ANTHELMINTHIC VACCINES

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1 Institute of Medicine ANTHELMINTHIC VACCINES The Antipoverty Vaccines Peter Hotez MD PhD

2 Drug Failure: Mebendazole for Hookworm ONLY 15% Cure Rates (Keiser & Utzinger JAMA 2008)

3 Rx with Mebendazole Does NOT Result in Improvement in Anemia Study ID SMD (95% CI) % Weight Albendazole Stephenson 1990 Adams 1994 Dossa 2001 Dossa 2001 Gilgen 2001 Gilgen 2001 Subtotal (I-squared = 0.0%, p = 0.997). Mebendazole Stolzfus 1998 Thi Le 2007 Thi Le 2007 Subtotal (I-squared = 0.0%, p = 0.730). Overall (I-squared = 0.0%, p = 0.885) 0.27 (-0.42, 0.95) 0.11 (-0.42, 0.64) 0.24 (-0.23, 0.71) 0.14 (-0.33, 0.62) 0.12 (-0.11, 0.36) 0.15 (-0.09, 0.39) 0.15 (0.01, 0.29) 0.03 (-0.06, 0.11) (-0.40, 0.22) (-0.34, 0.27) 0.01 (-0.07, 0.10) 0.05 (-0.02, 0.12) NOTE: Weights are from random effects analysis Smith and Brooker

4 R&D Funding for Neglected Diseases: Not the 10/90 Gap : The 1/99 Gap Disease R&D Support in 2008 % Funding Big Three $2.25 billion 73% HIV/AIDS $1.21 billion 39% Malaria $ 566 million 18% Tuberculosis $ 468 million 16% Non Big Three diseases $ 750 million 27% Kinetoplastid $ 146 million 05% Diarrheal disease $ 138 million 05% Pneumonia/meningitis $ 133 million 04% Helminth infections $ 69 million 02% Leprosy/Trachoma/Buruli $ 14 million 01% $3 Billion Total in Neglected Disease R&D but only 20%+ for the NTDs

5 THE RIGHT OF ACCESS TO INNOVATION A scientist who is also a human being cannot rest while knowledge which might reduce suffering rests on the shelf. Dr. Albert B. Sabin

6 Sabin Vaccine Development PDP for NTD Antipoverty Vaccines

7 Brazil s NTD Problem: Doenças de Jeca Tatu Disease % Burden LAC No. Cases in Brazil Trachoma 97% 1.06 million Leprosy 93% 44,436 Schistosomiasis 83% 1.5 million Kala-azar 67% 3,386 Hookworm 65% 32.3 million Dengue 63% 346,471 Ascariasis 50% 41.7 million

8 What we do? Major activities Develop a Target Product Profile (TPP) Antigen discovery through genomics/proteomics Process development 10 L fermentation scale Adjuvant formulation Technology transfer cgmp pilot manufacture with developing country manufacturers Regulatory filings (FDA and national regulatory authorities) Phase 1 and 2 clinical testing in disease endemic areas

9 Process Development: Bridging the Valley of Death Antigen Discovery Research & Development Product Development Strategy Process Development / Quality Control Technology Transfer to cgmp Facility cgmp Manufacture Phase 1 Clinical Trials

10 Transitioning into development Product Development Process Development Assay Development Yield Purity Adsorption In process testing Lot Release testing Stability testing Recombinant protein Vaccine Formulation Temp. Incursions Scalability and Consistency Technology Transfer & cgmp Manufacture Accelerated Long-term GLP Animal Toxicology Clinical Trials

11 Sabin-Brazil Technology Transfer

12 Sabin PDP Clinical Field Site: Americaninhas, Minas Gerais, Brazil Hookworm 32 million cases 68% Minas Gerais Schistosomiasis 2-7 million cases 45% Minas Gerais Hookworm and Schisosomiasis Co-Infections

13 Current Portfolio of Vaccines Human Hookworm Vaccine Bivalent recombinant protein vaccine: Na-GST-1 and Na-APR-1 Alhydrogel formulation + synthetic lipid A IND filing FDA Phase 1 testing Brazil Human Schistosomiasis Vaccine Monovalent recombinant protein vaccine: Sm-TSP-2 Alhydrogel formulation + synthetic lipid A cgmp pilot manufacture Brazil Transmission Blocking Malaria Vaccine Monovalent recombinant protein vaccine: AgAPN1 Alhydrogel formulation Process development USA

14 NTDs and Poverty Impair intellectual & physical development in children NTDs PROMOTE POVERTY Cause adverse pregnancy outcomes Reduce productive capacity/ worker productivity ANTIPOVERTY VACCINES Hotez PJ, Fenwick A, Savioli L Molyneux DH. Lancet 2009; 373: Property of the Global Network

