GHIT Fund
ANNUAL REPORT

2018

E

01

CONTENTS
Reaching New Heights

03

Advancing Portfolio

05

Clinical Candidates

07

SILVAMPTM TB-LAM

09

Pediatric Praziquantel (PZQ)

11

Fosravuconazole (E1224)

13

New Partnerships FY2018

15

Uniting Efforts for Innovation, Access, and Delivery

17

Co-Investment: Amplifying Impact for Patients

19

Finances

21

Leadership

23
02

ReachingNew
Heights
The 2018 fiscal year begins GHIT’s second five-year phase ofof the gold-standard drug for schistosomiasis (P.11-12), and aoperations after our successful replenishment in 2017. We are

long-lasting mycetoma drug (P.13-14). It is consistently clear topleased to report on our progress and performance, madeus that our development partners and co-investors share ourpossible by our partner’s unwavering efforts and commitment

mission, passion, and sense of urgency to progress. We aretoward developing game-changing innovations for global health.

committed to the registration of two products from our portfolio

We are thrilled to be closer than ever to graduating innovativeby 2022.

clinical candidates from our portfolio and putting them into thehands of those who need them most.

Because we recognize that innovation is valueless without
access, we also remain committed to the integration of access at

03

Since its 2013 establishment, GHIT has catalyzed the research

every step of the R&D process. In keeping with this core piece ofand development (R&D) of new global health innovations forour institutional DNA, this past year we co-launched—together

neglected patients by facilitating cross-sector partnerships

with the Government of Japan and the Access Delivery

between Japanese and non-Japanese entities. GHIT has invested

Partnership led and coordinated by the United Nations

USD 170M in 80 projects to date; currently, 22 discovery

Development Programme (UNDP) —“Uniting Efforts for

projects, 14 preclinical projects, and 8 clinical trials are under

Innovation, Access and Delivery,” a platform for dialogue andway in low- and middle-income countries (LMICs). Our mostcollaboration among key stakeholders involved in innovation,

advanced clinical candidates include a urine-based rapid

access, and delivery of health technologies for unmet healthtuberculosis (TB) diagnostic kit (P.09-10), a pediatric formulationneeds in LMICs (P.17-18).

All of these achievements build important momentum for theyear ahead. In 2019, Japan will host its first G20 Summit,
including the first-ever meeting between health and financeministers, as well as the Tokyo International Conference on
African Development VII. Both convenings represent critical
milestones for Japan’s and GHIT’s leadership in global healthand significant opportunities to further strengthen and expand
R&D and access partnerships. The UN High-Level Meeting on
Universal Health Coverage (UHC) in September 2019 also
provides a critical opportunity for Japan to show its leadership in
this area and increase momentum toward attaining UHC acrossthe globe.

Hiroki Nakatani

BT Slingsby

Board Chair & Representative Director

CEO & Executive Director(through March 31, 2019)

We are so grateful for the skill, passion, and steadfast
commitment that every single one of our governors, funding
partners, and development/access partners actively brings to thetable every day. They motivate us to reach higher, work better,and keep pushing toward our vision.
04

Clinical
Phase I

Phase II

Phase III

Registration

Product Development

Product Validation

Product Validation

Registration

Ib

IIa

IIb

IIb

▶P09-10Chagas disease

Schistosomiasis

IIb

III

▶P11-12MycetomaIIb▶P13-14

Investment Overview FY2013-FY2018

DiseaseMalaria

39.2%

Tuberculosis 13.5%
NTDs

Development
Stage

Intervention

47.3%

Drugs

68.3%

Discovery

16.1%

Vaccines

24.9%

Preclinical

56.0%

Diagnostics

6.8%

Clinical

27.9%

80

Total invested partnerships
44 Active investments
36 Completed investments

114

Total product development partners
43 Japanese partners
71 Non-Japanese partners

06

Clinical Candidates

BK-SE36/CpG
SJ733

Disease: Malaria
Intervention: Drug
Development Stage: Phase IIa
Country: To be determined
SJ733 is a chemically novel and potent
antimalarial candidate, based on adihydroisoquinolone platform and featuring goodoral availability, excellent safety tolerability, and alow propensity for resistance. The trial plans totest SJ733 in combination therapy to target special
needs groups (children and pregnant women).

Disease: Malaria
Intervention: Vaccine
Development Stage: Phase Ib
Country: Burkina Faso

This trial will assess the safety and reactogenicityof three doses of malaria vaccine candidate
BK-SE36, formulated with TLR9 ligand adjuvants
K3 CpG oligodeoxyribonucleotides (K3
CpG-ODN) in healthy African adults and children
exposed to the Plasmodium falciparum parasite.

Bolivia

E1224 Chagas disease/Drug

DSM265

Disease: Malaria
Intervention: Drug
Development Stage: Phase IIb
Country: To be determined
DSM 265 is a novel antimalarial candidate
anticipated to become a single exposure radical
cure and prophylaxis (SERCaP) product. The trial
aims to optimize oral formulation and lower theadministration volume for pediatric use.

E1224

Disease: Chagas disease
Intervention: Drug
Development Stage: Phase IIb
Country: Bolivia
This trial compares the safety and efficacy of oral
Fosravuconazole (E1224, a water-soluble
ravuconazole prodrug) regimens withbenznidazole in adults with chronic, indeterminate
Chagas disease. The novel candidate treatment
regimen is oral, easy to use, safe, and affordable.

07E1224

Disease: Mycetoma
Intervention: Drug
Development Stage: Phase IIb
Country: Sudan

PZQThis trial tests whether Fosravuconazole (E1224),
an azole-class antifungal drug discovered by Eisai,
may also be an effective and affordable treatment
for mycetoma. The trial is being conducted with
the WHO Collaborating Center on Mycetoma in
Khartoum.

