GHIT Fund

ANNUAL R E POR T

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E

GHIT Fund

ANNUAL R E PORT

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CONTENTS

01

Message from CEO & Chair

03

GHIT Fund Portfolio

05

Successful completion of
Phase III trial for arpraziquantel

07

Clinical Candidates

09

Invested Partnerships in FY 2021

15

Strategic Plan FY 2018-2022 Progress

19

Highlights in FY 2021

21

Finances

25

Leadership

27

Acknowledgment

29

Photo credit: Dominic Chavez

02

Message from CEO & Chair

Advancement, achievement,
and powerful partnerships
This year is marked by notable progress and a momentous

In addition, in FY2021 GHIT invested USD 23 million in

first for GHIT, despite the continuing Covid-19 pandemic.

14 innovative projects, such as the clinical development ofFirst, we want to begin with gratitude – to our development

placental malaria vaccine, preclinical development of apartners for their creativity, collaboration, and perseverance;

monoclonal antibody to prevent P. falciparum malaria,

to our longstanding and new funders and sponsors for theirpoint-of-care diagnostics for NTDs, and other innovative

unwavering support and partnership; and to the global

projects at the discovery stage (p.15-18). Twenty threehealth community for its unity and innovation as we

new partners joined us in FY2021 to accelerate R&D forcontinue the fight against neglected diseases. Your

neglected diseases, bringing our total investments to datecommitment and passion inspire us every day.

to USD 276 million in 114 projects and 166 R&D partners
in total since 2013.

The GHIT made a conscious decision during the pandemic
to double down on our commitment to product development

Our current portfolio features 28 discovery projects and 15

for malaria, tuberculosis (TB), and neglected tropicalpreclinical projects. With new incoming projects anddiseases (NTD). We continued existing support without

existing projects that entered clinical phase, there are 14

pause and made bold new commitments, inspire of the newclinical trials underway over in 20 countries (p.9-14). In

reality Covid-19 has introduced. Like so many organizations

the midst (fourth year) of our second five-year operational

across the world, we have continued to improve ourplan (FY 2018-2022), nearly all targets within our GHIT

operations to perfect virtual collaboration and ensure

2.0 (FY 2018-2022) strategic plan have already been

seamless services to our partners. This has also been a year

met--a year before its completion. The Covid-19 pandemicof achievement and new evolution.

made a significant logistical impact on clinical trials;
nevertheless, we remain firmly invested in meeting our

A watershed moment in GHIT's R&D history

original clinical targets, together with our partners, by the

In November 2021, the GHIT-invested Pediatric Praziquantel

end of March 2023 (p.19-20). The candidates in our

Consortium, a public-private partnership dedicated to thepipeline each hold enormous potential for patients and aredevelopment of arpraziquantel celebrating its 10th anniversary,

essential to global efforts to meet SDG Targets. Some ofannounced the completion of its pivotal Phase III trial in Côte

projects are expected to launch during GHIT 3.0 (FYd'Ivoire and Kenya for a new treatment option for2023-2027).

schistosomiasis in preschool-aged children. The successful

03

joint development of pediatric praziquantel represents one of

Ensuring Access & Delivery

GHIT's most significant R&D achievements to date. This

Alongside our investment in R&D, GHIT continues todefining success for Consortium partners Merck KGaA,

support the development of the respective access andAstellas, Lygature, Framanguinhosm, and other importantdelivery strategies for late-stage clinical candidates so thatpartners also embodies GHIT's steadfast commitment todeveloped innovation can effectively reach patients anddriving Japanese innovation and technology through global

healthcare professionals. In FY 2022 and beyond, GHIT will

partnerships. The new formulation and treatment option willcontinue pushing for smooth end-to-end delivery ofbring hope and health to more than 50 million preschool-aged

GHIT-funded products with a focus on impact-driven accesschildren in the coming years. Learn more about the history,

and delivery strategies. Furthermore, Uniting Efforts for

significance of this partnership and technologies, and access

Innovation, Access and Delivery (Uniting Efforts), a global

strategies to deliver this innovation (p.7-8).

platform co-convened since 2019 by the Government ofPhoto credit: Dominic Chavez

Japan, the UNDP-lead Access and Delivery Partnershipcollaboration with GHIT. We will continue to create greater(ADP) and GHIT, remains our main platform for GHIT's

impacts by further expanding our partnerships across sectors

Global Access strategy. In FY 2021, Uniting Efforts

and industries. (p.24)

cemented its collaboration with the World HealthOrganization (WHO) NTD Department through partnering

Vision for the GHIT 3.0

and co-funding the development of a toolkit for national

GHIT has initiated dialogue with our funders for ourinvestment cases for NTDs as a companion document for

replenishment and solidified our investment strategy for

the NTD roadmap. The toolkit prototype will be piloted in

GHIT 3.0 (FY 2023-2027), which we will announce laterselect countries by the end of 2022. (p.22)

this fiscal year. We look forward to further collaboration
with all stakeholders in coming years.

Stakeholders update
New Council, Board, and Selection Committee members
strengthened our governance. We also bade farewell to oneof GHIT’s conceptual founders and global health visionary,
Tadataka (Tachi) Yamada, and the former WHO NTD
Director, Mwele Malecela. Both were strong supporters of
GHIT. They provided unparalleled insight and wisdom to
our team and leadership over the course of many years. We
are proud to have worked with such global health legends
and express our regret and sorrow over their passing. Their
legacies renew our steady and strong commitment to global
health R&D and adds a new sense of urgency.
In FY 2021, GHIT welcomed new sponsors: ZVC JAPANK.K. (Zoom) and Diligent Corporation. GHIT’s sponsorshipprogram allows companies to engage global health inOsamu Kunii

Hiroki Nakatani

CEO and Executive Director

Board Chair & Representative Director

04

GHIT Fund Portfolio

As of March 31, 2022

Discovery
Drugs / Vaccines

Target Research

Diagnostics

Target ResearchPreclinical
Screening

Hit-to-Lead

Lead Optimization
Product Design

Chagas disease, Leishmaniasis

Chagas disease

Buruli ulcer

Leishmaniasis

Soil-transmitted helminths

Chagas disease
Leishmaniasis

NTDs
Mycetoma

Malaria

Tuberculosis

Co-investment

GHIT investment

12.8
18.9

05

2013

29.7
46.0

32.4
63.7

2014

2015

51.1

56.8

98.6

131.9

2016

2017

69.1
169.9

98.1

76.0

350.0
300.0
250.0
200.0

209.2

261.4

276.3

150.0
100.0
50.0

2018

2019

2020

2021

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)

374 M

USD

95.3

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

ClinicalPreclinical

Chagas disease

Drug

Phase I

Phase II

Phase III

Registration

Product Development

Product Validation

Product Validation

Registration

Buruli ulcer

Mycetoma

VaccineDiagnostics

Schistosomiasis

II

Dengue fever

Chagas disease

Visceral leishmaniasis

Cutaneous leishmaniasis
I

Leishmaniasis

Lymphatic filariasis, Onchocerciasis
I

Schistosomiasis

Ib

IIa

114

Investment Overview FY 2013-2021

Total invested
partnerships
DiseaseMalariaIntervention

44.7%

Tuberculosis 10.9 %
NTDs

44.5%

Development
Stage

USD

23M

Total investments

14

166

54.9 %

Total productdevelopment partners

New Japanese partners

27.8%

58 Japanese partners
108 Non-Japanese partners

New non-Japanese partners

Drugs

66.3%

Discovery

17.3%

Vaccines

23.7%

Preclinical
ClinicalDiagnostics 10.0 %57 Active investments
57 Completed investments

FY 2021

Invested partnerships

6

17

06

Successful completionof Phase III trial
for arpraziquantel
significant public health burden and economic impact. The
drug praziquantel – the current standard treatment developedin the 1970s – is safe, effective, and available for school-aged
children and adults. However, at present, around 50 millionpreschool-aged children have been left untreated in publichealth programs, primarily due to the lack of an appropriate
child-friendly formulation of the drug.
Derived from praziquantel, arpraziquantel is an orally
dispersible tablet (dissolves in the mouth). It was developedby Astellas Pharma Inc. in Japan, subsequently optimized by
Photo credit: Pediatric Praziquantel Consortium

Merck in Germany and transferred for clinical manufacturing
to Farmanguinhos in Brazil. The new tablet is small, has

