November 14-15, 2022 | Washington, DC 


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


The next Drug Repositioning & Repurposing Conference will be taking place in Spring/Summer 2024. Please check back in the coming weeks and months for updates.

Presentation Files

DAY ONE - October 23, 2012

8:00 Registration/Continental Breakfast

8:45 Chair's Opening Remarks

Carla M. Canuso, MD, Senior Director, External Innovation, Neuroscience Therapeutic Area, Janssen Pharmaceutical Research and Development, LLC

8:55 Case Study on Both DUEXIS and RAYOS - Why There was a Need to Fix an Already Good Thing
This presentation will discuss the story of both DUEXIS and RAYOS. Specifically, why there is a need for DUEXIS if the pieces have been around for 20 years, but just needed to be used together. In addition, this presentation will cover how to determine true patient need and the risks, challenges, and pathways for creating value from this need. This presentation will also discuss why reformulation means more than fixing convenience.

Todd Smith, EVP, Chief Commercial Officer, Horizon Pharma, Inc.

9:30 Repositioning an Underutilized Gold-standard Treatment by Leveraging Novel Delivery Technology:  A Case Study of Sumavel®DosPro® for Treatment of Migraine
This presentation will cover the following:

  • Connecting the market need with the right drug delivery solution
  • Differentiating the solution amidst entrenched branded and generic competition
  • Developing the drug/device combination product using the 505b2 regulatory pathway
  • Positioning the new solution for patient, physician, and payer acceptance
  • Ensuring well-rounded strategies and tactics to ensure adoption, uptake, and adherence

Steven Jenner, Vice President, Marketing, Zogenix, Inc.

10:05 Using Systematic Drug Repositioning to Map Drugs and Diseases Against Each Other
This presentation will cover the following:

  • The on- and off-target pharmacology of a drug can inform a systematic repositioning approach
  • Integrating multiple kinds of data leads to more robust drug repositioning
  • Case studies in rare as well as mainstream diseases will be described, including MS and Friedreich's Ataxia

Aris Persidis, Ph.D, President, Co-Founder, Biovista Inc.

10:40 Refreshment Break

11:05 Focused Patentomes and Literareomes - Text-Based Knowledge Mining for Drug Repurposing
Current methods for discovering potential new uses for known molecules focus on algorithmic bioinformatics and high-content screening. A much less popular alternative approach is driven by semantic and chemoinformatic analysis, placed in context with pharmacology and biological pathways. To advance the mining of a much-neglected information source we are building what we call "patentomes" - the totality of patent disclosures - for relatively small, well-defined fields of medicine. This presentation will discuss the patentome covering ocular drug therapy and biomarkers, and its potential applications to drug repurposing.

Hermann Mucke, Ph.D. (virtual presenter), Chief Executive, H.M. Pharma Consultancy

11:40 Repositioning Drugs Using Formulation and Delivery Technology
Drug repurposing can be a very attractive path for drug development compared to the traditional pharmaceutical drug discovery path as the latter is relatively slow, expensive, and risky even in late-stage development. This session will focus primarily on repurposing using the inhalation route and will include the following:

  • Repositioning of Drugs for Administration by Inhalation to Treat Lung Disease
  • Examples, including early asthma medications, fungal and bacterial infections in the lung, lung cancer, and prevention of organ (lung) rejection
  • Repositioning of a Drug for Administration by Inhalation for Systemic Effect, including proteins and peptides such as insulin, GH, EPO, PTH, and IFNs

Jim Blanchard, Principal Scientist, Preclinical Development, Aradigm Corporation

12:15 Luncheon

1:30 Computational Drug Repositioning for Novel Disease Indications
This presentation will cover the following:

  • Gene expression data can be used to create a molecular signature for disease
  • Signatures can be matched with signatures of drugs, to yield drug-disease relations
  • New indications for drugs can and need to be tested in disease models

Atul Butte, MD, Ph.D., Chief of the Division of Systems Medicine and Associate Professor of Pediatrics, and by courtesy, Medicine and Computer Science, Stanford University and Lucile Packard Children's Hospital

2:05 Establishing Internal Alignment to Support External Collaborations: A Case Study from Big Pharma
Drug repurposing holds promise in bringing much needed, new treatments to patients while minimizing the risk, cost, and timelines of traditional drug development. Increasingly government agencies, academic investigators, and pharmaceutical companies are partnering on such repurposing initiatives. While large pharmaceutical companies can be a great source of compounds ready for human testing, complex internal processes, business priorities, and team dynamics can create challenges in forging these collaborations. This case study will provide examples of how internal alignment can be achieved to overcome these challenges and successfully support government-sponsored drug repurposing initiatives.