Disease: Schistosomiasis
Intervention: Pediatric Drug
Development Stage: Phase III
Countries: Côte d'Ivoire, Kenya,
Zimbabwe
The Pediatric Praziquantel Consortium developed
the pediatric formulation of the gold-standard drug
for schistosomiasis to address the unmet medical
needs of infected preschool-age children. The
formulation under investigation is smaller,
exhibits an improved palatability, and is orally
dispersible, in contrast to the current commercial
formulation.

▶P13-14

Burkina Faso

BK-SE36/CpG Malaria/Vaccine

Sudan

E1224 Mycetoma/Drug▶P11-12

Kenya

Côte d'Ivoire

PZQ Schistosomiasis/Pediatric DrugPZQ Schistosomiasis/Pediatric Drug

Tanzania
Zimbabwe

DAR-901 TB/Vaccine

PZQ Schistosomiasis/Pediatric Drug

South Africa

DAR-901

TB-LAM TB/Diagnostic

Disease: Tuberculosis
Intervention: Vaccine
Development Stage: Phase IIb
Country: Tanzania

TB-LAM

Disease: Tuberculosis
Intervention: Diagnostic
Development Stage: Product validation
Country: South Africa

DAR-901 is the only novel TB booster vaccine
candidate based on inactivated whole cell derived
from Mycobacterium obuense that has shown
efficacy in humans. The DAR-PIA trial will be a
Phase II study of the DAR-901 booster conductedamong 13-15 year old adolescents in Tanzania.

This project aims to develop a highly sensitive,
rapid point-of-care TB test to diagnose the disease
from urine in high-risk, HIV-infected individuals.
Product validation studies based on samples fromSouth African townships are under way.

▶P09-1008

Clinical Candidate 1

SILVAMP TB-LAM
TM

Novel, urine-based, point-of-care TB test for people livingwith HIV in low-resource settings

One of the three most deadly infectious diseases theworld has ever known
TB infects 10 million people and kills 1.6 million annually.

SILVAMPTM TB-LAM uses patients’ urine to diagnose
TB, detecting low concentrations of LAM
1(lipoarabinomannan), which is found in the cell walls of

The dire socioeconomic consequences of this disease canmycobacterium tuberculosis.3 The partnershipdrive entire countries into poverty. Patients and theirsuccessfully leverages FUJIFILM’s proprietary silverfamilies endure severe stigma, and many are unable to work.

halide amplification technology, originally developed

This, coupled with the significant cost of treatment,for processing photographs, to create highly sensitive

contributes to a vicious circle for millions across the globe.

immunochromatography, which is capable of detecting

87% of all TB patients live in Africa and Southeast Asia,

viruses and bacteria. The SILVAMPTM TB-LAM isbut the disease affects every country in the world.

simple to use and does not depend on special equipment

Drug-resistant TB is a major global public health threat.

or require a stable power source. Therefore, thisinnovative diagnostic kit has the potential to be a

The combination of HIV and TB is particularly lethal, withgame-changing solution for detecting TB in endemic

each speeding the other’s progress. People living with HIV

areas.

are up to 30 times more likely to develop TB than those whoare HIV negative. The World Health Organization (WHO)Clinical and regulatory progress

estimates that 57% of TB cases among people living withThe SILVAMPTM TB-LAM complies with health, safety,

HIV are not diagnosed or treated.2

and environmental protection standards for products sold in
the European Economic Area and was CE marked in

Need for diagnostic innovation

November 2018. To obtain WHO endorsement to use theCurrently, TB diagnosis is made primarily based on sputum

test for TB diagnosis in HIV-positive patients in LMICs, theanalysis. However, because 20–60% of HIV-positive

product is undergoing a validation studies to performpatients presenting for TB diagnosis are unable to produce aprospective evaluations in settings of intended use and to

sputum sample, many patients cannot be diagnosed in time,

generate the required evidence for WHO policy

which results in high morbidity and high mortality. The

development. The partnership has also facilitated theUnited Nations' Sustainable Development Goals (SDGs) andSILVAMPTM TB-LAM’s transition to volume

End TB Strategy call for reducing TB mortality rates by

manufacturing.

90% and infection rates by 80% by 2030. To do this, theworld needs a non-sputum based, rapid diagnostic tool thatcan be used in LMICs and is effective for use with
HIV-positive patients.

Leveraging Japanese photograph processing
technology to transform diagnosis
GHIT has since 2016 invested in a partnership between
FUJIFILM Corporation and the Foundation for Innovative
New Diagnostics (FIND) to develop the SILVAMPTM
TB-LAM, a rapid, low-cost TB diagnostic with highsensitivity and specificity. Instead of sputum, the09

References
1. https://www.who.int/gho/tb/epidemic/cases_deaths/en/
2. https://www.who.int/hiv/topics/tb/about_tb/en/
3. http://www.fujifilm.com/news/n180927_02.html

South Africa

Disease: Tuberculosis
Intervention: Diagnostic
Development Stage: Product validation
Country: South Africa“FUJIFILM has applied its technology andknowledge originally developed for photographprocessing into problem solving in health andmedicine. the SILVAMPTM TB-LAM is suitable
for resource-limited countries with unreliable
power, and it delivers rapid and accurate
results by simply placing a urine sample into a
cartridge that does not require electricity. We
believe this will be a game-changing tool for the patients andhealthcare professionals who fight against tuberculosis.”“We are proactively working on R&D andaccess issues every day to develop better
innovations and deliver to those who are in
need. There's nothing that could make us
happier than knowing that our diagnostics arebeing utilized in the clinical settings in LMICs
and are contributing to health for as many
people as possible.”

