Peter S. Lu, M.D.
Company Background
Arbor Vita Corporation (AVC) was founded in 1998, based on technology initiated at Howard Hughes Medical Institute (HHMI) at Stanford University. AVC focuses on the development of novel diagnostics and therapeutics using the proprietary PDZ platform to improve health care worldwide. AVC was the first to obtain FDA clearance for a rapid test for H5N1 Avian Flu in 2009, and it is now moving into commercialization of the HPV E6 Test. AVC also maintains a program to develop a treatment for HPV-induced cancers. Currently the company has 30 employees and several consultants.
Technology Overview
PDZ protein domains play important regulatory roles in the cell. The AVC PDZ proteome platform combines informatics and chemistry to identify and validate important biological targets for development. Using its PDZ proteomic technology, AVC has developed one diagnostic product (H5N1 rapid test) 510(k) cleared by the FDA, a suite of diagnostics for HPV malignancy (one of which just completed a successful clinical trial), and a companion therapeutic for HPV cancer currently in pre-clinical development. The HPV E6 oncoprotein test platform addresses HPV neoplasia like cervical cancer, anal cancer, and oral cancers. In the clinical trial conducted by PATH (Seattle) and CICAMS (Chinese Academy of Medical Sciences, Beijing) the AVC E6 test achieved analytical sensitivity of less than 1,000 transformed cells, 99 percent specificity, and a positive predictive value (PPV) of 33 percent in a general screening population. In contrast current screening technologies (like Pap and HPV DNA/RNA) achieved lower specificity and a 5 percent PPV. The AVC HPV E6 rapid test that completed the clinical trial is suited for use in near point-of-care settings in developing countries and also physician’s offices.
Market Potential
The current cervical cancer screening market is more than $1 billion in the U.S. Current screening technologies lack specificity which leads to over treatment and more testing. The AVC E6 test addresses this concern with a test that is 99 percent specific. In addition, the improved analytical sensitivity permits earlier detection of cervical cancer (and potentially anal and head-and-neck cancers) that is crucial for survival and to decrease morbidity associated with surviving cancer. AVC plans to commercialize the HPV E6 Test within the next two years.
Competitive Advantage
Current cervical cancer screening (Pap and HPV DNA/RNA tests) suffers from low specificity, best captured by the term PPV. The 5 percent PPV typically seen in screening technologies means only 5 percent of the positives have disease and 95 percent do not. Low PPV leads to more testing, higher cost and greater morbidity. The HPV E6 Test achieves a 33 percent PPV for high-grade disease in the same general screening population. The improved PPV means better screening efficiencies, lower costs, and better outcomes for the patient.
Financial Overview
The HPV E6 oncoprotein detection platform is supported by SBIR grants and private investments. To accelerate the commercialization of this new technology, AVC will require an infusion of $10 to $20 million in new investments.
Intellectual Property
Most IP associated with the technology was developed by AVC and is patent protected in the U.S., Europe, and Asia. Ancillary patents to enable commercialization of this product have been licensed.
Commercialization Strategy
AVC’s HPV E6 oncoprotein detection platform can be used in point-of-care settings, high-throughput reference labs, and as augmentation to traditional pathology lab assays. The initial focus will be non-U.S. countries, with an emerging middle class, that have not invested in the health care infrastructure that exists in the U.S. and Europe. This approach favors new technologies able to bypass current practices to provide improved care at a lower cost. AVC also plans to introduce its technology in the U.S. initially through specialty labs, and then to established medical communities.
Pipeline Products
AVS’s initial focus is to commercialize the HPV E6 Test, followed by adapting the E6 platform to fit various environments. AVC also has a drug in pre-clinical development to address HPV-related cancer treatment and prevention.
Management Team
Peter Lu, M.D., Founder and CEO, trained at Caltech, Stanford, and University of Washington with a background in medicine, molecular biology, and oncology.
Johannes Schweizer, Ph.D., VP of Research and Development, trained at Institute Pasteur and Stanford with a background in genetics, molecular biology.