Carla M. Canuso, MD, Senior Director, External Innovation, Neuroscience Therapeutic Area, Janssen Pharmaceutical Research and Development, LLC

2:40 Entering the Golden Age of Repurposing:  NCE Pricing for Repurposed Drugs in Oncology
Common misperceptions of the reimbursement potential for repurposed drugs in oncology limit development funding by VCs and large pharmaceutical companies.  However, based on a the 1st ever repurposed drug survey of payers and their reimbursement algorithms,  repurposed drugs will likely gain coverage at NCE pricing in oncology.

This presentation will cover the following:

  • Why oncology reimbursement is unique and likely to remain so
  • Impact of ACOs in oncology and the rise of 340B hospitals and on-site pharmacies
  • Impact of clinical pathways in oncology
  • Importance of NCCN Guidelines in oncology
  • Importance of niche cancer indications
  • Importance of co-pay assistance programs

John Maki, President and CEO, Vicus Therapeutics

3:15 Refreshment Break

3:40 A Knowledge-Based Approach to Drug Repositioning
Pharmaceutical companies are continually seeking new avenues to increase revenues. One such avenue that has seen a significant increase in activity in recent years is drug repositioning, in which companies seek  new indications for existing drugs or late stage clinical assets. Repositioning encompasses everything from new indications for failed compounds to line extensions for existing drugs and is expected to generate up to $20 billion in annual sales in 2012.

One of the most profitable outcomes of repositioning is to successful reposition a drug for new indications.  Most often this is done by serendipity, an observation of a side effect of a clinical trial, or intimate knowledge of a particular disease. A more systematic, science driven approach is desirable. Using a variety of Thomson Reuters databases we have developed a knowledge based approach to repositioning which combines target and pathway analysis, analysis of molecular descriptors, and clinical trial proteomic information to identify new indications for existing compounds. The utility of the methodology will be demonstrated on several examples of compounds from the literature.

Richard Harrison, Ph.D., Director, Discovery Sciences, Thomson Reuters Life Sciences Professional Services

4:15 Orphan/Rare Drug Discovery Through Drug Repositioning
This presentation will cover the following:

  • Orphan/Neglected/Rare Diseases (Introduction)
  • Orphan Drug Act benefits – where was it, where it is and where it will be
  • Technologies and strategies for orphan drug discovery
  • How and where drug leads come
  • Outcomes of PPP
  • Patents, priority vouchers, etc
  • Current status of big pharma’s thinking (efforts)
  • Orphan drug repositioning success

Ramaiah Muthyala, Ph.D., Associate Director, Center for Orphan Drug Development, University of Minnesota

4:50 Rediscovery Research™ Impacts Patients with Rare and Neglected Diseases
Over 5000 inexpensive and safe generic drugs and safe nutriceuticals are approved for human use and every one of them has multiple applications. Non-profits can connect funders with researchers to quickly deliver safe and effective repurposed treatments from these already available drugs and nutriceuticals through publication of results of pilot clinical trials.  While the outcome might not create a direct economic profit, it will often reduce suffering and healthcare costs for the millions who go to sleep every night with no other hope of a treatment or cure. This presentation will cover the following:

  • How Partnership for Cures came to focus on Rediscovery Research
  • Validating the supply and demand side of rediscovery research
  • Success stories

Dr. Bruce E. Bloom, President and Chief Science Officer, Cures Within Reach (formerly known as Partnership for Cures)

5:25 Chair's Closing remarks for Day 1


DAY TWO - October 24, 2012

8:45 Chairperson's Opening Remarks

Aris Persidis, Ph.D, President, Co-Founder, Biovista Inc.

