Doing our part: Innovating to fight Neglected Tropical

Doing our part:
Innovating to fight
Neglected Tropical Diseases
April 2017
Designed by ACW, London
www.acw.uk.com
Contents
BIOPHARMACEUTICAL INDUSTRY
CONTRIBUTIONS TO THE GLOBAL
FIGHT AGAINST NTDs
4
8
Staying the Course: A Global
Commitment to Fight NTDs
- The scope of the NTD epidemic
- The London Declaration
16
18
Scaling Up Access to
Existing Treatments
Strengthening Health
Systems
Boosting Innovation
- Discovery
- Pipeline
- Funding R&D PIPELINE FOR NTDs
20
22
24
26
28
30
American trypanosomiasis
(Chagas disease) Chikungunya
Dengue
(dengue hemorrhagic fever)
Human African
trypanosomiasis
(sleeping sickness)
Leishmaniasis
Lymphatic filariasis
32
34
36
38
40
42
Mycetoma
(river blindness)
Onchocerciasis
(river blindness)
Rabies
Schistosomiasis
Trachoma
Abbreviations
STAYING THE COURSE:
A GLOBAL COMMITMENT
TO FIGHT NTDs
We are witnessing one of the greatest public health
achievements of the century. This year marks the 5th
anniversary of the World Health Organization’s (WHO)
Neglected Tropical Diseases (NTD) Roadmap1, establishing
targets and milestones for the control and elimination
of ten of the most prevalent NTDs by 2020.
Inspired by the WHO NTD Roadmap, a group of
20 partners from governments, intergovernmental
organizations, NGOs, foundations and R&D
biopharmaceutical companies came together to pledge
support to the WHO by signing the London Declaration2.
Today, the London Declaration’s endorsers have grown
to include over 200 organizations3.
“Today, we have joined together to increase
the impact of our investments and build on
the tremendous progress made to date.
This innovative approach must serve as a
model for solving other global development
challenges and will help millions of people build
self-sufficiency and overcome the need for aid.”
Bill Gates, co-chair of the Bill & Melinda Gates Foundation,
at the signing of the London Declaration in 2012
Now at the halfway mark of the WHO NTD
Roadmap’s timeline, this publication outlines how
the R&D biopharmaceutical industry is contributing
to this global agenda, with active research projects
to uncover new or improved treatments and vaccines.
It also provides context of how these R&D efforts
are part of an integrated approach to combatting
NTDs, including an unprecedented medicines
donation program of 14 billion treatments over
ten years and support for local capacity building.
THE SCOPE OF THE NTD EPIDEMIC
Although most NTDs are preventable and treatable,
they sadly continue to be a heavy burden on the
most vulnerable, disadvantaged people in the world.
One person in seven suffers from one or more NTDs
– comparable to the entire population of Europe –
with the vast majority of cases in low- and middleincome countries (LMICs).
NTDs are caused by a range of different parasites,
bacteria and viruses, which primarily thrive in subtropical
climates. They can be painful, blinding and disfiguring;
each year they lead to the poor-health, disability
and death of hundreds of thousands of people.
As they are often chronic and disabling, NTDs have
enormous educational and economic impacts, keeping
children out of school and adults out of work. At the
start of this millennium, it was estimated that in India
alone, an average of USD 1 billion was lost to lymphatic
filariasis each year due to healthcare costs and loss of
productivity. Research has shown that school children
infected with intestinal worms had a 20% lower
probability of school enrolment and, subsequently,
a 40% reduction in income as an adult4.
The landscape of neglected disease control is
continuously changing. Climate change is driving
diseases beyond their traditional geographies, resulting
in outbreaks of dengue and Chagas disease beyond the
tropics. Furthermore, complex changes in demography,
such as urbanization and globalization, impact upon
the spread of disease, as illustrated by the recent
outbreaks of Chikungunya, Ebola and Zika virus.
NTDs mire communities in a cycle of poverty, and hinder
progress towards the sustainable development agenda.
The increased commitments from national governments
and their partners to achieve the targets of the WHO
NTD Roadmap means significant improvements to the
health, wealth and quality of life of over 1 billion
people worldwide.
4
THE LONDON DECL AR ATION
The London Declaration represented a turning point in global
efforts to control and eliminate the most common NTDs. The
R&D biopharmaceutical industry remains a committed partner in this
agenda IFPMA signatories to the London Declaration are AbbVie, Bayer,
Bristol-Myers Squibb, Eisai, GlaxoSmithKline, Johnson & Johnson,
Merck, MSD, Novartis, Pfizer and Sanofi.
COMMITMENTS OF THE LONDON DECL ARATION ON NTDs
Sustain, expand and extend programs that
ensure the necessary supply of drugs and other
interventions to help eradicate some diseases
and to help control others by 2020.
Advance R&D through partnerships and provision
of funding to find next-generation treatments
and interventions for neglected diseases.
Enhance collaboration and coordination on NTDs
at national and international levels through
public and private multilateral organizations.
Enable adequate funding with endemic countries
to implement NTD programs necessary to achieve
these goals, supported by strong and committed
health systems at the national level.
Provide technical support, tools and
resources to support NTD-endemic countries
to evaluate and monitor programs.
AT THE HALFWAY MARK, THE WORLD IS ON TRACK TO
DELIVERING ON PROMISES OF THE LONDON DECL ARATION 5
• In 2015, biopharmaceutical companies donated an estimated 2.4 billion
tablets, enough for 1.5 billion treatments to prevent and treat NTDs –
an increase of 11.7% from 2014.
• Between 2012 and 2014, the number of people who needed treatment
decreased by 230 million.
• Since the London Declaration, there have been over 7.9 billion tablets
in pharma donations, which is enough for 5 billion treatments.
• 87% of countries in Africa have been fully mapped for the London
Declaration’s targeted NTDs.
1 http://www.who.int/neglected_diseases/NTD_RoadMap_2012_Fullversion.pdf
2 h ttp://unitingtocombatntds.org/sites/default/files/resource_file/london_
declaration_on_ntds.pdf
3 http://unitingtocombatntds.org/endorsements
4 h ttp://www.globalnetwork.org/sites/default/files/Social%20and%20
Economic%20Impact%20Review%20on%20Neglected%20Tropical%20
Diseases%20Hudson%20Institute%20and%20Sabin%20Institute%20
November%202012_1.pdf
5 http://unitingtocombatntds.org/report/fourth-report-reaching-unreached
5
DOING OUR PART:
COMPREHENSIVE EFFORTS
TO FIGHT NTDs
Scaling up access to
existing treatments
Donation of 14 billion treatments
over 10 years to help eliminate
or control 10 NTDs
Strengthening
health systems
Boosting innovation
Over 40 health
partnerships to build
capacity to fight NTDs where
they are endemic
109 active R&D projects to
develop the next generation
of medicines and vaccines
for NTDs
NTDs require a multi-stakeholder approach to drive further
research tailored to developing needs, improve health
policies and access to treatments, and boost healthcare
system capacity. That is why the R&D biopharmaceutical
industry combats NTDs in an integrated manner.
6
7
1
BOOSTING
INNOVATION
DISCOV ERY
There has been considerable progress in new technologies, including
medicines, vaccines, diagnostics and pesticides, to combat NTDs.
Since the launch of the London Declaration in 2012, a number of
new product approvals helped better address NTD challenges.
DENGUE (2015)
Dengue is a threat to nearly
half of the world’s population, yet
until recently there was no specific
treatment or vaccine available to
reduce the burden of this disease.
Dengue is the fastest progressing
vector-borne disease and can cause
massive outbreaks with a disruptive
effect on healthcare systems.
Mostly asymptomatic, dengue can
also lead to hospitalization, serious
illness and death among people
leaving in endemic areas in Asia
Latin America and Africa.
The newly approved dengue vaccine6
presents a major advance towards
the achievement of WHO objectives
to reducing dengue mortality and
morbidity by at least 50% and 25%
respectively, by 2020. Additional
dengue vaccine candidates are
in development.
6 Dengvaxia® (CYD-TDV), developed by Sanofi Pasteur.
7 VERMOX™ chewable (mebendazole chewable 500 mg tablets),
developed by Janssen Pharmaceuticals, Inc.
8 Eisai Co., Ltd. received prequalification from the WHO for diethylcarbamazine
citrate (DEC) 100 mg tablets in 2013.
8
SOIL-TR ANSMIT TED
HELMINTHIA SES (2016)
LYMPHATIC
FIL ARIA SIS (2013)
Soil-transmitted helminthiases,
otherwise known as intestinal
worms, are a chronic and debilitating
illness with particular impact
on children, stunting growth,
impairing cognitive development
and keeping children out of school.
Lymphatic filariasis is often
referred to as elephantiasis, due to
severe disfigurement, disability, and
swelling from fluid build-up caused
by improper functioning of the
lymphatic system. Infection
occurs when filarial parasites are
transmitted to humans through
mosquitoes. An estimated 120
million people worldwide live
with the disease.
