Archives for the month of: April, 2013

Last night I attended the launch of the Cystic Fibrosis Trust’s new research strategy. Our charity partner for 2013 marked the occasion with an international scientific conference, held at the Wellcome Trust, and it was good to see the UK biotech community well represented with companies like Vertex and Heptares and financiers like Abingworth all there. The UK’s Chief Medical Officer Professor Dame Sally Davies also spoke and it was good to catch up with her on NIHRs continued commitment to biomedical research.

Janet Allen

I’ll let Janet Allen the Director of Research at the CF Trust take up the story here:

“This launch is a big moment for the charity and reflects the exciting opportunities that exist in the area of cystic fibrosis and also in the wider research environment. The Cystic Fibrosis Trust has a strong and proud tradition of investment in research to advance treatment opportunities for people with cystic fibrosis. I hope this strategy will position the Trust to be able to take full advantage of these exciting times and to maximise the impact on the health and well-being of all people with cystic fibrosis and their families.

“The strategy has come out of extensive and widespread consultation with stakeholders in the UK and overseas, we are grateful to everyone for their generous time and input. The strategy will change the way we manage research investments. In particular, we will be more outward looking and seek to identify synergies and work with other funding agencies to increase the research awareness (whether government agencies such as NIHR and the research councils, such Medical Research Council, other research biomedical charities and industry).

“Our aim will be to manage a research portfolio that delivers solutions for all people with cystic fibrosis, so assisting in the delivery of the novel transformational treatments in the clinic whether they be small molecules, gene therapy or seizing new opportunities as they arise. But we also need to ensure we support research that slows the progress of the condition. Key features for the new strategy are to increase the quality and capacity for clinical trials, to recruit the brightest and best to cystic fibrosis research and to increase the voice of people with cystic fibrosis in the research agenda.

“These are exciting times in an era of unprecedented gains in treatment of the basic genetic defect that causes cystic fibrosis, we must forge a path forward in this new era and be at centre of driving research. Cystic fibrosis a fight we must win!”

The strategy, is published on the Cystic Fibrosis Trust website. A video and my previous blog on why the BIA has partnered with the CF Trust this year is here.

Ernst & Young Beyond Borders 2013Today, BIA member Ernst & Young published its 27th Beyond Borders report on the biotech sector.

It is pleasing to see that the UK still has the strongest pipeline in Europe, with more than 400 products in pre-clinical, phase I, II and III trials. Additionally, the UK led Europe in innovation capital raised – $467m – and was second to Germany (which has two significant financings by family offices) in venture capital raised – $220m.

The report highlights the need for companies developing new therapies to think at an early stage about the pricing, reimbursement and access profile of the candidates. This is something that has also been discussed at some of our recent conferences.

E&Y suggest that companies need to understand not only the safety and efficacy profiles of their products but also how it is differentiated relative to both current and prospective standards of care. Failure to do this can result in delays to partnerships or lower valuations.

The report suggests that many biotech companies do not fully appreciate this changing landscape and highlights five “myths” that companies perceive as well as proposing five “guiding principles” to help companies recalibrate their R&D commercialisation.

These guiding principles are:

  1. Define your value proposition
    • Even in early development, seek to understand your product’s contribution and why payers would value it
    • Solicit input from key stakeholders
    • Refine your value proposition as your product moves through phases of development
  2. Understand standards of care and value pathways
    • Understand the current and future standards of care
    • Map value pathways for individual diseases and understand the biggest causes of value leakage
    • Prioritize value leakages that payers most care about
  3. Identify new solutions for value leakages
    • Use approaches such as drug delivery technologies and personalized medicine to fill value leakages
  4. Design relevant clinical trials
    • Consider head-to-head trials to influence comparative effectiveness research
    • Consider adaptive trials to identify responding subpopulations while using capital efficiently
  5. Defend your product after launch
    • There is no finish line
    • Monitor evolving standards of care and be part of the conversation
    • Explore ways to conduct “value mining” by partnering with others

E&Y says that it will be necessary for companies to help patients by delivering new products but also to understand the patient’s journey through value pathways and identify their biggest unmet needs and to develop the solutions to these needs.

E&Y has also updated its European documentation this year and has been distributing a handout at the BIO Convention 2013 that includes recent tax changes in the UK including the patent box. It shows how the UK measures up as a globally great place to start a biotech business.

