DAY ONE - JUNE 21, 2016
7:25 am Registration/Continental Breakfast
8:00 am Chairperson's Opening Remarks
Dr. Bruce E. Bloom, President and Chief Science Officer, Cures Within Reach
8:05 am Overcoming Philanthropic and Commercial Roadblocks to Drug Repurposing, Repositioning and Rescue
Drug repurposing, repositioning and rescue can create solutions for unsolved diseases, but there are many economic and other obstacles prevent these potential treatments and cures from reaching patients. This presentation will describe these obstacles and then present a series of current and future initiatives designed to overcome these roadblocks and spur both philanthropic and commercial development of repurposed, repositioned and rescued therapies.
Dr. Bruce E. Bloom, President and Chief Science Officer, Cures Within Reach
8:35 am Genomics Driven Drug Repurposing in Neurodegenerative Diseases
Neurodegenerative therapeutic development is littered with clinical failures of multiple pharmacological mechanisms and treatment modalities. These failures can be attributed to several limitations. First, many clinical candidates were originally developed against targets with weak or no genetic link to human disease. Second, neurodegeneration is caused by complex interactions between hundreds of genes, and therefore reductionist drug discovery strategies focused on a single toxic target are unlikely to be effective at reversing disease progression. Verge Genomics has developed a sophisticated systems biology platform that uses network algorithms on genomic patient data to accelerate drug discovery for CNS diseases. They integrate large-scale gene expression, genetic variation, and molecular datasets to construct predictive disease network models to identify important gene networks driving disease progression, elucidate subtypes of syndromic diseases, and develop a novel “all-in-human” drug-screening platform when combined with patient-derived iPSCs.
Alice Zhang, Chief Executive Officer, Verge Genomics
9:05 am Drug Repurposing for Black Bone Disease
Black Bone Disease (also known as Alkaptonuria, or AKU) is a severely disabling rare disease caused by a malfunction in tyrosine metabolism. Nitisinone is a drug currently used to treat another rare disease called Hereditary Tyrosinemia Type 1. This talk will present how a team led by the Royal Liverpool University Hospital and the AKU Society patient group repurposed nitisinone for Black Bone Disease by creating an international consortium and a national treatment centre to drive clinical research.
Dr. Nicolas Sireau, Chairman, AKU Society, Findacure
Lakshminarayan Ranganath, Ph.D., Director, National AKU Centre, Coordinator, DevelopAKUre Consortium, Honorary Professor in Musculoskeletal Biology, University of Liverpool, Consultant, Royal Liverpool University Hospital
9:35 am A Corporate Venture Viewpoint on Investing in Repurposed Drug Companies
A pharma corporate venture's viewpoint on creating a winning strategy for drug repositioning, including a behind-the scenes view/case study on diligence for a venture investment in a repurposed Alzheimer's drug company. The discussion will center around scientific, commercial, IP, product development, regulatory and operational considerations that are typically considered during diligence for investment in repositioning opportunities.
Vikram Sudarsan, Head, Cipla Technologies, Cipla New Ventures
10:05 am Refreshment/Networking Break
10:30 am 505(b)(1) Processes Don't Work for Positioning 505(b)(2)s
One of the biggest mistakes made in developing a 505(b)(2) product is in applying 505(b)(1) FDA guidances and processes to 505(b)(2) drug repositioning. This mistake affects the timing, resources, and availability of funding, and causes inappropriate and wasteful study design. In his hour-long talk, Ken will outline a resource-efficient development plan that participants can apply toward their drug repositioning projects.
Kenneth Phelps, President and CEO, Camargo Pharmaceutical Services
11:30 am A Multi-pronged Approach to Identify Alternative Indications
The presentation will showcase collective efforts & benefits of utilizing our Global Repurposing Integrated Platform (GRIP), algorithms designed for repurposing analytics and visualization tools to identify the hidden associations between the drug-target-disease. We will also highlight the value generated for our partners and touch upon our Repurposing portfolio.
Nandu Gattu, Ph.D., VP Drug Repurposing, Excelra - Knowledge Solutions
12:00 pm Luncheon
1:00 pm The Development of Thalidomide and Subsequent Compounds
Thalidomide is the most notorious drug in pharmaceutical history. Unfortunately, its anti-emetic and sedative properties led to the discovery of thalidomide being a human teratogen. Thalidomide was subsequently discovered to have a number of human biological properties that resulted it being used to treat both chronic inflammatory diseases and neoplasms. This ultimately led to the drug’s use in treating multiple myeloma. Based on thalidomide’s activity for a variety of illness, successor compounds that were developed that have meaningful results in treating both neoplasms and chronic inflammatory diseases.
