9.05

Keynote address
Understanding what the business model needs to look like for AI products - Dendreon: Commercializing the first autologous cellular immunotherapy

• A new clinical paradigm: How has the strategy for development differed from that of traditional cytotoxic drugs?
• What data has had greatest influence throughout the regulatory approval process?
• What is the marketing and reimbursement pathway for a cancer vaccine?
• Persuading oncologists to go outside their comfort zone and adopt new modalities
  • How to displace, combine with or integrate the standard of care?
• Next steps - reigniting the platform to get other candidates into the clinic – what’s the rationale moving forward?

Dr Mitchell H. Gold, President & CEO, Dendreon Corporation

9.35

Questions & discussion

9.40

Financial markets perspective

• How will Dendreon and other successful phase III trials impact the market for AI products?
• How might the cancer vaccine market evolve?
  • What has been learned from failures to date?
• What business models are most likely to succeed in AI?
• What is the investment community’s influence on choice of indication for AI registration?
• What next after cancer vaccines – AI for chronic infectious diseases, allergy, autoimmune diseases?

Dr Philippe Pouletty, Co-Founder & General Partner, Life Sciences, Truffle Capital

10.05

Questions & discussion

10.10

Payor perspective

• How will Provenge™ be reimbursed? What impact will it have on CPT codes?
• When and how should companies start dialogue with payor, physician and patient groups to assess reimbursability of AI products?
• What is the likely requirement for outcomes research / health economics assessment with these new products?
• How will reimbursement decisions be made?
  • How will value be assessed?
• How should these technically complex AI products be positioned?

Dr Ulrich Granzer, Granzer Regulatory Consulting & Services

10.35

Panel discussion
Understanding what the business model needs to look like for AI products

• What are the critical components for building a business model to best fit the disease focus, technology, or potential investment group/partner?
  • How is it different from the traditional drug model?
• The global reimbursement landscape for AI products: What are major differences to be aware of in US/Europe and beyond?
  • How is it different from the traditional model?
• Demonstrating value to the prime influencers: What can industry do to make the field enticing and attractive to physicians and patient advocacy groups?
• Commercial scale-up of AI: What does it need to look like to achieve
  • a markedly increased patient volume?
  • the process being removed from a hospital/clinic environment?

Panellist:
Dr Habib Fakhrai, Executive Vice Chairman of the Board & CSO, NovaRX

11.10

Morning coffee in the exhibition area

12.00

Title to be announced

Dr Matthew W. Kalnik, Senior Vice President, Strategic Planning & Business Operations, Nabi Biopharmaceuticals

12.25

Questions & discussion

12.30

Title to be announced

12.55

Questions & discussion

Managing risk through a smarter phase II/III approach in AI

• Designing out discontinuity: Allowing the regulator to review data at interim stages
• Alternatives to doing the standard uncontrolled arm: Generating signals earlier
• Adopting randomized phase II trials: What are the pros and cons?
• How can novel flexible statistical approaches help with the choice of end point: Taking the initiative with non-conventional end points
• What benefits are these approaches bringing to AI?

1.00

Case study
Generating a well-supported phase III hypothesis in cancer immunotherapy studies: The importance of randomized, controlled phase II studies

• Size, structure and controls of cancer immunotherapy phase II studies
• Relevant surrogate endpoints and biomarkers – are there any?
• Examples of successful trial designs

Reiner Laus, MD, President & Chief Executive Officer, BN ImmunoTherapeutics

1.25

Questions & discussion

1.30

Buffet lunch in the exhibition area

2.40

Case study
How to minimize risk and time to approval of immunotherapeutic: the case of incorporating tumour stem cells in therapeutic vaccines

• Adaptive trial designs vs group sequential
• How many interim stages without jeopardizing the final statistics?
• Comparative studies in phase I-III
• Independent Data Monitoring Committees:
  • Stopping rules for futility
  • Early termination of the trial for efficacy
• Primary and secondary objectives
• Surrogate endpoints

Dr Habib Fakhrai, Executive Vice Chairman of the Board & CSO, NovaRX

3.00

Questions & discussion

3.05

Clinical development of AI combination therapies: Creative and accelerated trial design

• Designing early stage human trials for immunologic evaluation and initial activity assessment when combining experimental agents – chemo / biotherapy / immunotherapy
• Getting to combination phase II without multiple trials - phase 0/I - what’s the prevailing opinion on trial design?
• What should the control arm be for combination therapies: How to analyze the survival curve?
• Limitations in evaluating small cohorts and Bayesian design to achieve meaningful decision points for combination therapies involving AI
• Working with regulators to get early buy in for new combination studies

Dr Thomas A. Davis, CMO & Senior Vice President, Clinical Development, Celldex Therapeutics

3.25

Panel discussion
Debating the pros and cons of classical vs adaptive, flexible trial approaches for AI

• In what circumstances is adaptive design likely to be most effective and when is it likely to be over complicated?
  • What are the knock on effects in terms of ramping up patient numbers to power up the study?
• How can industry and regulators alike work to advance these data evaluation approaches to clinical development design?
• Comparing the experiences in oncology and other fields using AI

3.50

Chair’s closing summary

3.55

Afternoon tea in the exhibition area

12.00

Using an alternative animal model for toxicology of a novel Alzheimer’s disease immunotherapeutic

• Overview of the AD product candidate
• Preclinical rationale for animal models
• What are the alternative models for AD vaccines and immunotherapeutics?
• Do we need to be more open-minded regarding safety testing in other species?
• How do we allow for differences in immune systems and markers?

