The document summarizes a report by Scientia Advisors on the impact of DNA microarrays on molecular diagnostics. It provides an overview of the microarray market for molecular diagnostics, which is currently small but growing rapidly. Key drivers of growth include the ability of microarrays to profile multiple genes and pathways involved in complex diseases like cancer. The document also discusses regulatory requirements, reimbursement challenges, examples of microarray-based diagnostic tests, and emerging array technologies that could increase clinical adoption if assay time, ease of use, and reproducibility are improved.
Cellgen Diagnostics is an early stage venture that is developing a break through Companion Diagnostic platform that will enable Precision Medicine by determining whether a patients genetic profile is a match for the prescribed cancer therapeutic.
The document discusses liquid biopsies and next generation cancer molecular diagnostics. It summarizes that OncoCyte Corporation is focused on developing diagnostic tests for early cancer detection using liquid biopsies, with an initial focus on tests for lung cancer. Key points include that lung cancer diagnostics represents a large market opportunity and that OncoCyte's preliminary lung cancer diagnostic test shows strong performance in clinical trials with high sensitivity and specificity. The test has the potential to reduce risky follow-up procedures for patients and provide significant healthcare cost savings.
Survival guide to stem cell research and therapiesArete-Zoe, LLC
Survival guide to stem cell research and therapies provides comprehensive guidance to publicly available resource materials, libraries and registries for people who are interested in understanding currently available treatment options involving human stem cells.
Potential: The first section explains how stem cells are currently used in research, drug testing, and therapy, and how they have to be manipulated before transfer to make any treatments possible.
Classification: Origin and ability of stem cells to differentiate into different cell types determine how different types of stem cells are typically used.
Clinical Research: In this section, we will introduce the two most important registries of clinical trials: NIH registry ClinicalTrials gov and WHO International Clinical Trials Registry Platform. A project is part of this section to give students the opportunity to get hands-on experience with collecting and collating relevant information from registries and libraries, and interpretation of the findings. Real-time interactive sessions are included to allow students to ask questions and offer additional guidance.
Patient Demand: In this section, we briefly introduce challenges relating to marketing claims, objective outcome measures, advertising strategies, and patient autonomy.
Regulatory and Legal Framework: Stem cell therapies are regulated differently in various countries around the world. In this section, we will focus on regulations that govern stem cell research and therapies in the U.S. and in the European Union. Policies on stem cell research are driven by ethical concerns relating to research that utilizes human embryos. China recently announced new ethical guidelines and new rules for its stem cell clinics, regulating both trials and treatments.
Professional Societies: The last section explains the role of professional societies in stem cell research and therapies.
Oncoceutics leerink global healthcare 2015oncoceutics
- Oncoceutics has discovered a novel class of compounds called ONC201 that shows compelling efficacy against aggressive and refractory tumors in preclinical studies.
- Phase I/II clinical trials are underway at leading cancer centers to evaluate ONC201's safety and efficacy in hematological malignancies and solid tumors.
- ONC201 engages multiple critical cancer pathways without toxicity, demonstrating potential as a first-in-class therapeutic for treatment-resistant cancers.
Targovax is a biotechnology company developing oncolytic viruses and cancer vaccines to activate the immune system and fight cancer. The company has two clinical programs, ONCOS-102, an oncolytic virus, and TG, a neoantigen vaccine targeting mutated RAS cancers. Targovax is conducting clinical trials for both programs and has a strong cash position to complete its planned clinical trials into 2019.
Blueprints to blue sky – analyzing the challenges and solutions for IHC compa...Candy Smellie
Manual assessment of biomarker expression is associated with significant inter- and intra reader variability. In some cases there are also limitations when it comes to sensitivity and specificity of manual biomarker assessment.
In one example to the left, the “pure” contribution of inter-reader variability associated with Ki67 assessment was quantified across 20 tumors and 126 participating labs. In that study, it was demonstrated how image analysis can be used to significantly reduce inter-reader variability.
In a another study, the National Danish Validation study of Her2, it was demonstrated how improved sensitivity/specificity of quantitative HER2 protein expression wrt gene amplification lead to significant cost savings in reflex testing.
By automating aspects of stain quality control, it will become scalable to he point where EQA organizations may be able and willing to offer more frequent – perhaps even on-demand – proficiency testing and calibration services.
It is possible that objective and quantitative standards will contribute to improve compliance with protocol recommendations.
In clinical multi-center trials it will be easier to standardize and monitor data from each center.
And it is our hope tha larger diagnostic pathology labs will be able to benefit from such a method by closely monitoring drift in staining quality for biomarkers.
Cellgen Diagnostics is an early stage venture that is developing a break through Companion Diagnostic platform that will enable Precision Medicine by determining whether a patients genetic profile is a match for the prescribed cancer therapeutic.
The document discusses liquid biopsies and next generation cancer molecular diagnostics. It summarizes that OncoCyte Corporation is focused on developing diagnostic tests for early cancer detection using liquid biopsies, with an initial focus on tests for lung cancer. Key points include that lung cancer diagnostics represents a large market opportunity and that OncoCyte's preliminary lung cancer diagnostic test shows strong performance in clinical trials with high sensitivity and specificity. The test has the potential to reduce risky follow-up procedures for patients and provide significant healthcare cost savings.
Survival guide to stem cell research and therapiesArete-Zoe, LLC
Survival guide to stem cell research and therapies provides comprehensive guidance to publicly available resource materials, libraries and registries for people who are interested in understanding currently available treatment options involving human stem cells.
Potential: The first section explains how stem cells are currently used in research, drug testing, and therapy, and how they have to be manipulated before transfer to make any treatments possible.
Classification: Origin and ability of stem cells to differentiate into different cell types determine how different types of stem cells are typically used.
Clinical Research: In this section, we will introduce the two most important registries of clinical trials: NIH registry ClinicalTrials gov and WHO International Clinical Trials Registry Platform. A project is part of this section to give students the opportunity to get hands-on experience with collecting and collating relevant information from registries and libraries, and interpretation of the findings. Real-time interactive sessions are included to allow students to ask questions and offer additional guidance.
Patient Demand: In this section, we briefly introduce challenges relating to marketing claims, objective outcome measures, advertising strategies, and patient autonomy.
Regulatory and Legal Framework: Stem cell therapies are regulated differently in various countries around the world. In this section, we will focus on regulations that govern stem cell research and therapies in the U.S. and in the European Union. Policies on stem cell research are driven by ethical concerns relating to research that utilizes human embryos. China recently announced new ethical guidelines and new rules for its stem cell clinics, regulating both trials and treatments.
Professional Societies: The last section explains the role of professional societies in stem cell research and therapies.
Oncoceutics leerink global healthcare 2015oncoceutics
- Oncoceutics has discovered a novel class of compounds called ONC201 that shows compelling efficacy against aggressive and refractory tumors in preclinical studies.
- Phase I/II clinical trials are underway at leading cancer centers to evaluate ONC201's safety and efficacy in hematological malignancies and solid tumors.
- ONC201 engages multiple critical cancer pathways without toxicity, demonstrating potential as a first-in-class therapeutic for treatment-resistant cancers.