Abcodia_MikeFisher photo 18.4.13Biomarker validation company Abcodia provide the last in our series of guest blogs from BIO 2013 International Convention attendees. Mike Fisher, Abcodia’s Director of Strategic Alliances, describes how he hopes the BIO convention will benefit the company.

Abcodia Attendance at BIO 2013

Abcodia is honoured to have been judged one of the UK’s most innovative life science companies in a UK Trade & Investment competition, winning the opportunity to attend BIO 2013 as ambassadors of UK biotech innovation.

Curing Cancer by Catching it Early

Abcodia is a biomarker validation company with a focus on cancer screening. We passionately believe that diagnosing cancer early is the best strategy for improving mortality and enables the best treatment options. Our mission is to enable this through validated biomarkers for cancer screening and early detection.

Abcodia was formed in 2010 as a spin-out from University College London which operates a unique partnership model for provision of serum samples, patient data and experimental design expertise that does not exist anywhere else in the world. It has exclusive access to the largest blood serum biobank of its kind in the world.

The biobank contains more than 5 million longitudinal, pre-diagnosis serum samples collected as part of the UK Collaborative Trial in Ovarian Cancer Screening (UKCTOCS) which recruited 202,000 healthy women between 2001-2004. Additionally, 50,000 volunteers donated serum every year for up to 10 years. All volunteers gave informed consent for the samples to be used for research purposes, including commercial research.  The health of the volunteers has been followed over the years, during which time many women developed various diseases. For example, we now have 27,000 cases of cancer within this large cohort. This unique biobank is therefore ideal for assessing how biomarkers perform years ahead of a standard diagnosis as we can effectively go back in time with our longitudinal samples to identify those markers that are elevated early in disease progression.

Global Partners Wanted

Partnership is key in our mission. We are developing our own products and hope that our first screening test will be on the market within the next couple of years, but there’s so much more we could do in partnership with other companies and academics that are trying to develop biomarkers. We have the samples that can validate biomarkers without lengthy and costly clinical trials.

We are also interested in working with the biotech and pharmaceuticals industry as our samples can be used to look for drug pathways that are elevated early in disease onset. This work will give strong evidence that new therapies would ultimately be front-line and could be used early in disease treatment.

Attendance at BIO 2013

BIO is the major commercial biotech conference on the international calendar and we see it as the best platform for Abcodia to develop collaborative global partnerships which will help us achieve our goals. We will be taking part in business partnering meetings and as many networking opportunities as possible. Please get in touch (Mike.fisher[at]abcodia.com) if you’d like to explore partnership opportunities with us.

We would like to thank UK Trade & Investment and the UK BioIndustry Association for all their generosity and support and we hope that Abcodia can fly the flag for the UK biotech industry.

cell_therapy_catapult photo 18.4.13cell_therapy_catapult logo 18.4.13The Cell Therapy Catapult is attending the BIO 2013 International Convention this week. Emma Palmer Foster, Strategic Communications Consultant, outlines why international partners looking to access European cell therapy markets and innovation ought to look to the UK.

The UK is a great place for the development of cell therapies, which have the potential to treat and cure many debilitating diseases. It has a strong academic and commercial early-stage research base, access to clinical trial infrastructure/patients within the NHS and regulatory expertise, and is the home of the European Medicines Agency. Those are some of the reasons why the UK Government’s Technology Strategy Board established the Cell Therapy Catapult as a centre of excellence in 2012, with the aim of catalysing the development of a strong industry by providing expert services in this area.

What’s more, the Cell Therapy Catapult has significant funds at its disposal – £70m over the first five years – to help tackle some of the major challenges companies and other organisations in this area face in translating potential treatments into something that’s properly ready to enter Phase III. (We’ve been open for business since the end of 2012, and are already seeing strong demand for our services.)

The Cell Therapy Catapult is tackling three sets of challenges experienced in the sector:

  • the business model – factors such as health economics and perceived investment risk
  • manufacturing & supply chain – issues like COGS, scale-up and delivery
  • clinical & regulatory – understanding the regulatory environment & accessing the NHS

That’s why the Cell Therapy Catapult is establishing expert teams and state-of-the art translational labs in these areas. With an anticipated headcount of 50 by the end of 2013, and approaching 100 at full capacity, we plan to be one of the largest commercially focused cell therapy organisations in the UK.  There’s a huge amount of flexibility in the way we work with clients, with the ability to undertake a range of projects, from three-month suitability studies through to 12-month platform projects and three-year clinical trials. There’s flexibility too in how we structure interactions, from contract development to collaborations, meaning there’s a variety of ways for you to access our services.

