Your B-School Partner Using Your Business School to Enhance Both Technology Transfer Office and...

Your B-School Partner

Using Your Business School to Enhance Both Technology Transfer Office and

Educational Activities

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Introduction

AGENDA

1.

Overview of Discovery to Market2.

Perspective from partners3.

Sample student project4.

Discovery to Market (D2M)

2010• New MBA curriculum at Carey Business School • Year-long Discovery to Market (D2M) course required • Fundamentals of technology commercialization and entrepreneurship• No science background prerequisite

2011• D2M is launched as a part of the Global MBA curriculum• Builds on core MBA coursework.

2012• D2M is extended to other part time programs and Executive MBA

programs

Learning Model First Term – Boot Camp

Feasibility Analysis When is an idea an invention? Design theory and systemic generation of innovation Intellectual property – legal framework and strategy Market segmentation/sizing Competitive analysis Overcoming regulatory hurdles and barriers to entry Commercialization strategy Funding and financing

• Second Term– Experiential Project

MBA student teams (4-5 students) assigned prescreened inventions from project partner organizations

Inventor meeting Complete feasibility analysis

• Market Feasibility Analysis• Preliminary Recommendation on Go/No-Go

decision• Define Next Steps

Readings

CaseStudies

Team Projects

Learning the Business

Translating the Science

Tuning to the Market

Shared Learning

InteractiveLearningSession

ExpertsBriefing

Classroom Learning

Experiential

Learning

Project Partner Organizations Early-stage scientific innovations are sourced from research community at JHU and outside

organizations.

Johns Hopkins Technology Transfer OfficeJohns Hopkins Department of Medicine

Johns Hopkins School of EngineeringCenter for Bioengineering Innovation and

DesignKennedy Krieger Institute

Telemedicine and Advanced Technology Research Center

National Institute of Health University of Maryland

Local incubators, start up companies

Range of ProjectsMedical diagnostic

Medical devicesHealth ITRobotics

Life science therapiesVaccines

Renewable Energy

Critical Success Factors in Creating an Innovation Ecosystem for D2M

http://www.ncbi.nlm.nih.gov/pubmedhealth/PMH0002302/figure/d19e1387

Outcome Highlights

2010 2011 2012 20130

5

10

15

20

25

30

OthersNIHTATRCJHTTO

Project successTo date, student teams have worked on more than 50 technology commercialization projectsProjects are early stage so long term metrics are not available A number of projects have gained additional funding

Students successfully participated in business case competitionsStudents have gained new appreciation for technology commercialization and technology transfer -Students launched entrepreneurship club -Graduates from the inaugural class have pursued related careers

A standard course syllabus, policy (NDA, COI) and procedures were developed for a successful implementation of the D2M project

D2M curriculum is a part of funding proposal “The PhD Translational Excellerator: Transitioning from the lab to product” with Medical and Education Perspectives (“MEP”), a non-profit organization led by Hopkins Medical and Graduate Schools students to offer technology commercialization course to PhD students

D2M Project Example

With more specificity and less toxicity

ENGINEERED BACTERIAL TOXIN TOTARGET AND KILL CANCER CELLS

THE PROBLEM

Can we kill only cancer cells, sustain insignificant side effects, and leave healthy cells alone?

DeathA decline in heart functionIrregular heartbeatsHigh blood pressureSerious heart attackLung problemsSwelling of the lungs

tumor lysis syndrome (TLS) severe skin and mouth reactionsprogressive multifocal leukoencephalopathy (PML)hepatitis B serious infectionsheart problemslow blood cell counts

DeathA hole in the stomachWounds that don't heal Serious bleedingStroke or heart problemsSeizuresBlindnessKidney problemsInfusion reactionsFertility issuesSevere high blood pressure

Chemotherapies: Anemia, Appetite Changes, Bleeding Problems, Constipation, Diarrhea, Fatigue, Hair Loss, Infection, Memory Changes, Mouth and Throat, Nausea and Vomiting, Nerve Changes, Pain, Sexual and Fertility Changes, Skin and Nail Changes, Swelling , Urination Changes

Sources: http://www.herceptin.com/safety; http://www.rituxan.com/index.html; http://www.avastin.com/patient/lung/side-effects/serious; http://www.tarceva.com/patient/considering/effects.jsp; http://www.cancerresearchuk.org/cancer-help/about-cancer/treatment/cancer-drugs/methotrexate#common; http://www.cancer.gov/cancertopics/coping/chemo-side-effects

