C4XD and Garvan Institute of Medical Research report successful patient stratification approach using C4XD’s new precision medicine platform PatientSeek

First validation of C4XD’s patient sub-groups through a Garvan-led retrospective clinical trial analysis

Promising initial result paves way for novel approaches in Parkinson’s disease stratification

Launch of PatientSeek platform enables stratification of patients to identify the right patient for the right medicine

22 February 2023 – C4X Discovery Holdings plc (AIM: C4XD), a pioneering Drug Discovery company, announces the launch of a precision medicine platform, PatientSeek, following insights from a successful research collaboration with Garvan Institute of Medical Research (“Garvan Institute”).

In the collaboration, C4XD provided Garvan Institute with the genetic signatures for its PatientSeek sub-groups in Parkinson’s disease. Garvan researchers then applied this as part of a retrospective analysis of a failed Phase 3 Parkinson’s clinical trial that had not reached its primary endpoint, to assess if a genetic subgroup of participants showed a benefit from the therapeutic.  Very encouragingly, PatientSeek identified a subgroup that responded to the trial drug. These insights into Parkinson’s disease have the potential to help identify the most effective treatments and match them with groups of patients who are most likely to benefit, accelerating the drug development process.

These results provide the first validation of PatientSeek’s ability to identify patient subgroups to optimise patient selection, which in turn could lead to enhanced probability of targeted success in clinical trials. Whilst this study was focused on Parkinson’s disease the PatientSeek platform is disease agnostic and can be applied to any complex genetic disease. The results from this study will be submitted for publication in a peer reviewed journal in due course.

Garvan research partner, Associate Professor Antony Cooper, Research Director of the Australian Parkinson’s Mission, said: “This is a very important result which not only demonstrates that subgroups derived with PatientSeek are clinically relevant, but also reaffirms the important role that genetics play in Parkinson’s disease, including response to treatment. These findings have wide-reaching implications for patients, clinicians and researchers, and have advanced a key goal of the Australian Parkinson’s Mission of expanding precision medicine approaches to identify effective therapeutics for people with Parkinson’s disease. We will now focus on the mechanistic understanding of the differences between the patient subgroups identified to accelerate our understanding of the disease heterogeneity observed in Parkinson’s disease”.

Dr Richard Wyse, Director of Clinical Development at Cure Parkinson’s who introduced C4XD to Garvan Institute, said: “This work has enormous potential and paves the way for novel approaches to stratify patients in disease modifying Parkinson’s trials. Such approaches could inform clinical trial design, better select patients for their likelihood to be responders to specific therapeutics, and help enable development of companion diagnostics. This is a ground-breaking result and an important milestone in bringing precision medicine to patients.“

Dr Clare Murray, SVP Drug Discovery at C4XD, said: “Our new PatientSeek platform is the result of a successful Taxonomy3® collaboration with Garvan Institute. Taxonomy3® has historically been used for target identification, but this study clearly demonstrates the potential to use PatientSeek to apply our unique mathematical approach to stratify patients for novel treatments based on their genetics. C4XD has already identified PatientSeek subgroups in immuno-inflammatory disease and we will be exploring their application in bringing precision medicine approaches to these patient populations.”

C4X Discovery Holdings plc

Mo Noonan, Communications+44 (0)7876 444 977

Panmure Gordon (UK) Limited (NOMAD and Broker) +44 (0)20 7886 2500

Freddy Crossley, Emma Earl (Corporate Finance)
Rupert Dearden (Corporate Broking)

Consilium Strategic Communications

Mary-Jane Elliott, Chris Gardner, Matthew Neal +44 (0)203 709 5700

Garvan Institute of Medical Research

Carolyn Barry, Communications +61 (0)480 391 269

C4X Discovery (“C4XD”) is a pioneering Drug Discovery company combining scientific expertise with cutting-edge Drug Discovery technologies to efficiently deliver world leading medicines, which are developed by our partners for the benefit of patients. We have a highly valuable and differentiated approach to Drug Discovery through our enhanced DNA-based target identification and candidate molecule design capabilities, generating small molecule drug candidates across multiple disease areas including inflammation, oncology, neurodegeneration and addictive disorders. Our commercially attractive portfolio ranges from early-stage novel target opportunities to late-stage Drug Discovery programmes ready for out-licensing to partners and we have three commercially partnered programmes with one candidate in clinical development.

We collaborate with leading pharmaceutical and life sciences companies to enrich our expertise and take our assets through pre-clinical and clinical development. Through early-stage revenue-generating licensing deals, we realise returns from our high value pre-clinical assets which are reinvested to maximise the value of our Drug Discovery portfolio.  For further information see www.c4xdiscovery.com PatientSeek, powered by Taxonomy3®, is C4XD’s new platform for patient stratification based on clinically relevant genetic subgroups of patients to ensure the right drug is given to the right patient, based on their genetics. PatientSeek has the potential to de-risk drug development by identifying patient subgroups that are most likely to benefit from treatment and informing development of companion diagnostics. Garvan Institute of Medical Research brings together world-leading researchers and clinicians, collaborating locally and globally, to improve human health. Its mission is to harness the information encoded in the human genome to better diagnose, treat, predict and prevent disease. Through its key scientific strengths in data, genomics, cellular, translational and clinical science, Garvan aims to catalyse research from fundamental discovery to transformational impact. Garvan values its strong partnerships, most notably as a member of the St Vincent’s Health Innovation Precinct, and as a close collaborator with UNSW Sydney.

https://www.garvan.org.au Cure Parkinson’s is a UK-based charity that funds and facilitates research into disease modifying therapies to slow, stop or even reverse Parkinson’s. https://cureparkinsons.org.uk/