GHIT Fund expands investments in leishmaniasis, diagnostic tests

May 22 2015

GHIT Fund Also Announces Clinical Advancement of Antimalarial Drug and Malaria Vaccine Candidate

The Global Health Innovative Technology Fund (GHIT Fund), which in the last two years has funded almost $32 million for innovative tools to tackle global infectious diseases, today announced additional investments of nearly $11 million that bring its portfolio to approximately $43 million. GHIT Fund is expanding its technology scope to include diagnostic tests, its disease portfolio to include leishmaniasis, and its Screening Platform to include four additional Japanese companies and their unique chemical compound libraries.

The investments will include new funding for two of the world’s most neglected diseases, which overall threaten nearly 3 billion people—a little less than half of the world’s population. People are infected with schistosomiasis when they come in contact with contaminated water from which the parasite burrows into the skin, while leishmaniasis is spread by a parasite transmitted by infected sandflies.

GHIT Fund will invest in the development of a new point-of-care test for the diagnosis of schistosomiasis. Concurrently, GHIT will further invest in the continued development and registration of a pediatric formulation of praziquantel, the gold standard treatment for schistosomiasis. These two investments combined make the GHIT pipeline in schistosomiasis one of the most robust in the world.

Other investments include new funding for a new visceral leishmaniasis (VL) drug and the expansion of GHIT’s Screening Platform for malaria and tuberculosis to include four new Japanese entities: Daiichi Sankyo RD Novare Co., Ltd., Dainippon Sumitomo, Mitsubishi Tanabe Pharma, and Op Bio Factory Co., Ltd. of Okinawa.

GHIT Fund is a unique public health partnership that brings Japanese know-how and investment to the global fight against infectious diseases.

“We are proud that GHIT has been able to expand its investments into two new areas, leishmaniasis and diagnostics, as well as continue funding for current work that shows promise,” said GHIT Fund CEO Dr. BT Slingsby. “This is not only a symbol of our success in just two years, but also indicates the increased impact we can have on diseases that have, for far too long, threatened the world’s most vulnerable people.”  

First Investment in Diagnostics Further Fortifies Schistosomiasis Pipeline

GHIT is investing in four Japanese-, Philippines-, and US-based organizations to develop a faster and more effective test to detect schistosomiasis. The new point-of-care test, which would be the first of its kind to use recombinant proteins in diagnosing schistosomiasis, would be available for use in remote areas, allowing patients to be treated more quickly. This is expected to replace the traditional microscopic examination of stool to detect the worms’ eggs for schistosomiasis diagnosis, which is time-consuming and labor-intensive.

Schistosomiasis, also known as bilharzia, is caused by parasitic worms that live in specific types of freshwater snails. This is spread in freshwater contaminated with infective larvae that can penetrate the skin of susceptible mammalian hosts. Without treatment, chronic infection can cause enlarged liver, blood in the stool and urine, and can even lead to an increased risk of bladder cancer. This parasitic disease can be seen in 78 countries and affects more than 230 million people, including 100 million children―making it the second most important parasitic disease next to malaria.

The Schistosomiasis Detect Consortium includes Japan’s National Research Center for Protozoan Diseases (NRCPD)/Obihiro University of Agriculture and Veterinary Medicines and the Graduate School of Agricultural and Life Sciences/University of Tokyo, the Philippines’ College of Public Health, University of the Philippines-Manila, and the Seattle, US-based InBios International, Inc. NRCPD and Obihiro University have already identified target antigens for diagnosis, and the consortium will screen and select additional complementary targets to further increase the test’s sensitivity.

“New and better ways to diagnose schistosomiasis are desperately needed so that patients can quickly receive the best treatment available,” said Professor Shin-ichiro Kawazu of Obihiro University. “GHIT’s investment will allow us to develop a faster, more effective test that can diagnose patients on-the-spot, in even the most remote villages. Time is of the essence when it comes to treating neglected diseases, and this funding gets us closer to making a real impact.”

