Clinical validation of a bioMARKer for use in INtestinal PATHologies

Project Information

MARKinPATH

Grant agreement ID: 651065

DOI

10.3030/651065

Project closed

EC signature date 8 September 2014

Start date 1 October 2014

End date 28 February 2015

Funded under

• SOCIETAL CHALLENGES - Health, demographic change and well-being

Total cost

€ 71 429,00

EU contribution

€ 50 000,00

Coordinated by

VENTER PHARMA S.L.
Spain

Periodic Reporting for period 1 - MARKinPATH (Clinical validation of a bioMARKer for use in INtestinal PATHologies)

Reporting period: 2014-10-01 to 2015-02-28

Summary of the context and overall objectives of the project

The MARKinPATH project aims to undertake the clinical validation of Xylose, after ingestion of Gaxilose, as a Biomarker of intestinal conditions and pathologies. Currently, the measurement of Xylose after ingestion of Gaxilose is used to diagnose hypolactasia in adults. MARKinPATH will improve the diagnosis of hypolactasia, broaden the indication of pathologies to monitor and extend its use to the paediatric population. Gaxilose, marketed under the brand name LacTEST®, has proven in clinical studies to be a diagnostic assay for hypolactasia that has a superior diagnostic performance than current standard-of-care methods, hydrogen breathe test and intestinal biopsy, and is more cost-effective. It is also less invasive than the latter and avoids the discomfort that the former causes to patients.

Venter Pharma’s objectives include conducting clinical studies that will support extending the indications of LacTEST® to diagnosis of paediatric hypolactasia, monitoring of coeliac disease, and providing dietary advice to lactose intolerant patients. Additional goals are to improve the performance of the current test by validating the analysis of blood samples and providing better tools for the analysis of the metabolite in clinical laboratories. These improvements will facilitate its use as they make routine analysis, without changing any procedure or routine laboratory.

Venter Pharma has performed a feasibility study that has allowed Venter to acquire a better understanding of current and potential user needs, validated the existence of a profitable market and major unmet medical needs in the surveillance of conditions including coeliac disease diet management and paediatric hypolactasia. Our analysis has also been instrumental in identifying potential risks, bottlenecks and roadblocks. The main outcome has been a revised business plan including contingency plans and improved analytical frameworks and decision-making tools.

Venter Pharma is a highly innovative SME with more than 11 years focused on advanced research, development and commercialization of clinical solutions in the field of lactose intolerance. Venter Pharma holds title to a portfolio of intellectual property and an extensive background in these technologies. MARKinPATH represents a major advance in the growth and consolidation of Venter Pharma in the pharmaceutical market.

MARKinPATH meets EU objectives for innovation in healthcare by enhancing diagnosis and management of intestinal pathologies and conditions.

Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far

The following tasks have been conducted thus far:

1. We have acquired a better understanding of user needs through surveys collected from users (gastroenterologist and laboratory technicians). A major European-wide survey has been conducted in which 30 gastroenterologists from five countries participated in which they were questioned on how they currently manage hypolactasia and their reaction to LacTEST® and the clinical trials proposed in the MARKinPATH project.
2. We have undertaken a review of industry reports and we have also analyzed the implementation of several marketing tools such as gaps, value proposition, and market share analyses among others.
3. We have completed stability studies that support extending shelf life of the product.
4. We have completed development of enzymatic method Xylossay® that will automate determination of metabolite in clinical laboratories.
5. Designed protocols for clinical studies in paediatric, coeliac disease and diet monitoring in lactose intolerant individuals and secured conditions for manufacturing of batches for the clinical trials. This will allow Venter Pharma to initiate these studies promptly in a second phase of the MARKinPATH project.
6. We have established proof-of-concept of a biosensor method for metabolite detection. This is a fundamental milestone in the development of a point-of-care test.
7. A feasibility study has been performed employing analytical frameworks that produced a set of decision-making tools and a revised business plan as its principal outcome.

Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far)

Management of Venter Pharma expects that the outcomes of MARKinPATH will result in the advancement of major technical objectives.

• The paediatric trial will lead to the approval of LacTEST® for management of hypolactasia in children. This will result in better management of several intestinal conditions in children.
• Approval of the test for surveillance of coeliac patients will validate the use of the test in evaluating the evolution of the integrity of the intestinal mucosa. Eventually, use of the test could be extended to monitor other intestinal diseases such as Irritable Bowel Disease, Crohn´s Disease or Ulcerative Colitis. These conditions are becoming increasingly prevalent in the European population.
• Another outcome will provide gastroenterologists with a simple tool that will assist them in managing the diet of patients.
• The regulatory approval of the use of LacTEST® in blood samples and the development of a Point-of-care device for the determination of the result will facilitate the use of the test for both patients and physicians who will be able to reduce the time required for a diagnosis.
Nearly 20% of the European population has some degree of hypolactasia. In addition, coeliac disease has proven to be more prevalent than previously thought and increased awareness and better diagnosis is revealing the true extent of autoimmune diseases affecting the bowel. All these conditions are increasingly prevalent and cause major disabilities to affected patients. Healthcare providers need better tools to diagnose, monitor and manage patients of gastrointestinal conditions. Venter Pharma expects that the long-term impact of MARKinPATH will be a redefinition of the gold standard of hypolactasia diagnosis with a more cost-effective, non-invasive and better performing assay. LacTEST® will become a valuable tool for monitoring of gastrointestinal conditions. This will contribute to improved health outcomes for the European population and reduced costs for healthcare systems.

Venter Pharma is well positioned to serve the unmet needs in the field of intestinal pathologies and conditions. The MARKinPATH project will expand the current market and remove current hurdles and bottlenecks to market access.

Samples of LacTEST

Samples of enzymatic determination kit Xylossay