In November 2021, the Pediatric Praziquantel Consortium, achild-friendly flavor/ taste-masking properties, can be taken

public-private partnership dedicated to the development ofwith or without water, and withstands the hot and humid

arpraziquantel, a potential new treatment option forchallenges presented by a tropical climate.

schistosomiasis in preschool-aged children, announced thecompletion of its pivotal Phase III trial in Côte d'Ivoire andClinical development history

Kenya. The results of the trial, co-funded by the GHIT and the

The clinical program was set up in line with the EMA

European & Developing Countries Clinical Trials Partnershiprecommendations for pediatric development. It was designed(EDCTP), confirmed a favorable efficacy and safety profile

with the support of regulatory authorities and a panel offor arpraziquantel in children 3 months to 6 years of age,international experts, including clinicians from endemic

affected by this neglected tropical disease. This allows thecountries. Arpraziquantel required a full phase I-III clinicalprogram to advance towards regulatory file submission to thedevelopment program because of the new pediatric drug

European Medicines Agency (EMA).

candidate and the new target age group. The rationale for thePhase III study was based on data gathered from the clinical

New treatment option for millions of preschool-aged children

Phase I study in adult volunteers, a taste study in children 6-11

Schistosomiasis is one of the most damaging parasitic

years of age, and a Phase II dose-finding study in Schistosoma

diseases, affecting the lives of around 240 million people. It is

mansoni-infected children 3 months to 6 years of age,highly prevalent in sub-Saharan Africa and carries a

conducted in African countries.

Arpraziquantel development and funding history
2012

2013

2014

2015

Preclinical

2016

2017

Phase II in Côte d'Ivoire and Kenya
Phase I in South Africa
Taste study in
Tanzania

Pediatric PZQConsortium Launched

Grants provided
by R&D funders
07

BMGF US$1.15M
GHIT US$1.86M
GHIT Launched

GHIT US$4.9M

GHIT US$4.7M
EDCTP €1.99M

2018

Disease: Schistosomiasis
Intervention: Pediatric Drug
Development Stage: Phase III, Registration, Access
Country: Côte d’Ivoire, Kenya

The start of the COVID-19 pandemic brought significant

registered. ADOPT, a five-year program, aims to identify

disruptions of the clinical studies, setting back the pivotal

approaches for social mobilization, implementation models

Phase III clinical study in Côte d'Ivoire and Kenya.

and ensure wide acceptance and equitable access. With the

However, despite many challenges the trial resumed and was

support of GHIT, the consortium is also exploring thecompleted in October 2021. In the Phase III trial, children

expansion of the implementation research beyond ADOPT inaged 3 months to 6 years infected with S. mansoni or S.

collaboration with other partners and countries that have

haematobium were enrolled in different age groups andindicated an interest to conduct such studies. Merck has also

treated with a single dose of arpraziquantel. High efficacy

entered into a contract manufacturing agreement with

was observed with cure rates close to or above 90% for S.

Universal Corporation Ltd. in Kenya for the large-scalemansoni (at a dose of 50 mg/kg) and S. haematobium (at aproduction of arpraziquantel. Local manufacturing is meant

dose of 60 mg/kg). Arpraziquantel treatment at both doses

to ensure adequate supplies to meet the anticipated demanddemonstrated favorable safety, tolerability and improvedin an affordable and sustainable manner.

palatability among preschool-aged children. No new
potential risks or safety concerns were identified.
“With the completion of the Phase III trial, the PediatricAccess strategies for no one left behindPraziquantel Consortium demonstrates truly global

On behalf of the Consortium, Merck intends to apply for acollaboration with complementary expertise, bidirectional

scientific opinion by EMA under the EU-M4all procedureknowledge sharing, and mutual trust, is a key success factor to(previously known as Article 58) for high-priority medicines

develop and deliver safe and affordable treatments forfor human use intended for markets outside the European

schistosomiasis. With this milestone, we continue ourUnion. A positive opinion by EMA would facilitate thecommitment to eliminating schistosomiasis and ensuring all

inclusion of arpraziquantel in the World Health Organization

people affected by this neglected(WHO) list of prequalified medicinal products, as well as

tropical disease have access to aregulatory approvals in endemic countries. With the fulllife-saving therapy. Together withclinical development phase successfully completed, theour partners, we are steadfast in our

program has entered the regulatory filing stage. Meanwhile,

vision to bring new hope to thework is in progress to identify innovative mechanisms forworld’s most vulnerable children.”access and, through the Consortium’s dedicated accessprogram, ADOPT, also funded by GHIT and EDCTP, toprepare the potential delivery of arpraziquantel once it is

2019

2020

2021

2022

Dr. Jutta Reinhard-Rupp
Chair of the Pediatric Praziquantel Consortium Board
Head of the Global Health Institute at Merck

2023

2024

2025

Phase III in Côte d'Ivoire and Kenya
Access Program (ADOPT) in Côte d'Ivoire, Kenya, and Uganda
Regulatory Submission to EMA through EU-M4all

Access in firstendemic countries
in AfricaOldformulationNew

formulation

GHIT US$4.1M

GHIT US$2.6M
EDCTP €5.7M

Photo credit: PediatricPraziquantel Consortium
Pediatric Praziquantel Consortium. Where we stand: https://www.pediatricpraziquantelconsortium.org/

08Clinical Candidates
Impact on the ground

SILVAMP TBLAM
TM

Tuberculosis
Intervention: Diagnostics
Development Stage: Field Validation
Country: South Africa, Malawi,
Zambia, Tanzania,
Uganda, Vietnam,
Thailand
Disease:SILVAMPTM TBLAM developed by Fujifilm and FIND is a TB diagnostic assay with highsensitivity and specificity in HIV-positive patients using Fujifilm’s proprietary silver
amplification technology to detect LAM (Lipoarabinomannan) in urine. LAM is a
glycolipid found in mycobacterial cell wall and can be detected in urine in patients with
disseminated TB, particularly in severe immunocompromised states. Current TB
diagnostics rely on sputum, which are time-consuming, and require trained laboratory
personnel. Moreover, sputum samples are not always easy to get, for example in somepatient groups like children and people with severe HIV infection. In some cases ofextra-pulmonary TB, using sputum might be inappropriate. Therefore, SILVAMPTM
TBLAM could be a game-changing rapid diagnostic test (RDT). Despite the long-lasting
COVID-19 pandemic, this project has conducted field validation prospective study phase inSouth Africa, Malawi, Zambia, Tanzania, Uganda, Vietnam and Thailand. Additional
studies were also conducted by other partners. Product manufacturing optimization is to beaddressed by the end of 2022.

BP-AK urine LAM test
Tuberculosis
The only available non-sputum TB test in the market has insufficient sensitivity to be
Intervention: Diagnostics
used as a reliable diagnostic test, especially in the non-HIV-infected TB population.
Development Stage: Product Development
The BP-AK urine LAM test, co-developed by Asahi Kasei Corporation and Biopromic
Country: South Africa, Uganda
Disease:detects picogram amounts of LAM TB biomarker in the urine of TB patient,irrespective of HIV infection, in timely manner with high accuracy.

The BP-AK LAM test applies a novel proprietary sample preparation technology andunique detector particles that allow LAM TB biomarker detection at lower picogramlevel, which is 100 times higher than the Alere/Abbott, the only test available in themarket so far. The BP-AK LAM test for diagnosis of tuberculosis meets WHO TPP
criteria. The research team conducted validation study to evaluate BP-AK LAM
prototype by using well characterized frozen TB urine samples collected from TB
endemic area. In total 1050 urine samples collected from 5 cohorts (3 in Uganda and 2
in South Africa) were analyzed with the BP-AK urine LAM test. They have found thatthe overall pool sensitivity and specificity of BP-AK urine LAM prototype irrespectiveof HIV status were superior to the test available in the market.

