Neglected tropical diseases occur almost exclusively in impoverished populations in low- and middle-income countries. Barely known in industrialized nations, they attract little public attention and research funding. One example is schistosomiasis. Our aim is to help take urgent action to prevent and control this neglected disease as well as more familiar infections such as malaria.
Strategy for preventing and treating infectious diseases
Working hand in hand with our external partners, we seek to improve the health of underserved populations in low- and middle-income countries through our science and technology innovation. Our strategy is to develop and provide medicines, improve diagnosis, counter disease transmission, increase disease control, expand access to healthcare, and strengthen local health systems.
Our priority areas are eliminating schistosomiasis, developing health solutions for malaria and infectious diseases, expanding access to healthcare by strengthening health systems, and promoting capacity building along the value chain.
Our fight against schistosomiasis
Schistosomiasis, a tropical worm disease also known as bilharzia, is one of the most prevalent parasitic infections in Africa, placing a significant burden on public health and the local economy. The disease affects almost 240 million people worldwide, with more than 90% of cases occurring in sub-Saharan Africa. School-aged children are particularly vulnerable to infection. An estimated 200,000 people die every year from the long-term effects of schistosomiasis, such as liver and kidney infections, bladder cancer and anemia.
Our ultimate aim in all our schistosomiasis-related work is to eliminate the disease. To help achieve this goal, we adopted an integrated schistosomiasis strategy in early 2019 that we are implementing in close collaboration with multiple partners worldwide. The new approach focuses on five areas:
- Supplying medicine: We donate up to 250 million tablets of praziquantel per year to WHO. Nearly 50 years after its invention, praziquantel still remains the standard of care for the effective treatment of schistosomiasis around the world.
- Researching new solutions: We are collaborating with research institutions and through public and private sector partnerships to develop a new formulation of praziquantel for children under the age of six and identify diagnostics and vector control approaches.
- Working with partners: Collaborating with partner organizations through the Global Schistosomiasis Alliance (GSA), we are accelerating the progress towards schistosomiasis control and elimination.
- Improving water, sanitation and hygiene: We support WASH projects to prevent transmission of the disease through the development of infrastructure.
- Education and behavior change: We invest in education and behavior change projects and participate in health education initiatives that raise awareness of the causes and dangers of schistosomiasis and teach people how to prevent it.
Our fight against malaria
According to World Health Organization (WHO) estimates, nearly half of the world’s population is at risk of contracting malaria. More than 200 million cases of malaria and over 400,000 related deaths are recorded every year, with 70% in children under five years of age. Around 90 different countries are affected by the disease, with approximately 90% of deaths occurring in Africa.
There is an urgent need for new products to overcome the problem of increasing drug resistance and to achieve our goal of complete elimination. Through our company´s Malaria program, we are helping to deliver integrated and sustainable health solutions entailing treatments, diagnostics and prevention methods to fight malaria in endemic countries.
Schistosomiasis: Over one billion tablets donated
As part of our longstanding partnership with WHO, we have renewed our commitment to make annual donations of praziquantel tablets for distribution in 47 endemic African countries to treat school-aged children. In 2019, we donated approximately 233 million tablets for distribution in 35 countries, 32 of which in Africa. Also, we maintained our commitment by ensuring that we have sufficient production capacity to manufacture up to 250 million tablets a year. The latest numbers from WHO show that in 2017, 72% of all school-aged children in need of treatment in sub-Saharan Africa were treated.
Schistosomiasis health education project
Through our joint health education project with the NALA Foundation, we are investing nearly € 300,000 over a three-year period. Since the end of 2017, we have reached 250,000 people in southwestern Ethiopia, almost half of whom are school-aged children. The education programs will help promote long-term behavioral change in the drive to eliminate schistosomiasis and other neglected tropical diseases.
