29 May 2015

Pediatric Praziquantel Consortium Awarded USD4.9 Million Grant from Global Health Innovative Technology Fund

The second research grant from GHIT provides further support for the Phase II development of a pediatric formulation to combat schistosomiasis in pre-school children

The Netherlands, May 29, 2015 - The Pediatric Praziquantel Consortium, a Consortium dedicated to the development of a new pediatric formulation against schistosomiasis received almost USD4.9 million from the Japanese Global Health Innovative Technology (GHIT) Fund. The GHIT Fund reaffirms, with this second research grant, its confidence in the work being done by the consortium to offer a treatment solution for pre-school children and the future role that the Consortium will be playing for the elimination of schistosomiasis.

GHIT's mission is to facilitate international partnerships that bring Japanese innovation, investment, and leadership to the global fight against infectious diseases and poverty in the developing world. Dr. Slingsby, CEO & Executive Director of the GHIT Fund (Japan) says, "There is great need for a formulation of praziquantel created specifically for the pediatric population under six years old. Local healthcare centres and entire communities across Africa and South America (like in Brazil) are faced with a dilemma, where physicians are using an adult formulation for the needs of children. The Consortium is working to help fill the gap by developing and registering a formulation for pre-school-age children."

The existing 'gold standard' treatment for schistosomiasis is praziquantel (PZQ), which was developed by Merck KGaA, Darmstadt, Germany in the 1970s through a research collaboration. It is safe and effective, and is available in tablets, suitable for adults and school-age children. Proper treatment of preschool-aged children, including infants and toddlers suffering from schistosomiasis, is hampered due to the lack of a suitable pediatric dosage form that can be readily administered to children. This unmet medical need represents a significant gap toward the main goal of eliminating schistosomiasis. The Pediatric Praziquantel Consortium is working to develop orodispersible (fast disintegrating) tablets for children from three months old to six years old.

This research grant is intended to fund the costs for the conduct of a Phase II clinical study in Sub-Saharan Africa, and it will also cover additional costs such as those related to active pharmaceutical ingredient (API) process development activities.

"The Pediatric Praziquantel Consortium was founded on the belief that working together, public and private partners will make faster and more significant progress in developing a suitable treatment for schistosomiasis in children. This Consortium brings together high-caliber experts and some of the best-in-class capabilities in neglected diseases, paving the way for us to bring the new pediatric formulation where it is desperately needed," states Kathleen Ford, Chair of the Pediatric Praziquantel Consortium Board and Head of Global Clinical Operations at Merck Serono, the biopharmaceutical business of Merck KGaA, Darmstadt, Germany. "This additional GHIT grant will certainly contribute to the progress of the program and to the fight against schistosomiasis."

The first research grant from GHIT to the Consortium was awarded in 2014 and used to fund the Phase I drug development program.

About Schistosomiasis

Schistosomiasis, endemic in 78 developing countries, is a chronic inflammatory disease caused by parasitic worms. According to the World Health Organization, this disease affects at least 249 million people globally, of whom nearly half are children, and is commonly associated with poor sanitation and no access to drinking water. It is one of the most prevalent parasitic diseases in Africa, and one of the most important ones in terms of public health burden and economic impact. Left untreated, this poverty-related disease can lead to anemia, stunted growth, impaired learning ability and chronic inflammation of the organs, which can be fatal in the most serious cases. Today, there is no adequate treatment for children under the age of six, which are estimated to account for about 10% of the global population already infected.

Pediatric Praziquantel Consortium

The Pediatric Praziquantel Consortium was founded in July 2012. Its ultimate objective is to develop and register a new praziquantel pediatric formulation for the treatment of schistosomiasis in preschool-age children, including infants and toddlers. In this consortium of highly driven partners, Merck KGaA, Darmstadt, Germany, responsible for leading the program, also brings the necessary chemistry and manufacturing expertise, resources, and support related to praziquantel. In addition, it provides the preclinical, clinical and regulatory resources and experience necessary to efficiently and successfully execute the project. Astellas Pharma Inc. contributes by providing its innovative pharmaceutical technologies in the area of drug formulation development and clinical development in children. Swiss TPH brings extensive experience in helminths biological and pharmacological research, epidemiology and clinical research on drug effectiveness and efficiency in endemic regions. The governance is facilitated by TI Pharma, a non-profit organization and independent party with an extensive portfolio of international public-private partnerships in drug research and development, including in the area of neglected diseases. Farmanguinhos, the federal governmental pharmaceutical laboratory of the Fiocruz Foundation in Brazil, brings unique expertise to produce and distribute the new pediatric formulations product in endemic countries. Simcyp, a small UK research-based company, brings expertise in pharmacokinetic modelling for a better prediction for use in the pediatric clinical trials.

