Luminous double helix strands of abstract DNA, 3D render.
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UK drug discovery company C4X Discovery Holdings Plc. has announced the launch of PatientSeek, its precision medicine platform to identify patients most likely to benefit from a medication based on their genetics.

The launch follows its first validation for patient stratification using results from a failed Phase 3 clinical trial into Parkinson’s disease.

The retrospective analysis, conducted by Australia’s Garvan Institute of Medical Research in Sydney, identified a subgroup of patients that responded to treatment despite the trial’s failure to reach its clinical endpoint overall.

Findings from the study will be submitted to a peer-reviewed journal in due course, C4XD said.

“We believe the PatientSeek platform has huge potential to unlock precision medicine approaches for many different diseases,” C4XD senior vice president of drug discovery Clare Murray told Inside Precision Medicine.

She said the company was particularly excited about applying PatientSeek to immunoinflammatory diseases, which it is exploring through the analysis of datasets relating to inflammatory bowel disease and rheumatoid arthritis.

“The PatientSeek platform has the potential to identify biomarkers which could inform clinical trial design and enable the development of companion diagnostics,” Dr Murray explained.

“These could be highly complementary to our existing small molecule drug discovery pipeline and deliver greater value to eventual partners for our programmes.”

PatientSeek is a result of a collaboration with the Garvan Institute and utilizes C4XD’s Taxonomy3 platform. This novel platform performs highly sensitive in silico mining and uses proprietary algorithms that analyze complex genetic datasets to discover and validate potential drug targets.

Through its application to publicly available DNA databases, the technology can identify previously unknown genetic linkages and interactions between genes and biological pathways for a variety of diseases.

The approach allows the discovery of targets that cause underlying disease, rather than those merely associated with its symptoms and therefore provides a better starting point for drug discovery as well as patient stratification and biomarker identification.

The multivariate approach offers higher sensitivity and greater potential to identify disease targets than standard univariate approaches.

Taxonomy3 had historically been used for target identification, but this new application demonstrates that the company’s mathematical approach can be used to distinguish patients for treatment based on their genetic makeup.

Garvan research partner and Australian Parkinson’s Mission research director Antony Cooper said the findings reaffirmed the importance of genetics in Parkinson’s disease.

“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,” he said in a press statement.

UK charity Cure Parkinson’s director of clinical development Richard Wyse, who introduced C4XD to the Garvan Institute, added: “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.“

Earlier this year, C4XD announced positive, preclinical findings for the anti-cancer activity of its investigational inhibitor of MALT-1, a key regulator of B-cell and T-cell receptor signaling, indicating its potential for hematological cancers.

And in November, the company signed a global licensing deal potentially worth up to US$402 million with AstraZeneca for the C4XD NRF2 activator program.

AstraZeneca will develop and commercialize oral treatments for respiratory and inflammatory diseases, with a lead focus on chronic obstructive pulmonary disease.

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