CHI, Cambridge Healthtech Institute - Translational Medicine

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Tuesday, March 6, 2007

Dynamic Resource and Portfolio Management within the New Experimental Medicine Paradigm

8:30am Pre-Conference Tutorial Registration and Morning Coffee

9:00-12:00 Pre-Conference Tutorial
This workshop will explore why the changing world of pharma and biotech R&D requires a more dynamic approach to resource and portfolio management and how modern simulation approaches using Bayesian statistical ideas can help managers to get the best out of their portfolio while actively managing down individual project risks. With the introduction of exploratory clinical trials, adaptive protocols, and translational medicine, attrition rates are now (i) changing, (ii) increasingly uncertain, (iii) more amenable to active management through explicit investments to fill critical knowledge gaps. These gaps often include the real biological relevance of the target, side-effects pharmacology, or the need to identify likely responders in advance of treatment. This workshop will demonstrate how, by analyzing the information and time effects of experimental medicine on potential projects, and therefore understanding the impact of filling these knowledge gaps, managers can maximize both the potential of their drug portfolio and the early development technologies at their disposal, including how to:

I. Present and visualize the key therapeutic benefits and late failure liabilities - the benefit/risk profile of potential drugs - as a set of options within the portfolio
II. Visualize the uncertainty associated with each option and reveal the extent to which expected portfolio values are particularly sensitive to risk that might be controllable
III. Analyze the potential impact of new information on project and portfolio values, to see where new translational medicine tasks could add greatest value - which types of investigations, which candidates?
IV. Incorporate realistic views of time to execute and reliability of results (false negatives/ false positives)
V. Revise both the candidate portfolio and the specific commitments to experimental medicine to get the best out of both candidates (or licenses) and the development capabilities available

As well as introducing participants to the underling principles, and some working models for exploring these risks and benefits, the workshop will use specific examples of drug development, including R&D under extreme time pressure such as a pandemic, to explore the risk verses benefits of different R&D paths.
Presenters: Wade Speir, Principal Consultant, PA Consulting
Michaela Hajek, Consultant, Life Sciences, PA Consulting
John Grant, Consultant, Life Sciences, PA Consulting

Main Conference

12:00pm Main Conference Registration

OPENING SESSION: PHARMACEUTICAL
MODELS IN TRANSLATIONAL MEDICINE

1:00 Chairperson’s Opening Remarks and Presentation Translational Medicine-Strategies and Processes
T. Forcht Dagi, MD, MPH, FACS, FCCM, HLM Venture Partners & Harvard-MIT Division of Health Sciences and Technology (HST) and the MIT Sloan School of Management Faculty

1:30 Novartis' Approach to Incorporating Translational Medicine into an Explore-Confirm Agenda for Pharmaceutical R&D
Effective Translational Medicine relies on the appropriate integration of Early Development and Discovery Research. Broadly speaking this breaks down into a paradigm with a phase of Exploration (or Learning) followed by a phase of Confirmation (which can be iterated) and this replaces the old sequential approach to Drug Discovery and Development. This session provides an update on Novartis Pharma's approach towards Explore-Confirm in R&D.
This approach is based on programs that develop a deep understanding of the molecular biology of pathways involved in multiple disorders, and how that understanding can be used to select therapeutic options and patient stratification for Early Development. This presentation provides examples using this approach in several therapeutic areas. Lessons learned from the Novartis experience will be shared.
Robert Schmouder, MD, MPH, Executive Director, Translational Medicine, Exploratory Clinical Development, Novartis Pharmaceuticals Corporation

2:15 Enhancing Drug Development through Innovation
We will address a novel tool in translational medicine that could address known needs, e.g. optimizing drug candidate selection post-discovery and avoiding unanticipated side effects once in the clinic; discuss the challenge of incorporating new methodologies into a well-established industry process, and consider the potential impact of such tools on the economics and efficiencies of R&D.
Gerald Curtis, Ph.D., Chief Scientific Officer and Founder, Bowman Research
Frank Armstrong M.B.,Ch.B., President & CEO CuraGen Corporation
Hugo Stephenson, M.D., President, Strategic Research and Safety, Quintiles Inc.

2:15 ExVivoMetrics™ a New Tool in Drug Development Techniques
Gerald Curtis, Ph.D., Chief Scientific Officer and Founder, Bowman Research

2:25 Integrating New Approaches into the Pharmaceutical Development Process: Challenges and Opportunities
Frank Armstrong M.B.,Ch.B., President & CEO CuraGen Corporation

2:35 New Techniques in Translational Medicine: Narrowing the Development Gap
Hugo Stephenson, M.D., President, Strategic Research and Safety, Quintiles Inc.

