GHIT Fund

ANNUAL R E POR T

2020

E

GHIT Fund

ANNUAL R E PORT

2020

CONTENTS

01

Message from CEO and Chair

03

GHIT Fund Portfolio

05

SILVAMPTM TBLAM & Pediatric PZQ

07Clinical Candidates

09

Invested Partnerships in FY 2020

13

Strategic Plan FY 2018-2022 Progress

17

Highlights in FY 202019

Farewell Message from Prof. Kiyoshi Kita,
stepping down from GHIT SC

21“Together” is all about

23

Finances

25

Leadership

27

Acknowledgment

29

02

Message from CEO and Chair

Resilience and Partnership
During an Unparalleled Year
Covid-19 has drawn unprecedented global attention to theThis year, GHIT invested USD 41.6 million in 22 innovativevalue of innovation for life-threatening infectious diseases.

projects, which further strengthens our pipeline and

The extraordinary speed of pandemic product development

provides new hope for patients and healthcare professionals

over the past year demonstrates the innovation and scale thatin low-and middle-income countries. We successfully

is possible when stakeholders are incentivized to collaborate

expanded our portfolio, especially innovative point-of-caremeaningfully, despite enormous challenges in their path. We

diagnostics tools. Even in this difficult time, 15 new

at GHIT are committed to leveraging the power of thispartners have joined us to accelerate R&D for neglected

potential for R&D for neglected diseases. Over the pastdiseases. As of March 2021, our aggregate investments toyear, our development partners across the globe havedate total USD 251 million in 101 projects. Our current

demonstrated incredible tenacity, creativity, and grit in theportfolio features 23 discovery projects, 22 preclinicalface of global challenges. Our employees, too, haveprojects, and 7 clinical trials including 2 diagnostics fieldreimagined collaboration and efficiency in a new remotevalidation studies are in our portfolio (p.5-6). The World

working environment, and we are proud of their unwavering

Health Organization (WHO)’s endorsement and/or approvalenthusiasm and dedication.

from a stringent regulatory authority of our most advanced
clinical candidates, including a urine-based rapid TB

We recognize that it is GHIT’s duty to double down on ourdiagnostic kit and a pediatric formulation of thecommitment to innovation in service to the communities

gold-standard drug for schistosomiasis, are in sight. At thethat grapple daily with the threat and consequences ofsame time, we are actively discussing access and delivery

neglected diseases, and which are experiencing even morestrategies for late-stage clinical candidates with our

strain as a result of the pandemic. We know our partners anddevelopment partners to ensure patients and healthcare

stakeholders share our steadfast commitment to continue toprofessionals have access to innovative tools (p.7-8).

be a source of hope, inspiration, and collaboration in thefight against neglected diseases.

Notably, SILVAMPTM TBLAM’s proprietary silver
amplification immunochromatography (a platform

R&D: Keeping our foot on the pedal

technology developed by Fujifilm), was repurposed for the

In the midst of the COVID-19 crisis, product development

development of an antigen test kit for SARS-CoV-2

for malaria, tuberculosis (TB), and neglected tropicaldiagnosis. While GHIT does not invest in productdiseases (NTDs) has remained our – and our partners’ –development for COVID-19, this is a remarkable example of

crucial priority. While we spent much of the year online, we

how GHIT-invested innovation can also be utilized beyond

sustained our performance and improved the way we work.

its initial critical scope - for other infectious diseases andWe hosted two online Selection Committee (SC) meetings

how peacetime investments in innovative technologies andto identify investment recommendations to the Board forscientific advancement prepares the globe against future

approval. Applicants joined SC members for remotethreats.

interviews across multiple time zones. External reviewers

03continued to tirelessly evaluate proposals, helping ensure

Access & Delivery: Knowledge products

our ability to invest in critical innovation without pause.

In 2020, our Uniting Efforts for Innovation, Access &We are grateful for everyone’s strong commitment andDelivery (Uniting Efforts) partnership with the Government

generous support and would like to emphasize that theyof Japan and United Nations Development Programme

made our continuous investment possible.

(UNDP)-led Access and Delivery Partnership (ADP)© Dominic Chavez/World Bank

published two reports (p.19-20). The first, “Landscape ofthe rigorous standards and due diligence that have guided

funding and financing opportunities for access and delivery

the SC for the past eight years. Please take a moment toof health technologies for neglected diseases” summarizes

reflect on his important legacy and read about his passion

existing strategies to fund and finance access and delivery

for global health in our interview with him (p.21-22).

of health technologies for malaria, TB, and NTDs,
opportunities to augment funding, and proposals to improve

Looking forward, with gratitude

investment efficiency. The second report is the

As we continue to mobilize our network, knowledge, and“GUIDANCE NOTE for the development of national

experience in ever more creative and flexible ways toinvestment cases for neglected diseases”, which inform

advance progress and ensure continuity, we are deeply

national governments consider on the value of developing

honored by the partnership, solidarity, and support of ourinvestment cases to improve access and delivery of healthstakeholders. We are more inspired than ever to push harder,

technologies for neglected diseases and support

further, and with compassion toward our common goals,

implementation of national programmes.

#Together.

Strategic Plan 2.0 and beyond
In the midst (third year) of our second five-year operational
plan (FY 2018-2022), we are steadily approaching our goalsof GHIT’s Strategic Plan 2.0. Despite the impact of the
COVID-19 pandemic, GHIT and our partners haveprogressed steadily through R&D milestones and arefocused on meeting our original targets (p.17-18). Beyond
our strategic targets, these efforts represent the foundation
for GHIT 3.0 (FY 2023-2027), which will enable thecandidates in our current portfolio to flourish further.
Professor. Kiyoshi Kita steps down from the SC
This year also marks the final year of Professor. Kiyoshi
Kita’s expert service to and leadership of GHIT’s SC. As

Catherine Kaseri Ohura

Hiroki Nakatanifounding chair, Dr. Kita played a major role in establishing

CEO and Executive Director

Board Chair & Representative Director

04

GHIT Fund Portfolio
Discovery
Drugs / Vaccines

Target Research

Diagnostics

Target ResearchPreclinical
Screening

Hit-to-Lead

Lead Optimization
Product Design

Chagas disease, Leishmaniasis

Buruli ulcer

Chagas disease, Leishmaniasis

Chagas disease

Chagas disease

NTDs

Leishmaniasis
Mycetoma

Malaria

Tuberculosis

93.2

USD

51.1
12.8
18.9

2013
05

29.7
46.0

32.4
63.7

98.6

2014

2015

2016

GHIT investment

76.0

200.0

56.8
131.9
2017

300.0
250.0

69.1
169.9

350.0

209.2

250.8

150.0
100.0
50.0

2018

2019

2020

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

0

Cumulative investments USD (in millions)

344 M

Co-investment

Please visit GHIT website to find out more about each project and partner’s innovations.
https://www.ghitfund.org/investment/portfolio

ClinicalPreclinicalDengue fever

Drug

VaccinePhase I

Phase II

Phase III

Registration

Product Development

Product Validation

Product Validation

Registration

Cutaneous leishmaniasis

Mycetoma

Schistosomiasis

I

IIb

Diagnostics

III

Schistosomiasis
Leishmaniasis

Lymphatic filariasis,Onchocerciasis

Soil-transmitted helminths

Ib

IIa

Investment Overview FY 2013-2020

DiseaseMalaria

Tuberculosis 11.2 %NTDs

Development
Stage

Intervention

44.1%
44.7%

101

Total invested
partnerships
52 Active investments
49 Completed investments

143

Drugs

66.7%

Discovery

16.7%

Vaccines

24.0%

Preclinical

58.4%

Total productdevelopment partners

Diagnostics

9.3%

Clinical

24.9%

52 Japanese partners
91 Non-Japanese partners

FY 2020

USD

41.6M

Total investments

22

Invested partnerships

5

New Japanese partners

10

New non-Japanese partners

06

SILVAMP

TM

TBLAM
Novel, urine-based, point-of-care test for TB diagnosis forpeople living with HIV in low-resource settings

Disease:Tuberculosis

Intervention:

Diagnostics

Development Stage:

Field Validation

Country:South Africa, Malawi, Zambia, Tanzania, Uganda,
Vietnam, Thailand

immonocompromized states. Despite thedisruptions to the field studies due to
COVID-19, this project is currently in thefield validation prospective study phase inSouth Africa, Malawi, Zambia, Tanzania,
Uganda, Vietnam and Thailand.
Additional studies being conducted byother partners such as Médecins Sans
Frontiers, McGill University, and KNCV,
are under way. More data is also expectedfrom multiple independent studies led by
Fujifilm with expected completion by
Q3-2021, with targeted WHO Guideline
Development Group (GDG) review and
TB infects 10 million people and causes almost 1.5 million

launch in early 2022. The prospective field studies aredeaths every year. Projections show that the COVID-19

critical in providing evidence on assay sensitivity andpandemic’s disruption of just three months of TB service in

specificity, as well as stability and ease-of-use in thehigh-burden countries will result in an additional 1.3 million

real-life context which is critical for the evaluation by the

new cases and more deaths every year for the next five years.

