1 Financing and R&D for neglected diseases Role of Product Development Partnerships First meeting of Consultative Expert Working Group on Research and.

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Financing and R&D for neglected diseases Role of Product Development Partnerships

First meeting of Consultative Expert Working Group on Research and Development: Financing and Coordination –

Open Forum 6 April 2011

Contents

• Product development partnership (PDP) model

• Example of a PDP: Medicines for Malaria Venture (MMV)

PDP model

• Leverages public and philanthropic funds

• Engages the pharmaceutical industry and academic research institutions

• Undertaking R&D for diseases of the developing world that they would normally be unable or unwilling to pursue independently, without additional incentives.

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PDPs leverage the strengths of global partners to develop new tools for neglected diseases

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Philanthropy (funding, fund

raising ….)

Philanthropy (funding, fund

raising ….)

Public sector (early research, funding…)Public sector (early research, funding…)

Private sector (compound

libraries, facilities,

expertise …)

Private sector (compound

libraries, facilities,

expertise …)PDPs

(portfolio mngt, scientific due diligence, create links

across academia /industry; incentivize …)

PDPs(portfolio mngt, scientific due diligence, create links

across academia /industry; incentivize …)

The PDP model is vital to the R&D process for neglected diseases

• Access to best science, high tech facilities, skills compound libraries from Academia, Pharma and Biotech – e.g. 6 million compounds screened for malaria and 25,000 new starting points identified

• Innovative and cost-effective • Portfolio management allows flexibility to reallocate resources

• Apply rigorous industry style practices to progress projects through pipeline

• Shares the risk of R&D investment

• Uses funding to leverage further private sector assets, creating a virtuous circle

• Champions role in approaching unaddressed public health problems

• Trusted “broker” working across sectors and within countries

PDPs received less than 17% of global funding for neglected diseases in 2009

• Funding to PDPs in 2009 was $530.0m

• This represented 16.6% of global funding, 22.5% of global grant funding for neglected diseases

6 2010 G-FINDER report

Treatments: Activities of PDPs104 biopharmaceutical candidates in development

Notes: Includes products not funded by Gates Foundation. Biopharmaceutical candidates in development Include: IAVI, IPM, IVI, GATB, Aeras, MMV, MVI, MVP, PVS, DNDi, iOWH, PDVI, HHVI. Source: PDPs

59Pre Clinical

15Phase I

12Phase II

10Phase III

2Registration

6Launched

Drugs

Vaccines

Microbicides

# candidates

10%

12%

14%

57%

6%

2%

Slides source from:

Diagnostics and vector control: Activities of PDPs39 candidates

7Feasibility

7Test

Development6Evaluation

1Demonstration

6Country

Adoption

CD4FIND

IDRI

Notes: Includes products not funded by Gates Foundation. Biopharmaceutical candidates in development Include: IAVI, IPM, IVI, GATB, Aeras, MMV, MVI, MVP, PVS, DNDi, iOWH, PDVI, HHVI. Source: PDPs

4%

22%

26%

22%

Early Stage

In

Development

IVCC

# candidates

Diagnostics Vector control

26%

Slides source from:&

Medicines for Malaria Venture: PDP to discover, develop and deliver effective & affordable drugs

• Established in 1999 by WHO, World Bank, Governments, the Pharma Industry; subsequent funding from the Gates Foundation

• Swiss Foundation based in Geneva

• Largest ever antimalarial drug portfolio

• Launched 1 product (with Novartis); two under regulatory review by European Medicines Agency (with sigma-tau and Shin Poong)

• Supported prequalification of IV Artesunate for severe malaria (Guilin)

• Partners in ca. 130 countries

Medicines for Malaria Venture – Our Mission

MMV’s mission is to reduce the burden of malaria in disease-endemic countries by discovering, developing and facilitating delivery of new, effective and affordable anti-malaria drugs.

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Managing the MMV portfolio

• R&D priorities and Target Product Profiles evaluated and

agreed by Expert Scientific Advisory Committee (ESAC)

and published on MMV website

• Call for proposals published annually

• Assessment of proposals by MMV Science Team and

ESAC

• Grants awarded

• Projects managed on a daily basis in collaboration with

MMV scientific expert and assessed annually by ESAC

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MMV Governance structures

MMV Board of Directors

The highest policy and decision-making body of MMV which ensures that MMV’s objectives are efficiently executed by the management

MMV North America Inc. Board

Supports MMV’s Corporate Development activities in the USA

Expert Scientific Advisory Committee (ESAC)

Helps identify the best projects worthy of inclusion in the MMV portfolio and continues to monitor progress through an annual review of all projects

Access and Delivery Advisory Committee (ADAC)

Advises MMV on its global access activities to ensure timely and effective delivery of new antimalarial drugs in malaria endemic countries

Global Safety Board (GSB)

The Global Safety Board ensures adherence to scientific and ethical principles

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MMV global priorities

Malaria

•Increase the range of affordable quality ACTs to reduce morbidity and mortality

•Approval of quality treatment for severe malaria

Children

•Development of age-appropriate formulations to safely and effectively dose the youngest patients

Women and pregnancy

•Intermittent Preventative Treatment during Pregnancy

•Drug-drug interaction studies to support safe use with oral contraceptives

Resistance

•New drugs with better pharmacokinetic and safety profiles and activity against artemisinin-resistant parasites

Eradication: Transmission, relapse, long term protection

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Composition of the MMV portfolio 2011

RegistrationPreclinicalResearch Translational Development

Lead Opt Phase IIaPhase ILead Gen Phase IVPhase IIb/III

Novartisminiportfolio

Novartis2 Project

MK 4815(Merck)

IV artesunateGuilin

DHA-Piperaquine sigma-tau

Coartem®-DNovartis

GSKminiportfolio

Broad/Genzymeminiportfolio

AntimalarialsSt Jude/Rutgers

Other Projects12 Projects

sanofi aventisOrthologue screen

KinasesMonash

PfizerScreening

GSK2 Project

NITD609Novartis

OZ 439(Monash/UNMC/

STI)

AZCQPfizer

Pyramax®Shin Poong/University

of Iowa

ASAQ Winthropsanofi aventis/DNDi

AminoindoleBroad/Genzyme

AminopyridineUCT

QuinolonesUSF/ VAMC

PyrazolesDrexel

AstraZenecaScreening

GNF156Novartis

DHODHUTSW/UW/Monash

AN3661Anacor

TafenoquineGSK

MMV Access and delivery

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R&D

SRA1 RegistrationCountry of origin

WHO prequalification

National registration

Scale-upAcceptance

Outcome

Pricing (contractual)

Distribution

Pharmacovigilance

PolicyEssential Medicines List

WHO treatment guidelines(scientific)

Local guidelines / national policy1. Stringent Regulatory Authority

Results from MMV partnerships

• 55 million courses of paediatric Coartem distributed in Africa (MMV/Novartis)

• IV Artesunate WHO pre-qualified and registered in 23 countries through (MMV/Guilin)

• 2 new ACTs under regulatory review; paediatric formulations in advanced development (MMV/sigma-tau; MMV/Shin Poong)

• Studies ongoing to reduce burden of malaria in pregnant women (MMV/Pfizer)

• Programmes underway to improve understanding of malaria market dynamics (MMV/IMS/Vodaphone and Vodacom/Novartis)

• Portfolio of fifty projects including potential single-dose cure, drugs with activity against artemisinin-resistant parasites, transmission blocking, relapse and long-acting drugs

Partners include R&D-based Pharma, Generic manufacturers, academia, non-pharmaceuticals corporations, government …..