The new diagnostic combines well with the current GHIT investment in a pediatric formulation of praziquantel, the gold standard treatment for schistosomiasis. Today GHIT also announced an additional investment of $4.9 million for continued development and registration of this pediatric formulation of praziquantel in support of the Pediatric Praziquantel Consortium in implementing its Phase 2 development program. In March 2014, GHIT had provided a first round of funding to the Consortium to support its Phase 1 development program. The Consortium, a non-profit international public-private partnership, involves Top Institute Pharma of the Netherlands, Merck KGaA of Germany, Astellas Pharma Inc. of Japan, Swiss Tropical and Public Health Institute, Simcyp Limited of the UK and Farmanguinhos of Brazil.

New Drug Series to be Developed for “Ideal Treatment” for Visceral Leishmaniasis

GHIT is investing $4 million into a partnership with the Geneva-based Drugs for Neglected Diseases initiative (DNDi) and the Tokyo-based Takeda Pharmaceutical Company Limited to develop a new visceral leishmaniasis (VL) drug based on a novel series of aminopyrazoles, which recently demonstrated a proof of concept in an animal model.

Every single year, there are some 300,000 new cases of the most severe form of leishmaniasis: visceral leishmaniasis (VL), which causes fever, weight loss, enlargement of the spleen and anemia. If left untreated, it is almost always fatal. For those co-infected with HIV, the problem is even worse: VL accelerates the onset of AIDS. More than 90 percent of all VL cases are found in six countries in South Asia, Africa and Latin America: Bangladesh, Brazil, Ethiopia, India, South Sudan and Sudan.

Existing treatments for VL have serious drawbacks: toxic and costly, they usually require painful injections and intravenous infusions for long periods of time. Current drugs have also become increasingly ineffective against drug-resistant parasites, and have a high risk of failure for HIV co-infected patients. In fact, all co-infected patients will eventually relapse and die unless given antiretroviral therapy.

The targeted profile of the new drug would not only be short-course, safe and effective, but also easily delivered in the field by mouth, and would ideally also be an option for HIV co-infected patients. Researchers intend for the new drug to be used in combination with other oral therapies, providing a major step forward over existing monotherapies and combination therapies, potentially addressing drug-resistance issues. If effective, it could also be a potential candidate to treat other forms of leishmaniasis, and would help to reduce disease transmission.

With GHIT’s funding, DNDi and Takeda will collaborate on a “lead optimization program” of the aminopyrazole series, with the ultimate goal of identifying the best compound to treat VL, which will then move on to further preclinical development. They’ll continue designing and making improved candidates based on the early aminopyrazoles, select the most promising 4-6 leads, then paring them down to 2-3, and then eventually one that best meets the goals for a new, more effective drug.

“We’re excited about the possibility of this new drug series to tackle visceral leishmaniasis,” said Charles Mowbray, Head of Drug Discovery, DNDi. “With this funding, our partnership will endeavor to find the best compound from this new series that could meet all the requirements we have set out as the ideal treatment for visceral leishmaniasis.”

Opening Up Japanese Pharmaceutical Compound Libraries

Today GHIT welcomed four additional companies to its unique Screening Platform.

• GHIT has provided two investments of US$150,000 each for the New York-based Global Alliance for TB Drug Development (TB Alliance) to screen the libraries of Op Bio Factory Co., Ltd. of Okinawa and Daiichi Sankyo RD Novare Co., Ltd. of Tokyo for compounds that show potential to treat tuberculosis.

• GHIT has made two other investments for Geneva’s MMV to screen the libraries of the Osaka-based Mitsubishi Tanabe Pharma ($US150,000) and Sumitomo Dainippon Pharma Co., Ltd. ($US110,000) for new drug candidates for malaria.

The GHIT Screening Platform was launched in June 2013 to screen novel Japanese chemical compound libraries for potential new drug candidates for malaria, tuberculosis, Chagas disease and leishmaniasis. To date, GHIT Fund has financed the screening of over 250,000 compounds.

Advancing Clinical Candidates in the GHIT Pipeline for Malaria

Today GHIT announced progress on two key current investments in its malaria portfolio.
Takeda and Medicines for Malaria Venture’s (MMV) DSM265 antimalarial drug candidate has reached the Phase 2a GO milestone, meaning that decisions to fund the Phase 2a clinical trial are underway.

A malaria vaccine candidate called BK-SE36 will begin a Phase 1 clinical trial this summer. It is being developed by the European Vaccine Initiative (Germany), the Research Institute for Microbial Diseases at Osaka University (Japan), and the Centre National de Recherche et de Formation sur le Paludisme (Burkina Faso).