8:55 Bulletproofing Your Patent Estate to Withstand a Generic Challenge
Generic drug companies are running out of targets. No longer are generics waiting for an established market. Repurposed drugs are often seen as a relatively easy target. Repurposed drugs have only 3 years exclusivity from FDA. These are often existing generics for the active ingredient and thus have low technical hurdles. Mr. Israelsen will discuss proven strategies for deflecting a generic challenge, including:

  • Getting a good method of protection for the core indication
  • Considering composition patents for drug form or combinations
  • Filing multiple "label" patents covering aspects of the approved label

Ned Israelsen, JD, San Diego Managing Partner and Patent Attorney, Knobbe Martens Olson & Bear LLP

9:30 Crowds to the Rescue: Using Open Innovation for Repositioning and Indication Discovery
A new paradigm is emerging in drug repositioning and indication discovery based on a fusion of crowdsourcing, telemedicine, and full data transparency. This new model enables patients, physicians, researchers, and other stakeholders to contribute to the selection and design of clinical trials, and promises to slash costs of trial execution, thereby delivering more drugs with correct labels to the patients who need them.

Marc Foster, MBA, Co-founder and Chief Operating Officer, Transparency Life Sciences, LLC

10:05 Reduce Cost and Timelines with the 505(b)(2) Drug Approval Process
The era of the blockbuster drug is over; companies are facing the patent cliff. Pipelines are drying up and jobs are being cut. In order to thrive, you must lower development risk, reduce cost and increase speed-to-market.

The FDA’s 505(b)(2) approval pathway lets companies modify and reposition existing drugs to provide improved safety and efficacy for marketing clearance. However, before you can determine if it’s a fit for you, you must understand how it works. First, you need to understand its unique regulatory requirements, then the best process to approach the FDA with your 505(b)(2) development plan.

Discover how to navigate the intricate 505(b)(2) process. Through recent case studies, Phelps will illustrate the variety of global development programs available under 505(b)(2).

Learning Points or Questions to be Answered:

  • The regulatory differences between 505(b)(1), 505(b)(2) and 505(j) submissions
  • The risks and benefits of each type of submission
  • The drug development process requirements of a 505(b)(2) submission
  • The costs savings and shortened timelines of a 505(b)(2) submission
  • Current candidates for 505(b)(2) submissions
  • Global drug development and approval using 505(b)(2)

Ken Phelps, President and CEO, Camargo Pharmaceutical Services

10:40 Refreshment Break

11:05 Panel Session: A New Era for Drug Repositioning: How Successes and Partnerships are Changing the Drug Development Process

Chair: Barry Bunin, Ph.D., CEO & President, Collaborative Drug Discovery, Inc.

David Cavalla, Ph.D. (virtual presenter), Founder, Numedicus Ltd., Principal, ATPBio

Christine M Colvis, Ph.D., Program Officer, National Institutes of Health, National Center for Advancing Translational Sciences (NCATS)

Christopher Lipinski, Ph.D. (virtual presenter), Scientific Advisor, Melior Discovery

12:20 Luncheon

1:35 Chemical Biology: Creating New Opportunities From Roche Advanced, But Terminated Compounds
The global Chemical Biology Initiative (CBI) is fostering multiple projects with a remit to bring new and novel targets into the portfolio for the disease therapeutic areas of focus at Roche. One of the CBI projects is based on using Roche advanced and annotated clinical compounds that were terminated. This set of over 300 compounds with advanced PK properties and toxicity screening data are being used to leverage external proteomic, genomic and phenotypic platform screening technologies to identify novel targets.

Currently, Roche is focusing on creative collaborations with external institutes to take advantage of this Roche collection and to identify novel target-disease associations, to develop novel target identification technologies and to repurpose compounds for new indications. In this presentation, Dr. Kondru will discuss Roche's work in this area.

Rama Kondru, Ph.D., Associate Director, Department Head, Computational Chemistry and Data Mining, Hoffmann-La Roche

2:10 Pricing, Reimbursement, Access Challenges and Opportunities for Re-positioning Therapies
Key challenges and possible opportunities will be presented from an EU & US  Payer perspective. Where applicable, analogues will be drawn upon to demonstrate historical pitfalls/successes coupled with forward thinking strategies to mitigate the cost constrained economic environment that seems to be putting up more barriers than successes for re-positioned therapies.

Sasha K. Richardson BSc, PT, MBA, Vice President, GfK Bridgehead

2:45 End of Conference

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