15 The Global Distribution of Human Hookworm Infection Necator americanus is the predominant species 22 million DALYs ½ GBD of Malaria 65,000 deaths Intestinal blood loss: 25 Necator worms = 1 ml blood loss = 0.55 mg Fe = Child s daily iron intake 19-73% Global Iron Deficiency Anemia (IDA) East Africa, Nepal, Brazil, Vietnam (Stoltzfus & Others)

16 Pediatric Hookworm: 41% IDA & 57% Severe Anemia Pallor and Facial Edema Anasarca Loss in Intelligence Reduced growth, psychomotor development, physical fitness Developmental delays, lower IQ, Reduced school performance & attendance

17 The Human Hookworm Vaccine Anti-Enzyme Antibodies: Targeting Hookworm Blood Loss

18 APR-1 (Blood-feeding Aspartic Protease) hemoglobin (dg/l) APR-1 Control P= Anti-APR-1 Antibodies in Hookworm Gut LC Days post 1 st vaccination mutated Asp to Ala Anti-APR-1 antibodies reduce host blood loss APR-1 ASO3 70% (P = 0.05) 0 2,000 4,000 6,000 8,000 10,000 12,000 pepstatin Structure of Na-APR-1 red Na-APR-1 mut blue -pepsin Reduce fecal egg counts Loukas et al. PLoS Medicine 2005; 2: e295

19 APR-1 1 VACCINATION REDUCES HOST BLOOD LOSS 14 APR-1 Control 14 hemoglobin (g/dl) hemoglobin (g/dl) 8 Pre Post Pre Post 8 Hb concentrations in APR-1 dogs were significantly greater ( p = <0.05) than control canines Loukas, Bethony, Mendez et al. PLoS Medicine 2005

20 Na-GST-1: Heme Binding and Detoxification 3-D Structure 24 kda Homodimers Immuno- Localization of Na-GST-1 Modified Nu Class GST-1 Adapted for Blood-Feeding 40-71% Worm Burden Reduction Asojo et al. BMC Structural Biology 2007; Zhan et al. Infect. Immun. 2005

21 Na-GST-1 + Na-APR-1 (Co-Administered) Phase 1 NE Na-GST-1 (Adults) Phase 1 Na-GST-1 (Children) E E Phase 2 Na-GST-1 + Na-APR-1 (Children) E Trial Location: NE Phase 1 NE Na-GST-1 + Na-APR-1 (Adults) Phase 1 Na-GST-1 + Na-APR-1 (Children) Non-endemic (Belo Horizonte) E E E Endemic (Americaninhas) 21

22 A multi-antigen fusion protein vaccine Na-APR-1 is an efficacious antigen but is difficult to express at high yield Protection is thought to be mediated by antibodies that neutralize enzymatic function Neutralizing mabs map to fragment 5a We are now fusing APR-1 fragment 5a to the C-terminus of N. americanus Na-GST-1 to produce a chimeric antigen Fuse Na-APR-1 fragment 5a to C-terminus of Na-GST-1 fragment 5a Na-GST-1 Na-APR-1

23 Countries with High Hookworm & Schistosomiasis Country Hookworm Schistosomiasis Angola 11.3 million 6.1 million Brazil 32.3 million 2-7 million Cote d Ivoire 10.0 million 6.6 million DR Congo 31.0 million 14.9 million Ethiopia 11.2 million 5.0 million Ghana 5.5 million 15.1 million Kenya 9.2 million 7.4 million Nigeria 37.8 million 28.8 million Sudan 8.1 million 5.0 million Tanzania 9.9 million 19.0 million Uganda 9.1 million 5.3 million Zimbabwe 8.1 million 5.2 million

24 Completed Genome Projects Buruli Ulcer Chagas Disease Afr. Trypanosomiasis Leishmaniasis Leprosy Leptospirosis Lymphatic Filariasis Schistosomiasis Trachoma membrane Sm-TSP-2 EC-1 1 G/A K G/A G/A W/F/Y G C C E/Q E/Q C F/Y E/Q G/A The Schistosome Tetraspanins P C C G C EC-2 Hotez PJ, Ferris M. The antipoverty vaccines. Vaccine 2006

25 Suppression of tsp-2 2 mrna expression results in impaired tegument turnover in vitro Tran et al 2010, PLoS Path

26 Multivalent Anthelminthic Antipoverty Vaccines: Hookworm and Schistosomiasis 26.6 million DALYs 65-83% of disease burden in Brazil Drugs + Vaccines in Integrated Framework Human Vaccine-Linked Chemotherapy Alternative Health Delivery Mechanisms School-based Health Systems Child Health Days Co-Distribution of Hookworm and Schistosomiasis

27 Opportunity for Vaccine Diplomacy There are no better grounds on which we can meet other nations and demonstrate our own concern for peace and the betterment of mankind than in a common battle against disease John Gardner Hotez PJ. SCIENCE 2010

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