Olga Petrauskene, Ph.D., Director of Commercialization, formerly at ABI.
Charles Trimble, Chairman, founded Trimble Navigation and is a successful entrepreneur who has brought new technology into practical use, such as GPS.
Arbor Vita Corporation
Founder & CEO
Davide Marini, Ph.D.
Company Background
Firefly is introducing a universal technology platform for biomarker detection with a broad range of applications in life sciences, agriculture, veterinary medicine, and human diagnostics. Firefly BioWorks is a spin-out of the Chemical Engineering Department at the Massachusetts Institute of Technology (MIT), where the founders developed a novel method for high-throughput production of complex microparticles. The company began operations in 2010 and grew to seven full-time employees through a combination of SBIR awards and angel funding. The company is focused on developing multiplexed assays for biomarker detection that scale from discovery to clinical diagnostics. The company has adopted a lean startup approach. The first minimum viable product is being launched and operations will be scaled according to demand.
Technology Overview
Firefly develops and manufactures next-generation microparticles for biomolecule detection. The company’s first product, a kit for detecting microRNA aimed at the research market, has just been launched commercially. Firefly’s core technology, Optical Liquid Stamping, was developed by combining photolithography (typically used in microchip production) with microfluidics. This method allows fabrication of microparticles with virtually any shape, chemistry, and biofunctionality. Using Optical Liquid Stamping, Firefly developed a barcoded particle architecture that enables multiplexed biomarker detection on standard laboratory instrumentation. Through the support of the NCI SBIR Program, Firefly developed a particle-based assay for high-throughput profiling of microRNAs, a class of molecules with enormous potential for early diagnosis of cancer.
Market Potential
Firefly operates in the global market for biotechnology tools, a market that generates approximately $70 billion in worldwide sales. Firefly developed its first product for microRNA profiling, a market currently estimated at approximately $100 million and expected to grow very rapidly. Several market surveys indicate that bead-based assays for high-throughput/mid-multiplexing profiling are expected to address a critical need in the industry and grow at the fastest rate, especially in the area of microRNA.
Competitive Advantage
The Firefly platform enables detection of clinically relevant biomolecules with an unprecedented combination of performance, flexibility, throughput, and cost. Additionally, the assay developed by Firefly allows direct detection of miRNA in clinical samples without purification. This eliminates protocol discrepancies in RNA purification and will likely lead to more reliable diagnostics.
Beyond microRNA, Firefly’s platform has been used for the detection of disease-related proteins, mRNAs, and genomic DNA. The technology naturally lends itself to simple bedside or handheld devices that can be used in routine screens for early disease detection or point-of-care diagnostics.
Financial Overview
Initial funding was provided by two Phase I SBIR awards, for a total of about $500,000, followed by a seed round of $1 million from angel investors. In August 2011, Firefly was awarded a $2 million NCI SBIR Phase II award that catalyzed a $2 million second round of angel funding used for commercial development in the research market. Firefly seeks an industry partner to adopt the company’s technology and estimates additional funding in the $10 to $20 million range is required to expand its diagnostics capabilities.
Intellectual Property
Firefly has obtained an exclusive license from MIT for use of its technology in any research or clinical diagnostics application. The company’s current IP portfolio comprises four issued patents and 16 pending applications, covering the entire value chain of Firefly products from particle fabrication, encoding and bio-functionalization, to custom microRNA assays and readout in standard instrumentation.
Commercialization Strategy
Firefly’s long-term goal is to become a trusted provider of reliable and cost-effective solutions for clinical diagnostics. The company plans to first establish a presence in the research market and eventually enter the diagnostics field. Firefly intends to grow the company in three phases:
- Selling initial products to leading academic laboratories.
- Partnering with flow cytometry manufacturers for co-marketing of products.
- Expanding the product offering to include diagnostic applications by partnering with content developers in the cancer space.
