Genetic Engineering & Biotechnology News

JAN15 2018

Genetic Engineering & Biotechnology News (GEN) is the world's most widely read biotech publication. It provides the R&D community with critical information on the tools, technologies, and trends that drive the biotech industry.

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8 | JANUARY 15, 2018 | | Genetic Engineering & Biotechnology News Gail Dutton Lewis Carpenter, a running back for Vince Lombardi's NFL-champion Green Bay Pack- ers, had stopped playing football for decades before his relatives were confronted by obvi- ous signs that his health was in decline. He showed mood swings, memory loss, erratic behavior, and poor motor skills that are char- acteristic of chronic traumatic encephalopa- thy (CTE), a disease that can be definitively diagnosed only at autopsy. Early in 2011, postmortem test results confirmed that Car- penter had indeed suffered from CTE. The first chapter of that story was told late last year at the debut of the award-winning documentary, Requiem for a Running Back. The next installment involving this progres- sive, degenerative brain disease is being writ- ten in the scientific literature now, featuring technology developed by Quanterix. New technology has shown promise for detecting CTE and the approximately 150 protein biomarkers linked to conditions in neurology, oncology, cardiology, inflamma- tion, and infectious disease. Called Simoa™, it is a single-molecule array on a fully au- tomated digital immunoassay platform that detects single proteins or panels of pro- teins in the blood, saliva, and other biofluids and tissue. "We and our partners have invested some $150 million in developing the technology," says Kevin Hrusovsky, Quanterix executive chairman and CEO. The company expects to raise more than $64 million from the IPO it filed in December 2017. "Following the IPO, we have some very ex- citing things planned for the future of Quan- terix and precision health, including expand- ing our commercial base, improving our Si- moa technology, and supporting the launch of the new SR-X instrument, as well as expand- ing our corporate headquarters," Hrusovsky says. "We're also looking to expand into areas outside of life science research, including po- tentially lab-developed tests, in vitro diagnos- tic tests, and other markets." "The technology only becomes more mag- ical as we see its evolution through third-party publications," he continues. That magic has been reported by independent researchers in more than 129 journal articles so far. The ultimate benefits aren't just at the bedside. While it is being developed as a di- agnostic tool, Simoa is being used by leading drug developers to determine efficacy and identify autoimmunity issues. For example, Hrusovsky says, drug mak- ers are using Simoa to determine whether a drug affects such critical markers as amyloid beta, tau, and neurofilament light (NF-L). Companies also are using the technology for patient stratification and to identify second- ary markers that could be used to character- ize a drug's specificity, efficacy, and side ef- fects. Eventually, it may be used to monitor patient progress and as a health screen to identify diseases at their earliest stages, when they are most treatable. Quanterix, Repositioned "When I joined Quanterix in 2014, it was focused on becoming a diagnostic com- pany," Hrusovsky recalls. Gaining FDA approval for a diagnostic takes up to seven years, however. The company didn't want to wait. Therefore, it repositioned its technol- ogy for drug developers, offering a noninva- sive way to determine whether a drug was having the desired effect—and generating a revenue stream in the process. To make that happen, the company execut- ed a three-step plan. First, Quanterix focused on the engineering to ensure the product could deliver on its promise. Second, it secured bio- pharma validation, which meant putting the assay platform in the hands of scientists work- ing in their own, independent labs. "We've gone from having no commercial instruments placed in early 2014 to having nearly 160 in use today," he notes. Quanterix also runs customer studies on the eight instru- ments it maintains in house, to help biophar- ma clients learn whether the technology can be of value in their development programs. So far, it has run approximately 400 separate customer studies. The third step may have been the hardest. The company had to wait for results to be published in peer-reviewed journals. During that time, Hrusovsky renegotiated Quan- terix' diagnostics contract to increase the company's long-term opportunities. "It's a good growth model," Hrusovsky says. "Quanterix has grown 40–50% per year since January 2014. [In 2016], our rev- enue was nearly $18 million. By the end of the first half of 2017, we'd collected $11 mil- lion," demonstrating continued growth of more than 40% annually. Independent Research is Prized To support independent research using Simoa, Hrusovsky launched the Powering Quanterix Developing Biomarkers for Concussions Helping Solve the CTE Conundrum Corporate Profile Sitting beside the technician in this image is the SR-X Ultra-Sensitive Biomarker Detection System, an instrument developed by Quanterix. The SR-X is powered by the company's single-molecule array (Simoa) technology, which makes use of femtoliter-sized reaction wells that can isolate and detect single enzyme molecules. The SR-X is designed for multiplex detection of up to six analytes per well. A menu of over 70 Simoa assay kits will be available to measure critical biomarkers, says Quanterix, which adds that the kits will offer substantially higher sensitivity than standard immunoassay methods. BERG partnered with Massachusetts General Hospital (MGH) and Brigham and Women's Hospital (BWH) to focus on better understanding neurological disease pa- thology and to develop novel biomarker strategies for neurodegenerative disorders such as Alzheimer's disease. MGH and BWH will give the company access to a longitudinal collection of clinically annotated patient- derived biospecimens from the Harvard biomarker study. With this repository, BERG's Interrogative Biology® platform will be used to study the molecular basis and progression of Alzheimer's disease. The goal is to explore new approaches for the development of diagnostic and therapeutic products. The platform integrates patient multiomics profiling data with artificial intelligence analysis to streamline biomarker discovery, according to Niven R. Narain, BERG's president and CEO. "The neurodegenerative discovery program at BERG has made significant progress toward understanding the molecular basis of neurological diseases. We are excited to work with MGH and BWH to further this research and to collaborate on the potential develop- ment of novel treatments for neurological diseases like Alzheimer's disease," he says. "Through this agreement, we can address the significant unmet need to improve detection, diagnosis, and stratification of neurological diseases and speed earlier clinical intervention, which is especially important in the asymptomatic molecular stages through the early dementia phase of Alzheim- er's disease." "The Harvard Biomarkers Study is a powerful incubator for biomarkers discovery and precision neurology. This longitudinal biobank can speed up the biomarker test cycle from years to weeks. It comprises more than 2,500 participants, including patients at the earliest stages of cognitive decline," adds Clemens Scherzer, M.D., who co-founded the study and who heads the Neurogenomics Lab and Precision Neurology Program of BWH and Harvard Medical School. n BERG Enters Research Partnership Focused on Alzheimer's

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