Contact UsDiana Bartlett
Assistant Vice President,
Phone: (909) 607-9864
Email: diana_bartlett[at symbol]kgi.edu
Aemetis, Inc. is an industrial biotechnology company producing renewable chemicals and fuels using patented microbes and processes. Derived from the Scottish “Ae,” meaning “the one” and the Greek “Metis,” meaning “prudent wisdom,” Aemetis means “the one prudent wisdom,” referring to the prudence and wisdom of replacing petroleum with renewable chemicals and fuels. Focusing on the development and commercialization of innovative industrial biotechnologies, the Aemetis technology platform helps companies increase their brand value and operating margins while decreasing their environmental footprint. By converting existing bio-refineries and substantially reducing capital expenditures, Aemetis assists in the quick commercialization of technologies through joint ventures with existing biofuel plants.
Aemetis is currently in the process of investigating new biofuel feedstocks to supplement their existing corn ethanol plant in Keyes, CA. Initial tests have been completed and potential feedstocks identified. To determine the feasibility of making an industrial-scale transition, Aemetis asked the Aemetis TMP team to perform a detailed feasibility analysis.
Specifically, the Aemetis TMP team was asked to develop a financial feasibility study under the constraint of using one specific advanced feedstock. To accomplish this objective, the Aemetis TMP examined the economic, technical and regulatory aspects of this novel feedstock as a supplement for the existing ethanol process at the Keyes facility. The team examined three major components of the feedstock value chain: upstream, midstream and downstream. All three elements were included in the final detailed financial model. Ultimately, the team’s final report will provide Aemetis with an analysis which may help guide the use of this interesting feedstock.
Amicus Therapeutics is a Cranbury, New Jersey based biopharmaceutical company discovering and developing next-generation medicines to treat a range of rare and orphan diseases, with a focus on improved therapies for lysosomal storage diseases (LSDs). LSDs are a family of rare genetic metabolic disorders that are chronic, degenerative conditions which are usually fatal.
Amicus is interested in understanding the current status of newborn and related screening practices for LSDs in the United States. Of particular interest is how these practices relate to patient populations and diagnostic trends.
The Amicus TMP team was tasked with assessing the feasibility of implementing newborn and at risk population screening programs for LSDs. In conjunction with the Newborn Screening Branch of the U.S. Centers for Disease Control and Prevention, the team conducted a nation-wide survey to capture the attitudes of state health departments towards the mandated screening of LSDs in newborns. The team also conducted primary research to determine the mechanics of screening both family members of affected children and high-risk clinical populations. In their analysis, the team explored ethical concerns, technological hurdles, market dynamics, and future trends that may affect screening programs. Ultimately, the team recommended strategies for Amicus Therapeutics to facilitate national screening procedures and increase the detection of individuals with lysosomal storage diseases.
Astellas Pharma US, Inc.
Astellas Pharmaceuticals is a global pharmaceutical company with the aim of improving lives through innovative and effective medicines. Astellas Pharmaceuticals was formed by the merger of two established Japanese pharmaceutical companies in 2005. It is headquartered in Japan and has seven therapeutic areas of focus. They are currently global category leaders within the urology and the transplantation therapeutic areas.
Astellas has entered into the oncology therapeutic area and is focused on becoming a global category leader in oncology. To this end, it has allocated significant investments to enhance its presence within the oncology market. In support of its leadership goals in oncology, Astellas tasked the KGI TMP team to conduct a gap analysis comparing current oncology companies and Astellas.
To complete the goals set for the TMP, the project was divided into three phases. In Phase 1, the Astellas TMP team reviewed the larger oncology landscape and interviewed key members of the Astellas oncology leadership team. In Phase 2, pipeline analysis, image ranking and company profiles were built through interviews and surveys of oncology company executives and medical oncologists. In Phase 3, the Astellas TMP team identified existing gaps between Astellas and other global category leaders. Specific evidence based recommendations were provided to Astellas oncology leadership to bridge gaps and further their goal of becoming a global category leader in oncology.
Beijing Zhongmei Aochi Biotech Co. Ltd.
Zhongmei is the largest medical group in China, with its medical chain spanning across 7 major hospitals. In 2007, Zhongmei funded a subsidiary company geared to target the healthcare industry in China: Beijing Zhongmei Aochi Biotech Co. Ltd. Partnering with Blue-Bio Research Institute and KND. Co. Ltd, with $180 million in investment, Aochi Biotech produced collagen supplements, which have been the best-selling anti-aging supplement in China. Anti-aging therapies aim to increase longevity through the treatment of various age-related metabolic, cardiovascular, neurological, and immune diseases. As the aging population of China increases, such therapies have potential to expand the market.
