Global Health Focus: Repurposing for Rare Diseases and Orphan Drug Development

June 27-28, 2017 | Chicago, IL

2017 Agenda

June 27, 2017

7:45 am Registration/Continental Breakfast
8:25 am Chairperson's Opening Remarks
Aris Persidis, Ph.D., Chief Executive Officer, Biovista
8:30 am Massive Parallelization of Rare Disease Drug Discovery
Recursion's proprietary discovery platform brings together artificial intelligence with automated high throughput biology for therapeutic repurposing for rare genetic diseases. I will describe how we leverage these sophisticated screening methods to interrogate drugs against human disease models at scale.
Ron Alfa, MD, Ph.D., Vice President, Discovery and Product, Recursion Pharmaceuticals
9:00 am Presentation Title to be Announced
Speaker TBA, Elsevier
9:30 am A Complex Network Approach to Drug Repositioning
Computational methods are powerful tools for drug repositioning, and many such successful approaches are built around the complex network paradigm. Indeed, available drug datasets are very appropriate for describing structural relationships between pharmaceutical active substances such as chemical similarities or drug-target interactions, as well as behavioral connections such as drug-drug interactions. In this presentation, Dr. Bogdan will show how to extract previously unaccounted pharmacological properties using only drug-drug interaction networks.  By employing algorithmic clustering of drug-drug interaction networks, we recover recent and old repositionings but we can also indicate new potential drug repurposings.   
Paul Bogdan, Ph.D., Assistant Professor, Department of Electrical Engineering, University of Southern California
10:00 am Refreshment Break
10:25 am Presentation Title to be Announced
Berish Y. Rubin, Ph.D., Professor, Department of Biological Sciences, Fordham University
11:05 am A New Drug Discovery Paradigm Pioneered by Pharnext: PLEOTHERAPYTM
A new R&D platform pioneered by Pharnext, PLEOTHERAPY™, allows the identification and development of synergistic combinations of repositioned drugs at low doses. This approach is universal as it is applicable to any diseases and any compound whether in clinical development or off patent. A proof-of concept of PLEOTHERAPY™ was obtained with the lead PLEODRUG™, PXT3003, in Charcot-Marie-Tooth disease type 1A (CMT1A) through positive Phase 2 results. An international pivotal Phase 3 clinical trial is currently ongoing in 30 centers across Europe, the U.S. and Canada. PXT864, Pharnext’s second lead PLEODRUG™, with a novel mechanism of action, completed a Phase 2a for Alzheimer’s disease with positive encouraging data. In this presentation, Mr. Paoli and Dr. Hajj will:
♦ Describe the PLEOTHERAPYTM R&D platform based on network pharmacology
♦ Illustrate the proof of concept of this platform through the presentation of both clinical lead projects: CMT1A and Alzheimer’s disease
♦ Introduce Pharnext’s business model and growth strategy for years to come
Xavier Paoli, CCO and VP of R&D Operations, PharNext SA
Rodolphe Hajj, Ph.D., Chief Pharmacology Officer, PharNext SA 
11:35 am Drug Repurposing Meets Personalized Medicine
Pairnomix will address the convergence of genetic sequencing, patient-specific mutation models, and comprehensive drug repurposing screening to identify new therapeutic options in rare disease. They will present a case study for Nav1.6 sodium channels (SCN8A) as well as additional ongoing work in other ion channels, and will discuss future directions for therapy development beyond genetic epilepsy. 
Gregory Stewart, Ph.D., Chief Scientific Officer, Pairnomix
12:05 pm Luncheon
1:05 pm PANEL SESSION: New Technologies and Methods to Advance Systematic Repurposing
Paul Bogdan, Ph.D., Assistant Professor, Department of Electrical Engineering, University of Southern California
Ronald Alfa, MD, Ph.D., Vice President, Discovery and Product, Recursion Pharmaceuticals
Gregory Stewart, Ph.D., Chief Scientific Officer, Pairnomix
Rodolphe Hajj, Ph.D., Chief Pharmacology Officer, PharNext SA
1:40 pm De-risking, Not Discounting: Understanding the Economics of Drug Repurposing