AGENDA

BACKGROUND & INTRO

BUSINESS MODEL

STRATEGIC MARKET ANALYSIS

INTELLECTUAL PROPERTY

SUMMARY OF FEASIBILITY RECOMMENDATIONS

THE INVENTION

A protein toxin derived from Bacillus anthracis (Anthrax)

Engineered to be activated by 2 enzymes (MMP and uPA)

MMP and uPA are highly expressed in tumors

In vivo data demonstrates effectiveness in a broad range of cancers & a high therapeutic index (3-8)

Source: T. Bugge, S. Leppla and D. Guerrero. Personal communication.

VALUE PROPOSITION

Invention

High Specificity

Systemic Delivery

Non Mutagenic

Low or no relapse

rates

Few Side Effects

Low Toxicity

Bacillus anthracis

Anthrax Toxin Receptor 1/2

Furin?

Source: Image from www.textbookofbacteriology.net/Anthrax.html

PrAg LF

Protective Antigen

Lethal Factor

Cytotoxicity

MECHANISM OF ANTHRAX TOXIN

MODIFYING THE LF BINDING SITE

Figure Modified from Liu, S. et al. Intermolecular complementation achieves high-specificity tumor targeting by anthrax toxin. Nat Biotechnol. 23 (6), 725-30 (2005).

ASSESSMENT OF NOVELTY AND FREEDOM TO OPERATE

IP HIGHLIGHTS1 U.S. composition patent issued US7,947,289 Expires on 02/09/2024

1 U.S. method of treating diseases patent US8,388,933 Expires on 02/09/2024

12+ related U.S patents by the inventors

Concurrent PCT applications in Africa and Europe

5+ scientific publications in recognized journals

NOVELTY

Altered the enzymatic activation of PA by uPA and MMP instead of furin

Modified formation of LF binding sites within PA heptamer so that only altered PA can bind to LF

Required activation by BOTH uPA and MMP for formation of functional LF binding sites

Over 600,000 hitsBacterial Toxin, Protein Toxin,

Tumor, Cancer

Over 48,000 results Filters: Anthrax, Protective Antigen, Bacillus anthracis

Over 1000 results Filters: uPA MMP

Relevant Patents:

6

IP SEARCH

↓ Immunotoxin therapies

↓ Affinity-targeted conjugates

↓ Vaccines↓ Live cell

approaches

RELEVANT PATENTSPriority Date

Status Patent/App No.

Claim Assignee

04/30/1997 Application PCT/IL2011/000680

Ricin-like toxin variants for treatment of cancer, viral or parasitic infections. Ricin toxin A and B chains linked by a sequence that is cleaved by cancer associated proteases such that cleavage results in activation of the toxin.

Twinstrand Therapeutics

09/24/1999 Patent Issued

US 7468352 Mutated anthrax toxin protective antigen proteins that specifically target cells containing high amount of cell-surface metalloproteinases or plasminogen activator. Mutated anthrax toxin that is activated by cleavage of the cancer associated proteases uPA and MMP.

NIH


US 83889337947289

Multimeric protein toxins to target cells having multiple identifying characteristics. Modified anthrax toxin that kills cells only in the presence of high levels of both uPA and MMP proteases.

NIH

11/21/2005 Application PCT/CA2006/001900

Modified pore-forming protein toxins and use thereof. A pore forming toxin fused to an inhibitory peptide that is cleaved and activated by cancer associated proteases.

Protox Therapeutics Inc.

12/14/2006 Application PCT/US2007/087664

Human cancer therapy using engineered matrix metalloproteinase-activated anthrax lethal toxin that targets tumor vasculature. Use of MMP activated anthrax toxin to target the tumor vasculature.

NIH

04/5/2010 Application PCT/IB2011/051433

Protease-activatable pore-forming polypeptides. A pore forming toxin fused to an inhibitory peptide that is cleaved and activated by cancer associated proteases.

Bar-llan University


Activatable toxin complexes comprising a cleavable inhibitory peptide. Broad claims to concept of protease activated toxins. Specific embodiment are diphtheria and ricin toxins activated by the HCV protease NS3.

Tel Aviv University

RELEVANT PATENTSPriority Date

Status Patent/App No.