WHO GDG. WHO GDG endorsement is an important step

Of the 10 million estimated people with TB every year, almost

forward the technology’s inclusion in WHO and National

three million (30%) are missed and one of the reasons for thisTB diagnostic guidelines and to facilitate procurement.

is underdiagnosis. Current diagnostics rely on sputum, are

Preliminary analysis based on retrospective testing hastime-consuming, and require trained laboratory personnel.

shown that the assay has almost double the sensitivity ofMoreover, sputum samples are not always easy to get, forthe current LAM test available on the market. Although

example in such some patient groups like children and people

more research is still required, the test has also shown somewith severe HIV infection. In some cases of extra-pulmonary

promising results in HIV-positive people with higher CD4

TB, using sputum might be inappropriate. There are currently

counts, and even in some HIV-negative people, which

no reliable, accurate, point-of-care and rapid diagnostic tests

could revolutionise point-of-care diagnosis for TB.

(RDT) that can be used in remote settings. The only RDT
available has just 35% sensitivity and can only be used in a

Fujifilm and FIND, with GHIT support, have now started

small proportion of people who might benefit from such a test:

working on a comprehensive launch plan covering regulatory

HIV-positive, severely sick, and/or with very low CD4 cells.

process, manufacturing, procurement and supply. The
COVID-19 pandemic has caused disruptions to services and07

In response, Fujifilm and FIND have developed amodelling studies have shown that there will be at least 1.3

diagnostic assay with high sensitivity and specificity inmillion TB cases resulting from pandemic-related service

HIV-positive patients using Fujifilm’s proprietary silver

disruption increase the urgency to bring this technology toamplification technology to detect LAM

scale. To that end, Fujifilm has invested in technology transfer(Lipoarabinomannan) in urine. LAM is a glycolipid foundto a manufacturing plant in Vietnam that would facilitate

in mycobacterial cell wall and can be detected in urine involume manufacturing, which would significantly impact

patients with disseminated TB, particularly in severe

assay pricing and affordability.

Pediatric

PZQ
A new pediatric formulation of the gold-standard drug smaller in size, less bitter, and orally dispersible

Disease:Schistosomiasis

Intervention:

Pediatric Drug

Country:

Development Stage:

Phase III,
Registration, AccessCôte d’Ivoire, Kenya

Schistosomiasis is a parasitic disease affecting more than 200

the beginning of 2021, to assess and pilot different models of

million people worldwide every year, 28 million of themcare for treatment implementation. The goal: to identify

children. It is mostly prevalent in sub-Saharan Africa andapproaches for social mobilization and ensure wide

carries a significant public health burden and economic

acceptance and equitable access. The five-year program is

impact. Praziquantel is the current ‘standard of care’co-funded by the GHIT and the European & Developing

treatment. The drug is safe, effective, and available for adults

Countries Clinical Trials Partnership (EDCTP), eachand school-aged children. However, the current tablets are too

providing €2.1 million and €5.7 million respectively. A

big for smaller children, especially those under the age of 6

dosing and administration landscape analysis to assess(preschool age). Healthcare workers and caregivers must

community acceptability, operational aspects and practices ofoften crush the tablets, which is impractical in a mass drug

orally administered drugs in preschool-aged children, is also

administration (MDA) context. Crushing the tablets alsoplanned for 2021. Both the ADOPT program and the dosing

brings out the bitter taste of the medication making it

and administration landscape analysis will inform theunpalatable for preschool-aged children, leaving this very

development of WHO and national guidelines, as well as

vulnerable group of preschool-aged children untreated in

preparation for the registration and access and delivery scale

public health programs, primarily due to the lack of anup of the new pediatric medication in endemic countries.

appropriate, child-friendly formulation of the drug.
The Consortium has made significant progress on the launch
The Pediatric Praziquantel Consortium is developing apreparations, particularly with GHIT support for further

child-friendly formulation of the drug, adapted forrefinement of access and delivery strategies in preparationpreschool-age children up through the age of 6 years old. The

for launch. Merck KGaA, which will hold the marketing

newly created tablet using Astellas’s formulation technology

authorization for the pediatric praziquantel, is in advanced

is smaller, taste-masked, orally dispersible meaning it can bestages of discussions with a manufacturer in Africa for ataken with little or no water. The Consortium is conducting atechnology transfer deal that would ensure a sustainableclinical trial, now in Phase III, assessing the new formulation’s

procurement and supply system.

safety and effectiveness of the new formulation in Kenya andCôte d’Ivoire
The start of the COVID-19 pandemic broughtsignificant disruptions of the clinical studies,setting back the pivotal Phase III clinical studies
by several months and leading to the terminationof a supportive trial that was being set-up in
Zimbabwe. However, despite these challenges
the trial resumed and is expected to conclude in
Q3 2021, followed by EMA regulatory
assessment (Article 58) and WHO endorsement
anticipated by the end of 2023.
The Consortium has also launched an
Implementation Research Project, ADOPT, at
08

Clinical Candidates
From Phase I to Phase II

SJ733
Malaria
Drug
Development Stage: Phase IICountry: Peru
Disease:

SJ733 is a PfATP4 inhibitor that meets criteria for treatment of uncomplicated malaria.

Intervention:

Phase I human data shows an excellent safety profile and tolerability, good oral availability,
and moderate clearance. The project team has initiated an adaptive open label Phase IIa
study in Peru to examine the antimalarial efficacy, safety, and tolerability of SJ733 in adultpatients with uncomplicated P. vivax or P. falciparum blood-stage malaria monoinfection.
SJ733 will be administered orally once every day for three consecutive days, with orwithout a fixed dose of the pharmacoenhancer cobicistat. The beginning of recruitment wasdelayed due to the COVID-19 pandemic, but officially opened in March 2021 and the first
three patients enrolled have completed all doses. There are three cohorts with each cohort
containing two treatment arms, P. falciparum (a) and P. vivax (b) and they will enroll max
60 patients. Cohort progression will be managed independently for each treatment arm.
Interim analysis will determine whether the data for a given treatment arm meets the
success criteria, is inconclusive, or meets the failure criteria. Antimalarial efficacy will be
examined over the period of 42 days. Additional aims are to characterize the safety andpharmacokinetics of SJ733. The results of this trial will identify active, well-tolerated doses
for investigation in a larger Phase IIb clinical trial.

BK-SE36 / CpG & NPC-SE36 / CpG
MalariaIntervention: Vaccine
Development Stage: Phase Ib
Country: Burkina Faso
Disease:SE36 is a blood-stage malaria vaccine candidate that primarily targets young
children in endemic areas to reduce morbidity and mortality due to malaria. Though
in principle all stages of parasite development are potential vaccine targets, theblood stage is the one that causes the symptoms of malaria and its complications,and thus has a significant public health impact. Early-stage clinical trials havedemonstrated that the BK-SE36 vaccine (SE36 recombinant protein and aluminiumhydroxide gel) has acceptable reactogenicity, no unexpected safety signals and was
immunogenic. Vaccine responders experienced less malaria infection and havelower risk of developing clinical symptoms. A better immune response wasachieved in Japanese adults when CpG-ODN (K3) adjuvant was added to theformulation. A Phase Ib trial to test safety and immunogenicity of BK-SE36/CpG in
1 year-old children to adults in Burkina Faso has recently completed its Last
Subject Last Visit. Interim results indicate no unexpected safety concerns. Moving
forward, new lots of SE36 and CpG (renamed NPC-SE36/CpG) are being
manufactured for proof-of-concept studies. The aim is to show suitability of new
SE36 vaccine GMP lot for clinical trials and importation. Preparations are under
way for Phase IIb site selection and clinical trial design.

