Day 1 | Day 2
DAY TWO: WEDNESDAY, OCTOBER 14, 2009
Sponsored by
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7:45 am Continental Breakfast Presentation
505(b)(2): Not Just for Modifications of Approved Drugs
Ken Phelps, President & Chief Executive Officer, Camargo Pharmaceutical Services
Historically, most sponsors have used the 505(b)(2) pathway 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). This understanding will open up a vast source of potential drug products for development over the next decade. This Session will review the regulatory requirements for a 505 (b)(2) NDA and the kinds of information that may be used in support of an NDA submission, as well as the process needed to gain FDA buy-in to the 505 (b)(2) development plan and some of the products currently being developed without a U.S.-approved reference drug.
White Paper
Click here to dowload the "Understanding The 505(b)(2) Approval Pathway" white paper
8:30 Chairperson’s Opening Remarks
8:40 The CTSA Pharmaceutical Assets Portal
Kate Marusina, Ph.D., MBA, Manager, Industry Alliances, University of California, Davis
The Portal is the first systematic, large scale effort to expand the participation of the academia in drug repositioning by matching the scientific expertise of the CTSA academic scientists with the “shelved” compounds. The marriage of the academic and commercial goals would undoubtedly result in an increased number of approved drugs for new indications, and thus in considerable public benefit.
9:10 Screening Thousands of Drugs Directly in Leukemia Patient Samples
Juan Ballesteros, Ph.D., Chairman & Chief Scientific Officer, Vivia Biotech SL
Vivia is pioneering the screening of thousands of known drugs directly in fresh patient samples (ex vivo) of Hematological Malignancies using its proprietary ExviTech platform. A network of hospitals provide same day bone marrow and/or peripheral blood samples of patients being diagnosed. Up to 2,000 drugs and combinations can be screened per day in whole blood with minimal alterations following systems biology principles. Vivia has identified 4 non-cytotoxic very safe known drugs which kill selectively CLL leukemic cells ex vivo with the same efficacy as the approved cytotoxics, having minimal effect in healthy cells. PoC clinical trials will start this year using the commercialized formulation. Phases I/II using new formulations are planned for 2010.
9:40 What Else Should my Drug Do? Systematic Drug Repositioning and Adverse Event Profiling Sponsored by 
Aris Persidis, Ph.D., President, Biovista, Inc.
How do you know what the best indication for a drug or target is, beyond typical disease area constraints? Biovista screens the mechanism of action (MoA) of any drug or target against the MoA of 8,000 indications and 12,000 adverse events (AEs). This is simultaneous, unbiased indication discovery and AE profiling, and it is unique. Case studies of BVA-101, BVA-201 and BVA-601 in multiple sclerosis and epilepsy will be described, as well as the impact on lifecycle management and development costs.
10:10 Networking Coffee Break with Exhibit and Poster Viewing
10:50 Repositioning of Products to Explore New Therapeutic Potential and Markets: A CDRI Case Study
Zaka Imam, Ph.D., Scientist G & Head, TIILP Division, Central Drug Research Institute
In India, the Central Drug Research Institute (CDRI) is the only publicly funded institute which, after its inception in 1951, made the country proud in drug R&D on at least two counts: First, initiation of a systematic program for drug R&D for development of modern drugs, thus providing leadership in this area, in the early stages; Second, development and commercialization of in-house developed modern drugs. In spite of a leadership position acquired by the institute due to the large nursery of compounds and research findings it made, developing a blockbuster drug remained a distant dream for the institute. The paper examines repositioning approaches as a strategic exercise to retrieve different molecules and products lost -- for reasons of sustained follow-up, safety and efficacy concerns, or lack of commercialization will by licensee firms, during pre-clinical/clinical stages -- to develop valuable leads, and invite global cooperation to develop and commercialize the most potential entities. The repositioning exercise ultimately aims to bring to the laboratory bench known products with their associated knowledge, to undertake informed follow-up discussion and decision, and studies required for development of new and better products, at reduced cost and time. In addition, this strategy has the chance to develop products, or one/two blockbuster drugs, for global launching.
11:20 Knowledge Management Technologies for Drug Repurposing
Natalia Novac, Ph.D., Scientist, Research & Development, Knowledge Management, Merck-Serono
In my talk, I will tell about current knowledge management technologies we use at Merck-Serono for repurposing and extended profiling of internal compounds. In the course of several repurposing projects, knowledge management has developed a strategy towards hypothesis generation and validation. Several steps leading to the hypothesis generation for the top-ranked indication and subsequent hypothesis validation via clinical data analysis will be discussed to give the whole picture of the knowledge management support for repurposing projects. The immense amount of knowledge existing in proprietary and public domain is of great use for repurposing. The knowledge management technologies are absolutely required to drive through the huge amount of data in the search for new indication.
