An Irish startup team is developing a new protein-based drug to target inflammation, with the goal of eventually treating a range of diseases from multiple sclerosis to Alzheimer’s to sepsis.
Emer O’Shea, co-founder and CEO of Irish biotech company Khonsu Therapeutics, says more than half of the pharmaceutical industry’s pipeline is in these types of drugs, known as biologics. The global biologics market is predicted to be worth €340 billion by 2025.
Ms O’Shea, who has a master’s in Molecular Cell Biology with Bioinnovation from UCC, had been working in start-up Hooke Bio in the Stokes Research Institute at the University of Limerick when she was invited to join the team at Khonsu Therapeutics.
The UCC research group, including co-founder Dr Tom Moore, had been researching a particular protein for more than a decade and they had recognised its commercial potential. They had been accepted to take part in the SOSV-funded Rebel Bio accelerator.
Biologics are drugs made from living cells, which differ from chemically-derived drugs like aspirin in how they’re made, their use and their efficacy. The molecules in biologics are larger, and their effects are more specific and predictable.
The most successful biologic is Humira (adalimumab), approved for use in rheumatoid arthritis, psoriatic arthritis and other inflammatory diseases. Sales of the drug are predicted to reach €18bn by 2020.
When Ms O’Shea joined the team in April 2017, Khonsu Therapeutics planned to develop a drug for human use. However, early on they realised the challenges they faced in terms of cost and time of getting a new drug to market. The Tufts Center for the Study of Drug Development in the US puts the average cost of developing a new drug at €2.2bn and the average time at 10 to 15 years.
Ms O’Shea said: “As a startup company with a small team and very limited funding, and no track experience to leverage as validation, we were looking at how are we going to do it. Our protein is natural and produced by the body and exists in a lot of different animals, and it just happens to be in horses.”
The team decided to start with the veterinary route because the approval process is faster and significantly cheaper. Ms O’Shea says that they can potentially get the horse drug approved in two to three years at under €3m. The company plans to build a revenue stream with the veterinary drug, which will allow the team to work towards the ultimate goal of developing a human product.
Recent studies looking at diseases like Alzheimer’s, diabetes and depression have found that while the causes or pathogenesis are different, it’s all down to something gone wrong in the inflammatory response, Ms O’Shea says.
The recent ‘right to try’ Bill passed by the US Congress allowing the use of drugs that have passed safety tests, but are not fully approved, has created more incentive for drug companies to develop new drugs.
Another option for faster approval is developing a drug for an orphan disease like Motor Neurone disease, Ms O’Shea says. Drugs with the status of orphan disease — classified in the US as less than 200,000 people — have an accelerated approval pathway of seven to 10 years, instead of 10 to 15.
Khonsu Therapeutics received $100,000 (€85,000) in funding as part of the Rebel Bio, with $50,000 (€42,500) towards participation in the programme and $50,000 as an investment. The company was also granted two innovation vouchers from Enterprise Ireland of €5,000 each.
The company has conducted animal models testing where an anti-inflammatory and pro-repair mechanism were observed.
The tests showed promising results for the treatment of sepsis which causes 3,000 deaths in Ireland each year. With the sepsis model, the immune response was wiped out after six hours, and there was a thousand-fold difference between the drug and the control group, Ms O’Shea says.