Claim Assignee

04/30/1997 Application PCT/IL2011/000680

Ricin-like toxin variants for treatment of cancer, viral or parasitic infections. Ricin toxin A and B chains linked by a sequence that is cleaved by cancer associated proteases such that cleavage results in activation of the toxin.

Twinstrand Therapeutics


US 7468352 Mutated anthrax toxin protective antigen proteins that specifically target cells containing high amount of cell-surface metalloproteinases or plasminogen activator. Mutated anthrax toxin that is activated by cleavage of the cancer associated proteases uPA and MMP.

NIH


US 8388933 Multimeric protein toxins to target cells having multiple identifying characteristics. Modified anthrax toxin that kills cells only in the presence of high levels of both uPA and MMP proteases.

NIH


Modified pore-forming protein toxins and use thereof. A pore forming toxin fused to an inhibitory peptide that is cleaved and activated by cancer associated proteases.

Protox Therapeutics Inc.

12/14/2006 Application PCT/US2007/087664

Human cancer therapy using engineered matrix metalloproteinase-activated anthrax lethal toxin that targets tumor vasculature. Use of MMP activated anthrax toxin to target the tumor vasculature.

NIH

04/5/2010 Application PCT/IB2011/051433

Protease-activatable pore-forming polypeptides. A pore forming toxin fused to an inhibitory peptide that is cleaved and activated by cancer associated proteases.

Bar-llan University


Activatable toxin complexes comprising a cleavable inhibitory peptide. Broad claims to concept of protease activated toxins. Specific embodiments are diphtheria and ricin toxins activated by the HCV protease NS3.

Tel Aviv University

PRIMARY RESEARCHExpert Panel:Robert E. McBride, Esq. Thomas Woods, Esq. Dr. Daniel J. Nevrivy, Esq. Brian Andrea, Esq.

IP Considerations

How do very general claims to use anthrax toxin as a therapeutics impact FTO in other patents?

•Obviousness. Depends on where the claims end up as they are narrowed down. Would be concerned if the prior art says you can mutate in manner A and manner B for diseases. Then obviousness problem. If their method is similar (can tell by way of chemical compound search/amino acid sequence) then a problem….. There are likely a finite no of ways to modify the compound. If the technology patent has more than a few ways and not one way (substitutions of different variables in compound), then not obvious enough to infer from general claim. •Predictability test. is there a way to tell the new compound would work from the onset? If you can’t predict which one will work until you test and trial, then doesn't satisfy predictability test.

How do patents on MMP and uPA as diagnostics impact FTO?

•Patentable Subject Matter under Section 101. If there is something unique about detecting then very valid. They may have had a specific method in mind but realized the biomarkers (not the method) are key, so they may have felt the need to make the claims broader. If too broad then we can get into litigation about the patentable subject matter under Section 101. •Machine or Transformation Test. Recently the court tightened the standard for diagnostics – has to be tied into a particular machine or has to transform something to different state or thing. If claim has been allowed despite this and too broad; it is going to be something to flag. Doesn’t mean it is a 100% roadblock.

Would modifications to the protein sequence that reduced immunogenicity of the toxin qualify for a new patent with 20 year life, or only count as a divisional patent of the existing technology?

•There is a test. Test for new patent vs. Continuation: Look back at specifications if opportunity to change/reduce immunogenicity is disclosed, you can file continuation and add claims if you have an open/pending application. If not covered in prior specs and immunogencity is really new, you can either file a new application altogether or a continuation in part (claiming priority back to first application). If there was a continuation/app filed right before new patent was issued. Look at continuity data. What continuations or divisionals have been filed and if any still pending in PAIR. If new and non-obvious you get a fresh patent term 20 yrs. If you still have applications co-pending which we can claim priority to, then you can just do continuation or divisional but you forfeit the time. You can check to see if someone filed a divisional app right before patent issuing using PAIR.

