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New Drugs for Neglected Diseases New Hope for Forgotten Patients Jana Armstrong Executive Director, DNDi North America Par...
Overview Drugs for Neglected Diseases  initiative <ul><li>Neglected Patients </li></ul><ul><li>1 billion people afflicted ...
Disease Landscape Neglected Tropical Diseases (NTDs) <ul><li>13 diseases that affect the  poorest populations  in remote, ...
Disease Landscape Current Treatment Limitations Patients and Healthcare Workers Need Safe, Effective, Easy-to-Use Drugs Me...
Disease Landscape Human African Trypanosomiasis (HAT)  or Sleeping Sickness <ul><li>60 million at risk  in sub-Saharan Afr...
Disease Landscape Visceral Leishmaniasis (VL)  <ul><li>200 million at risk  worldwide (in 70 countries) </li></ul><ul><li>...
Disease Landscape Chagas Disease: A Silent Killer <ul><li>100 million at risk  in Latin America </li></ul><ul><ul><li>Kill...
Market Landscape A Fatal Imbalance <ul><li>World pharmaceutical market $712 billion in 2007* </li></ul><ul><li>Market-driv...
Market Landscape Global R&D funding in 2007 Source: Moran et al., G-Finder report, 2009 Neglected Diseases   $2.5 billion ...
DNDi “Business” Plan & Organization <ul><li>7 Founding Partners </li></ul><ul><li>Indian Council for Medical Research (ICM...
Strategy New compounds Screening of existing libraries >6 years Therapeutic switch “ Rediscovered” compounds 3-6 years New...
Exploratory Alternative formulations Amphotericin B  (VL) 6 to 8 new treatments Drug combination  (Chagas) Oxaborole  (HAT...
Accomplishments DNDi 6-Year Results <ul><li>2 new malaria treatments  delivered </li></ul><ul><li>1 new sleeping sickness ...
ASAQ (Malaria) Fixed-Dose Artesunate/ Amodiaquine NECT  (HAT) Nifurtimox - Eflornithine  Co-Administration ASMQ (Malaria) ...
Budget $187M of $410M  Secured (2003-2014)   <ul><li>Private Donors </li></ul><ul><li>Médecins Sans Frontières  (€29 M) </...
Budget ~$223M Still Needed for 3-5 More Treatments 2004-2014 Projected (€):
www.dndi.org By working together in a creative way,  we can bring innovation to neglected patients!
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DNDi: New Drugs for Neglected Diseases; New Hope for Forgotten Patients

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Jana Armstrong, Executive Director, DNDi North America, presents DNDi's research strategy at the 2009 Partnering for Cures meeting in New York.

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  • Primeiramente gostaria de parabenizar os organizadores pelo evento e agradecer a inclusão no programa de mini-conferências dando fala a novas iniciativas no âmbito da Doença de Chagas. Como já discutido na abertura por Tânia Araújo Jorge, Paulo Gadelha, assim como Prof. João Carlos Pinto Dias, devemos sim celebrar o centenário deste marco na histõria da ciência e da saúde brasileira que foi a descoberta da Doença de Chagas e comunicação ao mundo científico em 1909, assim como os progressos alcançados nos últimos anos, em particular o sucesso do controle vetorial. No entanto, é preciso seguir discutindo e agindo para preencher as lacunas existentes no conhecimento da Doença de Chagas, assim como a necessidade ampla utilização das ferramentas hoje disponíveis para seu diagnóstico e tratamento, assim como a necessidade de desenvolvimento de novas ferramentas para diagnóstico e tratamento do Mal de Chagas. Os gargalos existentes na Doença de Chagas, são comuns a várias das doenças negligenciadas, em especial as doenças de Tipo III, e resultam de falhas de mercado quanto de políticas públicas. Entre falhas de mercado, temos o d esenvolvimento de medicamentos enormemente concentrado na indústria farmacêutica com P&amp;D direcionada por lucros, onde os mais pobres s ã o negligenciados. Em paralelo, temos políticas públicas que não corrigem este desequilíbrio. Vivemos um momento de mudança no entanto, com aumento no investimento em P&amp;D para estas doenças, principalmente de países como o Brasil, discussão de novos mecanismos de financiamento e incentivo para pesquisa e desenvolvimento e criação de novas iniciativas, como parcerias para desenvolvimento de produtos. Como uma das iniciativas para lidar com as limitações existentes no diagnóstico e tratamento e com gargalos existentes na P&amp;D para doenças negligenciadas, a DNDi foi criada em 2003.