09

CpG-D35
Cutaneous leishmaniasis
Drug
Development Stage: Phase I
Country: UK, Turkey
Disease:Cutaneous leishmaniasis (CL), endemic in 87 countries worldwide, mainly affects poor

Intervention:

populations in developing countries. Approximately 0.6 to 1.2 million new CL cases are
estimated every year. While CL is not life-threatening, it is a disfiguring disease that results instigma and economic loss. Currently, there are no satisfactory treatments for any form of CL.
CpG-D35, as a combination therapy for the treatment of patients with CL, triggers the Toll-likereceptor (TLR) 9 expressed on plasmacytoid dendritic cells and thereby activates the innate andadaptive immune system of the host. Data generated to date support the hypothesis that
CpG-D35 alone or in combination with chemotherapy will reduce infection and accelerate thehealing of CL lesions, as demonstrated in preclinical studies with CpG-D35. This project aims
to; determine the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) after
single subcutaneous dose of CpG-D35 in healthy volunteers, compared to matching placebo(SAD study) and after multiple doses administered in subjects infected with L. major parasites(MAD study); refine the current active pharmaceutical ingredient (API) manufacturing process
to improve efficiency/overall quality of CpG-D35 substance; develop a more concentrated,affordable, and field-adapted subcutaneous dosage form for later-stage clinical trials; and further
optimize the CAL-1 potency assay used for CpG-D35 quality control and stability testing.

Vinicius Berger-DNDi

SCH-RDT
Schistosomiasis
The WHO’s 2021-2030 roadmap calls for the elimination of Schistosomiasis (SCH) as a public
Intervention: Diagnostics
health problem by 2030. In order to reach this target, the report explicitly highlights the importance
Development Stage: Product Developmentof developing necessary diagnostic tests, including standardized point-of-care diagnostics forCountry: Kenya, Philippines
Disease:effective MDA campaigns and continued micro-mapping. Current WHO guidelines for the diagnosis
of SCH recommend examination of stool and/or urine samples by microscopy, for the presence ofschistosome eggs. Whilst these tests are useful in settings with moderate- to high-intensity infections,they become futile in settings where prevalence and intensity are low, due to their poor sensitivity.
Sampling is repeated on multiple days and several slides are examined by trained microscopists,
rendering the methods time-consuming and challenging to deploy. To address this challenge, partners
are developing an easy-to-use, accurate and affordable SCH-RDT with a sensitivity comparable torepeated microscopy that detects the circulating anodic antigen (CAA), an antigen that is secreted
continuously by living schistosomes. The results of the test will be obtained within 20 minutes. This
project aims to; conduct field evaluations on semi-quantitative prototype SCH RDT; optimize the
RDT, design-lock and transfer to manufacturing; validate the performance of the RDT in thedetection of the major schistosome species, and determine its suitability as a replacement of current
microscopy-based diagnostic tests; and develop an access strategy for the SCH RDT. The program’s
R&D work is co-funded by Bill & Melinda Gates Foundation to further characterize and validate
CAA for s.haematobium and s.japonicum.

10E1224
Mycetoma
Drug
Development Stage: Phase IICountry: Sudan
Disease:

Mycetoma is a debilitating fungal or bacterial infection that leads to disfigurement,Intervention:

disability, and social stigma. The disease is mainly found in tropical and subtropicalregions of the world, including in Chad, India, Mali, Mauritania Mexico, Senegal, andSudan. However, there is extremely limited data on the prevalence and incidence of thedisease. Diagnosis requires a combination of clinical, imaging, histology, and molecular
testing (if available) procedures. Low- and middle- income countries (LMIC) often lackaccess to these diagnostic tools and/or the skilled expertise needed to conduct theprocedures. Additionally, no simple, rapid point-of-care diagnostic tools exist. Current
treatment for eumycetoma (fungal) is itraconazole which has a limited cure rate (26%),
long treatment duration, and is expensive for resource-limited settings. If not treated, in
extreme cases it can lead to amputation or death. Eisai and the Drugs for Neglected
Diseases initiative (DNDi) are conducting a Phase II trial to determine whether
Fosravuconazple (or E1224) , an azole-class antifungal drug discovered by Eisai (andalready approved for onychomycoses in Japan) may also be an effective and affordable
treatment for mycetoma. The trial is being conducted in Sudan with the WHO
Collaborating Center on Mycetoma in Khartoum. The study is on-going after a temporary
disruption due to the COVID-19 pandemic.

11

BENDITA Study
Chagas disease
Drug
Development Stage: Phase IICountry: Bolivia
Disease:The Lancet Infectious Diseases study shows that a two-week treatment forIntervention:

adults with chronic Chagas disease has similar efficacy and significantly
fewer adverse effects than the current standard eight-week treatment. This
study provides some hope for people affected by this neglected disease: ashorter treatment would remove some common caregiver and patient concerns,
as well as improve treatment adoption. These results are extracted from a
Phase II clinical trial that was carried out in three centers in Bolivia between
2016 and 2018. The GHIT-invested study was led by the DNDi in partnershipwith CEADES (Science and Applied Studies Foundation for Health andEnvironment Development), ISGlobal, Eisai and the Argentinian
pharmaceutical company Elea, among others. The BENDITA study(Benznidazole New Doses Improved Treatment & Therapeutic Associations)is the first placebo-controlled study to compare different durations and dosesof treatment with the standard treatment benznidazole, alone or in
combination with Fosravuconazole (or E1224) produced by Eisai.

“The BENDITA study provides hope for

Torrico F, Gascón J, Barreira F, Blum B,

people affected by this neglected

G, Correia E, Garcia W, Ortiz L, Parrado R,

Almeida IC, Alonso-Vega C, Barboza T, Bilbe

disease, as a shorter treatment will

Ramirez JC, Ribeiro I, Strub-Wourgaft N,

remove some concerns of caregivers

group. New regimens of benznidazole

Vaillant M, Sosa-Estani S; BENDITA study

and patients and will improvemonotherapy and in combination withtreatment adoption.”(BENDITA): a phase 2, double-blind,

Dr. Sergio Sosa Estani, Head of the Chagas ClinicalTHE

LANCET

Infectious Diseases

BENDITA study
Chagas disease

Global Healthfosravuconazole for treatment of Chagas disease
randomised trial. Lancet Infect Dis. 2021 Apr

Programme at DNDi and researcher at CONICET

6:S1473-3099(20)30844-6. doi:

(National Scientific and Technical Research

10.1016/S1473-3099(20)30844-6. Epub ahead ofCouncil) in Argentina

print. PMID: 33836161.

12Invested Partnerships in FY 2020
From Discovery to Preclinical

Towards the rapid diagnosis of malaria
hypnozoite infection: feasibility studies
Malaria / Diagnostics

Institute of Tropical Medicine, Nagasaki University (NU-ITM), National Institute of Technology,
Kumamoto College (NIT-KC), Biomedical Primate Research Centre (BPRC)

Target Research Platform

Screening Platform

Hit-to-Lead Platform

While clinical malaria cases in the Asia-Pacific and the Americas havegone down >90% in the last decade, a shift in malaria species composition
has been observed, with Plasmodium vivax now being the predominant
species outside Africa. This shift may relate to the unique biology of P.
vivax, including the relapsing phenotype from dormant liver stages.
Asymptomatic hypnozoite infections form a hidden parasite reservoir in
the human population that can give rise to new symptomatic andtransmissible malaria weeks, months or years after primary infection,
without new infection through mosquito bites. Proper diagnostic tools to
identify hypnozoite-infected individuals are currently lacking. Under

Product Development Platform

Prof. Osamu Kaneko
Nagasaki University

previous research, they pioneered an in vitro POC towards identifying
targets for diagnostic tools for malaria hypnozoites, exploiting our uniqueexperience in in vitro P. cynomolgi hypnozoite cultures, as well as insensitive metabolomics. Specific metabolites have been identified andprioritized based on the unique signatures found in hypnozoite-enriched
cultures. A second-phase in vivo feasibility study using the P.
cynomolgi-rhesus monkey model is warranted to determine whether the
specific signatures detected in the in vitro POC are confirmed in vivo andcan thus be pursued in the subsequent development phase of a RDT for
hypnozoite infection.

Evaluation of a malaria vaccine candidate comprisedof full-length recombinant CSP formulated with
SA-1 adjuvant, using RTS,S/AS01 as a benchmark
Malaria / Vaccine

PATH, Sumitomo Dainippon Pharma Co., Ltd., Ehime University

Target Research Platform

Screening Platform

Hit-to-Lead Platform

The world’s first malaria vaccine, RTS,S/AS01, is a recombinant VLP
containing truncated CSP as the target antigen and formulated with a
TLR 4/QS21-containing adjuvant (AS01). In a large multi-center Phase
III trial RTS,S/AS01 demonstrated a near 40% reduction in clinicalmalaria incidence in young African children, over four years offollow-up, and is currently undergoing pilot implementation in three
African countries. A next generation malaria vaccine with improved
efficacy and durability offers the potential for even greater public healthimpact. The project aims to rigorously test a novel vaccine candidate,flCSP/SA-1, comprised of a full-length recombinant CSP adjuvanted

Product Development Platform

Dr. C. Richter King
PATH

with a novel TLR7 agonist, SA-1, for superiority to the RTS,S/AS01
benchmark vaccine, to inform whether to advance flCSP/SA-1 topreclinical development. The team will conduct two tests: first, how
flCSP/SA-1, compares to flCSP/AS01, for induction of improved
durability of antibody responses in non-human primate (NHP) model.
NHPs are used as the test species because rodents do not adequately
model the rapid decline of antibody titer observed with RTS,S/AS01 in
humans. Second, testing will determine how the flCSP antigen
compares to RTS,S, for induction of stronger protective efficacy in wellqualified rodent challenge model.