For more information contact Hiliana Fienieg: hiliana.fienieg@tipharma.com M:+31 6 526 73 783

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About Astellas Pharma Inc.

Astellas Pharma Inc., based in Tokyo, Japan, is a pharmaceutical company dedicated to improving the health of people around the world through the provision of innovative and reliable pharmaceutical products. Astellas Pharma Inc. has approximately 17,000 employees worldwide. The organization is committed to becoming a global category leader in Urology, Immunology (including Transplantation) and Infectious Diseases, Oncology, Neuroscience, and DM Complications and Kidney Diseases. Astellas Pharma Inc. is committed to increasing access to health in developing countries through its partnership initiatives. Astellas Pharma Inc.'s pharmaceutical research and technology labs have contributed to improving convenience for use and the functionality of treatments by developing novel formulations and bringing additional value based on its excellent pharmaceutical technologies (e.g. solubilization, controlled release, oral disintegration tablet and drug delivery technologies).

For more information, please visit www.astellas.com/en

About Merck KGaA, Darmstadt, Germany

Merck KGaA of Darmstadt, Germany, is a leading company for innovative, top-quality high-tech products in healthcare, life science and performance materials. The company has six businesses - Merck Serono, Consumer Health, Allergopharma, Biosimilars, Merck Millipore and Performance Materials - and generated sales of around € 11.3 billion in 2014. Around 39,000 employees work in 66 countries to improve the quality of life for patients, to further the success of customers, and to help meet global challenges. Merck KGaA, Darmstadt, Germany, is the world's oldest pharmaceutical and chemical company - since 1668, the company has stood for innovation, business success and responsible entrepreneurship. Holding an approximately 70% interest, the founding family remains the majority owner of the company to this day. Merck KGaA, Darmstadt, Germany, holds the global rights to the Merck name and brand. The only exceptions are Canada and the United States, where the company operates as EMD Serono, EMD Millipore and EMD Performance Materials.

For more information, please visit www.merckgroup.com

About Swiss TPH

Swiss TPH is an associated institute of the University of Basel. Its mandate is to contribute to the improvement of public health at international, national and local levels through excellence in research, services, teaching and training. The strategy to pursue this mandate is based on a broad interdisciplinary approach for a sustainable iterative process between the laboratory, field, classroom and bedside. Swiss TPH addresses the continuum between innovation (developing concepts, methods and products), validation (providing evidence for what works) and application (strengthening public health systems & policies). Over 700 staff members from 62 nations are organized in two research and three service departments.

For more information, please visit www.swisstph.ch

About Farmanguinhos

Farmanguinhos is a technical-scientific unit of the Oswaldo Cruz Foundation. Bearing in mind the mission of "Acting with environmental responsibility in promoting public health through the production of medicines, research, technological development and general diffusion of knowledge", this unit is the largest public pharmaceutical company under the Brazil's Ministry of Health and are produced medicines for the treatment of diabetes, hypertension, malaria, AIDS, tuberculosis, among others. Farmanguinhos also has an important role in meeting emergency demands of the Ministry of Health.

For more information, please visit http://www2.far.fiocruz.br/farmanguinhos/

About Simcyp

Simcyp Limited was founded in 2001 by Professor Geoff Tucker, Professor Amin Rostami-Hodjegan and Mr. John Evans as a spin-out company from the University of Sheffield, UK. Early in 2012 Simcyp was acquired by Certara, a leading provider of drug discovery and development software and scientific consulting services. Simcyp operates as a stand-alone business unit within the Certara group which also includes Pharsight and Tripos. Simcyp provides platforms for the modeling and simulation of pharmacokinetics and pharmacodynamics in virtual human populations and virtual laboratory animals (rat, dog and mouse). The technology allows pharmaceutical researchers to predict in vivo outcomes from routinely generated in vitro data, to fit Simcyp models to observed clinical data and to assess inter-individual variability through 'real-life' simulations. Simcyp also runs educational workshops, on-site education and provides strategic consultancy in drug development. For more information, visit www.simcyp.com

About Top Institute Pharma

Top Institute Pharma (TI Pharma), a non-profit organization, enables groundbreaking pharmaceutical research for the development of new medicines by establishing and managing international public-private partnerships. Within an open innovation model, scientists, the business world, patient organizations and others collaborate in frontrunner, multidisciplinary research aimed at improving the development of socially valuable medicines.

For more information, please visit www.tipharma.com