Pipeline Products
Firefly’s first product is a custom 25-plex microRNA profiling kit that can be used on standard benchtop cytometers. The next generation of products will offer expanded multiplexing, a larger set of supported cytometers, and sensitivity that rivals polymerase chain reaction (PCR). Once the company has proven the technology in the field of microRNA, it will enter the protein market, with a set of companion products with relevance in both research and diagnostics.
Management Team
Firefly has built a team of executives and business advisors with deep expertise in molecular assays, microfluidics, materials, computer science, flow cytometery, and protein science, with roots in both start-ups and large life-science corporations. The team includes:
Operational Team:
Davide M. Marini, Ph.D., Co-founder, Chief Executive Officer
Daniel C. Pregibon, Ph.D., Co-founder, Chief Technology Officer
Andreas Windemuth, Ph.D., Chief Information Officer
Isaac B. Stoner, Vice President of Product Development
Andrea K. Bryan, Ph.D., Director of Engineering
Directors:
Roger Dowd and Rick Ryan
Business Advisory Board:
Joseph Gentile, Martin Madaus, Ph.D., Paul B. Manning, Ian Ratcliffe, and Michael P. Rubin, M.D., Ph.D.
Scientific Advisory Board:
David P. Bartel, Ph.D., and Patrick S. Doyle, Ph.D.
Firefly BioWorks
Co-Founder and CEO
Ray Martino
Company Background
Metabolomx is commercializing a non-invasive, rapid, and inexpensive breath test for cancer with the potential to revolutionize cancer diagnosis.
Technology Overview
Metabolomx has developed technology enabling the identification of lung cancer from its metabolomic fingerprint in exhaled breath, currently in a second round of efficacy trials at Cleveland Clinic and other distinguished clinical centers. At the heart of the system is a high dimensional array of diversely reactive chemical indicators that change color upon interaction with volatile species or mixtures.
Using the first generation of the Metabolomx sensor a recent Cleveland Clinic study (Mazzone et al., 2012, Journal of Thoracic Oncology) reported 85 percent specificity and sensitivity for lung cancer detection, comparable to a CT scan, the present gold standard. In the first quarter of 2012, the Cleveland Clinic and National Jewish Health Center in Denver (led by Dr. Jim Jett, Editor-in-Chief of JTO) began testing a Metabolomx sensor over 100 times more sensitive than the version used in the Journal study. The Mayo Clinic, led by Dr. David Midthun, is scheduled to begin testing in the second quarter of this year. Metabolomx’s technology will be in the clinic at the country’s three top-ranked lung care centers (U.S. News and World Report), reflecting the promise of this new paradigm for lung cancer assessment.
Market Potential
First indication: A companion diagnostic to CT scan
Metabolomx’s first FDA indication will be as a diagnostic adjunct to an inconclusive CT scan. Results from the 53,000 patient National Lung Screening Trial (NSLT) indicate that a CT scan screening of high-risk patients extends life expectancy over 20 percent (NEJM, June 2011). The study has already prompted a powerful shift toward widespread use of CT, with Wellpoint recently announcing it will cover CT screening of the enormous at-risk population (more than 55 years of age, more than 30 pack years) identified in the study. However, CT generates a large number of false positives (the NLST found 27% of the high-risk group had a positive CT). Millions of people who have a positive CT are faced with the difficult decision of whether to monitor with follow-up CT or submit to an invasive and expensive biopsy, magnifying the need for a diagnostic adjunct to inconclusive CT. The estimated market size of this first indication is 10 million units per year in the U.S., with revenue of $1 billion.
Pre-screen of high-risk population to triage who should receive CT
The financial costs of CT, risks involved with radiation exposure, and the enormous size of the at-risk population defined in the NLST calls for a non-invasive, inexpensive initial test to better triage who should be screened by CT. Metabolomx expects to gather data on more than 1,000 patients to confirm that the breath test is a candidate pre-CT screen of the high-risk population. The estimated market size to pre-screen the high-risk population is 25 million units per year in U.S., with revenue of $2 billion.