Junichi Katada
Manager, Research & Development Management Headquarters,

Kaoru Terashima

Medical Systems Research & Development Center, FUJIFILM Corporation

Corporate Vice President, In Vitro Diagnostics, FUJIFILM Corporation“The SILVAMPTM TB-LAM has already shown
great potential in identifying TB in people whoare HIV positive – this could be the first of a
new generation of rapid tests that could
transform TB diagnosis for all. Early
diagnosis enables faster linkage to treatment,
which positively impacts patients, theirfamilies, and everyone around them. Simply
speaking, lives are saved.”“Currently, diagnostic testing is not easily
accessible, sometimes not affordable, andresults often takes too long. Results fromsputum-based TB diagnoses take labs several
days to turn around. On the other hand, the
SILVAMPTM TB-LAM uses patient urine
samples and is very easy to use; results areready within one hour. If a test result is
positive, patients can receive lifesaving treatment that same day.”Catharina Boehme

Caroline Mateben

CEO, FIND

Research Nurse, Medical Microbiology, University of Cape Town

Product Development Partners

10Clinical Candidate 2

Pediatric Praziquantel(PZQ)
A new pediatric formulation of the gold-standard drug
for schistosomiasiss
Schistosomiasis: the pediatric challengeof the tablets, helps children swallow more easily, which

Endemic in 78 countries, schistosomiasis, also known as

facilitates compliance and efficacy. After a successful"bilharzia and snail fever," is a water-borne disease that affects

technology transfer from Astellas in Japan to Merck KGaA

nearly 240 million people and carries a debilitating health,in Germany and Farmanguinhos in Brazil, and successful

economic, and social footprint, particularly for children andclinical trials in South Africa, Tanzania, and Côte d'Ivoire,their families. Exposure leaves these children vulnerable tofurther formulation development and production processincreased risk for anaemia, stunting, and a reduced ability to

optimization activities were conducted to develop better

learn, although the effects are usually reversible with treatment.

tablets for the Phase III trials in Kenya and Côte d'Ivoire

Praziquantel (PZQ), the affordable, gold-standard drug, hasand future launch. Compared to the PZQ tablets available

existed for more than 30 years and transformed the lives oftoday, the new formulation contains only levopraziquantel

millions of adults and school-aged children.

(R-(-)-PZQ) as an active pharmaceutical ingredient.

4

Dextropraziquantel (S-(+)-PZQ), the inactive isomer that
Treatment for preschool-aged children has not been

contributes to the severe bitter taste, has been removed.

implemented due to several key obstacles: difficulty inswallowing and risk of choking give the large-sized

Clinical progresscommercially available tablets; high rejection rates due to bitter

In 2019, the Consortium will initiate Phase III trials intaste when pills are crushed and dispersed in liquids; andpreschool-aged children in Côte d'Ivoire, Kenya, andpotential of under-dosing, which leads to lower effectiveness of

Zimbabwe to test the safety, effectiveness, and acceptability ofcrushed tablet suspensions.

the new formulation. At the same time, the Consortium is

5

solidifying regulatory, access, and delivery strategies,

Pediatric PZQ consortium

including incorporation of feedback from regulatory

In response to this unmet need, since 2014 GHIT has invested

authorities, such as the European Medical Agency and key

in a catalytic global public-private consortium of eight partners

stakeholders from WHO, and planning for long-term financing

to develop, register, and provide access to the first pediatricand supply chain mechanism to ensure timely and sustainable

PZQ formulation for schistosomiasis in preschool-agedroll out.

children. Current partners include Astellas Pharma Inc.,
Farmanguinhos, Kenya Medical Research Institute, Lygature,
Merck KGaA, the Schistosomiasis Control Initiative, the Swiss
Tropical and Public Health Institute, and Université Félix
Houphouët-Boigny.

Formulation innovation
Astellas led the initial formulation development during
preclinical phases. The resulting pediatric tablets developed byAstellas are about a quarter of the size of the existing
commercial PZQ tablets and are orally dispersible (can betaken with or without water). This, together with agents thatminimize the bitterness caused by PZQ and improve the taste
11

References
4. https://www.who.int/schistosomiasis/disease/en/
5. World Health Organization. Report of a meeting to review the results of studies on the treatment ofschistosomiasis in preschool-age children. Geneva: World Health Organization; 2011.

Côte d'Ivoire
Kenya
Zimbabwe

Disease: Schistosomiasis
Intervention: Pediatric Drug
Development Stage: Phase III
Countries: Côte d'Ivoire, Kenya, Zimbabwe“Consortium partners, which hail from acrosssectors and countries, are so committed. No
one person or partner could do this alone. We
are all experts in drug development but notnecessarily in delivering medications to remoteparts of Sub-Saharan Africa. Thus, the global
community will play a critical role in helping
us provide the new drug to the vulnerable
children who need it most. We will facilitate a dialogue with theglobal community to guarantee sustainable access and delivery.”“The first time I heard about the project in2012, I asked myself what I could do for smallchildren as a formulation expert to address thesize and bitterness of the existing PZQ tablet.
We are proud that Astellas’s technology
successfully solved these problems. Thinking
about the future, and the smiles of the children
in Africa who will be impacted by thisinnovation, we are highly motivated to deliver the product as soon
as possible, in partnership with members of the Consortium.”

Elly Kourany-Lefoll

Hiroyuki Kojima

Head of Neglected Tropical Diseases Drug Development, Merck KGaA

Senior Director, Drug Product Development, Technical Operations,Astellas Institute for Regenerative Medicine“This clinical trial is very important. Pediatric
PZQ will solve a major problem we have hadfor a long time; we will finally be able toreach children under 5 years of age. As agovernment and as a Ministry of Health, we
made the policy decision to lend our full
support to this trial because it will not only
provide a national solution for our country,
but also a global solution for the world.”“Thanks to pediatric PZQ, it will soon bepossible to expand schistosomiasis treatment
to preschool-aged children – a critical part ofthe long-term process of eliminating thedisease and progressing in line with WHO’s
NTD roadmap for 2020 and beyond.”

