Companion diagnostic –A diagnostic test used to predict the likely clinical effectiveness and/or safety of a particular therapeutic intervention for a specific individual; the term is most often used to describe a molecular diagnostic test that stratifies a patient population with regard to the likelihood of response to, or the safety of, a pharmacologic therapy.Biomarker discovery for applications in drug discovery, preclinical studies of drug development and diagnostics research applications was the largest and accounted for nearly 48% of the total marketing in 2007. This market is expected to grow at a CAGR of 16.9% to reach nearly $6b by 2012, somewhat vindicating companies such as Vermillion (formerly Ciphergen) turning their back on instrumentation sales to focus on discovery programmes. The second biggest segment was found to be the use of biomarkers on molecular diagnostics applications which was estimated to be worth $2.3b in 2007. The report predicts this market will grow at a CAGR of 17.5% to be worth $5.2B by 2012. This market will provide the point-of-care and laboratory based diagnostic tools that enable a more informed ability to choose the right drug for a disease and patient.While currently the smallest business segment, having been valued at around $612M in 2007, the clinical trials market has been predicted to see the largest growth rate with a CAGR of 23.5%. This growth would see the market for biomarkers in clincial trials reach $1.8b by 2012, and has already been spotted by many CROs with Covance having luanched a biomarker expert team earlier this year.The report suggests that the use of biomarkers to aid the discovery, testing and prescription of oncology therapeutics will be one of the biggest application areas. Applications in the cardiovascular and centralnervous system areas are expected to grow during the next 7 years. Advances in technology that enable faster metabolomic, proteomic and genomic data acquisition as well as its integration with clinical trial data using bioinformatics software will all be major driving foces behind the growth of this burgeoning area.
From Wikipedia:The following are some of those functions:Glycoproteins found on the cell surface play a critical role in bacterial and viral recognition.They are involved in cellular signaling pathways and modulate cell function.They are important in innate immunity.They determine cancer development.They orchestrate the cellular fate, inhibit proliferation, regulate circulation and invasion.They affect the stability and folding of proteins.They affect the pathway and fate of glycoproteins.There are many glycan-specific diseases, often hereditary diseases.There are important medical applications of aspects of glycomics:Lectins fractionate cells to avoid graft-versus-host disease in stem celltransplantation.Activation and expansion of cytolyticCD8T cells in cancer treatment.Glycomics is particularly important in microbiology because glycans play diverse roles in bacterial physiology. Research in bacterial glycomics could lead to the development of:novel drugsbioactive glycansglycoconjugate vaccinesSeparation:BlotGlyco®—Ezose Sciences employs Glycoblotting technology, which is faster and easier than conventional methods. This rapid chemoselective process features an “all-in-one” bead-based platform technology that enriches oligosaccharides from crude mixture, enabling highly selective and high-throughput (profiling of 30–40 kinds of major glycoforms) purification of oligosaccharide. Only a small sample is needed for a full analysis. Results are proven comparable to standard methods.SweetBlot® Automated ProcessingSweetBlot®—Ezose Sciences’ SweetBlot technology provides for fully automated, high-throughput glycan sample processing, thus reducing operator error and standardizing results. (Current capacity of ~180 samples/day, and scalable)MALDI-MS Quantitative DetectionQuantitative MALDI-MS—Ezose Sciences uses a unique protocol in combination with MALDI mass spectrometry that enables sample identification and quantitation with reproducibility comparable to HPLC, yet at a far greater throughput. Both neutral and acidic oligosaccharides are simultaneously quantified.GlycanMap® ReportA detailed glycosylation profile that can be employed to achieve a variety of endpoints related to process development, formulation, stability, quality control and biomarker discovery.
OfferingUses its patented GlycanMap technology to provide high-throughput testing for clientsTesting of samples previously took days or even weeksEzose’s technology allows for a complete test to be done in a day
I would just summarize the statement as saying, 50% increase in contracts by leveraging their unique technologyAcceptance of more Fee-for-Service (Cost + 20%) contractsReturn business = future royalties/IP rights
Glycomics is a young, developing field.
