SCHISTOSOMIASIS PEDIATRIC PRAZIQUANTEL CONSORTIUM Treang preschool children www.pediatricpraziquantelconsorum.org THE IMPACT OF SCHISTOSOMIASIS • More than 700 million people at risk • At least 258 million people require treatment • Nearly half are school- age children • 120 million people with morbidity • Many chronically disabled children • High infecon rates in young children • No suitable treatment for preschool-age children Merck leads the program and provides experse and support relang to PZQ, including internal resources from different areas needed for clinical development – drug product manufacturing; preclinical; clin- ical and regulatory. Also responsible for the development and manu- facturing of the L-PZQ Acve Pharmaceucal Ingredient (API). Astellas Pharma Inc. (Japan) has developed the new pediatric PZQ formulaons, and provides expert advice on clinical development in children, and pharmacokinec modeling. Lygature, a Dutch not-for-profit foundaon, acts as the independent coordinator of the Consorum, providing governance in terms of progress, finance and collaboraon. Since 2006, Lygature has support- ed close to a hundred public-private partnerships in the field of life sciences & health, including poverty-related diseases. Swiss Tropical & Public Health Instute is a not-for-profit Instute in- ternaonally renowned for its research, services, teaching and train- ing in global health. It contributes with extensive experience in hel- minths biological and pharmacological research; epidemiology; and clinical research in endemic regions. SimCYP, a UK-based research company, provides pharmacokinecs modeling capabilies and experse. The Schistosomiasis Control Iniave at Imperial College London, works to provide treatments against schistosomiasis and three soil- transmied helminths to the rural poor in Sub-Saharan Africa and Yemen. SCI will facilitate preparaon and implementaon of the Access and Delivery plan. Farmanguinhos, the federal governmental pharmaceucal laboratory of the Fiocruz Foundaon in Brazil, brings unique experse to address- ing the producon and distribuon of new pediatric formulaons in endemic countries.
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SCHISTOSOMIASISPEDIATRIC PRAZIQUANTEL CONSORTIUM
Treati ng preschool children
www.pediatricpraziquantelconsorti um.org
THE IMPACT OF SCHISTOSOMIASIS
• More than 700 million people at risk
• At least 258 million people require treatment
• Nearly half are school-age children
• 120 million people with morbidity
• Many chronically disabled children
• High infecti on rates in young children
• No suitable treatment for preschool-age children
Merck leads the program and provides experti se and support relati ng to PZQ, including internal resources from diff erent areas needed for clinical development – drug product manufacturing; preclinical; clin-ical and regulatory. Also responsible for the development and manu-facturing of the L-PZQ Acti ve Pharmaceuti cal Ingredient (API).
Astellas Pharma Inc. (Japan) has developed the new pediatric PZQ formulati ons, and provides expert advice on clinical development in children, and pharmacokineti c modeling.
Lygature, a Dutch not-for-profi t foundati on, acts as the independent coordinator of the Consorti um, providing governance in terms of progress, fi nance and collaborati on. Since 2006, Lygature has support-ed close to a hundred public-private partnerships in the fi eld of life sciences & health, including poverty-related diseases.
Swiss Tropical & Public Health Insti tute is a not-for-profi t Insti tute in-ternati onally renowned for its research, services, teaching and train-ing in global health. It contributes with extensive experience in hel-minths biological and pharmacological research; epidemiology; and clinical research in endemic regions.
SimCYP, a UK-based research company, provides pharmacokineti cs modeling capabiliti es and experti se.
The Schistosomiasis Control Initi ati ve at Imperial College London,works to provide treatments against schistosomiasis and three soil- transmitt ed helminths to the rural poor in Sub-Saharan Africa and Yemen. SCI will facilitate preparati on and implementati on of the Access and Delivery plan.
Farmanguinhos, the federal governmental pharmaceuti cal laboratory of the Fiocruz Foundati on in Brazil, brings unique experti se to address-ing the producti on and distributi on of new pediatric formulati ons in endemic countries.
PEDIATRIC PRAZIQUANTEL CONSORTIUMTreati ng preschool children
Schistosomiasis is one of the most prevalent parasiti c diseases in Africa, and a very important one in terms of public health burden and economic impact. Left untreated, this poverty-related disease can lead to anemia, stunted growth and impaired learning ability, and chronic infl ammati on of the organs, which can be fatal in the most serious cases.
As eff orts focus on morbidity control and eliminati on, there is a pressing need to treat preschool children (under 6 years old). No suitable drug formulati on is made available for this high-risk group, which accounts for about 10% of the 258 million people already infected.
THE GOLD STANDARD
The existi ng ‘gold standard’ treatment for schistosomiasis is praziquantel (PZQ), which was developed in the 1970s. This oral anthelminti c is available as generic drug and currently donated through the Merck Praziquantel Donati on Program, via the World Health Organizati on (WHO), to fi ght schistosomiasis in Africa. It is safe and eff ecti ve, and a tablet formulati on is available for adults and school-age children but not for those below 6 years old.
The Pediatric Praziquantel Consorti um is developing a PZQ formulati on that is more suitable for younger children, including infants and toddlers. The formulati on should be smaller, orodispersible, and have improved taste properti es compared to the approved PZQ Cesol® 600 mg tablet.
THE NEW PZQ FORMULATION CANDIDATES
The existi ng PZQ formulati on is a racemic mixture of levopraziquantel (L-PZQ) and dextropraziquantel (D-PZQ). Only one of these components is pharmacologically acti ve: the L-PZQ enanti omer. The other component, D-PZQ has been shown to be inacti ve and signifi cantly contributes to the taste that makes treati ng young children diffi cult.
Two novel PZQ formulati on candidates have been developed by the Consorti um: a racemic mixture and an enanti opure ver-sion, both with improved taste properti es.
The new formulati on candidates are a quarter of the size of the current approved PZQ Cesol® 600 mg tablet. Because they are orodispersible, they can be dispersed in water and administered to younger children, infants and toddlers. Both formulati on candidates are in clinical testi ng. This allows the Consorti um to select the fi nal formulati on, and to acquire the clinical data required for its registrati on in endemic countries where the medicati on is urgently needed.
Scanning Electron Microscope image of a pair of Schistosoma blood worms. The adult female worm resides within the adult male worm’s gynaecophoric canal.
This Consorti um is supported by grants from the Bill and Melinda Gates Foundati on awarded in 2013, and the Global Health Innovati ve Technology (GHIT) Fund, awarded in 2014 and 2015.
THE PEDIATRIC PRAZIQUANTEL CONSORTIUM SET-UP
The new formulati on candidate (right) is a quarter of the size of the approved praziquan-tel Cesol® 600 mg tablet (middle). The 10 yen coin (left ) has a diameter of 2.35 cm or 0.93 inch.
The Consorti um
The Pediatric Praziquantel Consortium was founded in July 2012 as the first international, non-profit, public-private partnership in schis-tosomiasis, supported by world leading experts in tropical parasitic infectious diseases.
The Consorti um aims to develop, register and provide access to a pediatric prazi-quantel formulati on for treati ng schisto-somiasis in preschool-age children, with proven effi cacy and safety and acceptable
taste properti es. The formulati on should also withstand the challenges presented by a tropical climate. The program is currently in the clinical development phase.
The Pediatric Praziquantel Consorti um is the only partnership targeti ng the preschool age range.
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