Amadou Garba
Scientist, Schistosomiasis Control Programme,
WHO Department of Control of Neglected Tropical Diseases

Sultani Hadley MatendecheroHead, Kenya National Neglected Tropical Diseases Program, Ministry of Health

Product Development Partners

12Clinical Candidate 3

Fosravuconazole (E1224)
First-ever double-blind,randomized control trial for mycetoma drug

One of the most neglected of all tropical diseases

would drastically improve patient compliance and reduce

Endemic in numerous tropical and subtropical countries,

costs for individuals and health systems alike.

mycetoma’s main victims are poor teenagers and young adults
in rural areas. Most cases of mycetoma are reported from theBy January 2019, 84 patients had been enrolled, reachingso-called “mycetoma belt,” which includes Brazil, Mexico, andthe threshold for interim analysis to determine the weekly

Venezuela in Latin America; Chad, Ethiopia, Mauritania,

dosage (200mg or 300mg) of Fosravuconazole. Once theSenegal, Somalia, and Sudan in sub-Saharan Africa; Yemen in

analysis has been completed, patient enrollment in the trial

the Middle East; and India in Asia, among others. Though

is expected to conclude by the end of 2019. Follow-up

cases date back more than 300 years, mycetoma was only

should conclude in early 2021 to assess the effectivenessadded to the WHO’s official list of Neglected Tropicalof Fosravuconazole as compared to itraconazole.7

6

Diseases in 2016.

Khartoum Call to Action
Mycetoma slowly and progressively destroys soft tissue,

Working closely with WHO, Sudan’s Federal Ministry ofparticularly on the feet. The disease has two forms:

Health has led the way in fighting mycetoma internationally. In

Actinomycetoma, caused by bacteria, and Eumycetoma, caused

October 2018, WHO Director-General Tedros Adhanom

by a fungal infection. Eumycetoma has no effective treatment

Ghebreyesus visited the MRC, where he pledged to supportand is currently managed with sub-optimal, expensive drugs

more research into mycetoma epidemiology and diagnostics tocarrying strong side effects and often requiring surgery,produce a simple point-of-care test, and called for new,including amputation of affected limbs. In rare cases, it affects

cost-effective medicines and disease control.8

the lungs or brain and can be fatal. In all cases, patients areunable to work and face severe social stigma. An effective,

At the 6th International Conference on Mycetoma in Khartoum

affordable, and easy-to-administer treatment is urgently

in February 2019, WHO and the MRC launched the Khartoum

needed.

Call for Action, which was signed by governments of endemic
countries and organizations—including GHIT—engaged in

Clinical Progress

mycetoma research and advocacy. The call to action will lead

GHIT has invested in a partnership between Eisai Co., Ltd.,to a greater commitment of support for mycetoma research,and the Drugs for Neglected Diseases initiative (DNDi),

diagnosis, treatment, and care.9

together with the Mycetoma Research Center (MRC), a WHO
Collaborating Center in Khartoum, Sudan. This partnership is
conducting the world's first double-blind, randomized control
Phase IIb Proof of Concept clinical trial in mycetoma,comparing Eisai’s Fosravuconazole, originally developed totreat other fungal infections, with the current treatment,
itraconazole. Eisai supplied the drug for the clinical study. The
trial is taking place in Sudan, the country where the disease ismost prevalent and where the government is uniquelycommitted at the highest levels to addressing this scourge.
Fosravuconazole has a proven safety profile and shows strong
antifungal activity against mycetoma. This drug, if successful,
13

References
6. https://www.who.int/news-room/fact-sheets/detail/mycetoma
7. https://www.dndi.org/wp-content/uploads/2019/02/DNDi_Mycetoma_2019.pdf
8. http://www.mycetoma.edu.sd/index.php/archive-mrc/146-director-general-of-who-calls-for-moremycetoma-research-during-visit-to-mycetoma-research-centre
9. https://www.who.int/neglected_diseases/news/The-Khartoum-Call-for-Action.pdf?ua=1

SudanDisease: Mycetoma
Intervention: Drug
Development Stage: Phase IIb
Country: Sudan“Mycetoma patients are the poorest of the poor in
the most remote areas. People of low
socioeconomic status and manual workers such as
agriculturalists, laborers, and herdsmen bear thebrunt of the burden. Health and socioeconomic
impacts are severe, including significant disability
and pain, as well as financial burden, preventing
patients and their families from going to school
and finding employment. The disease can devastate entire communities.
I dream of a world free of mycetoma and the suffering it cases; goodsolutions for treatment and diagnosis would get us closer.”“There is so much need for funding for R&D
for diagnosis and treatment, forepidemiological studies, and for better accessto affordable treatment for immediate usage.
Partnerships are what is needed to provide
solutions and bring patients hope. If we canshow good efficacy of fosravuconazole in thisclinical trial, that would be a great hope forthe patients. It would transform their quality of life.”

Nathalie Strub Wourgaft
NTD Director, DNDi

Ahmed Hassan Fahal
Professor of Surgery, University of Khartoum and Director, MRC“As I learned more about mycetoma patients in
Sudan, I came to truly recognize the devastating
effects of this disease on their daily lives. As ascientist and with these patients—who travel very
far and with great difficulty from rural areas tothe capital city to receive treatment—top ofmind, I re-committed to do whatever I possibly
do to deliver a safe, effective drug for them assoon as possible.”

Katsura Hata
Senior Director, Global Health Research Section,“Adding mycetoma to WHO list of NTDs in2016 helped bring more attention to thedisease. It also made us realize how much we
still don’t know. WHO supports andencourages partners, especially funders, toaddress mycetoma’s root causes and use themodern tools of science to find solutions to thisproblem.”