DEPENDENT TECHNOLOGIES

Companion diagnostic

DNA recombinant techniques

Manufacturing processes

IP SUMMARY

Protection

Novelty

Non-Obvious

Freedom to Operate

TARGET MARKET

Non Small-Cell Lung Cancer

TARGET MARKETLUNG (NSCLC) HNSCC MELANOMA COLON

uPA / MMP YES YES YES YESIncidence HIGH (230K) MED (54K) MED (77K) MED (143K)Prevalence MED (390K) MED (350K) HIGH (875K) HIGH (1.1M)Deaths HIGH (160K) LOW (11K) LOW (10K) MED (51K)5 Yr Survival LOW (15%) MED (59%) HIGH (89%) MED (62%)

Lung Non-Small Cell Carcinoma (NSCLC)

Critical unmet medical need Large market

Potential for fast-track designation

from FDA

Potential off label usage for broad

range of cancers

STATUS QUO

Source: Mayo Clinichttp://www.mayoclinic.com/health/lung-cancer/DS00038/DSECTION=treatments-and-drugs

Surgery, sometimes chemotherapy

Surgery, chemotherapy, radiation

Combined chemotherapy and radiation, chemotherapy alone, surgery based

Chemotherapy, targeted drug therapy, clinical trials, supportive care

I

II

III

IV

Treat options for NSCLC at different stages

PRIMARY MARKET SEGMENT

Recurrent cases are extremely difficult to

treat

Poor prognosis; thus willing to enroll in experimental trials

62% of patients have high uPA and MMP

levels*

High MMP and uPA correlate with poor

prognosis*

New stage III/IV NSCLC patients

* Correlates with poor prognosis [Hofmann et al. Oncology Reports 16:587-595 ]

ESTIMATING MARKET SIZE

American Cancer Society report: Cancer Facts and Figures (2013)NCI SEER Database; Fast Stats on NSCLCHofmann et al. Oncology Reports 16:587-595Lou et al. General Thoracic Surgery. 145: 75-82

St. I/II(15%)

St. III(22%)

St. IV(56%)

Remission:(80%)

New Cases:230,000

Survivors:170,000

Recur.(20%)

uPA & MMPHIGH (62%)

111,000

uPAMMP

21,000

YEAR 2020

ESTIMATING MARKET SIZE

YR 2020111,000

Discount FactorsInsured

Willingness to treat

Contraindications Reimbursement

Penetration

4,286

Discount factors based on comparables (Herceptin) and US census data

NSCLC MARKET SIZE FORECAST

2020 2021 2022 2023 2024 2025 2026 20270

2000

4000

6000

8000

10000

12000

Best (50%)Worst (5%)

Num

ber o

f NSC

LC P

atien

ts

Note: Herceptin has 90% market share of market segment [eligible EGFR+ve Breast cancer patients]

Year

2020 2021 2022 2023 2024 2025 2026 20270

100200300400500600700800900

1000

BestWorst

Reve

nue

in M

illio

ns

* Source: STIFLING NEW CURES - The True Cost of Lengthy Clinical Drug Trials by Avik S. A. Roy Senior Fellow Manhattan Institute for Policy Research

NSCLC REVENUE OPPORTUNITY

Year

PRICING

Invention

Avasti

n

Erbitu

x

Herceptin

Gleevec

Rituxa

n

Tarceva

Nexava

r

Revlimid

020406080

100120140160180

80

45

8070

9280

6069

163

Cost

/ Y

ear

(In T

hous

ands

)

Drug name

Source: http://money.cnn.com/2013/04/25/news/economy/cancer-drug-cost/index.html

BENEFIT ANALYSIS

Invention Herceptin Erbitux Avastin0

102030405060708090 80

7080

45

13 9 11

Avg Cost / Yr ($'000s)Median OS*Median PFS*Number of MCAR*

Comparison against other block buster cancer drugs

OS – Overall Survival MonthsPFS – Progression Free Survival Months

MCAR – Most Common Adverse Reactions

Source: National Cancer Institute; Hofmann et al. Oncology Reports 16:587-595

PATIENT NEEDS ANALYSISTotal Customer

Costs

Psychological Cost

Short TermPhysical

Cost

Monetary Cost

Total Customer Benefit

Long TermQuality of Life

Benefit

Longer LifeBenefit

Time Spent In Treatment Cost

Time After Treatment

Benefit

Clarity Benefit

Source: http://blogs.webmd.com/cancer/2012/11/as-a-cancer-patient-what-do-you-want.html; http://www.cancerforums.net/threads/19080-Informnation-on-new-drug-Xalkori-(Crizotinib)-in-NSCLC?s=d344ffdd9e5c3bfaa69ddee16e1ce17b

BUSINESS MODEL

Preclinical Phase I Phase II Phase III Market Entry

•Mouse trials•Feline trials•Toxicology•Pharmacology•Manufacturing data •IND

Cost : $2M*

•Demonstrate low toxicity, absorption, metabolic distribution, excretion•Identify dosage

