Completion of Phase 3 trial for treatment of “Snail Fever” (aka schistosomiasis) is final step before regulatory and WHO submission; GHIT also announces new investment in novel malaria vaccine and therapeutic for dengue, continued support for innovative tools to tackle malaria and neglected tropical diseases
The Global Health Innovative Technology Fund (GHIT Fund), a unique Japanese public-private partnership formed to battle infectious diseases around the globe, today announced 11 new investments totaling US$23 million* that could help deliver a range of new innovative therapies for a host of debilitating conditions.
This latest round of targeted support includes funding for a Phase 3 clinical trial testing a pediatric formulation of a drug considered the gold standard for treating schistosomiasis, a water-borne parasitic disease linked to an assortment of acute and chronic health problems. Young children are most at risk, but the existing drug is so bitter and hard to swallow that kids often go untreated, leading to serious lifelong health and learning problems.
This clinical trial is one of the most advanced partnerships invested by the GHIT Fund, an organization that combines Japan’s historic leadership in global health and innovation with groundbreaking research from across the globe. The GHIT Fund also is making new investments in two malaria vaccine candidates, while accelerating work to find new drug treatments for malaria, dengue, Chagas disease, cryptosporidiosis and leishmaniasis.
“We’re reaching an exciting phase where GHIT’s approach to partnerships and drug and vaccine development is starting to produce tangible progress towards product deployment that could eventually lead to revolutionary breakthroughs,” said BT Slingsby, MD, PhD, MPH, who is CEO of the GHIT Fund. “We knew that combining Japan’s wealth of biomedical research talent and pharmaceutical capabilities with leading infectious disease experts near and far was likely to be a winning combination, and that’s been validated by the progress we are seeing across a rich diversity of projects.”
Schistosomiasis, sometimes called “snail fever” because it’s found in freshwater snails, leads to both acute and chronic disease. It’s caused by parasitic worms known as blood flukes and is usually transmitted through contact with infested water.
The disease is endemic in 78 developing countries and, according to the World Health Organization, more than 261 million people, including 100 million children, were infected with schistosomiasis in 2015. Some 90 percent of infections occur in Africa, where safe water is often scarce. While rarely fatal, left untreated, the disease can cause anemia, stunted growth, impaired learning ability and chronic inflammation of vital organs.
GHIT Fund’s investment of US$4.7 million, with co-funding from its partners, will support a Phase 3 clinical trial in Africa to evaluate a pediatric formulation of praziquantel (PZQ) in children aged three months to six years. Since the 1970s, the gold-standard of treatment for the disease has been a single oral dose of PZQ used to treat adults and school-aged children. But children under age five who are infected with schistosomiasis are not treated with PZQ under the current policy. And data on the treatment of these children has been sparse and insufficient to define and confirm the best dosing. In addition, the current tablets have a severe bitter taste and the large size of the existing pill makes it difficult or just impossible for small children to swallow.
A smaller, more palatable pill that could be administered to children as young as three months old is being developed by the Pediatric Praziquantel Consortium, a nonprofit international public-private partnership involving Astellas Pharma Inc. (Japan), Lygature (The Netherlands), Merck KGaA (Germany), the Swiss Tropical and Public Health Institute, Simcyp Limited (UK), Farmanguinhos (Brazil) and the Schistosomiasis Control Initiative (SCI, UK).
Previous GHIT Fund investments for this project took the formulation through a Phase 2 clinical trial in 2015 and 2016. If successful, the Phase 3 trial will pave the way for regulatory review and prequalification by the World Health Organization (WHO) that would deliver an affordable, effective, child-friendly formulation of PZQ to young victims around the globe.
Biting Back at Malaria
GHIT also announced today a new investment of US$600,000 to develop a unique vaccine for malaria that prevents the malaria parasite from being transmitted from an infected person to a mosquito. Although it won’t protect individuals from malaria, the formulation enables the vaccinated person to become a sort of “human shield” that breaks the vicious cycle of disease, which depends on the malaria parasite being able to move from mosquito to human and then back to mosquito. Known as a “transmission-blocking vaccine,” a successful formulation would be a significant weapon in the global push to eliminate malaria, which killed 438,000 people in 2015, most of them young children in sub-Saharan Africa.
Researchers from the PATH Malaria Vaccine Initiative (US) and Ehime University (Japan) will evaluate a protein (called Pfs230) found in the deadly P. falciparum malaria parasite that appears to produce antibodies that can block disease transmission from humans to mosquitos. The investment from GHIT allows the team to examine small regions of the extremely complex protein via an innovative research tool known as wheat germ cell-free protein synthesis technology. The goal is to harness the transmission-blocking features of the protein to drive development of new vaccine candidates.
GHIT Fund also is investing US$2.8 million to continue work on a promising malaria vaccine candidate being developed by researchers from Japan’s Research Institute for Microbial Diseases and the Medical Center for Translational and Clinical Research at Osaka University, the Germany-based European Vaccine Initiative (EVI), the Centre National de Recherche et de Formation sur le Paludisme (CNRFP, Burkina Faso), and Nobelpharma Co., Ltd. (Japan).
Their formulation, called BK-SE36, has produced encouraging results in early testing, generating an immune response in Japanese adults and in Ugandan volunteers aged 6-32 years. Currently, it is being tested in a Phase 1b clinical trial with young children aged 1-5 years in Burkina Faso, where malaria is rampant. This new investment from the GHIT Fund will enable researchers to evaluate a different formulation of the vaccine in healthy African adults and children, one that contains an additional substance known as an adjuvant that has shown promise in boosting the immune response to the vaccine.