The Cell Therapy Catapult is looking forward to BIO 2013 as a great opportunity to talk to cell therapy companies about how to help with rapid European commercialisation, as well as access to innovation.

So come and talk to us if you’re interested in:

  • accessing the regulators and understanding the framework
  • accelerating clinical trials
  • establishing a European manufacturing base
  • sharing development costs with experts

The Cell Therapy Catapult will be part of the UK BioIndustry Association and UK Department of Trade and Industry delegation on stand 4441 – please come and meet us there. Or get in contact beforehand if you want to make an enquiry or join our mailing list.

Critical Pharmaceuticals

Gareth King, CEO of Critical Pharmaceuticals, previews the company’s presence at the BIO 2013 International Convention, the world’s largest biotechnology event.

This week, licensing and business development leaders from pharmaceutical and biotechnology companies located world-wide will assemble in Chicago at the Business Forum of the BIO International Convention. I look forward to joining them, as CEO of Critical Pharmaceuticals, an innovative UK-based biotechnology company developing a pipeline of unique biological products utilizing our proprietary drug delivery technologies. Months before the BIO convention starts, the BIO One-on-One Partnering system has enabled me to maximise the time I have in Chicago by engaging with potential partners for our lead development programs and nasal and long-acting depot drug delivery technologies.

My key focus at BIO is to seek partners for Critical Pharmaceuticals’ clinical stage programs:

  • CP024 – intranasal human growth hormone for the treatment of endocrine disorders. CP024 has established clinical proof of concept from two phase 1 studies, with equivalent bioactivity to subcutaneous injection of marketed product. Moreover, it is the first intranasal human growth hormone product to induce the clinically important biomarker IGF-1 in man.
  • CP046 – intranasal teriparatide (parathyroid hormone or PTH 1-34) for the treatment of osteoporosis. This product has demonstrated increased activity in cell culture models, exceptional relative bioavailability in preclinical studies and a clinical trial in post-menopausal women will start shortly.

Both CP024 and CP046 have significant advantages over existing daily injectable products and are now at a stage that we want to secure partners for their further development and commercialisation on a world-wide basis.

BIO also offers a great forum for discussions with potential partners who are looking to develop novel and differentiated products utilizing our platform drug delivery technologies:

  • CriticalSorb – for non-invasive intranasal delivery.
  • CriticalMix – long-acting injectable depot technology.

Both these technologies are particularly well suited to the delivery of biological drugs: Biologics account for more than 30% of new drug applications and yet typically require frequent injections.

Critical’s technologies have been proven in clinical and preclinical proof of concept studies and further validated in our partnered programs with biopharmaceutical companies on their own novel protein and peptide therapeutics.

The BIO One-on-One Partnering system has enabled me to quickly establish the key interests of the attending pharmaceutical and biotechnology companies, including their focus therapeutic areas and types of opportunities they are seeking, and to secure meetings with members of their business development teams. My meeting schedule has filled up rapidly with only a small number of 30 minute ‘slots’ still available. This is a very efficient, albeit exhausting, way to meet a large number of potential partners to introduce Critical Pharmaceuticals or to update already interested partners on our drug programs and platform technology. I look forward to a very productive few days engaging with potential partners and collaborators in Chicago.

PRR_5056

The BIA held a breakfast roundtable discussion to examine French and UK life sciences  in the House of Commons on Wednesday 17 April which was kindly hosted by George Freeman MP, the Government Adviser on Life Sciences. What policies work (or don’t) and what experiences can we usefully share was the order of the day.

We were very fortunate to have been supported and joined by colleagues from Sanofi – with experience of operating in both countries –who were able through their excellent contacts to bring  Jean-Jacques Yarmoff, Director of International Partnerships at Oseo (roughly the equivalent of the Technology Strategy Board and the Business Bank in France) to London to speak.

There were 30 experts around the table to discuss the issues including chief executives of bioscience companies, investors, finance professionals and policymakers leading to a fascinating discussion around industrial strategy, funding schemes for innovative companies and a good chance to compare and contrast the different policies in the UK and France.

George Freeman MP, who is a key figure behind the launch and continued implementation of the Strategy for UK Life Sciences, outlined where we were up to and the challenges and opportunities presented by any industrial strategy. It is clear that there is interest in what the UK government is doing for life sciences across the channel.