The approval of a chewable
formulation of mebendezole7
supports global efforts to reduce
the burden of parasitic infections
in young children. Donations of this
treatment are planned to be rolled
out towards the WHO’s objective
to provide treatment to over 75%
of 870 million at-risk children.
The development and distribution
of diethylcarbamazine citrate
(DEC)8 through large-scale mass
drug administration supports the
WHO target of eliminating the
disease by 2020.
109
7
7 COMPOUNDS FOR
NTDs CURRENTLY
UNDERGO L ATE STAGE
TESTING
109 ACTIVE R&D
PROJECTS FOR NTDs
To ensure that new generations of improved treatments
and interventions are discovered, despite low commercial
incentives in the area of NTDs, the biopharmaceutical
industry engages in a variety of multi-sectoral research
models. The most common model sees NTD R&D through
partnerships. This innovative approach has proven to
enable the sharing of expertise and acceleration of research,
reduced risks and duplications, and sustainable financing.
90
%
OVER 90% OF THE ACTIVE
R&D PROJECTS FOR NTDs
ARE COLL ABOR ATIVE
EFFORTS
50
OVER 50 PARTNERS
( UNIVERSITIES, NGOs,
PUBLIC AND PRIVATE
SECTOR INSTITUTES )
Today, the industry’s NTD research efforts are done in
partnership with over 50 organizations, including renowned
universities, non-governmental organizations and public
and private sector institutes.
Biopharmaceutical R&D can be a lengthy process,
with instances of life-changing discovery amidst years
of hypotheses, setback and recalibrations. As the science
becomes more complex, research becomes more challenging.
As well as responding to emerging trends such as globalization
and climate change, local needs also demand innovation
in the space of pediatric formulations and medicines and
vaccines that can be distributed outside of the cold chain.
Research partnerships help streamline global efforts,
identify remaining R&D gaps and preventing the
duplication of efforts.
Adding to their R&D efforts and donations, companies
provide in-kind contributions that are specifically targeted
to NTDs R&D. This includes sharing intellectual property
assets such as compounds and compounds libraries for
research purposes, giving access to research facilities,
hosting scientists, and providing training. It also includes
transfer of technology, and building technical expertise
to develop, manufacture, register and distribute
NTDs products.
9
THE R&D PROCESS
RESEARCH
Practical Testing
Critical Trials
Regulatory
Review
Scale-up to
Manufacturing
Post-Marketing
Surveillance
4-6 YEARS
1 YEAR
PHASE IV
5
PHASE I
PHASE II
PHASE III
6-7 YEARS
NEW DRUG APPLICATION SUBMITTED
Target identification (TI)
Lead generation (LG)
Lead identification (LI)
Lead optimization (LO)
250
PATENT APPLICATIONS FILED
BASIC RESEARCH & DRUG DISCOVERY
5,000 - 10,000
COMPOUNDS
INVESTIGATIONAL DRUG APPLICATION SUBMITTED
PHASE 0
Early Phase
Research
DEVELOPMENT
ONE
MARKETED
MEDICINE
0.5-2 YEARS
CONTINUOUS
PIPELINE
IFPMA companies are involved in 109 active R&D projects for NTDs. Nearing
the end of what is on average a 10-15 year R&D process, IFPMA member
companies and their partners are approaching imminent NTDs breakthroughs,
with late stage testing (Phase III) of treatments and vaccines of 7 compounds,
within the disease areas of American Trypanosomiasis (Chagas disease),
dengue, Human African trypanosomiasis (sleeping sickness), lymphatic
filariasis, rabies, and trachoma.
In most cases, research undertaken in partnerships is conducted on a
not-for‑profit basis, with effective mechanisms to ensure access to treatments
in endemic countries. As stated by the 2016 Access to Medicines Index, there
are access plans in place for most high-priority pipeline products9.
“Overall, 56% of 151 high-priority, low-incentive
products in R&D have access plans in place. As
expected, there are more products with access
plans toward the end of the pipeline; there is a
marked increase as projects move into clinical
development, and then again between clinical
phases II and III. The majority (72%) of latestage projects have access plans in place.”
Access to Medicines Index 2016
9 https://accesstomedicineindex.org/
10
As more projects progress into later stages of clinical development,
continued support in the space of capacity building will be key to ensure
that clinical trials and regulatory infrastructures in low- and middle-income
countries are capable getting new medicines and vaccines to the people
that need them.
The final section of this publication provides the most up-to-date record
of active research and development for the next generation of medicines
and vaccines for the following NTDs: American trypanosomiasis (Chagas
disease), Chikungunya, dengue, Human African trypanosomiasis (sleeping
sickness), leishmaniasis, lymphatic filariasis, mycetoma, onchocerciasis
(river blindness), rabies, schistosomiasis, and trachoma.
R&D PIPELINE BY INDUSTRY AND PARTNER S FOR NTDs
Schistosomiasis
Rabies
Current projects: 5
Medicines: 5
Trachoma
Current projects: 1
Medicines: 1
Current projects: 1
Vaccines: 1
Onchocerciasis
(river blindness)
American trypanosomiasis
(Chagas disease)
Current projects: 28
Medicines: 26
Vaccines: 2
Current projects: 15
Medicines: 15
Mycetoma
Current projects: 1
Medicines: 1
Chikungunya
Current projects: 4
Medicines: 3
Vaccines: 1
Lymphatic filariasis
Current projects: 12
Medicines: 12
Dengue
(dengue hemorrhagic fever)
Current projects: 12
Medicines: 5
Vaccines: 7
Leishmaniasis
Current projects: 21
Medicines: 21
Human African trypanosomiasis
(sleeping sickness)
Current projects: 9
Medicines: 9
IFPMA companies are involved in 109 active R&D projects for NTDs
11
FUNDING
Eliminating NTDs requires sustained funding for
research and development of new treatments and
vaccines. According to the latest G-FINDER report
from Policy Cures Research, which measures global
investment into R&D of new products for 39 neglected
diseases (including malaria, tuberculosis, and HIV/
AIDs10), USD 3.04 billion was made available for
neglected disease research in 2015, as well as an
additional USD 631 million invested in Ebola and
other African viral hemorrhagic fever (VHF) research.
“2015 was the fourth year in a row that industry
has increased its investment in neglected disease
R&D – the only sector to have recorded
year-on-year growth for such a stretch.”
Policy Cures Research, G-FINDER 2016
• Industry investment in neglected disease R&D in 2015
was the highest ever recorded in the G-FINDER survey.
• In 2015 the pharmaceutical industry contributed USD
471 million to funding for neglected disease research.
10 Full list of diseases on page 11 of the G-FIN D ER report.
12
13
DEDIC ATED INTERNATIONAL INITIATIV ES FOR NTD RESE ARCH
GLOBAL HE ALTH INNOVATIV E
TECHNOLOGY FUND (GHIT ), FACILITATING
INTERNATIONAL R&D
The first of its kind in Japan, the GHIT Fund is a
public-private partnership between the Japanese
government, multiple pharmaceutical companies,
the Bill & Melinda Gates Foundation, the Wellcome
Trust, and United Nations Development Programme
(UNDP). GHIT Fund invests and manages a portfolio of
development partnerships aimed at neglected diseases
that afflict the world’s poorest people. GHIT Fund
mobilizes Japanese and international pharmaceutical
companies and academic and research organizations
to engage in the effort to get new medicines, vaccines,
and diagnostic tools to people who need them most.
IFPMA member companies involved in GHIT are:
Astellas, Chugai, Eisai, Daiichi Sankyo, Merck, Otsuka,
Shionogi, and Takeda.
For more information please visit www.ghitfund.org
WIPO RE:SE ARCH, A COLL ABORATIVE
PL ATFORM TO BOOST R&D
WIPO Re:Search was established in October 2011 by
the World Intellectual Property Organization (WIPO)
in collaboration with BIO Ventures for Global Health
(BVGH) and with the active participation of leading
pharmaceutical companies. The consortium now consists
of over 100 organizations from both the private and
public sectors, including non-profit organizations and
academic research institutes. WIPO Re:Search member
organizations share their intellectual property assets
such as compounds and compound libraries, technologies,
expertise, samples, and data to bridge research gaps,
reduce costs of product development, and advance drug,
vaccine, and diagnostic research and development for
NTDs, malaria, and tuberculosis.
To date BVGH has established 105 research collaborations
through WIPO Re:Search. Collaborations cover a range
of areas that include the sharing of data and services
(such as access to company research facilities, screening
of compounds, hosting of scientists) as well as other
forms of expertise.
IFPMA member companies involved in WIPO Re:Search
are: Eisai, GlaxoSmithKline, Janssen (Johnson & Johnson),
Merck, MSD, Novartis, Pfizer, Sanofi, and Takeda11.
