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FRIDAY, FEBRUARY 27

SELECTION AND DEVELOPMENT OF NON-CLINICAL SAFETY PROGRAMS FOR NEW THERAPIES 
(combined with Preclinical Development of Biologics Track)

8:30 Chairperson’s Remarks

8:35 Scientific Challenges in Preclinical Toxicology Studies to Support the Clinical Development of Biologic 
Barbara Mounho, Ph.D., Scientific Director, Toxicology, Comparative Biology and Safety Sciences, Amgen
The therapeutic advantage of biologics is their specificity, which provides them with a highly targeted therapeutic action. Due to the complex structural and biological nature of biologics, these molecules have several distinctive properties making them fundamentally different from conventional (small molecule) pharmaceuticals. The unique properties associated with biologics can create certain scientific challenges in conducting preclinical toxicology studies that typically are not issues for small molecules. The alternative approaches that toxicologists often use for the preclinical safety evaluation of these molecules will be discussed.

9:05 Safety of Molecularly-Targeted Biologics Versus Cytoxic Oncology Drugs: Utility and Limitations of Nonclinical Safety Assessment
Rakesh Dixit, Ph.D., D.A.B.T., Senior Director & Head, Biologics Safety Assessment, MedImmune, Gaithersburg, MD
The molecularly-targeted biologic (MTB) therapies have greatly improved the treatment of debilitating cancers. While MTB therapies are generally well tolerated, many MTB therapies with unique targets have also resulted in a wide spectrum of previously unrecognized and ill-defined toxicities in sensitive cancer patients. This presentation will discuss the potential mechanisms of toxicity of MTB versus standard cytoxic therapies in cancer treatment. The value and limitations of nonclinical safety assessment using animal models in identifying toxicities of newer MTBs versus cytoxics will also be presented. 

9:35 Streamlined Custom Non-Clinical Safety Programs for Biological Therapies
Lauren E. Black, Ph.D., Senior Scientific Advisor, Navigator Services, Charles River Laboratories
There is no “one size fits all standard” for biologic toxicology programs. For novel biologics, there may be more “gap” than “knowledge” of the treatment’s or the targets’ effects. To define appropriate safety programs but avoid excess studies, some processes can be cross applied. Knowing target structure, distribution, and function, we can anticipate many safety events. Historical cases, successful strategies for streamlining preclinical programs, and common hurdles to success will be discussed.

10:05 Sponsored Presentation (Opportunity Available)

10:20 Coffee Break

11:00 Qualification of New Safety Biomarkers for Use in Drug Development: Experience of the Predictive Safety Testing Consortium
Elizabeth Gribble Walker, Ph.D., Assistant Director, Toxicology, Predictive Safety Testing Consortium, Critical Path Institute 
Establishing a process for putting new biomarkers into routine practice for regulatory submission in drug development is a complex undertaking. While safety biomarkers that reflect injury to a particular target organ are applicable to developing both small- and large-molecule therapeutics, biologics possess unique considerations for safety testing. This talk will give an overview of the process and accomplishments of the Predictive Safety Testing Consortium, and highlight case studies and opportunities where such a collaborative consortium approach might advance biologic drug safety. 

11:30 Panel with Speakers: Developing Non-Clinical Safety Programs
Moderator: Vivek Kadambi, Ph.D., Director of Drug Safety Evaluation, Millennium: The Takeda Oncology Company 

• Lessons learned from in-vivo small molecules toxicology testing that can be applied to biologics or vice versa

• Points of intersection for nonclinical toxicology testing of biologics and small molecule therapies

12:00pm Luncheon Presentation (Opportunity Available) or Lunch on your own

COMPUTATIONAL TOOLS FOR PREDICTING COMPOUND TOXICITY

1:00 Chairperson’s Remarks

1:05 Impact of Target Selection on Drug Safety and Side-effect 
Michael Liebman, Ph.D., Managing Director, Strategic Medicine
We uniquely examine the “other side of the equation” in drug safety by analyzing the impact of target selection on predicting potential side-effects rather than focusing solely on the drug molecule’s profile for safety. These methods expand the target protein network using structural, functional and pathway relationships integrated with gene regulation and metabolomics to produce a complex network which can be analyzed through simulation-based approaches to identify considerations such as potential at-risk populations.

1:35 Knowledge-based expert systems, (quantitative) structure activity relationship tools and modeling approaches in preclinical safety studies 
Wolfgang Muster, Ph.D., Head of in silico and in vitro Screens, F. Hoffmann-La Roche Ltd. 
This presentation will illustrate how computation tools, deployed in the early drug development process, can help predicting toxicity and thereby optimize and select the best clinical candidates to move forward. A focus will be on in silico prediction methods roughly classified into so-called “expert systems” and “data driven systems”.

2:05 Compound Cytotoxicity Profiling and Characterization of Toxicity Mechanisms Using Quantitative High-Throughput Screening 
Ruili Huang, Ph.D., Research Scientist, Informatics, NIH Chemical Genomics Center 
A large library of compounds previously tested in traditional toxicological assays were profiled for cytotoxicity using quantitative high-throughput screening (qHTS). Combining data generated from these assays we designed a broader array of in vitro cell-based assays in order to screen large sets of compounds. Such assays should also elucidate mechanism of toxicity, prioritize compounds for further toxicological evaluation and predict in vivo biological response. As a proof of principle, we applied an unsupervised clustering method to this data set to identify mechanisms of action and evaluated the performance of this method by comparing the results against literature annotations of compound mechanisms.

2:35 To be Announced

3:05 Close of Conference

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For sales information, contact:
Carol Dinerstein
Tel: 781-972-5471 
email: Dinerstein@healthtech.com 
OR
Jon Stroup
Tel: 781-972-5483
email: jstroup@healthtech.com