BEST SCIENCE

1

FOR THE MOST NEGLECTED

GHIT PDP Webinar

Kaori Nakatani
Head of DNDi Japan
20 October 2021

www.dndi.org

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ORIGINS

Born on the frontlines
of medical action“DNDi was created in responseto the frustration of clinicians
and the desperation ofpatients faced with medicinesthat were ineffective, unsafe,unavailable, unaffordable, orthat had never beendeveloped at all.

The root of the problem?

The prevailing profit-oriented
model for medical researchand development (R&D) leaves
little incentive to develop
drugs for the poorest and mostvulnerable communities.

www.dndi.org

URGENT R&D NEEDS REMAIN UNADDRESSED

3For neglected diseases, a fatal imbalance remains

1975- 1999

1.1% of the 1,393 new drugs

were for neglected diseases
that represent
12% of the global disease burden

SOURCE: MSF & the DND Working Group, 2001. Fatal Imbalance: The Crisis in
R&D for Neglected Diseases. Médecins Sans Frontières.
www.dndi.org

4The “virtual orchestra”: a collaborative partnership model of R&D
ACADEMIA
We work together withuniversities, research institutes
and national research centres allaround the worldTREATMENT AND DIAGNOSTIC PROVIDERS
We partner with treatment and diagnosticproviders to ensure R&D responds to needs inthe field, and to encourage rapid deploymentof the new medical tools developed

ProductDevelopmentPartnerships(PDPs)PHARMACEUTICAL INDUSTRY
Close collaboration with pharmaceuticalcompanies, ranging from generics andbiotechs to “Big Pharma” on projects
spanning the whole drug R&D cycle fromdiscovery to access and delivery

PATIENTS AND COMMUNITIESPatient and community participation is key,in our projects we closely work withcommunity stakeholders

HEALTH MINISTRIES
Important partnerships involving definition of needs, co-sponsorship of clinical studies, andworking together to facilitate programme implementationwww.dndi.org

515 YEARS, 9 TREATMENTS, MILLIONS OF LIVES SAVED
We discover, develop, and accelerate access to urgently needed treatments for neglected patients, focusing on gaps forneglected tropical diseases and viral diseases that fuel cycles of poverty and disease in resource-constrained settings.
Treatments delivered

Clinical trials conducted
An average of 20
active clinicalstudies per year

field-adapted and affordable
treatments for 6 deadly diseases

R&D pipeline replenished

Research networks established

20+ NCEs•

5 clinical research networks•

4 million+
compounds screened•

4 lead optimization consortia•

800+ members COVID-19 ClinicalResearch Coalition

13 projects in Phase
III and registration

A healthy pipeline of drugcandidates for 8 deadly diseases

200+ partner
institutions in 40+
countries

Policies influenced
DNDi’s model, experience, andlessons learned documented
and disseminatedMobilizing any given year 1000+
partner FTEs (65% in Africa)••

Global partnerships forged

Diverse global team mobilized
A diverse global
team of 250+ staff
37 nationalities
across 4continents

New organization to fightdrug-resistant infections
DNDi joined forces with theWorld Health Organization(WHO) to create the
Global Antibiotic R&DPartnership (GARDP)

A unique footprint in LMICS
www.dndi.org

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WHAT DRIVES THE NEGLECT?

Our work is more urgent than everProfit-driven R&D leads to:••

Neglect to invest in unprofitable orchallenging segments, such as diseasesof poverty including neglected tropicaldiseases, paediatric formulations, andgender-responsive R&D

Emerging trends will affect the mostvulnerable people and the least
resourced settings most acutely:•Pandemic-prone diseases•

Climate-sensitive infectious diseases

Neglect to ensure timely, affordable,and equitable access worldwide,
including in resource-limited settings

www.dndi.org

3 Pillars

DNDi Strategic Plan

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We innovate
to save lives

We foster inclusive &sustainable solutions

We advocate
for change

We discover and develop urgently
needed treatments for neglectedpatients, and work to ensure they areaffordable, available, and adapted to thecommunities who need them

We work hand-in-hand with partners inlow- and middle-income countries
to power our progress and strengthen
innovation ecosystems that putpeople’s needs firstWe speak out for policy change toenable more effective and equitable
R&D and access to the fruits of science
for all people, no matter their income
or where they live

www.dndi.org

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DNDi – Innovating to save lives
We discover, develop, and accelerate access to urgently needed treatments forneglected patients, focusing on gaps for neglected tropical diseases and viral diseases
that fuel cycles of poverty and disease in resource-constrained settings.