Soumya Swaminathan
Chief Scientific Officer, WHO

hhc Data Creation Center, Eisai & Co., Ltd.

Product Development Partners

14New Partnerships FY2018

Malaria
Tuberculosis

Tuberculosis

Diagnostic

Drug

Development of Lateral Flow Assay platform
for improving sensitivity of point-of-careassays for malaria and tuberculosis

Hit-to-Lead development of novel anti-TB
natural products
Daiichi Sankyo RD Novare (Japan) & TB Alliance (USA)Asahi Kasei Corporation (Japan) & Biopromic AB (Sweden)
Target Research
Platform

Screening
Platform

Hit-to-Lead
Platform

Product Development
Platform

Target Research
Platform

Screening
Platform

Hit-to-Lead
Platform

Product Development
Platform

According to the WHO’s target product profile forThis project is the result of a GHIT-funded screening effort by

infectious disease diagnostics, a limited number ofDaiichi Sankyo RD Novare, TB Alliance, and the Research

point-of-care tests are available, meeting a fraction of theInstitute of Tuberculosis at the Japan Anti-tuberculosis

demand. This is due to the very low concentration of easily

Association (RIT/JATA). RIT/JATA evaluated bactericidal

accessible antigens in point-of-care samples. Current

activity against TB using Daiichi Sankyo RD Novare's original

Lateral Flow Assay (LFA) devices offer analyticalnatural product library, created from microorganisms such as

sensitivity above 1-5ng/ml while the concentration ofactinomycetes and fungi and consisting of 30,000 extracts and 600

antigens in TB and malaria patients’ samples is usually

purified natural compounds. After close collaboration, the partners

below 100pg/ml. To address the clear need for more

identified a group of hit compounds and subsequently determinedsensitive LFA systems, Asahi Kasei and Biopromic AB’s

the structures of the active components. The active components

project aims to develop a new LFA platform with antigen

were further studied for their biological properties, and partners

detection 50 times the current industry standard. Achieving

developed a research plan to generate additional fermentationsuch a high analytical LFA test sensitivity requires

products for structural modifications and biological evaluations.

significant improvement and integration of multiple

Today, the partners seek to identify lead compounds that cancomponents of the LFA system.

result in effective new TB drugs that feature shorter treatments
and cures for both drug-sensitive and drug-resistant TB.

https://www.ghitfund.org/investment/portfoliodetail/detail/140/en15

https://www.ghitfund.org/investment/portfoliodetail/detail/142/en

Malaria

Leishmaniasis

Drug

VaccineLead optimization of antimalarials
Mitsubishi Tanabe Pharma Corporation (Japan) &Medicines for Malaria Venture (Switzerland)

Target Research
Platform

Screening
Platform

Hit-to-Lead
Platform

Product Development
Platform

Immune therapy to prevent visceralleishmaniasis complications
University of Tokyo (Japan), International Center for
Diarrheal Disease Research (Bangladesh), and Infectious
Disease Research Institute (USA)
Target Research
Platform

Product Development
Platform

Several antimalarial hits from a diverse series of molecules

Intervention in visceral leishmaniasis (VL; Kala-azar) patients at

resulted from the screening of Mitsubishi Tanabe Pharma

greatest risk of treatment failure, relapse, or subsequent

Corporation’s approximately 50,000-member library of uniquedevelopment of post-Kala azar dermal leishmaniasis (PKDL) is

compounds. Development of structure-activity relationships forcritical for effective disease management. Published data has

three series of focus in a hit-to-lead project revealed that one ofidentified vaccine candidates that are as effective as a prophylaxis

those series is appropriate for lead optimization. The series isin advanced animal models. Project partners will build upon thisfast-killing, exhibits high antimalarial potency across thedata to determine the candidate antigen best suited for use inlifecycle, and has good physicochemical and pharmacokinetic

Bangladesh. They will also use a long-term preclinical model ofproperties. It also shows efficacy in the mouse model for malaria.

the L. donovani infection to develop immune/chemotherapeutic

The primary objective of this project is to identify between one

approaches to prevent complications of VL. To develop an

and three late leads within 18 months, as defined by Medicines

effective therapeutic vaccine for VL, selecting the right antigen(s)for Malaria Venture’s progression criteria, and to further profile

and adjuvant is important. It is also key that the vaccine does notthem in the final six months to select a preclinical candidate

compromise the effects of chemotherapy. Therefore, this project

capable of progression to first-in-human clinical trials. The

will comprise three major activities: patient-instructed selection of

second objective is to confirm the proposed mode of action toa vaccine antigen for prevention of PKDL; evaluation ofassess the likelihood that these compounds will have the ability tocompatibility of vaccines with amphotericin B treatment; andtreat malaria safely in areas of emerging drug resistance.

evaluation of immune therapy efficacy in the long-term VL model.

https://www.ghitfund.org/investment/portfoliodetail/detail/136/en

https://www.ghitfund.org/investment/portfoliodetail/detail/130/en
16

A new convening and communication platform
R&D and access are inextricably linked. Yet few
opportunities exist for biomedical R&D funders, innovators,
and access stakeholders to discuss the common challenges
they face and to jointly identify solutions. To address thisgap, in January 2019 the Ministry of Foreign Affairs ofJapan, UNDP-administered Access and Delivery Partnership(ADP), and GHIT co-hosted the conference entitled“Uniting Efforts for Innovation, Access and Delivery: A
Global Dialogue in Bangkok”. The conference engaged
more than 100 carefully selected R&D funders, researchorganizations, product development partnerships, research
institutes, and access platforms to discuss challenges and17

opportunities around the innovation, access, and delivery of

optimize the introduction of new treatments to those most in

health technologies. The dialogue represented a rare andneed.10 Participants in this inaugural meeting identified

much-needed opportunity for partners across the R&D

numerous specific possibilities for future actions at the

spectrum, each with essential perspectives and roles.

global, regional, and national levels.