THE EUROPE AN & DE V ELOPING COUNTRIES CLINIC AL
TRIAL S PARTNER SHIP ( EDC TP ) & THE SPECIAL
PROGR AMME FOR RESE ARCH AND TR AINING IN
TROPIC AL DISE A SES ( WHO/TDR ), SUPPORTING
RESEARCHERS AND CLINIC AL TRIAL RESEARCH TEAMS
FROM LOW- AND MIDDLE- INCOME COUNTRIES ( LMICs)
EDCTP and WHO/TDR offer a Clinical Research and Development Fellowship.
The overall objective is to strengthen collaboration between research
institutions, researchers and clinical staff in LMICs, pharmaceutical
companies, and Product Development Partnerships (PDPs)12.
By working together, they ensure harmonization and synergy through a
Joint Call for Proposals. The host organizations, pharmaceutical companies
and PDPs train scientists, for a period of up to 24 months to develop specialist
product development skills not readily taught in academic centers or public
research institutions. The fellows are expected to become an important
resource for institutional capacity development, to undertake and
manage clinical research in accordance with international regulatory
requirements and standards13.
The fellowship has increased the number of individuals trained and
helped facilitate common communication with researchers and clinical
staff, pharmaceutical companies, PDPs and research institutions.
IFPMA member companies involved in training fellows for the year
2015/2016 are: Astellas, Bayer, GlaxoSmithKline, Janssen (Johnson
& Johnson), Merck, Novartis, and Sanofi.
For more information please visit
EDTCP:
www.edctp.org/call/edctp-tdr-clinical-research-development-fellowships-2/
For more information please visit
TDR:
www.who.int/tdr/capacity/strengthening/career_development/en/
WIPO: www.wipo.int/research/en/
Dedicated centers for NTD research
11 http://www.wipo.int/research/en/
12 http://www.edctp.org/web/app/uploads/2016/07/EDCTP2-Workplan-2016.pdf
13 http://www.edctp.org/call/edctp-tdr-clinical-research-development-fellowships-2/
14
PHARMACEUTIC AL INDUSTRY CENTER S DEDIC ATED TO NTDs R&D
COMPANY
R&D CENTER
LOCATION
SINCE
AbbVie
AbbVie
North Chicago, IL
2009
Cambridge Biomedical Campus (CBC)
Cambridge, UK
2015
Alderley Park*
Macclesfield, UK
1999
Celgene
Celgene Global Health
Summit, NJ, USA
2009
GlaxoSmithKline
Diseases of the Developing World center
Tres Cantos, Spain
2002
Merck
R&D Translational Innovation Platform
“Global Health”
Geneva, Switzerland
2014
MSD
(operated as Merck & Co.,
Inc. in the USA and Canada)
MSD Wellcome Trust Hilleman Laboratories
New Delhi, India
2009
Novartis Institute for Tropical Diseases (NITD)
Singapore**
2002
Novartis Institutes for BioMedical Research
(NIBR)
Emeryville, CA, USA
2016
Genomics Institute of the Novartis Research
Foundation (GNF)
La Jolla, USA
2010
Eisai Inc. Andover Research Institute
Andover, MA, USA
1987
Eisai Pharmaceuticals India Pvt. Ltd.
Visakhapatnam, India
2007
Tsukuba Research Laboratories
(Ibaraki Prefecture)
Japan
1982
Marcy l’Etoile Research & development Campus
Lyon, France
Vaccines (dengue)
since the 90s
Medicines since
2015
AstraZeneca
Novartis
Eisai
Sanofi
The biopharmaceutical industry’s efforts are supported by R&D centers which are dedicated
solely to diseases that disproportionately affect people in low- and middle-income countries.
Some companies integrate these R&D activities within their broader R&D organization while
others provide financial and technical support to independent organizations.
* T he facility is planned to cease its operations in late 2016/early 2017; currently ongoing projects are those
involving AWOL, malaria and tuberculosis.
** In October 2016, Novartis has announced that NITD will move its operations to Emeryville, California, where it
will be co-located with the Infectious Disease Research team.
15
2
SCALING UP ACCESS TO
EXISTING TREATMENTS
As we search for the next generation of treatments and vaccines, we need
to continue to utilize to their full power the existing cost-effective and
high-impact treatments for NTDs.
The R&D biopharmaceutical industry is to delivering on its London
Declaration pledge of 14 billion treatments through 2020 to address the
10 diseases responsible for more than 90% of the global neglected disease
burden. In 2015 alone, biopharmaceutical companies donated an estimated
1.5 billion treatments to prevent and treat neglected tropical diseases –
an increase of 11.7% from 2014 and reaching 850 million people worldwide14.
By 2020, nearly USD 18 billion worth of medicines will have been
distributed, the largest medicine donation the world has ever seen.
By 2020, nearly USD 18 billion
worth of medicines will have been
distributed, the largest medicine
donation the world has ever seen.
Five of the 10 NTDs covered in the London Declaration
can be controlled through what is known as mass drug
administration (MDA) – large-scale population treatment
with safe and effective medicines to all people living
in high-risk areas. These diseases include: lymphatic
filariasis, onchocerciasis, schistosomiasis, soil-transmitted
helminthiases (hookworm, roundworm, and whipworm),
and trachoma.
The success of these drug distribution campaigns relies on an integrated
treatment approach. In the past, many countries used to conduct separate
treatment campaigns for individual diseases. Now, many programs provide
treatments for several diseases at the same time. For example, a national
program can treat all children in a region for intestinal worms, onchocerciasis
and lymphatic filariasis in a single school visit. This provides immediate
relief to any children that happen to carry the parasites, while at the same
time halting the spread of these diseases within a community. After a few
years of systematic mass drug administrations, a disease can be eradicated
from a country, and even eliminated entirely across the world.
Additionally, these large-scale campaigns offer a fantastic opportunity to
reach people with other health interventions. Partners are also looking at
ways to use these community distribution platforms in a more coordinated
way with other health programs.
The other five NTDs can be controlled by what is known as innovative and
intensified disease management (IDM) – individual diagnosis, treatment and
rehabilitation of people that have been infected. These include Chagas
disease, Guinea worm disease, Human African trypanosomiasis (sleeping
sickness), leprosy and visceral leishmaniasis.
14 http://unitingtocombatntds.org/impact.
16
17
3
STRENGTHENING
HEALTH SYSTEMS
NTDs control and elimination is a challenging task with a need for a
variety of locally-tailored solutions. Adding to their research and development
efforts and donations, IFPMA member companies are also involved in over
40 partnerships to assist countries to bolster the capacity of their health
workforce and medical infrastructures to meet the needs of people with
NTDs. Political commitment and leadership from national programs are
paramount. All projects involve collaboration with national governments
and community-based platforms.
Political commitment and
leadership from national
programs are paramount.
All projects involve collaboration
with national governments and
community-based platforms.
Programs focus on building health services that are
accessible and staffed with qualified healthcare workers
to enable the delivery of medicines and vaccines down
the last mile. They also provide people living in endemic
areas information to help prevent the spread of infection,
and infrastructure programmes to address water,
sanitation and hygiene.
So that every effort is made to support the WHO and
national governments in meetings targets of the NTD
Roadmap, the R&D biopharmaceutical industry is
committed to finding new ways to address gaps,
share best practices and advance solutions.
These partnerships include supporting local NGOs in establishing campaigns
around identifying the symptoms of dengue, supporting public health
institutes in development of clinical guidelines on treatment of leprosy,
and supporting governments in setting up mobile intervention teams that
can rapidly respond to outbreaks of Human African trypanosomiasis.
For more information about these programs, and to learn of many
other initiatives, please visit the IFPMA Health Partnerships Directory
(www.partnerships.ifpma.org), the most comprehensive international
database for health development programs involving the R&D
biopharmaceutical industry. Each partnership profile offers valuable
insights into why a specific program was developed, and the ways in which
it is helping to make a difference to communities around the world.
18
19
AMERICAN
TRYPANOSOMIASIS
Chagas disease
DISE A SE IMPAC T 15,16
American trypanosomiasis is caused by the kinetoplastid protozoan parasite
Trypanosoma cruzi, which is primarily transmitted by a triatomine bug –
an insect vector. The vector-borne transmission occurs primarily in
the Americas. Other ways of transmission are blood transfusion, organ
transplantation, congenital and oral transmissions. The disease has two
clinical stages: acute and chronic. Acute is characterized by fever, malaise,
facial oedema, generalized lymphadenopathy, and hepatosplenomegaly.
Chronic asymptomatic disease shows no signs of the disease, yet the
parasite remains transmittable to others. Chronic symptomatic disease
develops in 10% to 30% of infected patients. Trypanosoma cruzi infection is
curable if treatment is initiated soon after infection. In the chronic phase
antiparasitic treatment can also prevent or curb disease progression.
KE Y FAC TS17, 18
• A n estimated 6 million to 7 million people are infected worldwide, with the disease being endemic, but not limited
to 21 countries across Latin America.
