Life Science Partnerships With Patient Foundations: Best Practices - Part 2

By Wayne Koberstein, Executive Editor, Life Science Leader
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Voices of BayBio’s “Successful Public-Private Partnerships” Survey

Theory plus application yields real-world lessons. When life science companies and patient-advocacy groups come together to ensure development of new treatments for unconquered diseases, they typically draw on each other’s experience and inevitably learn some lessons on their own.

But this four-part series on the best practices for company-foundation partnerships, sparked by the large BayBio [UPDATE: In 2015, the BayBio Institute became the California Life Sciences Institute, and BayBio merged with the California Healthcare Institute to become the California Life Sciences Association (CLSA).] survey of players on both sides of such collaborations, seeks to spread some of the hard-earned knowledge over an even broader field of common interests.

In Part One of this series (June 2014), we examined what happens when two very different parties — a company and a foundation — establish their basic tenets, purpose, and goals in a common vision. In Part Two, we look at how the partners can avoid disruptive shifts and imbalances in their resources when they apply that vision to reality.

In addition to BayBio’s survey — conducted in collaboration with Merrill Datasite, BIO, and FasterCures — we draw from insights voiced by key people in companies and foundations participating in the survey. The experts represent a “core sample” of industry-foundation collaborations — most focused by their common involvement in neurodegenerative diseases.

Because industry-foundation partnerships cover such a wide range — from targeted data-only exchanges, to support for proof-of-concept studies, to full-scale funding of clinical trials — they often form asymmetrical relationships in size and resources. Recognizing the resource gap is the best first step each partner, and both partners together, can take. Although each one may be left alone to work out its own solution, ideally they will work together to align their resources.

The foundations that pioneered a shift to industry funding and collaboration strongly believe that support for product development is the most efficient and effective way to deploy their resources — that is, as long as all the parties involved give due diligence to “de-risking” their relationships. Realistic assessment of each partner’s assets, mapping out the development path, monitoring and measuring progress against clear milestones, and parallel adjustments to limit risk are the main best practices for ensuring resource alignment, as gleaned from the survey and its participating “voices.”

COMPLEMENT & LEVERAGE PARTNERS’ ASSETS
To make any rational use of resources, you must first know what you have, and in a partnership, you must also know what resources the other side can deploy. What does each party bring to the table? How and where can each partner best apply its assets to complement the other’s in reaching the common goal? Aligning precious resources along the most efficient path toward the ultimate goal is critical to leveraging them — a valuable lesson employed by one of the first patient foundations to partner with industry. In this case, the first few successes generated enough leverage for the group to expand its industry partnerships many times over.

SOHINI CHOWDHURY, Senior VP Of Research Partnerships, The Michael J. Fox Foundation For Parkinson’s Research (MJFF): We devote about $70 million toward research every year, but on the scale of industry drug development, that represents just a drop in the bucket. So we need to make sure we prioritize our funding to the most promising areas of therapeutic development, advancing a drug candidate to the point where it can be partnered with the groups that have more experience and deeper pockets to bring it to market — biotech and pharma. It took us a few years to staff up and plan how we could engage with industry and devote our resources to tackling related issues. In 2010, we launched the Parkinson’s Progression Markers Initiative, a public-private partnership for an observational, longitudinal study to identify progression markers for Parkinson’s disease. Though the study is in collaboration with industry, we are its sponsor and a primary funder. Sixteen pharmaceutical and biotech partners provide not only financial input, but also significant expertise and intellectual input to help us manage the study and to analyze the data coming out of it.

Although typically the size of funding from disease foundations, with notable exceptions, is relatively small in industry terms, its value in validating and attracting additional investment to small companies is evident. Less obvious, however, may be the effect of foundation support inside larger companies, as a legal expert specializing in industry-foundation partnerships points out.

DAVID LUBITZ, Partner, Schaner And Lubitz, PLLC: I have seen a number of medium to large biopharmaceutical companies come to the disease foundations for relatively small amounts of money because their programs must demonstrate internally not only that their technologies are promising, but also that they can attract seed funding to push them forward. So the disease foundations become de facto advocates for the technologies by funding them and participating in the collaboration within the companies. That is a vivid example of resource leveraging because, in a medium or a large company, which may receive at most a half-million dollars from some of the collaborations, the foundation funding may be enough to get the project off the ground, but its real value is to justify the project internally — to the folks who hold the purse strings.

In the case of the Myelin Repair Foundation (MRF), the currency of support is not money as it is with most other foundations, but knowledge. This group creates investigatory tools and produces data that guides discovery and development of drugs to repair damaged myelin, the protective sheath around nerves, primarily in multiple sclerosis. But though its native language is science, not business, the group invests many of its resources speaking to companies in terms the industry understands.

JENNIFER CHANG, Director Of Communications, The Myelin Repair Foundation: We don’t view industry as a collection of quasi-CROs to bring our compounds to the market, the traditional way many nonprofits have worked with industry. We realized we needed to replicate the academic research results and formulate comprehensive data packages that would interest industry. We opened up our own lab and have created our own proprietary myelin-repair drug assays that no one else in the world has, and because we’re a nonprofit, we’re able to work with many pharmaceutical companies to test any of their compounds that are interesting for myelin repair, to give them further evidence and support, whether or not they ultimately go into clinical development.

Besides funding, staff, and even knowledge itself, perhaps the most valuable asset a foundation might bring to a partnership from a company perspective is its ability to attract disease experts and key opinion leaders (KOLs) — another stake the MRF can place on the table.

CHANG: Traditionally, nonprofits have used a funding model based upon peer review, giving out individual grants in academic research and hoping the published papers would reach industry. But we leverage our nonprofit status to give us the freedom to operate with many more scientists and experts. We can bring people together at a table that universities may not. We can engage with the right people at every step of the drug development process, from the bench to FDA approval, to greatly accelerate the process.

Companies may also be the catalyst for KOLfueled collaborations. Amplimmune formed an alliance with Fast Forward, the industry-partnering subsidiary of the National Multiple Sclerosis Society (NMSS), and Northwestern University to move along the company’s early development of a molecule to tame abnormal immune responses. It found that teaming the partners’ hard assets with their “intangible” resources — all pushing in the same direction — brought quite tangible results.

MICHAEL RICHMAN, President And CEO, Amplimmune: Although people focus on the financial investment, the intangible value contributed by foundations is just as important, tapping into their network of professionals — exchanging and sharing ideas, obtaining access to materials such as patient blood and tissue samples that may be vital in one’s research. Foundations are learning how to use KOLs (key opinion leaders) in their network or their own in-house experts to evaluate partnering opportunities and monitor ongoing relationships. But the company can also bring in its own expertise; for example, in manufacturing, 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 product 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-inflection points in the critical path of developing 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 — controlled studies in patients for safety and efficacy. Foundations are providing preclinical 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 information and more data, to either discount a particular therapeutic avenue or key it up to the point where it’s been derisked enough that another group may pick it up. And so it requires us to actually understand the industry’s perception 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 proposal 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 collaborators, 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 trials, 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.