Beijing Zhongmei Aochi Biotech Co. Ltd. is interested in licensing advanced anti-aging and stem cell therapies. The Zhongmei Group has a commercial interest in developing these novel technologies for use in clinical settings in China. In support of this strategy, our TMP was asked to identify and assess anti-aging technologies from US universities and companies which might be further developed by Aochi Biotech and implemented in Zhongmei hospitals. Each technology was carefully evaluated with respect to effectiveness, IP protection, regulation, and market potential.
From our survey of numerous of cosmetic anti-aging technologies, we have created a database consisting of 20 potential candidates. Of these, five novel products and devices have been pursued further according to the sponsor’s request. The scope of our investigation has since expanded from cosmetic technologies to include advancements utilizing stem cells. We are thus engaged in surveying potential stem cell therapies in cardiovascular, neurological, and kidney diseases. This assessment aids in our development of a robust database consisting of both cosmetic and stem cell therapies that treat diseases prevalent in aging China. Upon project completion, we will have pursued technologies of particular interest to the sponsor, which will ultimately aid the Zhongmei Group in bringing therapies that can be adopted in the Chinese market.
BioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical Inc., of Novato, California, develops, manufactures and commercializes biopharmaceuticals for the treatment of rare diseases. BioMarin has successfully launched four products and is actively engaged in joint ventures and partnerships. With commercial operations in over forty countries and a robust clinical pipeline, BioMarin is a key player in the rare (orphan) disease market, providing access to treatments for patients with historically unmet medical needs.
Cold chain refers to a temperature-regulated supply chain, encompassing storage, handling, shipping, and distribution activities. Cold chain is a fast-growing part of the healthcare logistics industry, and providers are adapting to meet the ever-changing regulatory demands. In addition, biotechnology companies ship high-value and temperature sensitive products, often in experimental or developmental phases of clinical trials, the latter comprising the majority of industry costs. Cold chain compliance, although a non-revenue generating activity, is essential to the operation of modern biotechnology companies.
Currently, BioMarin has a very product-specific approach to the clinical product cold chain and is looking for a template strategy that will provide a consolidated, integrated and up-to-date solution to their cold chain needs. The BioMarin TMP team approached this project in two phases. First, the team determined the current landscape of pharmaceutical cold chain through conference attendance and interviews of subject matter experts, and gathered BioMarin’s requirements for their cold chain. The BioMarin TMP then applied these requirements through a decision matrix of vendor solutions to identify the most appropriate primary and tertiary containers for shipping of bulk clinical products. The project’s second phase focused on qualification testing of the container candidates, analysis of cargo carriers, as well as an examination of commercial cold chain requirements (bulk drug substance and vial shipping). Ultimately, our team provided a comprehensive, commercial-ready list of solutions to meet BioMarin’s cold chain needs for their pipeline products.
Castle Biosciences Inc.
Castle Biosciences Inc. is a privately held company specialized in developing diagnostics for rare cancers. Castle Biosciences works alongside leading cancer institutions to develop diagnostics designed to individualize treatment plans. Castle Bioscience’s mission is to provide improved prognostic guidance to medical practitioners for patients with rare cancers. The company’s proprietary diagnostic technology platform— DecisionDx—is the outcome of both technology in-licensing from centers of excellence in oncology as well as internal development. The company currently has four commercially available diagnostic tests for uveal melanoma, thymoma, glioma and glioblastoma. Castle Biosciences is currently pursuing the launch of a novel diagnostic for esophageal cancer (DecisionDx-EC) and a second diagnostic test for cutaneous melanoma (DecisionDx-Melanoma).
The first phase of the project involved a literature search to identify potential biomarkers for company-selected rare cancers, which might provide utility in predicting therapeutic response. To facilitate this goal, the team constructed a database which consolidated numerous biomarkers across a number of rare cancers, as well as a second database consisting of key opinion leaders with expertise in both cutaneous melanoma and esophageal cancer. These efforts are expected to support Castle Bioscience’s efforts to identify new diagnostic targets for portfolio expansion.
The second phase of the project involved demonstrating the economic value proposition for their esophageal cancer and cutaneous melanoma diagnostic tests. The Castle Bioscience TMP constructed and distributed surveys aimed at identifying a surgeon’s willingness to alter treatment strategies based on a predictive assessment of neoadjuvant therapy. Ultimately, the Castle Bioscience TMP will publish results from this survey as foundational support for the product launch. Additionally, the team built a budget impact model designed to identify the financial benefit to stakeholders providing care for patients diagnosed with cutaneous melanoma. The end goal for this objective is to provide a data-based economic rationale for the adoption of DecisionDx-Melanoma by clinicians who seek to better predict the occurrence of aggressive manifestations caused by cutaneous melanoma.