•5
% of children die with the acute stage of the disease, while chronic asymptomatic disease can last 10 years to lifetime.
• Up to 30% of chronically infected people develop cardiac alterations and up to 10% develop digestive, neurological
or mixed alterations, which may require specific treatment.
• Vector control is the most useful method to prevent Chagas disease in Latin America.
15 http://www.dndi.org/diseases-projects/chagas/
16 http://www.who.int/mediacentre/factsheets/fs340/en/
20
17 http://www.dndi.org/diseases-projects/chagas/
18 http://www.who.int/mediacentre/factsheets/fs340/en/
CURRENT R&D PROJEC TS
COMPA
COMPAN
NYY
PA
PARTNER
RTNERSS
PROJECTT
PROJEC
PHAASE
SE
PH
PE
TTYYPE
AbbVie
AbbVie
DNDi
DNDi
Compoundscreening,
screening,preclinical
preclinicalsupport,
support,
Compound
technicalconsulting
consulting
technical
Leadidentification
identification
Lead
Medicine
Medicine
Astellas
Astellas
AIST
AIST
Discovery
Discoveryof
ofanti-protozoan
anti-protozoanparasite
parasitedrugs
drugsfor
for
Trypanosoma
Trypanosomacruzi
cruzi
Discovery
Discovery
Medicine
Medicine
AstraZeneca
AstraZeneca
DNDi
DNDi
Focused
Focusedcompound
compoundlibrary
libraryscreening
screeningat
atSwiss
Swiss
TPH,
TPH,Inst.
Inst.Pasteur
PasteurKK
Lead
Leadidentification
identification
Medicine
Medicine
Bayer
Bayer
Company
company
Lampit,
Lampit,Nifurtimox
Nifurtimoxpediatric
pediatric(dosing
(dosingin
inchildren)
children)
Phase
PhaseIII
III
Medicine
Medicine
Development of treatments
Development of treatments
Lead optimization
Lead optimization
Medicine
Medicine
Screening program
Screening program
Screening program (Natural Products Library)
Screening program (Natural Products Library)
E1224
E1224
Hit identification
Hit identification
Hit identification
Hit identification
Phase II
Phase II
Medicine
Medicine
Medicine
Medicine
Medicine
Medicine
DNDi, GHIT (Shionogi,
DNDi,
GHIT,
(Shionogi,GHIT)
Takeda,
AstraZeneca,
Takeda, AstraZeneca, GHIT)
NTD Drug Discovery Booster (DDB-Chagas)
NTD Drug Discovery Booster (DDB-Chagas)
Lead generation
Lead generation
Medicine
Medicine
Broad Inst., GHIT
Broad Inst., GHIT
Focused compound library screening
Focused compound library screening
Drug discovery
Drug discovery
(optimization)
(optimization)
Medicine
Medicine
SVI, Baylor College, Aeras,
SVI,
GHITBaylor College, Aeras,
GHIT
Adjuvant to support vaccine development:
Adjuvant
to support
vaccine development:
Chagas
vaccine
with SVI
Chagas vaccine with SVI
Adjuvant to support vaccine development:
Chagas
vaccine
with Fiocruz
Adjuvant
to support
vaccine development:
Chagas vaccine with Fiocruz
Lead optimization project
Lead optimization project
Lead optimization III for Chagas disease
Lead optimization III for Chagas disease
Preclinical
Preclinical
Vaccine
Vaccine
Preclinical
Preclinical
Vaccine
Vaccine
Lead optimization
Lead optimization
Lead optimization
Lead optimization
Medicine
Medicine
Medicine
Medicine
Lead identification
Lead identification
Medicine
Medicine
Lead identification
Lead identification
Medicine
Medicine
Discovery
Discovery
Medicine
Medicine
Discovery
(tool)
Basic research
Medicine
Medicine
Lead identification
Discovery
Medicine
Medicine
Drug discovery
Discovery
Medicine
Medicine
Drug discovery
Basic research
Medicine
Medicine
Celgene
Celgene
Daiichi Sankyo
Daiichi Sankyo
Eisai
Eisai
DNDi, Antwerp
Antwerp Uni,
Uni, Epichem,
Epichem,
DNDi,
Monash Uni
Monash Uni
GHIT, DNDi
GHIT, DNDi
DNDi
DNDi
DNDi, GHIT
DNDi, GHIT
Fiocruz, GHIT
Fiocruz, GHIT
GlaxoSmithKline
GlaxoSmithKline
Dundee Uni
Dundee Uni
Wellcome
Wellcome
Wellcome/Dundee Uni
(Drug Discovery Unit)
Wellcome/Dundee
Uni
(Drug Discovery Unit)
Wellcome
Wellcome
TCOLF, Calibr
TCOLF, Calibr
Merck
TCOLF,Swiss
LSHTM
DNDi,
TPH
MSD
DNDi
TCOLF, Georgia Uni
Hit
Hitto
tolead
Leadidentification
identification Wellcome & Dundee
Uni (Drug Discovery Unit)
Hit to lead identification
Hit to Lead identification Wellcome
Develop novel therapies to prevent and treat
Develop&novel
therapies to prevent and treat
Chagas
leishmaniasis
Chagas & leishmaniasis
Mode of action and target identification of
Concept generation
hit finding
anti-Chagasic
compounds
Targeted
screening
structure–activity
Rapid
selection
of inand
vivohit
active
antirelationship
(SAR)
development
Trypanosoma cruzi compounds
company
DNDi, Swiss TPH
Chagas disease project 1: developing safe
Identify new molecules using high throughput
treatment for Chagas disease
screening of molecular libraries and genotypic
targets using CRISPR Cas9 technologies
Chagas disease project 2: developing safe
treatment for Chagas disease
Concept generation hit finding
CDIPD (UCSD), WIPO
DNDi
Re:Search
PDE5
In
vivoinhibitors
POC
Drug discovery
Discovery
Medicine
Medicine
CONICET
DNDi
Targeted screening and hit structure–activity
Compound screening
relationship (SAR) development
Leadidentification
optimization
Lead
Medicine
Medicine
Novartis
DNDi
Wellcome
Focused compound library screening
Developing safe treatment for Chagas disease
Lead generation
Drug discovery
Medicine
Medicine
Shionogi
Sanofi
GHIT, DNDi
DNDi
NTD Drug Discovery Booster
Focused compound library screening
Drug discovery
Lead generation
Medicine
Medicine
Shionogi
DNDi,
GHIT
GHIT, DNDi
Compound
screeningBooster
NTD
Drug Discovery
Drug discovery
Drug
(leaddiscovery
generation)
Medicine
Medicine
DNDi, GHIT
GHIT
DNDi,
Compound
screening Booster
NTD Drug Discovery
Drug
Drugdiscovery
discovery
(lead
(leadgeneration)
generation)
Medicine
Medicine
DNDi,
GHIT trypanosomiasis (Chagas
NTD Drug
Discovery
Booster
Total R&D projects for
American
disease):
27
Drug discovery
(lead generation)
Medicine
Janssen (J&J)
Novartis
Merck
Pfizer
MSD
Sanofi
Takeda
Takeda
company
GHIT, DNDi
Total R&D projects for American trypanosomiasis (Chagas disease): 28
21
CHIKUNGUNYA
DISE A SE IMPAC T 19,20
Chikungunya is a viral disease usually transmitted to humans by infected
mosquitoes Ae. aegypti and Ae. albopictus. Other insect vectors include
species of A. furcifer-taylori group and A. luteocephalus. There is evidence that
some animals, including non-primates, rodents, birds, and small mammals,
may act as reservoirs. The disease causes fever and severe joint pain.
Other symptoms include muscle pain, joint swelling, headache, nausea,
fatigue, and rash. Chikungunya disease does not often result in death, but
the symptoms can be severe and disabling. Some clinical signs are shared
with dengue, and it can be misdiagnosed in areas where dengue is common.
There is no cure for the disease. Treatment is focused on relieving the symptoms.
KE Y FAC TS21, 22
• I n 2016, about 31,000 cases were reported to the Pan American Health Organization.
•P
eople at highest risk for more severe disease include newborns, elderly (≥65 years old), and people with
medical conditions such as high blood pressure, diabetes, or heart disease.
•S
ymptoms usually begin 3-7 days after being bitten by an infected mosquito.
• Ae. aegypti mosquitoes are confined within the tropics and sub-tropics, Ae. albopictus kind has spread from Asia
to become established in areas of Africa, Europe, and the Americas, in the recent decades.