Targovax is a biotechnology company developing oncolytic viruses and cancer vaccines to activate the immune system and fight cancer. The company has two clinical programs, ONCOS-102, an oncolytic virus, and TG, a neoantigen vaccine targeting mutated RAS cancers. Targovax is conducting clinical trials for both programs and has a strong cash position to complete its planned clinical trials into 2019.
Blueprints to blue sky – analyzing the challenges and solutions for IHC compa...Candy Smellie
Manual assessment of biomarker expression is associated with significant inter- and intra reader variability. In some cases there are also limitations when it comes to sensitivity and specificity of manual biomarker assessment.
In one example to the left, the “pure” contribution of inter-reader variability associated with Ki67 assessment was quantified across 20 tumors and 126 participating labs. In that study, it was demonstrated how image analysis can be used to significantly reduce inter-reader variability.
In a another study, the National Danish Validation study of Her2, it was demonstrated how improved sensitivity/specificity of quantitative HER2 protein expression wrt gene amplification lead to significant cost savings in reflex testing.
By automating aspects of stain quality control, it will become scalable to he point where EQA organizations may be able and willing to offer more frequent – perhaps even on-demand – proficiency testing and calibration services.
It is possible that objective and quantitative standards will contribute to improve compliance with protocol recommendations.
In clinical multi-center trials it will be easier to standardize and monitor data from each center.
And it is our hope tha larger diagnostic pathology labs will be able to benefit from such a method by closely monitoring drift in staining quality for biomarkers.
This corporate presentation provides an overview of Oncolytics Biotech and its lead product REOLYSIN. Key points include:
- REOLYSIN is a proprietary reovirus being tested in combination with chemotherapy in multiple randomized phase 2 clinical trials for various cancers.
- Completed phase 2 trial REO 018 in head and neck cancer showed increased progression-free and overall survival when combined with chemotherapy.
- Ongoing trials include studies sponsored by the NCI in pancreatic, ovarian, lung, and breast cancers.
- REOLYSIN works by selectively replicating in Ras-activated cancer cells and is believed to have a favorable safety profile.
Introducing Drugs & Trials for Cancer DiagnosticsGolden Helix
When interpreting a variant using the AMP/ASCO guidelines for somatic variant interpretation, clinicians must determine whether the variant can be considered a biomarker that affects clinical care by predicting sensitivity, resistance, or toxicity to a specific therapy. Such a determination requires the investigation of multiple evidence sources, including clinical trials, FDA approved therapies and peer-reviewed studies. Unfortunately, strong evidence linking specific genetic biomarkers to FDA-approved therapies only exists for a small number of cancers. Thus, most variants require an exploration of clinical practice guidelines, peer-reviewed literature, and large-scale cancer mutation databases to effectively assess the clinical significance of a given mutation.
This webcast explores this new incorporation of Drugs & Trials Annotations in VSClinical's AMP Workflow covering:
Identification of relevant clinical evidence for drug sensitivity and resistance based on patient biomarkers and tumor type
Review of clinical trial information including inclusion criteria, trial status, and contact information
Management of citations associated with relevant, targeted therapies
Evaluation of a biomarkers clinical evidence tier based on available evidence for drug sensitivity and resistance
This editorial discusses regulatory issues with medical devices. It notes recalls of breast implants and hip implants that reflect failings in regulation. Medical device regulation in Europe and the US is described as having low evidentiary standards, often only requiring devices to be "substantially equivalent" to existing ones without clinical trials. This has led to unsafe devices being approved and problems going unnoticed. Stricter evidence requirements are needed prior to approval, especially for implantable devices, to better protect patients.
Gene Therapy / Cell Therapy / Stem Cells – Regulations for the "New Biol...wrtolbert
This document summarizes FDA regulations for gene therapy, cell therapy, and stem cell products. It discusses:
1) How these "new biologics" are regulated under different FDA centers and parts depending on their characteristics and risks. Products are either regulated solely under section 361 of the PHS Act or under both 361 and 351.
2) Key aspects of the new 21 CFR Part 1271 regulations including establishment registration and listing, donor eligibility, good tissue practice standards, and inspection authorities.
3) Issues related to specific cell therapies like stem cells, gene therapy vectors, and manufacturing challenges. The paradigm of regional manufacturing facilities for patient-specific products is presented as an optimal model.
1708 2 q presentation v6 uten back-upstargovax2017
This document provides a 3-sentence summary of a presentation by Targovax, a biotech company developing immunotherapies to treat cancer:
Targovax is developing two immunotherapy platforms, ONCOS-102 oncolytic virus and TG01 RAS peptide vaccine, to boost immune responses against cancer and has clinical trials ongoing or planned in several cancer types including pancreatic, melanoma, mesothelioma and others.
The presentation highlights interim clinical data from TG01 showing improved survival outcomes compared to historical controls in resected pancreatic cancer patients and outlines the company's clinical development plans and timelines over the next two years with multiple data readouts expected.
Targovax has sufficient cash runway into
Automating the ACMG Guidelines with VSClinicalGolden Helix
Clinical Genetic testing requires a complex analysis using the totality of our knowledge about the clinical relevance of a variant and a gene. This includes bioinformatic evidence as well as clinical evidence. The ACMG Guidelines provided a framework in which to score variants based on this evidence, and while some of those scoring criteria require close consultation of the clinical context for a given patient, much of it can be automated.
In this webcast, we review how VSClinical automates the ACMG scoring guidelines while integrating the collective lab expertise from previously classified variants and preferences about genes. We will cover:
Using the ACMG Auto Classifier as part the filtering strategy for gene panels and trio workflows
How gene preferences such as the default transcript, inheritance model, and disorder are updated and saved from VSClinical and used in all future analysis
How the per-variant recommendation engine builds on the auto-classification with descriptive reasons for answering each criterion yes or no
Using the auto-interpretation to present the evidence for all scored criteria in a human-readable paragraph
Working with VSClinical’s self-learning knowledgebase that incorporates previously classified variants and genes inform the interpretation of new variants!
As medical understanding of the genotype/phenotype correlation of a disease becomes clearer, genetic testing can be expected to become a mainstay in the clinical setting. While the application of genetic testing to the clinical setting is very much in line with the larger medical goals of preventative and personalized medicine, there are many unanswered questions with regard to genetic testing.
Speakers: Dr. Mansoor Mohammed, Genomics Portraits Inc., Dr. Brian Underdown, Managing Director, MDS Capital, Dr. June Carroll, Sydney G. Frankfort Chair in Family Medicine Mt.Sinai Hospital, Dr. Peter N. Ray, Head, Molecular Genetics Department of Paediatric Laboratory Medicine. HSC Professor, Molecular and Medical Genetics, University of Toronto
Avoiding Common Pitfalls in Cell and Gene Therapy TrialsMedpace
This webinar presentation discusses operationalizing advanced therapy clinical trials using lessons learned from past experiences. The webinar covers regulatory considerations, operational challenges, and case studies. Regulatory agencies require strategic engagement, assessment of regulatory readiness, and oversight of country requirements. Investigative sites face additional committee reviews and license applications. Manufacturing complex cell and gene therapies poses challenges around process transfer, scaling, and product availability. Aligning supply chain readiness, site capabilities, and an investigational product tracking process is key to avoiding delays. Developing a global strategy requires addressing requirements for manufacturing, stability data, labeling, and supply logistics early.