City of Hope
City of Hope (COH) is a non-profit institution dedicated to fighting life-threatening diseases using a three-pronged approach that combines biomedical research, treatment and education. COH’s research arm, the Beckman Research Institute, focuses on diabetes and HIV/AIDS, but has a particular emphasis on cancer research. This emphasis has produced results that have garnered national and worldwide recognition by being included within The National Cancer Institute of the National Institutes of Health designated Comprehensive Cancer Centers.
Recent advances in our understanding of the molecular basis of cancer have resulted in a surge in ‘personalized medicines’ for the treatment of cancer. These personalized medicines target specific molecular changes which cause different forms of cancer. Fortunately, some personalized medicines such as Gleevec® and Herceptin® have resulted in significant improvement in therapy while producing fewer side effects than older medicines. The Beckman Research Institute has invested heavily in research aimed at the expanding the number of personalized therapies. To better understand the impact personalized medicines, the Beckman Research Institute commissioned this TMP to investigate the impact of these medicines from a patient and payer perspective.
In order to create cost effectiveness models, the team investigated all personalized therapeutics from 1995 until today creating a short list of drugs which have made a major impact in the oncology disease space. A quantitative model was developed to determine the cost per life month added based on the clinical utility, adverse events and overall survival rate. The output from these models will be used to determine the cost-effectiveness ratios of major personalized medicine therapies and will be analyzed using cost effective thresholds around the world. It is hoped that these quantitative models will help guide COH to better understand the required cost-effectiveness ratios necessary to create revolutionary personalized medicines.
Edwards Lifesciences is the global leader in the science of heart valves and hemodynamic monitoring. Founded in 1958, Edwards has grown into a global company with a presence in approximately 100 countries and more than 7,800 employees around the world. Edwards focuses on medical technologies that address large and growing patient populations in which there are significant unmet clinical needs, such as structural heart disease and critical care monitoring.
Edward’s hemodynamic monitoring instruments measure cardiovascular system parameters that are derived from sampled data acquired by sensors. Typically, sensors are attached to, or integrated within catheters which are inserted into blood vessels. Occasionally, sensors may be placed externally to the patient to provide less invasive sensor information. Edwards has created a proprietary biomedical engineering platform to support the rapid development of biomedical algorithms for these hemodynamic monitoring instruments.
The core goal of the Edwards TMP is to facilitate the software design, implementation, and testing of Edwards biomedical engineering platform. The project focuses on two interrelated subprojects: physiological modeling and statistical performance testing of experimental/ simulation data. The key objective of physiological modeling is to complete development of a physiological mathematical model to generate realistic, simulated blood pressure signals coupled with cardiovascular parameters. The objective for the statistical performance testing is to develop a library of hemodynamic algorithms. In support of this project, the team has developed an application programming interface to read/ write patient signals and to maintain database archives encoded in the Edwards signaling format.
Eli Lilly and Company
Eli Lilly was incorporated in 1881 and even after more than a century, it continues to be at the forefront of pharmaceutical research. Lilly has been a leader in the industry for both small-molecule development as well as biologics research. It has pioneered many areas in the industry, being the first company to successfully produce and deliver penicillin on a large scale, as well as the first to use recombinant DNA techniques to manufacture insulin for production.
Traditionally, pharmaceutical projects have been managed, scoped and led through cross-functional internal project teams focused on delivering a new medicine that meets the requirements of two key documents: the Draft Launch Label (DLL) and the Manufacturing Marketed Forms Agreement (MMFA). Typically, these documents contain a set of manufacturing and launch requirements that are created by medical, marketing, regulatory, manufacturing, quality and product development. While this process can be effective, it is believed that additional value may be gained by using co-creation techniques with customers at time points earlier than the DLL and MMFA to create more robust and complete product design requirements. The hypothesis is that we often neglect to consider all of the stakeholder interests during development that could lead to a more valuable product in terms of improved patient outcomes and a lower cost of care.
In order to address these issues, the Lilly TMP team conducted research on application of co-creation to the Lilly drug design and development process. We then identified, developed, and refined six separate engagement platforms that will serve to incorporate Lilly customers more effectively in the design process as a means to improve the end value of their products. Towards that goal, we conducted interviews and a survey with the Lilly development teams as well as experts in Clinical Trials, Patient Advocacy, Legal and Information Technology to determine processes that would help Lilly interact more effectively with its customers. We are currently incorporating feedback from the interviews and the survey to define metrics for success and devise a plan for implementation of a pilot study at Lilly for the top two engagement platforms.
