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Target Health Inc.

Encouraging Development of Therapeutics for Neglected Diseases - Day 1

Day 1  |  Day 2 



7:30 am Registration and Morning Coffee

8:30 Chairperson’s Opening Remarks

Ian Gilbert, Ph.D., Professor, Drug Discovery Unit, University of Dundee

8:35 Facilitating R&D for Neglected Diseases: Fostering Collaboration and Navigating the Complex Interface among Diverse Stakeholders

Jacqueline B. Fine, Ph.D., CLPTM, Associate Director, Global Outlicensing and Asset Management, Merck Research Laboratories

The challenges we face toward significantly impacting global health, particularly in the developing world, are too complex for any single entity to solve alone. Partnership, as a critical pillar of Merck's global access strategy will be highlighted, with emphasis on depictions of how robust collaboration among individuals and organizations with diverse backgrounds and capabilities joined together by the mutual goal of improving human health can lead to true innovation. The route to achieving mutual goals may vary from partnership to partnership with respect to specific strategies, targets and needs of the parties; however, we believe the agreements supporting these collaborations should be aligned with current and evolving best practices and policies that include ensuring access on a sustainable basis. A spectrum of activities will be described, from licenses facilitating development of Merck assets for neglected disease indications by public-private partnerships, to a first-of-its-kind joint venture designed to be a sustainable R&D organization that operates like a business – but with a not-for-profit model – aimed at developing new vaccines in areas of unmet need and optimizing existing vaccines for resource-limited settings.

9:05 Drug Discovery for Neglected Diseases: Molecular Target-Based and Phenotypic Approaches

Ian Gilbert, Ph.D., Professor, Drug Discovery Unit, University of Dundee

I will describe the setting up of a fully integrated drug discovery unit within an academic sector, and how we are applying this for drug discovery for neglected diseases. The focus of the talk will be on human African trypanosomiasis, where we have used both molecular target-based and phenotypic approaches to discover starting points for drug discovery; I will discuss the relative merits of these approaches.

9:35 The Future of Industry-PDP Collaborations: Integrating Global Health into New Pharma Business Model?

Jean-Pierre Paccaud, Director, Business Development, Drugs for Neglected Diseases Initiative

The emergence of PDPs developing new treatments and vaccines against neglected tropical diseases provides hope for millions of patients awaiting life saving therapeutics the industry failed to develop because of the lack of a market. Although it took PDPs several years to succeed in involving industry into their activities, today most pharma companies advertise their relationship with one or several PDPs. Most of the collaborations are still at the discovery or pre-clinical stage, where pharma involvement does not generate significant costs for the industry. PDPs have demonstrated various achievements in R&D, developing products or vaccines with the help of their industrial collaborations, but they may face difficult challenges when it comes to industrial manufacturing, registration and distribution if they are not backed up by a strong industrial partner. At this stage, will industry be willing to engage in insuring sustainable drug/vaccine delivery at conditions enabling the neglected patients to access the drug? What can be the alternatives? What framework would be favorable for continued private sector involvement?

Sponsored by
10:05 SCYNEXIS Integrated Parasitology Screening: Focusing Biotechnology Innovation To Achieve Neglected Disease Solutions 
Yves Ribeill, Ph.D., President & CEO, SCYNEXIS, Inc.
Current strategies to identify new drugs for Neglected Tropical Diseases may overlook a rich source of innovation: compound collections from biotechnology companies.  By evaluating these compound collections for properties that are aligned with the Target Product Profiles of Neglected Diseases they can quickly be focused towards optimization.  The SCYNEXIS Integrated Parasitology Screening platform provides the rapid assessment of compounds against over thirty parasite species relevant to Global Health.

10:35 Coffee Break in the Exhibit Hall with Poster Viewing

11:05 Blockade of iNKT Cells as a Therapeutic Strategy for Sickle Cell Disease

Robert Mashal, Ph.D., CEO, Office of the President, NKT Therapeutics

Blockade of iNKT cells is a promising new therapeutic strategy for the treatment of SCD. NKT Therapeutics is developing NKTT120, a novel antibody which specifically blocks iNKT cell function.

11:35 Distributed Drug Discovery (D3): Addressing Large Neglected Disease Challenges with Multiple Small-Scale and Open-Access Resources

William L. Scott, Ph.D., Research Professor, Department of Chemistry and Chemical Biology, Indiana University Purdue University Indianapolis (IUPUI)

Distributed Drug Discovery (D3) capitalizes on the powerful potential that comes from linking worldwide educational resources in an open-access framework. It creates synthetic procedures and equipment to enable students in distributed global locations to reproducibly make new neglected disease drug-lead candidates selected from large D3 virtual catalogs. With the freedom that comes from open-access it simultaneously harnesses the expertise of synthetic, computational and biological researchers while educating the next generation of drug discovery scientists.

