Q&A: The role of disease focused foundations in bridging the drug discovery and development gap

Bio 2017 opens this week and Drug Target Review’s Niamh Marriott spoke with Richard Soll, Senior Vice President, Research Service Division, WuXi AppTec and Salvatore La Rosa, Vice President Research and Development, Children’s Tumor Foundation, to get their perspectives on one of the event’s most pressing trends. Both will be presenting during a key panel session that examines the increasingly prominent and positive role that disease focused foundations are playing in bridging the translational gap between discovery and drug development.

Disease focused foundations Q&A

How are disease-focused foundations bridging the funding gap between new targets being discovered and getting them into clinical trials?

SLR: Today disease-focused foundations present the opportunity to develop new models where they can play a niche role in their respective rare disease ecosystem. The Children’s Tumor Foundation (CTF) has developed a business model, which is based on two principles: Firstly, rare disease foundations occupy a unique intersection that includes patients, researchers, clinicians and pharma. Secondly, rare disease foundations can act as targeted investors, directing funding towards integrated platforms of critical R&D endeavours. These include things such as creating a disease registry for patient recruitment, a biobank to guarantee tissue availability, and an open data hub to share data and disseminate results.

Has this role evolved over the last few years to become more significant, and why?

RS: In discussions with foundations, we have seen a significant increase in interest toward becoming more engaged in R&D, particularly in light of pharma’s growing efforts in the development/commercial side, compared to R&D. There is direct funding of R&D projects by disease-focused foundations to academics, biotech and pharma companies, and this also includes participation by a CRO such as WuXi.

SLR: Giving for a ‘good’ cause is not enough anymore, as the goal for donors has become not only to invest in something they are interested in, but to also impact the field in a tangible way. In order to demonstrate the impact donors are looking for, the overall foundation landscape has drastically changed. Medical foundations have become increasingly innovative in participating in the R&D process, helping to get the right drug candidates through the clinic and to the market.

How do you see the role between biotech or early stage discovery company, foundations and research partner like WuXi?

RS: We view open access platforms as a critical component to any company’s R&D effort, whether we run a fully integrated programme or conduct a one-off study. Whereas the foundation may provide the capital, reagents, and other expertise related to a particular disease, it is the biotech that would typically drive towards key inflection points or milestones associated with a compound’s advancement in the biotech’s pipeline. WuXi supports this initiative through execution. We have been involved with these organisations in the following ways: hit-to-lead and lead optimisation programmes; manufacturing and GLP toxicology studies; pharmacology studies or pharmacodynamic studies; and early stage target ID. For neglected diseases, the targets tend to be validated already, so the sponsor is often looking for a composition that has some kind of advantage over existing therapies if available.

What are the long-term implications for biotechs, early stage discovery companies/institutions, and/or big pharma?

SLR: Modern medical foundations have shifted their focus towards facilitating all stakeholder interactions, and the ownership of specific resources and platforms will allow them to claim a seat at the table for strategy setting and negotiation with industry. Companies that are eager to enter a specific disease space will be attracted by ready-to-be-used tools that will allow them to relatively quickly reach a proof-of-principle, and to move development of products at a faster pace. This synergy will translate into more long-term collaborations, or partnerships, that will benefit both parties.

RS: Sustainability of efforts by the foundation throughout the entire drug development process is questionable. R&D, especially the latter phases of drug development, is costly so a foundation’s ability to participate will be dependent upon the available capital.

With respect to diseases, according to Faster Cures, there are circa 10,000 diseases today (likely more as we get to understand the genetic basis of disease) but there are only circa 400 treatments. Under the current approval rates of 20 – 40 drugs per year, it will take hundreds of years to achieve treatments for all diseases. Therefore, any help in ensuring targets get into drug development is obviously going to increase the chance of reducing this time frame.

How does WuXi ensure the targets move more quickly and cost-effectively into later stages of development? Can WuXi help ensure these vital donations and grants are spent wisely?

RS: It boils down to the value we bring for the sponsor. WuXi has a track record of cost-effective delivery, from the many tens of thousands of compounds it prepares every year for its customers to the delivery of more than 27 preclinical candidates to its partners over the last 2-3 years. Our biology business is built upon quality and speed, so that customers count on our teams to perform well and meet industry standards. Our measure of success is the success of the sponsor’s programme and their continuing use of the open access platforms.

On what basis do foundations make decisions as to whether to provide funding and/or which targets to provide it to?

SLR: Foundations are driven by their mission and they represent their patients. Although, in the past, scientific advisory boards and disease experts mainly drove decisions, conflict of interest remains one of the main concerns/challenges, especially in very small disease communities. Patients are becoming increasingly involved in all decision processes, working side-by-side with researchers and clinicians, and steering towards different approaches to studying a disease.

Can you envisage in the future disease-focused foundations taking a share in early stage companies or looking for returns, should a target be sold to a pharma company?

SLR: Some large foundations already have this type of approach; I see the vast majority of small and mid-size foundations securing capped returns or long-term revenues with those partners who agreed to partner and use their services. Even very small patient organisations could monetise a revenue from a collaboration where prompt access to patients for clinical trial recruitment, or disease experts for trial design, could speed up development programmes in a meaningful measureable way.