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 Messages
HIV 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

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

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

Vision for New Vaccines

Clear, shared understanding of the future:
Disease Impact/Public-health need
Epidemiology, morbidity, mortality and socio-economic impact

Demand
Realistic 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 Will
Shared commitment to need, priorities, demand, price and cost

Partnerships
Risk sharing Leveraging resources and expertise

Elements of a Target Product Profile (TPP)

Indications Populations Geographical coverage Route of administration Presentation Co-administration

Post-license activities

Vaccine R&D Timeline

File
Preclinical POC DISCOVERY Many years RESEARCH 2-4 years DEVELOPMENT 6-8 years Launch REGISTRATION 1 year 2 years LCM continue

Identification of target antigens Understanding of pathologies Natural history of disease Done mostly outside of the Big Pharma

Antigen production Assay development Animal model dev. Preclinical tox

Phase I Phase II a Phase III Safety Dose finding Large scale Initial Dose/schedule safety immunogenicity finding + Immunogenicity Lot to lot consistency Phase II b + Early POC Non inferiority (combos) or Efficacy

Industrial Investment
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Vaccine Development Decision Gates

Goals of Pre-Clinical Development

Know your product
Vaccine 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?

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 hurdles
Interplay 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.

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