Endologix develops, manufactures, and markets minimally invasive medical devices for the treatment of aortic disorders with a focus on the endovascular repair of abdominal aortic aneurysms (AAA). On December of 2010, the company finalized the acquisition of Nellix, a next-generation endovascular aortic repair (EVAR) technology designed to reduce the rate of secondary interventions and post-procedure surveillance. Unlike currently available technologies, the Nellix device seals the aneurysm sac by filling “endobags” with a biocompatible polymer to minimize the potential for aneurysm growth and rupture, and device migration.
The Endologix TMP team was tasked with conducting technical feasibility studies of the Nellix device for a new indication in the thoracic aorta, which has unique and dynamic biomechanical constraints. An initial assessment of the competitive landscape helped the team clarify the value proposition for the Nellix platform and identify areas where the Nellix device can lead to improved quality of life and reduced cost. Following this analysis, the team investigated the physiological constraints observed in the thoracic therapeutic in an effort to clarify design parameters and engineering constraints for a future Nellix EVAR device.
The Endologix TMP developed, and implemented experimental and computational protocols to test the ability of the Nellix polymer formulation to withstand the dynamic environment in the thoracic aorta. Based on the data collected, the team developed recommendations to optimize therapeutic performance, reduce operating costs, and enhance market strategy for a future Nellix EVAR device.
Gilead Sciences is a biopharmaceutical company that researches, develops and markets innovative treatments for life-threatening diseases. Gilead’s therapeutic areas of focus include HIV/AIDS, hepatitis, serious respiratory, cardiovascular, and metabolic conditions, cancer and inflammation. Since their founding 25 years ago, Gilead Sciences has grown rapidly, with a portfolio of 15 marketed products and a growing pipeline of investigational drugs and approximately 5,000 employees across four continents.
Process development for monoclonal antibody (mAb) production is a time-consuming, labor intensive and expensive effort due to the number and complexity individual steps involved. To prevent bottlenecks and ensure commercial viability of future monoclonal antibody manufacturing, it is important to accelerate the development of both upstream and downstream processes, as well as to improve process analytics at each step.
The Fall Gilead TMP provided an exhaustive assessment of recent innovations in mammalian cell line development for mAb production. Commercially available mammalian expression technologies for mAb products were identified and compared. The team provided a recommended list of top mAb expression systems which provide outstanding features and have a large client base. This analysis provides Gilead Sciences with cell line options which are friendly to regulatory agencies and have a proven performance in antibody production.
For the Spring TMP Project, the team identified, evaluated and made recommendations for high throughput analytical technologies which can be used for rapid bioprocess development at Gilead. The recommendations were based on the need to optimize the bioprocess design space (QbD enabling), easily integrate into high-throughput upstream and downstream technologies and provide resource efficiency. In support of this objective, the team explored high throughput technologies being developed at a number of academic institutions and companies. To provide a detailed analysis of each technology, the team conducted detailed interviews with scientists developing these high throughput platforms. Finally, the team provided Gilead with a report which included a detailed evaluation and recommendation of technologies which could be integrated into their processes and optimize their bioprocess design space.
Life Technologies Corporation
Life Technologies is a global biotechnology tools company formed in 2008 through the merger of Invitrogen and Applied Biosystems. The company supplies instrumentation, consumables and services to laboratories working across the biological spectrum. By focusing on supplying innovative technologies that drive the advancement of medicine, molecular diagnostics, environmental research, and forensics, Life Technologies hopes to improve the human condition.
Currently, Life Technologies is one of the leading suppliers of DNA testing systems to forensic laboratories. To maintain market leader status in the forensics and human identification market, the company is interested in expanding its product line to include rapid integrative DNA processing systems. This new technology gives personnel with limited training the ability to generate DNA profiles outside of a centralized laboratory. Although there are many theoretical benefits to decentralized rapid DNA profiling, the market need for this new technology has not been verified.
The Keck Graduate Institute’s Team Master’s Project (TMP) team was sponsored by Life Technologies to validate the needs of the potential market for rapid and decentralized DNA testing. To accomplish this objective, the TMP team investigated the current forensic DNA testing market and was able to identify key challenges with the existing workflow that rapid DNA profiling could address. Additionally, the team examined the forensic chain of custody for DNA profile generation and utilization in the justice system and identified the challenges to adoption of decentralized rapid DNA profiling. Finally, recommendations regarding the potential viability of the rapid and decentralized DNA testing market were made to Life Technologies to assist with the business decision to pursue or abandon this market.
