E!10126 Precision Cancer Medicine Pipeline and Services

Precision (or personalized) medicine is proposed to transition health care from the presently dominant "one-size-fits-all" treatments of cancer, which for many are completely inefficient, to targeted treatments tailored to the individual patient. Current cancer treatment is largely based on the "average patient" and currently used drugs in oncology are ineffective for a large proportion of patients - 75% or more - according to the US Food and Drug Administration (FDA). Although tremendous progress has been made in our understanding of cancer, for many common cancer types and especially for patients with cancer that has spread, progress has been very limited. Moreover, personalized medicine is not universally available, even in many highly developed countries. This is due to differing national policies regarding approval of new technologies and treatments, but also simply because of a lack of awareness and knowledge about personalized medicine. Personalized cancer treatment approaches may considerably improve both efficacy and reduce unwanted side effects. The main objective of the PCMPS project has been to develop a complete personalized cancer medicine pipeline, from Next Generation Sequencing (NGS) to clinical treatment support for cancer treatment. Focus has been on development, adaption, implementation, and evaluation of sequencing and health information technology. The project has built upon existing technologies from both partners PubGene AS and BGI-Europe. This has included routines for setting up and running the pipeline, bioinformatics tools/modules, pan-cancer panel and testing and validation of the pipeline. As part of this project we have together with the University Hospital in Bergen developed a tool where the user can comment, classify, validate results from different analysis and share this information with other users in custom defined studies. This tool has been used to evaluate the quality of our analysis and algorithms. We have also performed testing of the complete personalized cancer medicine pipeline with leading hospitals and cancer centers in the EU, and we have been able to develop and tune the product and begun to generate independent third-party validation. To evaluate and benchmark the cancer knowledge base and algorithms we have cross referenced our output with multiple modules of an analysis pipeline set up at the Radium Hospital (Oslo University Hospital). This confirms that our knowledge base and algorithms perform well and provide correct information, both with respect to mutation prediction and clinical relevance. To further strengthen the clinical validation we will analyse cohort of 20 patients with colorectal cancer with liver metastases. We have used available clinical information regarding treatment, quality of life, progression and survival in the benchmarking of the PCMPS pipeline. For the evaluation of the integrated solution we have used a medical expert panel consisting of one MD, one oncologist and three professors in molecular biology. This expert panel will also be involved in the extended evaluation of the overall solution using the abovementioned colorectal data set. Within 2019 the PCMPS product will be available on the personalized cancer treatment market. We will be able to offer products, ranging from "service for fee" to customized integration products covering the complete or individual parts of the PCMPS pipeline. Additionally, the new bioinformatics tools and the pan cancer panel will have significant spin off potential for the partners PubGene and BGI-Europe. Following the product launch, we will explore additional marketing strategies and seek opportunities for expanding the product range.

Precision medicine is proposed to transition health care from the presently dominant "one-size-fits-all" treatments of cancer to targeted treatments tailored to the individual patient. During this project we have performed beta-testing of the complete pipeline with leading hospitals and cancer centers in the EU. We have developed and tuned the product offering and begun to generate independent third-party validation. Within 2019 the final PCMPS Pipeline will be available on the personalized cancer treatment market. The partners will be able to offer a product range, ranging from "service for fee" to customized integration products covering the complete or parts of the PCMPS pipeline. The new bioinformatics tools and the cancer panels will have significant spin off potential for PubGene and BGI-Europe respectively. Following the product launch, we will explore additional marketing strategies and seek opportunities for expanding the product range.

In Europe, nearly 3.5 million people are newly diagnosed cancer cases, and more than 1.75 million deaths caused by cancer. In recent years, precision oncology has been driven forward by several key factors, like (1) the maturity of genomic sequencing technology; (2) the improved capability of bioinformatics tools; (3) increased patient awareness; (4) physician's willingness to incorporate new technology into clinical care; (5) positive changes in regulations and legislations; (6) reimbursement coverage towards genetic testing and targeted therapies. The enormous need and these positive developments bring burgeoning market opportunities in precision oncology. Effective clinical decision support has shown to be critical in improving health care quality. In this project our main goal is to develop a complete pipeline from Next Generation Sequencing (NGS) to clinical treatment support for cancer treatment. This will be achieved through the development, adaption, implementation, and evaluation of sequencing and health information technology. This pipeline will be able to sequence and analyze the tumor mutation profiles of each individual patient, find the ?actionable? mutations, and highlight the targeted drugs available either in the market or in clinical trials. This pipeline will provide the necessary tools for offering a complete service towards the personalized cancer medicine market. Successful implementation of this project will improve clinical decision making in precision cancer medicine, and therefore benefit for cancer patients and cancer care professionals. he PCMPS pipeline will be offered primarily as a premium service for fee. We also see the opportunities for offering individual analysis modules like the Clinical Trial Analysis module and the Pan-Cancer Gene Panel as separate products. For customer that have pre-established tumor care pipelines in place, we will offer integration services combined with site licenses