The document provides an overview and highlights from Targovax's first quarter 2018 presentation. Some key points:
- Targovax has two immuno-oncology programs in clinical development, ONCOS-102 and ONCOS TG.
- In the mesothelioma trial, the safety lead-in cohort of 6 patients was completed without safety concerns and showed signs of immune activation and early clinical responses in 3 patients.
- Targovax has a sound financial position with cash to fund its planned clinical program into 2019 and is listed on the Oslo Stock Exchange.
This report discusses Targovax's oncolytic virus and neoantigen vaccine programs for cancer immunotherapy. ONCOS-102, an oncolytic virus, has shown signs of efficacy in early clinical trials and is being studied in combination with chemotherapy for mesothelioma. Targovax is also developing TG, a neoantigen vaccine targeting mutated RAS cancers which accounts for many pancreatic and colorectal cancer cases. Targovax has an ongoing clinical program and aims to become a frontline treatment for mesothelioma with ONCOS-102 based on positive early data.
Targovax is a biotechnology company developing oncolytic viruses and cancer vaccines to activate the immune system and fight cancer. The company has two clinical programs, ONCOS-102, an oncolytic virus, and TG, a neoantigen vaccine targeting mutated RAS cancers. Targovax is conducting clinical trials for both programs and has a strong cash position to complete its planned clinical trials into 2019.
ACMG-Based Variant Classification with VSClinicalGolden Helix
Golden Helix is a global bioinformatics company founded in 1998 that provides software for variant annotation, filtering, and interpretation. Their flagship products are VarSeq Suite and VSClinical. VarSeq Suite allows for powerful variant filtering and annotation using curated public databases and premium annotations. VSClinical streamlines variant classification using American College of Medical Genetics and Genomics guidelines through a four-phased workflow involving variant filtering, evidence assessment, classification, and reporting. It provides consistent results and reduces learning curves for users. Golden Helix has over 400 customers globally and their software is cited in thousands of peer-reviewed publications.
Targeted genomic sequencing assay for comprehensive molecular characterizatio...Saba Anwer, MPH, MBA
The Quintiles Comprehensive Cancer Panel (QCCP) is a targeted sequencing assay that analyzes genomic variation in 223 cancer, DNA repair, and pharmacogenomic genes using Illumina sequencing platforms. It was analytically validated to CLIA standards, with a sensitivity over 99% for SNVs and indels at certain allele frequencies and a specificity over 99%. The QCCP can detect point mutations, insertions, deletions, and structural rearrangements to help identify biomarkers predictive of response to cancer therapies.
Pressure BioSciences is presenting their pressure cycling technology instruments and consumables. Their flagship product is the Barocycler 2320EXTREME instrument, which uses high pressure cycling to prepare biological samples for analysis. They have over 270 PCT systems installed worldwide and publications highlighting the advantages of PCT in biopharma sample preparation from discovery to clinical use. The company is poised for growth with their exclusive marketing agreement with SCIEX, expanded sales team, and focus on the multi-billion dollar biopharma market.
ROSALIND is a computer simulation program that uses a patient's tumor gene profile to evaluate all available cancer therapy options and predict the most effective treatments. It can simulate over 10,000 drug combinations in under 2 hours, allowing for a comprehensive and fast analysis. ROSALIND's predictions aim to personalize cancer treatment for each patient and help guide them to therapies with the highest chance of remission or available clinical trials when standard options are unlikely to work. The company envisions ROSALIND being widely adopted to achieve higher remission rates and lower healthcare costs through more personalized cancer care.
Presentation providing an overview of the recent milestones and next steps for getting Critical Outcome's p53-dependent cancer treatment, COTI-2, into clinical trials. This presentation was first given by Dr. Wayne Danter at Critical Outcome Technologies' annual shareholders meeting on October 21, 2014.
Critical Outcome Technologies Inc. is listed on the Toronto Venture Exchange under the trading symbol COT.
An overview of Critical Outcome Technologies' lead cancer drug candidate, COTI-2, which represents a potential breakthrough treatment for many types of cancer. Critical Outcome Technologies is listed on the TSX Venture Exchange under the symbol COT.
Exploring New Features and Clinical Reports in the ACMG Guideline WorkflowGolden Helix
For the past year, Golden Helix has been preparing a VarSeq release that includes ACMG Guidelines scoring and classification for not only single nucleotide variants but also copy number variants. In the past few months, our webcasts have introduced these guidelines and explored example CNVs that demonstrated the automated scoring of CNVs according to the new ACMG CNV Guidelines. However, there are many other new features and upgrades that have been incorporated into this VarSeq release. These upgrades have largely been driven by input from VarSeq users! That’s why I am very excited to explore these features together in the upcoming webcast “Exploring New Features and Clinical Reports in the ACMG Guideline Workflow”. In this webcast, we will walk through a workflow with SNVs and CNVs highlighting many of the new features and upgrades that will improve and streamline your ACMG workflow. Notably, we will see how these new features can also be incorporated into clinical reports.
Specifically, we will cover the following features:
New and improved ACMG Classifier algorithms
New and improved assessment catalogs for saving classifications and interpretations
New Project Options interface for creating patient evaluations
Now offering three new word-based clinical report templates
Inclusion of additional sections and features to the clinical reports
Enhanced report customization capability
Point of Care Diagnostics: Revenue Growth, New Entrants, InvestmentBruce Carlson
The document discusses point-of-care (POC) testing, which involves diagnostic tests performed near patients outside of centralized laboratories. It estimates the global POC diagnostics market was $17 billion in 2014 and is projected to grow to $18.7 billion by 2016. Key drivers of growth include rapid results to inform immediate treatment decisions, expanded test menus, and advances enabling quantitative lab-quality results. Major diseases addressed by POC solutions discussed are colorectal cancer, cardiovascular disease, and diabetes.
Targovax is developing two cancer immunotherapy drugs - ONCOS-102, an oncolytic virus, and TG01, a neoantigen vaccine. Data from clinical trials of ONCOS-102 showed it activated patients' immune systems against their tumors. Targovax has an ongoing clinical program testing ONCOS-102 in various cancer types and combinations. TG01 targets RAS mutations in pancreatic cancer and showed encouraging long-term survival rates in previous trials. A recent trial combining TG01 with chemotherapy showed improved median and 2-year survival over historical controls. Targovax is seeking a partner to advance TG01 into a late-stage trial aimed at registration.
Development of companion diagnostics - an FDA Perspective.pdfZhiqiangWang21
This presentation discusses several topics related to companion diagnostics from an FDA perspective, including:
- Co-development of drugs and companion diagnostics and challenges therein.
- When diagnostic tests require an Investigational Device Exemption, including considerations for determining risk level.
- Emerging concepts of complementary diagnostics that identify subgroups with different benefit-risk profiles but are not required for drug use.
- Opportunities and challenges for follow-on companion diagnostics, including lack of direct efficacy data and ensuring preserved drug effectiveness.