In 2019, we expanded the project to two further districts in Ethiopia and reached around 188,000 people, almost 40% of whom were school-aged children. To deepen our understanding of the knowledge, attitudes and practices of primary school children with respect to safe water, sanitation and hygiene, we conducted a survey that revealed that 58% of the children taking part had never heard of parasites that can infect the intestines. This prompted us to create a series of educational training sessions.
Thanks to our financial support, our on-the-ground partners were able to conduct training sessions in schools and among local communities, with the majority of the schools setting up hand-washing stations. Clean water and latrines are now available in these stations throughout the school year. Teachers also reported major improvements in students’ personal hygiene and general levels of cleanliness in the participating schools.
Central platform in the battle against schistosomiasis
The Global Schistosomiasis Alliance (GSA) is a coordinated, multi-sectoral effort to combat the complex disease schistosomiasis. In 2019, the GSA recruited additional international stakeholders as new members and contributed to WHO’s consultations ahead of the new NTD Roadmap expected to be passed by the World Health Assembly in 2020. The GSA took part in various projects aimed at driving local efforts to combat schistosomiasis as well as organizing several conferences and key meetings. The alliance is also helping to promote and support an international action plan to progress schistosomiasis control and ultimately eliminate the disease. The GSA continues with its efforts to raise awareness through coordinated campaigns.
Partners in schistosomiasis research
Over time, we have developed a portfolio of R&D projects on schistosomiasis. These include a new pediatric formulation of praziquantel to treat children under the age of six, identifying new drugs to prevent and treat schistosomiasis, developing innovative and highly sensitive schistosomiasis diagnostic methods, and defining approaches for vector control.
Praziquantel is an effective and well-tolerated drug, but it does not work in all developmental stages of the parasite. We continue to collaborate on research activities with many partners in developed and low- to middle-income countries. This work aims to discover new, long-lasting compounds to treat juvenile forms of the parasite, thereby improving efficacy and preventing reinfections. In 2019, we obtained promising assets from Salvensis and the London School of Hygiene & Tropical Medicine to identify potential new candidates for preventing infection and curing patients affected by schistosomiasis.
The need for more sensitive diagnostics is crucial in the fight against schistosomiasis. Since 2017, we have been collaborating with the Australian Institute of Tropical Health and Medicine at James Cook University in Townsville (Queensland, Australia) and with the Baylor College of Medicine in Houston (Texas, USA) on researching new biomarkers in order to develop diagnostic tools for schistosomiasis. The program achieved the preliminary identity of new schistosomiasis biomarkers for novel diagnostics.
As of early 2019, we also initiated our collaboration with the Foundation of Innovative New Diagnostics (FIND) and the Bill and Melinda Gates Foundation to develop a sensitive rapid diagnostic test (RDT) to improve mapping and case detection for schistosomiasis.
Beyond these efforts, we continued to explore technologies that control transmission factors through basic research activities, for example the elimination of the infectivity of snails through gene editing, or through access-to-water programs in Senegal. In 2019, we implemented initiatives to address female genital schistosomiasis (FGS), a major challenge to women’s health in Africa, and its impact on HIV/AIDS. In particular, we began supporting a trial to optimize therapeutic treatment for women suffering from FGS in Madagascar and conducted advocacy initiatives through workshops and training sessions.
Consortium for the development of a pediatric praziquantel formulation
If left untreated at a preschool age, schistosomiasis can have long-term effects on children such as anemia, stunted growth and impaired learning. It can seriously affect their lives and potentially cause chronic diseases, including bladder cancer or genital schistosomiasis. We are working with the Pediatric Praziquantel Consortium, which includes both public and private sector representatives, to develop and provide access to a pediatric formulation of praziquantel to children under the age of six.
Following initial Phase I studies and a taste evaluation, we completed the Phase II study in Ivory Coast in 2018. It assessed the efficacy and safety of two different formulations of orodispersible tablets (ODT) in schistosomiasis-infected children under the age of six. The results indicated that both formulations are well tolerated and helped us to identify the optimal formulation and dose to pursue until we can register the drug.