Jean-Jacques was able to outline some of the key policies used in France to support innovative companies. This includes the successful FCPI scheme on which the BIA has modeled our Citizens’ Innovation Funds (CIFs) proposal. He explained that at every stage of development the French government have a range of tools to support company growth whether through the tax regime or direct guarantee based schemes.

Iain Wilcock, a partner at the French Venture Capital firm Seventure which runs FCPI funds, was also able to provide his insights to the attendees on the shape of the scheme, how it operates and some of his observations from operating in the thick of life science investment.

I was also delighted that Iain Wright MP, the Shadow Business Minister with responsibility for innovation, was able to attend the discussion and share his enthusiasm for the sector and the exciting innovation underway in the UK.

I drew a few lessons from the conversations:

  • It remains important to look beyond borders for what others are doing on a global basis. Often the challenges we face are shared and the solutions can be also. Bioscience is already a very global sector but the policy development can be too.
  • There remains cross-party support in the UK for the life sciences sector in the UK.
  • There are subtle, yet very important, differences about the way government support can be structured which has a big impact on their performance. There were some interesting comparisons made between the FCPI regime and UK VCTs and the ability of the former regime to better create syndicates and attract inward co-investment.
  • The French have done a good job in linking the different aspects of government support together – whether its innovation grant funding, tax credit support or public bank investment. The UK could usefully learn some lessons here.
  • Technology transfer, how to best achieve it and what the policy objectives should be, remains an issue of hot debate across both sides of the channel.

Overall a very useful discussion which gave the BIA lots of food for thought and further policy areas to pursue with members in the coming months.

There are more than 400 delegates from the UK attending the 2013 BIO International Convention. These include delegates from four innovative life science companies – Abcodia, Critical Pharmaceuticals, Nanomerics, and PsiOxus Therapeutics – who won the recent UK Trade and Investment (UKTI) Innovation Competition and will showcase their technologies to an international audience in Chicago.

The UK presence at the convention will be centred on the UK pavilion (stand 4441) hosted by the BioIndustry Association (BIA) and UKTI and with 20 companies exhibiting. We invite conference attendees to visit the pavilion throughout the convention and to join our celebration of the 60th birthday of DNA on Tuesday 23 April at 5.00 pm.

Modern biotechnology started 60 years ago in the UK as a result of the discovery of the structure of DNA by Watson and Crick working at the Medical Research Council (MRC) laboratory in Cambridge.

This discovery underpins an industry – including medical biotechnology, industrial biotechnology, medical technology and pharmaceuticals – in the UK with 4,500 companies, 167,500 employees and a turnover of £50 billion. This is the largest life sciences sector in Europe and globally is second only to the US.

In the medical biotechnology segment there are:

  • 979 companies, of which 307 are developing therapeutics
  • 25,000 employees
  • 876 drugs in the pipeline – 510 of which are biologics

As well as discovering the structure of DNA scientists in the UK discovered penicillin and monoclonal antibodies, and one third of the human genome was mapped at the Wellcome Trust’s Sanger Centre. Additionally, Humira – the world’s biggest selling drug, with sales over $9 billion in 2012 – is one of many leading therapeutics discovered in the UK.

In 2015 the Francis Crick Institute , an interdisciplinary medical research institute which brings together the specialist knowledge, expertise and resources of the MRC, Cancer Research UK, the Wellcome Trust, UCL (University College London), Imperial College London and King’s College London, will open its doors. The institute, the result of a £650 million investment, aims to help understand why disease develops and find new ways to prevent and treat illnesses such as cancer, heart disease and stroke, infections, and neurodegenerative diseases.

We are fortunate that the UK government is supportive of life sciences and launched a Strategy for UK Life Sciences in December 2011, which aims to provide the foundation for a successful and sustainable future for the bioscience sector in the UK.

The strategy’s most successful initiative is the £180 million Biomedical Catalyst fund, jointly managed by the MRC and the Technology Strategy Board, which aims to nurture innovative technologies from the academic or commercial sector through to a point of concept in order to attract private equity. To date more than £90 million has been allocated and this funding has successfully leveraged additional match-funding of more than £40 million private sector investment in to UK biotech companies. This funding is enabling companies to do more research and grow faster in a strategically vital sector for the UK economy.

