Schistosomiasis

WASHINGTON, DC, USA I July 15, 2024 I Sabin Vaccine Institute launched a Phase 2 clinical trial for its vaccine against Sudan ebolavirus, with healthy volunteers receiving the single-dose vaccine at Makerere University Walter Reed Project (MUWRP) in Uganda. There are currently no approved vaccines for this strain of ebolavirus, which saw an outbreak end just last year. Ebolavirus disease kills on average half the people infected, according to the World Health Organization. Sudan ebolavirus is a filovirus, in the same family as Marburg virus disease and Zaire ebolavirus, which killed 11,325 in one outbreak in West Africa from 2014-16. Ebolavirus disease spreads between people via direct contact with the blood or other bodily fluids of infected people and is highly virulent, causing hemorrhagic fever. Based on the same cAd3 platform as its Marburg vaccine candidate, Sabin’s single-dose investigational Sudan ebolavirus 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 that lasted up to 12 months. This is Sabin’s second Phase 2 clinical trial partnership with MUWRP, based in Uganda’s capital, Kampala. A Phase 2 trial for a Marburg vaccine is already underway, having recently completed enrollment . Initial results from the Marburg trial are expected later this year. Dr. Betty Mwesigwa, deputy executive director of MUWRP, is once again the principal investigator (PI) for the Sabin-sponsored trial for the Sudan ebolavirus vaccine. In the coming weeks, participants will also be enrolled at the Kenya Medical Research Institute in Siaya, Kenya, with Dr. Videlis Nduba serving as PI for that site. In all, 125 volunteers will participate in the trial across the two countries. “We are delighted to advance a vaccine candidate that can thwart a deadly and devastating disease, especially one that caused a fairly recent outbreak and for which no approved treatments exist,” says Amy Finan, Sabin’s Chief Executive Officer. “Sabin’s vaccine candidate is backed by strong safety and immunogenicity data, and we hope this trial will yield further evidence to move the vaccine closer to licensure.” The most recent outbreak of Sudan ebolavirus occurred in the fall of 2022 in Uganda, after six suspicious deaths in the Mubende district. That outbreak ultimately resulted in 55 deaths. Sabin’s vaccine was the first to arrive in Uganda during that outbreak after the World Health Organization included it as one of three vaccines for possible use in an outbreak trial. The outbreak ended on January 11 , prior to vaccine being deployed for use. “Makerere University Walter Reed Project is pleased to partner with the Sabin Vaccine Institute once again,” says Dr. Mwesigwa. “Uganda has the most experience with Sudan ebolavirus outbreaks so we understand the importance of testing and researching an effective Sudan ebolavirus vaccine that could be used in the event of an outbreak.” The Phase 2 clinical trial will evaluate safety and immunogenicity for the vaccine among a larger group of individuals than in previous trials. 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 Sudan ebolavirus vaccine in the U.S. The Sudan ebolavirus 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. To date, Sabin has received around $216 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. 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 cAd3 Platform In August 2019, Sabin announced exclusive agreements with GSK for Sabin to advance the development of the prophylactic candidate vaccines against the deadly Zaire ebolavirus, Sudan ebolavirus 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 cAd3 platform, were further developed by GSK, including the Phase 2 development for the Zaire ebolavirus 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 1 and 2 vaccine clinical trials including the first Ebola vaccine trial in Africa. SOURCE: Sabin Vaccine Institute

In addition to supplying praziquantel, Merck KGaA’s four-pronged efforts toward “#MakingSchistoHistory” comprise developing new treatments for the disease, implementing health education and water safety initiatives and forming partnerships to bolster all of that work. After nearly two decades spent working with the World Health Organization (WHO) to supply billions of praziquantel tablets to help treat schistosomiasis, Merck KGaA has recently tacked on an additional awareness-raising component to its efforts to eliminate the parasitic disease also known as bilharzia. That project has been dubbed the Bilharzia Storytelling Lab, and it wrapped up its third iteration late last month, the drugmaker announced Tuesday. The latest lab took place in Ethiopia, following previous installments in Kenya and Rwanda. For this year’s lab, local participants were invited to a five-day workshop in Addis Ababa, during which they learned more about schistosomiasis and received instruction on storytelling from local and international experts. From there, the participants worked with health-focused and other nonprofit organizations to develop storytelling-based solutions that could help raise awareness about the disease in their communities. A panel of judges was tasked with selecting the best solution, which Merck will help implement throughout Ethiopia. The winning solution this year—which was awarded a grand prize of 10,000 euros—uses multimedia participatory theater to improve awareness of schistosomiasis, specifically targeting young men working in sugar factories. “We have seen great pitches today and it wasn’t easy to pick only one. In the end, we decided on Team Buna because it is an innovative and locally applicable solution, which is also scalable to surrounding communities,” Johannes Waltz, head of the Merck Schistosomiasis Elimination Program, said in a statement after judging was complete. The previous two winners included a card game that could educate young children about the disease through play and a school notebook that included information about disease prevention for both children and their caregivers, according to Merck. Schistosomiasis infections occur when the skin is exposed to parasites in bodies of water. After infection, parasite eggs that aren’t passed out of the body may become trapped within tissues, resulting in immune reactions and organ damage, per a WHO fact sheet. The organization has placed schistosomiasis in its group of neglected tropical diseases, as it is especially prevalent in poor communities within tropical and subtropical regions that don’t have ready access to clean water. The WHO estimates that at least 90% of the approximately 250 million people requiring treatment for the disease live in Africa. Since 2007, Merck KGaA has been working with the WHO to distribute praziquantel tablets, the recommended treatment for the disease, with efforts focused mainly on sub-Saharan Africa. Merck said in this week’s announcement that it provides the organization with up to 250 million tablets per year, for a total donation to date of 2 billion tablets, which have been used to treat around 800 million people. In addition to supplying praziquantel, Merck’s four-pronged efforts toward “#MakingSchistoHistory” comprise developing new treatments for the disease, implementing health education and water safety initiatives and forming partnerships to bolster all of that work.

- 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.