2:45 Q&A

3:00 Networking Refreshment Break

3:30 Question Based R&D
In a recent company wide R&D productivity improvement exercise (Bridging R&D) we implemented a question based template for our LO and exploratory development teams. The template formulates the questions that need to be addressed by these teams like organ exposure, target modulation, pharmacological effects etc. The teams are forced to address these questions and find satisfactory answers specifically related to their projects by measuring biomarkers. Measuring of target modulation in CNS probably requires different technologies (biomarkers)than f.e. in blood. The teams will have to identify these measuring methods and apply it to their projects. Finally the team needs to feed back clinical data into the project to further validate the methods.
Erik Sprengers, MD, Ph.D., Vice President, Translational Medicine, Organon nv

4:15 Panel Discussion with Session Speakers

How is Translational Medicine being funded?
Examples of successes and, more importantly, failures
Benefit vs, cost analysis
External Sources: What data are you willing to accept from small companies without repeating the validation?

Moderator: T. Forcht Dagi, MD, MPH, FACS, FCCM, HLM Venture Partners & Harvard-MIT Division of Health Sciences and Technology (HST) and the MIT Sloan School of Management Faculty
Panelists consist of session speakers

5:00 INTERACTIVE BREAKOUT DISCUSSIONS:

Roundtable 1: How Do We Stop Bad Drug Candidates from Slipping into the Pipeline?
Facilitator: Keith Ortiz, Partner, Vantage Point Consulting

Roundtable 2: Are Biomarkers the Answer to Shifting Failure Earlier?
Facilitator: Malorye Branca, Editor in Chief & Vice President, Pharmaceutical Discovery

Roundtable 3: What Are the Prospects for Translational Medicine in India?

Roundtable 4: What is the Role of Discovery in Shaping Clinical Attrition?

6:00 Networking Reception

7:00 Close of Day One

Wednesday, March 7, 2007

MORNING SESSION: INNOVATIONS in TRANSLATIONAL MEDICINE

8:15 Chairperson’s Opening Remarks and Roundtable Report Outs
Andrew Branca, Senior VP & GM, Advisory Services, Cambridge Healthtech Associates

8:45 Omics in COPD
	Omics experiment can be described as a genome wide “snapshot” of a biological system comparing functionally related stages of a studied mechanism (e.g. a disease or a treatment). COPD is defined as a non-reversible airflow limitation, that is progressive and associated with an abnormal inflammatory response of the lungs. The presented Project is an integrated approach to generate and analyze differential expression by RNA, protein and metabolomic approaches from clinical studies, animal- and cell-models. Different compartments are sampled simultaneously from subjects in the different studies. Data are integrated with publicly available data sets (knowlegde-based evaluation). The talk touches aspects in sample generation and handling, QC of samples and analysis results, data-management, -analysis and –integration, and illustrate how the project started and evolved as an inter-departmental and cross-disciplinary working effort within the company. A high level strategy for data-integration will be exposed.
Guido Grentzmann, Ph.D., Director, Biochemistry / Geneprofiling, ALTANA Research Institute, Nycomed

9:30 Establishing a Translational Medicine Collaborative Network in China
Translational Medicine is becoming an area of rapid growth across biopharma industry. Western biopharma companies are starting to look for China to set up translational medicine research collaborations; such collaboration have great potential but have unique challenges as well. Challenges include:

Identifying suitable sites
Accessing top talent pool
Interacting with existing clinical study infrastructure
Protecting IP
Following SFDA regulations on biosample processing

These challenges can be overcome by utilizing a systematic and integrated approach. In this presentation I will:

Familiarize attendees to the landscape for translational medicine in China
Teach attendees how to access the talent pool of translational medicine in China
Offer attendees strategies on how to overcome obstacles unique to translational research collaboration in China

Jingsong Wang, MD, Associate Director, Translational Medicine, Wyeth Research Labs

10:15 Networking Coffee Break

10:30 Translational Medicine: Where Are We? Where Are We Going? Do We Have the right infrastructures?
The development of key biomarker assays and their validation is often critical in defining and guiding development for several compounds. The First-in-Patient (often Phase 2a) proof-of-concept studies are so critical to future financing opportunities. However, there is often a tendency to include in the Phase numerous PD biomarkers in the hopes that one or more of these will show promising results. Still with multiple biomarkers, the complexity of the study increases. There is also the danger of “over-interpreting” the ensuing biomarker data that may be sparse and obtained using less robust exploratory assays.
In the development of the biomarker strategy as well as during method development, considerable attention should be given to balancing the need to simplify sample preparation procedures and minimize the number of clinical samples collected with the need to maximize the amount of useful information from each study. Is there an external option that would make all aspects of study planning and execution easier to manage?
To be Determined

11:15 INTERACTIVE PANEL: Moving Forward in Translational Medicine

Who are the Stakeholders and what are their needs?
FUNDING: From where will the $’s come?
Is an Association of Translational Medicine the answer?