CURRENT R&D PROJEC TS
COMPA N Y
PA RTNER S
PROJEC T
PH A SE
T Y PE
Sanofi
Vanderbilt Uni
Discovery: operations research
Lead optimization
Medicine
Celgene
A-WOL (Wolbachia), NEB
Compound screening
Screening
Medicine
MSD
USAMRIID
Targeted compound screening
Lead identification
Medicine
Takeda
Zydus Cadilla
Development
Preclinical & Phase I
Vaccine
Total R&D projects for Chikungunya (C): 4
19 https://www.cdc.gov/chikungunya/symptoms/
20 http://www.who.int/mediacentre/factsheets/fs327/en/
22
21 https://www.cdc.gov/chikungunya/symptoms/
22 http://www.who.int/mediacentre/factsheets/fs327/en/
CURRENT R&D PROJEC TS
COMPA N Y
PA RTNER S
PROJEC T
PH A SE
T Y PE
AbbVie
DNDi
Compound screening, preclinical support,
technical consulting
Lead identification
Medicine
Astellas
AIST
Discovery of anti-protozoan parasite drugs for
Trypanosoma cruzi
Discovery
Medicine
AstraZeneca
DNDi
Focused compound library screening at Swiss
TPH, Inst. Pasteur K
Lead identification
Medicine
Bayer
company
Lampit, Nifurtimox pediatric (dosing in children)
Phase III
Medicine
Development of treatments
Lead optimization
Medicine
GHIT, DNDi
Screening program
Hit identification
Medicine
DNDi
Screening program (Natural Products Library)
Hit identification
Medicine
DNDi, GHIT
E1224
Phase II
Medicine
DNDi, GHIT, (Shionogi,
Takeda, AstraZeneca, GHIT)
NTD Drug Discovery Booster (DDB-Chagas)
Lead generation
Medicine
Broad Inst., GHIT
Focused compound library screening
Drug discovery
(optimization)
Medicine
SVI, Baylor College, Aeras,
GHIT
Adjuvant to support vaccine development:
Chagas vaccine with SVI
Preclinical
Vaccine
Fiocruz, GHIT
Adjuvant to support vaccine development:
Chagas vaccine with Fiocruz
Preclinical
Vaccine
Dundee Uni
Lead optimization project
Lead optimization
Medicine
Wellcome
Lead optimization III for Chagas disease
Lead optimization
Medicine
Wellcome/Dundee Uni
(Drug Discovery Unit)
Hit to Lead identification Wellcome & Dundee
Uni (Drug Discovery Unit)
Lead identification
Medicine
Wellcome
Hit to Lead identification Wellcome
Lead identification
Medicine
TCOLF, Calibr
Develop novel therapies to prevent and treat
Chagas & leishmaniasis
Discovery
Medicine
Merck
DNDi, Swiss TPH
Concept generation hit finding
Basic research
Medicine
MSD
DNDi
Targeted screening and hit structure–activity
relationship (SAR) development
Lead identification
Medicine
company
Chagas disease project 1: developing safe
treatment for Chagas disease
Drug discovery
Medicine
company
Chagas disease project 2: developing safe
treatment for Chagas disease
Drug discovery
Medicine
CDIPD (UCSD), WIPO
Re:Search
PDE5 inhibitors
Drug discovery
Medicine
CONICET
Compound screening
Lead optimization
Medicine
DNDi
Focused compound library screening
Lead generation
Medicine
GHIT, DNDi
NTD Drug Discovery Booster
Drug discovery
Medicine
DNDi, GHIT
Compound screening
Drug discovery
(lead generation)
Medicine
DNDi, GHIT
NTD Drug Discovery Booster
Drug discovery
(lead generation)
Medicine
Celgene
Daiichi Sankyo
Eisai
GlaxoSmithKline
DNDi, Antwerp Uni, Epichem,
Monash Uni
Novartis
Pfizer
Sanofi
Shionogi
Takeda
Total R&D projects for American trypanosomiasis (Chagas disease): 27
23
DENGUE
Dengue hemorrhagic fever
DISE A SE IMPAC T 23
Dengue is a mosquito-borne viral infection which causes flu-like illness,
and occasionally develops into a potentially lethal complication – severe
dengue. The global incidence of dengue has grown dramatically in recent
decades. About half of the world’s population is now at risk. There is no
specific treatment for dengue/severe dengue. Dengue prevention and control
depends on effective vector control measures. A dengue vaccine has been
licensed by several National Regulatory Authorities for use in people 9-45
years of age living in endemic settings.
KE Y FAC TS24
• An estimated 390 million dengue infections occur worldwide each year, with about 96 million resulting in illness.
• Dengue is found in tropical and sub-tropical climates worldwide, mostly in urban and semi-urban areas.
• Severe dengue is a leading cause of serious illness and death among children in some Asian and Latin
American countries.
• Early detection and access to proper medical care lowers fatality rates below 1%.
23 http://www.who.int/mediacentre/factsheets/fs117/en/
24 https://www.cdc.gov/chikungunya/symptoms/
24
CURRENT R&D PROJEC TS
COMPA N Y
PA RTNER S
PROJEC T
PH A SE
T Y PE
COMPA N Y
AbbVie
PA RTNER S
DNDi
PROJEC T
Compound screening, preclinical support,
technical consulting
PH A SE
Lead identification
T Y PE
Medicine
Astellas
Celgene
AIST
A-WOL (Wolbachia), NEB
Discovery of anti-protozoan parasite drugs for
Trypanosoma
cruzi
Compound screening
Discovery
Screening
Medicine
Medicine
AstraZeneca
DNDi
Focused compound library screening at Swiss
TPH, Inst. Pasteur K
Lead identification
Medicine
Bayer
company
WRAIR, Fiocruz
DNDi, Antwerp Uni, Epichem,
Lampit,
pediatric
(dosing
in children)
Dengue Nifurtimox
purified and
inactivated
virus
vaccine candidate
Phase
Phase III
I
(WRAIR antigens)
Medicine
Vaccine
Development of treatments
Lead optimization
Medicine
DNDi, GHIT
E1224
Hit identification
Preclinical
(GlaxoSmithKline
Hit
identification
antigens)
Phase II
Medicine
WRAIR, Fiocruz
DNDi
Screening program
Dengue purified and inactivated virus
Screening
program (Natural Products Library)
vaccine candidate
Celgene
Daiichi Sankyo
GlaxoSmithKline
Monash Uni
GHIT, DNDi
Vaccine
Medicine
Medicine
DNDi, GHIT, (Shionogi,
Takeda, AstraZeneca, GHIT)
WRAIR, Fiocruz
Broad Inst., GHIT
Dengue therapeutic antibody
Focused compound library screening
SVI, Baylor College, Aeras,
GHIT
Wellcome, KU Leuven
Adjuvant to support vaccine development:
Chagas vaccine with SVI
Discovery new anti-viral small molecules
Preclinical
Discovery
Vaccine
Medicine
Fiocruz, GHIT
Adjuvant to support vaccine development:
Chagas vaccine with Fiocruz
Preclinical
Vaccine
Dundee Uni
Company
Wellcome
Lead optimization project
Tetravalent subunit
Lead optimization III for Chagas disease
Lead optimization
Phase I
Lead optimization
Medicine
Vaccine
Medicine
Wellcome/Dundee Uni
(Drug Discovery Unit)
NIAID
Wellcome
Hit to Lead identification Wellcome & Dundee
Uni (Drug Discovery Unit)
Tetravalent live attenuated
Hit to Lead identification Wellcome
Lead identification
Late stage
preclinical
Lead
identification
Medicine
Vaccine
Medicine
TCOLF, Calibr
Develop novel therapies to prevent and treat
Chagas & leishmaniasis
Discovery
Medicine
Merck
USAMRIID
DNDi,
Swiss TPH
Target compound
Concept
generation hit finding
Lead identification
Basic
research
Medicine
Medicine
MSD
DNDi
Targeted screening and hit structure–activity
relationship (SAR) development
Lead identification
Medicine
Novartis
Company
company
Discovering
newproject
anti-viral
treatmentssafe
Chagas
disease
1: developing
treatment for Chagas disease
Drug discovery
Drug discovery
Medicine
Medicine
Medicine
PDE5 inhibitors
Drug discovery
Phase III
(efficacy completed)
Drug discovery
CONICET
Compound screening
Lead optimization
Medicine
Hokkaido University
DNDi
Discovering new anti-viral treatment
Focused compound library screening
Drug discovery
Lead generation
Medicine
Medicine
Shionogi
GHIT, DNDi
NTD Drug Discovery Booster
Drug discovery
Medicine
Takeda
Takeda
DNDi,
GHIT
Company
Four-strain screening
recombinant viral vaccine
Compound
(TAK003)
Drug discovery
Phasegeneration)
III
(lead
Medicine
Vaccine
DNDi, GHIT
NTD Drug Discovery Booster
Drug discovery
(lead generation)
Medicine
Eisai
Janssen (J&J)
GlaxoSmithKline
MSD
Novartis
company
Sanofi
Pfizer
Sanofi
Shionogi
Company
CDIPD (UCSD), WIPO
Re:Search
NTD Drug Discovery Booster (DDB-Chagas)
Chagas disease project 2: developing safe
treatment for Chagas disease
Tetravalent live attenuated chimeric vaccine
Lead generation
Late state
preclinical
Drug
discovery
(optimization)
Medicine
Vaccine
Medicine
Vaccine
Medicine
Total R&D projects for American
trypanosomiasis
(Chagas
27
dengue/dengue
hemorrhagic
fever: disease):
12
25
HUMAN AFRICAN
TRYPANOSOMIASIS
Sleeping sickness
DISE A SE IMPAC T 25
Human African trypanosomiasis (HAT), also known as “sleeping sickness”,
is a parasitic disease transmitted by the bite of the “Glossina” insect (tsetse fly).
