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Adventures in Biotechnology

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Presentation on theme: "Adventures in Biotechnology"— Presentation transcript:

1 Adventures in Biotechnology
Boro Dropulic PhD, MBA

2 Overview Background Decision to Start a Biotech
Biotech Business Models Financing of Biotech Companies Modes of Productivity & Relationship to Business Models The relationship between Business Models and Investment Case Study: Lentigen Business Presentation

3 Background PhD in Australia (University of Western Australia)
Fogarty Fellow at NIH Bld 10 then Bld 4 Embryonic Stem Cells Gene Therapy Johns Hopkins University HIV vectors targeted to HIV infected cells (new IP) VIRxSYS First group to initiate and complete Lentiviral vector clinical trial (gene therapy for HIV infection) MBA (Johns Hopkins University) Lentigen Broad business Model

4 Starting a Biotech Company
Defining the technology Can the technology be used for multiple uses? Licensing strategy vs starting a biotech company If decision to start a company then: What are the objectives? Sell the company to large biotech or pharma Grow the company and license out products Organization and focus of the company Single product focus  short term exit strategy Multiple products  long term exit strategy

5 Therapeutic Biotech Business Model

6 Milestone driven value - technology

7 Financing of Biotech

8 Financing of Biotech OR
Derive revenue streams from the sale of product or services (research tools products & services) Smaller market potential - more difficult to attract investors - revenue streams are much smaller than the therapeutic or vaccine markets - requires organic growth strategies

9 The Very High Risk Nature of the Conventional Biotech Business Model
High risk = high failure rate Especially true for biotech focused upon development of biologics Failure not always related to science Investor understanding of the technology and product development cycle time is very important Financing strategy must match product development cycle time

10 Potential Saving Graces
Government Grants (SBIR etc) Private Foundation Grants Contracts (Government or Private/Foundation) Out-license portions of the technology Other financing models

11 Chimeric Business Models for High Risk Technologies
Long Lead Times to Therapeutic Markets Gene Therapy Cellular Therapies Stem Cell Therapy Vaccines (viral vector) RNAi (viral vector shRNAi) Therapeutics Therapeutics Validation / Growth Service / Research Products Service / Research Products Decreased Risk vs. Lack of Focus

12 Models of productivity
Geoff Race CFO Pangenetics Index Forum 2007 Stresa, Italy “Young Genius” “Old Master” Femme à la resille (Woman in a hairnet), 1938, by Pablo Picasso. Still Life with Carafe, Sugar Bowl, Bottle, Pomegranates, and Watermelon, 1906, by Paul Cezanne value Paintings most valuable = early work Paintings most valuable = late-in-life work

13 Comparison of Productivity Models
“Young Genius” Innovation Non-linear; concept Short term horizon Rapid, immediate Low # of objectives Single focus Singularity of Purpose Virtual; simplicity High Risk No track record “Old Master” Experimentation Process driven Long term view Quality mindset Higher # of goals Competing priorities Complexity/ Integrated infrastructure Low Risk Track record

14 Comparison of Business Models
Single product Biotech Company Innovation Short term horizon Low # of objectives Singularity of Purpose High Risk Concept Based Value Growth Multiple product Biotech Company Experimentation Long term view Higher # of goals Complexity/ Integrated Low Risk Linear Value Growth

15 Comparison of Business Models
Single Product Biotech Innovation Short term horizon Low # of objectives Singularity of Purpose Multiple Phases High Risk Concept Based Value Growth ACQUIRED Multi Product Biotech Experimentation Long term view Higher # of goals Complexity/ Integrated Life’s Work Low Risk Linear Value Growth IPO - ALLIANCES BOTH ARE VALID GROWTH STRATEGIES

16 Growth Strategy Needs to Match Investor Strategy
Investor Strategies Large number of singly focused companies vs few technological platform companies Shorter exit time for singly focused companies, but high risk Longer exit time for technological platform companies, but lower risk Investor Sophistication Important Choice of Investor Critical for ultimate success

17 Company Presentation January 2008

18 Safe Harbor and Confidentiality
The following information is confidential and should not be distributed without the permission of Lentigen Corporation. This presentation contains certain forward looking statements based on Lentigen’s current beliefs and expectations about future events. These statements are subject to numerous risks, uncertainties and assumptions and are not necessarily predictive of future results. Actual results may differ materially from those anticipated in this presentation depending upon a variety of factors including development of manufacturing procedures, market size and revenues, potential competition, regulatory compliance, operating effectiveness and other factors that may not be currently considered as material.

