Case Study: Research and Development
Pediatric Praziquantel Consortium, Merck Serono
At Merck, we are committed to expanding sustainable access to high-quality health solutions to underserved populations in developing countries. We leverage our core competencies, expertise, knowledge, and experience across the health value chain in order to address barriers that prohibit access to health in developing countries. We aim to achieve this goal through a strategic focus on our four As of access: availability, affordability, awareness, and accessibility. The Pediatric Praziquantel Consortium is part of our efforts around availability; it is working on the research, development, and refinement of health solutions that address unmet needs and are tailored to local environments across the spectrum of prevention, diagnostics, treatment, care, and support.
Launched in 2012, the consortium is an international, nonprofit public-private partnership that combines the best science and most experienced public and private partners to develop, manufacture, register, and launch a pediatric formulation of praziquantel. The consortium seeks to develop new formulations of this medicine that are suitable to be administered to preschool age children, including infants and toddlers, exhibit improved palatability, and are able to resist the challenges presented by a tropical climate.
Endemic in 78 developing countries, schistosomiasis, also known as bilharzia, is a chronic inflammatory disease caused by parasitic worms. It is the second most prevalent parasitic disease after malaria, affecting nearly 240 million people worldwide, including 100 million children, with 90 percent of cases occurring in Sub-Saharan Africa.
The consortium has addressed many scientific, regulatory, and access challenges in the area of global health by working as a team and involving external experts at key stages of development. It actively explores opportunities to extend the partnership into endemic countries (the partnership with FioCruz/Farmanguinhos in Brazil is an example) because, beyond acquiring important expertise for the program, it will contribute to capacity building in those countries.
This partnership has substantially evolved during the past two years—more partners have become involved—and the role of each partner has expanded.
The consortium fully supports the World Health Organization (WHO) objective of eliminating schistosomiasis as a global health problem and is composed of the following partners:
- Merck Serono, responsible for leading the program and principal investigator of the project, brings the necessary chemistry and manufacturing expertise and support related to praziquantel; it also provides preclinical, clinical, and regulatory resources to efficiently and successfully execute the development program. The consortium is governed by a board chaired by a top management representative of Merck Serono.
- Astellas Pharma (Japan) contributes with its innovative pharmaceutical technology, which helps to improve drug compliance and functionality, reducing or eliminating the bitter taste of praziquantel.
- Swiss TPH contributes with its in-the-field expertise of clinical trials in endemic countries and its knowledge on antischistosomal drug discovery.
- TI Pharma (the Netherlands) has a proven track record managing public-private partnerships in drug research and development.
- SimCyp (U.K.), a new partner as of 2014, brings its pharmacokinetic modeling capabilities.
- Farmanguinhos (Brazil), a new partner as of 2014, brings expertise in pharmaceutical manufacturing and distribution in endemic countries.
Results to date?
Since its implementation in July 2012, the consortium has accomplished the following:
- Process development and manufacturing of a Drug Substance GMP (Good Manufacturing Practices ) batch for the L-praziquantel enantiomer has been completed.
- New orally disintegrating Praziquantel formulation candidates have been developed. The prototype formulations are only a quarter of the size of the current commercial praziquantel tablet, Cesol (600 milligrams). A smaller size allows the tablets to be taken with or without water, allowing treatment, in principle, of very young children, including infants beginning at age three months.
- A preclinical package to support the initiation of the phase I studies is completed. So far, no toxicity issues have been reported.
- Bidirectional technology transfer of the pediatric formulation process to GMP manufacturers is almost completed.
- With the input of world-renowned clinical experts, a clinical development plan has been drafted and is being discussed with regulators.
- Funding from the Bill & Melinda Gates Foundation and Global Health Innovative Technology (GHIT) Fund were secured to finance some important gaps identified in the preclinical and clinical programs.
- A taste study involving African children, in which the newly developed praziquantel candidates will be compared to commercial praziquantel, is planned for the first half of 2015.
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