ExonHit provide RNA splicing progress report
conference in London giving investors updates on company activity
over the last year. The company are coming off the back of a patent
application, which covers a number of genes that could enable
differential diagnosis of Alzheimer patients from blood.
The French drug and diagnostic discovery company could become the first to develop a diagnostic test that can accurately identify patients with Alzheimer disease (AD). This may be too late for the 12m confirmed cases of AD worldwide.
ExonHit Therapeutics is a company that have made alternative RNA splicing its field of expertise. This is the process by which a single gene can lead to several proteins.
ExonHit's proprietary gene profiling technology, Differential Analysis of Transcripts with Alternative Splicing (DATAs) is utilised to identify genes whose splice variants produce abnormal proteins, which may trigger or contribute to the development of disease.
The information derived from this core technology is utilised in the fields of drug discovery, diagnostics and microarrays.
In a presentation, Bruno Tocque, CEO of ExonHit Therapeutics, said that ageing populations in industrialised countries has given rise to new needs in diagnostics.
"Diseases such as cancer or neurodegenerative disorders would be better managed to the benefit of both patients and society if diagnostic tools were available to predict the onset or progression of the disease."
The earlier the diagnosis is made, the greater the chance of controlling the probable course of the disease and early intervention in patients who develop AD would make it possible to significantly slow its progression.
Tocque briefed analysts at the presentation that its collaboration with bioMerieux involving development of cancer diagnosis using its blood diagnostics technology was progressing well.
Under the terms of the six-year collaboration, a minimum of five cancer programmes will be covered as well as a commitment to ongoing R&D funding, milestone payments and royalties on marketed products.
"It's an attractive market considering that it takes two-four years to arrive on the market compared to ten-twelve years for a drug," said Tocque.
"In addition, revenue can be generated whilst the product is still being developed."
ExonHit also provided details of its drug pipeline, in which it is concentrating on neurodegenerative diseases and cancer. The company has advanced drug candidates into clinical trials and is evaluating several promising pre-clinical compounds.
Its notable list of drug candidates include EHT 0202, a small molecule compound being developed for multiple indications in neurodegenerative disease and ophthalmology.
Currently, a phase IIa proof-of-concept study is being planned in AD patients.
DATAS applied to brain biopsies of AD patients led to the identification of multiple cascades of cellular events that ExonHit concluded are important in neurodegenerative conditions in the brain.
It's preclinical pipeline includes EHT 0206, a proprietary new chemical entity discovered and synthesized at ExonHit that was designed to inhibit the formation of senile plaques, largely composed of aggregated beta amyloid peptides
EHT 0101 blocks the activity of a small GTPase, and exhibits anti-tumoural and anti-angiogenic properties. This compound is being tested as a treatment in chronic myeloid leukaemia (CML).
Preclinical studies are currently underway.
