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., President, 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 Value Added Medicines: The European Experience with Repurposed Medicines
In this presentation Mr. Dylst will answer the following questions:
♦ What are value added medicines?
♦ How is Europe is dealing with repurposed medicines?
♦ What is preventing value added medicines from coming to market in Europe?
Pieter Dylst, Senior Manager Market Access and Value Added Medicines, Medicines for Europe
 
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 The Ability of Nutraceuticals to Alter RNA Processing: An Untapped Resource for the Treatment of Human Diseases
The alternative RNA splicing process, which enables the production of numerous gene products from a single gene, is dependent on specific nucleotide sequences present within the genome and the cellular expression of splice-regulating proteins. It has been estimated that approximately 60% of human genetic diseases are due to mutations that are located in sites that impact the splicing process. Dr. Rubin and his team have developed novel nutraceutical-based therapeutic modalities that reverse the impact of the RNA splice-altering mutation that causes Familial Dysautonomia (FD), a genetic disorder that compromises the development and survival of sympathetic and parasympathetic neurons. Their success at altering the RNA splicing process with nutraceuticals has led to efforts targeting the development of RNA splice-altering therapies for the treatment of Spinal Muscular Atrophy, Niemann–Pick disease and osteoporosis.
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
Panelists: 
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. 
Lieven 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., President, 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

 
8:00 am Chairperson's Opening Remarks
Dr. Bruce Bloom, President and Chief Science Officer, Cures Within Reach
 
8:05 am Repurposing Drugs in Oncology: Challenges and Opportunities
The scientific literature points to many FDA approved non-cancer drugs that could be re-purposed for cancer use. Lack of funding for definitive clinical trials is a major obstacle, since in many cases there is little financial return on investment. In this presentation, Ms. Sukhatme will discuss several other challenges including the need to formalize a comprehensive database of drug candidates, identify disease populations and biomarkers to predict in which patients these drugs will be most effective, design combinations based on synergistic mechanisms of action and conduct trials in a cost-effective way. Several criteria that could be used to prioritize promising therapies and treatment opportunities ready for patient use will also be provided.
Vidula Sukhatme, Founder & CEO, Global Cures
 
8:35 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
 
9:05 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
 
9:35 am Refreshment Break
 
9:50 am An Innovative Public-Private Partnership to Discover and Develop New Treatments for Cancer and Rare Diseases
The vision, mission and approach of our NCI Cancer Center to discovering and developing new as well as repurposed drugs for the treatment of cancers and rare diseases will be described.  Central to this strategy is leveraging the unique assets of the Kansas City region with a goal of growing the biotechnology community.  A unique public-private partnership has been established with a local investment group with a shared goal of developing and commercializing drug as well as diagnostic products.  The Ciclopirox Prodrug drug development program was presented at the 4th Annual Drug Repositioning, Repurposing and Rescue Conference as one approach to drug repurposing.  An update on this project will be presented illustrating how academic and for-profit partners are advancing this new cancer treatment to patients.  The presentation will lastly describe how this collaboration model is being applied to rare diseases in partnership with the patient-driven RareKC initiative.
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
 
10:20 am PANEL SESSION: Repurposing for Oncology - Emerging Candidates
Panelists:
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
 
10:50 am 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
 
11:20 am Novel Automated Workflow that Leverages Data Integration for Drug Repurposing
Challenges facing rare disease research include scarcity and scattered distribution of data relative to finding new treatments and/or repurposing existing ones. In collaboration with the UK charity Findacure, Elsevier developed an automated workflow utilizing literature-extracted disease, target, and bioactivity data, as well as key opinion leader analysis, to support current disease research, inform drug repurposing decisions, and advance clinical trials for rare diseases. An example of this workflow using congenital hyperinsulinism as a model will be discussed, as well as future applicability to other diseases.
Maria Shkrob Ph.D., Professional Services Consultant, Elsevier
  
11:50 pm Luncheon
 
12:50 pm Presentation Title to be Announced
Speaker TBA, DiscoverX
 
1:20 pm 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
 
1:50 pm Panel Session: The Pharma Perspective on Repurposing Using Multimodal Sources
In this panel session, our panelists will discuss the means by which the pharma industry is engaging with partners to repurpose. Topics will include:
♦ The pharmaceutical industry perspective and approach to repurposing
♦ Real World Evidence and drug similarity approaches
♦ Omic and molecular approaches to repurposing 
Chair: Daphna Laifenfeld, Ph.D., Senior Director, Personalized and Predictive Medicine, Teva Pharmaceutical Industries
 
2:35 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 (www.openinnovation.astrazeneca.com) 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
 
3:05 pm End of Conference

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