Life Technologies Corporation
Life Technologies Corporation is a global biotechnology tools company serving over 75,000 customers in more than 160 countries worldwide. Through constant innovation, Life Technologies is dedicated to improving the human condition. The company prides itself on consistently being at the forefront of scientific discovery, with novel technology development and commercialization. It has over 10,000 employees, approximately 50,000 products and over 4,000 patents and exclusive licenses worldwide.
Through the Applied Biosystems® and Ion Torrent™ brands, Life Technologies provides innovative equipment to labs around the world. Life Technologies also provides a broad range of consumables through world renowned brands including Invitrogen™, GIBCO®, TaqMan®, Novex®, Molecular Probes®, and Ambion ®. Life Technologies commits itself to providing products that enable lab functions in the basic, applied, and medical sciences. It is currently interested in expanding its market within the applied sciences sector, and wishes to learn about opportunities in water testing.
Water is an invaluable natural resource that is vital to all living beings. Proper handling, monitoring, treating and conservation are critical to protecting this essential resource. Water testing technologies are necessary to ensure safety of water usage and release into the environment. A variety of consumers with different needs makes up the water testing marketplace. They include private users, specialized laboratories, government agencies, utilities companies and industrial manufacturers. Life Technologies’ overarching goal for the team is to gain an understanding of the unmet needs of these consumers.
The team conducted primary and secondary market research to examine the water testing marketplace. Interviews with users of water testing products from different industries and organizations were found to be particularly valuable. The team also learned about regulatory guidelines in specific areas of interest. The information from these efforts allowed the team to perform a market segmentation analysis and identify unmet needs and opportunities. Equipped with an understanding of the unmet needs for different water testing market segments, the team provided Life Technologies with recommendations on which markets to target and how existing Life Technologies products and technologies could be leveraged to meet market needs.
Meditope Biosciences, Inc.
Meditope Biosciences is an early stage biotechnology company with a unique technology that can add molecules such as therapeutics or imaging agents onto monoclonal antibodies (mAbs) that allows specificity, control, and no changes to the antibody’s binding capacity. The company holds an exclusive global license to this platform technology which was developed at Beckman Research Institute of City of Hope. Often characterized as a ‘LEGO-like’ system, this technology has the potential for a broad set of applications such as: new 2nd generation antibody technologies (e.g., antibody-drug conjugates (ADCs) and bispecific antibodies), enhanced imaging agents for PET, and other technologies to allow more specific detection of cancers and other diseases. The precision and control afforded by Meditope’s technology in comparison to alternatives is expected to lead to enhanced efficacy and lower toxicity therapeutics, which are much needed in cancer treatment.
Meditope is interested in developing an ADC cancer therapeutic partnership with a company that has an existing mAb in either clinical trials or on the market. In doing so, Meditope’s main objective is to identify a lead candidate that will ensure speed to market, commercially attractive partnering opportunities, and capability of highlighting broader application of its technology optimally.
The Meditope TMP team was asked to evaluate the current universe of oncology mAbs in order to determine the top candidates for ADC development by Meditope, taking into account commercial potential, accelerated time to market, likelihood of partnership success, and features/capabilities of the Meditope technology. The team started by researching the current landscape of mAbs and formed weighted criteria through careful consideration of commercial, regulatory, and technical factors. These criteria shaped the creation of a scoring system used to filter the mAb universe yielding the top candidates through an iterative process. Each top candidate was assessed further to produce a small list of high priority partnering opportunities and strategies, including lead mAbs, clinical indications, and recommended regulatory path. Ultimately, the team provided Meditope with a comprehensive report on recommended partnering candidates and strategies as well as an adaptable selection tool for additional screening and evaluation.
Monsanto is a multinational agriculture and biotech company committed to meeting the needs of today while preserving the planet for tomorrow. It is the leading producer of genetically engineered seeds and herbicide, which is sold under the brand name Roundup. Monsanto is the largest American producer of corn, which is mainly used in animal feed and to produce high fructose corn syrup. Monsanto, which belongs to the materials sector and the agricultural chemical industry, actively pursues novel process enhancements to support their Biotechnology and Breeding pipelines for rapidly changing technologies in the agriculture industry.
The purpose of this project is to explore innovative seed treatment technologies to keep pace with evolving chemistries and needs. Existing technologies and processes for seed treatment are becoming outmoded with advancements in chemistries and in-field performance requirements. The team investigated novel seed treatment methods which demonstrate improvements over conventional processes in terms of delivery mechanisms and efficacy. The scope of this TMP is to deliver a prototype capable of producing coated seeds using various novel delivery methods for studying coating efficiency and efficacy. Additionally, the team has developed methods for studying the dynamics of liquid seed coating by using high-speed videos and supporting analytical techniques for generating delivery performance metrics. This will help Monsanto better understand the relevant fluid properties and physics to improve current coating systems.