In 2018, two new partners joined the Pediatric Praziquantel Consortium: the Kenya Medical Research Institute (KEMRI) and the Université Félix Houphouët-Boigny in Ivory Coast. Both play important roles in implementing the Phase III trial that started at the Homa Bay clinical center in Kenya in September 2019. This pivotal trial is designed to evaluate the efficacy and safety of the new child-friendly praziquantel ODT formulation in children three months to six years of age who are infected with schistosomes. The trial is being conducted in Kenya and Ivory Coast, and is co-funded by the consortium, the European & Developing Countries Clinical Trials Partnership (EDCTP) and the Global Health Innovative Technology (GHIT) Fund. The study represents the last step of the clinical development program, which, should it produce a positive outcome, will allow the clinical data package needed for registration to be completed. We expect the product to be available to the first endemic countries in Africa in 2022. Together with international key stakeholders, we are working on designing an innovative access path to ensure future affordability, availability and adoption of the new medicine.
Malaria: Enabling the treatment of children
As part of our company´s Malaria program, we are developing a new innovative drug (M5717) for the treatment of malaria in children. In 2019, we assessed the safety of the compound and gathered data to support clinical proof of principle by conducting a Phase I/Ib study in healthy volunteers in Australia. The program is progressing towards the next phase, where we will explore opportunities to develop the compound in combination with another anti-malarial compound to potentially serve as a single-dose treatment to cure or prevent malaria.
Developing new lead programs for antimalarials
In 2019, our strategic collaboration with the University of Cape Town in South Africa and the Medicines for Malaria Venture continued its screening activities with the aim of identifying new therapeutic solutions for malaria and building research capacity in and for Africa. Co-funded by the German Federal Ministry of Education and Research, this program continues to leverage our proprietary chemical library of nearly 100,000 compounds to identify new lead programs for the treatment of malaria. It targets liver-stage forms of the parasite and focuses on long-lasting compounds to improve post-treatment prophylaxis. Together with our partners, we have identified a promising chemical series to help declare potential lead candidates for drug discovery and development activities.
Through a collaboration with the Instituto de Biologia Experimental e Tecnologica (iBET) and the Instituto de Medicina Molecular (IMM) in Portugal, we made progress in developing a new cell model of liver-stage malaria infection. This new cell model could serve as a screening tool for novel anti-malaria drugs. The results have been published in peer-reviewed scientific journals.
Preventing and controlling malaria transmission
To help prevent the spread of malaria, we are working to improve access to insect repellent as a vector control method. Through internal and external collaborations, we are working towards demonstrating the efficacy of IR3535® against malaria in Africa. IR3535® is used in insect repellents for complementary prevention of vector-borne diseases such as dengue and Zika fever, Chikungunya and Lyme disease. The repellent is safe for all age groups, including children, pregnant women and nursing mothers.
We are partnering with the Infanta Malaria Prevention Foundation to support the Ghana Health Service’s National Malaria Control Program by exploring potential IR3535®-based solutions for malaria prevention in vulnerable communities. In 2019, we helped to broaden the scope of this initiative through an integrated, country-level approach, working with an established network of partners in Ghana. Through these efforts, we aim to improve health worker capacity to detect malaria cases through microscopy and continue our work to deploy IR3535® as a malaria preventive method for women and babies. Furthermore, we will increase our knowledge of the prevalence of asymptomatic patients suffering from malaria via an innovative pan-African network that maps the pathogenic parasite P.vivax.
Technologies to combat antimicrobial resistance
We have implemented new collaborative programs to assess the degree of resistance of identified bacterial pathogens. We have also focused our efforts on the development of new technological platforms to accelerate the assessment of infection types and test the validity of drugs. Since 2018, we have been partnering with Boston University to test, validate and optimize a new user-friendly technology to identify and quantify the active pharmaceutical ingredients of medicines sold in hospitals, health centers and pharmacies. This helps us in detecting fake medicines.