Pioneering antisense oligonucleotides as
long-acting malaria prophylactics
Malaria / Drug

Eisai Co., Ltd., University of California, San Diego (UCSD)

Target Research Platform

Screening Platform

Hit-to-Lead Platform

Development of effective new liver-stage prophylactic agents is apriority for the antimalarial field. Antisense oligonucleotides(ASOs) are well-matched to this unmet need, offering thepossibility of long-duration activity and benefiting from effective
delivery to hepatocytes using well-established conjugation
technology. Furthermore, ASOs are a platform technology thatenable highly selective targeting of essential Plasmodium genes,
with the potential to access previously undruggable targets andaccelerate development of additional drugs following initial
validation. Eisai has developed proprietary nucleic acid13

Product Development Platform

Dr. Elizabeth Winzeler
University of California, San Diegotechnologies that enhance these advantageous properties. This
project will undertake a rigorous investigation as a novel
antimalarial strategy, with the goal of demonstrating in vivo
proof-of-concept for ASOs as long-acting malaria prophylactics.
Well-validated malaria target genes will be examined for
tractability, and ASOs against the selected target will be
optimized using high-throughput cell culture assays at UCSD andEisai’s nucleic acid technologies. High-potency optimized ASOs
will be tested in a causal prophylaxis animal model to assess invivo efficacy.

Prolyl tRNA Synthetase Inhibitors
for New Antimalarials
Malaria / Drug

Takeda Pharmaceutical Company Limited, Medicines for Malaria Venture (MMV)

Target Research Platform

Screening Platform

Hit-to-Lead Platform

Takeda and MMV are working on prolyl tRNA Synthetase (PRS)
Inhibitors with the aim of identifying a potential new antimalarial
drug. This PRS chemical series was directly repurposed from theTakeda portfolio. At the beginning of the collaboration between
Takeda and MMV, screenings were performed at MMV testing
centers against the liver, blood asexual and sexual stages of themalaria parasite. Data showed that the Takeda PRS chemical
series has activity against both the asexual blood and liver stagesof the Plasmodium lifecycle. The main objective of the project is

Product Development Platform

Dr. Atsuko Ochida
Takedato transform PRS Inhibitors into lead series with proven in vivo
efficacy in relevant animal disease models so as to identify atleast one compound as an early lead molecule that meets the
GHIT/MMV criteria for progression to lead optimization stage
for prophylaxis. In this new phase the project team will perform
medicinal chemistry activity to optimise the PRS chemical seriesin terms of drug metabolism and pharmacokinetics (DMPK) andphysicochemical properties.

Hit-to-lead development of new antimalarial
compounds from DDI library
Malaria / Drug

The University of Tokyo, MMV

Target Research Platform

Screening Platform

Hit-to-Lead Platform

The University of Tokyo and MMV collaborate to generate
structurally optimized lead compounds effective against malaria
parasites, based on the hit compounds they identified with GHIT
support during 2018-2020. In the preceding project, the team identified
a number of hits that kill the malaria parasites under micromolar
concentrations, by phenotypic screening of 210,000 structurally
defined compounds from the Drug Discovery Initiative, Japan. In thepresent project, partners will conduct structural optimization of theselected six series to develop new antimalarial leads. The project will

Product Development Platform

Prof. Tomoyoshi Nozaki
University of Tokyo

generate a few series of new compounds that kill malaria parasites bystructural modifications of the initial hit series. The new compounds
will be further tested for efficacy against both drug-sensitive andresistant malaria strains, as well as for in vitro safety. It will be also
elucidated how the new compounds kill the parasites. Because malaria
parasites are transmitted between humans and mosquitoes, the teamwill also evaluate what developmental stages of parasites the newcompounds are efficacious to, e.g., parasites in the human liver,erythrocytes, mosquito gut, and the salivary gland.

First-In-Human Trial of the Pan-Malaria
Transmission-Blocking Vaccine AnAPN1
Malaria / Vaccine

Ajinomoto Bio-Pharma Services, GeneDesign (GeneDesign), CellFree Sciences Co. Ltd., Centre deRecherches Médicales de Lambaréné (CERMEL) , University of Tübingen (UKT) , The University of Florida

Target Research Platform

Screening Platform

Hit-to-Lead Platform

The anopheline mosquito midgut-specific alanyl aminopeptidase N(AnAPN1) is a luminal midgut surface protein involved in blood meal
digestion. At present, AnAPN1 is the only transmission-blocking vaccine(TBV) candidate, which blocks parasite transmission of P. falciparum andP. vivax in different Anopheles species. Working with a mosquito protein
further reduces the risk that the parasite could develop resistance against
the intervention, potentially allowing for a long-term use of the vaccine
under elimination settings. AnAPN1 has been studied extensively intransmission-blocking experiments, where the protein induced very hightiters in immunized animals. Because any TBV will require very highantibody titers within vaccinated individuals to be effective, development

Product Development Platform

Dr. Rhoel Dinglasan
University of Florida

of an optimized antigen to ensure transmission-blocking activity isessential. The optimized AnAPN1 immunogen, UF6b, has no purification
tags, and when formulated with the GLA-LSQ adjuvant elicits potent
transmission-blocking activity in mice and NHPs against natural P.
falciparum strains. Partners envision that during the two-stage project they
will complete preclinical manufacturing of UF6b and toxicology testing toobtain ethical approval for use of the vaccine and then enter directly into
Phase IA/B clinical trials in Gabon. At the end points of the clinical trial,safety and dose will be confirmed. Antibodies obtained from immunizedindividuals will be fully evaluated using a set of functional,
immunological, and biological assays.
14

Tuberculosis /Diagnostics

Product Development of LFA platform for
improving sensitivity of Point-of-Care assays for
infectious disease with main focus on Tuberculosis
Asahi Kasei Corporation, Biopromic AB

Target Research Platform

Screening Platform

Hit-to-Lead Platform

The only available non-sputum TB test in the market has
insufficient sensitivity to be used as a reliable diagnostics,
especially in the non-HIV-infected TB population. The RDT
co-developed by Asahi Kasei Corporation and Biopromic detects
picogram amounts of the LAM TB biomarker in the TB patienturine, irrespective of HIV infection, in timely manner with highaccuracy. The Asahi Kasei´s and Biopromic´s RDT test applies anovel proprietary technology regarding sample preparation andlabeling particles that allows LAM TB biomarker detection at

Product Development Platform

Dr. Beston Hamasur
Biopromic AB

lower picogram level, which is 100 times higher than the only test
available in the market so far. The main objection is to validate
the RDT which is currently developed by Asahi Kasei andBiopromic for diagnosis of tuberculosis in TB patients
irrespective of HIV status and ensure that it meets WHO TPP
criteria. Asahi Kasei´s and Biopromic´s RDT test will be
evaluated by analysis of more than 1000 well-characterized
clinical samples collected from South Africa and Uganda to
determine test accuracy.

Development of ‘all-in-one’ diagnostic kit for Buruli
ulcer using lateral flow DNA-chromatography
Buruli ulcer /Diagnostics

Teikyo University, Keio University School of Medicine, Tohoku Bio-Array (TBA) Co., Ltd,
Nagasaki University, FASMAC CO., LTD, Raoul Follereau Institute Côte d’Ivoire,
Pasteur Institute Côte d’Ivoire, Hope Commission International

Target Research Platform

Screening Platform

Hit-to-Lead Platform

No point-of-care diagnostic method for the tropical NTD Buruli
ulcer (BU), which results in delayed diagnosis and treatment.
Many patients must live with serious sequelae. In addition to
insufficient diagnostic resources, the low number of new BU
cases reported to the WHO (about 2,000 per year) is due to thefact that the disease is not widely known and often goesunrecognized. In many cases patients are left without proper
diagnosis, and in some cases inappropriate treatment. PCR,
currently the only diagnostic method recommended by WHO,

Product Development Platform

Prof. Koichi Suzuki
Teikyo University

requires a specialized laboratory with expensive equipment andtechnologists with a high level of knowledge and skills(unrealistic in many LMIC settings). This project aims to develop
the first point-of-care diagnostic kit for BU by combining
techniques for stably fixing and drying reagents, performing DNA
amplification without a special device, and DNA chromatographythat allows easy visual detection of its detection. The method is
simple and requires no special equipment or technology, and the
kit can be stored at room temperature for a long period of time.