Photo credit: Biopromic AB

09

Lung Flute ECO
Tuberculosis
Diagnostics
Development Stage: Field Validation
Country: Cameroon
Disease:The Lung Flute is a Positive expiratory pressure (PEP) device invented in early 2000s that

Intervention:

consists of a simple plastic flute with a vibrating reed for facilitating sputum expectoration. The
Lung Flute has lower risk of adverse events than isotonic saline-based sputum induction
devices, does not require an electrical supply, and is simple to use, making it particularly well
suited for primary care and community-based screening. The Lung Flute ECO is a recently
developed paper-based version of the Lung Flute that is inexpensive enough to be used as adisposable, point-of-care tool to aid in sputum production in low-resource settings. In this study,
the research team aims to evaluate the performance of the Lung Flute and Lung Flute ECO to
improve sputum-based TB diagnosis, primarily in vulnerable groups, in a TB endemic setting.
They will evaluate performance of the Lung Flute ECO and the Lung Flute® HR acrossmultiple sites in Cameroon. The study focuses on evaluating test access and accuracy in patientgroups with documented challenges to produce sputum on demand, including children 6-14
years of age, women, the elderly, people living with HIV, people admitted to hospital, andasymptomatic persons screening positive for TB by digital chest x-ray.

SJ733
MalariaIntervention: Drug
Development Stage: Phase IIa
Country: Peru
Disease:

SJ733 is a PfATP4 inhibitor that meets criteria for treatment of uncomplicated malaria. 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 adult patients with uncomplicated P.
vivax or P. falciparum blood-stage malaria monoinfection. SJ733 is being administered orally once
every day for three consecutive days, with or without a fixed dose of the pharmacoenhancer
cobicistat. 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. Recruitment opened in March 2021 and the firsttwo cohorts have been completed for P. vivax 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
and pharmacokinetics of SJ733. The results of this trial will identify active, well-tolerated doses forinvestigation in a larger Phase IIb clinical trial.

10

BK-SE36 / CpG & NPC-SE36 / CpG
MalariaIntervention: Vaccine
Development Stage: Phase IIb
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 allstages of parasite development are potential vaccine targets, blood stage vaccines target thedisease-causing stage of the P. falciparum life cycle, to protect against disease severity andreduce parasitemia while allowing development of acquired immunity. Early-stage clinical
trials have demonstrated that the BK-SE36 vaccine (SE36 recombinant protein andaluminium hydroxide gel) had acceptable reactogenicity, no unexpected safety signals andwas immunogenic. Vaccine responders experienced less malaria infection and had lower
risk of developing clinical symptoms. Immune response was stronger in Japanese adults
when CpG-ODN (K3) adjuvant was added to the formulation. A Phase Ib trial to test safety
and immunogenicity of BK-SE36/CpG in 1 year-old children to adults in Burkina Faso wascompleted. Results indicated that there were no unexpected safety concerns and the vaccine
was immunogenic. A follow-up observational study of this trial is assessing the long-term
persistence of the antibodies. New lots of SE36 and CpG (for the formulation renamed
NPC-SE36/CpG) are being manufactured for proof-of-concept trials and their comparability
to previous vaccine lots are being assessed. Simultaneously, preparations are under way for
the Phase IIb trial site selection and design. Finally, strategies are explored to improvedelivery of SE36 on virus-like particles.

SMAART-1
Malaria
Current malaria tests in the field, either RDTs or classical blood tests using microscopes, cannot
Intervention: Diagnostics
rapidly detect often sub-microscopic, asymptomatic malaria infections in individuals. While
Development Stage: Product Development
these individuals do not have any symptoms for the disease and thus do not seek medical support,
Country: Democratic Republic
they are still an important reservoir for the parasite, playing a key role in malaria transmissionof Congo
Disease:through mosquitoes. The project addresses this limitation by producing and validating a

Saliva-based Malaria Asymptomatic and Asexual Rapid Test (SMAART-1) that has high enoughsensitivity to diagnose asymptomatic cases with high reliability and greater acceptability,
designed especially for easier diagnostic screening of children. This test will for the very firsttime enable non-invasive testing of malaria parasite infections from saliva, avoiding the need forcollecting blood. Production of stable cells lines expressing antibodies used for the test anddevelopment of the commercial SMAART-1 are underway and are expected to be completed by
mid-2022. A usability study of the SMAART-1 in the Democratic Republic of Congo has been
conducted to evaluate the acceptability and potential for broader adoption of this testing modality
in comparison with blood-based rapid tests for malaria detection: results show that both lay andexpert users are able to effectively use the test; the SMAART-1 is a desirable alternative toblood-based test. Further field testing and evaluation of the SMAART-1 will be performed in
multiple countries by November 2022. The project aims to fulfill all the criteria for obtaining CE
marking and subsequent WHO prequalification of the SMAART-1 by 2024.

11

Fosravuconazole
Mycetoma
Drug
Development Stage: Phase IICountry: Sudan
Disease:

Mycetoma typically affects poor people who live in remote areas; the disease mainlyIntervention:

occurs in tropical and subtropical regions in Africa, Middle and South America, andAsia. There are two types: the bacterial type that is treated with antibiotics and the fungal
type that is treated with antifungals. Both present with a slowly progressive subcutaneousmass usually on the foot as it is thought that the microbes enter the skin for example after
a thorn prick. The treatment of the fungal type has been problematic for many years; thecure rates are low, and the choice of drugs is limited. Amputation of a limb after repeatedtreatment failure is not uncommon. DNDi, in partnership with Eisai Co., Ltd., Japan, hasconducted a Phase II trial looking at whether fosravuconazole, an azole-class antifungal
drug discovered by Eisai (and already approved for onychomycosis in Japan) may also be
an effective and affordable treatment for mycetoma. Fosravuconazole has the advantage
of better absorption, a long half-life (making once a week administration possible), andless side-effects. The trial has three arms; two arms with fosravuconazole in a high doseand low dose, and the comparator arm is with itraconazole, currently the standard
treatment. The treatment is for 12 months, with surgery (removal of the remaining lesion)
after 6 months. The trial was conducted between 2017-2021 in Sudan at the WHO
Collaborating Center on Mycetoma in Khartoum; the results are expected to be available
in July 2022.

CpG-D35
Cutaneous leishmaniasis
Intervention: Drug
Development Stage: Phase I
Country: UK, Columbia
Disease:Cutaneous leishmaniasis (CL), endemic in 87 countries, mainly affects poor populations.
Approximately 0.6 to 1 million new CL cases are estimated every year. While CL is not
life-threatening, it is a disfiguring disease that results in stigma and economic loss. Currently,
there are no satisfactory treatments for any form of CL. CpG-D35, as a combination therapy forthe treatment of patients with CL, activates the innate and adaptive immune system of the host.
The preliminary results of the single ascending dose (SAD) study conducted in the United
Kingdom showed that CpG-D35 was safe when administered subcutaneously at the tested
doses. Pharmacodynamics analysis showed dose-dependent increase of plasma CXCL10
concentrations, the most important biomarker in primate model of activity. There was noincrease nor in TNFα nor IL-6. A consistent pattern of haematological changes was observed ina dose-dependent manner. All changes were transient and considered non-clinically significant,and consistent with the expected pharmacological activity with migration of some white blood
cells at the site of injection and increase of production. The multiple ascending dose (MAD)study will be conducted in Colombia to assess the safety of CpG-D35 following multiple doses
administered to patient with uncomplicated CL either alone or in combination with miltefosine.
GMP batch using the improved manufacturing process complies with drug substancePhoto credit: Vinicius Berger-DNDi

specifications. Technical batch of lyophilized CpG-D35 powder also complies withspecifications. Drug product clinical batch will be manufactured in support of Phase 2 study.

12

AWZ1066S
Lymphatic filariasis /Onchocerciasis
Intervention: Drug
Development Stage: Phase I
Country: UK
Disease:Lymphatic filariasis and onchocerciasis are debilitating diseases affecting more than 150
million people globally. Both caused by parasitic worms, this particular drug, AWZ1066S,
has been designed to target Wolbachia, a bacterial symbiont which is essential to theworm’s survival, rather than the worm itself. Originally developed through the Bill &Melinda Gates Foundation supported A∙WOL consortium, the current work is a partnership
between Liverpool School of Tropical Medicine, the Department of Chemistry of the
University of Liverpool and Eisai, AWZ1066S was administered to the first participant
enrolled in a Phase I clinical trial being held at the NIHR Royal Liverpool and Broadgreen
Clinical Research Facility, based at Liverpool University Hospitals NHS Foundation Trust.
This first in human trial comes following the completion of pre-clinical safety tests and willassess the safety, tolerability and pharmacokinetics of the drug. The drug candidate,
AWZ1066S, also has the potential to be used in the whole population, including children
and pregnant women, providing a unique opportunity to make a large contribution tocommunities affected by these diseases.