The Monsanto TMP team designed and built a functional prototype to transport seeds and integrated the prototype with a seed formulation dispersal system. The prototype was tested and optimized to insure that it accomplished the project objectives. In addition to the testing process, the team successfully captured video of liquid droplets impacting seeds at 16,000 frames per second. With the ability to create these highspeed videos, the team investigated the dependency of fluid-seed surface interactions to dimensionless variables, including the Reynolds number and the Weber number. Finally, performance metrics were devised by the team to compare Monsanto TMP coating efficacy with efficacy studies performed at Monsanto internal trials.
Proteus Digital Health, Inc.
Proteus Digital Health is a pioneer in digital healthcare. By creating medical devices that communicate with digital technology, such as smart phones, tablets, and cloud computing, Proteus enables people to develop and sustain healthy habits and businesses to develop new information-based business models. Founded in 2003, Proteus’ flagship technology revolves around wearable and ingestible sensors. In July 2012, Proteus gained FDA clearance of their ingestible sensor that is powered by stomach fluid. Sensor-enabled pills communicate when they have been ingested by transmitting a signal to an adhesive, wearable sensor patch that also collects physiologic data such as heart rate, skin temperature, and physical activity. Data is transferred via Bluetooth to a paired device and uploaded to the Cloud. Users can then share their data with family, caregivers, and/or clinicians to build a community of individuals that help each other stay healthy.
The patch system was conceived to serve the elderly or chronically ill; however, Proteus’ long-term mission is to provide a range of lifestyle tools that serve individuals of all ages and stages of health. Proteus wanted to evaluate the potential uses of the patch in a competitive athletics environment.
The Proteus TMP team was tasked with identifying how the patch might be used in this new market and what user requirements needed to be changed to target this new audience. To accomplish this objective, the team met with coaches and athletic trainers from many different sports and conducted extensive research to evaluate the current state of athletic monitoring and identify ways the Proteus patch could address an unmet need. During this initial phase, the team identified swimming as a unique point of entry into the larger athletics market. The team then focused the remaining stages of the project on producing a working prototype of the patch and user interface for use as a training tool for swimmers and coaches. Through testing of the device on athletes from the Claremont-Mudd-Scripps swim team and garnering feedback from swim coaches, the Proteus TMP team will make recommendations regarding the features and marketability of the potential commercial product.
Questcor Pharmaceuticals, Inc.
Questcor Pharmaceuticals, Inc. is a biopharmaceutical company, which focuses on difficult-to-treat and severe autoimmune and inflammatory disorders. The company was formed in 1999 from the merger of Cypros Pharmaceuticals and RiboGene Inc. and has two main products; H.P. Achthar® Gel and Doral®. With current strategies in place, Questcor Pharmaceuticals is growing rapidly and the company posted net revenues of $268 million in 2011.
Questcor would like to investigate new market opportunities for various indications within the critical care setting. Additionally, the company would like to evaluate the management of pharmaceuticals within this critical care setting to more fully understand the challenges found in this marketplace.
The Questcor TMP team was tasked with performing a market assessment which included the identification of current unmet clinical needs for a number of clinical indications. Ultimately, the team identified high value market opportunities for consideration in Questcor’s strategic market development. To accomplish these goals, the Questcor Pharmaceuticals TMP team carried out extensive secondary research including interviews with Key Opinion Leaders (KOLs) in various specialties. Finally, the team produced a comprehensive case study analysis to aid in corporate planning.
Regeneron Pharmaceuticals, Inc.
Regeneron Pharmaceuticals, Inc. is a fully integrated biopharmaceutical company headquartered in Tarrytown, New York. Founded in 1988, Regeneron presently has three marketed products: EYLEA® (aflibercept), ZALTRAP® (ziv-aflibercept), and ARCALYST® (rilonacept). In addition to these marketed products, Regeneron has 13 product candidates in clinical development.
In an effort to reduce the risk of viral and mycoplasma contamination of current and future products, Regeneron has initiated efforts to bring viral barriers and testing methods into upstream cell culture processes. Various point-of-use viral barriers were investigated and Regeneron has decided to move forward with the implementation of High Temperature-Short Time (HTST) treatment of media and feeds in upstream cell culture processes. However, throughout these initial studies, many uncertainties have arisen regarding HTST implementation, particularly for legacy-approved processes.