Target validation and AI-guided identification of
trypanosoma cruzi phosphodiesterase inhibitors
for the treatment of Chagas disease.
Chagas disease / Drug

Eisai Co., Ltd.,Universidad Nacional de La Plata (UNLP)

Target Research Platform

Screening Platform

Hit-to-Lead Platform

There is an urgent need for new treatments for Chagas disease.
Existing medications lack effectiveness against chronic infection,require long regimens, and have several adverse effects. Given theirintegral roles in trypanosome signaling and low homology with humancounterparts, phosphodiesterases (PDEs) have been posited as drug
targets for Chagas disease. Given the paucity of identified targets andcritical need for new mechanism-of-action drugs, these enzymes meritdefinitive evaluation followed by efficient identification anddevelopment of inhibitors. This project aims to validate PDEs as drug

15

Product Development Platform

Dr. Fabian Gusovsky
Eisaitargets for Chagas disease using CRISPR/Cas9 technology and identify
selective inhibitors using a computationally-enhanced screening
cascade. Candidate inhibitors identified in machine-learning-based
virtual screens will be profiled experimentally, with promising
compounds advanced to animal studies. An accelerated drug
development path will be sought by focusing on repurposing
opportunities that can be rapidly progressed to clinical trials,
complemented by screening for new chemical matter from Eisai’s
compound library.

Cry5B optimization for Trichuris - whipworm
Soil-transmitted
helminth / Drug

Kao Corporation, PATH, University of Massachusetts Medical School

Target Research Platform

Screening Platform

Hit-to-Lead Platform

The last new drug to treat soil-transmitted helminth (STH)
infections was introduced more than 30 years ago. Increasing
resistance threatens the efficacy of the drugs most widely used in
MDA campaigns against STH infections and has already renderedsome nicotinic agents, such as levamisole, largely ineffective in thetreatment of livestock with parasitic nematodes. A significant gap
in the profile of all of the currently approved anthelmintic drugs isthat they are contraindicated for women in their first trimester ofpregnancy, which leaves the mother and her fetus vulnerable to theeffects of STH-induced anemia and malnutrition, and out of reach

Product Development Platform

Dr. Manjari Lal
PATHof a therapeutic intervention. As a non-absorbed therapeutic agent
that binds to a target known only in invertebrates, Cry5B is
expected to have an excellent safety profile for all patients,
including pregnant women and young children. Cry5B would be an
inexpensive and potent addition to the therapeutic options for
managing STH infections and can form part of a combination
therapy with existing drugs. It acts synergistically with some
nicotinic agents, and resistance to these can make STH
hypersensitive to Cry5B.

Hit-to-Lead development for NTDs
Chagas disease, Visceralleishmaniasis / Drug

Mitsubishi Tanabe Pharma Corporation, DNDi

Target Research Platform

Screening Platform

Hit-to-Lead Platform

Drug discovery and development at the early hit to lead stage for
Chagas disease and visceral leishmaniasis (VL) is prone to a
relatively high chance of failure. The objective of this project is to
identify at least one series that meets DNDi lead stage criteria for
Chagas disease and/or visceral leishmaniasis in line with DNDi
published Target Product Profiles (TPPs) for new chemical entities.
A critical mass of novel chemical series therefore needs to be
identified and progressed beyond Hit-to-Lead to build up a solid
portfolio of preclinical candidates aligned with the DNDi TPPs for
Chagas disease and VL. Mitsubishi Tanabe and DNDi have been

Product Development Platform

Dr. Jean‐Robert Ioset
DNDi

closely collaborating since September 2019 on a GHIT-funded
screening project, which has successfully identified nine series
mainly active against T. cruzi although a couple of them display dual
T. cruzi and Leishmania donovani activities. Three T. cruzi active
series have been prioritized. All series are novel to DNDi discovery
programs and satisfy all criteria set by the GHIT and DNDi,
resulting in successful progression into the Hit-to-Lead stage.
Medicinal chemistry efforts will be carried out in priority on thethree most promising series.

Lead optimization and preclinical candidate selection
from the NTD Drug Discovery Booster series S07 for VL
Visceral leishmaniasis/ DrugTakeda Pharmaceutical Company Ltd., DNDi

Target Research Platform

Screening Platform

Hit-to-Lead Platform

This project originates from a successful collaboration between
Takeda and DNDi through the GHIT-funded NTD Drug
Discovery Booster program, whose unique approach toidentification and progression of novel compounds as inhibitorsof protozoan parasites has ultimately led to the development ofcompounds with candidate-level efficacy in animal models of VL.
Lead compounds in the series S07 with good efficacy and safety
profiling have been identified and progressed in partnership with
Takeda. Medicinal chemistry optimization around the identified
leads and further development plans have been prepared to

Product Development Platform

Dr. Eric Chatelain
DNDi

support progression of at least one optimized lead to candidate
status. The S07 series is synthetically tractable, thestructure-activity- relationship is well-understood and extensivein vitro safety profiling has not highlighted any issues to date.
The overall objective is to expand current selection of compounds
meeting candidate level efficacy, investigate and determine
PK/PD drivers for the S07 series, support compound developmentwith preliminary CMC activities, and nominate one compound forpreclinical development IND enabling studies.

16

Strategic PlanFY 2018-2022 Progress
R&D: Investments in Product Development
Further leverage innovations from Japanese biopharma and academia
Diagnostics# targetedby March2023# achievedby March2021

5

Preclinical

Lead Identification

5ed
Ach iev

8ed
Ach ieved
Ach iev

Diagnostics identified
and funded

Hit-to-lead programs

Preclinical candidates
identified

7

10

9

15

# of potential
candidates

Candidates in target researchand hit-identification stage

8

Candidates inlead optimization stage

Our actions in FY 2020 to advance innovations
NTD scope expansion● Expanded in-scope NTDs from 10 to 20 (all WHO-identified NTDs) in GHIT Product Development Platform● Recruited and onboarded 30 new external reviewers (currently 150 in total)

Partnership formation for new innovations● Catalyzed partnerships between Japanese and non-Japanese entities through multiple consultations● Conducted a proposal writing seminar and engaged in various scientific conferences

Proactive portfolio management● Monitored progress and identified risks for each project through semiannual progress report call

¥ $
€ £
17

Supplemental budget injection● Provided supplemental emergency COVID-19 budget support for several projects

In GHIT 2.0’s third year, GHIT, we and our product development partners haveprogressed steadily toward key R&D milestones, despite the far-reaching impact of thepandemic. Additionally, we continued to identify and fund projects aligned withstrategic goals without disruption, as summarized below.

Advance R&D investment pipeline and prioritize late-stage candidates
Phase I

Phase II 〜 Phase III

Registration

5

3

2

First in Humanclinical trial conducted

Proof of Concept achieved

Approved by stringent
regulatory authority

1

1

1

13

6

Candidates inpreclinical stage

Candidates inPhase I or Phase II

3

Candidates inPhase II or Phase III
As of March 31, 2021

“Regular and ad-hoc calls with our development partners are the most important of ourportfolio-management activities. They help us review our strategy and take the best measures toensure we achieve R&D milestones together.”

Maki Kanesaka
Associate Director, Investment Strategy, Portfolio Development and Innovations“Exploring untapped technologies, connecting R&D partners, and catalyzing new innovations forneglected patients are our fundamental roles in service to our strategic plan. This goes
hand-in-hand with strengthening the global health R&D ecosystem’s R&D pipeline.”

Eriko Koyama
Manager, Investment Strategy & Business Development
18

Highlights in FY 2020
Ensuring continuity and excellence
GHIT’s first-ever virtual Selection Committee(SC) meeting took place in June 2020 due totravel restrictions during the COVID-19
pandemic. Incorporating the experiences andlessons learned in June, the second SC meeting
took place in January 2021. SC members andinvited applicants joined from over 20 countries
across multiple time zones for interactive,
in-depth interviews to inform SC investment
recommendations to the Board. Improved
operational efficiency made our meetings
successful, embracing a new form of humanconnection while ensuring adherence toappropriate investment processes.