Customer TypePharmaceutical and Biotech CompaniesRequest glycan mapping and/or biomarker analysis to assist their own preclinical/clinical researchMay use Ezose’s service to produce commercially viable therapeutic, diagnostic, or companion diagnostic productswould prefer to pay, as per industry standard, a fee-for-service of cost plus 20%may offer royalties or intellectual property rights for applications developed from the research
A purchase of a third GlycanMAP instrument by the end of 2012Increased research capacitySelf-sustainabilityStrategic Alliance:Being small can have its advantages – it offers flexibility to respond to changing market trends faster than larger competitors can and can also make for more personalized relations with the customer. However, being small can also have its limitations. One of the smaller CROs, Pacific Biomarkers, with a similar business model as Ezose, realized that clinical trial services are increasingly required to be global and that services offered by a single location often cannot meet sponsors’ turnaround time requirements, may be cost-prohibitive (due to shipping costs) or may not accommodate sample stability restrictions. Because of this, they teamed up with Quintiles Laboratories, one of the larger CROs, to match their core capabilities and expertise with the global logistics offered by Quintiles to successfully support large, multinational clinical trials (Ehlers). While Ezose does not need to form a strategic alliance with a larger company in the current environment, if the threat of industry consolidation and the dominance of larger CROs make survival on its own impossible, Ezose should look to partner with a company similar to Quintiles, such as Covance, PPD or Charles River.
Sense of urgency:Companies are currently in the market for Biomarker discoveryPharmaceutical and biotech companies are consolidating the number of firms they outsource work to
Ezose Sciences Inc. Final Presentation December 14th, 2011 1 Privileged and Confidential
Company Overview JEFF Relatively new company Centrally located in heart of pharmaceutical country Focus on glycomics and biomarker discovery Multiple collaborations completed / in progress Ezose laboratory - Pine Brook, New Jersey 2 Privileged and Confidential
Industry Snapshot JEFF 70,000 Pharmaceutical Industry Job Cuts Ezose: 60,000 Biotech industry 50,000 40,000 Discovery, Research & 30,000 Development market 20,000 Customers: pharma and 10,000 biotech companies 0 00 01 02 03 04 05 06 07 08 09 10 Major trends and drivers: Top 5 Drugs From 2011 and 2012 Coming off of Patent Protection Patent 2010 US Sales Drug Condition Company Expiration ($M) Expense control 2012 Plavix Anti-platelet BMS / Sanofi $6,154 2011 Lipitor Chloresterol Pfizer $5,329 Generic drugs 2012 Seroquel Antipsychotic AstraZeneca $3,747 2012 Actos Type 2 Diabetes Takeda $3,351 Companion diagnostics 2012 Enbrel Arthritis Amgen $3,304 2012 Singulair Asthma Merck $3,224 Partnerships 2011 Zyprexa Antipsychotic Eli Lily $2,496 2011 Levaquin Antibiotics J&J $1,312 Biomarkers 2011 Concerta ADHD/ADD J&J $929 2011 Protonix Antacid Pfizer $690 3 Privileged and Confidential
Industry Snapshot (cont.) JEFF $B Research & Development Market 180 160 140 17.5% 18.8% 15.4% 16.4% 120 14.6% 14.7% 100 80 60 40 20 0 2009 2010 2011 2012 2013 2014 Outsourced R&D In House R&D Growth Rates: R&D Market -1.2% 7.7% 4.3% 3.5% 3.5% 3.5% In-house R&D -2.6% 7.8% 3.2% 2.4% 2.2% 1.9% Outsourced R&D 7.7% 7.2% 10.3% 9.8% 10.6% 11.2% 2009 2014 5 Year Industry Mkt Value Mkt Value CAGR Pharmaceutical $664.6B $860.4B 5.3% Biotech $231.2B $366.7B 9.7% Contract Research $18.1B $28.9B 10.0% Biomarkers $8.4B $21.1B 20.2% 4 Verizon Privileged and Confidential
Biomarkers MATT Predict/prevent disease Predict drug Predict drug response safety Biomarkers Monitor drug Personalized response & safety medicine Rx-Dx combinations & theranostics 5 Verizon Privileged and Confidential
Glycomics MATTWhat is it? Study of sugars of a cell type or organism Involves complex, highly branched structures GlycansBenefits Underdeveloped and $B Global Biomarker Forecast underexplored field 18 16 Play critical role in bacterial and 14 viral recognition 12 10 Determine cancer development 8 6 Involved in cellular signaling 4 2 pathways and modulate cell 0 function Genomics 2010 Imaging Proteomics 2015 Bioinformatics Other 6 Verizon Privileged and Confidential