Jerome Zeldis, MD, Ph.D., Chief Executive Officer, Celgene Global Health, Chief Medical Officer, Celgene Corporation
1:30 pm Alliance for Lupus Research on Creating a Clinical Investigator Network and Working with Industry in Drug Repositioning
Al Roy, Executive Director of LuCIN (ALR's Lupus Clinical Investigator Network) will share with the audience how the Alliance for Lupus Research formed a network of investigators from 60 academic sites to help work on lupus clinical trials in drug repositioning. You'll leave this session with a working model to consider for governance structure and execution of trials through a network of sites. Kim Kaiser-Rachidi, Special Consultant to Alliance for Lupus Research on Industry Relations will speak about how they are reaching out to companies who make drugs they hope to reposition for lupus whether an already marketed product or still in the pipeline. She will also speak about some of their early challenges and successes in collaborations with industry to utilize LuCIN.
Al Roy, Executive Director, Lupus Clinical Investigators Network, Alliance for Lupus Research
Kim Kaiser-Rachidi, Special Consultant-Industry Relations, Alliance for Lupus Research
2:00 pm MRI Guided Laser Ablation of Prostate Cancer
Focal therapy has been successfully applied to treatment of various cancers in the body but is an emerging paradigm for prostate cancer. With the support of “Cures within Reach Foundation” and NIH, Dr. Oto's group has performed Phase I and Phase II clinical trials for MR guided laser ablation of prostate cancer and now started to offer this procedure as a standard of care alternative procedure to our patients. This presentation will cover their story and the role of focal laser ablation in prostate cancer.
Aytekin Oto, MD, Professor of Radiology, Section Chief, Abdominal Imaging, Chief of Body MRI, The University of Chicago
2:30 pm From the Past to the Future: How Repositioning is Evolving and Resetting Industry Norms
Repositioning has been around for a while, primarily as a serentiditous activity. Now, it is practiced more systematically. The recent ecosystem has evolved dramatically, with new technologies, new patent implications, new regulatory milestones and new types of business deals resetting the norms and expectations in the field.
Aris Persidis, Ph.D., President, Biovista
3:00 pm Refreshment/Networking Break
3:25 pm Big Data to Clinical Programs with a High Probability of Success
Dr Vimal’s presentation will showcase how his Company’s Bid Data Innovation Lab has established an ecosystem to identify new therapeutic candidates that have a high likelihood of clinical and commercial success. With a platform built on ten years of research and analytics we harness the power of proprietary algorithms that delve into the vast pharmaceutical knowledge space, including more than 7,000 active clinical pipeline candidates, discontinued and shelved clinical programs, and more than 10,000 marketed drugs to uncover novel connections between target, disease and pharmacological agents. Partners leverage our Lab for their portfolio expansion initiatives including the repurposing of shelved assets. Dr Mehta will also describe how internal efforts have yielded a portfolio of three clinical candidates that span cancer immuno-therapies and CNS orphan drugs.
Vimal Mehta, Ph.D., Chairman and Chief Executive Officer, BioXcel
3:55 pm The Discovery of AZT, the First Drug to be Used in HIV/AIDS: A Repurposing Success Story
In this presentation, Dr. St. Clair will discuss the discovery of the activity of AZT against HIV and the process resulting in the regulatory approval of AZT for the use in HIV infected persons.
Marty St. Clair, Ph.D., Clinical Virology, ViiV Healthcare
4:25 pm Notable Labs: A CLIA Laboratory Platform for Repurposing Combinations of FDA Approved Drugs for Individual Refractory Cancer Patients
Notable Labs is currently in clinical trials in relapse Acute Myeloid Leukemia testing the predictive power of a combinatorial repurposing screening platform. They've developed an individualized laboratory testing service for cancer patients and their doctors. They screen thousands of FDA-approved drugs against the patients own cancer cells to identify drug combinations that can be immediately prescribed by their doctor without a clinical trial. To ensure this translational system is predictive they’ve created a proprietary method to mimic the microenvironment of the human body. Their drug panel includes all approved chemotherapies and targeted agents, as well as generic non-oncology drugs that have published anti-neoplastic evidence for repurposing. They prioritize the safest combinations from their viability assay by estimating a therapeutic index with healthy cells. The end result of their process is a CLIA certified report which prioritizes therapeutic options that could be used by the physician and patient.
Matthew DeSilva, CEO, Notable Labs
4:55 pm Nutraceuticals as Repositioning Agents
Nutraceuticals (i.e., food-derived bioactive compounds) are a relatively untapped resource for novel drug discovery. Attributed mainly to limited regulatory control and a general lack of scientific rigor, these compounds are dismissed as potential therapeutics. However, given that these compounds possess biological activity, are used successfully clinically (in a few situations), and the existence of fundamental safety data, nutraceuticals are prime repositioning candidates. This presentation will focus on how nutraceuticals could be useful in therapeutic applications provided rigorous scientific assessment.
