our research very privately. We had a peripheral concept growing that was already available in the market and matched our scientific theory. There, we could do that concept work. By us understanding the mechanism that’s behind protein aggregation, we’ve been able to drive an enormous amount of academic publications through our support laboratories at Harvard, here in Melbourne at the Mental Health Research Institute and the University of Melbourne, and at some other collaborations we’ve had in various parts of the world.
INSIGHTS: It makes a lot of sense that a company like yours might be interested in Alzheimer disease, which has a very large market. Tell us why you are interested in Huntington disease.
KEMPLER: You imagine that companies always look at market size and market opportunity, and that’s true. I don’t want in any way diminish that as a focus. We know that cognition is one of the key areas of deficit in Huntington disease patients. We know that it’s a major aspect of Alzheimer disease, but we also know that there’s some normal cognitive decline that comes just with the aging process even if you don’t have either of these diseases. 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.