Cost: $1-$5M**

2013-2014 2015 2015-2016 2016-2019 2020-2021

•Controlled trials with patients for efficacy and side effects

Cost:$10-$40**

•Trials on large population of patients

Cost: $250-$400M**

•NDA •FDA approval•Manufacturing •Market launch • Marketing & Sales

MILESTONES TO MARKET

*Quotes from Bioreliance and Covance; ** STIFLING NEW CURES - The True Cost of Lengthy Clinical Drug Trials by Avik S. A. Roy Senior Fellow Manhattan Institute for Policy Research

PRIMARY RESEARCHExpert Panel:Dr. Art Frankel: S&W Memorial Hospital Dr. Susan Keating: CCS AssociatesDr. William Grady: FHCC Rosemarie Truman: RHT ConsultingDr. David Agus: USC

Technical Assessment• Innovative and promising technology• Need more realistic models, since native microenvironment is key• Compare effectiveness to existing drugs• Other protease dependent pro-drugs failed trials, may have side effects

due to uPA and MMP expression in normal tissues• Concerned about partial response or targeting small disseminated

tumors• Concerns about immune response to anthrax toxin proteins

PRIMARY RESEARCHExpert Panel:Dr. Art Frankel: S&W Memorial Hospital Dr. Susan Keating: CCS AssociatesDr. William Grady: FHCC Rosemarie Truman: RHT ConsultingDr. David Agus: USC

Licensing Considerations• Hard sell to “Big Pharma”

• Resistant to new ideas• Lung Cancer Pipeline “full”

• Pharmacology and toxicology data critical to progress• in vitro toxicity studies (test on normal cell types)• 14 or 28 day study in rats – Multiple doses toxicity (Systemic Delivery)

• Data supporting manufacturing questions• Scalability, reproducibility, scalability• Concern over the need to manufacture 3 components

PATHWAY TO COMMERCIALIZATION

Preclinical Phase I Phase II Phase III

Market Entry

PATHWAY TO COMMERCIALIZATION

Funding options

• SBIR phase I & II grants• Philanthropic

foundations and Patient advocacy groups

Venture capital

Business Model

• Partnerships with biotech companies having interest in similar technologies

• CRADA agreementsPotential

CompaniesBrian Oncore, Clovis Oncology, Biotech Synergy and Daiichi Sankyo

S m a l l P h a r m a

Potential acquisition by big Pharma

L i c e n s i n g

Acquisition

PANCREATIC CANCER TARGET MARKET SIZE

2018 2019 2020 2021 2022 2023 2024 2025 2026 20270

2000

4000

6000

8000

10000

12000

Best (50%)Worst (5%)

Num

ber o

f Pati

ents


YearNote: Based on the similar discount factors used for estimating the market size for NSCLC

PANCREATIC CANCER REVENUE OPPORTUNITY

2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 $-

$100 $200 $300 $400 $500 $600 $700 $800 $900

BestWorst

Reve

nue

in M

illio

ns

Preclinical Phase 1 Phase 2 Phase 3

< $0.5 M < $0.5 M < $1 M < $10 M

Estimated clinical trial expenditures for rare diseases*


Year

Assumed Treatment Cost - $100,000

FEASIBILITY SUMMARYTechnical feasibility

Freedom to operate

Remaining patent term

Legal, regulatory and reimbursement

Market opportunity (demand, revenue potential)

Funding

Overall – Needs more preclinical evidence

RECOMMENDATIONS

Publish all available toxicology and pharmacology data on OTT site

Seek venture philanthropy for pre-clinical

Consider orphan cancer indications for FDA fast track (even faster)

Consider optimizing immunogenic profile for possible new patent (new term)

Watch flagged PCT patent applications

ACKNOWLEDGEMENTSOncology:Dr. Art Frankel: UT SouthwesternDr. William Grady: FHCCDr. David Agus: USC

Intellectual Property:Robert McBride, Esq.Dan Nevrivy, Esq.Brian Andrea, Esq.Thomas Woods, Esq.

Reimbursement:Susan Downard, Medicare & Pharmacy Benefit Implementations at Kaiser PermanenteShaina Srivastava, Medicaid Director

Licensing and Commercialization:

Dr. Susan Keating: CCS Associates

Rosemarie Truman: RHT Consulting

Kristine Dehler: Covance

NIH Inventors:

Dr. Thomas Bugge

Dr. Stephen Leppla

Dr. Diane Guerrero

THANK YOU!!!

