Skip to main content

Rosetta Aims to Use miRNAs as Biomarkers for Pharmaceutical R&D


By Doug Macron

Rosetta Genomics' top official this week provided details about a new corporate effort to leverage the company's microRNA expertise in an area outside of the diagnostics field, which had long been the company's core focus.

Speaking at the Roth OC Growth Stock conference held in Dana Point, Calif., Rosetta President and CEO Kenneth Berlin said that the company has set its sights on developing ways to use miRNAs as biomarkers to improve drug research and development.

Berlin also noted that Rosetta continues to work in the miRNA therapeutics space, and indicated that the company's collaboration with Regulus Therapeutics to develop a liver cancer drug has officially ended.

During the conference, Berlin noted that Rosetta's primary, near-term focus remains on miRNA-based diagnostics, and that the company is on track to potentially move five new products onto the market by the end of 2011, although it is only publicly discussing three.

The first is a next-generation version of its miRview Mets test, which is designed to determine the source of cancers of unknown primary origin. In the currently available test, "we have 25 tumor types" that can be identified, Berlin said. "The next-generation test will have close to 50."

The follow-up test will be based on a microarray platform, rather than the PCR-based one used in the existing version of miRview Mets. The change "allows us to run more microRNA biomarkers … and also helps us [sidestep] certain royalty obligations that we currently have on the PCR platform," he noted.

For the next-generation miRview Mets, "we've already identified the biomarker candidates … [and are] about to exit discovery and go into development," Berlin added. The test is expected to reach the market in the second half of this year, and will be commercialized in the US by Prometheus Laboratories pursuant to the companies' 2009 agreement (see RNAi News, 4/16/2009).

The next two diagnostics in the pipeline — the miRview Bladder test, which is designed to predict the risk of superficial bladder cancer becoming invasive, and miRview FNA, a fine needle aspirate test to classify non-small cell lung cancer as either squamous or non-squamous — are both slated to reach the market by the end of 2011, Berlin said.

Meanwhile, Rosetta has begun to explore the possibility that miRNAs could be used to improve the overall drug-development process, creating opportunities for partnerships with big pharma and biotech.

"We believe microRNAs are good biomarkers for patient stratification," Berlin said at the event. "We believe that you can use microRNAs to differentiate patient populations, both in tissue samples, as well as body fluid samples."

Currently, Rosetta is in discussions with "a number of big pharma companies about using microRNAs as biomarkers for response to therapy, to identify patients who are likely to develop side effects, as well drug salvation" by identifying sub-cohorts in the total population who may respond well to a particular therapy, he added.

For example, "we worked with one pharma company on a drug that failed phase III. They said, 'Can you help us figure out which of these patients are responders and non-responders?' We were able to successfully do that," he said. "We're hoping to take that discussion to the next level with that company."

[ pagebreak ]

Rosetta is not alone in its efforts to find new uses for its core technology. Late last year, RNAi drug kingpin Alnylam Pharmaceuticals announced that it was looking to use its technologies to improve biologics manufacturing (see RNAi News, 11/19/2009).

Called Alnylam Biotherapeutics, the effort focuses on using RNAi to “improve the quantity and quality of biologics manufacturing processes using mammalian cell culture, such as Chinese hamster ovary cells,” the company said. If it is able to do so, Alnylam expects it will be able to strike partnerships with biotech drug developers and manufacturers.

Regulus Gone?

During his presentation at the Roth OC Growth Stock meeting, Berlin noted that Rosetta continues to work on miRNA therapeutics, an effort that had somewhat fallen to the wayside for the company as it increased work on its diagnostic programs.

According to Berlin, the company has two programs: one in liver cancer and one in ovarian cancer. While the ovarian cancer project is in the "initial, preclinical phase," he said that the company has already completed proof-of-concept experiments in liver cancer.

The liver cancer program had been one of Rosetta's earliest in miRNA drugs, and in 2006 the company struck a deal with antisense shop Isis Pharmaceuticals to discover and develop miRNA-targeting drugs for the disease (see RNAi News, 2/23/2006).

In 2008, Isis' role in the partnership was transferred to Regulus Therapeutics, an miRNA drugs joint venture between Isis and Alnylam Pharmaceuticals (see RNAi News, 9/25/2008).

However, in a January filing with the US Securities and Exchange Commission, Rosetta said that the "second research period of the collaboration" with Regulus had expired and that the two companies were in the process of "negotiating … regarding the structure of this collaboration moving forward."

This week at the conference, Berlin said that Rosetta is currently "evaluating [how] to take [the liver cancer] project to the next step," noting that it could perhaps "find a partner to help … take it to the next level" of an investigational new drug application filing, suggesting that negotiations with Regulus have ended without yielding another alliance.

Officials from Rosetta did not return a request for comment.

The Scan

Possibly as Transmissible

Officials in the UK say the B.1.617.2 variant of SARS-CoV-2 may be as transmitted as easily as the B.1.1.7 variant that was identified in the UK, New Scientist reports.

Gene Therapy for SCID 'Encouraging'

The Associated Press reports that a gene therapy appears to be effective in treating severe combined immunodeficiency syndrome.

To Watch the Variants

Scientists told US lawmakers that SARS-CoV-2 variants need to be better monitored, the New York Times reports.

Nature Papers Present Nautilus Genome, Tool to Analyze Single-Cell Data, More

In Nature this week: nautilus genome gives peek into its evolution, computational tool to analyze single-cell ATAC-seq data, and more.