2019 Conference Agenda

September 23, 2019
7:30 am Workshop Registration & Continental Breakfast
(separate registration required for workshop)

8:00 am Workshop: Intellectual Property (IP) Boot Camp – Basics on Protecting Your IP and Respecting Other’s IP
Intellectual property basics with a focus on repositioned drugs will be covered. This interactive and practical session will provide a basic overview of patents, copyrights and trademarks, while also answering questions about preparing patent applications in the drug repurposing space and how to file for a patent in the United States and abroad, with a focus on cost-saving and time-saving techniques, important legal updates for patent owners, and best practices for securing patent rights worldwide. IP strategies for positioning your company for a successful acquisition or exit will also be covered, along with IP concerns associated with commercializing your product. Entrepreneurs, investors, startups, inventors and anyone interested in learning how intellectual property and patents can help in the drug repurposing space will benefit from the workshop.
Workshop Leader: Rose Thiessen, JD, Ph.D., Partner, Knobbe Martens

10:00 am Main Session Registration Begins 


10:30 am Novel Approach to Drug Repurposing by Mining Safety Data and AI-based Simulation of Clinical Trials
In this presentation Dr. Schneider will present Molecular Health’s proprietary approach to identify repurposing candidates. Clinical observations have historically been proven to be one of the most prominent sources for drug repurposing hypotheses. Molecular Health has taken FDA’s vast set of clinical safety reports, cleaned and mapped it to their knowledge base of drugs, targets and pathways, and leverage the analytics of their software application MH Effect to rapidly identify drug candidates. He will present examples of repurposing projects in tuberculosis and neonatal health. Further he will showcase an in silico feasibility assessment, by simulating clinical trial success using our AI-driven software MH Predict, to support valuation and planning of the repurposed drug’s development program.
Armin Schneider, MD, VP Scientific & Medical Affairs, Molecular Health

11:00 am In Silico Drug Repositioning: From Cellular Networks to Personalized Treatment
The recognition that drug targets and disease mechanisms in the human sub-cellular interactome as finding the most possible configuration of cellular networks has shifted the focus of therapeutics and health care from the “one-drug, one-target” paradigm to the “multiple-drugs, multiple-targets” paradigm, which has a big impact at identifying actionable approaches for precision medicine. Dr. Cheng will present state-of-the-art network medicine approaches that seek to redefine human disease and therapeutics from an integrated perspective using innovative omics (i.e., genomics, proteomics, and interactomics), systems biology, and modern network science technologies, and along with large-scale patient longitudinal data (e.g., health insurance claims) – offers a non-invasive way to identify novel real-world data-driven actionable biomarkers and therapeutic targets for cancer and cardiovascular disease. Specifically, he will illustrate that a network medicine infrastructure that measures network interrelationship between drug targets and disease-gene products in the human protein-protein interactome for ultimately accelerating innovative therapeutics development (such as drug repositioning and network-based prediction of drug combinations).
Feixiong Cheng, Ph.D., Assistant Professor Molecular Medicine,
Cleveland Clinic Lerner College of Medicine

11:30 Open Innovation Through Partnership: Past, Present and Future
AstraZeneca pioneered a model of government-sponsored drug repositioning collaborations through its “Open Innovation” portal, which offers a range of compounds for which academic investigators can submit new repositioning ideas and translational research. Since its inception in partnership with MRC and NIH/NCATS 8 years ago, the program has spawned a significant number of preclinical and clinical drug development projects. The outcomes of these projects in turn delivered novel scientific insights, generated commercial value or new investment from out-licensing activities, and re-integrated assets back into AstraZeneca’s internal pipeline. In this presentation, I will highlight progress of selected high-impact clinical projects to exemplify the diverse approaches we have undertaken to conceive and advance therapeutic hypotheses. In addition, I will discuss strategies to overcome some of the challenges we faced in order to achieve sustained success of this platform or realize the full potential of drug repurposing.
Dong Liu, Ph.D., Associate Director of Translational Science, AstraZeneca

12:00 pm Amplifying Human Capability with Artificial Intelligence and Machine Learning to Accelerate and De-risk Drug Discovery and Development
The multifaceted nature of diseases presents an ideal opportunity to apply artificial intelligence (AI) and machine learning (ML) to uncover therapeutic insights that hone in on the “best-suited” treatment options and the ideal mode of pharmacotherapy (antibody, protein replacement, siRNA, mRNA, small molecule, cell and gene therapy, and gene-editing modalities). InveniAI’s platform, AlphaMeld®, generates testable hypotheses, therapeutic candidates, and insights that can be sorted based on disease severity, gene ontology, disease pathways, proteinopathies, standard of care, emerging innovation, and enabling technologies while factoring in medical, scientific, strategic, and commercial considerations. To serve as examples the following use cases will be presented: BXCL701, a DPP8/9 and FAP inhibitor, as an immuno-oncology candidate for treatment-emergent neuroendocrine prostate cancer (tNEPC), and BXCL501, a selective alpha 2a receptor agonist for the treatment of acute agitation.
Krishnan Nandabalan, Ph.D., CEO & President, Inveni AI