NTD webinar series2020 marked the launch of the WHO NTD
Roadmap 2030, which will serve as a key
guiding document for the global response to
NTDs over the next decade. In response to
the global movement, GHIT partnered with
the Japan Alliance on Global Neglected
Tropical Diseases (JAGntd) to host a
webinar series on NTDs focused on global
trends and Japan’s experience. During thesewebinars, Japanese experts on NTDs
presented their programs and activities anddiscussed challenges and opportunities
associated with the aim to eliminate NTDs
by 2030. All archived webinars are available
on JAGntd’s website.
https://jagntd.org/ntdwebinar2020

19

Virtual proposal
writing seminar
Our July 2020 online proposal writing
seminar helped future development
partners deepen their understanding of our
proposal process and investment
platforms. Participants from privatecompanies, academia, and research
institutions discussed the critical points tobe considered and/or addressed whencreating a proposal, with a special focus on
GHIT’s Target Research Platform and
Product Development Platform. Past
proposal writing seminar overviews areavailable on our website.
https://www.ghitfund.org/newsroom/films/en

Publications
Uniting Efforts
knowledge products

Scientific article
archive

Publications with
GHIT contributions

The Uniting Efforts partnership between
GHIT, the Government of Japan, and theUNDP-led ADP published two reports thisyear. “Landscape of funding and financing
opportunities for access and delivery ofhealth technologies for neglecteddiseases”, and “GUIDANCE NOTE forthe development of national investment
cases for neglected diseases” were
designed to support national governmentsin their efforts to improve access anddelivery of health technologies forneglected diseases and the implementationof national programs.

More than 40 scientific articles
have been published over the past
eight years as a result of
GHIT-funded research andclinical studies. Now available on
our website, this growing archiveof articles demonstrates ourpartners’ important scientificaccomplishments whileillustrating the progress of
GHIT’s investments. Articles areauthored by GHIT’s productdevelopment partners and refer toprojects that have been fully or
partially funded by GHIT.

Hooft van Huijsduijnen R, Kojima S, Carter D,
Okabe H, Sato A, Akahata W, Wells TNC,
Katsuno K. Reassessing therapeutic antibodies
for neglected and tropical diseases. PLoS Negl
Trop Dis. 2020 Jan 30;14(1):e0007860. doi:
10.1371/journal.pntd.0007860. PMID:
31999695; PMCID: PMC6991954.
https://journals.plos.org/plosntds/article?id=10.1
371/journal.pntd.0007860

https://www.unitingeffortsforhealth.org/resources

https://www.ghitfund.org/newsroom/
scientificarticles/jp

Katsuno K. Japan’s innovation for global health- GHIT’s catalytic role. Parasitol Int. 2021
Feb;80:102232. doi:
10.1016/j.parint.2020.102232. Epub 2020 Nov
2. PMID: 33144196.
https://www.sciencedirect.com/science/article/pi
i/S1383576920301823
Nakatani H, Katsuno K, Urabe H. Global healthlandscape challenges triggered by COVID-19.
Inflamm Regen. 2020 Sep 15;40:34. doi:
10.1186/s41232-020-00144-5. PMID:
32939226; PMCID: PMC7491013.
https://inflammregen.biomedcentral.com/articles/10.1186/s41232-020-00144-5

Promoting DEI
During a year of change and challenge, our commitment to diversity, equity, and inclusion(DEI) remained steadfast. We endorse Global Health 50/50’s evidence-based approach toadvance action and accountability for gender equality and DEI in global health and arehonored by GHIT’s inclusion in its annual Index. This initiative helps us improve ourpolicies, programs, and workplace. https://globalhealth5050.org/2021-report/

Respond to COVID-19
While we do not invest directly in product development for COVID-19,
GHIT served as a mediator, connecting our development partners whose
technologies and innovations have potential in the fight against the
pandemic with other funding agencies, resulting in some awarded
projects. In addition, TBLAM’s proprietary silver amplification
immunochromatography (a platform technology developed by Fujifilm
and funded by GHIT), was repurposed for the development of an antigen test kit for SARS-CoV-2 diagnosis. This exemplifies
how GHIT-invested innovation can also be utilized beyond its initial critical scope for other infectious diseases. Lastly, GHIT
joined the Access to COVID-19 Tools (ACT) Accelerator workstream to share our knowledge and perspective.

20

Farewell Message fromProf. Kiyoshi Kita,
stepping down from GHIT SC
Professor Kiyoshi Kita, inaugural chair and member of the GHIT Selection
Committee (SC) since its inception in 2013, retired from the Committee
following its January 2021 meeting. The GHIT staff members spoke withhim about his unique support to and reflections on GHIT’s evolution.
Together, we recognize his countless contributions to the institution and tostrengthening the relationship between the GHIT and Japanese academia.

Katsuno: You led the SC for eight

attributed to our constant recruitment ofyears, starting from GHIT’s

highly qualified experts. Personally, I

establishment. What stands out to you

think that GHIT is essentially aabout the beginning of the process?

Japanese entity, but it requires
international R&D collaboration,

When I heard GHIT was being

therefore it would be desirable to haveestablished, I had been researchingsomeone with a wealth of experience inseveral infectious diseases, and I

both Japan and overseas take the lead.

thought it would be a good opportunityand sincere about science. SC members

In this regard, I am very excited thatto get research grants. However, whenin the early days were a small group; we

Professor Naoto Uemura (Oita

officials from the Ministry of Health,recognized our strong need for more

University) has taken over as SC chair.

Labor and Welfare and the Ministry ofdiverse and objective opinions to

I believe this will lead to a further leap

Foreign Affairs came to see me andevaluate proposals fairly. Later, we

forward for GHIT. It is also vital for us

asked me to be the chair of the SC, it

introduced the “external reviewer”

to keep an eye open to find future

was the first time I recognized GHIT’s

system in which experts in productpotential SC members.

fund size. I appreciated that a fair anddevelopment and infectious diseases

equitable selection process wasfrom Japan and abroad peer-review

Urabe: How did the Japanese academic

essential to expedite good productproposals before the SC evaluates them.

community initially respond whendevelopment. When I received an offer

The objective comments of the external

GHIT was first established?

to lead that process I realized that myreviewers inform SC members and help

contribution to global health R&D

them make fairer decisions. We are

At that time, Japan’s infectious diseases

would not just be to do my own

sincerely grateful for the expertise andcommunity was brimming with highly

research, but also to advance productcommitment of more than 100 GHIT

motivated young researchers with adevelopment through helping GHIT

external reviewers.

hungry spirit. That hunger forinvest in good projects.
Katsuno: What do you see as the key
Katsuno: How would you summarize

characteristics and expertise of SC

the SC’s evolution over the past eight

members?

years?
In terms of experience and expertise, I

21

GHIT’s SC members have a wealth ofbelieve that we have a diverse andexperience in R&D and are very strict

almost ideal composition, which can beknowledge and discovery, for example

partnerships between Japan and global

Furthermore, I believe there are manyin the field of parasitology, only

entities. In the past, there have been amore things that GHIT can support

increased when GHIT was established.

number of projects that might have been

beyond its existing investment projects.

GHIT’s creation provided a tailwind forselected if the interview with SC

For example, why not support young

academia; in fact many researchers

members in English had been wellresearchers who may not have enoughhave submitted applications to GHIT,

managed. I know it is hard for Japanesedata but have great ideas? Additionally,

and the number of proposals actually

researchers who are not used to it, but I

supporting start-up projects could leadincreased. In order to further strengthen

hope that they will be able to overcome

to applications to the GHIT Fund in theits partnership with academia, however,their language challenges. I wish for

future. This will be good training for

GHIT needs to build more awareness of

their global success. I strongly hope that

researchers to prepare for the real

what it does and what it has to offer.

GHIT continues to encourage

application.

For example, at a symposium co-hosted

passionate researchers and help

by the University of Tokyo, MMV, andJapanese technologies can be utilized

Urabe: What message do you have for

GHIT in 2015, Nobel Laureate Satoshi

for global health.

researchers overseas and in Japan?

Omura, Distinguished Emeritus
Professor of Kitasato University, gave a

What I would like to tell overseas

keynote speech. This was well received

researchers is that Japanese researchers

by experts in Japan and abroad.

are resilient; they do not give up easily

Although it is difficult to hold such aand could be good partners. I would

large-scale event now due toalso like to see more international and

COVID-19, I hope that GHIT will

domestic acceptance of and intentional

continue to provide opportunities forcollaboration with young Japanese

researchers to convene.

Ohata: What are your hopes for the

researchers.