Dr Ronald W. Ellis, Senior Vice President, Research & Development, NasVax Ltd

12.25

Questions & discussion

12.30

State of the art in vitro systems: What novel approaches to predictivity are being adopted in AI?

• The XPRESIDENT platform: pre-clinical identification, selection and validation of relevant tumour antigens for cancer immunotherapy
• Identification and selection of clinically relevant antigens using human tumours as source material
• Predicting and benchmarking immunogenicity in vitro by an artificial APC approach
• Validation of immunogenicity in early clinical trials
• Validation of clinical relevance of antigens in mid-stage clinical trials

Dr Harpreet Singh, CSO, immatics biotechnologies GmbH

12.55

Questions & discussion

1.00

Case study
Immune interventions for chronic viral diseases - examples of CMV and HIV

• New antigenic targets, vaccine deliveries and immunologic endpoints
• Preclinical proof of concept - inferences from orthologous animal model systems
• Immune-based therapies and combined therapies to improve upon standard of care
• Considerations for the regulatory package moving forward

Dr Susan W. Barnett, Senior Director, Senior Project Leader, Viral Vaccine Research, Novartis Vaccines and Diagnostics, Inc

1.25

Questions & discussion

1.30

Buffet lunch in the exhibition area

Moderator: Professor Nikolai Petrovsky, CSO, Vaxine Pty Ltd

2.40

Case study – prophylactic vaccine
Establishing your preclinical rationale for animal models: To what extent can you trust mouse data?

• Identifying and utilising alternative animal models for preclinical efficacy in the prophylactic vaccine setting

Professor Dr Claire Boog, Scientific Director, Netherlands Vaccine Institute

3.00

Questions & discussion

Case studies
Revisiting the hardest targets - how are recent technological and scientific advances enabling fresh attempts to tackle major, long-standing unmet medical needs?

3.05

Case study – prophylactic vaccine - challenges for an RSV vaccine

• Attenuated virus vs. subunit vaccines
• Circumventing the effect of maternal antibodies against developing an immune response
• Approaches for avoiding potential immune pathologies associated with immunization

Dr Jan ter Meulen, Executive Director, Vaccines Research, Merck Research Laboratories

3.25

Questions & discussion

3.30

Case studies – prophylactic vaccines
Remaining vaccine challenges demand novel approaches

• Traditional vaccine approaches have been unsuccessful for diseases such as HIV, hepatitis C, and shigella
• Potential solutions may lie in better antigen or adjuvant selection, delivery route, or dosing regime
• Alternatively, these disease may only succumb to radical new vaccine approaches, examples of which will be discussed

Professor Nikolai Petrovsky, CSO, Vaxine Pty Ltd

3.50

Questions & discussion

3.55

Afternoon tea in the exhibition area

4.30

Panel discussion
Reaching a common understanding of which – and how much - data you need to do to get into the clinic: Is there a consensus?

• How to get regulatory buy-in earlier?

4.55

Moderator's closing summary

5.00

Close of session

4.40

Keynote presentation
How to develop the strategy and evaluate the key issues in development of AIs

• How does AI fit into the strategic plans for the business portfolio?
• Which applications are of greatest interest and why? How is their potential value assessed?
• How to make these expensive therapies more affordable and accessible by reducing cost of goods in any way possible – role of biosimilars
• Small molecule immunotherapeutics – will they be disruptive and replace biologics in AI? Will they drive down costs? Eg those targeting JAK-STAT pathways

Dr Helen Sabzevari, Global Head of Oncology-Immunotherapy, Merck Serono

5.05

Panel discussion
What is the extent of big pharma’s current involvement in AI and what form does it take?

• How does big pharma evaluate, assess and process novel AI products?
• What are they looking for from AI biotechs?
  • How should biotechs prepare for due diligence?
  • Changing expectations - partnering / collaborating rather than going for a deal from the outset – enabling big pharma to gain confidence in the technology first
• How are they are helping with regulatory / clinical development paths for AI products?
• Have we hit the inflection point yet: Are we spending enough money in the area to have success?
  • Are there enough biotechs involved?
• What are the differences between doing deals with big pharma with classical vaccine franchise versus those that don’t
• How are AI programs being integrated at big pharma?

Panellists:
Dr Axel Hoos, Co-Chair, Executive Committee, Cancer Immunotherapy Consortium (CIC) & Medical Lead, Ipilimumab Program, Bristol-Myers Squibb
Dr Karin Jooss, Head of Cancer Vaccines, Pfizer Global R&D
Jens-Peter Marschner, MD, Head, Immunologicals Programs, Global Early & Clinical Development Units Oncology, Merck KGaA

6.00

End of day 1 followed by a themed cocktail reception in the exhibition area