The disease invades the central nervous system, and is predominantly found
among the poor populations living in remote rural areas of Africa, exposed
to the tsetse fly though activities such as agriculture, fishing, animal
husbandry, or hunting. Other methods of transmission include mother-tochild infection through the placenta, mechanical transmission through other
blood-sucking insects, and transmission of the parasite through sexual
contact.Diagnosis and treatment of the disease is complex and requires
specifically skilled staff. Untreated, it is usually fatal. HAT exists in two
forms, depending on the parasite involved. Trypanosoma brucei gambiense
infects a person for months or years without major signs or symptoms of
the disease. The other form, Trypanosoma brucei rhodesiense causes an
acute infection with first symptoms appearing within weeks or months.
KE Y FAC TS26, 27
• The disease occurs in 36 sub-Saharan Africa countries, with 3,796 cases recorded in 2014.
• Trypanosoma brucei gambiense is found in 24 countries and accounts for more than 98% of reported cases.
• Trypanosoma brucei rhodesiense is found in 13 countries in eastern and southern Africa and accounts for under
2% of reported cases.
• Uganda is the only country that presents both forms of the disease in separate zones.
25 http://www.who.int/trypanosomiasis_african/disease/en/
26 http://www.who.int/trypanosomiasis_african/disease/en/
26
27 http://www.who.int/mediacentre/factsheets/fs259/en/
CURRENT R&D PROJEC TS
COMPA N Y
PA RTNER S
PROJEC T
PH A SE
T Y PE
COMPA N Y
AbbVie
PA RTNER S
DNDi
Compound
PROJEC T screening, preclinical support,
technical consulting
PH A SE
Lead
identification
TMedicine
Y PE
Astellas
AIST
Discovery of anti-protozoan parasite drugs for
Trypanosoma cruzi
Discovery
Medicine
AstraZeneca
AbbVie
DNDi
Focused compound library screening at Swiss
Compound screening
TPH, Inst. Pasteur K
Lead
identification
Screening
Medicine
Medicine
Bayer
company
Lampit, Nifurtimox pediatric (dosing in children)
Phase III
Medicine
Celgene
DNDi, Antwerp Uni, Epichem,
Monash
Uni
DNDi, Antwerp
Uni, Epichem,
Monash Uni
GHIT, DNDi
Development of treatments
Lead optimization
Medicine
Development of treatments
Screening program
Lead optimization
Hit identification
Medicine
Medicine
DNDi
Screening program (Natural Products Library)
Hit identification
Medicine
DNDi, GHIT
E1224
Phase II
Medicine
TCOLFGHIT, (Shionogi,
DNDi,
Takeda, AstraZeneca, GHIT)
Drug discovery
NTD Drug Discovery Booster (DDB-Chagas)
Discovery
Lead generation
Medicine
Medicine
Broad Inst., GHIT
Focused compound library screening
Drug discovery
(optimization)
Medicine
SVI, Baylor College, Aeras,
TCOLF, NEU, CSIC
GHIT
Adjuvant
to support
vaccine
development:
T. brucei drug
discovery:
ADMET
and PK
Chagas
with SVI
supportvaccine
for hit-to-lead
optimization
Preclinical
Lead optimization
Vaccine
Medicine
Fiocruz, GHIT
Adjuvant to support vaccine development:
Chagas vaccine with Fiocruz
Preclinical
Vaccine
Dundee Uni
Lead optimization project
Lead optimization
Medicine
Wellcome
TCOLF, Monash Uni
Hit tooptimization
lead optimization
kinetoplastid
Lead
III for for
Chagas
disease
diseases: single agents for Chagas and HAT
Lead
optimization
Discovery
Medicine
Medicine
Wellcome/Dundee Uni
(Drug Discovery Unit)
Hit to Lead identification Wellcome & Dundee
Uni (Drug Discovery Unit)
Lead identification
Medicine
Wellcome
Hit to Lead identification Wellcome
Lead identification
Medicine
Merck
DNDi, Swiss
TCOLF,
CalibrTPH
Develop
novel therapies
to prevent and treat
Concept generation
hit finding
Chagas & leishmaniasis
Basic research
Discovery
Medicine
Medicine
Merck
DNDi, Swiss TPH
Concept generation hit finding
Basic research
Medicine
MSD
DNDi
Targeted screening and hit structure–activity
relationship (SAR) development
Discovering (D) new affordable and
adaptable
NCEsproject 1: developing safe
Chagas
disease
treatment for Chagas disease
Lead identification
Drug discovery
(lead optimization)
Drug discovery
Medicine
Medicine
company
Chagas disease project 2: developing safe
treatment for Chagas disease
Drug discovery
Medicine
CDIPD
DNDi (UCSD), WIPO
Re:Search
Fexinidazole
(antiprotozoal compound)
PDE5
inhibitors
Phasediscovery
II / III
Drug
Medicine
Medicine
CONICET
Compound screening
Lead optimization
Medicine
DNDi
Focused compound library screening
Lead generation
Medicine
DNDi, DNDi
GHIT
GHIT,
Compound
screeningBooster
NTD
Drug Discovery
Lead identification
Drug
discovery
Medicine
Medicine
DNDi, GHIT
Compound screening
Drug discovery
(lead generation)
Medicine
Drug discovery
(lead generation)
Medicine
Celgene
Daiichi Sankyo
Eisai
GlaxoSmithKline
GlaxoSmithKline
Novartis
Wellcome
company
Novartis
Sanofi
Pfizer
Sanofi
Takeda
Shionogi
Takeda
DNDi, GHIT
NTD Drug Discovery Booster
Total R&D projects for Human African trypanosomiasis (sleeping sickness): 9
Medicine
Total R&D projects for American trypanosomiasis (Chagas disease): 27
27
LEISHMANIASIS
DISE A SE IMPAC T 28,29
Leishmaniasis exists in three main forms – visceral (also known as
“kala-azar”), a lethal form of the disease, cutaneous (the most common),
and mucocutaneous. Cutaneous leishmaniasis usually presents as ulcers on
exposed body parts (arms, legs, face). Mucocutaneous leishmaniasis affects
the skin and mucous membrane causing severe deformations. Leishmaniasis
is caused by the protozoan Leishmania parasites, which are transmitted by
the bite of infected female phlebotomine sandflies. The disease affects
some of the poorest people on earth, and is associated with malnutrition,
population displacement, poor housing, a weak immune system and lack of
financial resources. Leishmaniasis is also linked to environmental changes
such as deforestation, building of dams, irrigation schemes, and urbanization.
KE Y FAC TS30, 31
• A n estimated 900,000 to 1.3 million new cases occur annually with around 20,000 to 30,000 deaths occurring
in 98 countries.
• More than 20 species of the kinetoplastid protozoan parasite Leishmania can be transmitted to humans by some
30 species of phlebotomine sandflies.
• O ver 90% of new cases of visceral leishmaniasis occur within the 7 most affected countries – Bangladesh, Brazil,
Ethiopia, India, Kenya, Nepal, and Sudan.
• The majority of cases of cutaneous leishmaniasis occur in 10 countries – Afghanistan, Algeria, Brazil, Colombia,
Costa Rica, Ethiopia, Iran, Peru, Syria, and Sudan.
28 http://www.dndi.org/diseases-projects/leishmaniasis/
29 http://www.who.int/mediacentre/factsheets/fs375/en/
28
30 http://www.dndi.org/diseases-projects/leishmaniasis/
31 http://www.who.int/mediacentre/factsheets/fs375/en/
CURRENT R&D PROJEC TS
COMPA N Y
PA RTNER S
PROJEC T
PH A SE
T Y PE
AbbVie
DNDi
Compound screening, preclinical support,
technical consulting
Discovery
Medicine
AstraZeneca
DNDi
Focused compound library screening at Inst.