The event also marked the launch of Uniting Efforts for

Thanks to the support of the Government of Japan, GHIT and

Innovation, Access, and Delivery—a convening and

ADP have been grappling with these issues since 2013, on thecommunication platform to help improve the impact andone hand driving health technology innovation for malaria, TB,

efficiency of current and future R&D investments andand Neglected Tropical Diseases and on the otherpriorities, increase efficiencies and the integration of accessstrengthening policies, human capacities, systems, andconsiderations into R&D plans at an earlier stage, andregulations to promote access and delivery. Yet increased R&D

“No single country, sector, or organization can solve this problem. Let’s work together,so that patients can access innovative health products as soon as possible. Everyone is part of the solution.”

Manabu Sumi
Director, Global Health Policy Division
Ministry of Foreign Affairs of Japanefficiencies do not, on their own, lead to the introduction anduse of new health technologies. Greater progress toward
Universal Health Coverage and the Sustainable Development
Goal targets also require stakeholders across the globe to tackle
critical bottlenecks in LMIC health systems and enhance accessand delivery mechanisms.
In its first year, the platform is focusing on bringing
together key partners to define a common agenda for healthtechnology access and delivery preparedness in LMICs.
Over the long term, the aim is to establish a global networkof R&D funders, innovators, product development
partnerships, and delivery-focused organizations and to
evolve this network into a major platform for learning,
information exchange, and coordination and collaboration.
Reference
10. https://www.unitingeffortsforhealth.org/

18

Co-Investment:
Amplifying Impact
for Patients
In the same way that successful global health R&D is built
on partnership, so, too, is effective funding for that
innovation. Indeed, co-investment, where multiple funders
and partners collaborate to advance R&D, is a critical
approach to amplifying the impact of individual
investments.
GHIT is a proud co-investor in many partnerships alongsidemultiple funders around the world. For example, GHIT
co-invests with the European & Developing Countries
Clinical Trials Partnership (EDCTP) in the pediatric PZQ
phase III trial, which is providing clinical data and support

partners domestically and internationally to findfor registration of a new praziquantel tablet formulation toopportunities for co-investment and align investment

treat schistosomiasis in preschool-aged children. Other

strategy so that promising innovations can be developed andexamples include The Republic and Canton of Geneva

delivered to patients faster.

through its International Solidarity Office, which has
committed to support mycetoma activities in Sudan with the

By co-investing, funders decrease the risk associated withoverall goal of developing a new safe, effective, affordable

individual investment and amplify their impact

and field-adapted treatment for patients affected byexponentially. Moreover, by partnering with multiple

mycetoma. GHIT has worked with multiple funders andfunders, product development partners can accelerate

GHIT investment leveraged by co-investment

250.0

69.1

150.0

51.1
29.7
12.8
18.9

46.0

2013

2014

Co-investment

19

GHIT investment

32.4
63.7
2015

98.6
2016

131.9

2017

169.9

100.0

50.0

2018

Cumulative investments USD (in millions)

56.8

200.0

0

US dollar amounts represent conversions from Japanese yen, solely for the reader’s convenience, at JPY 100 = USD 1.
As of March 2019

progress by leveraging each funder’s unique networks androle in advancing product development.

resources. Finally, co-investment helps ensure that products
are part of the global product development portfolio.

GHIT aims to increase co-investment in its development
partnerships so that by the end of FY2022 its own

Importantly, co-investment is not restricted only to financial

contributions are matched by outside funding including

investments; in-kind donations and services can also be

in-kind contributions. In keeping with this goal, we are

critically important contributions. For example, private

actively exploring opportunities to join forces with othercompanies sometimes contribute human, technical, andco-investors and partners to leverage investment impact forother internal resources, each of which plays an essential

patients.

How Co-Investment Works

Funders

Leverage investment impact
Risk mitigation

Product Development Partners

Patients

Advance product development
Access funders’ network and resources

Enhance access toinnovations¥

Other Funders

¥

€

$

£
In-kind contributions comprisenon-monetary goods or services.

20

***For Translation Purposes Only***

Independent Auditor’s Report
To the Board of Directors, Global Health Innovative Technology Fund:<Audit of the Financial Statements>
We have audited the accompanying financial statements, which comprise the balance sheet, the statement ofincome, the notes to the financial statements, and the related supplementary schedules of the Public Interest
Incorporated Association Global Health Innovative Technology Fund (“the Organization”) applicable to theseventh fiscal year from April 1, 2018, through March 31, 2019. We conducted our audit in accordancewith the rules and regulations concerning the Act on the Authorization, etc. of Public Interest Incorporated
Associations and Public Interest Incorporated Foundations in Japan, under Article 23.
Directors’ Responsibility for the Financial Statements and the Related Supplementary Schedules
Directors need to ensure that the financial statements and related supplementary schedules were prepared andfairly presented in accordance with accounting principles generally accepted in Japan. Among others,
directors are responsible for designing and operating such internal control as directors determine is necessary
to enable the preparation and fair presentation of the financial statements and the related supplementaryschedules that are free from material misstatement, whether due to fraud or error.
Auditor’s Responsibility
Our responsibility is to express an opinion on these financial statements and the related supplementaryschedules based on our audit. We conducted our audit in accordance with auditing standards generally
accepted in Japan. Those standards require that we plan and perform the audit to obtain reasonableassurance about whether the financial statements and the related supplementary schedules are free frommaterial misstatement.
An audit involves performing procedures to obtain audit evidence about the amounts and disclosures in thefinancial statements and the related supplementary schedules. The procedures selected depend on theauditor’s judgment, including the assessment of the risks of material misstatement of the financial statements
and the related supplementary schedules, whether due to fraud or error. The purpose of an audit of thefinancial statements is not to express an opinion on the effectiveness of the Organization’s internal control,
but in making these risk assessments, the auditor considers internal controls relevant to the Organization’s
preparation and fair presentation of the financial statements in order to design audit procedures that areappropriate for the circumstances. An audit also includes evaluating the appropriateness of accounting
policies used and the reasonableness of accounting estimates made by directors, as well as evaluating theoverall presentation of the financial statements and the related supplementary schedules.
We believe that the audit evidence we have obtained is sufficient and appropriate to provide a basis for our
audit opinion.
OpinionIn our opinion, the financial statements and the related supplementary schedules referred to above present
fairly, in all material respects, the financial position and results of operations of the Organization applicableto the seventh fiscal year ended March 31, 2019, in conformity with accounting principles generally acceptedin Japan for Public Interest Incorporated Associations (similar to a 501(c)(3) in the United States).
<Opinion on the List of Assets and Liabilities>
We have audited the accompanying list of assets and liabilities for the seventh fiscal year of the Public
Interest Incorporated Association Global Health Innovative Technology Fund at March 31, 2019. We
conducted our audit in accordance with the rules and regulations concerning the Act on the Authorization, etc.
of Public Interest Incorporated Associations and Public Interest Incorporated Foundations in Japan, under
Article 23.
Directors’ Responsibility for the List of Assets and Liabilities
Directors need to ensure that the list of assets and liabilities was prepared and fairly presented in accordancewith accounting principles generally accepted in Japan and also in conformity with the public-interest
certification documents.

