July 13, 2011
8:30 - 9:00 Registration/Continental Breakfast
9:00 Chair's Opening Remarks
Aris Persidis, Ph.D., President, Biovista
9:10 Systematic Repositioning Using Mechanism of Action to Map all Drugs Against all Diseases
Using mechanism of action (MoA) at the core, it is possible to map the potential relevance of any drug against all 23,000 diseases and 6,000 adverse events tracked by modern biomedicine. Both efficacy and safety case studies will be presented. The impact on pipeline development in terms of growth and defense against competitor adjacency moves will be discussed.
Aris Persidis, Ph.D., President, Biovista
9:50 Knowledge Management Technologies in Discovery of New Indication Opportunities
An aspiration of global pharma companies to de-risk their pipelines, on one side, and rapid accumulation of knowledge in private and public domain, on the other, provides a great chance for knowledge management technologies to lead an exploration for novel business opportunities for existing compounds.
This presentation will illustrate the knowledge management approaches and strategies for leveraging information and generating new knowledge to drive the compound repositioning and extending profiling efforts at Merck-Serono.
Natalia Novac, MSc, Ph.D., Associate Manager, R & D Knowledge Management Operational Excellence, Merck Serono
10:30 Morning Refreshment Break
10:50 Developing Information Analytics & Search Solutions To Identify, Evaluate And Exploit New Opportunities For Drug Repositioning
Nick Brown, Associate Director Informatics Programme Manager, New Opportunities, AstraZeneca
11:30 Repurposed Drugs as a Novel, High-Value Product Life Cycle Management Option for Brand Managers: Case Study of VT-122 in Oncology
Repurposed drugs can provide a novel, high-value product life cycle management ("PLCM") option for brand managers. Repurposed drugs, co-administered with a branded drug, add new MOA to enhance the clinical value and market share of the branded drug. Unlike indication expansion or head-to-head PLCM options, repurposed drugs can be evaluated and gain clinical guidelines status within 2-years, for less than $5 million and without the risk of an adverse clinical finding that may undermine a brand franchise. In this talk, Vicus will provide a case of VT-122 in oncology and it’s positioning as a PLCM option for Nexavar and other leading oncology brands.
John Maki, President & CEO, Vicus Therapeutics
1:20 The Center for World Health & Medicine at Saint Louis University: A New Translational Research Model to Develop New Therapies for Neglected and Orphan Diseases and Other Unmet Medical Needs
This presentation will describe the mission, capabilities, disease target portfolio, and unique operating model of the Center for World Health & Medicine. It will highlight the Center's strengths in translational drug development and especially its model of multiple research collaborations and partnerships with international disease experts and Institutions. Particular emphasis will be on leveraging the Center's knowldege and experience with advanced drug candidates and re-purposing them toward orphan and neglected diseases and other unmet medical needs.
Peter G. Ruminski, Ph.D., Executive Director, Center for World Health and Medicine , Saint Louis University
2:00 Chemical Structure Based Virtual Screening Methods in Drug Repositioning
This presentation will describe the methods used to discover new indications based only in a ligand chemical structure, without taking into account any biological data on the molecule. The most important criteria for go/no-go decisions on which molecule to use, as well as which indication to push forward will also be reviewed, in order to choose those indications that minimize risks and add more business value.
Raúl Insa, MD, Ph.D., Chief Executive Officer, SOM Biotech
2:40 Drug Repositioning: Silenor Case Study
Silenor (doxepin) 3 and 6mg tablets was approved on March 17th, 2010 for the treatment of insomnia characterized by difficulty with sleep maintenance after two complete response letters and an appeal of the FDA’s decision. Doxepin was originally approved as an antidepressant (tricyclic antidepressant – TCA) in 1969 at doses of 75 to 150mg. This presentation will retrace the development and approval pathway of Silenor, identifying lessons learned and pitfalls in drug repositioning.
Brian Dorsey, Senior Vice President of Technical Operations, Somaxon
3:20 Afternoon Refreshment Break
3:40 Effective Drug Development with 505 (b) (2) Principles
Effective drug development begins with the end in mind—the requirements and preparation for an FDA approval in a streamlined, cost-effective fashion.
Candidate Identification: The four-step process for identifying suitable candidates for development: Criteria Selection, Criteria Evaluation, Candidate Narrowing, and Candidate Selection.
Approval Pathway Identification: What is the difference between a 505(b)(1) and a 505(b)(2)?
Historically, the 505(b)(2) pathway was used to make improvements to existing approved drugs. Yet, the regulations state that a 505(b)(2) can be used for any drug product where pivotal information is not conducted by the applicant. Thus, even a new chemical entity can be approved under 505(b)(2). Areas for discussion:
- Review the regulatory requirements for a 505(b)(2) NDA
- Review the kinds of information that may be used in support of an NDA submission
- Review products currently being developed without a U.S. approved reference drug
- Review the process needed to gain FDA buy-in to the 505(b)(2) development plan
Ken Phelps, President & CEO, Camargo Pharmaceutical Services
4:20 Computational Methods for Identifying Novel Disease Indications for New and Old Drugs
Typically drugs have been repurposed following serendipitous observations. Now, however, there are a number of computational methods that can evaluate and suggest new indications for a drug. Dr. Agarwal will discuss some of these that are based on text-mining literature, genetics, expression signatures and pathways.
