Schistosomiasis

- Astellas contributed to the development of a pediatric formulation to treat schistosomiasis as a member of the Pediatric Praziquantel Consortium - TOKYO, Dec. 17, 2023 /PRNewswire/ -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") contributed to the development of a pediatric formulation to treat schistosomiasis as a member of the Pediatric Praziquantel Consortium (Consortium). The Consortium announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive scientific opinion for arpraziquantel to treat one of the neglected tropical diseases, schistosomiasis, in preschool-aged children (3 months to 6 years of age) on December 15. EMA assessed arpraziquantel under the EU-Medicines for all (EU-M4all) *1 procedure for high-priority medicines intended for use in countries outside the European Union. For more details on arpraziquantel, please refer to the Consortium's press release. Continue Reading Astellas provided its innovative formulation technology to co-develop arpraziquantel as a member of the Consortium. The new pediatric formulation of arpraziquantel is palatable for very young children by reducing the bitter taste associated with the medication and administered via a 150 mg dispersible tablet that is dissolved in water. The prototype of the pediatric formulation has been developed by Astellas and further optimized by Merck (Germany) for global production using simple manufacturing processes yielding tablets that can remain shelf stable, even in hot and humid tropical climates. Farmanguinhos (Brazil) brings expertise in production and distribution and will be the manufacturing site for the future introduction of the new pediatric medication in endemic countries. The partnership with Universal Corporation Ltd. (Kenya) is also supporting the planned future large-scale local production to serve African countries. In parallel with this regulatory work, the Consortium's implementation research program (ADOPT)*2 is ongoing and preparing for the introduction of arpraziquantel in the first endemic countries in Africa. To ensure equitable and sustainable access, it is essential that new procurement and funding mechanisms are collaboratively explored and established. The intent is to make the product available at an at-cost basis in sub-Saharan African countries. Astellas has set a Strategic Goal to "deepen our engagement in sustainability" in the Astellas Corporate Strategic Plan 2021. To address the key sustainability issue of Access to Health (ATH), Astellas uses a comprehensive approach including, (1) Astellas' core business (Rx, Rx+), (2) Enhancing availability of Astellas products (Access to Medicines), and (3) Collaboration and support for the activities implemented by external partners. Initiatives in the Consortium apply to (3), and the positive CHMP scientific opinion by EMA of arpraziquantel represents a significant milestone in alignment with Astellas' Access to Health focus. Naoki Okamura, President and CEO, Astellas "We are so pleased that arpraziquantel received a positive scientific opinion by EMA, and also proud that Astellas could contribute to its development by providing our innovative formulation technologies. Access to Health is essential to the health of people around the world, and we recognize it as a material issue for Astellas. Through our VISION to be "On the forefront of healthcare change to turn innovative science into VALUE for patients", we are proactively taking a comprehensive approach to solving this issue." For more information on Astellas' contribution to the development of arpraziquantel, please see (). About schistosomiasis Schistosomiasis (also known as bilharzia) is one of the most prevalent parasitic diseases worldwide and a very important one in terms of public health burden and economic impact. It is a poverty-related disease that is widespread in tropical and subtropical regions where large sections of the population have no access to clean water. Flatworms transmit the disease and people become infected with the parasite through contact with freshwater, for example, while working, swimming, fishing, or washing their clothes. The minuscule larvae penetrate human skin, enter the blood vessels, and attack internal organs. The infection rate is particularly high among children. Schistosomiasis is a chronic condition and is classified by the World Health Organization (WHO) as one of 20 neglected tropical diseases (NTDs). About Arpraziquantel The current standard of care treatment for schistosomiasis is praziquantel. Praziquantel is safe, effective, and suitable for school-aged children and adults. Extending the range of options for the treatment of schistosomiasis, arpraziquantel is tailored for preschool-aged children against Schistosoma mansoni and Schistosoma haematobium. Tested in clinical development, under the responsibility of