Pasteur K, Dundee Uni, Swiss TPH
Lead identification
Medicine
Celgene
LSHTM, DNDi
Development of treatments
Lead optimization
Medicine
GHIT, DNDi
Screening program
Hit identification
Medicine
DNDi
Screening program
(Natural Products Library)
Hit identification
Medicine
DNDi, GHIT
(Shionogi, Takeda, AstraZeneca)
NTD Drug Discovery Booster (DDBLeishmaniasis)
Lead generation
Medicine
Wellcome/ Dundee Uni (Drug
Discovery Unit)
Hit to lead identification
Lead identification
Medicine
Wellcome
Hit to lead identification
Lead identification
Medicine
TCOLF, Calibr
Develop novel therapies to prevent and treat
Chagas & leishmaniasis
Discovery
Medicine
Wellcome/ Dundee Uni
(Drug Discovery Unit)
Lead optimization I for visceral leishmaniasis
Lead optimization
Medicine
Wellcome/ Dundee Uni
(Drug Discovery Unit)
Lead optimization II for visceral leishmaniasis
Lead optimization
Medicine
TCOLF, Uni Leon, CBMSO
In-vitro screening for treating leishmaniasis
Discovery
Medicine
Merck
DNDi, Swiss TPH
Concept generation hit finding
Basic research
Medicine
MSD
DNDi
Target screening and hit structure–activity
relationship (SAR) development
Lead identification
Medicine
Novartis
Wellcome
Leishmaniasis project: discovering a superior
treatment
Drug discovery
Medicine
DNDi
Focused compound library screening
Lead generation
Medicine
DNDi
Compound screening
Lead identification
Medicine
GHIT, DNDi
NTD Drug Discovery Booster
Drug discovery
Medicine
DNDi, GHIT
Compound screening
Drug discovery (lead
generation)
Medicine
DNDi, GHIT
NTD Drug Discovery Booster
Drug discovery (lead
generation)
Medicine
DNDi, GHIT
Lead optimization of the aminopyrazole series
for visceral leishmaniasis
Drug discovery (lead
optimization)
Medicine
Daiichi Sankyo
Eisai
GlaxoSmithKline
Sanofi
Shionogi
Takeda
Total R&D projects for leishmaniasis: 21
29
LYMPHATIC
FILARIASIS
DISE A SE IMPAC T 32
Lymphatic filariasis (elephantiasis) is caused by infection with nematode
parasites (roundworms) of the family Filariodidea. There are three types
of these thread-like filarial worms: Wuchereria bancrofti, Brugia malayi, and
Brugia timori. The disease can result in an altered lymphatic system and the
abnormal enlargement of body parts (lymphedema), causing pain, severe
disability, and social stigma. Infection occurs when filarial parasites are
transmitted to humans through mosquitoes. Infection is usually acquired
in childhood causing hidden damage to the lymphatic system and leading
to eventual permanent disability. Lymphatic filariasis can be eliminated by
stopping the spread of infection through preventive chemotherapy with
single doses of two medicines for persons living in areas where the infection
is present. A basic, recommended package of care can alleviate suffering
and prevent further disability among lymphatic filariasis patients.
KE Y FAC TS33
• A n estimated 15 million people are afflicted with lymphedema globally.
• 1.10 billion people in 55 countries worldwide remain threatened by lymphatic filariasis and require preventive
chemotherapy to stop the spread of this parasitic infection.
• 5.63 billion treatments have been delivered to stop the spread of infection since 2000.
• Wuchereria bancrofti is responsible for 90% of the cases.
• 25 million men globally suffer with genital form of the disease.
• 8 out of 73 endemic countries are currently under surveillance to demonstrate that elimination has been achieved.
32 http://www.dndi.org/diseases-projects/chagas/
33 http://www.who.int/mediacentre/factsheets/fs102/en/
30
CURRENT R&D PROJEC TS
COMPA N Y
PA RTNER S
PROJEC T
PH A SE
T Y PE
LSTM
Collaborative preclinical development
program
Discovery
Medicine
DNDi
Compound screening
Lead identification
Medicine
A-WOL, LSTM
Focused compound library screening and
DMPK support
Lead identification
Medicine
Monclair State Uni, DNDi,
NPIMR, Bonn Uni
Development of treatments
Lead optimization
Medicine
A-WOL (Wolbachia), NEB
Compound screening
Screening
Medicine
UCSD, UCSF, LSTM, Bonn Uni,
AbbVie, Anacor, Johnson &
Johnson, Eisai, Merck, Calibr,
DNDi, BMGF
Development of Macrofilaricide Drug
Accerelator (MacDA)
Lead identification
Medicine
Company
Development of diethylcarbamazine citrate
(DEC)
Post-approval
surveillance (stability
study for shelf-life
extension)
Medicine
LSTM, Liv Uni, GHIT
Novel compounds for anti-Wolbachia
Lead optimization
Medicine
DNDi, NPIMR
Concept generation hit finding
Basic research
Medicine
DNDi
Target compound screening
Lead identification
Medicine
DNDi
In vivo POC studies macrofilaricide
drug accelerator
Discovery
Medicine
DNDi
Focused compound library screening
Lead generation
Medicine
AbbVie
AstraZeneca
Celgene
Eisai
Merck
MSD
Sanofi
Total R&D projects for lymphatic filariasis: 12
31
MYCETOMA
DISE A SE IMPAC T 34,35
Mycetoma (also known as Madura foot / maduromycosis / maduramycosis)
is a slow-growing bacterial or fungal infection, which exists in two forms:
Actinomycetoma and Eumycetoma. It is a chronic, progressively destructive
morbid inflammatory disease usually of the foot but any part of the body
can be affected. Infection is most probably acquired when the causative
organisms of Mycetoma enter the body through minor trauma or a penetrating
injury. The disease is characterized by a triad of painless subcutaneous mass,
multiple sinuses, and discharge containing grains. It usually spreads to
involve the skin, deep structures, and bone, resulting in destruction, deformity,
and loss of function, which may be fatal. Mycetoma commonly involves the
extremities, back, and gluteal region. There is a clear relationship between
Mycetoma and individuals who walk barefoot. The disease has numerous
adverse medical, health and socioeconomic impacts on patients,
communities, and health authorities.
KE Y FAC TS36, 37
•M
ycetoma commonly affects young adults, particularly males aged between 20 and 40 years.
•A
ctinomycetoma form is a bacterial infection with an approximate 90% cure rate using antibiotics, Eumycetoma
form is a fungal infection with a 25-35% cure rate with antifungals and surgery.
•A
pproximately 40% of Mycetoma cases worldwide are Eumycotic.
•C
ausative organisms of Mycetoma are distributed worldwide but are endemic in tropical and subtropical areas in the
“Mycetoma belt”, which includes the Bolivarian Republic of Venezuela, Chad, Ethiopia, India, Mauritania, Mexico,
Senegal, Somalia, Sudan, and Yemen.
CURRENT R&D PROJEC TS
COMPA N Y
PA RTNER S
PROJEC T
PH A SE
T Y PE
Eisai
DNDi
E1224
Clinical (preparation for phase II)
Medicine
Total R&D projects for mycetoma: 1
34 http://www.who.int/buruli/mycetoma/en/
35 http://www.dndi.org/diseases-projects/mycetoma/
32
36 http://www.who.int/buruli/mycetoma/en/
37 http://www.dndi.org/diseases-projects/mycetoma/
CURRENT R&D PROJEC TS
COMPA N Y
PA RTNER S
PROJEC T
PH A SE
T Y PE
AbbVie
DNDi
Compound screening, preclinical support,
technical consulting
Lead identification
Medicine
Astellas
AIST
Discovery of anti-protozoan parasite drugs for
Trypanosoma cruzi
Discovery
Medicine
AstraZeneca
DNDi
Focused compound library screening at Swiss
TPH, Inst. Pasteur K
Lead identification
Medicine
Bayer
company
Lampit, Nifurtimox pediatric (dosing in children)
Phase III
Medicine
Development of treatments
Lead optimization
Medicine
GHIT, DNDi
Screening program
Hit identification
Medicine
DNDi
Screening program (Natural Products Library)
Hit identification
Medicine
DNDi, GHIT
E1224
Phase II
Medicine
DNDi, GHIT, (Shionogi,
Takeda, AstraZeneca, GHIT)
NTD Drug Discovery Booster (DDB-Chagas)
Lead generation
Medicine
Broad Inst., GHIT
Focused compound library screening
Drug discovery
(optimization)
Medicine
SVI, Baylor College, Aeras,
GHIT
Adjuvant to support vaccine development:
Chagas vaccine with SVI
Preclinical
Vaccine
Fiocruz, GHIT
Adjuvant to support vaccine development:
Chagas vaccine with Fiocruz
Preclinical
Vaccine
Dundee Uni
Lead optimization project
Lead optimization
Medicine
Wellcome
Lead optimization III for Chagas disease
Lead optimization
Medicine
Wellcome/Dundee Uni
(Drug Discovery Unit)
Hit to Lead identification Wellcome & Dundee
Uni (Drug Discovery Unit)
Lead identification
Medicine
Wellcome
Hit to Lead identification Wellcome
Lead identification
Medicine
TCOLF, Calibr
Develop novel therapies to prevent and treat
Chagas & leishmaniasis
Discovery
Medicine
Merck
DNDi, Swiss TPH
Concept generation hit finding
Basic research
Medicine
MSD
DNDi
Targeted screening and hit structure–activity
relationship (SAR) development
Lead identification
Medicine
company
Chagas disease project 1: developing safe
treatment for Chagas disease
Drug discovery
Medicine
company
Chagas disease project 2: developing safe
treatment for Chagas disease
Drug discovery
Medicine
CDIPD (UCSD), WIPO
Re:Search
PDE5 inhibitors
Drug discovery
Medicine
CONICET
Compound screening
Lead optimization
Medicine
DNDi
Focused compound library screening
Lead generation
Medicine
GHIT, DNDi
NTD Drug Discovery Booster
Drug discovery
Medicine
DNDi, GHIT
Compound screening
Drug discovery
(lead generation)
Medicine
DNDi, GHIT
NTD Drug Discovery Booster
Drug discovery
(lead generation)
Medicine
Celgene
Daiichi Sankyo
Eisai
GlaxoSmithKline
DNDi, Antwerp Uni, Epichem,
Monash Uni
Novartis
Pfizer
Sanofi
Shionogi
Takeda
Total R&D projects for American trypanosomiasis (Chagas disease): 27
33
ONCHOCERCIASIS
River blindness
DISE A SE IMPAC T 38
Onchocerciasis or “river blindness” is caused by the parasitic worm
Onchocerca volvulus. It is transmitted to humans through exposure to
repeated bites of infected blackflies of the genus Simulium. Symptoms are
caused by the microfilariae, which move around the human body in the
subcutaneous tissue and induce intense inflammatory responses when they
die. Infected people commonly suffer from severe itching, disfiguring skin
conditions, and visual impairment, as well as permanent blindness.