21***For Translation Purposes Only***

Auditor’s Responsibility
Our responsibility is to express an opinion on the said list of assets and liabilities which was prepared andfairly presented in accordance with auditing standards generally accepted in Japan and also in conformity
with the public-interest certification documents.
OpinionIn our opinion, the list of assets and liabilities referred to above present fairly, in all material respects, inaccordance with auditing standards generally accepted in Japan and also in conformity with the
public-interest certification documents.
<Conflicts of Interest>
We have no interest in the Organization which should be disclosed in compliance with the Certified Public
Accountants Act.

Ernst & Young ShinNihon LLC.
May 8, 2019
End-of-Document

2018 Financial Summary (Audited)
Balance Sheet
Assets (in million)

JPY

USD

Current Assets
Fixed Assets

1,156.2
4,097.8

10.4
36.9

Total Assets

5,254.0

47.3

JPY
935.6
292.4

USD
8.4
2.6

Total Liabilities

1,228.0

11.0

Net Assets (in million)
Designated Net Assets
General Net Assets

JPY
4,026.0
-

USD
36.3
-

Total Net Assets
Total Liabilities and Net Assets

4,026.0
5,254.0

36.3
47.3

JPY

USD

Grants Received
Contribution Received
Misc. Income

3,296.0
764.2
2.8

29.7
6.9
0.0

Total Ordinary Income

4,063.0

36.6

Operating Expenses
Management Expenses

3,865.8
197.2

34.8
1.8

Total Ordinary Expenses

4,063.0

36.6

JPY

USD

Grants Received and Others
Governments, NGOs, Multilateral Organizations
Foundations
Contributions Received

3,338.6
1,629.6
834.4

30.1
14.7
7.5

Total Grants and Contributions Received

5,802.6

52.3

Liabilities (in million)
Current Liabilities
Non-current Liabilities

Net Assets Variation Statement
Change in General Net Assets (in million)
Ordinary Income
incomeOrdinary Expenses

Change in Degignated Net Assets (in million)

The US dollar amounts in this section represent translations of Japanese yen, solely for the reader’s convenience, at PY 111 = USD 1, the exchange rate as ofMarch 31, 2019.
This fnancial summary is an excerpt from the GHIT Fund's audited fnancial statements, which are audited by Ernst & Young ShinNihon LLC.. The GHIT Fund isa Pubic Interest Incorporated Association and is registered in Japan.
22Jeremy Farrar, MD, PhD, FRCP

Astellas Pharma Inc.

Chugai Pharmaceutical Co., Ltd.

Director
Wellcome

Yoshihiko Hatanaka
Representative Director
Chairman of the Board

Tatsuro Kosaka
Representative Director
President and CEO

The Board of Directors consists of global health experts and management professionals. In addition to overseeing operations by the Leadership Team, it also resolves important businessmatters including approval of major rules, strategic plans, annual operational plans/budget, and funding decisions based on recommendations from the Selection Committee.

Chair & Representative Director

Hiroki Nakatani, MD, PhD, MHPEd

Project Professor
Global Research InstituteKeio University

Vice ChairPeter Piot, MD, PhD

Director and Professor of Global HealthLondon School of Hygiene and Tropical Medicine
Former Executive Director, UNAIDS

BT Slingsby, MD, PhD, MPH

CEO & Executive Director
Global Health Innovative Technology Fund
*Through March 31, 2019

Catherine K. Ohura, MS, PMP

CEO & Executive Director
Global Health Innovative Technology Fund
*From April 1, 2019

Daikichi Momma

Former Director-General, International Bureau
Ministry of Finance
Former Executive Director
International Monetary Fund representing Japan

Ex-Officio
Statutory Auditor
INSPiRE Corporation

Ex-Officio

Andrin Oswald, MD

Director, Life Sciences Partnerships
Bill & Melinda Gates Foundation

23

Lecturer at Law, Harvard Law School
Former Senior Vice President
and General Counsel, World Bank

Stephen Caddick, PhD

Director, Innovations DivisionWellcome

Co-Chair

Kiyoshi Kita, PhD

Co-ChairDennis Schmatz, PhD

Professor Emeritus, The University of Tokyo
Professor and Dean, Nagasaki University School of
Tropical Medicine and Global Health