DISCOVERY TO MARKET CLASS

SREE NAMPALLYELAINE RUBINO

JON FRANCA-KOHSUSMITA SRIVASTAVA

APPENDIX

OVERVIEWProblem: Current treatments have significant adverse effects because their targets are either non-specific or are cells that play important roles in normal cells.

Need: Treatments with specificity, that kill cancer cells completely, have minimal side effects

Invention: Novel biotherapy to target only cancer cells without harming non-cancerous cells using engineered bacterial toxin.

Recommendation: Licensing currently not feasible.

SWOT

Value propositions

Excellent laboratory results

Licensed prior version

Long road to marketCompetition

Uncertain immune responsePublic perception

Growing target marketBroad Use / Potential off label

usage

FundingValley of death

Unfavorable clinical trial outcomesPatent term expiring soon

S W

O T

VALUE CHAIN

Vast OptionsService Culture

Rich Heritage

Suppliers Purchasers

Fiscal /ProductIntermediaries

Providers

SUPPLIERS


Rich Heritage

NIH(Bacillus anthracis spores)

Manufacturing Co.

Laboratory Equipment Vendors

Pharm/BioTech

PURCHASERS


Rich Heritage

Government Philanthropic Orgs.

HospitalsIndividuals

FISCAL INTERMEDIARIES


Rich Heritage

Medicare Insurance Companies

PROVIDERS


Rich Heritage

Hospitals Clinics

PhysiciansMCOs/HMOs

PRODUCT INTERMEDIARIES


Rich Heritage

WholesalersGroup Purchasing Organization

STRATEGIC MARKET ANALYSIS

WORLD MARKET

Revenues CAGR2013 ~ 43 Billion 2010-2015 ~ 10%2015 ~ 53 Billion

Source: Kalorama Information 2010

Roche; 54.4%

Novar-tis;

15.3%

Celgene; 6.0%Pfizer; 4.2%

Bristol-Myers Squibb;

3.8%

John-son & John-son; 3.5% Others;

12.9%

Market Share

RocheNovartisCelgenePfizerBristol-Myers SquibbJohnson & JohnsonOthers

LEGAL ISSUESBudget Control Act of 2011 affected the most expensive drug categories – cancer biotherapeutics

Biosimilar User Fee Act of 2012 (BsUFA), authorizes FDA to assess and collect fees for biosimilar biological products from October 2012 through September 2017

The Biologics Price Competition and Innovation Act of 2009 (BPCI Act) enacted as part of the Affordable Care Act of 2010, established new abbreviated approval pathway for biosimilars

Obama Healthcare Plan: mandates FDA to approve biosimilars, to render existing biotherapeutics drugs significantly affordable when their patents expire

PHARMACOVIGILANCE

US FDA Center for Biologics Evaluation and Research (CBER)

WHO standards through the Expert Committee on Biological Standardization (ECBS) serve as a basis for setting national requirements for production, quality control and overall regulation of biotherapeutics and biosimilars.

FDA

The IND application must contain:» Animal Pharmacology and Toxicology Studies » Manufacturing Information » Clinical Protocols and Investigator Information

FDA CHALLENGES

Q. Do the results of well-controlled studies provide substantial evidence of the treatment's effectiveness?

A. Murine studies are well controlled and showed excellent results – no side effects and low

toxicity. Feline studies in progress. Q. Do the results show the product is safe under the conditions of use in the proposed labeling? In this context, "safe" means that potential benefits have been determined to outweigh any risks.

A. Addressing safety of toxin by changing its molecular properties. Must prove this in human testing.

REIMBURSEMENT

Biotherapy physician administered only• Covered by Part B• No new CPT or ICD codes required• CPT code is 162.x, with the fourth-digit subcategory identifying the specified site of cancer

Not all costs covered by insurance

Strategy to improve reimbursement• Bundled payments for based on common protocol• Value-based insurance design • Shared savings models linked with pay-for-performance programs reward adherence to

standard protocols• Genentech has a Co-Pay Card program that covers some the out-of-pocket costs for patients

enrolled

Source: Strategies to Reduce Cancer-Care Costs: http://jnci.oxfordjournals.org/content/early/2013/01/30/jnci.djt020

