The most effective way to achieve progress and develop new approaches to fighting neglected diseases, is to work through robust public-private partnerships, innovative alliances and interdisciplinary approaches to health and development.
In January 2014, the biopharmaceuticals business launched the Global Health Innovation Platform to address unmet medical needs for neglected diseases in children from developing countries. The Global Health Innovation Platform has the goal of developing innovative, affordable, and integrated health solutions leveraging from the Group's cross-business competencies. This R&D platform is based on public-private partnerships (PPP) and collaborations with leading Global Health institutions and organizations in both developed and developing countries. The R&D activities for the Global Health Innovation Platform focus on schistosomiasis and related helminth diseases, as well as malaria. The Global Health Innovation Platform operating model functions through a series of partnerships and collaborations. Our partners include (and its extended network across the globe), the Swiss Tropical and Public Health Institute, the African Institute of Biomedical Science & Technology () in Zimbabwe, the Liverpool School of Tropical Medicine, the London Center for NTD Research, and Saint George’s University in the United Kingdom. We have furthermore signed a Memorandum of Understanding with both the Kenya Medical Research Institute (KEMRI) as well as the University of Makerere in Uganda. We are also a leading partner of the .
Through the Global Health Innovation Platform, the biopharmaceuticals business promotes and sponsors educational programs. Some examples of this include grants for post-doctoral fellowships at the AiBST in Zimbabwe and grants for healthcare worker disease awareness at the University of Namibia. Through these efforts, we are striving to help build capacities in endemic countries. Additionally, Biopharmaceuticals participates in international fellowship programs that host post-docs from developing countries. The post-docs are trained on clinical operations practices and clinical trial execution across all Phases (I-IV). More specifically, the fellowship places successful candidates with leading product development organizations, including pharmaceutical companies and product development partnerships, for a period of up to 24 months. On returning to their academic institutes, the fellows are expected to become an important resource for institutional capacity development and to undertake as well as manage clinical research in accordance with international regulatory requirements and standards.
Through this Innovation Platform, we are currently focusing on two priority issues in its research and development (R&D) activities: the pediatric praziquantel formulation for the treatment of pre-school age children suffering from schistosomiasis, and an anti-malaria drug. Initiatives are ongoing to assess new opportunities for additional projects to build up our portfolio in both areas. In addition, interdisciplinary approaches in health and development are being fostered through internal R&D cross-business collaboration, such as the diagnostics being co-developed by Biopharmaceuticals and Life Science. They are currently exploring the development of a malaria diagnostic kit from the existing Muse, a diagnostic point of care (IVC) used for the detection of HIV/AIDS. This initiative aims to allow co-diagnosis of HIV and malaria in countries where the co-infection rate is high.
Praziquantel pediatric formulation
Since July 2012, the Pediatric Praziquantel (PZQ) Consortium, an international, non-profit public-private partnership, has been operating with the aim to develop, register, manufacture and launch a pediatric formulation of praziquantel, the gold standard treatment for schistosomiasis. To date, the oral praziquantel tablets have only been available for adults and children over the age of 6. However, the high-risk age group of pre-school age children (from 3 months to 6 years old) accounts for about 10% of the estimated 249 million people already infected with schistosomiasis worldwide. The pediatric formulation will bridge the current treatment gap. Initiated and led by Merck KGaA, Darmstadt, Germany, consortium partners include: Astellas Pharma GmbH (Japan), the Dutch pharmaceutical research enabler TI Pharma, the Swiss Tropical and Public Health Institute, Farmanguinhos (Brazil) and Simcyp (UK).
Significant progress has been achieved within the Pediatric PZQ formulation program: upon successful completion of the pre-clinical phase, a clinical development strategy was defined, and the first Phase I clinical trial in healthy volunteers was completed in South Africa. Work is ongoing to implement the second Phase I trial in South Africa as well as a taste study in Tanzania, and to plan for a clinical Phase II/III trial in African countries. In 2014, the company's Bioethics Advisory Panel (MBAP) discussed how to specifically guarantee that the children's parents and guardians be informed of the risks of trial participation in an ethically responsible manner and give their consent. In particular, the MBAP expert discussion provided guidance to shape the clinical development plan with regard to aspects such as ancillary care, and drew attention to key aspects such as reimbursement.
Malaria remains a long-standing public health problem and has a major impact on vulnerable populations in over 100 countries: more than 3 billion people are at risk of infection and more than 200 million cases are recorded per year. An estimated 627,000 deaths are caused by malaria every year, primarily in African children under 5 years of age. Thus, despite an existing product portfolio and pipeline, there is an urgent need for new products to overcome the problem of increasing resistance and to achieve the overall goal of eradicating the disease.
In 2014, the biopharmaceuticals business and Medicine for Malaria Venture (MMV), an internationally recognized, Swiss-based not-for-profit organization, renewed the contract of their partnership on the anti-malarial long-acting compound program, which was originally signed in April 2013. The partnership allows them to reach out to a large key expert network including the in Spain, Monash University in Australia, and the Imperial College of London. Merck KGaA, Darmstadt, Germany and the MMV aim to develop urgently needed anti-malarial compounds through their lead optimization program, which comprises chemical series that have shown significant anti-malarial potential. The main component of this collaboration between the company and MMV is a new chemical series that entered the exploratory development phase in 2014. Under the biopharmaceuticals business’ leadership, this alliance is aiming to have at least one pre-candidate drug move into the non-clinical phase by the end of 2015.
Over the last two years, we have been participating in a series of conferences focused on schistosomiasis and malaria. These conferences have fostered the dialogue and interactions with key stakeholders and scientific experts in these two fields, from both public as well as private sectors. In particular:
- The 13th International Symposium on Schistosomiasis (Belo Horizonte, Brazil, 2012) opened an important dialogue with governmental representatives; it led to one new partner (Farmanguinhos, the federal governmental pharmaceutical laboratory of the Fiocruz Foundation in Brazil) joining the Pediatric Praziquantel program, thereby bringing its unique expertise for the production and distribution of the new pediatric formulation in endemic countries;
- The 11th Malaria Meeting at the end of 2013 (Aachen, Germany), which fostered a new dialogue and partnership with Saint George’s University (UK);
- The 7th EDCTP (European and Developing Countries Clinical Trials Partnership) Forum in mid-2014 (Berlin, Germany);
- The Harvard University Symposium on Malaria and Tuberculosis at the end of 2014 in Boston, MA (USA), during which the biopharmaceuticals business presented their innovative business model and fostered a number of future collaboration opportunities.