Merck, arpraziquantel contains the pharmacologically active enantiomer of praziquantel. In developing arpraziquantel, the Pediatric Praziquantel Consortium established a pediatric drug development program, divided into four major steps: preclinical development, clinical development, registration, and access. All details can be found on the Consortium website. About the Pediatric Praziquantel Consortium The Pediatric Praziquantel Consortium is an international public-private partnership that aims to reduce the global disease burden of schistosomiasis and improve child health by addressing the medical needs of infected preschool-aged children. Its mission is to develop, register, and provide access to a suitable pediatric drug for treating schistosomiasis in children 3 months to 6 years of age. For more information, and to see an overview of all Consortium partners, visit the Consortium website. Consortium Partners Merck (Germany) Astellas Pharma Inc. (Japan) The Swiss Tropical and Public Health Institute (Switzerland) Lygature (The Netherlands) Farmanguinhos (Brazil) Unlimit Health (United Kingdom) Kenya Medical Research Institute (Kenya) Université Félix Houphouët-Boigny (Côte d'Ivoire) Klinikum rechts der Isar der Technischen Universität München (Germany) Ministry of Health Côte d'Ivoire (Côte d'Ivoire) African Institute for Health and Development (Kenya) Other collaborators that contribute to the mission of the Pediatric Praziquantel Consortium: Makerere University School of Public Health (Uganda) Ministry of Health Kenya, Division of Vector Borne and NTDs (Kenya) Ministry of Health Uganda, Vector Borne and NTDs Control Division (Uganda) About Astellas Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into VALUE for patients. For more information, please visit our website at . Cautionary Notes In this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management's current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas' intellectual property rights by third parties. Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice. SOURCE Astellas Pharma Inc.

EMA assessed arpraziquantel under the EU-M4all procedure for high-priority medicines intended for use in countries outside the European Union Merck KGaA, Darmstadt, Germany, is committed to contribute to the elimination of schistosomiasis as a public health problem by 2030 Merck KGaA, Darmstadt, Germany, a leading science and technology company, and the Pediatric Praziquantel Consortium today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive scientific opinion for arpraziquantel for the treatment of schistosomiasis in children aged 3 months to 6 years. The application was submitted by Merck KGaA, Darmstadt, Germany, on behalf of the Consortium, under the EU-M4all procedure for high-priority medicines for human use intended for countries outside the European Union. “With this positive response by EMA, a critical milestone was achieved to broaden the range of options for the treatment of schistosomiasis so as to address the needs of millions of preschool-aged children”, said Peter Guenter, Member of the Executive Board and CEO of Healthcare at Merck KGaA, Darmstadt, Germany. “I thank all partners of the Pediatric Praziquantel Consortium for this achievement and for the continuous collaboration. Together, we strive to contribute to the elimination of schistosomiasis as a public health problem by 2030, as part of our commitment to drive health equity through our innovations and products.” Arpraziquantel is derived from praziquantel, the standard of care treatment of schistosomiasis developed in the 1970s. Extending the range of options for the treatment of schistosomiasis, arpraziquantel is tailored for use in preschool-aged children. The tablet is administered by dissolving it in water and has an improved taste to make it more palatable for very young children. In addition, the 150mg tablet has been specifically designed to withstand the hot and humid conditions presented by a tropical climate. The positive CHMP scientific opinion by EMA is the basis for a potential inclusion of arpraziquantel in the World Health Organization’s list of prequalified and essential medicinal products. Together with the positive scientific opinion, the planned prequalification will support the regulatory pathway in African countries. In Brazil, regulatory submission is under preparation by Consortium partner, Farmanguinhos. As the federal governmental pharmaceutical laboratory of the Fiocruz Foundation in Brazil, Farmanguinhos brings expertise in production and distribution, and will be the manufacturing site for the future introduction of the new pediatric medication in endemic countries. For planned future large-scale local production to serve African countries, Merck KGaA, Darmstadt, Germany, has also partnered with Universal Corporation Ltd., Nairobi, Kenya . In parallel, within the Consortium, the implementation research program (ADOPT) is currently ongoing, preparing for the introduction of arpraziquantel in the first endemic countries in Africa. To support equitable and sustainable access, it is essential that new procurement and funding mechanisms are collaboratively explored and established. The intent is to make the product available at an at-cost basis in sub-Saharan African countries. The collaborative program on arpraziquantel is part of the integrated approach of Merck KGaA, Darmstadt, Germany, to contribute to the elimination of schistosomiasis as a public health problem. Indeed, through its Schistosomiasis Elimination Program, Merck KGaA, Darmstadt, Germany, addresses the key requirements of the WHO’s 2021-2030 Roadmap for Neglected Tropical Diseases and contributes to the United Nations’ Sustainable Development Goals, especially Good Health and Well-Being (Goal 3) and Partnerships (Goal 17). The program also aligns with the Sustainability Strategy of Merck KGaA, Darmstadt, Germany, and the company’s commitment to expand sustainable and equitable access to medicines by bringing its existing products and innovative portfolio to patients in low- and middle-income countries recognizing the needs and the specificity of the health systems in each of the country. About EU-M4all Through the EU-M4all procedure , the European Medicines Agency (EMA), in cooperation with the World Health Organization (WHO), can provide scientific opinions on high-priority human medicines, including vaccines, that are intended for markets outside of the European Union (EU). The procedure was previously known as the Article 58 procedure, as the legal basis is Article 58 of Regulation (EC) No 726/2004. More information can be found here . About Arpraziquantel The current standard of care treatment for schistosomiasis is praziquantel, listed on WHO’s list of essential medicines , and suitable for school-aged children and adults. Extending the range of options for the treatment of schistosomiasis, arpraziquantel is tailored for preschool-aged children against Schistosoma mansoni and Schistosoma haematobium . Tested in clinical development under the responsibility of Merck KGaA, Darmstadt, Germany , arpraziquantel contains the pharmacologically active enantiomer of praziquantel. It is a 150mg dispersible tablet. The prototype of its pediatric formulation was developed by Astellas in Japan and further optimized by Merck KGaA, Darmstadt, Germany, in Germany. The manufacturing process served to produce clinical trial supplies from Merck KGaA, Darmstadt, Germany, and Farmanguinhos in Brazil. Future manufacturing is planned to be done by Farmanguinhos and Universal Corporation Ltd., in Kenya, which is preparing for extensive local production capacities in and for Africa. About Schistosomiasis Schistosomiasis (also known as bilharzia) is one of the most prevalent parasitic diseases worldwide and a very important one in terms of public health burden and economic impact. It is a poverty-related disease that is widespread in tropical and subtropical regions where large sections of the population have no access to clean water. Flatworms transmit the disease and people become infected with the parasite through contact with freshwater, for example, while working, swimming, fishing, or washing their clothes. The minuscule larvae penetrate human skin, enter the blood vessels and attack internal organs. The infection rate is particularly high among children. Schistosomiasis is a chronic condition and is classified by the World Health Organization (WHO) as one of 20 neglected tropical diseases (NTDs). About the Schistosomiasis Elimination Program of Merck KGaA, Darmstadt, Germany, Merck KGaA, Darmstadt, Germany, initiated its Schistosomiasis Elimination Program in cooperation with WHO back in 2007. Since then, more than 1.9 billion tablets have been donated, enabling the treatment of over 760 million people, mainly school-aged children in 47 endemic countries. Merck KGaA, Darmstadt, Germany, has committed itself to maintaining its efforts in the fight against the tropical disease until schistosomiasis is eliminated as a public health burden. To this end, each year Merck KGaA, Darmstadt, Germany, provides up to 250 million tablets to WHO. In addition, Merck KGaA, Darmstadt, Germany, has adopted an integrated schistosomiasis strategy that is implemented in close collaboration with partners worldwide and focuses on: treatment, research & development, WASH (Water, Sanitation & Hygiene), advocacy & partnerships. More information about the Schistosomiasis Elimination Program of Merck KGaA, Darmstadt, Germany, is available at www.makingschistory.com . About the Pediatric Praziquantel Consortium The Pediatric Praziquantel Consortium is an international not-for-profit partnership that aims to