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LIFESCIENCELEADER.COM JULY 2014 35 leaders) in their network or their own in-house experts to evaluate partnering opportunities and monitor ongoing rela- tionships. But the company can also bring in its own expertise; for example, in manu- facturing, regulatory affairs, and working with the clinical operation groups such as CROs and universities. We take the partner's resources and integrate them with the company's resources, creating the alignment, and then we put all of it on a path that hopefully leads to a new prod- uct at the end. So, the resource alignment is very clear — it is focused on the specific objectives and the goals we share with the foundation. MAP OUT THE DEVELOPMENT PATH Best practices in industry-foundation partnerships commonly overlap; they are not always sequential or completely separate actions. For example, moving between evaluating partners' assets to planning goals and objectives is a dynamic, two-way process. Both actions — evaluating assets and setting goals — involve defining the scientific, financial, and other practical challenges. But mapping out a clear path for the collaborative project will identify key points where partners might work together to de-risk development, as in preclinical or proof-of-concept studies, patient selection, and clinical trial design. Whether the aim is lab tools or clinical trials, a clear common goal, well-defined milestones, and scientific results must guide all planning, monitoring, and decision making from the earliest point of the partnership on. RICHMAN: There are two key value-inflec- tion points in the critical path of develop- ing a potential product. One is preclinical validation, based on cellular biology work or testing in animal models. The second is clinical proof-of-concept trials — con- trolled studies in patients for safety and efficacy. Foundations are providing pre- clinical proof-of-concept investments so that a company can generate the data sets needed to secure additional funding from investors and take things further into clinical trials. CHOWDHURY: The concept of de-risking is really central to why and how we think about placing our money and working with industry — to generate further infor- mation and more data, to either discount a particular therapeutic avenue or key it up to the point where it's been de- risked enough that another group may pick it up. And so it requires us to actu- ally understand the industry's percep- tion of Parkinson's disease, what they consider the critical hurdles to investing in Parkinson's, and to figure out what is their threshold for risk in obtaining the product. MONITOR PROGRESS AND REROUTE AS NEEDED Trust but verify — de-risking also requires diligent monitoring and steering of development projects. For Ceregene and MJFF, as for other voices in this series, continuous oversight by the foundation partner proved vital to the relationship right from the start, enabling the partners to align and leverage their resources for repeated expansions and successes. JEFFREY M. OSTROVE, Former CEO, Ceregene: With Ceregene's Parkinson's drug, we were designing our Phase 1 clinical trial in 2004 and we wrote a grant propos- al to The Michael J. Fox Foundation to fund parts of the trial because there were some really interesting endpoints — not necessarily ones that would be required by the FDA, but important scientifically. Though the group was new to funding industry research, our application was fast-tracked when the Foundation's head of research received positive feedback on the drug from outside experts, and our chief scientist was allowed to present our case directly to the Foundation's review committee. They awarded us the grant of about $750,000. Since then, Ceregene has been awarded approximately $7 million from the MJFF, but all of the grants were driven by milestones, and we worked like a family with the Foundation and our col- laborators, in order to make sure we all stayed on track to achieve them. Some of the money that we really needed from MJFF was to extend the timing of our clinical endpoint. We wanted to have up to a 24-month endpoint in one of our tri- als, as opposed to a 12-month or 15-month endpoint, because the experts believed it would generate a richer data set. The Foundation gave us a $2.5 million grant. It was milestone-driven: enroll the first cohort of patients, enroll the second, get some more money, and so on. Had the FDA stopped the trial for any reason, which they didn't, the Foundation wouldn't have made the next payment. CHOWDHURY: We are clear with all of our awardees — we are not a bank. We don't just provide a check and walk away. We are very tailored in our approach to research. We view ourselves as partners in the research endeavor. All of the payments are linked to milestones accomplished, and there are frequent assessment calls or in-person meetings that happen with every awardee. Managing expectations, whether it be with industry or academia is critical to resource alignment. Thus ends Part Two of our four-part series, "Industry Partnerships With Patient Foundations — The Best Practices." Watch for Part Three, "Partnership Structure," in next month's Life Science Leader. Many thanks to Travis Blaschek-Miller at BayBio and the BayBio team for their help with this article series. (See BayBio's white paper on the survey at http://baybio.org/?s=public-private+partnerships.) L 0 7 1 4 _ B a y b i o _ 2 . i n d d 4 0714_Baybio_2.indd 4 6 / 2 0 / 2 0 1 4 1 2 : 2 8 : 2 5 P M 6/20/2014 12:28:25 PM