Life Science Leader Magazine

JUN 2014

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LIFESCIENCELEADER.COM JUNE 2014 46 EXCLUSIVE LIFE SCIENCE FEATURE leaders MICHAEL RICHMAN President And CEO, Amplimmune: When your project is aligned with the objectives of the foundation in a given disease, it's a win-win situation for all involved because you're synergizing your financial resources, access to informa- tion, ideas, and materials. All that came together in experiments that would help us figure out whether our molecule had some potential application with MS. Fast Forward brought in scientists from Northwestern University in Chicago who were doing research that could help us, so we triangulated a collaboration with the university and Fast Forward. Then, all three of us were synergizing financial and experimental resources, and all of us focused on a development plan for products to treat MS and other autoim- mune diseases. A leader of the major MS group explains why his organization — once mainly dedicated to patient care, advocacy, and academic research — founded Fast Forward, which soon inter- sected with Amplimmune in the beginning of a beautiful friendship. TIM COETZEE Chief Advocacy, Services, And Research Officer, National MS Society: Along with many other organizations, we now believe the leads from discov- ery in the university laboratory setting, which are vital, also require a commer- cial partner and a translation from one part of the value chain to the next. So we made the decision in 2007 to expand our footprint in research and to incorporate a strategy that focuses on investing in research happening in the commercial setting, typically at small biotech com- panies, though our portfolio has not been strictly limited to small biotech. That was the impetus behind the creation and launching of Fast Forward within the National MS Society. Meanwhile, another group, the Myelin Repair Foundation (MRF), came at MS from a differ- ent direction, creating its own area of research to push the industry into a new therapeutic approach. JENNIFER CHANG Director Of Communications, The Myelin Repair Foundation: Our founder, who has MS, realized that a lot of the research in academia had a dif- ficult time getting out of academia, so he began identifying the barriers in medical research that prevented novel therapeu- tics from reaching MS patients. The stan- dard industry approach to MS was to sup- press the entire immune system to lessen its symptoms, a treatment that causes its own set of severe symptoms. There was absolutely no focus on how to repair the neurological system once the disease dam- ages it. With his business background, he also noticed a lot of disease organiza- tions didn't have ambitious time lines for achieving their goals. So he started the MRF to change how research was done in this area. Identify/validate potential treatments/solu- tions. For foundations, sorting through and selecting projects from among numerous companies, development candidates, or other industry-partnering prospects require sufficient scientific expertise and methods to vet candi- dates in opportune areas of intervention. On the company side, it requires preparation and responsiveness to the foundation's validation approach. RICHMAN: Like any investor, the founda- tions will do due diligence. We had to sub- mit an application to Fast Forward, and the application was reviewed by an expert committee made up of scientists, neurolo- gists, and autoimmune experts working in the MS space. In fact, they rejected our initial submission because of the study design, but we revised and resubmitted our application and secured grant approv- al about a year later. Through their due diligence, they can evaluate a company to determine whether it has the right exper- tise, infrastructure, tools, molecules, and the means to carry out the experiments. And if you don't, they may help you cre- ate "collaborative clusters," working with experts in the field. One well-known foundation goes further than being a catalyst for developmental research — it has always been a research-oriented organization, parallel to several large patient- support groups, but it is now arguably the lead player in the push for new products to help Parkinson's patients. SOHINI CHOWDHURY Senior VP Of Research Partnerships, The Michael J. Fox Foundation For Parkinson's Research (MJFF): We have a scientific advisory board and 10 scientists and one neurologist on staff, so we set priorities and drive the science toward our goals. We look at the science always through the prism of a patient's eyes because our funding comes from patients, by and large. We take a portfolio approach toward the research that we fund, looking both at the here and now in terms of trying to improve symptomatic treatment for patients, as well as a longer- term vision of that holy grail of a way to slow or halt the disease progression. It is important to remember that the founda- tion's vetting process can result in more than a financial boost. Many companies benefit more immediately from the network a founda- tion can house, as in this example from Fast Forward's initiative with Amplimmune. JEFFREY OSTROVE Former CEO, Ceregene: One of our first product candidates was nerve growth factor (NGF), in which we delivered the gene for the factor directly to the brain of patients with Alzheimer's disease in Phase 1 and Phase 2 clinical studies. Not only did we hope to see symp- tomatic improvement in patients, but we also believed we could slow down the neurodegenerative process. Yet we would have to prove it in our clinical trials. Our Alzheimer's program and clinical stage Parkinson's disease programs were all we could afford with the venture money we raised. A new potential treatment for amy- otrophic lateral sclerosis (ALS) using a viral vector-delivering insulin-like growth factor 1 (IGF-1) discovered by Fred Gage and Brian Kasper at the Salk Institute was very exciting and complemented our other programs. Unfortunately, we did not have the resources to carry out develop- ment of this potential drug. Fortunately, Project ALS gave us hundreds of thou- sands of dollars to allow us to start work- VOICES OF BAYBIO'S "SUCCESSFUL PUBLIC-PRIVATE PARTNERSHIPS" SURVEY By W. Koberstein 0 6 1 4 _ B a y b i o 3 . i n d d 3 0614_Baybio3.indd 3 5 / 2 1 / 2 0 1 4 1 2 : 5 9 : 3 8 P M 5/21/2014 12:59:38 PM

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