ExonHit Therapeutics claimed that EHT 0202 has a novel mechanism of action when compared to existing Alzheimer’s disease therapeutics and stimulates the a-secretase pathway, thus enhancing the production of the procognitive and neuroprotective sAPPa fragment of APP (Amyloid Precursor Protein).
The EHT 0202 poster presentation reports preliminary results from a study designed to determine whether patients that responded best to EHT 0202, during the recently completed Phase IIa study, have a distinct blood-based gene expression profile.
The data showed that ExonHit’s Genome-Wide SpliceArray expression profiling technology is able to clearly separate clinical patient subpopulations. Patients whose condition improved while on EHT 0202 have a different gene expression profile from those whose condition declined, and these gene expression profile differences are specific to EHT 0202. These data raise the possibility of identifying expression profiles that could allow the stratification of patients who will benefit from EHT 0202 therapy from those who won’t prior to study initiation .
ExonHit Therapeutics said that applying this approach to other clinical development programs could significantly increase the likelihood of successfully achieving primary endpoints and could help identify the most relevant patient populations for a given therapeutic product.
Additionally, a poster on AclarusDx, ExonHit’s first research-use-only molecular test for AD launched in December 2009, entitled ‘Identification of patients with Alzheimer’s disease using molecular signatures derived from splice variant expression profiles from peripheral blood” was also presented.
Loic Maurel, president of the management board at ExonHit Therapeutics, said: “Identifying patients that will respond to a treatment before its initiation is an approach that will be increasingly used in the coming years. ExonHit has the technology necessary to developing biomarkers that will be the cornerstone of tomorrow’s personalized medicine.”