12:30 pm Luncheon

1:30 pm Lupus Research Alliance and Drug Repurposing: A Patient Advocate Organization's Approach
Please join us to hear how a patient advocacy organization that focuses on research is approaching and utilizing drug repurposing to find appropriate treatments for a disease with a dearth of treatment options currently. Al Roy the Executive Director of Lupus Therapeutics, an affiliate of Lupus Research Alliance (the largest private funder of lupus research in the world) will share what the LRA has accomplished recently in this area with a number of drugs through corporate and academic investigator collaborations and more on what new partnership opportunities in lupus drug repurposing may be available going forward.
Al Roy, Executive Director, Lupus Therapeutics (an Affiliate of the Lupus Research Alliance)

2:00 pm Phenotypic Screening Is a Powerful Approach Towards Drug Repositioning, Repurposing and Rescue
We will first provide an overview of an approach that we have been using for the last 15 years to uncover otherwise unpredicted new uses of existing drugs, drug candidates, and uncharacterized chemical matter. A basic rationale for the approach, outline of the methods, experience summaries, and lessons learned will be provided. We will then illustrate the approach and highlight its capabilities with a more detailed account using an example of our lead compound which has successfully progressed in clinical development. The example will especially serve to illustrate why this compound – therapeutic area linkage may only have been uncovered by phenotypic screening.
Andrew Reaume, Ph.D., MBA, President & CEO, Co-Founder, Melior Discovery
Christopher Lipinski, Ph.D.
, Scientific Advisor, Melior Discovery

2:40 pm AIs That Make Sense, Those That Don’t, and the Massive Use in (re)Positioning
AI is the only realistic platform for the data needs of healthcare going forward. However, despite the market being saturated with only one form of AI, machine learning (ML), not all AIs are created equal. We need to look beyond the key limits of ML and match the right tool for the job. The major use case of (re)Positioning will be covered, as well as others.
Aris Persidis, Ph.D., President, Biovista

3:10 pm Refreshment/Networking Break


3:40 pm From Data Science to Machine Learning in Drug and Target Repurposing
In this presentation, Dr. Oprea will:
  • Discuss the knowledge-based classification of human proteins and its applications in target repurposing discovery, with potential applications for Rare Diseases
  • Cover his team's on-going Machine Learning efforts to predict novel protein-disease associations for Alzheimer’ Disease, cardiac phenotypes, and Type 2 Diabetes
  • Address the limitations and potential of machine learning in target and drug repurposing
Tudor Oprea, Ph.D., Prof. of Medicine, Chief, Translational Informatics Div., Dept. of Internal Medicine, University of New Mexico

4:10 pm Systematic Drug Repurposing: Our Experiences with Computational Approaches and Experimental Verification
In this presentation Dr. Agarwal will discuss the following:
  • His team's experience running a small biotech within pharmaceutical R&D focused on computational repurposing
  • Recently published examples on the use of sEH inhibitor for IBD and wound healing
  • Current methods for computational drug repurposing
Pankaj Agarwal, Ph.D., Senior Fellow & Senior Director, GlaxoSmithKline

4:40 pm Data Repurposing for Drug Repurposing
This presentation will focus on how existing data can be harnessed to “guide” or “inform” translational medicine Using case studies, Dr. Jegga will present how available data can be repurposed to identify preclinical candidate therapeutics or understand the molecular basis for drug response.
Anil Jegga, DVM, Professor, Division of Biomedical Informatics, University of Cincinnati College of Medicine

5:10 Cocktail Reception

September 24, 2019

7:45 am Continental Breakfast

8:15 am Making Better Repurposing Decisions: Integrating Evidence from Multiple Sources Including Human Experience
Drug repurposing aims to reduce risk of failure in drug development, as well as reduce cost and time to market entry. There are more potential opportunities in drug repurposing than resources to develop them, and decision making is all the more important because early studies are likely to be expensive. Human evidence of the effects of drugs outside their primary indication can be derived from retrospective trials in a number of areas, including cancer, sepsis, Alzheimer disease and depression. Lower quality information is also increasingly available from mining of social media. This approach can be combined with pathway analysis to provide first-in-class treatments for complex diseases. Alternatively, it can be combined with prospective preclinical studies to uncover a validated mechanism for a new indication, after which a repurposed molecule is chemically optimized.
David Cavalla, Ph.D., Executive Director, Exvastat, Founder, Numedicus

8:45 am Effective Formal Interactions with US-FDA for 505(b)(2) Regulatory Pathway – Our Experience
In this talk, Dr. Jadhav will elaborate on the importance of having the right drug development strategy (clinical, formulation, TPP) for specific formal interactions with the different divisions of the US FDA. Setting the right path can fulfill the aim of getting maximum benefits/waivers from the Agency. Dr. Jadhav will also cover specific case studies to illustrate this point.
Manoj Jadhav, Ph.D., FCP, Translational Clinical Pharmacologist, CRC Pharma, LLC

