VIRTUAL | November 8-10, 2021 

Learn about the latest technologies, partnerships and advances propelling the field of repurposing and re-shaping drug discovery and development

 

2014 Agenda

DAY ONE - JULY 15-16, 2014

8:00 Registration/Continental Breakfast


8:40 Chairperson's Opening Remarks

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


8:45 Why High Quality Phenotypic Screening is So Well Suited for Drug Repositioning

Over the last several year there has been a burgeoning appreciation for phenotypic screening as a viable drug discovery strategy. The term has also come to refer to an in increasingly diverse array of activities from very high throughput, in vitro-based, approaches to certain approaches in the clinic. As regards the endeavor of working with a privileged candidate (a molecule with proven safety, tolerability and an array of drug-like properties) there is a rationale for employing high quality (i.e. in vivo models of disease) forms of phenotypic screening in order to uncover otherwise unpredictable 

Andrew Reaume, Ph.D., MBA, President & CEO, Melior Discovery, Inc.


9:15 Social Investing to Support Rediscovery Research™

Rediscovery Research improves lives by taking drugs, devices and nutriceuticals approved for one disease and repurposing them to create a “new” treatment in a totally different disease.  When the repurposed drug is generic and able to be taken in the current dosage and formulation, there is often little or no profit to be gained, so industry does not fund these repurposing projects.  

Currently, philanthropy funds a large number of them, and that funding is hard to get. One potential way to create a huge incentive for investors to finance these life-saving Rediscovery Research projects is through Social Investing vehicles such as Social Impact Bonds (SIBs). A SIB is an arrangement between one or more government agencies and an external organization, called a Social Impact Bonding Organization (SBIO) where the government specifies an outcome and promises to pay the external organization a pre-agreed sum if it is able to accomplish the outcome. Rediscovery Research provides a perfect fit for SIBs and other Social Investing because it creates improved healthcare outcomes and significant healthcare cost savings, two huge government needs. And ICD-9/10 coding allows tracking of healthcare costs for individuals and disease groups. This presentation will discuss in detail the potential for Social Investing to transform the way Rediscovery Research impacts health.

Dr. Bruce Bloom, President and Chief Science Officer, Cures Within Reach


9:45 The Challenges of Repositioning Drugs: 10 lessons From Black Bone Disease

Nitisinone is an enzyme inhibitor originally developed as a herbicide before being used to treat successfully the rare disease hereditary tyrosinemia type 1. It works on the same metabolic pathway as Black Bone Disease, a rare genetic disorder also known as Alkaptonuria (AKU). Our patient group, the AKU Society, has been at the core of a consortium of hospitals, universities, pharma and biotech companies to repurpose nitisinone for Black Bone Disease through cell models, an animal model, a natural history study, as well as phase 2 and phase 3 trials. We have also screened a library of FDA-approved drugs and identified six small molecules that increase the enzymatic activity in Black Bone Disease. To accomplish all this, we have worked on new models of fundraising and patient-led collaboration in order to drive the quest for treatments.

Nicholas Sireau, Ph.D., Chairman and CEO, AKU Society, Co-Founder and Chairman, Findacure


10:15 Refreshment Break


10:45 The Drug Repurposing Patent Landscape: A Hotbed of Pharmaceutical Creativity

The presentation will discuss, based on a pattern analysis of recent patenting, how inventors from academia and industry constantly probe for new ground while taking another look at known active ingredients. We will show that the landscape is neither flat (some drug classes lend themselves to repurposing more than others) nor clearly delineated (it is constantly growing in new directions, obvious ones as well as some that are less predictable). Typical profiles of drug repurposing patents, their applicants, and claim-supporting data will also be discussed.
 
Hermann Mucke, Ph.D. (virtual presenter), Chief Executive, H.M. Pharma Consultancy

11:15 Prioritizing and Integrating Existing Methods into Cost-effective and Efficient Pipelines for Drug Repositioning

Drug repositioning accounts for about 30% of the newly US FDA-approved drugs and vaccines in recent years. The prevalence of drug repositioning studies has resulted in a variety of innovative computational methods for identification of new opportunities for use of old drugs. Questions often arise from customizing or optimizing these methods into cost-effective and efficient drug repositioning pipelines for alternative applications. To address these questions, it requires comprehensive understanding of the available methods by evaluating both biological and pharmaceutical knowledge and the elucidated mechanisms-of-action of drugs. This talk intends to provide guidance for prioritizing and integrating drug repositioning methods for specific drug-repositioning pipelines.