  • Transcript of "DNDi: New Drugs for Neglected Diseases; New Hope for Forgotten Patients"

    1. 1. New Drugs for Neglected Diseases New Hope for Forgotten Patients Jana Armstrong Executive Director, DNDi North America Partnering for Cures 2009 Thursday, Dec 3, 2010
    2. 2. Overview Drugs for Neglected Diseases initiative <ul><li>Neglected Patients </li></ul><ul><li>1 billion people afflicted </li></ul><ul><li>530,000 deaths/year </li></ul><ul><li>Poor, marginalized populations </li></ul><ul><li>Most Neglected Diseases </li></ul><ul><li>Ineffective, toxic, expensive, archaic treatments </li></ul><ul><li>No financial incentives to motivate profit-driven pharma industry </li></ul><ul><li>Insufficient public sector leadership </li></ul><ul><li>Non-profit drug R&D organization </li></ul><ul><li>Addressing the needs of the most neglected patients </li></ul><ul><li>Harnessing resources from public institutions, private industry and philanthropic entities </li></ul><ul><li>Three new treatments delivered since 2003 </li></ul>DNDi: an alternative model for drug development “ an experiment borne out of extreme frustration about health care for the poorest”
    3. 3. Disease Landscape Neglected Tropical Diseases (NTDs) <ul><li>13 diseases that affect the poorest populations in remote, rural areas, urban slums or conflict zones </li></ul><ul><li>Urgent patient needs: </li></ul><ul><ul><li>improve access & improve innovation </li></ul></ul><ul><li>DNDi Focus: innovation for the most neglected patients and diseases </li></ul><ul><ul><li>Comprehensive drug development strategy for “most neglected” diseases </li></ul></ul><ul><ul><li>Respond to patient needs for efficacious, safe and accessible treatments </li></ul></ul>Visceral Leishmaniasis (VL) Sleeping Sickness (HAT) Chagas Disease Malaria
    4. 4. Disease Landscape Current Treatment Limitations Patients and Healthcare Workers Need Safe, Effective, Easy-to-Use Drugs Melarsoprol (arsenic-based IV drug) “Fire in the Veins” Benznidazole: adult tablets fractioned or macerated for pediatric use <ul><li>Ineffective (resistance) </li></ul><ul><li>Toxic </li></ul><ul><li>Expensive </li></ul><ul><li>Painful when delivered </li></ul><ul><li>Difficult to use (i.e. IVs requiring round-the-clock nursing) </li></ul><ul><li>Not registered in endemic regions </li></ul><ul><li>Restricted by patents </li></ul><ul><li>Lack of pediatric formulations </li></ul>
    5. 5. Disease Landscape Human African Trypanosomiasis (HAT) or Sleeping Sickness <ul><li>60 million at risk in sub-Saharan Africa </li></ul><ul><li>Transmitted by the tsetse fly </li></ul><ul><li>Difficult to diagnose ; many patients go undiagnosed until late stage of disease </li></ul><ul><li>Fatal if untreated </li></ul><ul><li>Needs: </li></ul><ul><ul><li>A safe, effective, and practical stage 2 treatment </li></ul></ul><ul><ul><li>A simple stage 1 treatment </li></ul></ul>
    6. 6. Disease Landscape Visceral Leishmaniasis (VL) <ul><li>200 million at risk worldwide (in 70 countries) </li></ul><ul><li>Transmitted by the sandflies </li></ul><ul><li>Symptoms: prolonged fever, enlarged spleen & liver, substantial weight of loss, progressive anemia </li></ul><ul><li>Fatal if untreated </li></ul><ul><li>Current drugs: antimonials, Amphotericin B, AmBisome ® , miltefosine, paromomycin </li></ul><ul><li>Needs : </li></ul><ul><ul><li>Oral, safe, effective, low-cost and short-course treatment </li></ul></ul>