IP EXPERTS

DRUGS APPROVED FOR NSCLC

1. Abitrexate (Methotrexate)2. Abraxane (Paclitaxel Albumin-stabilized

Nanoparticle Formulation) 3. Alimta (Pemetrexed Disodium)4. Avastin (Bevacizumab)5. Bevacizumab6. Carboplatin7. Cisplatin8. Crizotinib9. Erlotinib Hydrochloride10. Folex (Methotrexate)11. Folex PFS (Methotrexate)12. Gefitinib13. Gemcitabine Hydrochloride14. Gemzar (Gemcitabine Hydrochloride)

15. Iressa (Gefitinib)16. Methotrexate17. Methotrexate LPF (Methotrexate)18. Mexate (Methotrexate)19. Mexate-AQ (Methotrexate)20. Paclitaxel21. Paclitaxel Albumin-stabilized

Nanoparticle Formulation22. Paraplat (Carboplatin)23. Paraplatin (Carboplatin)24. Pemetrexed Disodium25. Platinol (Cisplatin)26. Platinol-AQ (Cisplatin)27. Tarceva (Erlotinib Hydrochloride)28. Taxol (Paclitaxel)29. Xalkori (Crizotinib)

Source: National Cancer Institutehttp://www.cancer.gov/cancertopics/druginfo/lungcancer

Therapy Marketer Phase

Cetuximab (Erbitux) ImClone III (U.S.)

Vadimezan (DMXAA, ASA404)

Novartis II (E.U.)

Afatinib (BIBW 2992) Boehringer Ingelheim Pharmaceuticals

II, III ongoing (U.S. , E.U., Korea)

motesanib (AMG-706) Amgen II complete (U.S.)

sorafenib (Nexavar) Bayer II complete (U.S.)

sunitinib (Sutent) Pfizer II complete (U.S. , Canada, E.U)

Exelbine (ANX-530) Adventrx Pharmaceuticals Registration (U.S.)

NEW DRUGS

NSCLC CLINICAL TRIALS

All the major pharmaceutical players

Over 120 drugs and biologics in clinical trials

Stage 1 Stage 2 Stage 3 Stage 40

50

100

150

200

250

300

NSCLC REVENUE OPPORTUNITY

• Estimated investments prior to the market launch in 2020 are about $1 Billion. • This investment funds the pre-clinical development and Clinical Trials

2020 2021 2022 2023 2024 2025

Total Revenue 343$ 470$ 733$ 1,135$ 1,676$ 1,206$ Less: Cost of Revenue (COGS) 202$ 216$ 337$ 454$ 670$ 362$ Gross Profit 141$ 254$ 396$ 681$ 1,005$ 844$ Operating Expenses (70)$ (83)$ (85)$ (100)$ (115)$ (130)$

Research & Development (50)$ (50)$ (40)$ (40)$ (40)$ (30)$ SG &A (20)$ (33)$ (45)$ (60)$ (75)$ (100)$

EBITDA 71$ 171$ 311$ 581$ 890$ 714$ EBIT 71$ 171$ 311$ 581$ 890$ 714$ Provisions For Taxes 25$ 60$ 109$ 203$ 312$ 250$ % of EBIT 35.0% 35.0% 35.0% 35.0% 35.0% 35.0%Net Income ($) 46$ 111$ 202$ 378$ 579$ 464$

($ in Millions)

STAKEHOLDER ANALYSIS

Patients

DoctorsResearchers

FDA

Govt/Public Health

Patient Advocate Groups

Funders

National Cancer

Institute

Insurance

POTENTIAL LICENSEESAmbit Biosciences

ARIAD PharmaceuticalsGlobeImmuneHeat Biologics

Idera PharmaceuticalsActive Biotech

Oncolys BiopharmaOncoGenex Pharmaceuticals

GenprexBryan Oncor

ZIOPHARM OncologyTalon Therapeutics

VasGene Therapeutics

Criteria: Small Pharm, NSCLC, Phase I Trials, $$

STANDARD LICENSE TERMS

• If there is licensee interest:» An options based deal» Requires an upfront payment of approximately $3 million» In exchange if NIH achieves clinical proof of concept, Pharm

co. has option to pay $80 million for an exclusive license for the compound.

» Pharm co. would take over late-stage development and commercialization while NIH collects development, regulatory and commercial milestone payments totaling $400 million

» Eligibility to receive tiered royalties on worldwide product sales

Your B-School Partner Using Your Business School to Enhance Both Technology Transfer Office and...

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