The Pistoia Alliance is examining the challenges of the Faster Safe Companion Diagnostics (CDx) by Aligning Discovery & Clinical Data in the Regulatory Domain.
The slides discuss whether the data standards used in the research environment be aligned better with the data standards used in the regulated environment? If so, the time and cost of the development of NGS-based CDx could be reduced.
This corporate presentation provides an overview of Oncolytics Biotech and its lead product REOLYSIN. Key points include:
- REOLYSIN is a proprietary reovirus being tested in combination with chemotherapy in multiple randomized phase 2 clinical trials for various cancers.
- Completed phase 2 trial REO 018 in head and neck cancer showed increased progression-free and overall survival when combined with chemotherapy.
- Ongoing trials include studies sponsored by the NCI in pancreatic, ovarian, lung, and breast cancers.
- REOLYSIN works by selectively replicating in Ras-activated cancer cells and is believed to have a favorable safety profile.
Introducing Drugs & Trials for Cancer DiagnosticsGolden Helix
When interpreting a variant using the AMP/ASCO guidelines for somatic variant interpretation, clinicians must determine whether the variant can be considered a biomarker that affects clinical care by predicting sensitivity, resistance, or toxicity to a specific therapy. Such a determination requires the investigation of multiple evidence sources, including clinical trials, FDA approved therapies and peer-reviewed studies. Unfortunately, strong evidence linking specific genetic biomarkers to FDA-approved therapies only exists for a small number of cancers. Thus, most variants require an exploration of clinical practice guidelines, peer-reviewed literature, and large-scale cancer mutation databases to effectively assess the clinical significance of a given mutation.
This webcast explores this new incorporation of Drugs & Trials Annotations in VSClinical's AMP Workflow covering:
Identification of relevant clinical evidence for drug sensitivity and resistance based on patient biomarkers and tumor type
Review of clinical trial information including inclusion criteria, trial status, and contact information
Management of citations associated with relevant, targeted therapies
Evaluation of a biomarkers clinical evidence tier based on available evidence for drug sensitivity and resistance
This editorial discusses regulatory issues with medical devices. It notes recalls of breast implants and hip implants that reflect failings in regulation. Medical device regulation in Europe and the US is described as having low evidentiary standards, often only requiring devices to be "substantially equivalent" to existing ones without clinical trials. This has led to unsafe devices being approved and problems going unnoticed. Stricter evidence requirements are needed prior to approval, especially for implantable devices, to better protect patients.
Gene Therapy / Cell Therapy / Stem Cells – Regulations for the "New Biol...wrtolbert
This document summarizes FDA regulations for gene therapy, cell therapy, and stem cell products. It discusses:
1) How these "new biologics" are regulated under different FDA centers and parts depending on their characteristics and risks. Products are either regulated solely under section 361 of the PHS Act or under both 361 and 351.
2) Key aspects of the new 21 CFR Part 1271 regulations including establishment registration and listing, donor eligibility, good tissue practice standards, and inspection authorities.
3) Issues related to specific cell therapies like stem cells, gene therapy vectors, and manufacturing challenges. The paradigm of regional manufacturing facilities for patient-specific products is presented as an optimal model.
1708 2 q presentation v6 uten back-upstargovax2017
This document provides a 3-sentence summary of a presentation by Targovax, a biotech company developing immunotherapies to treat cancer:
Targovax is developing two immunotherapy platforms, ONCOS-102 oncolytic virus and TG01 RAS peptide vaccine, to boost immune responses against cancer and has clinical trials ongoing or planned in several cancer types including pancreatic, melanoma, mesothelioma and others.
The presentation highlights interim clinical data from TG01 showing improved survival outcomes compared to historical controls in resected pancreatic cancer patients and outlines the company's clinical development plans and timelines over the next two years with multiple data readouts expected.
Targovax has sufficient cash runway into
Automating the ACMG Guidelines with VSClinicalGolden Helix
Clinical Genetic testing requires a complex analysis using the totality of our knowledge about the clinical relevance of a variant and a gene. This includes bioinformatic evidence as well as clinical evidence. The ACMG Guidelines provided a framework in which to score variants based on this evidence, and while some of those scoring criteria require close consultation of the clinical context for a given patient, much of it can be automated.
In this webcast, we review how VSClinical automates the ACMG scoring guidelines while integrating the collective lab expertise from previously classified variants and preferences about genes. We will cover:
Using the ACMG Auto Classifier as part the filtering strategy for gene panels and trio workflows
How gene preferences such as the default transcript, inheritance model, and disorder are updated and saved from VSClinical and used in all future analysis
How the per-variant recommendation engine builds on the auto-classification with descriptive reasons for answering each criterion yes or no
Using the auto-interpretation to present the evidence for all scored criteria in a human-readable paragraph
Working with VSClinical’s self-learning knowledgebase that incorporates previously classified variants and genes inform the interpretation of new variants!
As medical understanding of the genotype/phenotype correlation of a disease becomes clearer, genetic testing can be expected to become a mainstay in the clinical setting. While the application of genetic testing to the clinical setting is very much in line with the larger medical goals of preventative and personalized medicine, there are many unanswered questions with regard to genetic testing.
Speakers: Dr. Mansoor Mohammed, Genomics Portraits Inc., Dr. Brian Underdown, Managing Director, MDS Capital, Dr. June Carroll, Sydney G. Frankfort Chair in Family Medicine Mt.Sinai Hospital, Dr. Peter N. Ray, Head, Molecular Genetics Department of Paediatric Laboratory Medicine. HSC Professor, Molecular and Medical Genetics, University of Toronto
Avoiding Common Pitfalls in Cell and Gene Therapy TrialsMedpace
This webinar presentation discusses operationalizing advanced therapy clinical trials using lessons learned from past experiences. The webinar covers regulatory considerations, operational challenges, and case studies. Regulatory agencies require strategic engagement, assessment of regulatory readiness, and oversight of country requirements. Investigative sites face additional committee reviews and license applications. Manufacturing complex cell and gene therapies poses challenges around process transfer, scaling, and product availability. Aligning supply chain readiness, site capabilities, and an investigational product tracking process is key to avoiding delays. Developing a global strategy requires addressing requirements for manufacturing, stability data, labeling, and supply logistics early.
The document provides an overview and highlights from Targovax's first quarter 2018 presentation. Some key points:
- Targovax has two immuno-oncology programs in clinical development, ONCOS-102 and ONCOS TG.
- In the mesothelioma trial, the safety lead-in cohort of 6 patients was completed without safety concerns and showed signs of immune activation and early clinical responses in 3 patients.
- Targovax has a sound financial position with cash to fund its planned clinical program into 2019 and is listed on the Oslo Stock Exchange.
This report discusses Targovax's oncolytic virus and neoantigen vaccine programs for cancer immunotherapy. ONCOS-102, an oncolytic virus, has shown signs of efficacy in early clinical trials and is being studied in combination with chemotherapy for mesothelioma. Targovax is also developing TG, a neoantigen vaccine targeting mutated RAS cancers which accounts for many pancreatic and colorectal cancer cases. Targovax has an ongoing clinical program and aims to become a frontline treatment for mesothelioma with ONCOS-102 based on positive early data.