In its December 2012 update of the Strategy for UK Life Sciences the government announced it would be establishing a National Biologics Industry Innovation Centre. This £38 million investment will be a centre to address challenges in process development and manufacturing of biologics.

Although not specifically for the life sciences sector the government has also introduced other policies to ensure the UK is a destination for inward investment:

  • Corporation tax is 24%, with a reduced rate of 20% for SMEs, and will be cut to 20% by April 2015
  • The Patent Box will allow innovative companies in the UK to benefit from a reduced corporation tax rate of 10%.
  • An above-the-line R&D tax credit of 10% for larger companies
  • SME R&D tax credit relief at 225% of costs

The UK is also home to the European Medicines Agency which is responsible for the scientific evaluation of medicines developed by pharmaceutical companies for use in the European Union. Additionally, the European Union recently adopted a Unified Patent which covers the 27 member states, and will establish the life sciences section of its Unified Patent Court in London.

Over the last year or so there have been a number of funds established to support innovative bioscience in the UK:

Johnson & Johnson has chosen London as the location for the first of its four global innovation centres and is looking forward to collaborating within the UK, which it considers to be home to one of the world’s thriving life sciences ecosystems.

With its world-leading science base, government support and a knowledgeable investor community the UK is a globally competitive location for bioscience companies. Those companies are working to develop their pipeline of innovative products to help address the needs of patients. Please drop by the UK pavilion (stand 4441) to find out more.

It was great to see stakeholders from across the life sciences ecosystem come together with a common agenda of making it easier to bring innovative drugs to patients at the Empower Halpin protocols launch at the Houses of Parliament on Monday.

As there were so many eminent speakers on the panel I confined my remarks to commenting on what would help Les not only someone with Motor Neurone Disease, but also as a successful businessman with a track record of successful investment and Chairman of Halpin Neurosciences, a privately-owned biotech company.

I argued that we need to change two sets of rules at the same time to get more novel therapies to patients quicker:

  • First we need to change the rules that govern drug development to reduce the time and cost of bringing drugs to market.
  • Second we need to change the rules governing investment management to also fit the risk and return profile for the good of society.

If the drugs could reach patients earlier and be applied to a wider number of conditions, and if the testing regime was simpler, this would help spur significantly more companies into developing new drugs.
Here I echoed the comments of others on the panel – the regulatory regime is out of kilter with the public mood. The risk appetite of patients with terminal diseases is different to that of regulators who have a legal responsibility to regulating medicines.

However, regulators need to change to reflect that fact and patients have a key role to play in helping them on that journey. The US Food and Drug Administration and the European Medicines Agency have started to get patients involved in key committees, but, their pace is glacial. I wondered if this was an agenda UK policymakers could take up?

Speeding things up is vital. To create greater incentives for truly innovative new medicines the BIA has long advocated for the new routes that can be used to get breakthrough medicines through to patients sooner and we have outlined this in the early access scheme we have proposed to the UK government.

We’ve already seen successes using conditional approval and flexibility in the evidence needed in orphan diseases. It was great to hear from Simon Lem of Vertex about how their Cystic Fibrosis drug Kalydeco is a great recent example of how patients can benefit from breakthrough products when things go well.

Although some good work has been done we need to keep going. It inspires those of us in industry to know that patients want this to happen and I believe this campaign can deliver the speed and focus to ensure regulators start to listen.

While the BIA has led the charge on an earlier access scheme, which could be used to deliver existing drugs to more patients in need, there is a second set of rules (and attitudes) that also need to be challenged: the rules that govern investment in risk from big financial institutions – like pension funds. Currently, there is no incentive for pension funds in the UK to invest in what matters to patients – it’s considered too risky.

It would be pointless to have lots of people starting companies but being unable to fund them as there will always be a cost for developing drugs however much we speed up the process or change the regulations.
However in the USA there are a lot more investors prepared to invest in innovative drugs because in part investment rules force big pension funds to invest a small part of their funds in riskier investments like biotech.

I believe we should campaign to ensure all UK long term investments have some degree of risk investment (which is where growth is most likely for returns). This should provide a greater pool of capital to invest in breakthrough science for treatments, especially for the smaller companies who are most likely to take the biggest risks. Here Les’ career expertise as an investor is vital – to make a real breakthrough we need to make investments in biotech as speedy and attractive as an investment into an app or technology platform.

If we can change the regulators approach to risk and speed up drug development and change the change the risk attitude of investors we will deliver more innovative products to patients sooner.

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