Moderator: Francesco M. Marincola, MD, Editor-in-Chief, Journal of Translational Medicine, and Director, Transfusion Medicine, Clinical Center, NIH
Panelist: Malorye Branca, Editor in Chief & Vice President, Pharmaceutical Discovery
John Ferbas, Ph.D., Associate Director, Clinical Immunology, Amgen
Guido Grentzmann, Ph.D., Director, Biochemistry / Geneprofiling, ALTANA Research Institute, Nycomed
Dominic Spinella, Ph.D., Global Translational Medicine Lead, Oncology, Pfizer, Inc.


12:15 Networking Luncheon	Sponsored by
Dynamic Arrays for Real-Time qPCR Fluidigm has introduced nanofluidic chips called dynamic arrays, which provide experiment density in a class with microarrays yet consume orders of magnitude less sample and reagents compared to microplates. Dynamic arrays have been introduced for real-time qPCR. Each chip accepts 48 cDNA samples and 48 TaqMan assays, creating 2,304 (48x48) parallel reactions. Dynamic arrays for real-time qPCR are a significant advance for gene expression analysis as they provide a highly efficient means to validate expression changes for a set of genes on samples from many individuals. Marc Unger, Director, Research & Development, Fluidigm Corporation

AFTERNOON SESSION: MODELS FOR BRIDGING THE TRANSLATIONAL GAP

1:45 Using Analytical Approaches to Enable Translational Medicine Programs
	Flow cytometers are versatile instruments that have been traditionally used to enumerate cell subsets in the setting of infectious diseases (e.g. HIV) and to diagnose a number of malignancies. Recent technical advances in these platforms have paralleled analytical method development such that these instruments can be used to perform multiplexed immunoassays, probe intracellular compartments for cytokine production, define intracellular signaling pathways (detection of phosphorylated signaling molecules) and perform functional assays including cell proliferation and cell and antibody-mediated cytotoxicity. In this session, the widespread utility of flow cytometry for biomarker development will be reviewed with specific examples to underscore the value of integrating this approach into clinical development programs.
John Ferbas, Ph.D., Associate Director, Clinical Immunology, Amgen

2:30 Effective Translation of Pharmacokinetic/Pharmacodynamic Information in Support of mAbs Clinical Development
	With the advancement in genetic engineering and manufa turing approaches in generation of monoclonal antibodies (mAbs) during the recent years, clinical application of therapeutic mAbs has witnessed an increase in popularity. Currently, various forms of antibodies, including mouse, chimeric, humanized or human antibodies, targeted against a diverse array of pharmacological targets, have been either marketed in US or are currently in clinical trials. Similar to what has been traditionally practiced for advancement of classicsmall molecule therapeutics, an important consideration for translation of preclinical data to clinic and effective development of protein therapeutics, such as antibodies, is the basic knowledge of the pharmacokinetics (PK), understanding of the underlying factors influencing the disposition of antibody therapeutics and mAb Pharmaco/toxicodynamics (PD) across species. This presentation will focus on: Important considerations for development of fully human mAbs such as affinity, pharmacokinetics and antibody cross-reactivity Effective translation of preclinical information to clinic using information available for various marketed antibodies
Mohammad Tabrizi, Ph.D., Associate Director, Development DMPK and Bioanalysis, AstraZeneca Inc.

3:15 Networking Refreshment Break

3:30 Translational Oncology: Strategy and Tactics
Dominic Spinella, Ph.D., Global Translational Medicine Lead, Oncology, Pfizer, Inc.

4:15 Translational Update of Probability of Success in Drug Development
p(success) is a key indicator for portfolio prioritization and valuation. Translational medicine adds intermediate steps into drug developments, at which p(success) is updated. To take advantage of this effort for better and faster
decision making, it is important to rely on good p(success) estimates. Here, we address this point and show how p(success) may be updated between trials. We illustrate the ideas with real case examples coming from neuroscience.