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PROGRAMA DE ESTRATÉGIA EM P&D DE MEDICAMENTOS PARA FARMANGUINHOS

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Page 1: Seminario iv

PROGRAMA DE ESTRATÉGIA EM P&D

DE MEDICAMENTOS PARA

FARMANGUINHOS

Page 2: Seminario iv
Page 3: Seminario iv

ESTRATÉGIA NA PROTEÇÃO DE

RESULTADOS

Page 4: Seminario iv
Page 5: Seminario iv

Custos de desenvolvimento de uma droga

• Custos podem alcançar US$ 800 milhões

para uma droga

• O prazo de desenvolvimento aproximado é

de 15-16 anos

• 75% deste custo é atribuído às taxas de

falha

• 90% de todas as drogas desenvolvimento

não chegam ao mercado

Page 6: Seminario iv

Onde, como e quando investir

nossos recursos de forma satisfatória?

Page 7: Seminario iv
Page 8: Seminario iv
Page 9: Seminario iv
Page 10: Seminario iv

Alocação de Recursos

Page 11: Seminario iv

Alocação de tempo (em anos)

Page 12: Seminario iv
Page 13: Seminario iv
Page 14: Seminario iv

TIME

Degre

e o

f In

tere

st

Idea

Research

Proof

Of

Concept

Demonstration

Pre-production

Success

in market

Academic interest

Commercial

interest

After Chisholm

O Enigma

Page 15: Seminario iv

Quem tem interesse em preencher o gap?

TIME

Degre

e o

f In

tere

st

Academic interest Commercial

interest

Page 16: Seminario iv

O desafio: ausência de valor para preencher o gap!

TIME

Degre

e o

f In

tere

st

After Chisholm

Page 17: Seminario iv

138

318

802

1318

0

200

400

600

800

1000

1200

1400

$ m

illi

on

1975 1987 2001 2006

Year

Estimated full cost of bringing a new chemical or biological entity to market

($ million – year 2005 $)

Source: J.A. Di Masi and H.G. Grabowski, ‘The Cost of

Biopharmaceutical R&D: Is biotech Different? Managerial

and Decision Economics 28 (2007): 469-479

São 15 anos para desenvolver uma nova droga

De cada 5-10,000 moléculas sintetisadas & separadas por sua atividade, apenas 250 chegam ao pré-clinico, apenas 5 chegam a pesq. Clínica e apenas 1 alcança o mercado

Custo de P&D em 2006: $ 1.318 bilhões

Apenas 2 de 10 drogas disponíveis

comercialmente produzem retorno que correspondam aos custos de P&D

Fármacos são geralmente baratos e fáceis de copiar – empresas de genéricos acessam mercados maduros desenvolvidos pelo 1o. Entrante, sem custo de entrada

Desenvolvimento de drogas é caro e arriscado

Page 18: Seminario iv

Drug Discovery Pipeline

Target

Identification

and

Validation

Assay

Development

Lead

Generation

Hypothesis

Generation Candidate Development Commercialization

Phase

III

Submit Global

Launch

Global

Optimization

Lead

Optimization

First

Human

Dose

Phase

IA

Phase

IB/II

Compound patent filed at Lead Op

Subsequent patents filed over 10-15 years

Page 19: Seminario iv

Função das Patentes na Inovação

Competitiva

Page 20: Seminario iv

Product A

75 80 85 90 95 00

A

B C

D E F G J H I K L M

Q R

N O

P

Basic patent/product lifecycle

Patent filings leading to granted patents

Page 21: Seminario iv

Classe de compostos, sais, formas cristalinas, usos (1o uso e

subsequentes), formulações, processo de obtenção, métodos de

purificação, rotas de administração, perfis de liberação e

combinações

Tipos de Patentes que refletem

o processo de P&D

Page 22: Seminario iv

O escritório Europeu conduz um dos exames mais rigorosos

Estatísticas de 2005:

128,754 depósitos

163,144 buscas empreendidas

104,433 exames

Apenas 53% das patentes foram concedidas

O prazo médio de aprovação é de 44.3 meses

Um portfólio de patentes de um produto pode ter entre 20 e 40 patentes

Obter uma Patente não é um processo trivial

Page 23: Seminario iv

No USPTO

Estatísticas de 2005:

417,508 depósitos

207,867 depósitos (nacionais)

