The project will leverage Healx’s extensive expertise in tech-driven drug repurposing for rare diseases and combine it with the PKD Charity’s unparalleled patient and disease insight to progress novel therapies for the conditions towards the clinic.
ADPKD is the most commonly inherited kidney disorder in the world, and affects roughly 3 in every 10,000 people across the EU and the UK. The condition is dominantly inherited, meaning there is a 1 in 2 chance of passing it on to children, and is caused by a genetic fault that disrupts the normal development of some of the cells in the kidneys and other organs, such as the liver and pancreas. ADPKD is characterised by the formation of multiple fluid-filled cysts on these organs. In the case of the kidneys, the cysts grow and multiply over time, replacing normal healthy tissue to the point that the kidneys start to fail. As these cysts grow, kidneys can enlarge up to four times their regular size and can weigh up to 100 times more. Alongside kidney failure, this can cause a range of problems including large cystic livers (which can require transplantation), chronic back and abdominal pain, urinary and cyst infections, high blood pressure, brain aneurysms and kidney stones.
ARPKD, the rarer form of PKD, also affects the kidneys and liver. It is most commonly diagnosed during pregnancy and in babies and young children. ARPKD occurs in roughly one in 20,000 live births, and is caused by an alteration in the gene PKHD1, which is passed on to a child through recessive inheritance by both parents. Parents of children with ARPKD don’t have the disease themselves but are ‘carriers’ of the disease and critically, around one in every 70 people is an ARPKD carrier.
Both ADPKD and ARPKD are currently incurable, and treatments vary in their effectiveness. In the case of ARPKD, there are no proven treatments to slow the progression of the disease but there are treatments to address other symptoms of the condition. For ADPKD, there are treatments available that can both slow kidney function decline and reduce the symptoms, but there remains a significant unmet need for treatments that can be better tolerated by the patient. By combining the PKD Charity’s patient and disease knowledge with Healx’s extensive drug discovery expertise and AI technology, this partnership aims to identify, and accelerate to clinic, new therapies for these conditions.
This tech-driven repurposing approach is central to Healx’s work and means patients can get access to life-changing treatments more quickly than traditional drug discovery and development methods allow. AI enables incomparable speed and lateral thinking on a scale that is impossible for humans to achieve alone and, in the case of drug discovery and development, can make the process quicker, cheaper and more efficient. In this project, Healx will be using Healnet – its AI-powered drug discovery platform – to interrogate drug-disease data and quickly predict the most effective drugs that could be repurposed into mono- and combination therapies for the condition. By focusing on known drugs, the team is able to further accelerate and de-risk the progression to clinical trials and reduce the likely side effects of treatments since the safety profile of the drug is already known.
Commenting on the partnership, Tess Harris, CEO at the PKD Charity, said: “We are delighted to be collaborating with Healx in this pioneering partnership which has the potential to accelerate the development of much-needed treatments for both forms of PKD. We know that the patients and families we support will hugely welcome this initiative. They are eager and willing to contribute their insights and experiences in the hope of new therapies that can slow down the relentless progression towards kidney failure in ADPKD and kidney and liver failure in ARPKD, as well as reduce the symptom burden and improve overall quality of life – not just for themselves but for future generations.”
Bruce Bloom, Chief Collaboration Officer at Healx, says: “We are incredibly proud to be partnering with the PKD Charity, the UK’s leading charity for polycystic kidney disease awareness and research, to find repurposed therapies for these life-altering conditions. Putting patients at the heart of the drug discovery and development process is central to all of our work at Healx, and we are looking forward to working with the PKD Charity to integrate their expertise and insight right from the start.”
The partnership with the PKD Charity marks the latest in a growing number of collaborations between Healx and patient groups across the globe, and the team is eager to extend its expertise in the area by collaborating with other groups working on polycystic kidney diseases. The partnership also extends the number of disease areas within Healx’s portfolio. Earlier this year, Healx announced that it would be working with Mission: Cure to identify the world’s first treatments for chronic pancreatitis, and in 2020 the team launched partnerships with the Foundation of Angelman Syndrome Therapeutics, Muscular Dystrophy UK and The Children’s Tumor Foundation.
Patient, industry and academic groups interested in collaborating with Healx to unlock the power of repurposed drugs for rare diseases are encouraged to apply to Healx’s Rare Treatment Accelerator (RTA). The RTA is Healx’s partnering programme for groups and organisations with promising drug repurposing opportunities for rare diseases who are looking for financial or clinical support to take the drugs to trial. Working together with selected applicants, Healx will leverage its AI, drug discovery and clinical expertise to accelerate novel therapies towards clinical trials in a typical timeframe of 6-12 months. To learn more about the RTA, please go to https//healx.io/rta.