Open Laboratory Drug Discovery at GSK: Collaborating on Medicines for Diseases of

the Developing World

Martin PanDiscovery Medicine

Purpose of this Presentation

• GSK Commitment to Diseases of the Developing World

• Introduce the Tres Cantos open laboratory concept

• What does success look like?

To improve the quality of human life

do morefeel betterlive longer

Our Mission

by enabling people to

The Challenge

• Africa has 24% global disease burden, but only 3% of the world’s health workers

• Healthcare spending (annual per capita)– $34 - minimum required for

fundamental healthcare – <$12 - 35/46 countries in Africa

spend – $2,000 - high income countries

• Access to essential medicines– <30% - Africa

GSK’s Response - Why?Ethical imperative

– Build a new, sustainable business model to address the needs and capabilities of the developing world

Image and reputation imperative

– Demonstrate to financial institutions & external stakeholders effective GSK management of non-financial risks and thereby meet the expectations of society

Business imperative

– Defend importance of IP framework for innovation

– Responsibly pursue commercial opportunitiesIt’s the right thing to do for patients, society

AND for our business

GSK’s Contribution

Pre Clinical R&D

Pre Clinical R&D

Clinical R&D

Clinical R&D

New Business Models

New Business Models

Post LaunchPost

LaunchHealth SystemsHealth Systems

• R&D PPPs• Knowledge Pool• Open lab• Malaria screen• TB, malaria

• R&D PPPs• Knowledge Pool• APJEM R&D• Malaria vaccine• HIV

• Tiered pricing• Price caps• MIC Pricing• Branded generics• 5% return on malaria

vaccine• ViiV Healthcare• Brazil JV• AMC

• CME• Voluntary

licencing

• 20% reinvestment• LF elimination• African malaria

Partnership• PHASE• Positive Action and

PAFC• Humanitarian relief

GSK, Tres Cantos, Spain

• Focused on R&D in malaria, TB and other neglected diseases

• Collaborative with key funding partnerships

• Projects prioritised by unmet medical need - not commercial benefit

• Approx. 120 scientists in Tres Cantos• Full access to GSK resources

GSK Diseases of the Developing World

What is our Vision for Tres Cantos?

• A campus that is recognised as a leading place to translate ideas into medicines for Diseases of the Developing World (DDW)

• The first port of call for any academic, institute or pharma company wishing to partner in drug discovery for DDW

• A pipeline of innovative transformational medicines for malaria, resistant TB and other Diseases of the Developing World

January, 2010

Open Innovation for Neglected Tropical Diseases

• Comprises three main strands:

– Being more flexible with our IP and know-how - Knowledge Pool

– Enabling access to our resources - open laboratory

– Opening up access to our compounds and data

Current Antimalarial Therapy Covers a Very Limited Number of Biochemical Pathways

MitochondrionDrug:Atovaquone

Apicoplast

Drug:Azi, antibiotics

Food vacuole

Drug:4-aminoquinolines

Cytosol

Drug:Antifolates artemisinins

• Resitance emerging to all current classes of antimalarials

• Sequencing of P. falciparum genome has revealed more than 5000 genes

• Whole cell approach allows screening of all relevant blood-stage targets

• Assures a minimum of pharmacological requisites

• Membrane permeability

• Activity against target in its intracellular context

• Identification and validation of novel anti-malarial targets

Fidock, D.A. et al, Vol. 3, 2004, 509-520

Data-sharing: Release of 13,500 Malaria Chemical Starting Points

• > 2 Million compound GSK corporate collection screened for inhibitors of malaria parasite growth– Whole cell approach under BSL3 containment– Year long effort involving multiple groups at GSK

• Over 13,500 hits identified – structures and data released on widely accessible internet sites

• Leadership step to engage worldwide scientists to fully exploit data and promote neglected disease research

• Jumpstart research on urgently needed novel antimalarial chemotypes and targets for drug discovery

13

EMBL-EBI Database for Mining Malaria Compound Setwww.ebi.ac.uk/chemblntd

What is the Tres Cantos Open Lab?

• A concept intended to facilitate open collaboration between academia or other institutions and GSK

• An opportunity for external scientists to work alongside GSK drug discovery scientists in Tres Cantos

• A way for GSK to demonstrate commitment and seek new approaches for stimulating research in the field of neglected diseases

General Principles of the Tres Cantos Open Lab

• Incoming projects will be aligned with our interests in TB, malaria or other neglected diseases

• Projects will benefit from the collaboration with DDW Drug Discovery

• An open approach to innovation in neglected diseases (intellectual property)

Open Lab

Collaborations and Alliances

Governing Board

Chair of the Governing Board

DDW Campus Head

High-level Operating Model for the Tres Cantos Open Lab

Tres Cantos Open Lab – `Open for Business´

• First open laboratory project has started; others being negotiated

• In the short-term, success for GSK will be that we have fostered the science and drug discovery in neglected diseases

• In the longer-term, success for GSK will be that we played our part in bringing transformational new medicines to people who need them

Open Lab possibilities for TB?

Open Architecture Approach to Research on Neglected Tropical Diseases

• Significant research funding devoted to neglected tropical diseases, but research largely fragmented

• Need for more concerted, collaborative approach to reduce redundancy and increase chance of success

• Neglected tropical disease research is an ideal area to explore innovative data sharing approaches

• Open architecture approach could be model for future pharmaceutical R&D

21

The over-arching priority ...

Focus on the Patient

The over-arching priority ...

Focus on the Patient