NanoString Secures Option from The Broad Institute for Exclusive Worldwide License of Liver Cancer Gene Signature

NanoString Technologies Secures Option from The Broad Institute for Exclusive Worldwide License of Liver Cancer Gene Signature

SEATTLE, Wash. | April 17, 2013 – NanoString® Technologies, Inc., a privately held provider of life science tools for translational research and molecular diagnostic products, today announced that it has secured an option to an exclusive worldwide license for a 186-gene signature that could be used to determine the prognosis of patients diagnosed with the most common type of liver cancer, hepatocellular carcinoma (HCC), or with hepatitis C-related early-stage cirrhosis. HCC is the third leading cause of cancer mortality worldwide and Hepatitis C cirrhosis-related HCC is the most rapidly increasing cause of cancer related death in the United States.

NanoString secured the option to an exclusive worldwide license from The Broad Institute, a leading non-profit research institute of genomic medicine in Cambridge, Massachusetts, acting on behalf of the inventors’ institutions. The HCC gene signature was invented by Todd Golub, MD, Chief Scientific Officer of the Broad Institute and a group of researchers from the Dana-Farber Cancer Institute, the Icahn Sinai School of Medicine at Mount Sinai, Massachusetts General Hospital, Massachusetts Institute of Technology, The Hospital Clínic of Barcelona, Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), Institució Catalana de Recarca i Estudis Avançats (ICREA), and Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas (CIBERehd).

During the period in which the option can be exercised, NanoString plans to assess the feasibility of developing an in vitro diagnostic assay based on the HCC gene signature for use on the nCounter® Analysis System.

A paper in the New England Journal of Medicine in 2008 by Hoshida, et al, described the HCC gene signature in connection with a method for conducting gene expression analysis on RNA extracted from liver tissue adjacent to HCC tumors. Using this method, the authors discovered a 186-gene signature that identifies those HCC patients who have a poor prognosis because of a high rate of recurrence after primary treatment. This gene signature was highly correlated with survival in a training set of 82 Japanese patients and was validated in an independent set of 225 patients from the United States and Europe. A paper recently published online in Gastroenterology by Hoshida et al, demonstrated that this same 186-gene signature also identifies those patients with hepatitis C-related early-stage cirrhosis who have a poor prognosis because of their high rate of developing HCC.

Josep M. Llovet, MD, Professor of Medicine and Director of the HCC Program, Liver Diseases, Mount Sinai, Professor ICREA at IDIBAPS-Hospital Clínic, and a co-inventor of the HCC gene signature, commented: “By identifying those HCC patients who are at the greatest risk of recurrence, doctors may choose to monitor these patients more regularly or to enter them into clinical trials in the adjuvant setting to reduce the risk of HCC recurrence. The signature also identifies patients with hepatitis C-related early-stage cirrhosis at high risk of developing HCC who are good candidates for entry into surveillance programs or clinical trials of new agents in the chemo-preventive setting.”

Yujin Hoshida, M.D., Ph.D., who led the discovery of the signature while a postdoctoral fellow in Todd Golub's laboratory at the Broad Institute and is on the faculty at Mount Sinai, commented: “This platform could provide the multiplexed gene expression capabilities needed for clinical diagnostic use of this HCC gene signature, especially given the potential for a large scale global surveillance testing opportunity. We are excited to take this next step in translating the liver cancer gene signature we have applied in research into a clinical diagnostic available to patients worldwide.”

“NanoString is dedicated to helping patients and physicians by providing information that can inform major clinical decisions to improve patient care,” said Brad Gray, President and CEO of NanoString. “On the heels of launching our Prosigna breast cancer assay in the European Union and Israel, we are excited at the prospect of once again partnering with a customer of our Life Sciences business to translate their discovery into a diagnostic product.”

About Hepatocellular Carcinoma
Hepatocellular carcinoma (HCC), the most common form of liver cancer, is an increasingly prevalent clinical problem and is the third most common cause of cancer-related death globally. The incidence rates of HCC in the United States have historically been lower than in many countries. However, in recent decades, HCC age-adjusted incidence rates have doubled and primary liver cancer mortality rates have increased faster than mortality rates for any other leading cause of cancer. HCC develops from advanced fibrosis of the liver, or cirrhosis, which is estimated to affect one to two percent of the world’s population. The prognosis for patients with advanced HCC is poor, with a reported five-year survival rate of approximately 10 percent due to the high rate of recurrence after initial treatment of the primary tumor. Like many cancers, patient outcomes are better when the disease is detected and treated at an early stage.

About NanoString Technologies, Inc.
NanoString Technologies is a privately held provider of life science tools for translational research and molecular diagnostic products. The company’s nCounter® Analysis System, which has been employed in basic and translational research since it was first introduced in 2008 and cited in more than 200 peer-reviewed publications, has also now been applied to diagnostic use. The system offers a cost-effective way to easily profile the expression of hundreds of genes, miRNAs, or copy number variations, simultaneously with high sensitivity and precision. The company’s technology enables a wide variety of basic research and translational medicine applications, including biomarker discovery and validation. The nCounter-based Prosigna™ Breast Cancer Prognostic Gene Signature Assay is the first in vitro diagnostic kit to be marketed through the company’s recently formed diagnostics business.

The nCounter Analysis System is currently available for “Research Use Only” in North America. For more information, please visit www.nanostring.com.

The NanoString Technologies logo, NanoString, NanoString Technologies and nCounter are registered trademarks, and Prosigna is a trademark, of NanoString Technologies, Inc.

As co-inventors of the HCC gene signature, Drs. Llovet and Hoshida would be entitled to a portion of future royalty payments from NanoString.