Indications Discovery & Drug Repositioning Summit - Day 1

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Day 1 | Day 2 



7:30 am     Morning Coffee

8:35           Chairperson’s Opening Remarks

Aris Persidis, Ph.D., President, Biovista, Inc.



8:40           Repurposing Kinase-Targeted Medicinal Chemistry for Neglected Tropical Disease Drug Discovery

Mike Pollastri, Ph.D., Associate Professor, Chemistry & Chemical Biology, Northeastern University

This presentation will discuss the target purposing approach for neglected disease drug discovery, highlighted by some examples in my lab where we have found anti-parasitic activities in classes of human kinase inhibitors.

9:10           Synopsis of Breakout Discussions

Sponsored by
9:30           Systematic Drug Repositioning for Neglected Diseases and Not Only

Aris Persidis, Ph.D., President, Biovista, Inc. 
Drug repositioning is emerging as a major strategy to accelerate the development of drugs in rare or neglected diseases and beyond. The potential for innovation and novelty is significant and a number of case studies will be discussed, including Friedrich's Ataxia, Leber's neuropathy, progressive multiple sclerosis and others.

10:00         Networking Coffee Break in the Exhibit Hall with Poster Viewing

10:40         Drug Repositioning: An Opportunistic Approach to Identifying New Drugs for Malaria?

Julie Lotharius, Ph.D., Associate Director, Translational Medicine, Medicines for Malaria Venture

Medicines for Malaria Venture has tested a vast number of approved, investigational and discontinued drugs in an in vitro assay of human Plasmodium falciparum blood stage infection. Several hits with low micromolar activity and acceptable human safety and pharmacokinetic profiles were then followed up by in vivo testing in a humanized mouse model of P. falciparum infection. In addition, target and genome-based searches comparing the mechanism of action of known drugs to pathways responsible for parasite survival and propagation have been employed to identify compounds from the clinical development pipeline that may have anti-malarial activity. Data generated from these activities will be presented.

11:10         Repurposing Drug Candidates - Fexinidazole as a Promising Treatment for Visceral Leishmaniasis

Susan Wyllie, Senior Research Associate and Lecturer, Division of Biological Chemistry and Drug Discovery, University of Dundee

We report the potential for therapeutic switching of fexinidazole, currently in phase I clinical trials for treatment of human African trypanosomiasis, as a novel oral treatment for Visceral Leishmaniasis. This 2-substituted 5-nitroimidazole is rapidly oxidized in vivo to sulfoxide and sulfone metabolites. These metabolites were both active against Leishmania donovani amastigotes grown in macrophages, whereas fexinidazole itself is inactive. A once daily regimen for 5 days at 200mg kg-1 caused a 98.4% suppression of infection in a VL mouse model, superior to the clinical drugs miltefosine or pentostam. In African trypanosomes, the mode of action of nitrodrugs involves reductive activation via an NADH-dependent bacterial-like nitroreductase. Overexpression of the leishmania homologue in L. donovani increased sensitivity to fexinidazole by 19-fold indicating that a similar mechanism is involved in these parasites. These findings illustrate the potential of fexinidazole as a much needed oral treatment for VL.

11:40         Repurposing Drugs for Tropical Diseases: Case Studies and Open-Source Screening Initiatives

Curtis R. Chong, M.D., Ph.D., M.Phil., Partners Cancer Care Hematology & Oncology Fellow, Dana Farber, Brigham and Women’s Hospital, Massachusetts General Hospital

12:10 pm   Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own

1:40           Chairperson’s Remarks

Pankaj Agarwal, Ph.D., Director, Computational Biology, Molecular Discovery & Development, GlaxoSmithKline



1:45           Applying Computational Methods for Drug Repositioning

Pankaj Agarwal, Ph.D., Director, Computational Biology, Molecular Discovery & Development, GlaxoSmithKline

2:15           Computational Drug Repurposing Using Compendia of Public Molecular Data

Joel Dudley, Ph.D., Director, Informatics and Co-Founder, NuMedii, Inc.

Public molecular data repositories, such as the NCBI Gene Expression Omnibus (GEO), contain billions of molecular measurements, many of which are relevant to human disease. Although these repositories are often intended to serve as data archives, it is now possible to apply large-scale integrative informatics approaches to leverage the aggregate molecular data in these repositories towards evaluating new types of biomedical hypotheses. In this talk, I will present our recent work in developing and applying a systematic computational approach to identify novel drug indication relationships using public gene expression profiles, which recently led to the identification and pre-clinical validation of novel drug repositioning candidates for lung cancer and inflammatory bowel disease.

Sponsored by
2:45           What is 505(b)(2) an the Growing Importance of it Today in Drug Development?

Ken Phelps, President and CEO, Camargo Pharmaceutical Services

In the fiscal year 2006, approximately 20% of new small-molecule drugs were approved through the 505(b)(2) process; two years later, more than half of the small-molecule new drugs approved in the United States were based on this strategy. Judging from this rate it is expected that the percentage of 505(b)(2) approvals will be greater than 80% within the next few years. We will walk through and explain the reasons behind the remarkable success of the 505(b)(2) process.

3:15           Networking Refreshment Break in the Exhibit Hall with Poster Viewing

3:45           Repositioning Two Ways: Integrating Large-Scale Molecular Docking and High-Throughput Screening

Yvonne Li, Ph.D., Scientist, Canada’s Michael Smith Genome Science Center

We have developed a virtual screening platform to predict novel binding interactions between existing drugs and drug targets, with multiple levels of false positive filtering. We present an example of this approach for the breast cancer target p90RSK and validated our results using both high-throughput screening and cellular assays.

4:15           Repurposing of NSAIDs as Anticancer Chemopreventive Agents

Jie Zheng, Ph.D., Associate Member, Department of Structural Biology, St. Jude Children’s Research Hospital

4:45           Focused Breakout Discussions

In this interactive session, several topics will be offered for discussions and delegates are invited to choose a breakout topic of interest and join the moderated discussion at hand. In this informal setting, participants are encouraged to share examples from their work, vet ideas with peers and be part of a group problem-solving endeavor. We emphasize that this discussion is an informal exchange amongst scientists and is not meant to be, in any way, a product discussion.

Topic 1: Systematic Drug Repositioning

 Moderator:  Aris Persidis, Ph.D., President, Biovista, Inc.

1. Is systematic drug repositioning nice to have or need to have?
2. Is the best time to apply SDR sooner or later in the development process?
3. Where should leadership for SDR come from in a company?

Topic 2: Computational Drug Repositioning 

 Moderator: Joel Dudley, Ph.D., Director, Informatics and Co-Founder, NuMedii, Inc.


1. Types of data needed to improve computational drug repositioning approaches (e.g., "negative" data from clinical trials)
2. Getting buy-in for computational drug repositioning approaches from traditional stakeholders in pharma
3. Computational approaches for improving the probability of therapeutic success beyond drug-disease molecular relationships (e.g., modeling side effects, trial and regulatory success, patient subsets) 

Topic 3: Open Source Screening Initiatives 

Moderator: TBA

6:00           End of Day

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