This one-semester Team Master’s Project was commissioned to survey and benchmark the biopharmaceutical industry to learn more about how leading companies are addressing viral barriers and what, if any, hurdles they have experienced in implementation. Our study surveyed nine leading biopharmaceutical companies, as well as six industry-leading consultants, who were able to share insights into the regulatory landscape and potential hurdles, scalability challenges, best practices for viral inactivation and validation studies, as well as the impact to cost of goods. Results were compiled and shared in a white paper that will serve as a guide for viral risk mitigation efforts, at both established and start-up biopharmaceutical companies
Terumo BCT, Inc.
In 2011, CaridianBCT and Terumo Transfusion merged to create Terumo BCT, a company that has become a global leader in cellular and blood component technologies. It is the only company that has the unique combination of apheresis collections, manual and automated whole blood processing, and pathogen reduction coupled with leading technologies in therapeutic apheresis and cell processing. With commercial operations in over 120 countries and a robust product pipeline, Terumo BCT is a key player in the blood industry, continuing to unlock the potential of blood to benefit patients even more than it does today.
Terumo BCT has developed the Mirasol® System, a pathogen reduction technology (PRT) capable of inactivating leukocytes and pathogens in blood products. It has received the CE-mark in Europe and the company is currently seeking approval of the technology in the US. With the military’s interest to use Mirasol treated whole blood in combat and trauma settings, Terumo BCT would like to evaluate the feasibility of Mirasol-treated whole blood for civilian applications. Granted that no PRT is approved in the US and the blood supply is considered very safe, Terumo BCT would also like to understand the requirements, barriers and the economic impact of introducing a new medical device in the US.
To accomplish these objectives, the Terumo BCT TMP team performed a two-phase analysis to characterize the market opportunities for the Mirasol System. The first phase involved a thorough evaluation of the U.S whole blood transfusion market, including an analysis of market size and attractiveness for the Mirasol System. In parallel, the team conducted interviews with key opinion leaders and industry experts. The combined research results provided a foundation for a market survey which provided critical data for the next part of the project. During the second phase, additional rounds of surveys and key opinion leader interviews were completed to generate a market definition report citing the team’s strategic recommendations. Hence, the team provided Terumo BCT with actionable information for future marketing activities and strategic planning in support of the Mirasol System.
US Script, Inc.
Corporation in 2006, US Script, Inc. is a full-service Pharmacy Benefit Manager (PBM) that provides a comprehensive suite of cost-effective prescription drug benefit services to its members. US Script serves over 3 million members with 2.5 million Medicaid members and the remainder from behavioral health programs, third party administrators, labor unions and self-funded employers. US Script is headquartered in Fresno, CA with additional offices in St. Louis, MO; Tempe, AZ; Ft. Worth, TX; Texarkana, TX; and Atlanta, GA. US Script provides customized services to clients through claims processing, flexible plan design, pharmacy network management, formulary and rebate management, clinical programs, reporting, 24/7/365 customer service support, specialty and mail order pharmacy, and data integration services.
The PBM marketplace has become highly commoditized with competitors developing technical solutions to improve customer service. US Script is looking to develop a strategy to become the leading innovator of PBM self-service technologies that will revolutionize patient empowerment, decrease prescription costs, and ultimately create value for their clients. The US Script TMP was divided into two phases: (I) surveying the industry landscape and (II) recommending a strategy to deploy a novel self-service technology platform.
The US Script TMP team began Phase I by performing a comprehensive analysis of competitor technologies. The team then identified emerging trends in the PBM and healthcare sectors by attending industry conferences, interviewing key stakeholders, and monitoring social media and industry publications. The results of Phase I were compiled to guide the development of a marketing strategy for self-service technology deployment in Phase II. Finally, the team interviewed potential internal and external partners to provide a final recommendation and process for future execution.
Veracyte, Inc., (San Francisco, CA) is a privately held molecular diagnostics company pioneering the emerging field of molecular cytology. The company discovers, develops and commercializes molecular diagnostic solutions that enable physicians to make more informed early treatment decisions, thus helping patients avoid unnecessary invasive procedures while reducing healthcare costs. Veracyte’s first product – the Afirma® Thyroid FNA Analysis – combines specialized cytopathology assessment with the Afirma® Gene Expression Classifier, a genomic test that clarifies inconclusive thyroid nodule results as benign or suspicious for cancer.
Veracyte, Inc., is currently focusing on opportunities to add to their product pipeline of minimally invasive diagnostic tests for newer indications. The TMP team at KGI is focusing on validating the unmet need in the early diagnosis of one of these indications, by assessing the market potential for a novel minimally invasive diagnostic technology. An early diagnostic test enables doctors to make more informed treatment decisions thereby improving the quality of patient care which, in turn, leads to costs savings for the healthcare system.
