Science and TechnologyWritten evidence submitted by Drug Discovery Centre, Imperial College London

Imperial College London embodies and delivers world-class scholarship, education and research in science, engineering, medicine and business, with particular regard to their application in industry, commerce and healthcare. The Imperial College Drug Discovery Centre, aims to translate the wealth of Imperial’s disease-related research into novel therapeutics using pharmaceutical industry expertise.1

1. What are the difficulties of funding the commercialisation of research, and how can they be overcome?

1. The discovery and development of novel therapies for diseases with significant unmet need is a difficult and high-risk activity, taking in excess of ten years. However, there is a great need for innovative treatments with high societal impact such as Alzheimer’s disease, cardiovascular diseases (eg heart failure), antibiotic resistant infections (eg MRSA) and others.

2. Data from Pharmaceutical companies suggests that only one in twenty five small molecule projects results in a commercial product. This level of productivity is much lower than any other industry. Further, current data suggests that each new drug costs $700 million–$1.5 billion to reach the market and 10–15 years to create. The long-term investment required for this high–risk, high cost, low productivity paradigm means that investment in this space is almost impossible to achieve. However, there remains a moral imperative to find innovative medicines for un-met need that bring with it very significant commercial opportunity, as evidenced by the historic success of the Pharmaceutical (Pharma) Industry.

3. The Pharma Industry response to this situation is to increasingly withdraw from the earlier and undoubtedly riskier (Pre-Clinical) phases of research. Most Companies are now planning on >50% of their pipeline being sourced from Academia and Biotech. However they, their shareholders and Investors are failing to invest in these phases, preferring to acquire new products that have been fully validated by others.

4. Further, these companies anticipate that other agencies, such as Research Councils and Charities, will support these high-risk activities until validated (de-risked) clinical candidates are identified. However, current translational schemes are inadequate to support academia and Biotech in achieving such candidates. There are very few investors prepared to provide funds for these high-risk stages.

5. The ability of the UK to effectively translate the wealth of its publically funded basic health research is declining as UK Pharma dismantles its own early stage R&D. Against this, the availability of the required industry skills (eg Chemistry, Pharmacology) due to job losses in the UK is high. However, this scenario will only exist for a limited time before the UK’s capabilities in this space will decline rapidly as will UK leadership in Pharmaceutical Sciences.

6. It is therefore urgent that the UK addresses the lack of funding in the therapeutic translation space. Many UK Research-based Institutions are currently using their own resources to create the capabilities that address this. We believe that resources from the BioMedical Catalyst Fund should be used to establish key “Centres of Excellence” for Research & teaching in Therapeutic Discovery; linked directly to the AHSC/BRCs for Clinical evaluation as the most efficient way of effectively translating basic research into validated novel therapies with significant commercial potential. This position has also been laid out in the Royal Society of Chemistry’s “Healthcare Innovation in the UK” position paper.

7. The Imperial Drug Discovery Centre (DDC) is an example of this: a small multi-disciplinary group of ex-industrial drug discovery scientists focusing on the validation of novel targets from basic and clinical research and then translating them to clinical validation. In four years the DDC has succeeded in pump priming >25 novel projects; progressing four to five towards clinical validation, creating one spinout company to date, with potential for a second in the near future. However, the work of the DDC is severely threatened by lack of funding.

8. There have been significant levels, of public investment (eg MRC, EPSRC, BBSRC) in basic health research and recently in Clinical research (eg NIHR, NOCRI). However, support for truly translational therapeutic activities linking these areas, such as that as that covered by DDC, is severely limited. The University funding model is not well placed to support this activity.

9. In order that the potential of the public investment in basic research is to be realized, it is essential that therapeutically aligned translational capabilities, such as the DDC are supported and made available to all UK Research institutions via “Centres of Excellence”. Such capabilities will directly enable the creation of commercialisable entities for development in the UK Biotech or Pharma Sector.

2. Are there specific science and engineering sectors where it is particularly difficult to commercialise research? Are there common difficulties and common solutions across sectors?

10. As discussed above the Life Sciences and in particular the Pharmaceutical, Biotech sectors have very significant challenges. The high risk and long development times of this work makes it a particularly difficult area in which to secure investment and to effectively commercialise products.

