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Vaccine Development: From the Lab to the Clinic

Jim Tartaglia, PhD

Vice-President, R & D

Sanofi Pasteur

AIDS Vaccine 2011

Bangkok, Thailand

September 12, 2011

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HIV Vaccine Development: An Industry Perspective

Key MessagesHIV vaccine development follows same principles as with other vaccine efforts

HIV vaccine development has certain unique hurdles

HIV vaccine development/deployment requires public/private partnerships

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Vaccines – How Do They Differ from Drugs?

Biologicals vs. Chemicals

Preventative rather than therapeutic

Considered by many to be commodities

Impacts compliance, willingness to pay and acceptance

of side effects

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Challenges Facing the Vaccine Industry

Success of vaccines – people no longer fear many diseases

Consumers expect perfect vaccines

Highly effective

No side effects

Increased complexity and difficulty of regulatory environment

• Increased cost and length of development

• Increased resource drain associated with maintaining marketed products

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Vision for New Vaccines

Clear, shared understanding of the future: Disease Impact/Public-health need

Epidemiology, morbidity, mortality and socio-economic impact

DemandRealistic product profile, production & presentation

Realistic assessment of development costs and timelines

Clear vision of future demand, price and financing

Realistic assessment of public-health value, in the context of other preventive measures

Political WillShared commitment to need, priorities, demand, price and cost

PartnershipsRisk sharing

Leveraging resources and expertise

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Elements of a Target Product Profile (TPP)

Indications

Populations

Geographical coverage

Route of administration

Presentation

Co-administration

Post-license activities

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Vaccine R&D Timeline

Many years 2-4 years

DISCOVERY RESEARCH

• Antigen production• Assay development• Animal model dev.• Preclinical tox

PreclinicalPOC

6-8 years

DEVELOPMENT

Phase I•Safety•Initial•immunogenicity

Phase II a• Dose finding• Dose/schedule

finding• Immunogenicity

Phase II b• Early POC

Phase III• Large scale

safety +• Lot to lot

consistency +• Non inferiority

(combos) or• Efficacy

Industrial Investment

1 year 2 years continue

REGISTRATION LCM

Launch

File

• Identification of target antigens

• Understanding of pathologies

• Natural historyof disease

• Done mostly outside

of the “Big Pharma”

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Vaccine Development Decision Gates

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Goals of Pre-Clinical Development

Know your productVaccine candidates should be appropriately characterized to insure material can be consistently produced

Is the product safe ?Vaccines are given to healthy individuals, especially pediatric populations, limited tolerance to adverse events

Is the product immunogenic in animals?

Is the product effective in animal models?

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Clinical Development Plan (CDP):

Vaccine formulation

Recommended vaccination schedule (primary course,

booster)

Safety

Immunogenicity

Efficacy

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Phases of CDP:

Phase I

Phase IIa

Phase IIb

Phase III

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HIV Vaccine R & D Challenges

Need for novel technologies/complex regimens

Significant global clinical development hurdlesInterplay between clinical research and clinical development

Defining investment milestones

Challenges with increasingly complex partnerships

Strict traditional industry development paradigm is insufficient

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Sustained industry partnership for all components is essential to success

It is imperative to secure industry partnership to

ensure a cohesive strategy for:

Product profile

Regulatory strategy

Clinical supply

Approaches to access

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No Company, Government or NGO Alone Will be Able to Carry the Burden

Governments, academia, NGOs, donors and industry must work together to allow for the most effective means for developing and providing access to a vaccine(s) for

those who need it the most, as quickly as possible.