143,806 depósitos estrangeiros

Apenas 48% das patentes foram concedidas

O prazo médio de aprovação é de 35 meses

Obter uma Patente não é um processo trivial

Page 24: Seminario iv

Obter uma Patente não é um processo trivial

INPI

Page 25: Seminario iv

Obter uma Patente não é um processo trivial

Tempo médio 8,1 anos com tendência de redução para 6 anos

Page 26: Seminario iv

Competition in Therapeutic

Innovation

• The first mover is rarely the most successful

• The period of exclusivity for first movers is

shrinking

Page 27: Seminario iv

The period of exclusivity for first entrants to

a therapeutic class is decreasing (US data)

Source: DiMasi & Paquette (2004)

1.2

3

4.1

7.2

10.2

7.7

1.8

2.8

5.1

5.9

8.2

7.2

1995-98 (n=18)

1990-94 (n=15)

1985-89 (n=14)

1980-84 (n=5)

1970s* (n=9)

1960s (n=8)

First-

in-C

lass A

ppro

val Period

Years

Mean

Median

Page 28: Seminario iv

Without patents there would be no innovation

• Given the costs & risks of drug development, without a period of exclusivity against copyists there would be no investment in pharmaceutical innovation

• Pharma do not seek therapeutic area exclusivity (anti-virals, antibiotics)

Patent protection promotes therapeutic & innovative competition

Page 29: Seminario iv

Compound differentiation

• New drugs tested against “gold standards”

• Patent competition drives improvements: Increased Efficacy

Decreased Side-effects

Decreases ADRs

Decreased drug-drug interactions

Decreased dosing

Specialised drug delivery systems

• Patients benefit from a range of products with differing characteristics

Page 30: Seminario iv

Quarterly Enalapril Sales in the UK

0

5,000

10,000

15,000

20,000

25,000

QTR

SEP 1

992

QTR

MAR 1

993

QTR

SEP 1

993

QTR

MAR 1

994

QTR

SEP 1

994

QTR

MAR 1

995

QTR

SEP 1

995

QTR

MAR 1

996

QTR

SEP 1

996

QTR

MAR 1

997

QTR

SEP 1

997

QTR

MAR 1

998

QTR

SEP 1

998

QTR

MAR 1

999

QTR

SEP 1

999

QTR

MAR 2

000

QTR

SEP 2

000

QTR

MAR 2

001

QTR

SEP 2

001

QTR

MAR 2

002

Sale

s (

£ m

illi

on

)

Protection

Expiry

Source: IMS Health MIDAS database

Page 31: Seminario iv

Changing face of Innovation

• The Past….Large Corporate R&D facilities

Secretive smart scientists, low level of collaboration

Closed Innovation

• The Present…..leaner business units Increased levels of collaboration

More outsourcing, more partnerships

Open Innovation

Page 32: Seminario iv

“Closed” Innovation “Open” Innovation

“No matter who you are, most of the smartest people work for someone else”

Bill Joy, Sun Microsystems

Page 33: Seminario iv

Changing Landscape of I.P • More small companies owning & licensing basic

IP

• Many companies not in manufacturing, only generating technology/IP

• More patent aggregators, who take on patents from universities & small companies

• Patents used as bargaining chips

Page 34: Seminario iv

Patent strategy and biotechnology

• Biotech industry (and sometimes universities) use broad and basic and research-tool patents in marketing to Pharma and seek “reach-through royalties,” to obtain a share of the ultimate rent.

Page 35: Seminario iv

Observations on the Academic IP

Page 36: Seminario iv

Technology Strategy

• Must concentrate on seeking competitive advantage, not on scientific interest

• Must be consistent with overall strategy – e.g. R&D programs shouldn't focus on product performance if the firm is

pursuing a cost leadership strategy

• Shouldn't focus exclusively on product design or manufacturing technology if this means ignoring other areas of the firm (info systems, materials handling, office automation)

• Must recognize risk/return tradeoffs

• Must be consistent with industry/product lifecycle

Page 37: Seminario iv

Sustainability of the technological lead

• Depends on: – Faster, more successful innovation than competitors

• managing internal vs external sources

• scale and scope economies in R&D

• superior technological skills

– Slowing the rate of diffusion to competitors

• preventing reverse engineering

• restricting technology transfer

• obtain/enforce IP

• loyalty of employees, non-disclosure agreements etc

• vertical integration

– Lock-in of customers

• building in buyer switching costs

• control of standards

Page 38: Seminario iv

Mechanisms undermining first-mover advantage

• Free-riding by late-comers

• First-mover locked in to the wrong strategy

• Competition

Page 39: Seminario iv

Free-riding

Latecomers can use the first-mover’s investments in:

