ICE COLD-PCR-Enriched Results Identify All Mutations Detected by Standard Tissue Biopsy PCR; ICP Also Identified Additional Mutations Missed by Conventional Tissue Biopsy

Study Confirms ICE COLD-PCR’s High Sensitivity, Which Enables Use of Plasma-based Liquid Biopsies for Cancer Mutation Detection and Monitoring                                    

OMAHA, Neb. (May 16, 2016) — Transgenomic, Inc. (NASDAQ: TBIO), today announced new concordance study data confirming the superior performance of its ICE COLD-PCR (ICP) enrichment technology over standard biopsied tissue PCR results for detection of tumor mutations in cancer patients. The data show that plasma-based liquid biopsy testing using ICP is 100% concordant with an established approach that uses standard PCR. The matched patient data further indicate that the ICP-enabled liquid biopsies detected more tumor mutations than tissue biopsy testing using standard PCR. Transgenomic is in the process of submitting the study to a peer-reviewed scientific publication.

The concordance study was reviewed by Anil Vachani, MD, MS, Associate Professor of Medicine at the Hospital of the University of Pennsylvania and the Veteran’s Administration Medical Center, and a Director of Clinical Research at the University of Pennsylvania Perelman School of Medicine. Dr. Vachani’s research includes the study of methods to optimize the use of targeted cancer therapies. Dr. Vachani noted, “High sensitivity detection of genetic biomarkers from cell free DNA has the potential to provide guidance to clinical oncologists for personalized, precision cancer treatment. Liquid biopsies are a source of genetic information when there is no available tumor tissue and they make it possible to monitor treatment effectiveness and the emergence of drug resistance on an ongoing basis, thereby enabling more effective real-time treatment decisions. This can be critical for patients with metastatic disease. Based on these initial encouraging results, ICP may be a useful technology for helping to facilitate the broad use of cell free DNA liquid biopsies in cancer. I look forward to seeing the results of further confirmatory studies now underway.”

In this study, Transgenomic analyzed 22 matched samples from chemo-naïve late-stage colorectal cancer (CRC) patients. The purpose was to compare the concordance between tissue biopsy results using traditional PCR and Sanger sequencing with plasma liquid biopsy results obtained using ICE COLD-PCR and Sanger sequencing. The study also compared mutation detection results from the matched tissue/FFPE samples analyzed with Sanger sequencing and either standard PCR or ICE COLD-PCR.

The concordance between the conventional tumor biopsy results and the matched ICP-enriched plasma sample liquid biopsy results was 100%. In addition, the ICP-enriched biopsies identified seven mutations missed by the conventional PCR approach–four additional mutations were identified in the ICP-enriched plasma samples and three additional mutations were identified in tissue samples that were analyzed with ICE COLD-PCR.

Transgenomic President and CEO Paul Kinnon commented, “We are delighted with the latest results from our studies, which show 100% concordance between ICE COLD-PCR-based liquid biopsy results and tissue-based results from the same patients, as well as ICP’s superior performance in identifying tumor mutations that were missed by conventional PCR methods. This new data further validates the clinical utility of ICP as a robust method to detect and monitor actionable mutations from a liquid biopsy sample, an essential component needed for adoption of precision medicine. We expect to submit additional data to peer reviewed scientific settings in the coming months.”

Transgenomic’s ICP cancer assays exponentially amplify targeted regions of exons that have been determined to be predictive and/or prognostic for many types of cancer, including colorectal cancer, melanoma, breast cancer and non-small cell lung cancer. The 17-target mutation panel matches the set of mutation data currently used routinely in clinical settings for identifying and selecting therapeutic or clinical trial options for patients being treated for multiple cancer types. Additional mutations are being added as the science advances.

ICE COLD-PCR achieves its ultra-high sensitivity through selective amplification of mutant DNA. The result is up to a 500-fold increase in sensitivity in identifying mutations with the most precise sequence alteration detection rates available. ICP was originally developed by the laboratory of Dr. Mike Makrigiorgos at the Dana-Farber Cancer Institute, which has exclusively licensed rights to the technology to Transgenomic.

For more information about Transgenomic’s cancer assays, click here.

About Transgenomic

Transgenomic, Inc. is a global biotechnology company advancing personalized medicine in cardiology, oncology, and inherited diseases through advanced diagnostic technologies, such as its revolutionary ICE COLD-PCR™. Transgenomic also provides specialized clinical and research services to biopharmaceutical companies developing targeted therapies. Transgenomic’s diagnostic technologies are designed to improve medical diagnoses and patient outcomes.

Forward-Looking Statements

Certain statements in this press release constitute “forward-looking statements” of Transgenomic within the meaning of the Private Securities Litigation Reform Act of 1995, which involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. The known risks, uncertainties and other factors affecting these forward-looking statements are described from time to time in Transgenomic’s filings with the Securities and Exchange Commission, including in Transgenomic’s Annual Report on Form 10-K, filed with the Securities and Exchange Commission on April 14, 2016. Any change in such factors, risks and uncertainties may cause the actual results, events and performance to differ materially from those referred to in such statements. Accordingly, the Company claims the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 with respect to all statements contained in this press release. All information in this press release is as of the date of the release and Transgenomic does not undertake any duty to update this information, including any forward-looking statements, unless required by law.




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Barbara Lindheim



Transgenomic Investor Relations