Taking its name from the Rosetta Stone, which paved the way to decipher ancient Egyptian hieroglyphics, Rosetta Genomics (NASDAQ:ROSG) is unlocking the relevance of microRNA within the human genome to develop next generation diagnostic tests for personalized medicine.
“MicroRNAs are master gene regulators of almost every activity in the cell, and when something goes wrong with microRNA regulation, this often leads to disease,” president and CEO, Ken Berlin, says in an interview with BioTuesdays.com.
“By measuring microRNA expression profiles, we can understand a lot about the state of a particular disease,” he adds.
Mr. Berlin explains that there are thousands of peer-reviewed papers that reference the central role of microRNAs in cancer and, specifically, how microRNAs are aberrantly expressed in all cancers.
“We know microRNAs are tissue specific, which is why they are such sensitive biomarkers,” he adds. “By establishing the type of cancer people have and where the tumor started is how we help physicians personalize treatment and care.”
Founded in 2000, Rosetta is the first company to focus on these extremely sensitive microRNA biomarkers. “We have the industry’s most validated, accurate and prolific microRNA biomarker platform,” Mr. Berlin contends.
The company has discovered hundreds of biologically validated novel human microRNAs through a platform that combines bioinformatics and lab processes at R&D facilities in Israel. Its 6,000-square-foot commercial lab in Philadelphia is certified by the Centers for Medicare and Medicaid Services under Clinical Laboratory Improvement Amendments (CLIA) and accredited by the College of American Pathologists as part of their oversight.
Frost & Sullivan recognized Rosetta with the 2012 North American Next Generation Diagnostics Entrepreneurial Company of the Year Award.
Rosetta’s business model is to drive three sources of revenue growth, including currently marketed products, a pipeline of new products scheduled to be launched each year starting in 2015 and pharma/biotech collaborations.
Rosetta has developed six diagnostic assays, including two second-generation assays, for four assays that are generating revenue today. The company plans to launch a new assay for indeterminate thyroid nodules in 2015. Its current lead product is the Rosetta Cancer Origin Test, on which 90% of the company’s commercial effort is focused, according to Mr. Berlin. The company also has launched tests for kidney and lung cancer, as well as a test for mesothelioma.
According to Mr. Berlin, the Cancer Origin Test can identify the tumor of origin in what is referred to as Cancer of Unknown or Uncertain Primary (CUP) and other difficult-to-diagnose, or -manage, metastatic cancers.
“It can identify one of 49 cancer origins, which is important for treatment decisions, allowing a more informed and more targeted treatment strategy, as well as greater certainty around diagnoses,” he adds.
The test has a high level of accuracy, with overall specificity up to 99%, along with sensitivity for a single answer of 90%, and overall sensitivity of 85%.
“Our post-marketing validation studies with world-class CUP medical centers has demonstrated 88% to 92% concordance in actual CUP patients, compared with published data from competitors that use different approaches, of 45% to 75% concordance,” he claims.
“We believe our competitive advantage is in using microRNAs, which yield higher accuracy and lower failure rates.”
The company estimates that the market opportunity for the CUP test is $600-million a year in the U.S., based on 200,000 CUP patients and an average selling price of $3,000 per test. Medicare coverage was obtained 12 months ago and progress is being made in obtaining coverage and reimbursement from commercial payers, Mr. Berlin notes. The company has a direct sales team of 12 territory managers who are currently promoting the Cancer Origin Test.
Mr. Berlin says Rosetta hopes to have Medicare coverage for its kidney and lung cancer tests in 2015. “Once this is achieved, our commercial effort will include these tests,” he adds.
The Rosetta Kidney Cancer Test distinguishes between benign oncocytoma and malignant renal cell cancer (RCC), and it further sub-classifies RCC into clear cell, papillary and chromophobe sub-types. It utilizes 24 microRNAs on a proprietary microarray platform, with overall sensitivity of 95%.
The Rosetta Lung Cancer Test discriminates between squamous cell cancers and adenocarcinomas for therapeutic guidance, and it also identifies small cell carcinomas and carcinoids, with a sensitivity of 95%. There are 226,000 new cases of lung cancer in the U.S. each year.
Mr. Berlin says the Rosetta Mesothelioma Test, which distinguishes mesothelioma from carcinomas in the lung or pleura, has a sensitivity approaching 100% and is currently being marketed and used in connection with asbestos exposure litigation.
In June, Barrington Research analyst, Charley Jones, initiated coverage of Rosetta with an “outperform” rating and price target of $7. He raised his target to $10 later in June after the company received a U.S. patent for microRNA miR-205 for the prognosis and treatment of prostate cancer, a previously unpublished area of interest, he said.
Mr. Berlin says the new biomarker could potentially be used for predicting the aggressiveness of prostate tumors and for helping to determine the most appropriate course of treatment. The biomarker, which is over-expressed in primary prostate tumors, could also be potentially used as a therapeutic target for prostate cancer.
Mr. Jones described the patent announcement as a “big deal and is likely to have a meaningful impact on Rosetta’s value.” Shares of Rosetta closed at $3.72 on Friday.
Mr. Berlin points out that microRNAs also play a central role in diseases other than cancer. Several weeks ago, Rosetta teamed up with a global pharmaceutical company in the area of Alzheimer’s disease (AD) diagnostics to provide its microRNA profiling to assist in the development of a microRNA-based diagnostic for the early detection of AD.
The goal is to develop a diagnostic that can identify AD patients earlier so treatment can begin earlier. “Currently, we are diagnosing AD patients too late in their disease to have a meaningful impact with treatments on the market, as well as those in development,” he adds.
In addition to neurological disorders, he notes that microRNAs play a central role in other diseases, such as cardiovascular diseases, immune response conditions, infectious diseases, and diabetes and obesity.
At the beginning of 2014, Rosetta signed an accord with a global biopharmaceutical company to provide microRNA profiling to assist in the development of new therapeutics. It also is collaborating with Marina Biotech on neuromuscular disorders and dystrophies in order to find predictive biomarkers and drug targets.
Rosetta is planning to launch its thyroid neoplasia test before the end of 2015 to diagnose indeterminate fine needle aspirates (FNAs) from the thyroid, which may reduce unnecessary surgeries and health care costs. “By measuring microRNA expression levels, we can differentiate malignant nodules from benign nodules in the thyroid,” Mr. Berlin says.
The National Comprehensive Cancer Guidelines encourage physicians to use molecular testing instead of diagnostic surgery when a thyroid FNA result is indeterminate.
Mr. Berlin points out that there are 450,000 thyroid FNAs performed in the U.S. annually, with up to 30% indeterminate, resulting in a more than $400-million market opportunity.
He says that, in the coming months, Rosetta will select a new cancer indication for its next diagnostic, likely to be launched in 2016. Among the oncology indications under consideration are bladder, ovarian and breast cancers.
In addition to AD, Rosetta also is working on how microRNAs can be applied to address chronic rejection of transplanted kidneys and heart failure—specifically early detection and risk stratification immediately after myocardial infarction.
“At the end of the day, we have more than 50 publications in peer-reviewed journals of studies conducted with our platform, which speaks to the robust science behind our launched products, new products in development and collaborations with Big Pharma,” Mr. Berlin says.