Targovax is a biotechnology company developing oncolytic viruses and cancer vaccines to activate the immune system and fight cancer. The company has two clinical programs, ONCOS-102, an oncolytic virus, and TG, a neoantigen vaccine targeting mutated RAS cancers. Targovax is conducting clinical trials for both programs and has a strong cash position to complete its planned clinical trials into 2019.
ACMG-Based Variant Classification with VSClinicalGolden Helix
Golden Helix is a global bioinformatics company founded in 1998 that provides software for variant annotation, filtering, and interpretation. Their flagship products are VarSeq Suite and VSClinical. VarSeq Suite allows for powerful variant filtering and annotation using curated public databases and premium annotations. VSClinical streamlines variant classification using American College of Medical Genetics and Genomics guidelines through a four-phased workflow involving variant filtering, evidence assessment, classification, and reporting. It provides consistent results and reduces learning curves for users. Golden Helix has over 400 customers globally and their software is cited in thousands of peer-reviewed publications.
Targeted genomic sequencing assay for comprehensive molecular characterizatio...Saba Anwer, MPH, MBA
The Quintiles Comprehensive Cancer Panel (QCCP) is a targeted sequencing assay that analyzes genomic variation in 223 cancer, DNA repair, and pharmacogenomic genes using Illumina sequencing platforms. It was analytically validated to CLIA standards, with a sensitivity over 99% for SNVs and indels at certain allele frequencies and a specificity over 99%. The QCCP can detect point mutations, insertions, deletions, and structural rearrangements to help identify biomarkers predictive of response to cancer therapies.
Pressure BioSciences is presenting their pressure cycling technology instruments and consumables. Their flagship product is the Barocycler 2320EXTREME instrument, which uses high pressure cycling to prepare biological samples for analysis. They have over 270 PCT systems installed worldwide and publications highlighting the advantages of PCT in biopharma sample preparation from discovery to clinical use. The company is poised for growth with their exclusive marketing agreement with SCIEX, expanded sales team, and focus on the multi-billion dollar biopharma market.
ROSALIND is a computer simulation program that uses a patient's tumor gene profile to evaluate all available cancer therapy options and predict the most effective treatments. It can simulate over 10,000 drug combinations in under 2 hours, allowing for a comprehensive and fast analysis. ROSALIND's predictions aim to personalize cancer treatment for each patient and help guide them to therapies with the highest chance of remission or available clinical trials when standard options are unlikely to work. The company envisions ROSALIND being widely adopted to achieve higher remission rates and lower healthcare costs through more personalized cancer care.
Presentation providing an overview of the recent milestones and next steps for getting Critical Outcome's p53-dependent cancer treatment, COTI-2, into clinical trials. This presentation was first given by Dr. Wayne Danter at Critical Outcome Technologies' annual shareholders meeting on October 21, 2014.
Critical Outcome Technologies Inc. is listed on the Toronto Venture Exchange under the trading symbol COT.
An overview of Critical Outcome Technologies' lead cancer drug candidate, COTI-2, which represents a potential breakthrough treatment for many types of cancer. Critical Outcome Technologies is listed on the TSX Venture Exchange under the symbol COT.
Exploring New Features and Clinical Reports in the ACMG Guideline WorkflowGolden Helix
For the past year, Golden Helix has been preparing a VarSeq release that includes ACMG Guidelines scoring and classification for not only single nucleotide variants but also copy number variants. In the past few months, our webcasts have introduced these guidelines and explored example CNVs that demonstrated the automated scoring of CNVs according to the new ACMG CNV Guidelines. However, there are many other new features and upgrades that have been incorporated into this VarSeq release. These upgrades have largely been driven by input from VarSeq users! That’s why I am very excited to explore these features together in the upcoming webcast “Exploring New Features and Clinical Reports in the ACMG Guideline Workflow”. In this webcast, we will walk through a workflow with SNVs and CNVs highlighting many of the new features and upgrades that will improve and streamline your ACMG workflow. Notably, we will see how these new features can also be incorporated into clinical reports.
Specifically, we will cover the following features:
New and improved ACMG Classifier algorithms
New and improved assessment catalogs for saving classifications and interpretations
New Project Options interface for creating patient evaluations
Now offering three new word-based clinical report templates
Inclusion of additional sections and features to the clinical reports
Enhanced report customization capability
Point of Care Diagnostics: Revenue Growth, New Entrants, InvestmentBruce Carlson
The document discusses point-of-care (POC) testing, which involves diagnostic tests performed near patients outside of centralized laboratories. It estimates the global POC diagnostics market was $17 billion in 2014 and is projected to grow to $18.7 billion by 2016. Key drivers of growth include rapid results to inform immediate treatment decisions, expanded test menus, and advances enabling quantitative lab-quality results. Major diseases addressed by POC solutions discussed are colorectal cancer, cardiovascular disease, and diabetes.
Targovax is developing two cancer immunotherapy drugs - ONCOS-102, an oncolytic virus, and TG01, a neoantigen vaccine. Data from clinical trials of ONCOS-102 showed it activated patients' immune systems against their tumors. Targovax has an ongoing clinical program testing ONCOS-102 in various cancer types and combinations. TG01 targets RAS mutations in pancreatic cancer and showed encouraging long-term survival rates in previous trials. A recent trial combining TG01 with chemotherapy showed improved median and 2-year survival over historical controls. Targovax is seeking a partner to advance TG01 into a late-stage trial aimed at registration.
Development of companion diagnostics - an FDA Perspective.pdfZhiqiangWang21
This presentation discusses several topics related to companion diagnostics from an FDA perspective, including:
- Co-development of drugs and companion diagnostics and challenges therein.
- When diagnostic tests require an Investigational Device Exemption, including considerations for determining risk level.
- Emerging concepts of complementary diagnostics that identify subgroups with different benefit-risk profiles but are not required for drug use.
- Opportunities and challenges for follow-on companion diagnostics, including lack of direct efficacy data and ensuring preserved drug effectiveness.
The Pistoia Alliance is examining the challenges of the Faster Safe Companion Diagnostics (CDx) by Aligning Discovery & Clinical Data in the Regulatory Domain.
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Nw biotech fundamentals day 2 session 4 medical devices and diagnosticsNicholas Weston Lawyers
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Similar to Scientia Advisors Impact Of Microarray In M Dx Oct 2008 (20)
Joint San Diego Chapters CLMA AACC / May 16 2010 Mtg Robert Parson
Scientia Advisors Impact Of Microarray In M Dx Oct 2008
1. 2008
Scientia Advisors, LLC
Boston ■ Palo Alto
Scientia Advisors LLC
One Main Street, 7th Floor
Cambridge, MA 02142
www.scientiaadv.com
Impact of DNA Microarrays on Molecular Diagnostics
By Scientia Advisors LLC
2. All materials copyrighted and can not be used without explicit permission2Scientia Advisors LLC
Agenda
• Market Overview
• Product case study, Regulatory, and Reimbursement Overview
• Technology Overview and Emerging Array Technologies
• Conclusions
• About Scientia Advisors and Contact Details
1
3
2
4
5
3. All materials copyrighted and can not be used without explicit permission3Scientia Advisors LLC
Microarrays for molecular diagnostics (MDx)
Definition
Microarrays for MDx
Microarrays consist of biological material hybridized to a solid surface or a bead
bound probe in aqueous suspension. They are used to profile gene expression,
compare genomic hybridization, determine protein binding sites on a gene, detect
single nucleotide polymorphisms, assay the expression of alternative splice forms of a
gene and to empirically detect expression of transcripts or splice forms that may not
have been previously known or predicted.
