Magazine Article | June 2, 2014

Life Science Partnerships With Patient Foundations: Best Practices - Part 1

Source: Life Science Leader

By Wayne Koberstein, Executive Editor, Life Science Leader
Follow Me On Twitter @WayneKoberstein

Voices of BayBio’s “Successful Public-Private Partnerships” Survey

One by one, life sciences companies and patient-advocacy groups have been getting together, bound by a common purpose — to ensure development of new treatments for unconquered diseases. Not always an easy marriage, these unions have grown and benefitted from experience.

Such were the basic assumptions underlying industry group BayBio’s [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 — in collaboration with Merrill Datasite, BIO, and FasterCures — to assess the state of “public-private” partnerships and tease out a set of best practices that would guide new partnerships in the future. Helped by further input from some key companies, foundations, and people involved in the BayBio survey, each part of this four-part series will illustrate the best practices in one of the following areas:

  1. vision and goal alignment
  2. resource alignment
  3. partnership structure
  4. management paradigms.

In part one, we examine what happens when the two very different parties, a company and a foundation, come together and establish the basic tenets of their relationship. It is a time when the parties must bring their vision and goals for the partnership into alignment while recognizing and reinforcing each other’s interests.

Nowadays, functioning public-private partnerships have proliferated in such a variety of forms and fashion, it is impossible to draw from all of their experiences in this context. Partnerships range from targeted data-only exchanges to support for proof-of-concept studies to full-scale funding of clinical trials, and often asymmetrical relationships in size, resources, and other aspects between partners.

Here, we introduce three industry executives and three foundation leaders, all with extensive experience in multiple public-private partnerships, some in partnerships with each other, and every one concerned enough about best partnering practices to have helped with the BayBio project.

Examining the “core sample” these experts represent — further focused by their common involvement in neurodegenerative diseases — keeps the picture as simple as possible while suggesting the great variety of possible partnerships. Hopefully, what these experts have to say will pique your interest in life sciences public-private partnerships, whether for further learning or involvement or for implementation of new, successful partnering entities. However, many of them caution that their learnings can’t always be transferred wholesale to a different setting.

Toward A Common Vision And Goal
From the BayBio survey and the words of those quoted below emerge some essential, arguably “best-practice” steps companies and foundations should take to find the right partners, work their way toward a common vision, and establish shared goals — the genesis of a productive and mutually beneficial alliance.

Rally Around Development
Foundations deal with complex diseases that typically affect patients’ lives in many ways. And at any time, scientific progress may open up new mechanisms of action and disease targets that need further exploration and proof-of-concept. Typically, a company and a foundation come together to target development of new interventions in areas where science offers an opportunity and their interests intersect. Amplimmune found a match in Fast Forward, the industry-partnering subsidiary of the National Multiple Sclerosis Society, in its early development of a molecule to tame abnormal immune responses.

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 information, 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 autoimmune 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 intersected 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 discovery in the university laboratory setting, which are vital, also require a commercial 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 companies, 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 different 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 difficult time getting out of academia, so he began identifying the barriers in medical research that prevented novel therapeutics from reaching MS patients. The standard industry approach to MS was to suppress 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 damages it. With his business background, he also noticed a lot of disease organizations 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/solutions. 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 candidates 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 foundations will do due diligence. We had to submit an application to Fast Forward, and the application was reviewed by an expert committee made up of scientists, neurologists, 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 approval about a year later. Through their due diligence, they can evaluate a company to determine whether it has the right expertise, infrastructure, tools, molecules, and the means to carry out the experiments. And if you don’t, they may help you create “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 longerterm vision of that holy grail of a way to slow or halt the disease progression.

It is important to remember that the foundation’s vetting process can result in more than a financial boost. Many companies benefit more immediately from the network a foundation 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 symptomatic 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 amyotrophic 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 development of this potential drug. Fortunately, Project ALS gave us hundreds of thousands of dollars to allow us to start working on this new product. We then formed a consortium with them, along with the ALS Association and the Robert Packard Foundation and Dr. Jeffrey Rothstein, M.D., a neurologist and world-class ALS expert at Johns Hopkins Medical School. This led to the generation of extensive preclinical data.

COETZEE: One of our advantages as a major funder of bench-to-bedside MS research is that we have an unbiased view about what’s exciting and happening in a particular disease and, importantly, where the gaps and opportunities are. As in the case of Amplimmune in those early years, we start looking at opportunities if the scientific underpinnings of the program are sound, have strong potential, are innovative, and have a clear runway for development. We also look at whether they have a strong business and science team. Then we step through a process of bringing together scientific and business experts in a VC-like due-diligence process, where we give them feedback on where we see positives and negatives in their program and where it might be improved in order for us to make our investment. We even facilitated a connection between Amplimmune and an academic collaborator, Dr. Steve Miller of Northwestern University.

The MRF focuses its support for drug development even further back in the PoC process, functioning as a nonprofit research laboratory churning out new discovery, translational, and preclinical tools, such as a new mouse model, for the focus area of the Foundation: to support the drug discovery of myelin repair MS therapeutics. Then it essentially makes the tools readily available, with a low-cost barrier, to any company to test a new drug for myelin repair. It also sponsors and advises MS clinical trials in myelin repair.

CHANG: When MRF began in 2004, there was no pharma company investigating myelin repair in MS. Our five-year goal then was to attract pharmaceutical interest; in 10 years, we wanted to get to Phase 1, and by 15 years, bring a drug to market. In our founder’s view, if we don’t meet those ambitious time lines, we deserve to fail, but so far we are on track, even ahead of schedule. In our expert research meetings, when the academic researchers present their latest findings, the industry experts on our advisory boards are there to say, “If you want this to reach patients, you also need to think about X, Y, and Z.”

Harmonize constituents. Matching partners’ vision and goals also often requires managing the expectations of their various constituents. Foundations have their patient, contributor, and scientific groups to satisfy. Companies have investors and shareholders, boards, regulators, and so on. It’s an ongoing challenge, but both sides can anticipate many of the challenges by communicating and defining realistic expectations during the partnership’s conception and thereafter.

CHOWDHURY: You don’t want to alienate your bases, so while you may begin to do more with industry, you want to make sure it’s not at the expense of important activities in academic research labs. Managing patients’ expectations is always critical, but the need varies between disease areas and foundations based on the state of the science. At MJFF, we are very optimistic, because the pipeline in Parkinson’s disease is incredibly robust. But we are also very grounded in reality. We explain to our patient and supporter community how difficult drug development is and how much is still unknown about the disease. Still, you don’t need to know everything to find a solution.

Experience in partnering with foundations at his previous and current companies tempered one executive’s enthusiasm with a clear-eyed view of partnering challenges and taught him the need for discernment among the diversity of foundations, a wider view of partnering goals, and an adherence to high principles.

ANDREW GENGOS President And Ceo, Immunocellular Therapeutics:
The patient populations and not-for-profit disease groups out there are all very different. Some are way ahead in thinking about formal relationships with industry in drug development, and others are in their infancy. If your company is working on a particular disease, you have to understand the landscape of groups that are focused on that disease. I wanted to position our company with the right group that would provide support and potentially amplify our influence with Congress and the FDA. What I finally settled on were the grassroots patient groups. The reciprocity for your partner is honesty about your company and your drug development. You have to be honest, transparent, and build a trust-based relationship. And you’ve got to be genuinely caring about their plight, as individuals and as a group, so if you ever need their support, they will be there for you.

Thus ends part one of our four-part series. Watch for part two, “Resource Alignment,” in next month’s Life Science Leader. Many thanks to Travis Blaschek-Miller at BayBio for his help with this article series.