HD Insights™

Vol. 12 - Fall 2015

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8 HD Insights, Vol. 12 Copyright © Huntington Study Group 2015. All rights reserved. HD INSIGHTS: You also saw some changes on imaging. ZAUDERER: Yes: for example, we observed reduced loss of brain volume. One characteristic feature of HD is loss of about 10 percent of both white matter and grey matter volume. Incidentally, this is also true in progressive MS. We demonstrated that use of VX15/2503 significantly reduced the loss of brain volume in YAC128 HD transgenic mice. HD INSIGHTS: You recently launched a Phase II clinical trial of VX15/2503 in HD called SIGNAL. What do you hope to learn from this study? ZAUDERER: We are enrolling patients who are in the prodromal and early manifest stages of HD. Our goal is to see whether treating patients with VX15/2503 delays or prevents the onset of disease. We actually just enrolled our first patient about a week ago, so it is too early to know. However, we have tested VX15/2503 in about 40 patients at various stages of MS, and it has had a good safety profile to date. We are hopeful that we will continue to have a good safety profile and be able the judge the efficacy of the drug as well in this study. The neuropathology and the behavioral and cognitive symptoms of HD continue to progress after clinical diagnosis. About one-third of the patients we will be working with will be in the early manifest stage of HD, because we want to see whether our VX15/2503 continues to have an impact on disease progression. HD INSIGHTS: Changing gears, Vaccinex recently announced an agreement with Five Prime Therapeutics. Are there any implications for HD? ZAUDERER: That was an antibody development agreement. Vaccinex has a novel human antibody selection technology that has been of interest to a number of other companies, including Five Prime Therapeutics, and Janssen Pharmaceuticals. In those relationships we select fully human antibodies against targets of interest to those companies. It is a technology collaboration. HD INSIGHTS: Before you founded Vaccinex, you were a highly successful academic. Can you describe what motivated the change from academia to private enterprise? ZAUDERER: I very much enjoyed my academic research career. I was on the faculty at Columbia University, then at the University of Rochester Cancer Center for a total of 25 years before founding Vaccinex. It was a very enjoyable time in my life. As it happened, my laboratory developed some novel technologies that were of commercial interest. I was approached by a group of investors who were very interested in founding a biotechnology company based on those technologies, and I thought this would be an interesting challenge, and indeed it has been. It was an opportunity to do something different in my life. HD INSIGHTS: Any regrets? ZAUDERER: No, none at all. On the contrary, it has been a very enriching experience. The qualities you need and that you cultivate as an academic scientist are different from the qualities that you need and cultivate as an entrepreneur. I really feel this has been a growth opportunity for me. HD INSIGHTS: Can you elaborate on those qualities? ZAUDERER: In academic life, the most important value is innovation on an individual level; it is the basis for publication, the basis for promotion, and for grants. When you are at a biopharmaceutical company, the process of bringing a product from the laboratory to the clinic is so complicated that you very quickly realize that you cannot possibly do it without a lot of cooperation with other people. So, although innovation continues to be important, cooperation is equally important, and that involves a whole different set of relationships and skills. I feel fortunate to have been put in a situation where I have had to develop and cultivate those skills. HD INSIGHTS: Do you have any advice for academics who are thinking of making the transition to industry? ZAUDERER: It is much more challenging than I realized when I first jumped into this. There are numerous organizational, financial, and development challenges. You need a very strong group of supporters in order to be able to do this successfully. HD INSIGHTS: Dr. Zauderer, thank you very much for your time and good luck with your efforts in HD. For more on Vaccinex, please visit their website. For more on the SIGNAL trial, please visit SIGNAL trial. Meet Maurice Zauderer, cont... 1 Southwell AL, Franciosi S, Villanueva EB, et al. Anti-semaphorin 4D immunotherapy ameliorates neuropathology and some cognitive impairment in the YAC128 mouse model of Huntington disease. Neurobiol Dis. 2015;76:46-56. doi: 10.1016/j.nbd.2015.01.002 H D I N S I G H T S

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