Stephen Wong, Ph.D., PE, Founding Chair for Department of Systems Medicine and Bioengineering, The Methodist Hospital Research Institute, Weill Cornell Medical College at Texas Medical Center


11:45 Luncheon


1:00 Comparison of Different Development Paths, Challenges and Profiles of Three Re-positioned Drug Products

NeuroHealing is a private virtual company established to develop drug therapies for the untapped post-acute and chronic neuro-rehabilitation market. We use novel formulations and drug delivery strategies to reposition approved neurologically-active small molecule drugs for specialty indications. Using insights into the pathobiology and patient needs, the company has brought three product candidates to human testing. Dr. Farber will directly compare and contrast the starting insights and divergent product & patient needs of these product candidates under development. The talk will be illustrated with detailed counterbalancing examples of the risk attenuation features versus technical, IP, market size, sale force, regulatory and financing issues.
 
Neal M. Farber, Ph.D., Chief Executive Officer, NeuroHealing Pharmaceuticals, Inc.

1:30 The NCATS Pharmaceutical Collection: Overview, Screening Processes, and Examples of Use in Cancer and Infectious Diseases

Anton Simeonov, Ph.D. (tentative), Acting Deputy Scientific Director, National Center for Advancing Translational Sciences, National Institutes of Health


2:00 Drug Repurposing In Pediatrics: A Clinician's Perspective

Most pediatric chronic diseases meet the definition of rare or orphan diseases, with prevalences of  <200,000 in the US. Particularly for children with cancer, the effectiveness of current therapies, frequent ineligibility for phase I clinical trials, and a desire of families to continue care close to home have limited the effectiveness of current algorithms for drug development. A first step to using drug repurposing as an alternative approach in children is an understanding of what drugs are available to this population. “The Children’s Pharmacy Collaborative™” is a comprehensive listing of more than 1200 drugs for which there are safety profiles and recommended dosing for children. This database will be the basis for in vitro screening and drug repurposing efforts for a range of pediatric indications. Dr. Blatt will discuss the development of the database and its preliminary application to high throughput phenotypic screening in pediatric cancer.  

Julie Blatt, MD, Professor,  Pediatric Hematology Oncology, University of North Carolina School of Medicine


2:30 Refreshment Break


3:00 Repositioning: Validating a Drug Target for a New Indication through Genetics, Expression, and Phenotypic Screening

Drug repositioning offers the potential to create value for patients in a quicker time frame. It is also perhaps one of the better ways to validate targets for a new indication. We will examine how data from across the drug discovery can suggest additional indications. We will also discuss some computational methods that can systematically identify disease indications for drugs; in particular 1) genome wide association studies (GWAS), 2) connectivity map (expression based drug signatures), and 3) data from phenotypic screens.
 
Pankaj Agarwal, Ph.D., Director, Systematic Drug Repositioning (SyDR), QSci Computational Biology, RD Projects, Clinical Platforms, & Sciences, GlaxoSmithKline

3:30 In Silico Drug Repurposing for Cystic Fibrosis

Cystic fibrosis (CF) is a rare genetic disorder affecting ~70,000 individuals worldwide. The treatment option for CF is very limited; the principle treatment approved by the FDA in 2012 only affects ~4% of the CF population and is very expensive. It has been demonstrated that the feed-forward loops (FFLs), a specialized regulatory motif, plays a major role in many diseases. Built on this observation, we developed an in silico approach to construct a set of FFLs for CF. With these FFLs, we examined all the market drugs for their potential use for the treatment of CF based on the drug repositioning principle. As a result, a set of promising drug candidates were identified for CF. While marketed drugs for the treatment of CF was our focus, the developed strategy can be applied to other diseases for the repurposing of drugs.
 
Zhichao Liu, Ph. D., PI, Division of Bioinformatics and Biostatistics, National Center for Toxicological Research (NCTR), FDA

4:00 Panel Discussion


4:45 Wine & Cheese Reception


DAY TWO - JULY 16, 2014

7:45 Continental Breakfast


8:40 Chairperson's Opening Remarks

Dr. Bruce Bloom, President and Chief Science Officer, Cures Within Reach


8:45 Celgene Global Health:  Focusing on Those Who Can Least Afford to Live by Re-focusing a Proprietary Chemical Library

Celgene Global Health (CGH) is a division of Celgene Corporation “focusing on those who can least afford to live”. CGH is exploring the potential of Celgene’s chemical library and pipeline to address diseases of the developing world (DDWs) and neoplasms prevalent in lower income settings. CGH is operating as a virtual company partnering with foundations, product development programs, universities, institutions, and other companies to discover and develop its assets. The goal is create meaningful medications for these illnesses and by doing so CGH will transform into a self-sustaining “Social Business” that continues to perform this type of activity.

The research currently being conducted spans the entire range of pharmaceutical development from discover, pre-clinical and clinical development. CGH is currently conducting research on the kinetoplastid diseases (Human African Trypanosomiasis, Chagas Disease, Leishmaniasis), malaria, lymphatic filariasis, tuberculosis, babesiosis, viral hemorrhagic fevers (Lassa Fever, Rift Valley Fever, Argentina Hemorrhagic Fever), HIV/AIDS, Kaposi’s Sarcoma, and leprosy. In his presentation, Dr. Zeldis will discuss Celgene’s and CGH’s efforts to combat DDWs.