• R&D

• moving down the learning curve

• employee training

• infrastructure development

• obtaining regulatory approval

• finding, educating customers & suppliers

Page 40: Seminario iv

Lock-in to the wrong strategy

• ex ante choices may prove to be wrong ex post

• First mover can be locked in to the wrong technology or marketing strategy because of – incumbent inertia

• sunk costs: plant and equipment, marketing and distribution channels, advertising and reputation

• reluctance to cannibalize existing product lines

– organizational inflexibility

• organizational routines

• corporate culture doesn't prize innovation

• doctrinal views about the world

• internal political dynamics

– established relations/contracts with other organizations

Page 41: Seminario iv

R&D Budgeting

• Technology strategy usually implemented by R&D budgeting decisions

– How much to spend?

– Where to spend it?

Page 42: Seminario iv

Where do R&D budgets go?

Breakdown for US manufacturing

• long run v. short run 25:75

• fundamental v. incremental 8:92

• product v. process 65:35

• basic v. applied v. development 5:25:70

– “basic” means advancement of knowledge without specific commercial objectives

– “applied” means research with specified commercial objectives

– “development” means embodiment of research results into products/processes

Page 43: Seminario iv

R&D Budgeting: General Considerations • R&D is an investment, not an expense

– evaluate R&D projects like any other investment project?

• R&D budgeting decisions entail making trade-offs against competing uses of funds – should R&D always over-ride other claimants?

• Continuing commitment to high levels of R&D spending often distinguishes leaders from followers – can a firm become a technology leader just by increasing R&D/Sales ratio?

• Adoption of a formal budgeting process often marks start-up companies’ transition to “adolescence”

• Can the firm find a project selection process which promotes innovative success?

Page 44: Seminario iv

Industry Practice in R&D Project Selection

• Diversity: no standard practice – most firms use home grown techniques

• Ambivalence towards quantification – many R&D managers are predisposed by background to seek objective

quantitative criteria for decision-making, but the weakness of available techniques and complexity of problems make them reluctant to give these a dominant role

• Some lessons: pay close attention to the process -- who should be involved? what weight should be placed on various inputs? how should conflicts be resolved?

Page 45: Seminario iv

Some Analytical Techniques for R&D Project Selection

• index models: compare probability-weighted benefit/cost ratios

• discounted cash flow models: recognize impact of distribution of costs and benefits over time, incorporate appropriate risk premium

• portfolio models: risk and return tradeoffs. CAPM ???

• scoring/profile models: compare projects against checklist of desired characteristics

• real options: “financial engineering at Merck”, recognize option value of continued funding, use Monte Carlo simulations to bound likely returns

Page 46: Seminario iv

Example: pharmaceutical R&D portfolios

• Typical firm runs 8-10 major research programs

• Discovery phase: $2m-$25m / year

• Development phase: $50m+ / year

• Manager’s problem:

– How much to spend on each program?

– How to select new projects?

– How to know when to stop projects?

Page 47: Seminario iv

Why is this tough?

• Very risky:

– 10,000 candidate molecules

– 10 go into development phase

– 1 makes it to the market place

• Hard to measure performance

• Spillovers: project successes are correlated

• Economies of scope and scale

Page 48: Seminario iv

Implications for real life R&D strategy

• In head-to-head competition:

– Try not to enter a race you aren't sure of winning: if you have to work too hard to win, it wasn't worthwhile

– Have eyes in the back of your head: be well-informed of your competitor's position, and let them know it

Page 49: Seminario iv

Effective strategies answer three key questions:

How will we Create value?

How will we Capture value?

How will we Deliver value?

Page 50: Seminario iv

• How will we create value?

– How will the technology evolve?

– How will the market change?

• How will we capture value?

– How should we design the business model?

– Where should we compete in the value chain?

– How should we compete if standards are important?

• How will we deliver value?

– How do we manage the core business and growth simultaneously?

– How do we use our strategy to drive real resource allocation?

Page 51: Seminario iv

Outline: • Why do I need an innovation strategy?

• How will we create value?

• How will we capture value?

• How will we deliver value?

• Doing strategy in practice

Page 52: Seminario iv

Why have a strategy?

Page 53: Seminario iv

The Timing and Impact of Management Attention

Phases

Influence

High

Low

ACTUAL

ACTIVITY MANAGEMENT

PROFILE

Acquisition Investigation Basic

Building Production Manufacturing

ABILITY TO INFLUENCE OUTCOME

Page 54: Seminario iv

Why is it so hard to kill project #26?