Monitor efficacy of chemotherapy
Metabolomx is gathering data to confirm that the breath signature declines when chemotherapy is effective in curtailing tumor growth. The estimated potential market to monitor treatment is 15 million units per year in the U.S., with revenue of $1.5 billion.
Correlation between metabolomic breath fingerprint and effective treatment
Metabolomx is compiling an unmatched database allowing the post-hoc assessment of whether there is a predictive correlation between the metabolomic breath fingerprint and the efficacy of a particular treatment. With each treatment often tailored to intervene in a particular metabolic pathway, Metabolomx predicts that the metabolomic fingerprint in exhaled breath will allow individualized selection of treatment.
Competitive Advantage
The technology is a five minute, inexpensive (less than $100), non-invasive test for lung cancer and other cancers, based on the VOC profile present in the bloodstream and picked up in exhaled breath. Each cancer has its own “smell,” and canines have been documented more than a 95 percent accurate in detection across stages, signaling the extraordinary promise of this new paradigm.
Financial Overview
Metabolomx has received several government development contracts, including a NCI Phase I/Phase II Fastrack award of $1.135 million. The company seeks a $5 million Series A round to reach clinical quantification of the level of accuracy of the second generation lung cancer detection system (building on the 85 percent accuracy of the first generation system) and submission to the FDA. Financial information on Metabolomx is available to interested parties under NDA.
Intellectual Property
The company holds exclusive, worldwide rights to an estate of fundamental enabling technology, including both the sensor itself and application IP. Additional private information is available to interested parties under NDA.
Commercialization Strategy
The Metabolomx breath analysis instrument will be sold or leased to the same hospitals and testing centers that use CT. The bulk of the company revenue is from the disposable sensor arrays and the balance from equipment and support. The company’s first FDA indication will be as a diagnostic adjunct to indeterminate CT scan.
Pipeline Products
The first product is a breath test for lung cancer, but tests for other cancers will follow.
Management Team
Paul Rhodes, Ph.D., CEO leads a diverse group of technology companies, which have received $13 million in DARPA contracts to develop next-generation sensory systems.
Ray Martino, COO, who, during a 20-year career at Symbol Technologies was General Manager of its mobile business ($500 million in division sales) and then CTO of Symbol prior to its acquisition by Motorola for $3.9 billion in 2007.
Sung Lim, Ph.D., Chief Scientist, is a co-inventor of the company’s proprietary nanoporous pigment array optical sensing technology, along with Metabolomx’ co-founder, University of Illinois Professor Ken Suslick.
A Clinical Advisory Board has been formed, and includes deep practical expertise in FDA approval processes.
Metabolomx
COO
Thomas Neumann
Company Background
Nortis is dedicated to developing a new generation of in vitro systems that are based on small segments of tissues and organs grown from human-derived cells in disposable, chip-like devices. Such human “body-on-a-chip” systems represent urgently awaited alternatives to laboratory animals and are expected to become the gold standard for the testing of drugs, vaccines, toxic compounds, cosmetics, and warfare countermeasures.
The Nortis project started in 2005 as a division within VisionGate, a bio-imaging company. In 2011, Nortis, Inc., was officially spun-off and subsequently moved into its new facilities at a biotech incubator on the University of Washington campus. As of January 2012, the Nortis team consists of 10 full- and part-time employees and consultants.
Technology Overview
Nortis is developing products that will overcome a crucial bottleneck in the development of therapeutic drugs and vaccines. Due to the lack of reliable in vitro assays, drug development depends heavily on animal testing for predicting efficacy, safety, and pharmacokinetics in humans. This is problematic for several reasons. Testing in animals is expensive and fraught with ethical concerns. Most importantly, the results obtained with animals often don’t translate to humans.