11. Models in which Open-Innovation and data sharing are actively pursued have been widely used and successful in Industries such as IT. Effective data sharing would increase the efficiency of this high cost but ultimately high value activity. However, there have been historical difficulties in establishing a significant common knowledgebase, that includes information on both successful and unsuccessful approaches, as most of this data is embargoed by competing companies.

3. What, if any, examples are there of UK-based research having to be transferred outside the UK for commercialisation? Why did this occur?

12. Even for projects with candidate compounds achieved with charity support, we have had difficulty in securing sufficient funds for the pre-clinical studies required to assess safety and efficacy in man. We have one example of a candidate for breast cancer for which no UK funding could be secured and which we are eventually partnering with a US institution. In accepting funds to enable clinical studies with this partner we will lose significant UK ownership of the asset.

4. What evidence is there that Government and Technology Strategy Board initiatives to date have improved the commercialisation of research?

13. The Funding delivered by the MRC for translational activities ie the MRC DPFS/DCS scheme, MICA, MRC/AZ, have and are supporting translation of therapeutics and devices. However, despite clear evidence to the contrary, their funding decisions suggest that they believe that early small molecule drug discovery continues to lie predominantly within Pharma’s remit. As such, they are not effectively supporting the development of new approaches to drug discovery from the academic community and addressing the gap left by the decline of Pharma R&D.

14. TSB schemes directly and effectively address areas such a Stratified Medicines very well for those who are successful in securing funding and industrial partners. Such schemes do appear to support commercialisation effectively.

15. However, there are too few schemes in the early therapeutics space partly due to the lack of available early R&D industrial partners. TSB calls are usually available for a very limited time and this makes it difficult to secure industrial partners for early phase therapeutic projects.

5. What impact will the Government’s innovation, research and growth strategies have on bridging the valley of death?

16. The UK benefits from an integrated healthcare system that should be capable of translating ideas from the bench to the clinic in a manner which cannot be done elsewhere. However, our understanding of important diseases like Alzheimer’s is not yet good enough to predict which drug targets which will deliver benefit in the clinic. Investment in fundamental disease biology understanding provides the seed corn for initiating drug discovery, but we can only validate these hypotheses by creating specific chemical and biological tools. The Universities are ideally suited to link these areas directly to the clinic, in a way that is outside the usual commercial and academic funding sources. This effectively excludes translation of these ideas.

17. The recently announced Strategy for UK Life Sciences has been widely welcomed. However, it is essential that initiatives be specifically targeted to the key “valley of death”. Specifically this refers to the Phases between basic disease research and identification of a suitable quality candidate therapeutic.

18. If these phases are suitably supported to create “Centres of Excellence for drug and therapeutics discovery”, this will enable better selection of robust novel targets, reducing project attrition and increasing the likelihood of achieving candidate therapeutics with commercial potential.

19. It is notable that much of this research sits outside the Commercial sectors, but within the academic space. As such suitable academic (pre-company formation) funding schemes, which enable such translational, research, the development of skills and training and of robust commercialisable candidates is key.

6. Should the UK seek to encourage more private equity investment (including venture capital and angel investment) into science and engineering sectors and if so, how can this be achieved?

20. The culture of philanthropic and early investors in countries such as the USA has a significant impact on the availability of funding for early translational research. A result of this is the lively and close relationship between Research Institutions and investors, which enables commercialization of new inventions from the institutions. The Boston area is an excellent example of this.

21. The government should actively encourage early, philanthropic and private investors with tax and other incentives (Consortium Relief, EIS). The Citizens Innovation Funds (CIF) suggested by the BIA is an interesting suggestion aimed at securing investment from mid-net worth individuals as well as high-net worth individuals.

22. It is essential that those who ultimately benefit from commercialized products be also encouraged to invest in their creation. This should include downstream Pharma companies (the customer for most early therapeutics) as well as patient bodies and advocacy groups. These groups are increasingly becoming an essential source of research funding in the USA.

7. What other types of investment or support should the Government develop?

23. Funding schemes that directly support the creation of academic “Centres of Excellence, for drug and therapeutics discovery” directly linked to the patients via the AHSCs/BRCs are absolutely key to this. Direct support of this area will enable greater numbers of validated therapeutics to be commercialized via spinout companies or UK based Biosciences Companies.

February 2012

1 www1.imperial.ac.uk/medicine/research/institutes/drugdiscoverycentre

Prepared 11th March 2013