help improving child’s health by addressing the medical needs of preschool-age children with schistosomiasis for potentially reducing the global burden caused by this disease. Its mission is to develop, register and provide access to a suitable pediatric drug for treating schistosomiasis in children three-months to six years of age. For more information, and to see an overview of all Consortium partners, visit the Consortium website: www.pediatricpraziquantelconsortium.org The Consortium is financially supported by Merck KGaA, Darmstadt, Germany; in-kind contributions from the Consortium’s partners; and grants from the Bill and Melinda Gates Foundation (2012), the Global Health Innovative Technology Fund (GHIT) (2014, 2015, 2016, 2019 & 2020), and the European & Developing Countries Clinical Trials Partnership (EDCTP), under its second program supported by the European Union (2018 & 2021). All Merck KGaA, Darmstadt, Germany, press releases are distributed by e-mail at the same time they become available on the EMD Group website. In case you are a resident of the USA or Canada, please go to www.emdgroup.com/subscribe to register for your online, change your selection or discontinue this service. About Merck KGaA, Darmstadt, Germany Merck KGaA, Darmstadt, Germany, a leading science and technology company, operates across life science, healthcare and electronics. More than 64,000 employees work to make a positive difference to millions of people’s lives every day by creating more joyful and sustainable ways to live. From providing products and services that accelerate drug development and manufacturing as well as discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2022, Merck KGaA, Darmstadt, Germany, generated sales of € 22.2 billion in 66 countries. The company holds the global rights to the name and trademark “Merck” internationally. The only exceptions are the United States and Canada, where the business sectors of Merck KGaA, Darmstadt, Germany, operate as MilliporeSigma in life science, EMD Serono in healthcare and EMD Electronics in electronics. Since its founding in 1668, scientific exploration and responsible entrepreneurship have been key to the company’s technological and scientific advances. To this day, the founding family remains the majority owner of the publicly listed company.

WASHINGTON, Oct. 19, 2023 (GLOBE NEWSWIRE) -- The Sabin Vaccine Institute has launched a Phase 2 clinical trial for its vaccine candidate against the lethal Marburg virus. Healthy volunteers received the single-dose vaccine at Makerere University Walter Reed Project (MUWRP) in Kampala, Uganda today. There are currently no vaccines or antiviral treatments approved to treat Marburg virus disease. Marburg is a filovirus, in the same family as the virus that causes Ebola. Like Ebola, Marburg virus disease spreads between people via direct contact with the blood or other bodily fluids of infected people, is highly virulent, and causes hemorrhagic fever. The disease has a fatality rate of up to 88%. Based on the ChAd3 platform, Sabin’s single-dose investigational Marburg vaccine was found to be promising in Phase 1 clinical and non-clinical studies, with results showing it to be safe, while eliciting rapid and robust immune responses. Dr. Betty Mwesigwa, deputy executive director of MUWRP, is the principal investigator for the Kampala portion of the Sabin-sponsored trial. Participants will also be enrolled a few weeks later at a second site at the Kenya Medical Research Institute in Siaya, Kenya, with Dr. Videlis Nduba as principal investigator. In all, 125 volunteers will participate in the trial. “We have an extraordinary opportunity here to improve our preparedness to save lives and protect people from a deadly and unforgiving disease that typically strikes under-resourced countries first and most,” says Amy Finan, Sabin’s Chief Executive Officer. “Sabin’s Phase 2 clinical trial builds on a solid safety and immunogenicity foundation and we are hoping it will generate the information needed to move the vaccine toward licensure.” The number of Marburg outbreaks in Africa has climbed steadily in recent years. Two outbreaks of Marburg virus disease have occurred already this year: Equatorial Guinea reported its first ever documented Marburg outbreak, which killed 12 people, followed by Tanzania, where six people succumbed to the virus. Communities in Uganda and Kenya are familiar with Marburg virus disease, having been ravaged by outbreaks over multiple years in the last few decades. “Makerere University Walter Reed Project (MUWRP) is delighted to partner with the Sabin Vaccine Institute to launch the clinical testing for a preventive Marburg vaccine,” says Dr. Mwesigwa. “Most Marburg virus disease outbreaks have originated in Africa. Uganda alone has registered 4 outbreaks of the disease. We urgently need a vaccine