Includes DNA and microsphere microarray diagnostic tests; multiplexed diagnostic microarray tests;
automated diagnostic microarray analyzers; microarray kits; microarray consumables
Excludes sample preparation; splice variant microarray technologies; microarray technologies used in
drug discovery
Source: Scientia analysis
Focused arrays are those that are capable of multiplexing 25-50 targets
Arrays are typical high density microarrays capable of multiplexing 50+ targets
4. All materials copyrighted and can not be used without explicit permission4Scientia Advisors LLC
Microarrays in MDx
Small market today with attractive growth prospects driven by multiplex tests
for complex diseases such as cancer
$0
$100
$200
$300
$400
$500
$600
$700
2007 2012
WW Microarray MDx Market
Infectious Disease Breast Cancer CF HLA Other
US $ MM
Key Trends & Growth Drivers
• Array market today is limited to genetic testing such
as CF, HLA, transplantation rejection, and PGx^
(cancer market is small)
• Since many diseases and potential treatment
outcomes are determined by the function of
multiple genes and pathways; molecular assays will
require multi-dimensional analysis of genetic
alterations, pathways, and processes. This
has resulted in a pressing need for reliable
technologies such as microarrays with high
multiplexing technologies
» A large number of microarray based diagnostic
products such as those for breast cancer prognosis,
sepsis are set to enter the market over the next few
years and drive further growth
» Tests for sepsis, breast cancer prognosis are high value
tests that command premium price due to their ability
to address unmet needs and change clinical paradigm
• Breast cancer arrays are set to grow at a rapid pace
driven by products from Agendia, Veridex, and
Roche. Both Veridex and Roche are expected to
launch products soon and will be based on
Affymetrix GeneChip
• Autoimmune, cardiovascular, and diabetes represent
long-term opportunities (as biomarkers still need to
be validated)
$160M
$660M
33%
Source: Scientia analysis
* Other includes PGx^, other cancer types, and other emerging tests
^ PGx = Pharmacogenomics
*
5. All materials copyrighted and can not be used without explicit permission5Scientia Advisors LLC
Competitive landscape
Luminex has a head start but market is likely to become fragmented in the
future as numerous companies are positioned to enter the market
Key Observations
Source: Scientia analysis
2007 WW Microarray MDx Market: $160M
* Other includes Autogenomics, Nanosphere, Osmetech,
Pathwork diagnostics, and other emerging companies
• Today the microarray market is dominated by Luminex,
with its own product in CF and platform partnerships
with One Lambda and Tepnel lifecodes for HLA typing
• Although, Agendia MammaPrint Dx for breast cancer
recurrence was the first IVDMIA to be approved; the
product is not as well accepted as Genomic Health’s
OncoType Dx
• Although Roche/Affymetrix was the first to enter the
market with FDA approval; reception of the AmpliChip
has been lukewarm
• Currently, Innogenetics (now Solvay Pharmaceuticals) CF
product is based on its line probe array and will be
switched to its array 4-Mat microarray platform soon
• XDx is gaining traction with strong performance of
AlloMap to monitor heart transplant rejection
• Roche and Veridex (JnJ) are expected to launch several
assays in the oncology area in the near future using the
Affymeterix GeneChip
• Emerging companies such as Almac, Autogenomics,
Combimatrix, ExonHit, GeneWave, Nanosphere,
Osmetech, Pathwork Diagnostics, and Xceed Molecular
are positioned to enter the market with offerings in
oncology, pharmacogenomics, genetic testing , diabetes
and inflammation
•Agendia
•Innogenetics
•Roche/Affymetrix
•Tepnel/Luminex
•Tm-Luminex
•XDx
•Other
Luminex/One
Lambda
6. All materials copyrighted and can not be used without explicit permission6Scientia Advisors LLC
Agenda
• Market Overview
• Product case study, Regulatory, and Reimbursement Overview
• Technology Overview and Emerging Array Technologies
• Conclusions
• About Scientia Advisors and Contact Details
2
3
1
4
5
7. All materials copyrighted and can not be used without explicit permission7Scientia Advisors LLC
Roche’s AmpliChip CYP450 Case Study
This test has had limited adoption due to a lack of physician education and
reimbursement
• Although the test was FDA approved; acceptance
of the test has been lukewarm
• One of the key reasons the test had limited
acceptance was lack of sufficient evidence for
incorporation of into guidelines for clinical practice
» The professional journal American Family
Physician stated in August 2007: “As new
genetic testing technologies are approved and
made available for clinical use, it is important to
emphasize that FDA approval is based on test
accuracy rather than on demonstration of
improved clinical outcomes. Availability of
technology should ideally be paralleled by
availability of evidence regarding its impact in
clinical practice (through methodologically
sound studies), which unfortunately has not yet
happened in the case of CYP450 genotyping.”
» Most insurance companies do not cover the cost
of the test due to uncertainty of cost benefits
• Array companies seeking to launch products need
to keep in mind that besides regulatory approval;
reimbursement and physician education through
clinical outcomes studies are paramount
Key Learnings
The Roche AmpliChip Cytochrome P450 Genotyping test based on the
Affymetrix GeneChip Microarray aims to identify slow and fast drug
metabolizers by capturing genetic variations in two genes, CYP2D6 and
CYP2C19 in the cytochrome P450 system.
Source: Scientia analysis, Roche, American Family Physician, Aetna
8. All materials copyrighted and can not be used without explicit permission8Scientia Advisors LLC
Regulatory Requirements
Clinical burden varies by regulatory filing; EU less stringent than the U.S.
US Regulatory Bodies EU Regulatory Bodies
IVD Directive
Guides
requirements for
clinical diagnostic
products and
manufacturing
practices (device
classification)
CE Mark
Seal of approval to
sell clinical
product – to be
renewed every 5
years
CLIA
Extends regulation
for devices for
performance sites
Source: Scientia analysis, FDA
Regulates
diagnostic
products and
related
manufacturing
practices
• Research use only (RUO)
• Investigational use only (IUO)
• Special purpose reagents (SPRs)
• 510(k) [Class I or II]
• Premarket Approval (PMA) [class
III]
• In vitro Diagnostic Multivariate
Index Assay (IVDMIA)
• Analyte Specific Reagent
(ASR)
» Can only be sold to high
complexity CLIA labs
» Prohibited from making
any statement regarding
analytical or clinical
performance
• Manufacturer’s self-
declaration of conformity to
the Medical Devices Directive
(MDD) and its essential
requirements
» Intervention of a
notified body is not
required
» Firms are subject to
inspection for
conformance to ISO
standards
• Device classification is based on
adherence to Annex II, IV, V, VI,
and VII
• These devices must not only
be compliant with the basic
requirements under the EU IVD
law, but also require a
conformity assessment by a
notified body
FDA
9. All materials copyrighted and can not be used without explicit permission9Scientia Advisors LLC
Source: Scientia analysis, FDA, Nature, HHS
• On September 7, 2006 the FDA issued a draft guidance
interpreting and explaining the ASR* rule, and on July 26,
2007 the In vitro Diagnostic Multivariate Index Assays
“IVDMIAs^”
» Agendia’s MammaPrint Dx for breast cancer recurrence
was the first IVDMIA^ test cleared by the FDA in February
2007
• With these new guidance and policies the FDA intends to
regulate the novel, high value multiplex MDx tests from the
moment they are introduced into the market. Manufactures
will no longer be allowed to register them as ASRs
• The FDA is requiring more pharmacogenetics information.