Photo credit: Kate Dodd

BU-MYCOLAC
Buruli ulcer
The research team developed a RDT for Buruli ulcer (BU) diagnosis. This test targets
Intervention: Diagnostics
mycolactone, the toxin produced by Mycobacterium ulcerans the causative organism of theDevelopment Stage: Product Development
disease. The test is configured as a competitive assay. In current activities, the prototype wasCountry: Cameroon, Côte d'Ivoire
Disease:evaluated in feasibility studies in Cameroon and Côte d'Ivoire, using clinical samples from morethan 200 suspected patients. Seven swab samples were collected per person and allocated to
PCR, ELISA and RDT at different sites. All the identified BU patients were put on treatment.
The initial data indicate a 92% specificity and 38% sensitivity, and poor concordance between
the different tests. The samples used being the last of seven samples collected from each lesion,
the concentration of mycolactone determined through calibration curves was unexpectedly low
and may explain the poor clinical sensitivity of the RDT. The research team hypothesizes thatthe discrepancies observed can be ascribed to sample-to-sample variations, efficiency of sample
collection and the nature of the sequential collection of the swabs per lesion. Evaluation of the
RDT on 19 different bacteria species cultured from non-BU patient samples shows the RDT to
be specific to M. ulcerans. Significant improvements were made to the test, allowing 100%validity of the RDT and improved consistency of the results demonstrated with duplicate swabs.
However, wound swabs being an unusual sample matrix, improvements to sample collection

Photo credit: FIND

13

and processing will be the focus of upcoming studies prior to a reevaluation of the improved
RDT in the field.

Trypanosoma cruzi-LAMP
Chagas disease
According to PAHO, an estimated 1.12 million women of childbearing age are infected by
Diagnostics
Trypanosoma cruzi (T. Cruzi), and around 9,000 infected babies are born each year, accounting forDevelopment Stage: Product Development
more than 20% of all new cases of Chagas disease in the region. The validation of highly sensitive
Country: Argentina, Bolivia,
and specific diagnostics that provide an early access to diagnosis for mothers and newborns will
Paraguay
Disease:Intervention:

greatly contribute to control the disease impact. Furthermore, already available drugs have a very
high cure rate (~100%) in T. cruzi-infected newborns if administered early upon infection.

However, the current algorithm to detect congenital T. cruzi infection involves microscopy-basedparasitological methods that lack sensitivity and thus a serological study must be performed
several months later once mother-derived anti-T. cruzi immunoglobulins have waned. But, inmany endemic regions people live far from referral centers, which entails that a large proportion of
infants rarely go back for diagnosis confirmation and treatment, if infected. Consequently, they
evolve to the chronic phase of the disease with the risk of developing severe manifestations that
affect the cardiac and/or digestive tissues. In contrast to microscopy-based methods,
molecular-based diagnostics have a very high sensitivity to detect congenital T. cruzi infections.
The problem is that most laboratories in Chagas disease highly endemic regions are not equippedto perform them. With the aim to provide a suitable point-of-care (POC) test for the timely
Photo credit: CONICET-INGEBI

diagnosis of congenital Chagas disease in these settings, the research team is now conducting theoperational validation of EIKEN T. cruzi loop-mediated isothermal amplification (LAMP)
prototype in nine maternity hospitals in Argentina, Bolivia and Paraguay.

SCH CAA RDT
Schistosomiasis
In 2021, the project reached its first milestone, where the schistosomiasis circulating anodic
Intervention: Diagnostics
antigen rapid diagnostic test (SCH CAA RDT) prototype was field-tested in two schistosomiasis
Development Stage: Product Development
endemic countries; Kenya and the Philippines. The test was used to detect the presence of S.
Country: Kenya, Philippines
Disease:mansoni, S. haematobium and S. japonicum. In addition, in each country, a non-endemic site waschosen to evaluate the specificity of the test. In total, 1182 individuals were enrolled in the fieldstudy; 3 sites in Kenya and 2 sites in the Philippines. To run the assay, only 50µl of finger-prickblood is needed, and the result is available after 20 minutes. In the absence of a gold standard, theperformance of the prototype was evaluated against a composite reference standard comprised of 3days of repeated microscopy, a highly sensitive lab-based assay (termed the UCP-LF CAA assay)
and PCR. The results are very encouraging and will inform further development prior to thedesign-lock and transfer to manufacturing. In parallel, the partners have developed a high-level
access strategy for the SCH CAA RDT that will be further developed and implemented. Throughthe support of the Bill & Melinda Gates Foundation and Merck KGaA, molecular characterizationof the analyte itself has been undertaken in addition to understanding the secretion dynamics of thecirculating anodic antigen (CAA) for each of the three Schistosoma species. In addition, thepartners are developing synthetic CAA standards, which will be necessary for lot-to-lot testing as
the product enters the transfer to the manufacturing phase.

14

Invested Partnerships in FY 2021
From Discovery to Preclinical

Identification of novel dual-acting bactericidal drug targets
against Mycobacterium tuberculosis
Tuberculosis / Drug

Fujita Health University, Hokkaido University, Nagoya University,
Research Institute of Tuberculosis, Harvard University, University of Minnesota

Target Research Platform

Screening Platform

Hit-to-Lead Platform

The emergence of multi-drug resistant and extensively drug-resistantstrains of Mycobacterium tuberculosis (Mtb) jeopardizes the efficacy of
TB treatment. This project aims to identify novel targets for TB drugs thatcan rapidly and strongly kill Mtb through two independent antimicrobial
mechanisms (dual-acting). The researchers have already identified suchdual-acting candidate genes, and they will characterize the candidate
genes in vitro and in vivo by utilizing a newly developed gene silencing
technology for Mtb, the mycobacterial CRISPR interference system. They
will also perform their unique whole cell target assays using an in-house

Dr. Yusuke Minato
Fujita Health University

Product Development Platformchemical library to identify small molecules that kill Mtb due to inhibiting
one of their genetically validated targets. Identified hit compounds will beclustered by structural similarity, and representative molecules of eachcluster will be derivatized. These compounds will be used as probe
compounds to perform chemical validation of their targets. The teamconsists of highly skilled experts in bacterial genetics, bacterial
physiology, chemical biology, medicinal chemistry, and pharmaceuticalsciences. The team shows their strong commitment to developing a new
drug to improve TB treatment.

Identification and Validation of potential Plasmodium E3
Ligases for PROTAC Platform
FIMECS, Inc., National Center for Genetic Engineering and Biotechnology (BIOTEC)
Malaria / Drug
Target Research Platform

Screening Platform

Hit-to-Lead Platform

A new drug design approach is needed to expand the repertoire of
antimalarial targets and the drug arsenal to exploit previously
untouched vulnerabilities in the parasite. Instead of using drugs thatact as inhibitors of target functions, drugs designed as protein
degrader, also known as PROteolysis-TArgeting Chimeras or
PROTACs, can destroy target proteins. The protein degrader work byhijacking the proteasome (a protein complex present in every cell thatnaturally breaks down old or damaged proteins) to degrade a target
protein. Protein degraders are designed with one “warhead” that binds
the target and another that binds ubiquitin E3 ligase, a protein that

Product Development Platform

Dr. Nitipol Ken Srimongkolpithak
BIOTEC

marks other proteins for degradation. This approach has the advantagethat the protein degraders can bind anywhere on the target, without the
restriction of binding to sites important for target function. Therefore,
the protein degraders can be designed against proteins previously notconsidered as drug targets. BIOTEC and FIMECS have successfully
established the protein degradation screening system in transgenicparasites and also generated our unique and diverse PROTAC library.
Next stage would be the screening of the library and identification ofpotential parasite E3 ligases for PROTAC platform.

Development of a P. vivax multistage vaccine
effective both for protection and transmission blocking
Malaria / Vaccine

Hokkaido University, Jichi Medical University, Kanazawa University, Kyoto University, University of Toyama,
University of Cambridge, Instituto Leônidas & Maria Deane (ILMD) and The Fundação de Medicina Tropical
Doutor Heitor Vieira Dourado (FMT-HVD)

Target Research Platform

Screening Platform

Hit-to-Lead Platform

This project aims to develop multistage vaccine against P. vivax by
applying vaccine platform technologies utilized to develop
multistage vaccine against P. falciparum previously funded by
GHIT. The viral-vectored vaccine against P. falciparum developedby the research team induced humoral and cellular immune
responseseffectively, showed desired protection rate in animalmodel, and has a transmission-blocking efficacy. By utilizing the15

Product Development Platform

Prof. Shigeto Yoshida
Kanazawa University

technologies, they will develop two viral-vectored vaccines
expressing both pre-erythrocytic-stage and sexual-stage antigens of
P. vivax. This project is conducted by an intercontinental
consortium involving researchers from Japan, the United Kingdom,
Brazil, and Burkina Faso. The research team will also conduct
research for the development of a bivalent vaccine effective bothfor P. vivax and P. falciparum.

