Horizon Reference Standards Used in FDA Approval of New Oncology Companion Diagnostic Test
Horizon’s reference standards supported assay development and subsequent clinical trial
2 Jul 2017Horizon Discovery Group plc, a leader in the application of gene editing technologies, has announced the use of its reference standards in a recent successful premarket approval application (PMA), filing with the FDA for a companion diagnostic in oncology by a leading next generation sequencing company. In the development and subsequent clinical trial, Horizon’s Reference Standards were used as positive controls to support the program and to help establish the performance specifications of the assay.
Horizon is a leading provider of highly characterized genetic materials used as reference standards for the development and quality control of molecular assays. Increasingly, the company works with assay developers early in their process to support the efficient development and validation of new tests, often with the reference standards becoming part of the kit and Horizon receiving a portion of revenues, as was the case in a previously announced agreement with a global market leading Next Generation Sequencing (NGS) platform company in May 2016.
Horizon has played a major role in the identification and application of the critical biomarkers of cancer since its inception. Horizon’s genetic markers are made available to researchers via its significant catalogue of products in the form of gene edited cell lines, and to clinical labs in the form of reference standards that mimic real patient samples.
Colorectal cancer is one of the most commonly diagnosed cancers in both men and women, with an estimated 135,000 new cases expected to be diagnosed in 20171 in the United States alone. Current front-line colorectal cancer drugs exist in the forms of Erbitux and Vectibix, however not all patients respond to the therapeutics. Studies from Professor Alberto Bardelli (a Horizon co-founder) and Dr. Sabine Tejpar (a Horizon SAB member), published in the Journal of Cancer Research (March 2007 and February 2009) and the Journal of Clinical Oncology (October 2008), first made the link between K-RAS mutations and resistance to these drugs in up to 65% of colon cancer patients2. This work provided critical evidence that led to the requirement for clinicians to test patients for K-RAS mutation status prior to their prescription, not only enabling patients to obtain the right drug for their cancer, but also saving an estimated $750 million/year through avoidance of prescriptions that would provide no patient benefit.
Horizon continued to build on this work through the COLTHERES consortium, a four year €6 million funded collaboration of EU-based clinical centers and translational researchers, that identified additional biomarkers in the 30-40% of patients who are resistant to Cetuximab, a therapeutic for the treatment of advanced colorectal cancer. This work led to multiple ongoing clinical trials for combination therapies, and resulted in several technical innovations including the first practical use of liquid biopsy for diagnostics, the full impact of which is now being realized.
Dr. Darrin M Disley, Chief Executive Officer, Horizon Discovery, commented: “Due to our deep-seated experience, Horizon is a world-leader in the development of cell-based models of human disease and their application in a wide range of fields, including to support the accurate and efficient provision of critical oncology diagnostic testing. We are pleased that our Reference Standards have proven such a valuable component of this seminal clinical trial and FDA approval with one of the world’s leading players in the genomics and diagnostics market. We are currently supporting many similar trials and products and look forward to making an impact on the lives of patients through our products.”