• It’s a “good” project!

• Good managers can meet stretch goals

(and I’m a good manager)

• Making difficult decisions takes time & energy

It’s very hard to kill projects without a strategy

Page 55: Seminario iv

Reasons to have a strategy:

2. To be able to change it

Page 56: Seminario iv
Page 57: Seminario iv

Simple

molecules

<1nm

IBM PowerPC 750TM

Microprocessor

7.56mm×8.799mm

6.35×106 transistors

semiconductor

nanocrystal (CdSe)

5nm

10-10 10-5 10-9 10-7 10-6 10-8 10-4 10-3 10-2

m

Circuit design

Copper wiring

width 0.2m

red blood cell

~5 m (SEM) DNA

proteins

nm

bacteria

1 m

Nanometer memory element

(Lieber)

1012 bits/cm2 (1Tbit/cm2)

SOI transistor

width 0.12m

diatom

30 m

Page 58: Seminario iv

Inventors Authors

Universities

Governments

Companies

Traditional IP Stakeholders

New Stakeholders are focusing on IP

Page 59: Seminario iv

Regulators Companies Start-ups

Universities Technology

Transfer Companies

VCs Investors

Governments Banks Financial

New IP Stakeholders + New interests =

More confusion and disputes

Multiple Inventors Authors

Indigenous Populations

Page 60: Seminario iv

E.g., Pharma Industry Requires Strong IP Protection

Source: Boston Consulting Group: “A Revolution in R&D” 2001

• 70% of R&D Costs Are Incurred before Clinical Trials • Cannot raise money without IP and cannot afford to get IP wrong!

Stage of Development

$165 $205 $40 $120 $90 $260

$165

$410

$530

$620

$880

$0

$100

$200

$300

$400

$500

$600

$700

$800

$900

Target ID

1 yr

Target Validation

2 yrs

Screening

1.1 yrs

Optimization

2 yrs

Pre-clinical

1.6 yrs

Clinical

7 yrs

Do

lla

rs in

Mil

lio

ns

Stage Cost

Cumulative Cost $370

Biology

3 years

Chemistry

3.1 years

Development

8.6 years

= TOTAL

14.7 years

Page 61: Seminario iv

The Increased Visibility Of IP: What Is At Stake?

The value of IP is growing but cannot be accurately forecast in an increasingly global and technological world. Our valuation

methodologies and laws are inefficient. This will lead to more IP disputes

“It is estimated that by 2007, as much as 90% of the value of the

world’s top 2000 enterprises will consist of intellectual property” Building and Enforcing Intellectual Property Value,

An International Guide for the Boardroom 2003

PriceWaterhouseCoopers

“How appropriate is our system – developed for a world in which

physical assets predominated – for an economy in which value

increasingly is embodied in ideas rather than tangible capital?”

Alan Greenspan April 4, 2003

Page 62: Seminario iv

What is the nature of an IP asset?

• Bundles of national and territorial rights

• Rights to exclude others (NB, not to practise)

• Rights considered as property (financial assets), which can be pledged and securitized

THE CHALLENGE = How to convert national, legal « rights to exclude » into global, commercial

revenue-generating assets?

Page 63: Seminario iv

Invention Protection of invention

Transfer of technology

Entrepreneur Start-up creation

Seed funding

Business plan

Proof of concept

Development of technology / product

First round financing

Management/Structure of company

Strategic partnerships

More rounds of financing

Company grows

Traditional Thinking: IP is done at the beginning

Product development

Sales & markets

Regulatory strategy & clinics

EXIT TO SUCCESS!

Page 64: Seminario iv

EXIT TO SUCCESS!

Invention Protection of invention

Transfer of technology

Entrepreneur Start-up creation

Seed funding

Business plan

Proof of concept

Development of technology / product

First round financing

Management/Structure of company

Strategic partnerships

More rounds of financing

Company grows

But, IP is important throughout

IP + Money = O2: Lifeline of the company

Product development

Sales & markets

Page 65: Seminario iv
Page 66: Seminario iv

Possible Revenue-Generating Strategies Using IP

• Sell Products: Usually a “one-way” street, with all IP rights exhausted (internationally or domestically) (e.g., INTEL)

• License/Rent Product: Better to retain rights & maintain some control (e.g., transgenic mice, software: restrict access to source code & use)

• Sale of IP: a) Assignment of IP assets: May be simplest (e.g., 3M Post-It)

b) License of IP Assets: How?