Hit-to-Lead development of novel Astellas compoundswith antimalarial activity
Malaria / Drug

TCGLS, Astellas, MMV

Target Research Platform

Screening Platform

Hit-to-Lead Platform

With funding from GHIT, between 2018-2021 MMV and Astellas
successfully completed a high throughput screening project. The project
involved the screening of an Astellas 20,000 compound library in aphenotypic whole cell P. falciparum growth inhibition assay. Initial
profiling of the active compounds identified four chemically andbiologically attractive starting points for a malaria drug discovery project.
The goal of the current Hit-to-Lead project is to identify at least one
novel compound series with clear potential for further development andprogression to Lead Optimization. The project will consist of two phases.

Product Development Platform

Dr. James Duffy
MMV

The first 6-month phase will involve synthesizing and profiling a small
set of compounds designed around each of the four hit compounds. The
compounds will be designed to explore both structure-activity
relationships and the scope for structural modification to improve thecompound profile, such as potency, DMPK, and safety. Two series will
be selected for Hit-to-Lead studies and the second 18-month phase of the
project will be to optimize the compounds, aiming to identify afrontrunner compound meeting the MMV-GHIT Early Lead criteria.

Preclinical development of a monoclonal antibody toprevent P. falciparum malaria
Malaria / Drug

PATH, GSK, Eisai Co., Ltd., Ehime University

Target Research Platform

Screening Platform

Hit-to-Lead Platform

RTS,S/AS01 (RTS,S), the world’s first malaria vaccine recommended
for use in young children living in areas of moderate to high malariatransmission by the WHO, functions primarily by inducing high titerantibodies to the invariant repeat region of the P. falciparum
circumsporozoite protein (CSP). It affords relatively high-level
short-term protection and functionally potent monoclonal antibodies(mAbs) in protective antisera are likely to be promising tools forpreventing infection. This project aims to develop a potent anti-CSP
mAb for malaria prevention and is enabled by access to a panel of potent

Product Development Platform

Dr. Ashley Birkett
PATHanti-CSP mAbs, identified from volunteers who were protected against
P. falciparum challenge following immunization with RTS,S. Applying
in silico, in vitro, and in vivo methodologies, hundreds of candidate
mAbs have been evaluated and optimized lead and backup molecules
meeting pre-defined performance characteristics have been identified.
This funding will support completion preclinical development of a leadcandidate, the development of a clinical development plan, and enable
regulatory interactions in support of a future IND submission for a
proof-of-concept clinical trial.

Clinical development of placental malaria vaccine candidates

Malaria / Vaccine

Ehime University, European Vaccine Initiative (EVI), University of Copenhagen,Institut national de la santé et de la recherche médicale (Inserm), Institut de recherche pour le développement,Groupe de Recherche Action en Santé (GRAS), Noguchi Memorial Institute for Medical Research

Target Research Platform

Screening Platform

Hit-to-Lead Platform

Malaria infection during pregnancy can lead to the development ofplacental malaria (PM), accounts for an estimated 200,000 infant
deaths annually and is predicted to contribute to nine maternal deaths
per 100,000 live births. An effective vaccine would be an attractive
tool to control PM on its own or to complement the existing yet
imperfect tools. The two PM vaccine candidates PAMVAC andPRIMVAC consist of recombinant proteins encompassing theplacental binding region of VAR2CSA, using similar but
complementary approaches. Previous clinical trials demonstrated thatboth adjuvanted vaccine candidates are safe and well-tolerated andinduce good homologous immune responses. Preclinical and clinical

Product Development Platform

Dr. Flavia d'Alessio
EVI

trial results show lasting immune responses, supporting thedevelopment of a PM vaccine. However, VAR2CSA is a diverse
antigen, and it is essential to optimize cross-reactivity against
different VAR2CSA variants and further evaluate the longevity of theimmune response prior to embarking on costly large-scale phase II
clinical trials. This project aims to characterize the longevity of the
PRIMVAC-induced immune response in women in malaria-endemicareas and the capacity of the vaccine to boost and broaden a naturalacquired immune response. Additionally, the partners will undertakethe pre-clinical development and evaluation of PAMVAC-cVLP,
which is an improved version of PAMVAC.
16

Autophagy as a novel drug development target
for Chagas disease
Chagas disease / Drug

National Institute of Advanced Industrial Science and Technology (AIST), DNDi

Target Research Platform

Screening Platform

Hit-to-Lead Platform

One of the main difficulties in attempting target-directed drug
discovery approaches in Chagas disease is the lack of information onmolecules vital to the survival and proliferation of the parasite T. Cruzi.
This project is based on the outcome of the previous research where
the research team conducted CRISPR/Cas9-aided exhaustive
knockouts on over 500 T. cruzi genes and identified 25 essential
molecules for the parasite proliferation. Each of these 25 molecules
was carefully examined for its potential as a drug target for Chagas
disease: the researchers investigated whether host cell-derived
metabolites in the amastigote (the proliferating stage of this parasite
in animals) will rescue dysfunction of the target molecules andProduct Development Platform

Dr. Koji Furukawa
AIST

whether the structural features of the active sites are suitable for the
discovery of T. cruzi-specific drugs. Finally, they selected an
autophagy-regulating factor as a potential target of interest for thisChagas disease drug discovery project. This project aims to obtain
initial hit compounds amenable for further development as novelanti-T. cruzi drugs acting through inhibition of the parasite
autophagy-regulating factor. To validate whether thecompounds/target are amenable to drug development, they will
determine the structural binding mode between the compounds andprotein target by X-ray crystallography.

Preclinical development of DNDI-6174,
a drug candidate for leishmaniasis
Visceral leishmaniasis/ DrugEisai Co., Ltd., DNDi

Target Research Platform

Screening Platform

Hit-to-Lead Platform

Visceral leishmaniasis (VL) is a complex neglected tropical diseasecaused by more than 20 different species of the Leishmania parasite
and accounts for 50,000 to 90,000 new cases and 10,000 deaths
annually. The long term goal of DNDi’s leishmaniasis program is tocontribute to the WHO NTD roadmap goal of eliminating VL as apublic health problem and reaching zero deaths globally due toprimary VL by 2030. In this perspective, the project aims to develop
a drug candidate that is orally active, safe, effective, short-course,

Product Development Platform

Ms. Stéphanie Braillard
DNDi

and field-adapted to treat VL and potentially cutaneousleishmaniasis. DNDI-6174 is an ideal candidate as it belongs to anovel chemical class, acts through a new mechanism of action andoffers the possibility of avoiding adverse effects due to a predicted
low human dose and a very promising safety margin. They willcomplete preclinical development of DNDI-6174 within two years toenable its nomination as a clinical candidate ready for Phase I
studies in healthy human volunteers.

