Exosome Diagnostics Presents Clinical Performance Data For Exodx Lung(ALK) Liquid Biopsy Test And New Data On Detection Of EGFR Activating Mutations And T790M Resistance Mutation

ExoDx™ Lung(ALK) and ExoDx™ Lung(EGFR T790M) are only liquid biopsy tests based on exosomal RNA

Plasma-based assays overcome key challenges associated with tissue-based approaches, enabling sensitive, accurate, real-time mutation detection in non-small cell lung cancer patients

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Exosome Diagnostics, Inc., a developer of revolutionary, biofluid-based molecular diagnostics, today announced new clinical performance data for its ExoDx™ Lung(ALK) liquid biopsy test. The data demonstrated high sensitivity and specificity of the novel exosome-based liquid biopsy test in patients with non-small cell lung cancer (NSCLC) suspected to be positive for EML4-ALK translocations. Building upon a previously presented data set, the company also presented new data showing the ability of its proprietary exosomal RNA (exoRNA) plus cell-free DNA (cfDNA) platform to detect with high sensitivity EGFR activating mutations and the EGFR T790M resistance mutation in blood plasma of patients with NSCLC.

“We’re extremely pleased with these new data and the strong clinical performance of ExoDx Lung(ALK)”

ExoDx Lung(ALK) and ExoDx Lung(EGFR T790M) are the industry’s first and only liquid biopsy tests based on exosomal RNA to enable more sensitive, accurate and real-time mutation detection.

The data were presented at the following poster sessions at the 16^th World Conference on Lung Cancer, taking place September 6 – 9, 2015 in Denver, Colorado:

• “Exosomal RNA based liquid biopsy detection of EML4-ALK in plasma from NSCLC patients,” being presented today from 9:30 a.m. to 4:30 p.m. local time; and
• “EGFR activating and T790M resistance mutation in plasma exoRNA and cfDNA, detected with single-step isolation columns and targeted resequencing,” presented yesterday.

“Physicians currently rely on tissue biopsies and fine-needle aspirates, or archived samples, to detect mutations in non-small cell lung cancer, but we know these techniques are significantly constrained by surgical complications, availability of tissue, and sample heterogeneity,” said Vince O’Neill, M.D., Chief Medical Officer, Exosome Diagnostics. “Our non-invasive liquid biopsy tests for non-small cell lung cancer offer physicians and their patients a number of critical advantages to help ensure maximum detection sensitivity. These tests will help guide more informed treatment choices at baseline, and serially and longitudinally monitor patient progress and mutation status over the course of treatment.”

About ExoDx Lung(ALK) and New Data

The ExoDx Lung(ALK) liquid biopsy test, which has been validated in Exosome Diagnostics’ CLIA certified laboratory, is a laboratory developed test (LDT) designed to isolate and analyze exoRNA from a blood sample to detect EML4-ALK fusion transcripts, with the goal of informing more individualized treatment decisions for patients.

In the clinical performance data presented today, the ExoDx Lung(ALK) test demonstrated the ability to determine the variant-specific expression profile of EML4-ALK fusion transcripts in plasma samples from 24 NSCLC patients with known ALK tissue status by FISH. Specifically, the test demonstrated 88% sensitivity and 100% specificity, with the ability to differentiate between v1, v2, v3a,b,c fusion-specific transcripts. These represent the vast majority of all EML4-ALK-positive cases.

Because it has the ability to detect specific fusion transcripts of EML4-ALK, ExoDx Lung(ALK) may provide more actionable clinical information than liquid biopsies which utilize cfDNA only. In addition, today’s standard-of-care tissue-based diagnostics, such as fluorescence in situ hybridization (FISH) and immunohistochemistry (IHC), frequently do not accurately reflect the current disease state and have been associated with technical challenges of limited sensitivity and subjective interpretation. In addition, neither FISH nor IHC offers variant-specific detection of ALK rearrangements.

“We’re extremely pleased with these new data and the strong clinical performance of ExoDx Lung(ALK),” said Dr. O’Neill. “The ability to detect specific fusion transcripts of the ALK gene represents a critical advance in the detection of this mutation. With the test, we believe we will be able to give physicians the most complete molecular information they need in order to direct patients to the most targeted and appropriate available treatment or clinical trial.”

Patients with NSCLC in whom the EML4-ALK mutation is accurately detected can be matched to treatment with approved ALK kinase inhibitors. In addition, there are several other therapies in clinical development currently being evaluated to target this mutation. Different variants of EML4-ALK may have varying response rates to particular treatments.

About the EGFR T790M Data

In the study, Exosome Diagnostics used its ExoDx Solid Tumor mutation detection panel, which extracts exoRNA and captures cfDNA in a single step, to analyze 47 blood plasma samples from NSCLC patients collected at the time of clinical resistance to EGFR tyrosine kinase inhibitor (TKI) therapy. The samples were EGFR-genotyped on time-matched tissue from a repeat biopsy. The exoRNA and cfDNA were then simultaneously analyzed utilizing targeted ultra-deep sequencing (UDS) of select genes. The positive concordance of EGFR mutations in metastatic disease was 86.0 percent for the activating mutations EGFR L858R and del19, and 64.7 percent for the resistance EGFR T790M mutation. In patients with intra-thoracic disease (M0/M1a), these mutations have proven challenging to detect utilizing cfDNA alone. However, by combining exoRNA and cfDNA, Exosome Diagnostics achieved a 72.7 percent concordance for activating EGFR mutations.

The company also presented new data demonstrating that co-isolating exoRNA and cfDNA in NSCLC, as well as other cancer types, substantially increased the number of gene copies available for low abundant somatic mutation detection. The co-isolation technology approximately doubled the amount of detectable molecules in comparison to cfDNA only, translating to a potentially major advance in the development of clinically relevant liquid biopsies.

Patients with EGFR activating mutations often initially respond to treatment with tyrosine kinase inhibitors (TKIs). However, eventually, many patients’ lung cancer continues to progress because they develop new, also known as acquired, mutations, including the T790M resistance mutation. Due to these acquired mutations, many patients stop responding to TKI therapy, rendering it ineffective. There are currently several therapies in clinical development that target these acquired mutations in NSCLC.

ExoDx Lung(EGFR T790M), a laboratory-developed test (LDT) analyzed in Exosome Diagnostics’ CLIA certified laboratory, is designed to give medical oncologists a critical new tool to non-invasively and with high sensitivity detect the development of this resistance mutation over time through a simple blood draw, helping inform the most appropriate, targeted and timely treatment decisions for patients as their disease progresses.

About Exosome Diagnostics

Exosome Diagnostics is a privately held company focused on developing and commercializing revolutionary biofluid-based diagnostics to deliver personalized precision healthcare that improves lives. The company’s novel exosome-based technology platform can yield comprehensive and dynamic molecular insights to transform how cancer and other serious diseases are detected, diagnosed, treated and monitored. Visit www.exosomedx.com to learn more.

Exosome Diagnostics and ExoDx are registered and unregistered trademarks of Exosome Diagnostics, Inc.