Paul Spagnuolo, Ph.D., Assistant Professor, University of Waterloo
5:25 pm Cocktail/Networking Reception
DAY TWO - JUNE 22, 2016
7:25 am Continental Breakfast
7:55 am Chairperson's Opening Remarks
Aris Persidis, Ph.D., President, Biovista
8:00 am Data Integration in Drug Repurposing: Marrying Literature-based, Laboratory, and Algorithmic Outputs
Systematic drug repurposing is increasingly dominated by in silico and bioinformatics approaches, driven by sophisticated algorithms that are designed to identify new drug-target relations across the human and pathogen genomes and transcriptomes. On the other hand, high-content cell-based drug screening approaches can generate not only quantitative biomarker data, but also a huge amount of "soft" imaging data. Finally, contextual mining of the published literature (peer review papers, patent documents, and congress reports) for implicit information can be nested with our knowledge of biological pathways to provide yet another perspective. These three approaches ("silicon", "wet," and "text/network") generate vastly disparate outputs for which few integrative paths are evident. This presentation will discuss how we could eventually move from a sequential to a holistic strategy of information extraction to support the redevelopment of known active ingredients.
Hermann Mucke, Ph.D., Principal, HM Pharma Consultancy
8:30 am Drug Repositioning by a Computational Approach: Two Successful Cases
Dr. Horimoto’s team discovered two drug candidates for prostate and breast cancers, which are resistant to each of base anti-cancer drugs, by using a computational approach. The drug for prostate cancer resistant to docetaxel is now under investigator-initiated clinical trial in Keio University Hospital, and that for breast cancer resistant to paclitaxel showed high efficacy in xenograft test. These case studies highlight an original technique to estimate the signature between patients sensitive and resistant to base anti-cancer drugs, which well utilizes the information on clinical outcome.
Katsuhisa Horimoto, Ph.D., Deputy Director, Molecular Profiling Research Center for Drug Discovery, National Institute of Advanced Industrial Science and Technology
9:00 am Focus Session on the Business and Finance of Drug Repurposing
Overview of Business Perspectives in Drug Repurposing
There is an emerging consensus that the impact of Drug Repurposing on the pharmaceutical industry is real and sustainable. These efforts can only be maintained and expanded if the dynamic variables of viable and creative business models are identified and understood. In this work we describe the lessons that can be learnt from surveying the landscape of the industry. This analysis includes both the successes and the failures of past repurposing companies. We introduce the various stakeholders that are shifting the decision process of implementation and acceptance away from the pharmaceutical industry. In addition the component pieces necessary to enhance the value of a drug repurposing company are discussed and we assess and compare an assortment of business models.
Stephen Naylor, Ph.D., Chairman & CEO, MaiHealth Inc.
Financial Model Considerations for Drug Repurposing Companies
Drug repurposing is faster and decreases risk compared with traditional drug development. However, such efforts afford somewhat ambiguous patent protection. The various factors that affect return on investment for a pharmaceutical company pursuing repurposing versus traditional methods can be modeled using Monte Carlo methods and Net Present Value (NPV) analysis, to quantify the financial benefits of repurposing. Factors to be considered include failure rate at various stages of clinical development, steps that can be skipped, and extent of market exclusivity post approval.
Daniel Sem, Ph.D., MBA, Dean and Professor of Business, Professor of Pharmaceutical Sciences, Concordia University Wisconsin
Comprehensive Survey Analysis of Drug Repurposing Sector
We explore how drug repurposing companies can generate new innovation opportunities by using existing resources in the industry. We will present our results predicated on 30 interviews that we conducted with 25 experts who are involved in drug repositioning activities in big pharmaceutical companies, drug repositioning companies, academia, contract research organizations, data analytics companies and governmental agencies over the past year. In concert with archival data in the form of literature review materials we provide an overview of the drug repurposing sector as it pertains to opportunity creation and knowledge re-use directed towards the pharmaceutical industry.
Burcu Kucukkeles, Research Associate & Doctoral Candidate, Department of Management, Technology and Economics, Chair of Strategic Management and Innovation, ETH Zurich
10:00 am Refreshment/Networking Break
10:25 am Repurposed Drugs: An Investor's View
Significant societal benefit from developing new therapeutic uses for off-patent drugs remains unrealized because the existing incentives for investing in new pharmaceutical R&D fails to provide most investors with a clear path towards a viable business model for investing in repurposing drugs. The presentation will discuss ways investors can evaluate if an exclusive marketing position for the repurposed drug does or could exist, and how to go about making the case for the investment.