Jerome B. Zeldis, MD, Ph.D., Chief Medical Officer, Celgene Corp., CEO, Celgene Global Health


9:15 Integrating the Digital Universe of Information for Drug Discovery and Repurposing

This talk will show illustrate how Dr. Dudley’s lab is combining integrative genomics, network biology, and translational bioinformatics to integrate and model large molecular and clinical data repositories to enable new insights into drug mechanisms, side effects, and indications.

Joel Dudley, Ph.D., Assistant Professor of Genetics and Genomic Sciences, Director of Biomedical Informatics, Mount Sinai School of Medicine


9:45 Rethinking Design and Execution of Clinical Trials for Repositioned Drugs

Recent advances in web-based crowdsourcing and telemonitoring technologies, along with a shift toward greater data transparency, portend a radical restructuring in the economics of clinical development for repositioned drugs. Crowdsourcing enables patients, physicians, researchers and other stakeholders to contribute to the design of clinical trials, resulting in protocols that are focused on parameters most relevant to clinical decision-making and patient needs. Leveraging advances in information technology and mobile health allows for trials that are far more cost-effective than current approaches, while reducing burdens on subjects and sponsors and enhancing data quality. Providing data transparency throughout the development process builds credibility with diverse stakeholders and produces better results.

Marc Foster, MBA, Chief Operating Officer, Transparency Life Sciences


10:15 Refreshment Break


10:45 Repurposing as a Viable, Risk-managed Business Model

Rediscovery Life Sciences aims to develop new medicines through drug repurposing.  We see repurposing as having a fundamentally better risk/reward profile, and thus it can be a great de-risking tool for life science investors. Their business model insures low startup costs, effective risk management and triple-bottom-line value creation. They achieve this through disciplined scouting and due diligence, entrepreneurial translational development, and a project-focused portfolio approach.  

Daniel Behr, MBA, Co-founder and Partner, Rediscovery Life Sciences


11:15 Next Generation Repositioning: Taking the Serendipity Out

In this presentation, Dr. Persidis will illustrate the following critical thoughts regarding repositioning today:

  • Serendipitous discovery can only achieve so much
  • Current advances in technology allow a much more systematic and unbiased exploration of the link between any drug and any disease
  • Linking mechanism to medical records allows for very interesting subpopulation effects to become visible  

To do repositioning can be easy, quick, and cheap: to do high quality repositioning, however, requires different approaches

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


11:45 Development of a Topical Anti-inflammatory Compound Using Smart Re-Profiling

In this presentation, Dr. Scoffin will give a case study of re-profiling using a platform termed “Smart Re-Profiling,” which is the combination of computational chemistry approaches to identifying potential re-profiling opportunities with deep domain expertise in pharmacology to triage the ideas generated, and a network of biological experts to help validate the triaged opportunities in real disease models. This case study will focus on the identification and development of RP0217 which is their lead compound for topical inflammatory conditions.

Dr. Robert Scoffin, Chief Executive Officer, Cresset, Co-Founder and CEO, Re-Pharm


12:15 Luncheon


1:15 Repositioning of Drugs for Renal Cell Cancer Using Transcriptome Signatures

Since survival outcome for metastatic clear cell renal cell carcinoma (ccRCC) remains poor, new therapeutic strategies are essential. We are applying a new individualized bioinformatics analysis of ccRCC transcriptome data and sunitinib resistance gene signatures in conjunction with analysis of the Connectivity Map (C-MAP) database to reposition FDA-approved drugs for anti-cancer therapy. This strategy enabled us to prioritize multiple drugs as candidate new therapeutic agents against ccRCC. Detailed mechanistic analyses confirm that these drugs target relevant cancer pathways in ccRCC. We demonstrate that one of these drugs slows tumor growth while enhancing survival in a ccRCC xenograft mouse model, highlighting the potential of C-MAP analysis to repurpose drugs for anti-ccRCC therapy.
 
Towia Libermann, Ph.D., Associate Professor of Medicine, Director, BIDMC Genomics, Proteomics, Bioinformatics, and Systems Biology Center, Beth Israel Deaconess Medical Center

1:45 Pushing the Boundaries of Science Together Through Open Innovation Partnerships

AstraZeneca has recently announced a Open Innovation platform (www.openinnovation.astrazeneca.com) to invite collaborations with investigators and clinicians across all stages of drug discovery – from innovative early ideas through to clinical validation. Collaboration with AstraZeneca means 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 and, most importantly, the opportunity to push the boundaries of medical science and deliver life-changing medicines to patients in need. The specific modules (offerings) in this platform as well as the associated benefits and challenges will be described with particular focus on drug repositioning.

Craig D. Wegner, Ph.D., Executive Director, Translational Science, Emerging Innovations Unit, Scientific Partnering & Alliances, AstraZeneca Pharmaceuticals LP


2:15 Panel Discussion


3:00 End of Conference

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