The agency relabeled Warfarin to accompany
pharmacogenetic testing by approving the first genetic test
for Warfarin sensitivity, “Verigene” in September 2007. The
test can determine an individual’s ability to metabolize the
anti-coagulant medication, this information is critical to
determining safe and appropriate dosing
» Osmetech’s Warfarin sensitivity test was cleared by the
FDA in July 2008
• These moves indicate that the agency recognizes MDx as
being paramount for personalized medicine and better health
outcomes
» This outlook from the FDA will drive pharma to change
their perspective on personalized medicine tests
^ In Vitro Diagnostic Multivariate Assays multiplexed tests that use a computer software to
analyze the test results to make a final diagnosis or diagnostic recommendations)
* Analytic Specific Reagent
Cleared by FDA on February 2007
Key Takeaways
The FDA is Being Proactive About Array based MDx Tests
Array based test was the first approved IVDMIA
10. All materials copyrighted and can not be used without explicit permission10Scientia Advisors LLC
IVD Reimbursement
Reimbursement differs by geography; better policies are needed for new high
value diagnostics*
Source: Scientia analysis, The Lewin group
xx
xx
U.S.
EU
• Reimbursement in the U.S. is determined by CMS and private insurers
• CMS pays for each test based on a fee schedule using CPT codes determined by American Medicare Association
(AMA)
• CMS decisions can influence private insurers
• Many new laboratory tests that are substantially similar to existing tests may be assigned to the same code
• New tests can petition for a new code from the AMA, but this process can be lengthy and often results in delays.
Outcome studies are essential to prove perceived value of tests; The AMA in conjunction with a panel of experts,
may assign substantially new tests a new code. CMS then decides on a method of payment
• MDx companies such as Genomic Health and XDx are blazing the path for reimbursement of high value MDx tests
such as breast cancer prognosis. Third party payers such as the California Medicare contractor, National Heritage
Insurance Company, United and Aetna are starting to reimburse these tests
• The reimbursement of in vitro diagnostic tests in Europe is determined by individual government bodies. European
nations have compulsory health insurance for citizens, high levels of coverage and public ownership of hospitals
• In U.K, the pricing environment is affected by budget constraints, as budgets are based on historical spending rates
rather than future demand. NHS local lab directors decide how to spend their allocated diagnostic budget. NICE is
starting to screen diagnostics and make recommendations as part of broader disease management plans
• The DRG^ payment system in Germany is developing and can considerably affect diagnostic reimbursement. Office-
based payment for tests may depend on findings of a technology assessment committee
• The French reimbursement process is also made via DRG and requires an assessment of medical benefit by a
government commission
*=Tests that provide critical information to help physicians make clinically relevant decisions; as a result command premium price
^ Diagnosis related group: A new reimbursement system based on the number and type of procedures rather than bed days
11. All materials copyrighted and can not be used without explicit permission11Scientia Advisors LLC
MDx Tests Reimbursement
Although codes and regulations are evolving, better policies are still needed
** Includes relevant old and new codes
* Hospital associated infections
^Secretary’s Advisory Committee on Genetics, Health, and Society
• Historically several MDx reimbursement claims have been denied
due to inaccurate codes use
• Recent changes to MDx coding, including the addition of eleven
new codes in the 2006 and 2007 CPT are expected to partially
improve this situation
• Several organizations such as SACGHS^, the Lewin Group have
questioned Medicare’s coding for MDx and believe
reimbursement needs to be re-adjusted to reflect their added
value
• Under new Medicare regulations, hospitals will no longer receive
higher payments for the additional costs associated with treating
patients for certain hospital- acquired infections. These new rules
will go into effect in October 2008 and will compel hospitals to
improve care by migrating to fast turnaround time MDx tests
• When reimbursement bodies switches to DRG^^, only MDx tests
showing significant clinical utility will be reimbursed
• MDx companies such as Genomic Health and XDx are blazing the
path for reimbursement of high value MDx tests such as breast
cancer prognosis
• Reimbursement agencies need to implement better policies to
better accommodate high value multiplex MDx tests* that are
changing the healthcare paradigm (currently there are no codes
available to bill for multiplex tests such as MDx tests for sepsis
causing pathogens)
Key Takeaways
CPT Code
Total # of
claims
submitted
% of
Claims
Denied
83890 (extraction) 162,297 48%
83896 (DNA Probe) 422,628 25%
83898 (amplification) 639,134 40%
83901 (Multiplex
Amplification)
85,105 36%
83902 (Reverse
transcription)
42,670 30%
87798 (infectious agent) 54,939 10%
Procedure CPT Code
Molecular diagnostic assays 83890 - 83914
Assays which identify or quantify
infectious agent antigens
87470 - 87660
DNA based studies on culture
material
87149
Lack of an organism specific code
for a DNA based infectious agent
assay
87797-99
Total number of claims for molecular diagnostic laboratory tests
submitted to all Medicare carriers, ’00-’04
Relevant CPT Codes**
Source: Scientia analysis, CMS, other industry reports
^^ Diagnosis related group
*=Tests that provide critical information to help physicians make clinically relevant decisions; as a result
command premium price
12. All materials copyrighted and can not be used without explicit permission12Scientia Advisors LLC
Agenda
• Market Overview
• Product case study, Regulatory, and Reimbursement Overview
• Technology Overview and Emerging Array Technologies
• Conclusions
• About Scientia Advisors and Contact Details
3
1
2
4
5
13. All materials copyrighted and can not be used without explicit permission13Scientia Advisors LLC
Microarrays in MDx
Arrays are invaluable multiplexing tool; however, widespread use in MDx will
require reproducible, fully automated and easy to use array platforms
Characteristics of a well engineered microarray platform: miniaturization, massive
scale-up, low costs, reproducible data, complete automation and minimal biological
sample requirements
Source: Scientia analysis, IVDT
Workflow of developing a diagnostic array
• During the past decade, DNA microarrays have been
used in every facet of life science research, from basic
science to the study of clinically relevant diseases
• In MDx, arrays have been historically viewed as
powerful research tools to identify markers; However,
with the recent advent of commercially available,
low-cost, reproducible, technically simple arrays, and
easy-to-use analytical software, the direct use of
microarrays as diagnostic platforms have emerged
• With their ability to gain more information from a
limited clinical sample by using highly parallel
expression-analysis techniques; arrays are powerful
MDx tools. Since many diseases and potential
treatment outcomes are determined by the function
of multiple genes, DNA microarrays will be important
in advancing molecular diagnostics
14. All materials copyrighted and can not be used without explicit permission14Scientia Advisors LLC
Comparison of MDx technologies
Although arrays score high in multiplexing; TaT^, ease of use, and ability to
quantitate need to be improved for wide spread clinical adoption
Real Time PCR
Other Amp.