Screening project
Takeda Pharmaceutical Company Ltd., DNDi

Chagas disease / Drug

Target Research Platform

Screening Platform

Hit-to-Lead Platform

Product Development Platform

Screening Platform

Hit-to-Lead Platform

Product Development Platform

Screening Platform

Hit-to-Lead Platform

Product Development Platform

Screening projectDaiichi Sankyo Company Limited, DNDi

Chagas disease / Drug

Target Research Platform

Screening project
University of Tokyo, DNDi

Leishmaniasis / Drug

17

Target Research Platform

18

Strategic PlanFY 2018-2022 Progress
R&D: Investments in Product Development
Further leverage innovations from Japanese biopharma and academia# targetedby March
2023

Diagnostics

Lead Identification

Preclinical

5

5

8

Diagnostics identified
and funded# achievedby March
2022

11

ed
Ach iev

Hit-to-lead programs

17ed
Ach iev

Preclinical candidates
identified

14ed
Ach iev

Diagnostics

1. Fujifilm SILVAMP TB LAM – A Sensitive point-of-care Tuberculosis Test [G2017-207]
2. Commercial development of a Saliva-based Malaria Asymptomatic and Asexual Rapid Test (SMAART-1) [G2019-105]
3. Production, validation and use of Leishmanin skin test (LST) for detection of Leishmania exposure and immunity [G2019-213]
4. A schistosomiasis rapid diagnostic test to support control programs in monitoring treatment impact and reassessment mapping [G2020-104]
5. Product Development of LFA platform for improving sensitivity of Point-of-Care assays for infectious disease with main focus on Tuberculosis [G2020-201]
6. A Buruli ulcer mycolactone (BU-MYCOLAC) rapid diagnostic test to enhance early diagnosis and treatment [G2020-202]
7. Field validation of Trypanosoma cruzi-LAMP: a molecular point-of-care test for the control of congenital Chagas disease [G2020-203]
8. Viability & Value of the Lung Flute ECO for Sputum Sample Collection and Tuberculosis Testing in Vulnerable Groups (3V Trial) [G2021-114]
9. MycEXomics: Development of field-friendly point-of-care diagnostic test for mycetoma [T2019-260]
10. Towards the rapid diagnosis of malaria hypnozoite infection: feasibility studies [T2020-153]
11. Development of ‘all-in-one’ diagnostic kit for Buruli ulcer using lateral flow DNA-chromatography [T2020-161]

Lead identification

19

1. Structured-based approach to develop a novel mechanism-of-action antimalarial with multistage activity [G2020-112]
2. Preclinical development of a new class of Plasmodium DHODH inhibitor for the treatment of malaria [G2018-112]
3. Hit-To-Lead development for Neglected Tropical Diseases (DNDi and Mitsubishi Tanabe) [H2020-202]
4. Hit-to-Lead Development of Novel anti-TB Natural Products [H2018-202]
5. Live attenuated prophylactic vaccine for leishmaniasis [G2018-201]
6. Prolyl tRNA Synthetase Inhibitors for New Antimalarials [H2020-101]
7. Hit-to-Lead development of novel Astellas compounds with antimalarial activity [H2021-201]
8. Lead optimization and preclinical candidate selection from the NTD Drug Discovery Booster series S07 for visceral leishmaniasis [G2020-213]
9. Hit-to-Lead Development of Hits Identified in the Phenotypic Screening against Mycobacterium tuberculosis [H2020-203]
10. Hit-to-lead Development of 6-Deoxy-8-O-methylrabelomycin Analogues [H2019-201]
11. Hit-to-Lead Development of Phenotypic and Mechanism-based Screen Hits [H2019-107]
12. Optimisation of multistage inhibitors of P. falciparum lysyl-t-RNA synthetase for the treatment of malaria [G2020-115]
13. Hit-to-lead development of new antimalarial compounds from DDI library [H2020-201]
14. Mitsubishi Tanabe Lead Optimization of Anti-malarial [G2018-202]
15. Lead optimization of a candidate series active against Chagas Disease [G2019-209]
16. Proteasome inhibitors as new potent antimalarials [H2019-101]
17. Development of nucleoside sulfamates as novel antimalarials [H2019-104]

In GHIT 2.0’s fourth year, we and our product development partners have progressed
steadily toward key R&D milestones, despite the far-reaching impact of the pandemic.
Additionally, we continued to identify and fund projects aligned with strategic 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 conducted6ed
Ach iev

Proof of Concept achieved

Approved by stringent
regulatory authority21
As of March 31, 2022

Preclinical

1. Lead optimization of novel azetidine-based tryptophan synthase inhibitors as new mechanism of action treatment of tuberculosis [G2017-101]
2. The development and production of cGMP lots of a novel tetravalent dengue virus-like particle (VLP) vaccine [G2018-103]
3. Optimization and pre-clinical development of a Trypanosoma cruzi Cyp19 knock-out strain as a live vaccine for Chagas disease [G2019-102]
4. Preclinical development of malaria transmission-blocking vaccine candidate Pfs230D1+ formulated with SA-1 adjuvant [G2019-205]
5. Preclinical and Clinical Development of SJ733, a Novel PfATP4 Inhibitor for the Treatment of Severe Malaria [G2019-215]
6. Preclinical studies of potent Gwt1p inhibitor toward IND for antimalarial agent with novel mechanism of action [G2019-219]
7. Preclinical development of DNDI-6174 a drug candidate for leishmaniasis [G2020-116]
8. First-In-Human Trial of the Pan-Malaria Transmission-Blocking Vaccine AnAPN1 [G2020-208]
9. Clinical development of placental malaria vaccine candidates [G2020-214]
10. Preclinical development of a monoclonal antibody to prevent P. falciparum malaria [G2021-111]
11. Co-delivery of Pfs230C1 and CSP with CoPoP, a versatile, potent liposomal adjuvant system for multistage malaria vaccine [G2019-111]
12. Immune therapy to prevent VL complications [G2018-111]
13. Preclinical development of a novel mechanism-of-action antimalarial drug with multistage activity [G2018-207]
14. Preclinical development of an anti-Dengue virus antibody that neutralizes all four serotypes [G2018-106]

Phase I

1. Clinical development of CpG-D35 for combined treatment of cutaneous leishmaniasis [G2020-108]
2. Development of AWZ1066S, A Small Molecule anti-Wolbachia Candidate Macrofilaricide Drug [G2019-202]
3. A schistosomiasis rapid diagnostic test to support control programs in monitoring treatment impact and reassessment mapping [G2020-104]
4. Field validation of Trypanosoma cruzi-LAMP: a molecular point-of-care test for the control of congenital Chagas disease [G2020-203]
5. A Buruli ulcer mycolactone (BU-MYCOLAC) rapid diagnostic test to enhance early diagnosis and treatment [G2020-202]
6. Product Development of LFA platform for improving sensitivity of Point-of-Care assays for infectious disease with main focus on TB [G2020-201]

POC

1. Adoption of Levo-Praziquantel 150mg for schistosomiasis by endemic countries [G2020-102]
2. A new treatment for Chagas disease [G2013-218]

Registration

1. Fujifilm SILVAMP TB LAM – A Sensitive point-of-care Tuberculosis Test [G2017-207]

20

Highlights in FY 2021

GHIT- PDPs
WE BINARS E R IE S

COVID-19 has drawn unprecedented global attention to the value of innovation
for life-threatening infectious diseases. The extraordinary speed of pandemic tool
development over the past two years demonstrates the innovation and scale thatare possible when stakeholders are incentivized to collaborate meaningfully, inspite of unrivaled challenges. We at GHIT are committed to leveraging the powerof this potential for R&D for neglected diseases.

Partnering with PDPs yields results
Over the last two decades, PDPs, which areinternational, non-profit organizations thatdevelop health technologies—drugs, vaccines,
diagnostics, vector controls, and devices—for
neglected diseases, have played a pivotal role in

To maintain momentum and foster dialogue about the R&D community’s role,

the global health R&D ecosystem. PDPs havechallenges, and opportunities in the fight against neglected diseases during—andsuccessfully registered and launched more thanfollowing— the COVID-19 pandemic era, GHIT convened a set of webinar series

85 innovative, new life-saving products inwith six Product Development Partnerships (PDPs) in FY 2021: Medicines for

LMICs. Recognizing the unique capabilities andMalaria Venture (MMV), Drugs for Neglected Diseases initiative (DNDi), TB

strengths of these entities, GHIT catalyzes andAlliance, Foundation for Innovative New Diagnostics (FIND), European Vaccine

invests in partnerships between PDPs andInitiative (EVI), PATH Malaria Vaccine Initiative (PATH MVI), and their

Japanese pharmaceutical companies, academia,

Japanese partners.

and research institutions dedicated to creating

During the webinar series, R&D experts from these PDPs addressed the criticalrole of each organization, unpacking their innovative R&D partnership models forneglected diseases and responses to future global health threats by utilizing theirexpertise, portfolios, and partner networks. Speakers reiterated that lessons learned
from the pandemic experience must be adopted for R&D for neglected diseases.
Please visit our website athttps://www.ghitfund.org/newsroom/webinar/pdp/en
and watch archive videos on YouTube.

innovative products for malaria, tuberculosis,
and NTDs by harnessing untappedchemical/natural compounds, technologies andinnovations in Japan. Since 2013, R&D
partnerships between PDPs and Japanese
partners accounts for over 60% of GHIT total
investment amount and 67% of invested
partnerships and multiple promising candidates
in GHIT’s portfolio, several of which will
emerge from the pipeline in the near future.