    7. 7. Disease Landscape Chagas Disease: A Silent Killer <ul><li>100 million at risk in Latin America </li></ul><ul><ul><li>Kills more people in region than malaria </li></ul></ul><ul><ul><li>Patient number growing in non-endemic, developed countries </li></ul></ul><ul><li>Transmitted by ‘ kissing bug ’ , blood transfusion, organ transplantation, as congenitally or orally </li></ul><ul><li>Majority of patients undiagnosed until late stage </li></ul><ul><li>30% will develop life-threatening heart disease or gastrointestinal complications </li></ul><ul><li>Needs: </li></ul><ul><ul><li>An affordable, age-adapted, safe, and efficacious paediatric strength </li></ul></ul><ul><ul><li>A new drug for early chronic stage </li></ul></ul>
    8. 8. Market Landscape A Fatal Imbalance <ul><li>World pharmaceutical market $712 billion in 2007* </li></ul><ul><li>Market-driven, private enterprise focused on ROI and profitable drugs </li></ul><ul><li>Neglected Diseases: </li></ul><ul><ul><li>affect poorest populations in developing countries </li></ul></ul><ul><ul><li>lie outside the world “market” </li></ul></ul><ul><ul><li>patient needs remain unaddressed due to market and public policy failures </li></ul></ul>*Source: IMS Health, 26.2.2008 Chart Data Source: Chirac P, Torreele E. Lancet . 2006 May 12; 1560-1561.
    9. 9. Market Landscape Global R&D funding in 2007 Source: Moran et al., G-Finder report, 2009 Neglected Diseases $2.5 billion (US) Kinetoplastids $125 million (US)
    10. 10. DNDi “Business” Plan & Organization <ul><li>7 Founding Partners </li></ul><ul><li>Indian Council for Medical Research (ICMR) </li></ul><ul><li>Kenya Medical Research Institute (KEMRI) </li></ul><ul><li>Malaysian MOH </li></ul><ul><li>Oswaldo Cruz Foundation Brazil </li></ul><ul><li>Medecins Sans Frontieres (MSF) </li></ul><ul><li>Institut Pasteur France </li></ul><ul><li>WHO/TDR (observer) </li></ul>7 worldwide offices <ul><li>Objectives </li></ul><ul><li>Deliver 6 - 8 new treatments by 2014 for neglected diseases, with robust pipeline (malaria, Chagas, sleeping sickness, leishmaniasis ) </li></ul><ul><li>Use and strengthen research capacity in disease endemic countries </li></ul><ul><li>Raise awareness and advocate for increased public responsibility </li></ul><ul><li>Key features </li></ul><ul><li>Patient-needs focused </li></ul><ul><li>Founding partners and governance include research institutes in disease-endemic countries </li></ul><ul><li>R&D project management team with strong pharma, biotech, drug development experience </li></ul><ul><li>Global but lean core team managing a variety of partnerships & consultants </li></ul><ul><ul><ul><li>120+ scientific partners, including large pharmaceutical firms, small biotechs, universities, and public institutions </li></ul></ul></ul><ul><li>Investment required: ~$400M / d iversified sources </li></ul>Brazil India Kenya Malaysia USA DRC Japan Geneva Coordination Team + consultants
    11. 11. Strategy New compounds Screening of existing libraries >6 years Therapeutic switch “ Rediscovered” compounds 3-6 years New formulations (FDC) Geographical extensions Co-administration  3 years R&D Strategy DNDi Portfolio-Building Model Long-Term projects Medium-term projects Short-term projects
