TOKYO, March 29, 2017 /PRNewswire-USNewswire/
-- 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."
A Snail Fever Drug That's Easy to Swallow
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.
GHIT will also continue to support four projects that are
pursuing new malaria drugs, which are urgently needed to fight the
spread of parasites that have become resistant to existing
therapies. Over the last few years there has been an emergence of
P. falciparum malaria parasites—first in Southeast Asia and just recently in
Africa—that can survive an assault from previously powerful
antimalarial drugs. Researchers are now racing to develop new
treatments that can kill the parasite in a single dose. GHIT's
investments in this work include:
- US$750,000 for an HTLP
partnership (Hit-To-Lead Platform) between Eisai Co. Ltd.
(Japan) and Medicines for Malaria
Venture (MMV, Switzerland) to
further explore a series of "hits" that emerged from screening
20,000 compounds in Eisai's library for potential activity against
malaria. MMV and Eisai have been evaluating potential activity
against different stages of the malaria parasite to identify
compounds that could be "lead" candidates for antimalarial
treatments. This new investment will allow further investigation of
one of these "hits" while supporting "hit-to-lead" work with a new
series of compounds.
- US$483,000 for a second HTLP
partnership, this one between Takeda Pharmaceutical Company Limited
(Japan) and MMV, to examine a new
series of compounds in Takeda's chemical library that might have
the potential to fight malaria.
- US$2 million to the Broad
Institute of MIT and Harvard (US), Eisai and MMV to build on promising
work to identify a series of compounds that can quickly defeat
drug-resistant strains and prevent their spread by blocking
transmission of the parasite. The new funding will allow
researchers to narrow their focus and identify two or three
compounds that warrant further study.
- US$4.5 million to the
University of Kentucky (US), Eisai and
MMV for a Phase 2a study of an antimalarial compound called SJ733,
which is intended for use in combination with other malaria
medicines to provide a fast-acting treatment that also prevents
relapse. In addition to supporting a Phase 2a study in adults, this
investment will allow researchers to investigate potential drug
formulations and conduct "challenge" trials, in which healthy
volunteers are given malaria under controlled circumstances and
then treated with the drug to see if it is effective. This work
will set the stage for further Phase 2 studies in children and
pregnant women—populations most at risk for malaria.
Going After the World's Most Overlooked Diseases
In its latest round of investments, GHIT is continuing to
confront some of the most neglected diseases in the world—diseases
that burden more than 1 billion of the world's poorest people and
keep them impoverished by causing a range of chronic mental and
physical problems. Fighting these overlooked diseases has been a
long-time priority for Japan, and
for GHIT, which officially endorsed the London Declaration on
Neglected Tropical Diseases in 2014, pledging ongoing funding to
defeat them once and for all.
GHIT announced today the following investments in neglected
diseases:
- US$5.3 million for Chugai
Pharmaceutical Co., Ltd. (Japan)
and A*STAR's Singapore Immunology Network (SIgN) to pursue
pre-clinical development of a therapy that could treat the symptoms
associated with the four types of dengue virus, as well as prevent
lethal and severe cases of the disease. The global burden of this
mosquito-borne disease is increasing, with about half of the
world's population now at risk of contracting dengue. Dengue causes
flu-like symptoms, extreme joint pain and can progress to a deadly
hemorrhagic fever.
- US$780,000 to support a
hit-to-lead collaboration between the Drugs for Neglected Diseases
initiative (DNDi) and Daiichi Sankyo in the areas of
leishmaniasis and Chagas disease. The project aims to progress
promising hit series, all of which were previously identified
through a GHIT-supported screening venture, into potential
treatment leads for these diseases.
- US$550,000 in continued funding
to DNDi, Eisai, Shionogi & Co. Ltd. (Japan) and Takeda for continued work with the
Neglected Tropical Diseases Drug Booster program—a groundbreaking
initiative committed to accelerating early-stage drug discovery for
treatments for leishmaniasis and Chagas disease. Visceral
leishmaniasis (VL) causes fever, weight loss, enlargement of the
spleen and anemia. If untreated, it is almost always fatal. Chagas
disease, which kills more people in Latin
America than any other parasitic disease, causes severe
heart damage and intestinal problems. This new investment will
allow partners to continue the work of the booster program, which
seeks to overcome early-stage intellectual property barriers and
enable DNDi to search for new leads by evaluating thousands
of unique compounds simultaneously.
- US$980,000 for an effort to
identify novel drug targets for a group of deadly parasitic
diseases: malaria, Chagas disease and leishmaniasis, and also for
cryptosporidiosis, which is a major cause for severe diarrhea in
infants and toddlers. This work will be led by the RIKEN Center for
Sustainable Resource Science (Japan), the Structural Genomics Consortium at
University of Toronto (Canada), the University
of Melbourne (Australia),
McGill University (Canada), MMV and DNDi.
The first of its kind in Japan, the GHIT
Fund is a public-private partnership between the
Japanese government, multiple pharmaceutical companies, the Bill
& Melinda Gates Foundation, the Wellcome Trust, and
UNDP. Launched in April 2013
with an initial commitment of more than US$100 million
and now with capital of over US$140
million, the organization taps Japanese research and
development (R&D) to fight neglected diseases. GHIT Fund
invests and manages a portfolio of development partnerships aimed
at neglected diseases that afflict the world's poorest people. GHIT
Fund mobilizes Japanese pharmaceutical companies and
academic and research organizations to engage in the effort to get
new medicines, vaccines, and diagnostic tools to people who need
them most, with Japan quickly
becoming a game-changer in global health. For more information,
please visit http://www.ghitfund.org
*All amounts listed at the exchange rate of USD1 = JPY100
For more information, contact:
Katy Lenard at +1-301-280-5719 or
klenard@burness.com
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SOURCE Global Health Innovative Technology Fund (GHIT Fund)