Former Head, Infectious Diseases Research
Merck Research Labs, USA
Former Head, Research, MSD-Japan

Ann Mills-Duggan, PhD

Ken Duncan, PhD

Head, Seeding Drug Discovery FundBusiness Development, Innovations
Wellcome

Gerd Michel, PhD

Chief Scientific Officer
Vela Diagnostics

Deputy Director
Discovery & Translational Sciences
Bill & Melinda Gates Foundation

Ralf Clemens, MD, PhD

Independent Vaccine Expert

Ken Ishii, MD, PhD

Professor, Institute of Medical Science
University of Tokyo
Professor, the Laboratory of Vaccine Scienceat the Immunology Frontier Research Center(IFReC), Osaka University

Naoto Uemura, MD, PhD

Professor, Department of Clinical Pharmacology
and Therapeutics
Oita University Faculty of Medicine

Members provide strategic advice to the Board of Directors and to the Leadership Team.

Harvey V. Fineberg, MD, PhD

President, Gordon and Betty Moore Foundation
Former President
Institute of Medicine of the National Academies

Kumi Sato

President and CEO
Cosmo Public Relations Corporation

Dai Hozumi, MD, MSM, MPH
Chief Technical Officer
VP for Center for Technical Excellence
IntraHealth International

Michael R. Reich, PhD

Taro Takemi Professor
International Health Policy
Harvard School of Public HealthLorenzo Savioli, MD, DTM&H, MSc

Former Director, Department of
Neglected Tropical Diseases
World Health Organization

Leadership Team is responsible for the design and development of business and investment strategies and, upon Board approval, the execution of strategies, administrative operations,and organizational growth of GHIT.

BT Slingsby, MD, PhD, MPH

CEO*Through March 31, 2019

Masayuki Sato, MBA

Vice President, External Engagement

Director, Finance

Catherine K. Ohura, MS, PMPCEO*From April 1, 2019

Chief Operating Officer

Senior Director, Investment Strategy& Government Relations

Senior Director, Brand Communications

Hayato Urabe, PhD, MPIA
Director, Investment Strategy
Planning & Management

As of March 31, 201924

LEADERSHIP
EXTERNAL REVIEWERS
The work of the GHIT community could not progress without vital support from these experts and their institutions.

25

Richard Adegbola

Takashi Fujitsu

Dennis Kyle

Judy Sakanari

Yukihiro Akeda

Tamio Fujiwara

Nancy Le Cam Bouveret

Dirk Schnappinger

Pedro Alonso

Nisha Garg

James LeDucAmi Shah Brown

Peter Andersen

Ricardo Gazzinelli

Carole Long

George Siber

Rip Ballou

Birgitte Giersing

Timothy Lu

KJ Singh

Lewellys Barker

Ann Ginsberg

John Mansfield

Peter Smith

Michael Barrett

Daniel Goldberg

James McCarthy

Lynn Soong

Clif Barry

Glenda Gray

Joseph McCune

Gerald SpaethDavid Bell

Brian Greenwood

James McKerrow

Nathalie Strub-Wourgaft

Marleen Boelaert

Sanjay Gurunathan

Carl Mendel

Yasuhiko Suzuki

Maria Elena Bottazzi

R. Kiplin Guy

Charles Mgone

Marcel Tanner

Tom Brewer

Lee HallGerd Michel

John Telford

Martin Brusdeilins

Yoshihisa Hashiguchi

Toshiyuki Miura

Tetsuya Teramoto

Nick Cammack

Thomas Hawn

Valerie Mizrahi

Kaoru Terashima

Simon Campbell

Chris HentschelKatsuhiko Mochizuki

Katsushi Tokunaga

Eric Chatelain

D. Gray Heppner

Kouichi Morita

Nadia TornieporthPhilip Cole

Philip Hill

Charles Mowbray

Bruno Travi

Stewart ColeToshihiro Horii

Peter Myler

Takafumi Tsuboi

Simon Croft

Sanjay Jain

Christian Ockenhouse

Moriya Tsuji

Roy Curtiss

Stephen Johnston

Tsuyoshi Ogiku

Mickey Urdea

Peter Dailey

Takushi Kaneko

Giuseppe Pantaleo

Stephen Ward

Julian Davies

Niranjan Kanesa-thasan

David Persing

Tim Wells

Christine Debouck

Shigeyuki Kano

Meg Phillips

Bruce Weniger

Thierry Diagana

Subhash Kapre

Punnee Pitisuttithum

George Whitesides

Thomas Dick

Paul KayeDavid Pompliano

Samuel Wickline

Carter Diggs

Naoto Keicho

Dominick Pucci

Judith Wilber

Boro Dropulic

David Kelso

Regina Rabinovich

Elizabeth Winzeler

Filip Dubovsky

Kent Kester

Rino Rappuoli

Dyann WirthDavid Edwards

Akinori Kimura

Zarifah Reed

Michael Witty

Sabine Ehrt

Sue Kinn

Rebecca Richards Kortum

Paul Wyatt

Hiroyoshi Endo

Harajeshwar Kohli

Paul Roepe

Kazuhisa Yoshimura

Alan Fairlamb

Somei Kojima

Polly Roy

Takeshi Yura

Hermann Feldmeier

Hidehito Kotani

Eric Rubin

Fidel Zavala

David Fidock

Peter KremsnerPeter Ruminski

Donato Zipeto

JoAnne Flynn

Sanjeev Krishna

Philip Russell

Michael Free

Michael KurillaDavid Sacks

FUNDING PARTNERS & SPONSORS
Full Partners

Associate Partners

Affiliate Partners

Sponsors

26

Ark Hills Sengokuyama Mori Tower 25F
1-9-10 Roppongi, Minato-ku
Tokyo 106-0032 Japan
TEL: +81-36441-2032 FAX: +81-36441-2031
www.ghitfund.org