Adriann Sax, MBA, Entrepreneur in Residence, Fortress Biotech
10:55 am Drug Combination Therapy for Improving the Success Rate of Drug Repositioning by Drug Repurposing Screens
A drug repurposing screen is one of the methods for drug repositioning that usually uses high throughput screening to identify active compounds for new indications. It significantly shortens the process of drug development if an approved drug is identified for a new indication. However, most of the identified compounds from drug repurposing screens exhibit low potency which limits their immediate applications. Dr. Zheng and his team found that a combination of two or more compounds with different mechanisms of action is an alternative approach to increase the success rate of drug repurposing screens. Two cases of repurposing screens for drug resistant bacteria and Ebola virus will be discussed in this presentation.
Wei Zheng, Ph.D., Leader, Biology, Therapeutics for Rare and Neglected Diseases, Division of Pre-Clinical Innovation, National Center for Advancing Translational Sciences, National Institutes of Health
11:25 am Drug Discovery and Repurposing at the University of New Mexico Center for Molecular Discovery
Dr. Sklar’s team introduced high throughput flow cytometry (HyperCyt) as a technology for drug discovery, patenting applications of the technology as well as the identification of small molecule probes, new chemical entities, and repurposed drugs. The high throughput flow cytometry platform for drug discovery has been associated with multi-target screening for both cellular and molecular targets such as efflux transporters, integrins, GPCRs, and GTPases which have been accompanied by repurposing screens and association with the NIH Molecular Libraries Program and various other programs. Experimental and computational methods have led to the identification of small molecules as first in class chemical probes, leads for drug discovery, and repurposed drugs. These molecules and technologies have been described in more than 100 publications as well as 30 patents, and have contributed to several clinical trials and start-up companies. Dr. Sklar's team has conducted repurposing studies in neurological, cardiovascular and infectious diseases as well as cancer that are beginning to show promising results. They envision future repurposing contributions to personalized medicine.
Larry Sklar, Ph.D., The Maralyn S. Budke and Robert E. Anderson Distinguished Endowed Chair in Cancer Drug Discovery, Director, UNM Center for Molecular Discovery, The University of New Mexico School of Medicine
11:55 pm Luncheon
12:55 pm Drug Repositioning in the Era of Precision and Systems Medicine
With the advent of Big Data revolution, diverse teams from Drug/Indication Discovery to Global Clinical Development/Medical Affairs are faced with massive amount of potential signals from clinical, -omic, and imaging data collected from pre-clinical/translational and controlled clinical studies. This presentation highlights some of the potential and challenges in the wake of Big Data application in finding hidden therapeutic gems.
Jason Tsai, MD, MS, VP Translational Medicine, Numedii
1:25 pm New Uses for Existing Drugs - Exclusivity Strategies
Existing drugs are an attractive research target for new uses, in that safety testing has already been conducted. However, exclusivity in the form of patent protection, which is typically necessary to secure investment, may be difficult to obtain. Strategies for obtaining strong patent protection, defending against generics, and securing other forms of exclusivity for new uses of existing drugs will be explored, along with the impact of recent U.S. case law regarding patentable subject matter on the ability to secure patent rights for such uses.
Rose Thiessen, Ph.D., Partner, Knobbe, Martens, Olson & Bear, LLP
1:55 pm Leveraging Unique Capabilities and Patient Access for Systematic Identification of Repurposing Opportunities
Combining advances in genomics, large-scale Electronic Medical Records, and sophisticated Big Data analytics enables the identification of novel applications for existing molecules. Teva has developed a sustainable, ever-learning repurposing platform to systematically evaluate existing molecules, based on multiple data sources and modalities.
Daphna Laifenfeld, Ph.D., Director, Personalized and Predictive Medicine and Big Data Analytics, Teva Pharmaceuticals
2:25 pm Pushing the Boundaries of Scientific Collaboration to Accelerate Novel Repurposing Opportunities
Building on more traditional partnerships, we at AstraZeneca are taking a multi-pronged approach to collaboration in order to foster novel discoveries and speed the development of new medicines for patients in need. Through our Open Innovation platform (www.openinnovation.astrazeneca.com), we invite external investigators and clinicians across all stages of drug discovery to collaborate. AstraZeneca provides access to optimized compounds, compound libraries, technologies, multi-disciplinary science, services and/or know-how with the prospect of joint publications in high profile journals. Through additional partnerships, we are applying integrative genomics, network biology, and other translational bioinformatics approaches to gain insight into novel connections between drugs and indications for the repositioning discontinued compounds. Our approaches, as well as the associated benefits and challenges, will be illustrated by specific examples with a particular focus on drug repositioning.
Leslie Cousens, Ph.D., Associate Director, Translational Medicine, Emerging Innovations, AstraZeneca
2:55 pm End of Conference