Rosetta Genomics Reports First Ever In-Vivo Efficacy Data for a Systemic MicroRNA Therapeutic for Liver Cancer

Rosetta Genomics reported today the first ever in-vivo efficacy data for a systemic microRNA-based cancer therapeutic

PHILADELPHIA, PA, USA and REHOVOT Israel | February 12, 2009 | Rosetta Genomics Ltd. (Nasdaq: ROSG) reported today the first ever in-vivo efficacy data for a systemic microRNA-based cancer therapeutic. The animal-model study demonstrated a marked suppression of a microRNA that was found to be over expressed in human hepato-cellular carcinoma (HCC), and a significant two-fold reduction of tumor mass. These data are being presented at the Keystone Symposium, Therapeutic Modulation of RNA Using Oligonucleotides, at Lake Louise, Alberta Canada, on February 12th.

Liver cancer is the third cause of cancer death globally, affecting over 19,000 patients annually in the US alone. Prognosis is extremely grim, with nine out of ten patients dying within 5 years. Sorafenib (Nexavar(R)), the only oral anti-cancer drug currently approved for systemic therapy for HCC, extends survival by approximately 3 months.[1]

Rosetta's scientists first identified miR-191, a microRNA that is abnormally over-expressed in human liver cancer cells, and which when inhibited effectively reduces cell proliferation and promotes cell-death of these liver tumor cells. The scientists then used an in-vivo model called Orthotopic Xenograft, in which an artificial tumor fragment derived from a human liver cancer cell line is transplanted into livers of mice, in order to evaluate the efficacy of systemic microRNA-based treatment in these mice. Chemically modified antisense oligos (ASOs) were delivered systemically, targeting the over-expressed microRNA. Using this animal model, the scientists have shown that the targeted microRNA was markedly decreased in mice livers and was practically undetectable within the tumor itself, and that the tumor mass was significantly reduced two-fold, within 40 days of treatment.

Interestingly, the researchers showed that Dioxin, one of the most toxic substance ever identified and a known liver cancer carcinogen,[2] markedly increases the expression of the targeted microRNA.

"We view this study as a landmark event in the development of microRNA based cancer therapeutics" said Amir Avniel, President and CEO of Rosetta Genomics. "While Rosetta's clear focus is on developing diagnostics, this collaborative project with Regulus is an example of how we intend to leverage the tremendous potential that microRNA holds in therapeutics and other domains. Every one of our diagnostic biomarkers is also a potential drug target, and we intend to form strategic alliances with suitable biopharma partners to leverage and commercialize these opportunities."

The data presented at the conference is now available for downloading on the company's website at:

The study is supported by a grant from the BIRD foundation.

[1] Llovet et al., Sorafenib improves survival in advanced Hepatocellular Carcinoma (HCC): Results of a Phase III randomized placebo-controlled Trial (SHARP trial), Journal of Clinical Oncology, 2007 ASCO Annual Meeting Proceedings Part I. Vol 25, No. 18S (June 20 Supplement), 2007: LBA1
[2] Health Risks from Dioxin and Related Compounds, National Research Council, National Academies Press, 2006

Nexavar(R) (sorafenib) is a registered trademark of Bayer HealthCare Pharmaceuticals Inc.

About Rosetta Genomics

Rosetta Genomics (Nasdaq: ROSG) is a leading developer of microRNA-based molecular diagnostics. Founded in 2000, the company's integrative research platform combining bioinformatics and state-of-the-art laboratory processes has led to the discovery of hundreds of biologically validated novel human microRNAs. Building on its strong IP position and proprietary platform technologies, Rosetta Genomics is working on the application of these technologies in the development of a full range of microRNA-based diagnostic tools. The company's first three microRNA-based tests, miRview(TM) squamous, miRview(TM) mets, and miRview(TM) meso, are commercially available through its Philadelphia-based CLIA-certified lab. Rosetta Genomics is the 2008 winner of Wall Street Journal's Technology Innovation Awards in the medical/biotech category.

About MicroRNA

MicroRNAs (miRNAs) are recently discovered, naturally occurring, small RNAs that act as master regulators and have the potential to form the basis for a new class of diagnostics and therapeutics. Since many diseases are caused by the abnormal activity of proteins, the ability to selectively regulate protein activity through microRNAs could provide the means to treat a wide range of human diseases. In addition, microRNAs have been shown to have different expression in various pathological conditions. As a result, these differences may provide for a novel diagnostic and therapeutic strategy for many diseases.

SOURCE Rosetta Genomics Ltd

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