Tech.
Signal Amplifi-
cation
Focused
Arrays*
Arrays**
Sequencers
(incl. Nex Gen)
Multiplexing
Quantitation
Automation
TaT^
Accuracy (Sens. &
Spec.)
Ease of Use
Low Med High
Source: Scientia customer survey and analysis
* Focused arrays are those that are capable of multiplexing 25-50 targets
** Arrays are typical high density microarrays capable of multiplexing 50+ targets
^ TaT = Turnaround time
Scientia Confidential
15. All materials copyrighted and can not be used without explicit permission15Scientia Advisors LLC
Emerging Array Technologies
Several promising technologies on the horizon
Nanosphere
Illumina
Asuragen
• Illumina’s BeadArray Technology is based on 3-
micron silica beads that self assemble in micro wells
on either of two substrates: fiber optic bundles or
planar silica slides
» Allows for stringent quality control, high reproducibility
» Allows for multi sample testing
NimbleGen
High throughput, cost effective array
High sensitive, easy to use array
mi RNA array for diagnostics
Epigenetics array
• NanoSphere’s Verigene system has many
improvements
» Utilizes nano-sized magnetic beads
» Two mechanisms of signal amplification for analytes
with low abundance, reports 2-3 fold greater sensitivity
Source: Scientia analysis
• Asuragen’s miRNA arrays are based on Agilent and
Affymetrix arrays
» Allows for high sensitivity, specificity, and
reproducibility
• NimbleGen HD2 arrays offer the highest density
microarray platform currently available for CGH and
ChIP analysis
» Fewer arrays , less sample are required leading to a
significant reduction in cost
» Greater flexibility with increased sensitivity, specificity,
and reproducibility
16. All materials copyrighted and can not be used without explicit permission16Scientia Advisors LLC
Microarray
Planar
Array
BeadArray Probe-
based
(TaqMan)
Sanger
Sequencing
"Next Gen"
Sequencing
Affymetrix (AFFX) x
ABI x x x
Helicos x
Illumina (ILMN) x x x
454 / Roche (ROHHY) x
ATTRIBUTES
Flexibility for custom content -- + ++ --
Robustness of fixed content + + -
Accuracy / Reproducibility - ++ ++ ++ ++
Throughput: # GT's sample + ++ - -- ++
Throughput: Samples / Study -- + -- +
Per Genotype Cost + ++ - -- -
Per Sample Cost - ++ ++ - -
APPLICATIONS SUITABILITY
Candidate Gene Association + ++ -- -- --
Whole Genome Association + ++ -- -- --
Disease Panels / Pathways ++ ++ + + ++
Clinical diagnostics - ++ + -- +
NexGen Sequencer
Next Generation (NexGen) Sequencing
Potential to displace arrays as they become faster and cost effective
Key Takeaways
• NexGen sequencers have favorable features such as accuracy and suitability to clinical diagnostics (as highlighted in the
table)
• In its current state, arrays fare better than NexGen Sequencers in MDx
• Ultimately, when the price per base goes down , throughput increases, and informatics solutions improve NexGen
Sequencers have the potential to displace arrays
Source: Scientia analysis
Scientia Confidential
17. All materials copyrighted and can not be used without explicit permission17Scientia Advisors LLC
Agenda
• Market Overview
• Product case study, Regulatory, and Reimbursement Overview
• Technology Overview and Emerging Array Technologies
• Conclusions
• About Scientia Advisors and Contact Details
4
3
2
1
5
18. All materials copyrighted and can not be used without explicit permission18Scientia Advisors LLC
Conclusions
• The microarray market is small today with tests in genetic testing and
pharmacogenomics. The market is expected to grow rapidly due to increasing need
for multiplexing and expected launch of several high value tests such as breast
cancer prognosis and sepsis
• Luminex is currently the market leader; however market is likely to become
fragmented as several companies including Veridex (JnJ) are positioned to enter
the market
• Regulatory, reimbursement, and physician education are hurdles to adoption
• Although arrays are exceptional in multiplexing, inability to quantitate and lack of
ease of use is likely to hinder wide spread adoption when compared to MDx
technologies such as real time PCR
• Although current forms of next generation sequencing may not be threatening;
ultimately, when the price per base goes down , throughput increases, it has the
potential to displace arrays
• Several promising new array technologies are on the horizon that could address
limitations of current products on the market
• All in all, arrays are powerful MDx tools and is likely to continue its overall growth
over the next 5-10 years
19. All materials copyrighted and can not be used without explicit permission19Scientia Advisors LLC
Agenda
• Microarray Diagnostic Market
• Product case study, Regulatory, and Reimbursement Overview
• Technology Overview and Emerging Array Technologies
• Conclusions
• About Scientia Advisors and Contact Details5
3
2
4
1
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Science, Knowledge and Skill for your competitive advantage.
Scientia Advisors is an international strategy and management consulting firm
with a concentration in life sciences. We have one mission: To ensure that our
clients consistently outperform the market and their competitors.
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» We help the world's best and most innovative life science
companies make decisive improvements in their direction
and performance.
» Our highly collaborative work style converts insight into
strategy that substantially impacts performance.
» We continually strive to generate experience-based insight into
what drives value creation and competitive advantage
in our clients' businesses.
» We earn the trust of our client teams
by consistently delivering impact.
22. All materials copyrighted and can not be used without explicit permissionConfidential and Proprietary - Do not distribute
Our Capabilities Include:
Corporate Strategy
• growth strategy
• core growth
• white space growth strategy
Customer Strategy
Mergers and Acquisitions
• strategy
• acquisition screening
• strategic due diligence
• divestitures
• joint ventures and alliances
Venture Capital/Private Equity
• due diligence
• portfolio strategy
• exit strategy
Emerging Companies
• post investment growth
strategy
• program prioritization
• market strategy and
positioning
• valuation
23. All materials copyrighted and can not be used without explicit permission23Scientia Advisors LLC
CONTACT INFORMATION
info@scientiaadv.com
Office: +1- 617-299-3000
Fax: +1-617-812-0315
One Main Street , 7th Floor
Cambridge, MA 02142
THIS PRESENTATION IS MEANT TO BE ACCOMPANIED BY COMMENTARY &
VIEWPOINTS BY SCIENTIA ADVISORS
THIS IS NOT MEANT TO BE A STANDALONE DOCUMENT ON WHICH TO BASE THE
FINAL VIEWPOINTS OF SCIENTIA ADVISORS OR ON WHICH TO BASE THE
FINAL AND DEFINITIVE GO-FORWARD DECISIONS