Pankaj Agarwal, Ph.D., Director, Computational Biology, Molecular Discovery & Development, GlaxoSmithKline
July 14, 2011
8:00 - 8:30 Continental Breakfast
8:30 Chair's Opening Remarks
Ken Phelps, President & CEO, Camargo Pharmaceutical Services
8:40 Virtual Panel Session: Competing & Collaborating to Achieve Successful Drug Repositioning (Sponsored by Collaborative Drug Discovery)
The panel will explore ways to simultaneously compete and collaborate - and consider alternative business models for rare, neglected, and commercial drug discovery.
Christopher A. Lipinski, Ph.D., Scientific Advisor, Melior Discovery
Noel Southall, Ph.D., Informatics Scientist, NIH Chemical Genomics Center
David Cavalla, Ph.D., Founder, Numedicus Ltd., Principal, ATPBio
Chair: Barry A. Bunin, Ph.D., CEO and Board Director, Collaborative Drug Discovery (CDD)
9:40 Screening Drugs Directly in Primary Cells: From the Bench to the Clinic
Dr Ballesteros will discuss the following topics:
- System's Biology driven Reprofiling, evaluating the activity of thousands of drugs directly in patient samples
- Novel reformulations with better IP, for example Vivia009 that reaches selectively lymph nodes for blood cancers
- Phama-friendly Reprofiling approach, where enantiomers of drugs & metabolites are the candidates with better protection and pricing, and the known drug enable quick clinical PoC
Joan Ballesteros, Ph.D., Chairman & Chief Scientific Officer, Vivia Biotech SL
10:20 Morning Refreshment Break
10:40 Predicting the Unpredicted with Phenotypic Screening
Drug repositioning of clinical stage development and approved drugs has emerged as a viable, but underutilized mechanism for filling the pharmaceutical industry innovation gap. Although the process of drug repositioning can utilize in silico, in vitro or cell based biological systems, the majority of drug repositioning events are driven by therapeutic observations made in in vivo biological settings. These in vivo settings include both pre and post-marketing human clinical trials and preclinical experimental animal models of disease.
This presentation will describe the processes of drug repositioning and describe an optimized preclinical in vivo efficacy platform for repositioning preclinical and clinical stage drug candidates.
Andrew Reaume, Ph.D., MBA, Chief Executive Officer, Melior Discovery
11:20 Case Study: ADL6906, a 5-HT2 Serotonin Receptor Antagonist & Norepinephrine Reuptake Inhibitor (SANRI), for the Treatment of Pain
ADL6906 was previously studied by a pharmaceutical company in late-stage clinical trials in over 1,000 patients for the treatment of depression, where it demonstrated a good safety profile. Adolor Corporation is repositioning ADL6906 as a potential treatment for pain conditions and is currently conducting Phase 1 clinical evaluation of the compound. ADL6906 is a dual norepinephrine (NE) reuptake inhibitor and 5-HT2 serotonin receptor antagonist, referred as SANRI (5-HT2 Serotonin Receptor Antagonist & Norepinephrine Reuptake Inhibitor), giving ADL6906 a novel, unique and potentially differentiating pharmacological profile for treating pain. Dr. Le Bourdonnec will discuss the process by which Adolor re-discovered this compound and will outline the company's new clinical development program for it.
Bert Le Bourdonnec, Ph.D., Senior Director, Program Management & Discovery, Adolor Corporation
1:10 Repositioning Drugs for Parkinson’s Disease: Perspective from the Michael J. Fox Foundation
The presenter will cover the following:
- Past opportunities e.g. case studies such as nicotine and piaglitazoneOpen, investigator-initiated solicitation by launching the RFA-Repositioning Drugs for PD 2011
- How the Repositioning RFA was publicized and announced
- The process for preproposals, full applications as well as criteria to determine what to fund
- RFA – how many preproposals came in, and which eventually got funded
- Lessons learned
Kuldip Dave, Ph.D., Associate Director - Research Programs, The Michael J. Fox Foundation for Parkinson's Research
1:50 Maintaining your Re-position: a Patent Primer
Mr. McLaren will discuss the following topics:
- Patentable subject matter
- Patent law requirements (utility, novelty, non-obviousness, written description, enablement) Market forces
- Regulatory forces
- A survey of recent cases (Strattera(r), Gemzar(r), Enbrel(r)) to avoid some pitfalls
Kevin L. McLaren, Ph. D., Esq., Of Counsel, Intellectual Property Department, Barnes & Thornburg LLP
2:30 Repositioning a Monoclonal Antibody for Niche Markets
Bertilimumab, an anti-eotaxin-1 human monoclonal antibody with 126-patient history, was exclusively licensed from AstraZeneca/MedImmune. This case study will illustrate the unique opportunity to reprofile a biologic in an ophthalmic setting, while also allowing for the development of multiple, differentiated product candidates.
Andrew J. Rae, MBA, President, CEO and Director, iCo Therapeutics Inc.
3:10 Rationale Drug Repositioning
Whilst many repositioning strategies rely on mining the literature and databases for therapeutically useful responses of known drugs, Celentyx Ltd hunts for utilised drug targets on human immune cells. In particular a differential increasing expression of the target across resting, activated and pathological human immune cell types encourages greater investigation of impacting the immune cell type with known target ligands for potential therapeutic benefit. Integral to the strategy is the investigation of key immune cell subsets (e.g. Tregs, Th17s) that although often present in miniscule fractions, orchestrate considerable influence upon the immune system with relevance to inflammation and autoimmunity. This strategy creates clear inventive step facilitating patent protection of projects. The talk will use a representative example of one of Celentyx’s projects.
Professor Nicholas Barnes, Co-founder & CEO, Celentyx
3:50 End of Conference