Nortis has pioneered proprietary techniques for the in vitro creation of human tissues and organs in disposable chip-like devices. These organ microenvironments are designed as disposable modules, to be used as single assays or integrated in fluidic circuits that connect several different organ modules in various configurations as needed for the testing of drug/vaccine efficacy, toxicity, and pharmacokinetics. Nortis established proof-of-principle for their technology through a completed SBIR Phase I grant. Nortis anticipates their first products to enter the market within two to three years.
Market Potential
Market analysis for the first two Nortis assays, a model of the blood-brain-barrier assay and an angiogenesis assay, were prepared by Foresight Science & Technology, a leading technology commercialization and transfer firm. The combined yearly revenue potential for the two assays was predicted to exceed $400 million. The market potential of the other tissue/organ assays has not yet been evaluated, but is expected to have the same magnitude.
Competitive Advantage
Nortis’ tissue and organ models differ significantly from other body-on-a-chip approaches. The competitive advantage of Nortis’ technology arises from the integration of living vasculature, which can be directly perfused to mimic blood flow. This unique feature allows for the study of vascular growth and function in real time, reducing the need for expensive and laborious animal testing. Vasculature is a structural and functional key element of almost every tissue. Thus, Nortis’ assays are poised to produce test results that better replicate in vivo conditions and predict clinical outcome. Notably, the Nortis system is ideally suited for administering test compounds either through the vessel lumen or through the surrounding microenvironment. This is especially important for the testing of drugs, toxins, and vaccines. Nortis’ assays are modular and can be set up in flexible configurations with anticipated widespread adoption in various research areas, including high-throughput drug screening.
Financial Overview
The development of Nortis’s first commercial assays were supported by three NCI SBIR Phase I grants. The company aims to raise an additional $1 million in private investment this year to support R&D efforts until SBIR Phase II grants are secured in 2013.
Intellectual Property
Nortis owns two issued U.S. patents, and two U.S. applications that were filed on this technology to cover additional features and techniques. International patent applications in important global markets are currently undergoing the examination process. Nortis is the sole owner of all related IP.
Commercialization Strategy
Nortis plans to introduce the first assays and basic perfusion platforms into the scientific research market during the second half of 2014 — ideally by partnering with a company with established sales structures in this area. Nortis anticipates that the success in the scientific research market will translate to adoption in the area of commercial drug development.
Pipeline Products
The first line of products will include a blood-brain-barrier assay, an angiogenesis assay, a metastasis assay, as well as a perfusion platform in which the assay modules can be inserted.
Management Team
Thomas Neumann, M.D., is President and CEO of Nortis. His career path includes clinical work, academic appointments, and leadership positions in industry, where he has gained extensive experience in directing multidisciplinary teams. He and Dr. Nelson are the founders of Nortis.
Alan Nelson, Ph.D., Chairman, held multiple prestigious academic positions. He is a dynamic serial entrepreneur. His first biomedical company, Neopath, won landmark FDA approval in 1995, had an initial public offering (IPO) in 1996 and was sold to Becton Dickinson in 2000.
Nortis Inc.
CEO and President
Daniel Pregibon, Ph.D.
Company Background
Firefly is introducing a universal technology platform for biomarker detection with a broad range of applications in life sciences, agriculture, veterinary medicine, and human diagnostics. Firefly BioWorks is a spin-out of the Chemical Engineering Department at the Massachusetts Institute of Technology (MIT), where the founders developed a novel method for high-throughput production of complex microparticles. The company began operations in 2010 and grew to seven full-time employees through a combination of SBIR awards and angel funding. The company is focused on developing multiplexed assays for biomarker detection that scale from discovery to clinical diagnostics. The company has adopted a lean startup approach. The first minimum viable product is being launched and operations will be scaled according to demand.
