Earlier this year, 8 March 2017, Parkinson’s UK and the University of Sheffield launched a new £1 million virtual biotech company, Keapstone Therapeutics, as part of a research programme to create novel medicines for Parkinson’s disease. Arthur Roach, Director of Research and Development at Parkinson’s UK spoke to AMRC about the partnership and the resulting spin-out company.
Parkinson's UK is the UK's leading charity supporting those with the condition. Its mission is to find a cure and improve life for everyone affected by Parkinson's through cutting edge research, information, support and campaigning.
Why did you decide to partner?
“Scientists at the University’s Sheffield Institute for Translational Neuroscience (SITraN) have spent more than a decade working on a research project that has identified a possible in-built defence system that helps protect brain cells from oxidative stress. This stress is caused by a damaging build-up of free-radicals and is found in the brain cells of people with Parkinson’s.
“We felt the project was so promising that a full scale drug discovery approach was warranted to develop it further, and that launching a virtual biotech company was the best method for this. The virtual biotech will effectively ‘own’ the research project and act as an investment vehicle for further funding. Since Sheffield was already the owner of the starting intellectual property, they were key players in the start-up.”
How did this partnership come about?
“The collaboration was based on the idea that biotech companies are the most effective way to accelerate drug discovery at the very high standard needed to attract investment from large pharma companies during late-stage clinical studies.
“Rather than give a one-off grant to the university, we wanted to try and create a spin-out biotech company that would ensure that the research programme would be in the best possible position to be progressed beyond the next stage of drug development.
“We approached the University of Sheffield with the idea, agreeing that we would fund the company to get off the ground, and potentially follow the company for several years with further funding if needed. They thought about it for less than a day and decided that would be an interesting model to try.”
What is a virtual biotech company?
“It is a biotech company that has no staff, no fixed premises and no expensive laboratory equipment. It completes the work required with staff that have been seconded to the company or by contracting work to research organisations – but it still has a legal structure and retains the rights to all Intellectual Property created.
“Setting up drug discovery and development projects in this way will allow Parkinson's UK to deliver new treatments at a fraction of the cost. The charity can use its funding, donated by supporters, to find the most promising leads for impactful new treatments, and then to bring together the best scientists, companies and investors to develop those leads into new treatments.
“It also means Parkinson’s UK can stay agile - rapidly investing in the most promising, emerging, or neglected projects, and cutting off those projects that turn into dead ends to maximise the use of every penny spent.
“This new way of working will accelerate drug development for Parkinson's and create as much compelling value as possible in each project, to attract funding partners who will carry the project on their own or in partnership with continued funding - putting successful research in the best possible position to be taken forward.
“Furthermore, Parkinson’s UK will retain a share in any commercialisation rights – so if any of the research leads to actual treatments the charity will benefit financially. This could enable Parkinson’s UK to invest further into research or to provide further support to improve the lives of people living with Parkinson’s.”
How does the partnership work?
“Parkinson’s UK has invested £1 million in Keapstone Therapeutics’ first stage of research. This £1million will fund 16 months of work, to be conducted by a chemistry specialist company called Sygnature Discovery, which will develop research that SITraN started alongside the European Lead Factory.
“The research is currently in the Hit to Lead stage of development, so it’s still very early days – but the hope is that the research will remain promising enough to attract further investment and progress to the next level of drug development.”
What impact will this partnership have?
“We believe this type of partnership will be more effective at moving promising university research into drug testing, so in the end more drug trials, and more new treatments should result. If successful, the approach will also produce income, perhaps significant income, for the charity some years down the road.
“We believe we can act as a kinder, gentler source of seed financing for Parkinson’s - directed projects in small companies, and we are actively encouraging university investigators to take the plunge. This could give some of them alternate career options, while promoting the innovation-based sector of the UK economy.”
What challenges have you faced so far?
“Our challenges have mostly been the organisational and institutional buy-in issues that we expected, but it has taken a bit longer than we had hoped. We have had to find new partners and develop the confidence necessary to move into areas that are new to us as a charity. One area that has not been a challenge is fundraising. We have had a very good response from supporters, who are excited to see us taking this more business-like approach in one area of our research.”
What advice would you give to others considering launching a biotech company?
“Get people in your team who have worked in biotechs in a previous life; find well-recommended advisers in the domains that are new to you (for example biotech investment deals, intellectual property). Also, talk to trustees and supporters early and often about the initiative – I believe it is a great and motivating story!”