Collective voice from CEOs of six PDPs
“GHIT’s unique partnership model connects the Government of Japan and the Japanese pharmaceutical industry with global
foundations, academia, UN agencies and, of course, PDPs. It has grown from strength to strength, catalyzing synergies between
our organizations, establishing new scientific collaborations with Japanese R&D entities and providing us with much-needed
access to Japanese innovations, capacity, and investment. This has been crucial in supporting us, as PDPs, to develop a morerobust pipeline to fight against infectious diseases.”21

R&D without accessis valueless
The primary objective of GHIT’s Access and Delivery
strategy is to facilitate a seamless end-to-end passage of
products we fund for maximum impact and benefit to theintended populations. R&D and Access and Delivery are two

Research &Development

Leverage
Influence
Network
Catalyze

Acccess &DeliveryInnovation

Patient

GHIT provides funding to stimulate, catalyze
and incentivize research and development ofinnovative health technologies.

GHIT supports the development of robust
launch strategies (access and delivery plans)by the product development partners..

GHIT ensures that access considerations are
imbedded in the R&D process as early as
possible to facilitate future access & delivery.

GHIT Leverages its funding and Networks to
Influence and Catalyze strategic partnerships
for access and delivery to facilitate accessand delivery of innovative healthtechnologies to the intended population.

GHIT Funding de-links R&D costs from final
cost of the products.

sides of the same coin. R&D is the first step to access.
Through funding, GHIT stimulates, incentivizes, and catalyzes

clinical studies for arpraziquantel, the pediatric drug for

R&D of innovative technologies. While GHIT does notschistosomiasis, as well as the ongoing process for EMA

directly fund access and delivery, we work with productregulatory submission, planned for the Q4 2022. We continue

development partners to help them develop robust launch

to support the Pediatric Praziquantel Consortium in finalizing

strategies and establish strategic, product-focused partnerships

access and delivery plans, in part through co-funding thefor access and delivery.

ADOPT program with EDCTP, which is designed to assessthe different models of delivery for pediatric Praziquantel at

Fostering a global enabling environment

the country level. Additionally, GHIT continues to support the

GHIT works with global stakeholders with a focus on fostering

clinical development of the SILVAMPTM TBLAM rapid

an enabling environment for the uptake and scale-up of vital

diagnostic assay for tuberculosis and Fosravuconazole, ainnovative health technologies. Uniting Efforts for Innovation,

treatment option for eumycetoma, as well as the development

Access and Delivery (Uniting Efforts), a global platformof the respective access and delivery strategies for both ofco-convened since 2019 by the Government of Japan, thethese new health technologies. Furthermore, GHIT aims to

UNDP-lead Access and Delivery Partnership (ADP) and GHIT,

launch three diagnostic tools during GHIT 3.0 (FY

is the main platform for GHIT’s Global Access strategy. Uniting

2023-2027); these include the Chagas LAMP, the malaria

Efforts brings together, and promotes dialogue and partnerships

RDT, and the schistosomiasis RDT.

among, key stakeholders in funding, innovation, and access anddelivery of health technologies for neglected diseases. The aim

Looking toward the future

is to identify barriers to access, discuss solutions, drive greater

In 2022 and beyond, we continue our focus on the smoothcollaboration and progress for aligned action across theend-to-end delivery of GHIT-funded products, with a focus on

innovation–access–delivery continuum, and support the

impact-driven access and delivery strategies. Our global strategy

development of efficient, critical pathways for healthfor 2022 and beyond will leverage GHIT’s R&D funding andtechnology, from R&D to access and delivery, for maximum

networks to initiate, catalyze, and mobilize other stakeholders

impact. In 2021, Uniting Efforts cemented its collaboration withfor the paradigm shift we believe must take place in order tothe WHO NTD Department through partnering and co-fundingachieve sustainable access and delivery of health technologies

the development of a toolkit for national investment cases forfor neglected diseases. This shift features increased global

NTDs as a companion document to WHO’s 2021-2030 NTD

attention on, and investment in, R&D and access and delivery

roadmap. The toolkit prototype will be piloted in selectfor neglected infectious diseases. Where possible, GHIT will use

countries by the end of 2022. Uniting Efforts also commissioned

the products our funding has helped to create to showcase ana landscape analysis and technical paper on strategies to

end-to-end pathway from innovation to the patient.

1

improve the procurement of health technologies for NTDs.
Ending the neglect to attain the Sustainable Development Goals: a road map
for neglected tropical diseases 2021–2030. Available from:https://www.who.int/teams/control-of-neglected-tropical-diseases/ending-ntds-together-towards-2030

1

Product-specific access and delivery
This year, GHIT supported the successful completion of22

Highlights in FY 2021

Forbes Japan × GHIT FundFighting Infectious Diseases for a Sustainable Society:

What’s GHIT's Mission

23

In 2021, GHIT and Forbes JAPAN launched a series of

how Japan can contribute to global health through its

featured articles on Forbes JAPAN's website. The "Fighting

strengths and expertise in R&D, and each player’s pivotal

Infectious Diseases for a Sustainable Society: What’s GHIT'srole in the R&D ecosystem, as well as the significance ofMission" series collaboration engaged a wide range ofinternational collaboration and cross-sector partnerships. As

stakeholders with strong interests in contributing to SDG and

GHIT approaches its 10th anniversary in FY 2022,

ESG (Environment, Social, and Governance) issues, as wellstakeholders also discussed GHIT’s unique role, theas helped empower the next generation of aspiring global

significance of public-private and global partnerships, andhealth professionals. GHIT stakeholders, including experts on

their expectations of GHIT in the future. This content isglobal health R&D, shared their personal perspectives about

available in Japanese only.

the necessity of product development for neglected diseases,

https://forbesjapan.com/feat/ghitfund/

Article 1

Article 2

Article 3

Interview with Prof. Ken Ishii
Professor, Institute of Medical Science
University of Tokyo

Interview with Ms. Mihoko Kashiwakura
Head of East Asia Relations
Bill & Melinda Gates Foundation

Interview with Dr. Isao Teshirogi
President and CEO
Shionogi & Co., Ltd.

Article 4

Article 5Special Article

Interview with Dr. Wataru Akahata
CEO and Founder
VLP Therapeutics

Interview with
Mr. Fuminori Saga, President, ZVC JAPAN (Zoom)

Interview with
Dr. Hiroyuki Kojima, Senior Vice President,
Pharmaceutical Research and Technology Labs,
Pharmaceutical Technology, Astellas Pharma Inc.

Mr. Kuniaki Takemoto, Sales Director
Diligent Asia Pacific

Mr. Fumiya Domoto, Sustainability,
Astellas Pharma Inc.

ZVC JAPAN and Diligent Corporation
join as GHIT Sponsors

Building on our strong shared vision to contribute to

reduce its administrative costs, enabling us to allocate more

addressing global health challenges, we welcomed ZVC

resources to R&D investments.

JAPAN (Zoom) and Diligent Corporation as new sponsors
in FY 2021. Together, GHIT and these companies will

We will continue to create greater impacts by further

create new synergies to leverage cross-sectoral partnerships

expanding our partnerships across sectors and industries.

and improve our impact.
Our sponsorship program brings tangible benefits to bothsponsors and GHIT. For sponsor companies, the program
carries unparalleled opportunities for engagement in global
health issues in collaboration with GHIT. This public-private
partnership with GHIT is not only an opportunity forsponsors to contribute to the SDGs, especially SDG Goal 3

SDG Goal 3: Good health and well-being
Target 3.3 by 2030 end the epidemics ofAIDS, tuberculosis, malaria, andneglected tropical diseases and combat
hepatitis, water-borne diseases, and othercommunicable diseases& target 3.3, but it also serves their stakeholders from an
ESG perspective, especially in addressing “Social” issues.
For GHIT, these partnerships help us strengthen andSponsors

accelerate our activities by harnessing in-kind services andproducts that sponsors are uniquely positioned to provide.
These in-kind contributions also help GHIT significantly

Strengthen GHIT activities
and reduce administrative costby harnessing sponsors’ in-kind support

Contribute to the SDGs
in collaboration with GHIT

"We are pleased that Zoom's communication platform will seamlessly and securely connect GHIT
to the world, facilitating new ways of working and collaborating to further advance drug
development for the world's neglected people."
Mr. Fuminori Saga
President, ZVC Japan“We are very proud to be a part of the GHIT's mission to develop life-saving products forneglected infectious diseases in low-and middle- income countries. We recognize this is avery high-priority agenda in the SDGs. We at Diligent empower leaders, like GHIT, to drive
greater impacts, and strongly support the GHIT's innovative activities through our modern
governance tool.“