SLEEPING SICKNESSLEISHMANIASISCHAGAS DISEASEFILARIA (RIVER BLINDNESS)

MYCETOMAHIV

Advance progress toward
breaking the cycle oftransmission

Prevent devastating
amputation and disability

HEPATITISCOVID-19/ PANDEMIC
PRONE DISEASES

Accelerate sustainable disease
elimination

Help make treatment a reality
for millions of people waiting
for a cure

Deliver safer, simpler treatments
to save lives and reduce socialstigma

Speed tools to save lives,
especially in resource-limited
settings

Contribute to eliminating Chagas asa public health problem

Ensure access to lifesaving
treatment for children and people
with advanced HIV

NEW AREAS TO BE
EXPLORED: Dengue,
schistosomiasis, snakebitewww.dndi.org

JUNE 2021 R&D PORTFOLIOJapan Office activity

Role of DNDi Japan office
DISCOVERY
ScreenHit-to-lead

TRANSLATION
Lead
optimization

Pre-clinical

Phase IDEVELOPMENT
Phase IIa/
Proof-of-concept

SCYX-1330682
SCYX-1608210

SLEEPING
SICKNESS
Leishmaniasis
Hit-to-lead
NTD Drug Discovery
Booster Hit-to-lead

LEISHMANIASIS

Screening

CF series
LeishmaniasisS07 series
L205 series
DNDI-5421
DNDI-5610

DNDI-6174
GSK3494245
DDD1305143

DNDI-0690

Chagas disease
Hit-to-lead
NTD Drug Discovery
Booster Hit-to-leadOxaborole
C205series profiling

Treatment accessFexinidazole for T.b.gambiense*

Fexinidazole forT.b. rhodesiense

Nifurtimox-eflornithine combination
therapy (NECT)*New VL treatments(Latin America)
New treatments
for HIV/VL

SSG&PM (East Africa)*
New VL treatments (South Asia)*

Miltefosine +paromomycin
combination (Africa)

CpG-D35 for CL

Biomarkers

Registration

IMPLEMENTATIONAcoziborole

New treatments
for PKDL

GSK899 DDD853651
Novartis LXE408

Amino pyrazoles

Phase IIb/III

New CLcombination

DNDI-6148

GSK245 DDD1305143
CHAGASDISEASE

9Fexinidazole

New benznidazole
regimens

Benznidazole paediatric dosage forms*

UW series
Daiichi Sankyo
seriesScreening

Macrofilaricide
CC6166

FILARIA:RIVER
BLINDNESS

Oxfendazole

Emodepside
TylAMac(ABBV-4083)
Fosravuconazole

MYCETOMA
5 FC (cryptococcal
meningitis)HIV

4-in-1(ABC/3TC/LPV/r)Super-booster therapy for children
with HIV/TB*
2-in-1 LPV/r pellets and ABC/3TC or
AZT/3TC

Ravidasvir +sofosbuvir

HEPATITIS C
COVID 19

Hit-to-lead

Pre-Clinical

ANTICOV
Fixed-dose combination ASMQ*MALARIA
Implementation transferred to the Medicines for Malaria Venture in 2015

Ravidasvir

NCE (New chemical entity)* Treatments delivered by DNDi

www.dndi.org

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IMPACT OF GHIT ON DNDI

Summary of DNDi Japan projects (mostly GHIT funded!)
Start

2021

2020

2019

2018

2017

PartnerStage

DiseaseMitsubishi Tanabe

HTL

Leish/Chagas

TakedaLO

Leish (VL)

Eisai

PreC

Leish

U Tokyo

Scr

Leish (VL)U Tokyo, GeneDesign

Clin

Leish (CL)

LOChagas

Scr

AMR

Daiichi Sankyo Novare

Scr

Leish/Chagas

Mitsubishi Tanabe

Scr

Leish/Chagas

Eisai
TakedaScr

AMR

TakedaPreC

Leish (VL)

Eisai
Shionogi
TakedaAstellas

HTL

Leish/Chagas(Booster III)

Daiichi Sankyo Novare

Scr

Leish/Chagas

Eisai
GeneDesign

Clin
PreC

Mycetoma
Leish (CL)Eisai
Shionogi
TakedaHTL

Leish/Chagas(Booster II)
Leish/Chagas
Leish/Chagas

Daiichi Sankyo

Daiichi Sankyo
Riken

HTL
TRP

Start

2016

Partner
GeneDesign
Daiichi Sankyo Novare

StagePreC
Scr

DiseaseLeish (CL)
Leish/Chagas

Daiichi Sankyo

Scr

Leish/Chagas

Eisai

Scr

Leish/Chagas

Eisai

Clin

Mycetoma

TakedaLO

Leish (VL)

Eisai, Shionogi, Takeda

HTL

Leish/Chagas(Booster I)

2015

Osaka U, Gene Design,PreC
Nagasaki U

Leish (CL)

Eisai

Clin

Chagas

Eisai

Scr

Leish/Chagas

TakedaScr

Leish/Chagas

Kitasato U

Scr

Leish/Chagas

IMC

Scr

Leish/Chagas

GeneDesign

TRP

Leish/Chagas

U Tokyo, JICA

CapB

Leish (VL) (Bangladesh)

2009

Eisai

Clin

Chagas2006

Kitasato U

Scr, CapB HAT, Leish

2005

Kitasato U

Scr

2014

2013

2012

HAT

www.dndi.org