• M&A: Sell company including the IP in it (e.g., a holding entity)

• Joint Ventures: Alliances that pool their IP resources into a new company (e.g., Nanonics)

• Franchise: Package concept (e.g., McDonalds): what is the IP bundle (TM + © + Know-How + patents)? Quality Control and brand management issues?

• Create Market: Offer for free and then charge using installed base (e.g., Skype)

• Open Source/Freeware/Shareware & then charge for improvements (e.g., .php)

• Covenant not to sue? (e.g., Two start-ups to avoid depleting resources)

• IP Holding companies? Who should hold IP? Tax and financing issues: inter-company pricing and royalty considerations.

Page 67: Seminario iv

San Francisco (CN), February 24, 2012

Verinata Health and Stanford University sued Sequenom, in a dispute to determine who owns the rights to a noninvasive prenatal test that uses DNA sequencing to search for abnormal fetal chromosomes.

The context: a case study

Patent Fight Over Fetal DNA Sampling

Page 68: Seminario iv

• Verinata has just completed clinical trials of the test for aneuploidy, a genetic defect.

– The most common birth defect associated with aneuploidy is Down syndrome.

– Verinata claims the new test is more accurate than the maternal serum screening tests now available and less dangerous to the fetus than amniocentesis.

– Verinata licensed-in this technology from Stanford University.

– The company has spent "tens of millions of dollars in the research, evaluation, and development”.

Patent Fight Over Fetal DNA Sampling

Page 69: Seminario iv

• Sequenom has a patent (licensed-in) for "Non-Invasive Prenatal Diagnosis," which was issued in 2001 (the so-called '540 patent).

– in 2010 Sequenom's lawyers sent a letter alleging that "'the practice of non-invasive prenatal diagnostics, including diagnosis of the Down Syndrome and other genetic disorders, using cell- free nucleic acids in a sample of maternal blood infringes' the '540 patent.“

– Verinata's predecessor, Artemis Health, responded by stating that Sequenom's infringement claims were "unsupported by the patent".

Patent Fight Over Fetal DNA Sampling

Page 70: Seminario iv

• Verinata says that:

– since then, "Sequenom has repeatedly stated to the public that anyone who performs a non-invasive prenatal test using cell-free DNA circulating in the blood of a pregnant woman would infringe the '540 patent.

– these statements, which misrepresent the scope of the '540 patent, are intended to broadly convey that:

– no one other than Sequenom has the freedom to perform non-invasive prenatal testing under the '540 patent

– with the goal of deterring potential competitors from entering the market and deterring doctors and healthcare providers from using anyone other than Sequenom for those services."

Patent Fight Over Fetal DNA Sampling

Page 71: Seminario iv

NOW…

• Verinata seeks declaratory judgment that its test does not infringe on Sequenom's patent.

• And it claims that Sequenom itself infringes on Verinata's patents for determining chromosomal abnormalities (two patents), by manufacturing and marketing its test.

– Sequenom's infringement of both patents has been "deliberate and willful, warranting increased damages and attorney's fees," Verinata says.

– Verinata and Stanford University seek declaratory judgment that both patents have been infringed, a permanent enjoinder from further infringements, damages and treble damages.

Patent Fight Over Fetal DNA Sampling

Page 72: Seminario iv

Patent Fight Over Fetal DNA Sampling

The Message

• The key point is not the patents as a way to block competitors

– Few SMEs can afford litigation to enforce anyway

– Few SMEs care about controlling a monopoly!

• The patents protection (strength) of the actual business (product) is essential

• Freedom to operate is the key (this is the real value of your patents)

Page 73: Seminario iv
Page 74: Seminario iv

Translational Application of Novel Withanolides for the Treatment of

Advanced and Drug-Resistant Cancers

Mark S. Cohen, MD, FACS

Associate Professor of Surgery and Pharmacology

University of Kansas Medical Center

Barbara N. Timmermann, PhD

University Distinguished Professor and Chair

Dept. of Medicinal Chemistry, The University of Kansas

Page 75: Seminario iv

Portfolio of Novel Compounds

• We have identified many of the important anticancer activities of the withanolide, Withaferin A.

• Our strong medicinal chemistry team has recently identified 40 natural and semi-synthetic withanolide analogs from the local Physalis plant

– Unique properties through structure-activity relationships (SAR)

– Each has a unique anticancer activity profile

– Abundant selection of novel drug candidates for translation and proof-of-concept.

• Successful SAR has improved solubility and potency of drug analogs for ongoing in vivo studies.