The team performed an extensive literature review and conducted interviews with Key Opinion Leaders (KOLs) to gather data on general trends and practices in the diagnosis and treatment of the study indication. This work allowed the team to determine the patient population served and the location where the diagnostic test would be most efficiently used. The team also designed expanded surveys to gather larger data sets in an effort to understand patient dynamics for the specified indication. From the literature and initial interviews, the team identified a potential market opportunity for a minimally invasive early diagnostic test for the study indication. Finally, additional interviews and results from expanded surveys were performed to validate results obtained by the literature review.
Veracyte Inc. is a company based in San Francisco founded in 2008 with a vision to transform molecular cytology. The company is committed to enable physicians to improve the quality of patient care and generate savings to the healthcare system. Veracyte develops molecular tests for oncology, for example related to thyroid cancer, that are designed to clarify ambiguous cytology results.
According to the American Cancer Society, there are approximately 60,220 new cases of thyroid cancer annually. The diagnoses entail cytopathology of samples obtained via Fine Needle Aspiration (FNA) from the thyroid. It is estimated that 450,000 FNA samples are collected in the United States, of which 100,000 yield indeterminate results and most are referred for thyroid surgery at a cost of $12,000 per procedure. However, approximately 70% of post-surgical pathology tests suggest nodules are actually benign and improperly diagnosed. The Veracyte Afirma® FNA Analysis addresses this unmet need, by enabling these indeterminate results to be re-classified preoperatively, thereby reducing the number of unnecessary surgeries. The Afirma® FNA Analysis combines specialized cytopathology and the novel Afirma® Gene Expression Classifier (GEC) test, which entails microarray based quantification of mRNA extracted from the sample. Both approaches, however, require FNA samples with a sufficient number of thyroid follicular cells, and a sufficient amount of extractable RNA. Inadequate sampling for the cytopathology and Afirma® GEC test occurs in a small number of cases, causing a “no result” to be reported. This often results in a repeat of the FNA procedure, with substantial time delay, negatively impacting the patients. Therefore, it would be ideal for the adequacy of the sample acquisition to be determined immediately at the point-of-care.
The Veracyte II TMP team was tasked to investigate technologies that could be used to determine, at the time of FNA sample acquisition, whether a sufficient number of thyroid cells have been collected for the cytopathology and GEC tests. To accomplish this objective, the Veracyte II TMP: is clarifying the requirements for this Sample Adequacy (SA) test; is evaluating the suitability of different biomarkers for FNA SA testing; and, is gathering information on technologies that can appropriately detect these biomarkers with suitable analytical and clinical performance in the targeted use settings. Finally, upon consolidating the data from literature research and feedback obtained from key opinion leaders, the team will recommend the most suitable approaches or combinations of technologies.
Jingdong Yumei Kidney Disease Hospital
The Zhongmei healthcare group is a multi-facility organization within several cities throughout China, and includes medical schools, anti-aging clinics, and specialty hospitals. One such hospital is the Yumei Kidney Disease Hospital in Yanjiao, Hebei, China. This hospital explores the interface of Western therapies with traditional Chinese medicine, and is exploring new therapies in the kidney disease sector. Kidney disease affects millions of people globally, and can also be linked to several ailments including diabetes and autoimmune disorders. Current therapeutic options either involve invasive treatments to filter the blood (dialysis), supplements to slow the progression, or full kidney replacement surgery. While each of these improves the quality of life, it is often a short-lived improvement. Many researchers and companies are investigating the potential of stem cells and tissue engineering for regenerative therapies in the treatment of renal failure.
The goal of KGI’s Jingdong Yumei Kidney Disease Hospital TMP was to work with the liaisons and investigate research groups and companies exploring kidney repair through reconstructive methods. The TMP then evaluated these technologies according to Yumei’s strategic and therapeutic goals. Ultimately the TMP team made a prioritized list of potential technologies and facilitated communication with the final group of investigators responsible for the technologies of interest. To accomplish these tasks, the team began their efforts by building a portfolio of innovative kidney disease technologies that were in development or approaching market approval. By attending conferences for nephrology and discussing new advances in the nephrology with KOL’s, the most interesting list of technologies was narrowed to a final list of potential collaborative opportunities. Through additional evaluation, as well as communication with inventors and investigators, contacts were then established to facilitate potential partnerships with the Jingdong Yumei Kidney Disease Hospital. Progress so far has suggested that there are numerous opportunities within the United States to develop a regenerative approach for the treatment of kidney disease.
As a result of this TMP, the Jingdong Yumei Kidney Disease Hospital in China made several connections to different institutions exploring regenerative medicine for kidney disease. Thus, our efforts have provided the opportunity for collaborative research or licensing agreements between US research groups or US companies and the Jingdong Yumei Kidney Disease Hospital.