against Marburg because of its potential to cause epidemics with significant death rates. It is imperative for us to test candidate vaccines in Uganda, a country prone to these outbreaks. This work will contribute new knowledge to inform the scientific discovery for an effective vaccine against the deadly Marburg virus.” The Phase 2 clinical trial for Sabin’s Marburg vaccine will continue to evaluate safety and immunogenicity for the vaccine, this time among a larger group of individuals. This is a randomized, placebo-controlled, double-blind study, meaning that neither the participants nor the researchers will know whether trial participants receive a vaccine dose or a placebo dose until after the trial is over, an approach used to help reduce experimental bias. Participants in the clinical trial will be monitored for a full year and will include both younger (18-50 years) and older age groups (51-70 years). Interim results are expected next year. In addition to the current trial in Uganda and Kenya, Sabin plans to conduct a similar Phase 2 clinical trial for Marburg in the U.S. The Marburg vaccine trials are supported by the Biomedical Advanced Research and Development Authority (BARDA), part of the Administration for Strategic Preparedness and Response within the U.S. Department of Health and Human Services, under multi-year contracts between the organizations, including most recently a $36.4 million award for vaccine development and production. Similarly, BARDA has invested in Sabin for advancing ChAd3 Sudan ebolavirus vaccine candidate, including awarding $28 million this August for Phase 2 clinical trials in the U.S. To date, Sabin has received around $215 million in contract awards from BARDA for furthering vaccine research and development against Sudan ebolavirus and Marburg virus diseases. BARDA and Sabin began working together in September 2019 to develop the two monovalent vaccine candidates. Sabin’s Sudan ebolavirus vaccine candidate was the first to arrive in Uganda last year during the disease outbreak that left 55 people dead. Sabin has also initiated plans for a Phase 2 Sudan ebolavirus vaccine clinical trial in Uganda and Kenya. This project has been supported in whole or in part with federal funds from the Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority (BARDA), under contract numbers 75A50119C00055 and 75A50123C00010. About the Sabin Vaccine Institute The Sabin Vaccine Institute is a leading advocate for expanding vaccine access and uptake globally, advancing vaccine research and development, and amplifying vaccine knowledge and innovation. Unlocking the potential of vaccines through partnership, Sabin has built a robust ecosystem of funders, innovators, implementers, practitioners, policy makers and public stakeholders to advance its vision of a future free from preventable diseases. As a non-profit with three decades of experience, Sabin is committed to finding solutions that last and extending the full benefits of vaccines to all people, regardless of who they are or where they live. At Sabin, we believe in the power of vaccines to change the world. For more information, visit www.sabin.org and follow us on X, @SabinVaccine.About Sabin’s Vaccine R&D Using the ChAd3 Platform. About Sabin’s Vaccine R&D Using the ChAd3 Platform In August 2019, Sabin announced exclusive agreements with GSK for Sabin to advance the development of the prophylactic candidate vaccines against the deadly Ebola Zaire, Ebola Sudan and Marburg virus. The three candidate vaccines were initially developed collaboratively by the U.S. National Institutes of Health and Okairos, which was acquired by GSK in 2013. The candidate vaccines, based on GSK’s proprietary ChAd3 platform, were further developed by GSK, including the Phase II development for the Ebola Zaire vaccine. Under the agreements between GSK and Sabin, Sabin exclusively licensed the technology for all three candidate vaccines and acquired certain patent rights specific to these vaccines. About the Makerere University Walter Reed Project MUWRP is a non-profit biomedical research organization with a mission to mitigate disease threats through quality research, health care and disease surveillance. The project’s scope includes among others; clinical research in infectious and non-infectious diseases such as HIV, Ebola, Marburg, COVID-19, Influenza and Influenza-like illnesses, and neglected tropical diseases such as Schistosomiasis, among others. A major part of the clinical research are clinical trials, where the MUWRP has conducted more than 12 phase I and II vaccine clinical trials including the first Ebola vaccine trial in Africa. Media Contact: Monika Guttman Media Relations SpecialistSabin Vaccine Institute +1 (202) 662-1841 press@sabin.org A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/0ecbc982-493a-4ecf-9393-e203708aa3ea