19 Summary Lentiviral Vector (LV) gene delivery technology
Four phase I/II clinical trials in 2008/9 Eight NIH/DOD grants supporting future therapeutic pipeline Alliance with ThermoFisher - LV RNAi research tools Dominant Intellectual Property Position Experienced management team and board of directors Upcoming Milestones

20 Lentigen Overview Established 2004 Location: Employees: 40
Gaithersburg, Maryland 2008Q1 25,000 ft2; multi-product GMP facilities Employees: 40 Technology platform focus Lentiviral vector mediated gene delivery Areas of application Vaccines & Therapeutics Research and Drug Discovery Tools

21 Lentiviral vector (LV) gene delivery
Most efficient, stable gene delivery system known LentiMax™ LV system High titer, low toxicity Scalable manufacturing process Bench to clinic translatability

22 Efficient & stable gene delivery with LV-GFP (Green Fluorescent Protein)
Cells before addition of LV-GFP 98.7% Cells after gene delivery with LV-GFP Human kidney cells

23 Vaccine & Therapeutic Pipeline

24 Glioblastoma 18,000 per year (USA) cases with comparable market in Europe 50% malignant with 40 week survival Market size estimate - $300 million+ Easily addressable patient population Patients concentrated at surgical centers Limited sales force Targeted physicians No good current treatment available Treatments limited by toxicity Temezolomide (TMZ) effectively treats disease but prolonged TMZ treatment results in severe lymphopenia and thrombocytopenia  death

25 Glioblastoma therapy using LV-MGMT
Protection of stem cells from TMZ toxicity  longer TMZ treatment  better outcomes for patients LV-MGMT can protect stem cells from effects of TMZ Proof of concept shown in dogs and monkeys Clinical trial endpoints are short – 3 weeks CWRU (Dr S. Gerson) Broadly applicable CML, AML, HIV etc IP exclusively licensed TEMEZOLOMIDE LV MGMT KILL TUMOR PROTECT STEM CELLS STEM CELLS Bone Marrow Transplantation

26 Graft versus Host Disease (GVHD)
In 2005: 7,250 allogenic transplants in USA and 10,000 worldwide 50% develop chronic GvHD Market size estimate - $300M to $500M Potential for huge savings to healthcare system Easily addressable patient population Patients concentrated at transplant centers Limited sales force Targeted physicians No good current treatment available

27 GVHD therapy using LV-TMPK
Two technological advances LV efficient gene delivery TMPK human safety gene Proof of concept shown in primary human T cells Short clinical trial endpoints 3 weeks to 3 months CRADA with NIH; 2 sites; Drs Gress, Fowler, others Broadly applicable to other diseases – cancer, HIV etc IP exclusively licensed LV TMPK T CELLS DLI + BMT AZT STEM CELLS RECIPIENT IF GVHD THEN + AZT KILL ALLO DONOR

28 Pandemic & Seasonal Influenza
Influenza infects 10-20% of the population every year and causes 500,000 deaths Approximately 300 million doses produced globally Market Size = $1B in 2004, rapidly expanding market Need for vaccine MFG efficiencies Egg manufacturing not cost effective Cell culture with L.A. virus is limiting Need for development of a rapid and efficient cell culture based vaccine manufacturing platform

29 LV-IFVX Influenza VLP vaccine
Synthesized gene cloned into LV 1 week Rapid manufacturing platform weeks not months MFG time Virus Like Particles (VLP) highly immunogenic Exact strain match no genetic drift during production Continuous production of VLP advantage over batch production Safe VLPs are genetically inactive IP protected LV manufacture and QC testing 1 week LV transduction 1 day Scale Up cells 3 weeks Vaccine Purification 1 day