Mr. Kuniaki Takemoto
Sales Director
Diligent Asia Pacific24Finances
FY 2021 Financial summary
Balance Sheet
Assets (in millions)

JPY

USD

Current Assets
Fixed Assets

31.4
6,404.7

0.3
52.3

Total Assets

6,436.1

52.6

Liabilities (in millions)

JPY

USD

Current Liabilities
Non-current Liabilities

83.5
1.3

0.7
0.0

Total Liabilities

84.8

0.7

Net Assets (in millions)

JPY

USD

Designated Net Assets
General Net Assets

6,351.3-

51.9-

Total Net Assets
Total Liabilities and Net Assets

6,351.3
6,436.1

51.9
52.6

The US dollar amounts in this section represent translations of Japanese yen, solely for the reader’s convenience, at JPY122.41 = USD1, the exchange
rate as of March 31, 2022. This financial summary is an excerpt from the GHIT Fund's audited financial statements, which are audited by Ernst & Young
ShinNihon LLC..

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
Exchange Gain
Misc. Income

2,598.6
381.9
203.0
0.4

21.2
3.1
1.7
0.0

Total Ordinary Income

3,183.9

26.0

Operating Expenses
Management Expenses

3,101.2
135.1

25.3
1.1

Total Ordinary Expenses

3,236.3

26.4

Extraordinary Income

52.4

0.4

Total Extraordinary Income

52.4

0.4

Change in Designated Net Assets (in millions)

JPY

USD

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

2,747.9
901.9
764.6

22.4
7.4
6.2

Total Grants and Contributions Received

4,414.4

36.0

Ordinary Expenses

Extraordinary Income

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.

Takeshi Akahori

Hajime Inoue, MHS, MPH, DrPH

Jeremy Farrar, MD, PhD, FRCP

Astellas Pharma Inc.

Chugai Pharmaceutical Co., Ltd.

Director
Wellcome

Yoshihiko Hatanaka
Representative Director
Chairman of the Board

Tatsuro Kosaka
Senior Advisor

Daiichi Sankyo
Company, Limited

Eisai Co., Ltd.

Shionogi & Co., Ltd.

Haruo Naito
Representative Corporate Officer
and CEO

Isao Teshirogi, PhD
President and CEO

Ambassador, Director-General for
Global Issues Ministry of Foreign Affairs

George Nakayama
Senior Advisor and Corporate Advisor

Assistant Minister for
Global Health and Welfare
Ministry of Health, Labour and Welfare

Trevor Mundel, MD, PhD
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

Handa Professor of Global Health,and former Director, London School ofHygiene and Tropical Medicine

CEO, GHIT FundPeter Piot, MD, PhD

Mahima Datla

Satoshi Ezoe, MD, MPH, MPA, PhD

Daikichi Momma

Ann M. Veneman, JD

Vice Chairman
Institute for International Economic Studies

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

Visiting Professor
Keio University School of Medicine

Managing Director
Biological E. Limited

Supervisory Board Member

Saori Nakamura

Attorney at Law
Hirayama Nagareya Shirai Law Office

Ex-Officio

Richard Seabrook, PhD, MBA
Independent Senior Advisor,
Innovations, Wellcome
CEO 360Biomedical Ltd

27

Vice Chair

Hiroki Nakatani, MD, PhD, MHPEd

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

Supervisory Board Member

Ko-Yung Tung, JD

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

Osamu Kunii, MD, PhD, MPH

Yoshitaka Kitamura, MSc, EMBA
Director
Office of Global Health Cooperation
Ministry of Health, Labour and Welfare

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

Ann Mills-Duggan, PhD

Naoto Uemura, MD, PhD

Ralf Clemens, MD, PhD

Ken Duncan, PhD

Ken Ishii, MD, PhD

Timothy Jinks, PhD

Hiroo Koyama, PhD

Gerd Michel, PhD

Dennis Schmatz, PhD

Anna-Karin Tidén, PhD, MRSC

Rieko Yajima, PhD

Independent Consultant

Independent Vaccine Expert

Head, Infectious Diseases Interventions
Wellcome

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

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

Deputy Director
Discovery & Translational Sciences
Bill & Melinda Gates Foundation

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

Independent Medicinal Chemistry Expert

Independent Diagnostic Expert

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

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.

Osamu Kunii, MD, PhD, MPH

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, 2022
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.

Adriano Casulli

Hiroyoshi Endo

Kaoru Terashima

Roy Curtiss

Harajeshwar Kohli

Eric R. Houpt

Keertan Dheda

Rino Rappuoli

Ann Ginsberg

Yukihiro Akeda
Akinori Kimura

Alexandre Cuenat
Ami Shah Brown
Lewellys Barker
David BellBirgitte Giersing
Bruce Weniger
Boro Dropulic

Maria Elena Bottazzi
Brian Greenwood
Bruno Travi

Simon Campbell
Clif Barry

Christian Mandl
Carole Long

Christine Debouck
Charles Mowbray

Christian Ockenhouse
Charles Mgone
David Persing
David Sacks

David W. DenningDavid W. Dowdy
David Edwards
Dennis Kyle

David Fidock
Dyann Wirth

Dinesh Mondal

Dominick Pucci

Marco Albonico

Donald Peter McManus
Donato Zipeto

David Pompliano
David Soane

Lisette van Lieshout
Eric Chatelain
atlas support

Enrico Brunetti
Eric Rubin

Elizabeth Winzeler

Francesca Tamarozzi
Filip Dubovsky

Hermann Feldmeier

Federico Gomez de las HerasFred Luciano Neves Santos
Michael Frimpong
Fidel Zavala

Geoffrey N. GobertGovert J. van Dam
Carter Diggs

Gerald Spaeth

Gerd Pluschke
Gerd Michel

Glenda Gray

Giuseppe Pantaleo
Daniel Goldberg

D. Gray Heppner

Greg Matlashewski
Greg Matlashewski
Grant TheronGeorge Whitesides
Chris HentschelToshihiro Horii

James McCarthy
Julian Davies

Jennifer Keiser

Jennifer GardinerJames McKerrow
JoAnne Flynn

James A. Platts-Mills
Judy Sakanari
Judith Wilber
James LeDuc

Katsuhiko Mochizuki
Joseph Kamgno

Shigeyuki Kano

Subhash Kapre
David Kelso
Kent Kester

R. Kiplin Guy
KJ Singh

Peter Andersen
Lee HallLynn Soong

John Mansfield
Marcel Tanner
Meg Phillips

Martin Brusdeilins
Michael Free
Michael Free

Michael Witty

Michael Barrett

Joseph McCune
Kouichi Morita
Moriya Tsuji
Ivo Mueller

Mickey Urdea

Nancy Le Cam Bouveret
Nisha Garg

Niranjan Kanesa-thasan
Naoto Keicho

Nathalie Strub-Wourgaft
Nadia Tornieporth
Tsuyoshi Ogiku

Paul L.A.M. Corstjens
Philip ColePaul Kaye

Pedro AlonsoPeter RuminskiPeter KremsnerPeter Myler
Philip Hill

Peter Dailey
Polly Roy

Peter U. Fischer

Rashidul Haque
Ricardo Gazzinelli

Rebecca Richards Kortum
Paul Roepe
Sue Kinn

Sabine Ehrt

Sanjay Gurunathan
Sanjay Gurunathan
Sanjay Jain

Alejandro Gabriel Schijman
Sanjeev KrishnaGeorge Siber
Simon Croft

Somei Kojima
Stephen Ward
Stewart Cole

Yasuhiko Suzuki
Samuel Wanji

Takashi Fujitsu

Tamio Fujiwara
Takeshi Yura

Takushi Kaneko
Teun Bousema
Thomas Hawn

Thierry Diagana
Thomas Dick
Timothy Lu

Katsushi Tokunaga
Tom Brewer

Toshiyuki Miura

Takafumi Tsuboi

Wendy van de Sande

Mark Wansbrough-Jones
Tim WellsSamuel Wickline

Yoshihisa Hashiguchi
Kazuhisa Yoshimura
Zarifah Reed30

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
Photo credit: Dominic Chavez