Technology Overview
Firefly develops and manufactures next-generation microparticles for biomolecule detection. The company’s first product, a kit for detecting microRNA aimed at the research market, has just been launched commercially. Firefly’s core technology, Optical Liquid Stamping, was developed by combining photolithography (typically used in microchip production) with microfluidics. This method allows fabrication of microparticles with virtually any shape, chemistry, and biofunctionality. Using Optical Liquid Stamping, Firefly developed a barcoded particle architecture that enables multiplexed biomarker detection on standard laboratory instrumentation. Through the support of the NCI SBIR Program, Firefly developed a particle-based assay for high-throughput profiling of microRNAs, a class of molecules with enormous potential for early diagnosis of cancer.
Market Potential
Firefly operates in the global market for biotechnology tools, a market that generates approximately $70 billion in worldwide sales. Firefly developed its first product for microRNA profiling, a market currently estimated at approximately $100 million and expected to grow very rapidly. Several market surveys indicate that bead-based assays for high-throughput/mid-multiplexing profiling are expected to address a critical need in the industry and grow at the fastest rate, especially in the area of microRNA.
Competitive Advantage
The Firefly platform enables detection of clinically relevant biomolecules with an unprecedented combination of performance, flexibility, throughput, and cost. Additionally, the assay developed by Firefly allows direct detection of miRNA in clinical samples without purification. This eliminates protocol discrepancies in RNA purification and will likely lead to more reliable diagnostics.
Beyond microRNA, Firefly’s platform has been used for the detection of disease-related proteins, mRNAs, and genomic DNA. The technology naturally lends itself to simple bedside or handheld devices that can be used in routine screens for early disease detection or point-of-care diagnostics.
Financial Overview
Initial funding was provided by two Phase I SBIR awards, for a total of about $500,000, followed by a seed round of $1 million from angel investors. In August 2011, Firefly was awarded a $2 million NCI SBIR Phase II award that catalyzed a $2 million second round of angel funding used for commercial development in the research market. Firefly seeks an industry partner to adopt the company’s technology and estimates additional funding in the $10 to $20 million range is required to expand its diagnostics capabilities.
Intellectual Property
Firefly has obtained an exclusive license from MIT for use of its technology in any research or clinical diagnostics application. The company’s current IP portfolio comprises four issued patents and 16 pending applications, covering the entire value chain of Firefly products from particle fabrication, encoding and bio-functionalization, to custom microRNA assays and readout in standard instrumentation.
Commercialization Strategy
Firefly’s long-term goal is to become a trusted provider of reliable and cost-effective solutions for clinical diagnostics. The company plans to first establish a presence in the research market and eventually enter the diagnostics field. Firefly intends to grow the company in three phases:
- Selling initial products to leading academic laboratories.
- Partnering with flow cytometry manufacturers for co-marketing of products.
- Expanding the product offering to include diagnostic applications by partnering with content developers in the cancer space.
Pipeline Products
Firefly’s first product is a custom 25-plex microRNA profiling kit that can be used on standard benchtop cytometers. The next generation of products will offer expanded multiplexing, a larger set of supported cytometers, and sensitivity that rivals polymerase chain reaction (PCR). Once the company has proven the technology in the field of microRNA, it will enter the protein market, with a set of companion products with relevance in both research and diagnostics.
Management Team
Firefly has built a team of executives and business advisors with deep expertise in molecular assays, microfluidics, materials, computer science, flow cytometery, and protein science, with roots in both start-ups and large life-science corporations. The team includes:
Operational Team:
Davide M. Marini, Ph.D., Co-founder, Chief Executive Officer
Daniel C. Pregibon, Ph.D., Co-founder, Chief Technology Officer
Andreas Windemuth, Ph.D., Chief Information Officer
Isaac B. Stoner, Vice President of Product Development
Andrea K. Bryan, Ph.D., Director of Engineering
Directors:
Roger Dowd and Rick Ryan
Business Advisory Board:
Joseph Gentile, Martin Madaus, Ph.D., Paul B. Manning, Ian Ratcliffe, and Michael P. Rubin, M.D., Ph.D.
Scientific Advisory Board:
David P. Bartel, Ph.D., and Patrick S. Doyle, Ph.D.
Firefly BioWorks
Co-founder and CTO