30 Potent Immune Response in Mice
0.000 0.200 0.400 0.600 0.800 1.5 2 2.5 3 3.5 4 4.5 5 Log Serum dilution Absorbance Vaccine only Vaccine with adjuvant Prebleeds Strong immune response generated in mice against Lentigen’s H5N1 VLP vaccine (blue) and vaccine with adjuvant (purple); pre-bleed controls (red)

31 Biogenerics & Proprietary Biologics
Biologics that are coming off-patent are worth >$30B Erythropoietin, cytokines, coagulation factors, antigens, McAbs COGS critically important for competing in the protein MFG markets Efficiencies gained by decreasing time to market Efficiencies gained by increasing yield from cell lines LVs can reduce time to market and decrease COGS High copy numbers in cells without gene amplification Integration in areas of open chromatin  higher expression

32 Erythropoietin production
Gold Standard Efficient Bulk CHO Cells > 2.5 g/L No cell cloning No selection No enhancement of cell growth conditions Levels of 5 – 10+ g/L attainable with isolation of cell clones

33 Research & Drug Discovery Tools Pipeline

34 SMARTvector RNAi: Effective gene silencing
GAPDH knockdown 9 days post-transduction with SmartVector shRNAi Lentiviral vector particles SMARTvector Control SMARTvector GAPDH RNAi SHSY5Y neuronal cells Fixed and stained with GAPDH antibody (red) and Hoechst (blue)

35 Effective gene silencing in a wide variety of cells
Stem cells Neuronal cells Suspension cells Suspension cells 75% Difficult-to-Transfect

36 Intellectual Property
Dominant patent estate for Lentiviral vectors Lentigen (GBP IP) has acquired the entire Cell Genesys Lentiviral vector IP portfolio Non-provisional patent coverage of: Novel LV compositions and methods Use of LVs for Influenza VLP manufacturing Use of Novel LVs for protein manufacturing Lentigen has exclusively in-licensed several key payload technologies TMPK LentiSafe™ technology MGMT LentiSelect™ technology Lentigen continues to develop and in-license additional key proprietary intellectual property

37 Grants Seven (7) SBIR Grants from National Institutes of Health
Leading Clinical Centers & Investigators Oncology Infectious Diseases STTR Grant from Department of Defense Efficient Manufacture of Monoclonal Antibodies in the National Stockpile MIPS Grant Development of new animal models for cancer

38 Experienced Management
Boro Dropulic, PhD, MBA - Founder and CEO 19 years experience Founder & CSO of VIRxSYS, Johns Hopkins University, NIH Sponsor Investigator of first Lentiviral Vector Clinical trial in humans Adam Sachs, MSE – President, Commercial Operations 23 years experience Life Technologies (now Invitrogen), Qiagen, Origene Global Director for Manufacturing at Qiagen Gregory Feulner, PhD, JD – VP, Business Development NIH, Johns Hopkins, Gene Logic Inc., Galileo Genomics, 3M Yung-Nien Chang, PhD – VP, Vector Development 22 years of experience Johns Hopkins University, VIRxSYS, NIH, GTI (now Novartis)

39 Board of Directors David Wetherell – Chairman of the Board
Former Chairman, CMGI, Founder, Greenwich Biotech Ventures Boro Dropulic, PhD, MBA Douglas Lind, MD Managing Partner of Accendx Management LLC Former Senior Biotech. Equity Analyst at Morgan Stanley & PaineWebber John McMullen, JD, MBA Managing Principal of Cambridge Meridian Group Republican Nominee for Senate, VT (2004) Robert Breyer Former President/COO of Alkermes Former President and General Manager of Eli Lilly Italy Barrie Carter, PhD EVP and CSO of Targeted Genetics Former Chief of the Lab. of Mol. & Cell Biology at the NIH

40 Upcoming Milestones Initiate phase I/II clinical trials for GVHD & Glioblastoma Complete pre-clinical studies for Influenza vaccine and Erythropoietin protein product Continue development of pipeline for therapeutic & vaccine products, leveraging grant funding mechanisms Expand alliance with ThermoFisher and other partners Certify Lentigen’s new GMP manufacturing facilities Complete additional financing


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