2017 Agenda

The Harrods Food Court Problem: Picking & Choosing from the Delightfully Arrayed Immuno-Oncology Delicacies
Columbus Room
While not a new problem in oncology, the sheer number of shiny options and the fervor for accessing these (and the price tag) has ramped up the complexities in building cancer portfolios: balancing scientific risk of early/novel IO targets with the commercial risk of the Nth checkpoint inhibitor or a later entry CAR-T cell therapy. This panel will be a lively discussion around the pros and cons of different strategies that BioPharma are pursuing, and how that affects key decisions for early stage biotechs in telling their story, and in their clinical development choices.

Moderator: Jeffrey M. Bockman, PhD, Senior Vice President,Oncology Practice Head, Defined Health

• Panelists:
  Christopher Mortko, PhD, Executive Director, Oncology Search & Evaluation Lead, Merck
• John Mumm, PhD, Director of Immunoncology R&D, MedImmune
• Tingting Zhang-Kharas, PhD, Advisor – Oncology External Innovation, Eli Lilly and Company

Put Your Money Where Your Heart Is – Investing in Novel Cardiovascular Therapies in Myocardial Infarction, Heart Failure and Peripheral Artery Disease
Columbus Room 
Cardiovascular disease remains the largest cause of mortality, life-long morbidities and healthcare expenditure, for which we have a societal responsibility to address. Presently, R&D efforts have been channeled to rare disorders and oncology, where much progress is being made in complex therapeutic approaches such as gene and cell therapy. These investments are being rewarded, as several gene and cell therapeutics are expected to be launched into the US market starting late this year. Such substantial technological validation could potentially drive Pharma investment back into regenerative medicines for cardiovascular disease given the size of the commercial opportunity; however, distinct challenges must be addressed.Join us as we review historical setbacks in novel therapies and how development risk might be mitigated in new regenerative approaches, and discuss the following questions:

• Are we testing novel therapies in patients who have travelled too far in terms of disease progression to benefit from regenerative therapies?
• What novel biomarkers are in development to ensure better efficacy in selected patient subsets?
• Where are we in terms of our understanding of genetics in these various spaces?
• What fresh approaches to clinical trials may help to lead to better therapies for the right patient, at the right time?
• What can be leveraged from advances in implementing commercial models of complex therapeutics for rare disorders and oncology?

Moderator: Michael C. Rice, MS, MBA, Principal, Defined Health

• Jonathan Garen, Chief Business Officer, uniQure
• Timothy Hemesath, PhD, Director, External R&D Innovation, Site Lead New York Centers for Therapeutic Innovation, Pfizer In
• Douglas W. Losordo, MD, FACC, FAHA, Senior Vice President, Clinical, Medical and Regulatory Affairs and Chief Medical Officer, Caladrius Biosciences
• Barry Ticho, MD, PhD, Chief Medical Officer, Stoke Therapeutics, Inc.

Deconstructing Neurodegeneration: Orphans as Entrées to Larger Markets
Columbus Room 
Orphan neurodegenerative diseases that share underlying pathological characteristics with broader indications are emerging as potential templates for accelerated drug discovery and development. For example, advances in clinical diagnosis, neuropathology, genetics, biomarkers and tau-related therapeutic approaches for the treatment of PSP, an orphan disease, promises to inform development of therapies for other tauopathies, including Alzheimer’s disease. Pursuit of orphans in parallel with or as a precursor to their more prevalent counterparts is attractive for a number of reasons, including speed to market, patient homogeneity, reduced clinical trial expense, MOA elucidation, and staged de-risking of clinical programs. While time will tell if such an approach is broadly able to bear fruit, early examples seem to provide at least some high-level takeaways for those currently in the space or contemplating such an approach. Join us on as we take a deep-dive into some recent cases and their implications for clinical development strategy in neurodegenerative disease.

Moderators: Ginger S. Johnson, PhD, CEO, Defined Health & Joel S. Sandler, PhD, Associate Principal, Defined Health

Panelists:

• Sean Ekins, President, Chief Executive Officer, Co-Founder, Phoenix Nest Inc.
• Dana C. Hilt, MD, Chief Medical Officer, Lysosomal Therapeutics Inc.
• C. David Nicholson, PhD, EVP & Chief R&D Officer, Allergan
• Todd Sherer, PhD, Chief Executive Officer, The Michael J. Fox Foundation for Parkinson’s Research

Strategic Partner Presentations
Columbus Room 

National Institutes of Health 
MedImmune 
Eli Lilly and Company
Merck 
Pfizer