GHIT’s future?
Ohata: Have you seen any changes

For Japanese researchers, especiallyamong the mindsets or priorities of

After working with all of you on a daily

those who will lead the next generation:

Japanese researchers over the years?

basis, I strongly feel that GHIT as analways give 100% in your research

institution and a team of talented

field. Research ranges from usefulness

I think they have made tremendous

individuals is highly motivated andto the world and people to the research

progress. In the early days, proposals

sincere about the goals it has set. I

for the truth. Strive to find your originalfrom organizations with Japanesebelieve that you will be able to continue

research that only you can do, no matter

principal investigators were often

working at this speed and further

where you are. Good research will

inadequate in terms of explanation andstrengthen collaborations with various

surely contribute to society and people

content in English. However, over thepartners to achieve your goals.

someday. I hope that you will keep inpast years I feel that the proposals have

Innovative partnerships have been mademind that only 1% of your new

become more and more robust, and thethrough GHIT. Please continue

discoveries will be for your own

gap between the level GHIT is looking

match-making good R&D partners andenjoyment, and the remaining 99% will

for and the proposals submitted byprojects.

be for the benefit of those in need.

applicants is closing. The result has
been increasingly strong proposals anda more competitive selection process.
In addition, the number of young
researchers with a global mindset has
increased, and their success has been
truly remarkable. I believe this can be
attributed to GHIT’s investment
prerequisite that projects must be

From left to right: Dr. Hayato Urabe (Senior Director, Investment Strategy, Portfolio Development & Innovations), Prof.
Kiyoshi Kita (Dean, the Graduate School of Tropical Medicine and Global Health, Nagasaki University), Mina Ohata(Manager, Investment Strategy, Portfolio Development & Innovations), Dr. Kei Katsuno (Senior Director, Investment
Strategy, Business Development)

22

“Together” is all about

GHIT’sCultureand Future

From left to right: Daisuke Imoto (Vice President, External Affairs & Corporate
Development), Nana Arai (Manager, Brand Communications), Ayaharu Moriwaki(Associate Director, HR & Office Management)

In FY2020, the COVID-19 pandemic transformed the world and disrupted organizations in every industry. GHIT, now in its
eighth year of operation, also experienced many changes. The pandemic significantly impacted our investment projects andshifted our working style. Three GHIT staff members reflected on GHIT’s evolution during the pandemic and discussed ourorganizational strategy, human resource (HR) strategy, culture, and hoped-for future.

Advancing innovation,
Together-and our stakeholders in overcoming

which organizations can create

this crisis and continuing to movesynergies with GHIT and find new

forward to achieve our mission ofopportunities for collaboration.

Imoto: Eight years have passed sincecreating accessible products for

GHIT was established, and we arepeople in need.

currently working on about 50 R&D

Government of Japan and the

projects, including several late-stage

Arai: In order toUNDP-led ADP, launched “Uniting

candidates which are approachingcreate new innovations

Efforts for Innovation, Access andlaunch. We are excited to show

and advance product

Delivery” which aims to build aresults. We live and breathe “opendevelopment, it is important to step

global platform bridging from drugs,

innovation” at GHIT and are strongly

outside of existing boundaries andvaccines, diagnostics, other novel

driven to further strengthencommunities, collaborate with people

medical technologies, and innovations

collaboration with diverse

who have never been involved before,that address unmet health needs instakeholders around the world toand take dynamic action. This is also

low- and middle-income countries toadvance and bring innovations totrue when you are faced withaccess and delivery.

neglected patients as soon as we can.

challenges you have neverexperienced before. I think

Moriwaki: In 2020, the world was“Together” motivates us to moveengulfed in turmoil due to theforward and involve many people.

The team player spirit of“Together”-

Imoto: The spirit of

COVID-19 pandemic. One of the23

For example, GHIT, together with theways the pandemic disrupted GHIT’s

Imoto: In order to increase the“Together” is deeply(and our partners’) activities was bynumber of promising R&D projects,

embedded in GHIT

putting a temporary halt to clinical

GHIT not only issues public calls forstaff culture. I know that all staff feeltrials all over the world. “Together”

proposals, but it also proactively

a sense of responsibility and pride inbecame our slogan against thisreaches out to potential partners with

contributing to the organization as a

backdrop to help us share ourgood compounds and technologies,

member of the team. I think this is a

aspirations internally and externally. I

and helps facilitate and build

culture that took root in GHIT’s early

think the “Together” slogan

partnerships. We actively participate

days. I joined GHIT last year, anddemonstrates the solidarity andin international and domestic

have really felt how open themutual support between GHIT staff

gathering moments to try to identify

environment is--even mid-careeremployees can easily speak up and beenvironment, I believe that people

staff, and promote a working styleheard. The decision-making process is

who embody the “Together” (e.g.,that uses time effectively andalso much faster than in other“team player”) spirit will have a great

efficiently. The COVID-19 hadorganizations I have experienced.

opportunity to play an active role.

many downsides, but it alsopresented an opportunity to expand

Arai: I think the reason all this is

Arai: There are a lot of things that are

possible is because all GHIT staff

required of our staff, but we nevermembers have flexible mindsets andfeel negatively pressured. I think thisArai: Since the start ofdiverse experiences, and theyis because we all have the desire toworking-from-home, I have notnaturally accept each other’s

work “Together”, rather than solving

experienced any major problems in

differences. I believe that diversity,difficult issues alone. More thancommunication. Each staff member isequity, and inclusion (DEI) are among

anything, it is because we want to do

at a different stage in life (some live

GHIT’s greatest strengths.

our best to realize GHIT’s mission ofoverseas in different time zones, somedelivering products to neglectedhave families) but we all understandpatients.

the importance of work-life balance

Imoto: GHIT is a Japaneseorganization, but also like aninternational organization. We cannot
achieve our mission without
cooperating with many stakeholders

these ways of working all at once.

and support each other as we move

A year in which change was
encouraged-

things forward. This is possible
because we trust each other, andbecause GHIT is an organization that

Moriwaki: Our whole team started

respects the mental and physicalfor GHIT to be successful, it isworking-from-home in February

health of its staff.

important for our entire staff to have

2020, even before the COVID-19

excellent international sensibilities,

pandemic was declared. We had

language skills, and expertise. It ispreviously introduced

also essential for us to have a deep

working-from-home once a week onunderstanding of what DEI means ina test basis from the winter of 2018

had an enormous impact, but wepractice, and to be able to carry out

as part of ourwould like to promote dialogue andour duties with the “Together” spirit.

workstyle reforms,

collaboration with our stakeholders on

so although thiswhat we will do now through thewas our firstdifficulties and what we need to do

experience withfor the future.

from diverse backgrounds. In order

GHIT’s vision for itself-

Toward the future-

Imoto: It is true that the COVID-19

Moriwaki: GHIT’s has a team of only

working remotely every day, it was

20 people, but it needs to create a big

a smooth transition. It allowed us to

We have all experienced the pandemic

impact, so a high level oftake appropriate countermeasuresand have seen how well governments,

self-discipline and professionalism

against infectious diseases, and webusinesses, academia, international

are required. On top of that, from anhave been able to improve theorganizations, and all sectors can

HR perspective, we believe that byefficiency and digitization of ourwork together and in solidarity tobringing people into the organization

work by going online. I think weovercome various difficulties andwho are passionate about contributing

have created an environment thatquickly develop and deliver products

to global health, as well as people

allows us to achieve a goodto people. GHIT wants to realize thiswho have unique experiences and awork-life balance while

potential for neglected diseases. To

high level of expertise, regardless ofmaintaining high performance. In

achieve this, we share the spirit ofrace, nationality, gender, sexual

order to increase productivity and“Together” with our stakeholders in

preference, age, culture, or any othercreate maximum value as an

Japan and overseas, and work more

attribute, we can continuously

organization, we need to constantly

diligently than ever.

revitalize the organization and create

review and improve our work

new challenges and value. In addition,

environment with the deepest

because of the diversity of theconsideration for the health of our24

Finances
FY 2020 Financial summary
Balance Sheet
Assets (in millions)

JPY

USD

Current Assets
Fixed Assets

467.1
4,970.8

4.2
44.9

Total Assets

5,437.9

49.1

Liabilities (in millions)

JPY

USD

Current Liabilities
Non-current Liabilities

520.0
0.5

4.7
0.0

Total Liabilities

520.5

4.7

Net Assets (in millions)

JPY

USD

Designated Net Assets
General Net Assets

4,917.4-

44.4-

Total Net Assets
Total Liabilities and Net Assets

4,917.4
5,437.9

44.4
49.1

The US dollar amounts in this section represent translations of Japanese yen, solely for the reader’s convenience, at JPY110.71 = USD1, theexchange rate as of March 31, 2021. This financial summary is an excerpt from the GHIT Fund’s financial statements, which were audited by Ernst& Young ShinNihon LLC. The GHIT Fund is a Public Interest Incorporated Association and is registered in Japan.