11:50 Intradermal Drug Delivery: A Novel Approach for Drug Repositioning Using Nanotechnology
Bai Xu, Ph.D., President & Chief Executive Officer, Nanomed Devices, Inc.
About $70 billion of brand drugs are affected by patent expiration. Pharmaceutical companies need innovative tools for product life-cycle management. An intradermal drug delivery device based on nanofabrication technology is one of these tools. This device, enabled by microneedles that can temporarily compromise the skin barrier layer, is designed to safely and more effectively deliver drugs, including protein drugs that, until now, must be delivered by needle injection. It overcomes the limitation of traditional transdermal patch.
12:10 pm Lunch Presentation (Opportunity Available) or Lunch on Own
1:25 Chairperson’s Remarks
Aaron Schimmer, M.D., Ph.D., FRCPC, Staff Physician and Scientist, Princess Margaret Hospital, Ontario Cancer Institute; Assistant Professor, Departments of Medicine and Medical Biophysics, University of Toronto
1:30 Identification of FDA-approved Drugs with new Activity Against Leukemia
Aaron Schimmer, M.D., Ph.D., FRCPC
To identify FDA-approved drugs with previously unrecognized anti-cancer activity, we screened an in-house library of on-patent and off-patent drugs for potential anti-leukemia agents including those cytotoxic to leukemia stem cells. From these screens we have identified 4 compounds that are advancing into phase I clinical trial for leukemia, including the anti-fungal ciclopirox olamine.
2:00 Case Studies of Drug Repositioning in Japan
Tohru Mizushima, Ph.D., Director, Research Institute for Drug Discovery; Professor, Graduate School of Medical and Pharmaceutical Sciences, Kumamoto University
Japan is working to develop a consortium (government, academia and industry) to further the field of drug repositioning in Japan. The consortium focuses on the regulation (such as drug price) and liaison to scientific meetings, such as drug delivery system. In this year’s drug repositioning summit, we will present a drug repositioning study about non-steroidal anti-inflammatory drugs (NSAIDs) and geranylgeranylacetone (GGA), a leading anti-ulcer drug on the Japanese market. For NSAIDs, we showed that both COX inhibition at gastric mucosa and membrane permeabilization activity of NSAIDs are required for the production of gastric lesions and we developed NSAIDs that do not exhibit membrane permeabilization activity as safer NSAIDs for the gastrointestinal tract. Based on these results, we concluded that analysis of clinically using drugs would provide useful information about molecular mechanism of their actions and drug repositioning studies.
2:30 Networking Refreshment Break with Exhibit and Poster Viewing
3:10 Sponsored Presentation (Opportunities Available)
3:40 Drug Repositioning and Neglected Diseases and The Johns Hopkins Clinical Compound Screening Initiative
Curtis R. Chong, M.D., Ph.D., Internal Medicine, Massachusetts General Hospital
The Johns Hopkins Clinical Compound Screening Initiative aims to reduce the cost and time necessary for new drug development by finding new uses for existing drugs. Our goal is to collect all of the 11,000 drugs ever used in medicine and relentlessly test this collection to identify new uses for existing drugs. Currently our collection of 3,100 existing drugs is being screened by several dozen collaborators at Hopkins and elsewhere on a variety of diseases including malaria, angiogenesis, cancer and HIV. We eagerly welcome collaborators who are as enthusiastic as we are about bridging the gap between the lab and the clinic by finding new uses for existing drugs.
4:10 Collaborative Opportunities in Huntington’s Disease
Hyunsun Park, Ph.D., Director, Translational Biology, CHDI Foundation
Huntington’s disease is a fatal inherited neurological disorder characterized by motor, cognitive, and psychiatric symptoms. Tetrabenazine is a recent example of an old drug repositioned for symptomatic treatment of the chorea associated with HD. CHDI Foundation has been laying the groundwork for conducting clinical trials of disease-modifying agents and we have collected target validation data on a long list of potential targets. We have a robust in vitro/in vivo HD assay platform and an experienced clinical team. We can lower the barrier for companies to reposition their pre-development compounds and failed drug candidates for HD by providing reagents, domain knowledge, and funding.
4:40 Close of Drug Repositioning Summit
For more information, please contact:
Christina Lingham
Cambridge Healthtech Institute
T: 781-972-1346
E: clingham@healthtech.com
For partnering and sponsorship information, please contact:
Arnie Wolfson
Business Development Manager
781-972-5431
awolfson@healthtech.com