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Non invasive Cell Based Liquid Biopsy Platform for Bladder Cancer

Periodic Reporting for period 1 - OneRNA4Bladder (Non invasive Cell Based Liquid Biopsy Platform for Bladder Cancer)

Reporting period: 2017-12-01 to 2019-05-31

The objective of the OneRNA4Bladder project is to save lives and reduce cost of care for bladder cancer treatment – the most expensive kind of cancer to treat due to expensive diagnostic procedures and ineffective drugs, leading to recurrence rates of up to 70%. This objective is achieved by integrating 3 technologies to completely redefine the management of bladder cancer by:

1) Substituting the use of cystoscopy with the OneRNA4Bladder for the detection of bladder cancer recurrence;
2) Validating the OneRNA™ platform’s ability to predict responders to current standard of care and the newly approved checkpoint inhibitors;
3) Developing data to support the use of the OneRNA4Bladder system as a liquid biopsy platform (e.g. measuring signals for response to treatment)

Bladder cancer is the 6th most common cancer with an estimated 357,000 new cases worldwide every year, of which 180,000 are European. The high incidence, coupled with it’s relapsing nature, results in the highest lifetime treatment costs per patient of all cancers. A significant part of this cost burden stems from routine use of highly invasive and expensive procedures, such as cystoscopy, in diagnosis and recurrence testing.

Substituting routine cystoscopy with the OneRNA4Bladder system could cut the cost of diagnosis and managing bladder cancer in half. In Europe the cost of diagnosing bladder cancer is €1.8 billion/year, thus European Healthcare payers could save €900 million/year. Perhaps even most striking, the costs associated with an untimely death due to bladder cancer (i.e. the ‘‘value’’ of life lost) approach €7 billion annually. OneRNA4Bladder is expected to significantly minimize bladder cancer burden.

The high bladder cancer recurrence rate can be interpreted as simply lack of effective treatment. Genomic Expression has developed a way to sequence RNA fast and inexpensively and link the statistically changes to drugs that could provide more durable response. This technology is called OneRNA™. In OneRNA4Bladder, OneRNA™ will be deployed as a liquid biopsy platform to effectively measure response, recurrence and provide insight into novel treatment options.
Detect: The OneRNA4Bladder project is based on a prospective fully randomized and blinded clinical study demonstrating that the OneRNA4Bladder system for urine-based detection of bladder cancer outperforms cystoscopy in terms of sensitivity and specificity and, therefore, may substitute this invasive and expensive procedure in the diagnostic work up for bladder cancer. To our knowledge no other technology has successfully met this end point. Most other non-invasive tests are adding cost to the management of the disease instead of reducing cost simply because they cannot replace cystoscopy, but can only provide a supplement.

Treat: Genomic Expression is a clear first mover in developing this kind of treatment navigation platform using RNA sequencing. mRNA is coding for proteins and proteins are targets for drugs. Today RNA is also directly targets for drugs (gene therapy) and used as therapeutics (e.g. Moderna) as well as vaccines. The OneRNA™ platform enables a paradigm shift from ONE disease, ONE marker, and ONE drug to ONE patient, MANY markers, and MULTIPLE treatment options that go beyond just using drugs. This could disrupt the current standard of care regime. OneRNA is being validated in 3 other clinical studies and typically identify 5 actionable markers in 100% of the patients translating into 5 already existing drug options. That means that OneRNA™ could theoretically improve outcome in 100% of patients and should be applied prior to treatment selection.

Optimize treatment: The selected markers in the DNA assay have been selected because they are markers for disease however they are not targets for drugs (today). The emerging liquid biopsy concepts is a much more dynamic treatment model where the doctor select a treatment and then design/select a test to monitor its effectiveness instead of waiting 3 month to do another scan. In this project, the concept “liquid biopsy” is the concept that by adding actionable markers to the DNA test and/or using OneRNA™ RNA sequencing of the captured cancer cells, we could potentially add information to the test such as which treatment would be most applicable and we could use that test to inform the physician if that choice is effective.

This initial stage of the project has focused on the following activities:
1) Getting a prototype of the Urine Cancer Cell Filtration Device (UCCFD) developed into a production version, manufactured and tested.
2) Developing a testing methods to QC the UCCFD and ensure quality among various batches.
3) Developing consensus among the Principal Investigators in the project regarding the details in the clinical protocol
4) Getting Payer and KOL support
5) Developing OneRNA4Bladder as a liquid biopsy platform enabling therapy selection and monitoring of response in cancer cells collected with the UCCFD
6) Create HIPAA/GDPR compliant Cloud platform for personalizing treatment based on DNA and RNA data
7) Getting the approvals for starting the clinical study
8) Enrolling sites in the study

The prototype of the Urine Cancer Cell Filtration Device has now been tested and cleared and the clinical trials initiated.
Substituting routine cystoscopy with the OneRNA4Bladder system could cut the cost of diagnosis and managing bladder cancer in half. In Europe the cost of diagnosing bladder cancer is €1.8 billion/year, thus European Healthcare payers could save €900 million/year.

Impact on patients
Cystoscopy is not only expensive but also a very invasive uncomfortable procedure with significant risks involved. Every follow-up cystoscopy is associated with time consume for the patient, risk of infection, pain and discomfort, and a risk of strictures. A risk of need for sick leave for a few days in patients still at work must also be expected following every follow-up cystoscopy. A non-invasive alternative to cystoscopy is therefore urgently needed. Urine tract infections happens as a result of the cystoscopy in 5% of the procedures. Because cystoscopy is so uncomfortable, not all patients comply with the care guidelines. According to studies in the USA the number of patients that doesn't comply is 18% and the consequence is a doubling in progression of the disease. The OneRNA4Bladder solution allows the patient to monitor for recurrence in the comfort of his/her home by using the UCCFD device and mailing it to a central lab that sends the result to his/her doctor. An immense improvement over having to schedule visits and undergo the cystoscopy procedure between 4 and 8 times/year.