Our Funding Partners and Sponsors
Full Partners

PublicFull Partners

25

PrivateNet Assets Variation Statement
Change in General Net Assets (in millions)
Ordinary Income

JPY

USD

Grants Received
Contribution Received
Misc. Income

3,350.9
463.1
0.5

30.3
4.2
0.0

Total Ordinary Income

3,814.5

34.5

Operating Expenses
Management Expenses

3,694.3
120.2

33.4
1.1

Total Ordinary Expenses

3,814.5

34.5

JPY

USD

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

1,003.3
1,645.2
788.2

9.0
14.9
7.1

Total Grants and Contributions Received

3,436.7

31.0

Ordinary Expenses

Change in Designated Net Assets (in millions)

Full Partners

Associate Partners

Affiliate Partners

Sponsors

26

LeadershipCouncil
The Council consists of the Japanese government, various foundations, and private companies that provide funding to GHIT. The Council resolves important matters as provided byapplicable laws and regulations or the Articles of Incorporation, including appointment and dismissal of members of the Council and the Board of Directors, amendment of the Articles
of Incorporation, and approval of financial statements.

Keiichi Ono

Yasuyuki Sahara, MD, MPH

Trevor Mundel, MD, PhD

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
ChairmanDaiichi Sankyo
Company, Limited

Eisai Co., Ltd.

Shionogi & Co., Ltd.

Haruo Naito
Representative Corporate Officer
and CEO

Isao Teshirogi, PhD
President and CEO

Assistant Minister/Director-General andAmbassador for Global Issues
Ministry of Foreign Affairs

George Nakayama
Senior Advisor and Corporate Advisor

Senior Assistant Minister for
Health Security and Technology
Ministry of Health, Labour and Welfare

President, Global HealthBill & Melinda Gates Foundation

Takeda Pharmaceutical
Company Limited
Christophe Weber
Representative Director
President and CEO

Board of Directors
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

Executive Director

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

CEO, GHIT FundPeter Piot, MD, PhD

Mahima Datla

Daikichi Momma

Satoshi Ezoe, MD, MPH, MPA, PhD

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

Director, Global Health Policy DivisionInternational Cooperation Bureau
Ministry of Foreign Affairs

Kazuho Taguchi

Ann M. Veneman, JD

Visiting Professor
Keio University School of Medicine

Managing Director
Biological E. Limited

Director
Office of Global Health Cooperation
Ministry of Health, Labour and Welfare

Supervisory Board Member

Ko-Yung Tung, JD

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

27

Vice Chair

Hiroki Nakatani, MD, PhD, MHPEd

Former Executive Director, UNICEF
Former Secretary
United States Department of Agriculture

Ex-Officio

Stephen Caddick, PhD

Professor, University College London
Former Director of Innovations DivisionWellcome

Catherine K. Ohura, MS, PMP

Supervisory Board Member

Hikaru Ishiguro, LLM
Statutory Auditor
INSPiRE Corporation

Ex-Officio

Katey Einterz Owen, PhD

Director, Neglected Tropical Diseases
Pharmaceutical Industry Leadership
Engagement
Bill & Melinda Gates Foundation

Selection Committee
The Selection Committee (SC) consists of domestic and international experts who have extensive knowledge and experience in research and development of drugs, vaccines, anddiagnostics. This committee evaluates investment proposals and reports from development partners and recommends investments to the Board of Directors. This SC includes no privatecompany representatives to avoid any conflict of interest between our backers and development partners.

Co-Chair

Co-ChairSophie Allauzen, PhD

Dennis Schmatz, PhD

Naoto Uemura, MD, PhD

Ralf Clemens, MD, PhD

Ann Mills-Duggan, PhD

Ken Duncan, PhD

Ken Ishii, MD, PhD

Hiroo Koyama, PhD

Gerd Michel, PhD

Anna-Karin Tidén, PhD, MRSC

Rieko Yajima, PhD

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

Independent Vaccine Expert

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

Independent Medicinal Chemistry Expert

Professor, Department of ClinicalPharmacology and Therapeutics
Oita University Faculty of Medicine

Partner, Innovations
Wellcome

Platform Unit Leader
Drug Discovery Chemistry Platform Unit
RIKEN Center for Sustainable Resource Science

Independent Diagnostic Expert

Deputy Director
Discovery & Translational Sciences
Bill & Melinda Gates Foundation

Chief Scientific Officer
Vela Diagnostics

Director, Drug Discovery Innovation
SPARK Program in Translational Research
Stanford University School of Medicine

Leadership TeamThe 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.

Catherine K. Ohura, MS, PMP

Kio Yamabe, MBA

Daisuke Imoto, MBA

Kei Katsuno, MD, MPH

Miho Takazawa, MBA

Bumpei Tamamura, MPH

CEO

Senior Director, Investment Strategy
Business Development

Chief Operating Officer

Senior Director, Finance & Operations

Vice President
External Affairs & Corporate Development

Senior Director, Brand Communications

Hayato Urabe, PhD, MPIA
Senior Director, Investment Strategy
Portfolio Development & Innovations

As of March 31, 2021
28

Acknowledgement
Funding Partners & Sponsors
Support from our generous funding partners and sponsors helps GHIT’s investments and operations advance and create meaningful impact.

Full Partners

Associate Partners

Affiliate Partners

Sponsors

29External Reviewers
The GHIT community’s work could not progress without the vital support of these experts and their institutions.

Yukihiro Akeda

Takashi Fujitsu

Nancy Le Cam Bouveret

Wanji SamuelPedro Alonso

Jennifer Gardiner

Carole Long

Alejandro Gabriel Schijman

Marco Albonico
Peter Andersen

Lewellys Barker
Michael Barrett
Clif BarryDavid BellMaria Elena Bottazzi
Teun Bousema
Tom Brewer

Enrico Brunetti

Martin Brusdeilins
Simon Campbell
Adriano Casulli
Eric Chatelain
Philip Cole

Stewart ColePaul L.A.M. Corstjens
Simon Croft

Alexandre Cuenat
Roy Curtiss

Peter Dailey

Julian Davies

Christine Debouck

David W. Denning
Keertan Dheda

Thierry Diagana
Thomas Dick
Carter Diggs

David W. Dowdy
Boro Dropulic

Filip Dubovsky
David Edwards
Sabine Ehrt

Hiroyoshi Endo

Hermann FeldmeierDavid Fidock

Peter U. Fischer
JoAnne Flynn
Michael Free

Michael Frimpong

Tamio Fujiwara
Nisha Garg

Ricardo Gazzinelli
Birgitte Giersing
Ann Ginsberg

Geoffrey N. Gobert
Daniel Goldberg

Federico Gomez de las Heras
Glenda Gray

Brian Greenwood

Sanjay Gurunathan
R. Kiplin Guy
Lee HallRashidul Haque

Yoshihisa HashiguchiThomas Hawn

Chris Hentschel

D. Gray Heppner
Philip Hill

Toshihiro Horii
Eric R. Houpt
Sanjay Jain

Joseph Kamgno

Takushi Kaneko

Niranjan Kanesa-thasan
Shigeyuki Kano
Subhash Kapre
Paul KayeNaoto Keicho

Jennifer KeiserDavid Kelso
Kent Kester

Akinori Kimura
Sue Kinn

Somei KojimaPeter Kremsner

Sanjeev Krishna
Michael Kurilla
Dennis Kyle
Ole LagatieJames LeDuc
Timothy Lu

Christian Mandl
John Mansfield

Greg Matlashewski
James McCarthy
Joseph McCune

James McKerrow

Donald Peter McManusCharles Mgone
Gerd Michel

Toshiyuki Miura
Valerie Mizrahi

Katsuhiko Mochizuki
Dinesh Mondal

Kouichi Morita

Charles Mowbray
Ivo MuellerPeter Myler

Daniel Neafsey

Christian Ockenhouse
Tsuyoshi Ogiku

Giuseppe Pantaleo
David Persing
Meg Phillips

Punnee Pitisuttithum

James A. Platts-Mills
Gerd Pluschke

David Pompliano
Dominic Pucci
Rino Rappuoli
Zarifah Reed

Rebecca Richards Kortum
Paul Roepe
Polly Roy

Eric Rubin

Peter Ruminski
Philip RussellDavid Sacks

Judy SakanariFred Santos

Dirk Schnappinger
Ami Shah Brown
George Siber
KJ Singh

Peter Smith

Lynn Soong

Gerald SpaethNathalie Strub-Wourgaft
Yasuhiko Suzuki

Francesca Tamarozzi
Marcel Tanner

Kaoru Terashima
Grant Theron

Katsushi Tokunaga
Nadia Tornieporth
Bruno Travi

Takafumi Tsuboi
Moriya Tsuji

Mickey Urdea

Govert J. van Dam

Wendy van de Sande
Lisette van Lieshout
Samuel Wanji

Mark Wansbrough-Jones
Stephen Ward
Tim Wells

Bruce Weniger

George Whitesides
Samuel Wickline
Judith Wilber

Elizabeth Winzeler
Dyann Wirth

Michael Witty
Paul Wyatt

Kazuhisa Yoshimura
Takeshi Yura
Fidel Zavala

Donato Zipeto30

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