What is the promise of drug repurposing? Commercial and philanthropic drug developers both tend to answer this question in economic terms: it is supposed to be significantly cheaper and quicker than developing a new chemical entity because it allows the re-developer to „piggyback“ on what the original developer has already done. The savings potential is often reported as being dramatic. This presentation will discuss the extent of cost-cuts and development acceleration that is achievable during the stages of development, depending on the status of the compound being re-developed for a new indication. It will also make the case that the most significant advantage offered by drug repurposing lies not so much in direct savings but rather in de-risking of early-stage and mid-stage development, and should be handled as part of a comprehensive risk management strategy.
Hermann Mucke, Ph.D., Chief Executive, H.M. Pharma Consultancy
2:10 pm Shots on Goal: Repositioning and Repurposing for Fragile X
Since 1994, FRAXA Research Foundation has been funding research on disease mechanisms of fragile X syndrome, the most common genetic cause of intellectual disability and autism. Translational studies have identified many promising therapeutic targets, including quite a few which can be accessed with available drugs. Many clinical trials of available and investigational drugs have emerged from this work, but results have been mixed. This presentation will discuss the lessons learned from efforts to repurpose available drugs and reposition investigational drugs for the treatment of fragile X. 
Michael Tranfaglia, MD, Medical Director, Chief Scientific Officer, FRAXA Research Foundation
2:40 pm Refreshment Break
3:05 pm The Story of Fenfluramine in Dravet Syndrome
In this presentation Dr. Legae will discuss his team’s involvement in the discovery of the SCN1A mutation in Dravet syndrome; how they found out that the few photosensitive patients responding to fenfluramine actually were children with Dravet; how they then designed their clinical study (paper Epilepsia 2012); how they are now working together with Zogenix to get fenfluramine on the market; and how they started translational research in a zebrafish model to confirm the efficacy. 
Liven Lagae, MD, Ph.D., Professor, Paediatric Neurology, Katholieke Universiteit Leuven, President, European Society for Paediatric Neurology
3:35 pm Repositioning in the Age of Big Data and Artificial Intelligence
Positioning drugs and targets across multiple possible uses is a tactical advantage many companies are working to embrace. In the age of Big Data and AI, new technologies make it possible to capture opportunities that traditional screening would be too long and too expensive to do. Such technologies show how we can re-imagine how drugs are developed.  
Aris Persidis, Ph.D., Chief Executive Officer, Biovista
4:05 pm Crowdsourcing Wisdom About Drug Repurposing/Repositioning Challenges
In this presentation, Dr. Austin will discuss the following:
♦ Overview of NCATS New Therapeutic Uses Programs
♦ Challenges in need of solutions
♦ Crowdsourcing audience ideas
Bobbie Ann Austin, Ph.D., Program Officer, Drug Development Partnership Programs, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Office of the Director
4:35 pm Lead Repurposing as an Effective Approach for Neglected Tropical Disease Drug Discovery
Dr. Pollastri’s current research has been broadly focused on addressing parasitic disease by repurposing known classes of investigational agents that have been successfully pursued for other indications. For example, entire families of kinase inhibitors discovered for cancer indications can often be redirected and optimized for potency against parasite cells. Dr. Pollastri and his team’s efforts to repurpose kinase-targeting chemotypes as new lead compounds for neglected tropical diseases will be presented. In particular, their model of distributed drug discovery, which involved pharma industry collaborators, will be highlighted.
Michael Pollastri, Ph.D., Associate Professor, Chair of Chemistry & Chemical Biology, Northeastern University
5:05 pm End of Day's Presentations

