RNS Number : 5980J
e-Therapeutics plc
06 December 2018

E-therapeutics and C4X collaboration identifies new pathophysiological mechanisms in Parkinson's disease


Early results point to novel approaches to drug discovery for this debilitating disease 



Oxford, UK, 6 December 2018:  e-therapeutics plc (AIM: ETX, "e-therapeutics"), the network-driven drug discovery (NDD) company, and C4X Holdings PLC (C4X.L) ("C4XD"), are pleased to announce an update on their collaboration (announced 1st May 2018) to identify novel intervention strategies for the potential treatment of Parkinson's Disease (PD).


PD is a progressive neurological disease which affects up to 10 million people worldwide and incidence is increasing as the global population ages[1]. Whilst to date some PD drivers have been identified, this has not resulted in any novel therapies.


C4XD's proprietary genetic target discovery technology Taxonomy3® has found multiple novel disease-associated genes in Parkinson's Disease (PD) in addition to identifying discrete patient sub-groups that could potentially provide an opportunity in stratified medicine. It has been estimated that selecting genetically supported drug targets should double the success rate of investigational new drugs in clinical development[2].  These genetic discoveries provide a significant opportunity to uncover biological processes central to the causation and progression of disease.


e-therapeutics proprietary Network-Driven Drug Discovery (NDD) approach has been applied to this unique and rich dataset.  Using e-therapeutics' cutting edge technology, harnessing the latest mathematical and data analysis techniques to augment and interrogate this complex biological information, the study analysed approximately 200 PD-associated genes identified by C4XD's Taxonomy3 genomics platform.


Initial results derived using the advanced computational analytics of the e-therapeutics NDD platform have identified novel potential pathophysiological mechanisms relevant to PD as well as highlighting known mechanisms. The implications of these insights are now being explored further and suggest a number of novel intervention strategies that could be taken forward using ETX's NDD approach as well as providing another basis on which to select targets derived directly from C4XD's Taxonomy3® platform. Ultimately this approach might yield new drugs to treat this debilitating disease.


Importantly, the combination of network aware approaches and genetic target discovery has identified novel relevant biological pathways and processes that when targeted may subsequently address the pathophysiological phenotype. Key to this is the ability of the network perspective to tie together complex genetic information. This leads us to believe that the synergistic power of NDD with direct genetic findings will lead to new disease insights and identification of better treatments for PD.


Both C4XD and e-therapeutics believe their Taxonomy3® and NDD platform approaches are particularly relevant to diseases with poorly or partially characterised genetic predispositions, including PD, Alzheimer's, and inflammatory disorders such as multiple sclerosis. Both companies consider that further work in this area has the potential to transform the treatment landscape for these disorders, where there remains a significant unmet need.


Alan Whitmore, Head of Discovery Biology at e-therapeutics, said: "The initial results from this collaboration highlight the critical importance of considering biology in a network context to gain insights into clinically relevant biological mechanisms in complex disease. The ability to link genetic data to disease mechanism remains one of the greatest challenges of our industry. By using the advanced computational analytics of our NDD platform, we have been able to confirm the centrality of a number of known mechanisms in PD and, importantly, identify potential new ones. This in turn, opens up the prospect of new approaches to the discovery of effective novel drugs to tackle this and other undertreated, debilitating conditions."  


Craig Fox, Chief Scientific Officer at C4XD, said: "Following the identification of novel drug targets for the treatment of PD from the direct findings from our Taxonomy3® platform we recognise there still remains untapped potential in this proprietary analysis.  By working with the team at e-therapeutics and utilising their NDD platform, we have been able to access cutting-edge mathematical and data analysis techniques to augment and interrogate the vast amount of biological information currently available in both public and private databases.  This combination has identified additional novel biological pathways for the treatment of PD and we look forward to moving these findings forward to initiate new drug discovery programmes."




For more information, please contact:


e-therapeutics plc

Ray Barlow, Chief Executive Officer

Steve Medlicott, Finance Director


Tel: +44 (0)1993 883 125 


Numis Securities Limited

Michael Meade/Freddie Barnfield (Nominated Adviser)

James Black (Corporate Broking)


Tel: +44 (0) 207 260 1000


FTI Consulting

Simon Conway/Brett Pollard

Tel: +44 (0) 203 727 1000


About e-therapeutics


We are an Oxford, UK-based company with a unique and powerful computer-based drug discovery platform and a specialised approach to network biology.


Our novel network-driven methodology allows us to discover new and better drugs in a more efficient and effective way.


We use our highly productive drug Discovery Engine to develop our own IP-protected, pre-clinical drug discovery programmes which will be of interest to partners looking to acquire or in-license novel and differentiated assets. We are currently developing two programmes in immuno-oncology and have a number of partner-ready projects in areas such as fibrosis and tumour microenvironment.


Because of our novel network-driven drug discovery (NDD) approach, we believe there is potential to enter into several different types of collaborative partnerships with biotech, pharma and other technology companies to create sustainable mutual value.


About Network-Driven Drug Discovery (NDD)


e-Therapeutics' proprietary NDD platform comprises a suite of powerful computational tools to augment and interrogate the vast amount of biological information currently available in both public and private databases.


Our NDD platform is founded on sophisticated network science and employs techniques such as machine learning, artificial intelligence (AI) and state-of-the-art data analysis tools. Using our biological expertise, we can create and analyse network models of disease to identify likely proteins that could effectively be disrupted to treat the disease.


We believe that our network-driven approach more realistically reflects the true complexity of disease, with its multiple and often interconnected cellular pathways. By modelling and analysing disease networks and considering the pattern of connections between proteins, and not just single pathways, we more efficiently select the very best drug-like compounds for screening and for subsequent medicinal chemistry and pre-clinical testing. With our novel methodology, significant numbers of active molecules can be identified and tested quickly. Our approach is highly productive and consistently generates hits that have been progressed into potent, selective and novel drug molecules.


Our overall aim is to discover more efficacious drugs more effectively. By using more biologically realistic, cell and tissue-based assays we can choose and design compounds that are more likely to translate into better, more clinically efficacious drugs.

[1] Parkinson's News Today. (2018). Parkinson's Disease Statistics - Parkinson's News Today. [online] Available at: [Accessed 19 Nov. 2018].

[2] Nelson et al, The support of human genetic evidence for approved drug indications.  Nature Genetics volume 47, pages 856-860 (2015)

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