Page 76: Seminario iv

Benefits of Novel Withanolides Novel Withanolides

from Physalis

Potent, highly selective anticancer activity, orally bioavailable

Induces apoptosis, cell cycle shift to G2M (potential radiosensitizer) and down-

regulates several key signaling pathways(RET, BRAF, mTOR, notch, BRCA, HSF-1) in melanoma, breast CA, thyroid CA, Head and Neck CA, and leukemias

Low Toxicity Profile,

clean hERG and AMES

Standard Chemotherapy

Potent, majority given i.v. which are often non-selective, resistance is

common (imatinib, cisplatin)

Targeted agents as monotherapies have problems with resistance through

alternative survival pathways

Systemic toxicities common (often dose- and treatment-limiting)

Novel withanolides effectively treat resistant cells and can synergize with imatinib

or cisplatin to decrease dose/toxicity

Page 77: Seminario iv

Natural withanolide X001 inhibits notch signaling

in breast cancers (even triple-negative tumors)

Novel withanolides induce apoptosis, shift the cell cycle to G2M,

and inhibit key signaling pathways in multiple cancer cell-lines

Page 78: Seminario iv

Withanolides are highly effective vs. MTC in vivo

________

Page 79: Seminario iv

Withanolides Effectively Treat

Melanomas in vivo with

Reversal of Metastatic Disease

Balb-C mice injected with aggressive B16F10 murine melanoma cells develop metastatic disease in controls (top

right figure) but treatment with low dose WA(2.5 mg/kg/d) results in partial response (top left) with prevention of

metastases or complete response with high dose treatment (5mg/kg/d) with reversal and cure of

metastatic disease (right bottom figure). Graph shows complete tumor response in 60% of low dose and 80% of

high dose treated mice sustained even 5 weeks after treatment ceased.

2.5mg/kg/d Control

Pre-treatment

After 5mg/kg/d x 3 wks

Page 80: Seminario iv

Market Analysis

• Melanoma, head & neck CA, ALL, glioblastoma, MTC, breast, pancreatic CA

• 350k patients/year in US, 2.5M worldwide

Potential Cancers for Withanolide

Treatment

• Orphan indication

• US market of 20K pts/year

• Currently PEGylated IFN is $20K per course, Yervoy is $120K per patient and Zelboraf is $112k/year

Current Novel Melanoma Therapies

•$50k per complete course of therapy due to superior safety & efficacy

• Withaolides are safer than other cytotoxics and targeted agents

• Can synergize with either cytotoxics, BRAFi or other TKIs for combo therapy

• Phase 1 human trial anticipated for 2014

Melanoma Treatment with Withanolides

2018 2019 2020 2021 2022

Market share 5% 15% 30% 40% 50%

# of courses

of therapy

1,000 3,000 6,000 8,000 10,000

Revenue @

$50k per

$50M $150M $300M $400M $500M

Page 81: Seminario iv

Business Development Strategy • IP

– University has both composition of matter and method-of- use patents filed for novel withanolides from Withania and Physalis (licensing arrangement for partner/start up)

• Capitalization – $150K grant from Inst. for Advancing Medical Innovation

– Seeking additional $5M to get orphan status, IND, and complete phase 1 data in melanoma

• Development Model – Start-up to move lead withanolide (already identified for

potency in melanoma/ pathway specificity and solubility by SAR) through Phase I and then partner with larger pharma company to reach market

– Early partnership/licensing opportunity with pharma company to move lead candidate(s) into market.

Page 82: Seminario iv

Withanolide Development Milestones

Page 83: Seminario iv

Reasons to Invest in Novel Withanolides

• Strong Scientific Validation

– In vitro / in vivo anticancer mechanism, potent efficacy, low tox

– Orally bioavailable analogs with enhanced solubility and potency for cancers (melanoma, thyroid, ALL)

• Solid IP portfolio

– 40 novel anticancer compounds with composition of matter and method-of-use patents filed (licensing arrangement for partner/start up)

• Accelerated Market Entry Timeline

– Orphan drug pathway in advanced melanomas, GLP scalability to start human Phase I in 2014, potential market entry by 2018

• Early Partnership/Licensing Opportunity – Start-up can complete Phase I with $5M invested then

partner/license with pharma to reach market

Page 84: Seminario iv
Page 85: Seminario iv

Summary

• Strong I.P essential & is the foundation of any technology driven organisation

• Portfolios of multiple patents held by different parties do not impede innovation

• There would be no competitive therapeutic innovation

without patents

• To be successful in taking new medicines into the clinic we need to dramatically increase our partnering activity to drive innovation through new business models