Industry news that matters to you.  Learn more

ExonHit Presents Promising Data on Genomic Biomarkers in Alzheimer’s Disease Clinical Trials

Reading time: 2 – 4 minutes

ExonHit Therapeutics announced today that promising data regarding the use of blood-based genomic biomarkers in Alzheimer’s disease (AD) clinical trials were disclosed in two distinct oral presentations at the Third Conference of Clinical Trials on Alzheimer’s Disease (CTAD) from November 3rd to November 5th in Toulouse, France.

The CTAD 2010 brought together current thought leaders involved in AD clinical trials to discuss in particular new results, new drug development, and methodological items (disease modifying outcomes, biomarkers, etc…).

To date, clinical trials designed to evaluate a new treatment for Alzheimer’s disease select patients based on clinical criteria, using psychometric scales, brain imaging and measurements in cerebrospinal fluid. AD being a complex disease with multiple clinical manifestations, clinical trial results are sometimes difficult to interpret due to the heterogeneity of the patient population. Similarly, treatment response in AD is mainly assessed using psychometric scales with results that also vary according to the rater’s experience or the patient’s disposition.

“Including the use of a simple blood-based molecular biomarker such as AclarusDx™ in Alzheimer’s disease trials may facilitate the recruitment of a more homogeneous Alzheimer patient population and thus contribute to reduce random noise in trial results, hence making it easier to evaluate a drug’s potential efficacy,” stated Professor Serge Gauthier, M.D. from McGill University in Montreal. “In addition, drawing blood from patients can be done almost anywhere and is much easier than collecting cerebrospinal fluid in an aged and fragile patient population.”

“Thanks to the development of comprehensive profiling technologies such as our SpliceArray™ platform, pharmacogenomic analysis can be applied in clinical trials to develop predictive or monitoring biomarkers that can be used to identify patients who will benefit from a treatment or to follow their response to that treatment. This is what we have done with EHT 0202, our Phase II compound in Alzheimer’s disease,” added Matthew Pando, PhD, Executive Vice President, Therapeutics of ExonHit Therapeutics.

The oral presentation given by Professor Gauthier was entitled “How biomarkers can help investigators and the pharmaceutical industry in AD clinical trials. From concept to application” and described the different ways of considering the use of genomic biomarkers in AD clinical trials, in particular for patient selection, stratification and recruitment. Adding genomic homogeneity to the patient selection criteria could contribute to improving the study power to detect a real effect or, equivalently, reducing the number of patients needed to detect such an effect (1).

The EHT 0202 oral presentation by Matthew Pando entitled “Identification of blood transcriptomic signatures in AD patients related to EHT 0202 treatment response and efficacy” highlighted how a drug response can be correlated to genomic expression profiles. ExonHit’s proprietary SpliceArray™ technology identified a potential blood-based transcriptomic signature specific to treatment response to EHT 0202 (2).

Both presentations underlined how the use of clinically relevant biomarkers could improve the chances of getting exploitable results when conducting AD trials. AD patients are a fragile population because of their age and of the associated pathologies; expanding the use of simple, non-invasive biomarkers in addition to current standard methods could significantly contribute to the successful development of new treatments for AD.

Source: ExonHit