9:15 am Oncology Repurposing Engine: Creating New and Affordable Cancer Treatments Using AI to Repurpose Non-Cancer Generic Drugs
In this presentation, Dr. Kleiman will discuss AI-powered synthesis and prioritization of drug repurposing data from scientific literature and real-world evidence. She will also discuss new models for funding and conducting clinical validation, and for implementing repurposed therapies as standard of care. Finally, she will cover the importance of collaborating across disciplines and sectors for social impact.
Laura B. Kleiman, Ph.D.
, Founder and Executive Director, Cures Within Reach for Cancer

9:45 am New and Existing Incentives to Support Drug Repurposing

Drug repurposing has grown to be a significant part of therapy development. This presentation will review some of the existing incentives that support repurposing, and will describe other programs that could be implemented to further support repurposing, including differential pricing, blockchain technology, social impact bonds, and new regulatory drug approval processes.
Dr. Bruce E. Bloom, Founder, Cures Within Reach

10:15 am Refreshment/Networking Break

10:45 am Drug Life-cycle Management: Challenges, Opportunities and Strategies
Launching a new drug is lengthy and costly process that leaves only a small amount of time for Pharma companies to generate revenues before the drug becomes generic. While the clinical benefit to patients continues, the profitability to pharma companies ceases soon after the expiry of the patent. Maximizing the product value through innovative Life-cycle management (LCM) strategies is essential for the drugs to preserve exclusivity in the market and continue to fuel the revenue that supports further R&D development. A thorough understanding of the drug molecules and their approvals across regulatory markets provides market expansion opportunities. In addition to the drug repurposing, the presentation will discuss various LCM challenges, opportunities and strategies.
Nandu Gattu, Ph.D.
, Sr. Vice President, Pharma Analytics, Excelra

11:15 am Panel Session Hosted by HealX 
Panelists to be announced soon

12:15 pm Luncheon

1:15 pm EspeRare: Groundbreaking Pivotal Testing of an Antenatal Treatment for a Genetic Disease
As a patient-centered nonprofit model, EspeRare is bringing to life and fast-forwarding innovative therapeutic interventions for rare diseases using a drug rescue approach. This is illustrated by its ER004 program, currently in pivotal development for X-link ectodermal dysplasia using an antenatal administration approach. The presentation will discuss how the program was rescued and the key drivers of success of this breakthrough program.
Caroline Kant, Founder and CEO, EspeRare Foundation

1:45 pm Patent Law Roundup: What You Need to Know about Patentable Subject Matter and Other Legal Developments Impacting Exclusivity for Repurposed Drugs
2019 has seen many new developments in the area of patentable subject matter, including new USPTO guidelines and proposed legislative reform in Congress. Learn about how these developments, as well as other legal decisions, will impact your ability to pursue patent protection or other forms of exclusivity on repositioned drugs.
Rose Thiessen, JD, Ph.D., Partner, Knobbe Martens

2:15 pm CURE ID: Capturing Clinician’s Experiences of Repurposing Drugs to Identify Innovative Treatments for Infectious Diseases Lacking Adequate Therapy
CURE ID is an FDA-NIH collaborative endeavor that deploys a web-based platform and mobile app, providing clinicians throughout the world an effective mechanism by which to collect and share real-world experiences treating patients for infectious diseases that lack adequate treatment options. This may include neglected tropical diseases, emerging infectious threats, and infections caused by antimicrobial-resistant organisms. By inputting case reports via an easy to fill-out case reporting tool, real-world clinical outcome data will be accumulated which can directly assist in identifying potential drug candidates for more formalized clinical studies. Because these drugs are already approved for one or more indications, significantly more is known about them and the cost and time of completing a formal drug development program should be reduced. Moreover, the increasing accumulation of clinical case-report data (that is systematically collected and aggregable) by using this platform may aid clinicians in underserved populations with making individual patient treatment decisions in the absence of established safe and effective drug treatment options.
Heather Stone, Health Science Policy Analyst, Office of Medical Policy, Center for Drug Evaluation and Research, US Food & Drug Administration

2:45 pm How Machine Learning, Genomics and AI are Revolutionizing Cancer Drug Development
Machine learning and artificial intelligence are reshaping and transforming every industry, including the high stakes world of drug discovery and development. It’s a unique point in time for the biopharma industry due to: the tremendous decrease in computing costs; the exponential increase in the speed and power of computing resources; widely available genomic data and real world patient data; and regulatory pathways that have been created to accommodate personalized and precision medicine. The traditional, expensive and time consuming process of drug development is being transformed and made more efficient by leveraging AI and rescuing or revitalizing drug compounds that had failed earlier clinical trials due to low efficacy in a general population or been abandoned by companies due to lack of understanding about the mechanisms of action. Lantern Pharma is harnessing modern AI advances and genomic data to revitalize cancer therapies by pinpointing the patients that will respond and benefit from the drug. This precision oncology drug development approach leverages the insights from Lantern’s AI platform, RADR, to both identify patient populations suitable for Lantern’s drug portfolio and aid in the rapid development of companion diagnostics.
Panna Sharma, CEO, Lantern Pharma

3:15 pm End of Conference