    12. 12. Exploratory Alternative formulations Amphotericin B (VL) 6 to 8 new treatments Drug combination (Chagas) Oxaborole (HAT) Nitroimidazole backup (HAT) Fexinidazole ( HAT) ASAQ (Malaria) Fixed-Dose Artesunate/ Amodiaquine ASMQ (Malaria) Fixed-Dose Artesunate/ Mefloquine Combination therapy (VL in Asia) Paediatric benznidazole (Chagas) Azoles (Chagas) Exploratory <ul><li>Combination therapy (VL in Africa) </li></ul><ul><ul><li>Paromomycin </li></ul></ul><ul><ul><li>AmBisome® </li></ul></ul><ul><ul><li>Miltefosine </li></ul></ul><ul><li>8-aminoquinolines – in preparation (VL) </li></ul><ul><li>Sitamaquine </li></ul><ul><li>Tafenoquine </li></ul>Combination therapy (VL in Latin America) – in preparation NECT Nifurtimox - Eflornithine Co-Administration Stage 2 HAT <ul><li>2 HAT LO Consortium </li></ul><ul><li>Scynexis </li></ul><ul><li>Pace Univ </li></ul><ul><li>Chagas LO Consortium </li></ul><ul><li>CDCO </li></ul><ul><li>Epichem </li></ul><ul><li>Murdoch Univ </li></ul><ul><li>VL LO Consortium </li></ul><ul><li>Advinus </li></ul><ul><li>CDRI </li></ul>Reference screening centres: LSHTM, Swiss Tropical Institute, University of Antwerp Exploratory <ul><li>Compound mining </li></ul><ul><li>E.g.: nitroimidazoles, … </li></ul><ul><li>Chemical classes </li></ul><ul><li>E.g.: macrolides, GSK, Merck, … </li></ul><ul><li>Target-based </li></ul><ul><li>E.g. Dundee’s Drug Discovery Unit (DDU), … </li></ul><ul><li>Screening </li></ul><ul><li>E.g. natural products (Kitasato, Eskitis), new technology (Institut Pasteur Korea), DDU at Dundee, … </li></ul>R&D Strategy Robust Portfolio – November 2009 a robust pipeline
    13. 13. Accomplishments DNDi 6-Year Results <ul><li>2 new malaria treatments delivered </li></ul><ul><li>1 new sleeping sickness combination delivered </li></ul><ul><li>Largest pipeline ever for the kinetoplastid diseases </li></ul><ul><li>Clinical research platforms in Africa </li></ul><ul><li>$187M of $410M needed raised </li></ul><ul><li>On track to deliver new treatments per business plan </li></ul>
    14. 14. ASAQ (Malaria) Fixed-Dose Artesunate/ Amodiaquine NECT (HAT) Nifurtimox - Eflornithine Co-Administration ASMQ (Malaria) Fixed-Dose Artesunate/ Mefloquine Accomplishments 3 New Treatments Delivered Farmanguinhos (Brazil) Cipla (India) sanofi-aventis (France) National Control Programs MSF WHO 2007 2008 2009 Partners <ul><li>Easy to Use </li></ul><ul><li>Affordable </li></ul><ul><li>Field-Adapted </li></ul><ul><li>Non-Patented </li></ul>
    15. 15. Budget $187M of $410M Secured (2003-2014) <ul><li>Private Donors </li></ul><ul><li>Médecins Sans Frontières (€29 M) </li></ul><ul><li>Bill & Melinda Gates Foundation (€42M) </li></ul><ul><li>Other Private Foundations </li></ul><ul><li>Public Donors </li></ul><ul><li>United Kingdom - DFID (€29 M) </li></ul><ul><li>France – AFD & MAEE (€8 M) </li></ul><ul><li>Spain – AECID (€7.5 M) </li></ul><ul><li>Netherlands – DGIS (€3 M) </li></ul><ul><li>USA – NIH/NIAID (€1 M) </li></ul><ul><li>Germany – GTZ (€1 M) </li></ul><ul><li>Switzerland - Canton de Genève & DDC </li></ul><ul><li>European Union - FP 5, 6,7 </li></ul><ul><li>Italy - Region of Tuscany </li></ul><ul><li>Funding Strategy: Independence through diversified sources of funding </li></ul><ul><li>Approximately 50% of funding from public institutional donors in line with DNDi’s advocacy objective (public responsibility for NDs) </li></ul><ul><li>Approximately 50% from private sector (foundations, major donors, general public) </li></ul><ul><li>Key contributions to come from Founding Partners </li></ul><ul><li>Maximum of 25% per donor </li></ul>
    16. 16. Budget ~$223M Still Needed for 3-5 More Treatments 2004-2014 Projected (€):
    17. 17. www.dndi.org By working together in a creative way, we can bring innovation to neglected patients!
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