June 28, 2017

8:20 am Chairperson's Opening Remarks
Dr. Bruce Bloom, President and Chief Science Officer, Cures Within Reach
8:25 am Repurposing Generic Drugs as New and Affordable Cancer Treatments
Cures Within Reach for Cancer aims to fund and facilitate the development of repurposed generic drugs for cancer. In this presentation, Dr. Kleiman will discuss plans for 1) a KnowledgeBase to systematically assess and prioritize repurposing opportunities, 2) Phase II/III clinical trials, and 3) advocacy for the treatments to be incorporated into standard of care so all cancer patients can access and benefit from them. 
Laura Kleiman, Ph.D., Executive Director, Cures Within Reach for Cancer
8:55 am Repurposing Drugs in Oncology: Challenges and Opportunities
Vidula Sukhatme, Founder & CEO, Global Cures
9:25 am Case Study: The Development of APL-1202 and Its 2nd-Generation APL-1301 for the Treatment of Genito-Urinary Cancers
Asieris Pharmaceuticals is a biotech company focusing on transmuting knowledge from old drugs for the development of innovative drugs. APL-1202, an obsolete antibiotic approved for urinary tract infections, is currently in a pivotal clinical trial in China for non-muscle invasive bladder cancer (NMIBC). Using APL-1202 and its 2nd-generation APL-1301 as examples, the presentation tries to elucidate Asieris’ approaches of how to capitalize the advantages while addressing the challenges from drug repositioning.
Kevin Pan, Ph.D., Co-Founder, Asieris Pharmaceuticals
9:55 am Refreshment Break
10:15 am Presentation Title to be Announced
Scott Weir, Pharm.D., Ph.D., Frank B. Tyler Cancer Reseach Professor in Therapeutic Discovery, Director, Institute for Advancing Medical Innovation, University of Kansas, Associate Director – Translational Research, University of Kansas Cancer Center
10:45 am PANEL SESSION: Repurposing for Oncology - Emerging Candidates
Laura Kleiman, Ph.D., Executive Director, Cures Within Reach for Cancer
Kevin Pan, Ph.D., Chief Executive Officer, Asieris Pharmaceuticals
Vidula Sukhatmae, Founder and Chief Executive Officer, Global Cures
11:15 How to Build and Nurture Collaborations in Repurposing Research 
Strategic partnerships are critical in repurposing research.  Each stakeholder adds value and resources. From identifying academic collaborators to incorporating the latest technology to securing funding, bringing the right stakeholders together can help successfully drive repurposing solutions to patients. Cures Within Reach has experience creating collaboration tools and opportunities, and this presentation will describe successes and failures, best practices and a framework for future collaborations. 
Dr. Bruce Bloom, President and Chief Science Officer, Cures Within Reach
Clare Thibodeaux, Ph.D., Director of Scientific Affairs, Cures Within Reach
11:45 am Presentation Title to be Announced
Pieter Dylst, Senior Manager Market Access and Value Added Medicines, Medicines for Europe
12:15 pm Luncheon
1:15 pm Presentation Title to be Announced Using Human Genetic Variation to Repurpose Existing Medications for New Diseases
BioVU, a DNA database linked to Vanderbilt University Medical Center (VUMC) de-identified electronic health records (EHRs), is a large-scale resource for deep genome-phenotype information captured within a health care delivery system. Dense genome-phenome associations contained in BioVU can be utilized to predict previously unappreciated clinical outcomes of modulating a given gene product with a pharmaceutical. As illustrated by the case studies, our interdisciplinary approach, which we term Human Biotarget Discovery, is being implemented to accelerate repositioning or repurposing of existing pharmaceuticals for important unmet clinical needs. 
Charles Hong, MD, Ph.D., FAHA, Associate Professor of Cardiovascular Medicine, Pharmacology, and Cell and Developmental Biology, Chair, Accelerating Drug Repurposing Incubator, Vanderbilt University
1:45 pm Presentation Title to be Announced
Daphna Laifenfeld, Ph.D., Senior Director, Personalized and Predictive Medicine, Teva Pharmaceutical Industries
2:15 pm Open Innovation: Harnessing the Power of Industry/Academic Partnerships 
Drug development is a daunting task, and the inherent challenges will require us to re-think our approach. A shining example of pioneering innovation is the Open Innovation Initiative at Astra Zeneca where we have sanctioned the vision of openly collaborating with the academic, government, and medical community key stakeholders to leverage existing resources and compounds to deliver value to patients in a novel way. Through our Open Innovation platform ( we invite external investigators and clinicians across all stages of drug discovery to collaborate. This model, despite being only three years old, is proving to be a success. For example, 6 of 22 ongoing phase II studies have completed with 4 of those yielding positive outcomes with one that will be discussed in a case example. Our unique approach illustrates a successful model of collaboratively repurposing to meet the unmet needs of patients.
Chad Nivens, Ph.D., Director, Translational Science, Innovative Medicines & Early Development Biotech Unit, AstraZeneca Pharmaceutical LP
2:45 pm End of Conference

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