12:05 pm Challenges in the New Drug Discovery for Tuberculosis - A TMC207 Perspective and Update

Nacer Lounis , Ph.D., Microbiologist, Department of Antimicrobial Research, Janssen Research and Development, Johnson and Johnson Pharmaceuticals

  • Discovery  and developmental challenges for identifying new TB drugs.
  • Update on TMC207 : A new investigation drug in clinical studies for MDR-TB.
  • Energy metabolism as a new target pathway  for mycobacterial drug research

12:30 Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own

1:55 Chairperson’s Remarks

Julie Lotharius, Ph.D., Associate Director, Translational Medicine, Medicines for Malaria Venture

2:00 Recent Advances in Visceral Leishmania Vaccine Development Using Genetically Altered Live Attenuated Parasites

Hira Nakhasi, Director, Division of Emerging and Transfusion Transmitted Diseases, OBRR/CBER/FDA

Leishmaniasis is a major Neglected Tropical Disease. There are no vaccines against Leishmania. Strategies such as killed parasites with or without adjuvants, subunit vaccines (recombinant proteins or DNA vaccines) have not resulted in a viable Leishmania vaccine. However, people who have recovered from infection develop a protective immunity against future infections. Hence we have used the strategy of developing live attenuated parasites which do not cause disease but develop protective immunity against various species of Leishmania.

2:30 Discover New Biologics for Protection Against Infection by Different and New Viruses

Grace Wong, Ph.D., CEO, (former Genentech, Millennium, AstraZeneca and Serono), ActoKine Therapeutics

We have identified ActoKine-2 (AK-2) which protects cells against infection by a broad spectrum of viruses. AK-2 was highly active against different viruses including West Nile viruses, bird flu, Rift Valley fever virus, Punta Toro phlebovirus, HIV, Yellow Fever Virus, Tacaribe virus, and Venezuelan equine encephalomyelitis (VEE) virus. AK-2 could be administered sub-lingually or nasally to prevent infection through the nose or mouth or intravaginally to prevent against sexually transmitted diseases. These novel routes of administration can be safer, cheaper, and logistically easier than traditional injections for disease prevention.

3:00 Expanding Elimination and Control Programs for the Five NTDs: The Challenge with Determining Needs

Mawuli Nyaku, Scientist, Division of Parasitic Disease and Malaria, Center for Global Health, CDC

3:30 Refreshment Break in the Exhibit Hall with Poster Viewing

4:15 Partnerships in Neglected Disease Research: A Pharmaceutical Industry Perspective

James Brown, Ph.D., Director, Computational Biology, Quantitative Sciences, GlaxoSmithKline

Malaria, tuberculosis and the various kinetoplastid diseases, trypanosomosis and leishmaniasis, collectively account for the most widespread and serious infectious diseases.  To address this global health burden, GlaxoSmithKline (GSK) is actively pursuing open lab research initiatives which include opening our relevant data and research, engaging in private-public partnerships and offering research spaces in our labs to scientists and academics from universities, non-profit organisations and other research institutes.  Besides the external facing partnerships, internal collaborations across GSK R&D units are also contributing to neglected disease research, such computational biology analyses of potential targets and drug repositioning opportunities.  In this presentation, the wider view of pharmaceutical company research partnerships, both internal and external, to address neglected disease research will be discussed. 

4:45 Focused Breakout Discussions

Topic 1: Clincial Research Software Tools to Enhance Development of Drugs for Neglected Diseases

Moderator: Dean Gittleman, Senior Director, Operations, Target Health, Inc.

  • Issues associated with data collection & management in remote locations
  • How can available technology be part of the solution?
  • How direct data entry and eSource electronic files can mitigate the risk of using paper records

Topic 2: Pricing of Drugs for Neglected Tropical Diseases

Moderator: Jean-Pierre Paccaud, Director, Business Development, Drugs for Neglected Diseases Initiative

  • How to engineer mechanisms that would entice the private sector to participate, although knowing that only minimal margins could be made on such products?
  • What are considered as "acceptable margins", or price for patients? 
  • How to take into account local conditions in pricing a product (extended tiered pricing)? 
  • What other mechanisms would enable the private sector to contribute?

Topic 3: Developing Robust Collaborations

Moderator: Jacqueline B. Fine, Ph.D., CLPTM, Associate Director, Global Outlicensing and Asset Management, Merck Research Laboratories

Topic 4: Strategies for Developing Vaccines

Moderator: Hira Nakhasi, Director, Division of Emerging and Transfusion Transmitted Diseases, OBRR/CBER/FDA

6:00 End of Day

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