GlycanMap Technolgoy MATT 7 Verizon Privileged and Confidential
Cost Tech Location Revenue Resources Reputation(aka Industry Standard) Speed/Throughput STRONG Ezo s e Competitors Om ic R Pro &D (pr C ote teom omp om e Sc any ic s : ), T i ence (ge ran s Gl y no sge com mic no s) mi c Vir ic R& dan D C te, o Mo mpan Gl y m y cot enta : ec h , I n- Ho u Ro se B che i g P Di a har gno maVerizon Privileged and Confidential stic R&D s, M : Un erc ive rsi t k yR MATT J oh &D ns Ho : Duk Wa pki eU shi n . ngt s U., , on WEAK U. Go ve r nm ent R FD &D (A A, NIH ir Fo Re ): rce , sea (Un rc h i ve Ho r Cle sity H spita ve os ls Un l and, pi tal ive s rsi t Natio of yH nal os p ita l): 8
Ezose - Strengths and Weaknesses ERICStrengths Weaknesses Technology: GlycanMap allows for Size: limited number of projects high-throughput glycan analysis they can take on due to having only one location, low headcount, and a Small size: flexible; customizable small amount of machines services Reputation: Company is only a Location: centrally located in New couple of years old, is still trying to Jersey, the top pharmaceutical build up its reputation state in the U.S. Inefficiencies: New company, still Staffing: talented and experienced needs to work out their staff inefficiencies 9 Verizon Privileged and Confidential
Opportunities ERIC Plans to Outsource Biomarker Discovery: Within the Year Within a Few Years Not at All Source: Primary Research $B Biomarker Market 35 30 25 20 15 10 5 0 2009 2010 2011 2012 2013 2014 10 Verizon Privileged and Confidential
Business Strategy ERIC Strategic Statement Ezose strives to increase contracts by 50% in 2012, while becoming a self- sufficient company that is recognized as the premiere name in glycomics by 2014. This will be accomplished by leveraging Ezose’s revolutionary proprietary glycan technology to discover and develop new biomarkers quicker and at a lower cost than their competitors, while retaining intellectual property rights for themselves and their parent company Shionogi. Objective: To increase market awareness of its unique services while focusing on internal growth and increasing the number of research contracts 11 Privileged and Confidential
Critical Success Factors ERIC Promote Ezose throughout the biotech and pharmaceutical industry Industry Recognition Do you have a firm understanding of Biomarker Discovery? Do you have a firm understanding of Glycomics? Have you heard of Ezose? If Yes, do you have a firm understanding of what they do? Yes No Industry recognition estimated based off of primary research as well as collection of secondary research Obtain key partnerships with mid/large sized pharmaceutical or biotech companies Expand into a second lab as more partnerships are obtained 12 Privileged and Confidential
Recommendations JEFF Marketing Plan 1. Build upon current actions • Trade Shows, Public Relations Firm, Publications 2. Hire a customer relations management (CRM) team 3. Target specific companies 4. Aggressive pricing Reasons for Outsourcing R&D Cost-effective Lack of In-house capacity Vendor expertise Time savings Regulatory certification Lack of in-house knowledge Quality of results From Science Advisory Board 13 Verizon Privileged and Confidential
Recommendations(cont.) – Target Companies JEFF 14% Biomarker Deal Activity by Therapeutic Focus 12% 10% 8% % of Deals 6% 4% 2% 0% Industry Perception of High- throughput Glycan Analysis Autoimmune / Inflammation Cardiovascular Dermatology Endocrine / Metabolic Systems Gastrointestinal Infectious Disease Neuroscience Oncology Renal Other More Beneficial Less BeneficialTarget companies with focus on Oncology, Autoimmune / Inflammation & Neuroscience 14 Verizon Privileged and Confidential
Summary MATT The Pharma/Biotech Industry is trending in the right direction: Outsourcing of R&D, Biomarkers, Companion Diagnostics, Partnerships GlycanMap technology is revolutionary Must act with a sense of urgency The plan: Increased market awareness Increased revenue through new contracts Increased growth 16 Privileged and Confidential