KE Y FAC TS39
• More than 99% of infected people live in 31 African countries.
• In July 2015, Mexico became the third country in the world after Colombia in 2013 and Ecuador in 2014 to be
declared free of onchocerciasis after successfully implementing elimination activities for decades.
38 http://www.who.int/mediacentre/factsheets/fs374/en/
39 http://www.who.int/mediacentre/factsheets/fs374/en/
34
CURRENT R&D PROJEC TS
COMPA N Y
PA RTNER S
PROJEC T
PH A SE
T Y PE
LSTM
Collaborative discovery program
Discovery
Medicine
DNDi
Compound screening
Discovery
Medicine
LSTM, DNDi
Preclinical candidate, scientific engagement
Preclinical
Medicine
Macrofilaricide Drug
Accelerator (MacDA)
Compound screening
Discovery
Medicine
AstraZeneca
A-WOL, LSTM
Focused library screening and DMPK support
Lead identification
Medicine
Bayer
DNDi
Development of emodepside
Preclinical
Medicine
Monclair State Uni, DNDi,
NPIMR, Bonn Uni
Development of treatments
Lead optimization
Medicine
A-WOL (Wolbachia), NEB
Compound screening
Screening
Medicine
LSTM, Liv Uni, GHIT
Novel compounds for anti-Wolbachia
Lead optimization
Medicine
UCSD, UCSF, LSTM, Bonn Uni,
AbbVie, Anacor, Johnson &
Johnson, Eisai, Merck, Calibr,
DNDi, BMGF
Development of treatments with
Macrofilaricide Drug Accelerator (MacDA)
Lead identification
Medicine
DNDi, NPIMR
Concept generation hit finding
Basic research
Medicine
Buea Uni, WIPO Re:Search
Screening for macrofilaricides for
onchocerciasis
Screening
Medicine
Macrofilaricide Drug
Accelerator (MacDA)
Compound
Discovery
Medicine
DNDi
Target compound screening
Lead identification
Medicine
DNDi
Focused compound library screening
Lead generation
Medicine
AbbVie
Celgene
Eisai
Merck
MSD
Sanofi
Total R&D projects for onchocerciasis: 15
35
RABIES
DISE A SE IMPAC T40
The rabies virus is contracted through wounds (e.g. scratches from an
infected animal) or by direct contact with mucosal surfaces (e.g. bite from an
infect animal). Once inside the body, the virus replicates in the bitten muscle
and gains access to motor endplates and motor axons to reach the central
nervous system. The virus then travels to the central nervous system, where
a majority of the clinical symptoms manifest as an acute encephalitis or
meningoencephalitis. Half of the global population lives in canine rabiesendemic areas and is considered at risk for contracting rabies.
KE Y FAC TS 41
•A
n average of 60,000 people die from rabies annually, and more than 15 million people receive post-exposure
prophylaxis every year, averaging to 40% of children in Asia and Africa aged 5-14 years.
• I n more than 99% of all cases of human rabies, the virus is transmitted via dogs.
•T
he incubation period averages 2-3 months and death occurs within 2 weeks after the appearance of clinical
symptoms if intensive care is not sought.
•4
out of every 10 deaths due to rabies occur in children younger than 15 years old.
CURRENT R&D PROJEC TS
COMPA N Y
PA RTNER S
PROJEC T
PH A SE
T Y PE
Sanofi
Company
VRVg: purified serum-free vero cell rabies vaccine
Phase III
Medicine
Total R&D projects for rabies: 1
40 http://www.who.int/rabies/human/en/
41 http://www.who.int/rabies/human/en/
36
CURRENT R&D PROJEC TS
COMPA N Y
PA RTNER S
PROJEC T
PH A SE
T Y PE
AbbVie
DNDi
Compound screening, preclinical support,
technical consulting
Lead identification
Medicine
Astellas
AIST
Discovery of anti-protozoan parasite drugs for
Trypanosoma cruzi
Discovery
Medicine
AstraZeneca
DNDi
Focused compound library screening at Swiss
TPH, Inst. Pasteur K
Lead identification
Medicine
Bayer
company
Lampit, Nifurtimox pediatric (dosing in children)
Phase III
Medicine
Development of treatments
Lead optimization
Medicine
GHIT, DNDi
Screening program
Hit identification
Medicine
DNDi
Screening program (Natural Products Library)
Hit identification
Medicine
DNDi, GHIT
E1224
Phase II
Medicine
DNDi, GHIT, (Shionogi,
Takeda, AstraZeneca, GHIT)
NTD Drug Discovery Booster (DDB-Chagas)
Lead generation
Medicine
Broad Inst., GHIT
Focused compound library screening
Drug discovery
(optimization)
Medicine
SVI, Baylor College, Aeras,
GHIT
Adjuvant to support vaccine development:
Chagas vaccine with SVI
Preclinical
Vaccine
Fiocruz, GHIT
Adjuvant to support vaccine development:
Chagas vaccine with Fiocruz
Preclinical
Vaccine
Dundee Uni
Lead optimization project
Lead optimization
Medicine
Wellcome
Lead optimization III for Chagas disease
Lead optimization
Medicine
Wellcome/Dundee Uni
(Drug Discovery Unit)
Hit to Lead identification Wellcome & Dundee
Uni (Drug Discovery Unit)
Lead identification
Medicine
Wellcome
Hit to Lead identification Wellcome
Lead identification
Medicine
TCOLF, Calibr
Develop novel therapies to prevent and treat
Chagas & leishmaniasis
Discovery
Medicine
Merck
DNDi, Swiss TPH
Concept generation hit finding
Basic research
Medicine
MSD
DNDi
Targeted screening and hit structure–activity
relationship (SAR) development
Lead identification
Medicine
company
Chagas disease project 1: developing safe
treatment for Chagas disease
Drug discovery
Medicine
company
Chagas disease project 2: developing safe
treatment for Chagas disease
Drug discovery
Medicine
CDIPD (UCSD), WIPO
Re:Search
PDE5 inhibitors
Drug discovery
Medicine
CONICET
Compound screening
Lead optimization
Medicine
DNDi
Focused compound library screening
Lead generation
Medicine
GHIT, DNDi
NTD Drug Discovery Booster
Drug discovery
Medicine
DNDi, GHIT
Compound screening
Drug discovery
(lead generation)
Medicine
DNDi, GHIT
NTD Drug Discovery Booster
Drug discovery
(lead generation)
Medicine
Celgene
Daiichi Sankyo
Eisai
GlaxoSmithKline
DNDi, Antwerp Uni, Epichem,
Monash Uni
Novartis
Pfizer
Sanofi
Shionogi
Takeda
Total R&D projects for American trypanosomiasis (Chagas disease): 27
37
SCHISTOSOMIASIS
DISE A SE IMPAC T42
Schistosomiasis is an acute and chronic parasitic disease caused by
blood flukes (trematode worms) of the genus Schistosoma. People are
infected during routine agricultural, domestic, occupational and recreational
activities, which expose them to infested water. There are two major forms
of schistosomiasis – intestinal and urogenital – caused by five main species
of blood fluke. Intestinal schistosomiasis can result in abdominal pain,
diarrhea, blood in the stool, and enlargement of organs. The urogenital
schistosomiasis results in blood in urine, fibrosis of the bladder and
ureter, kidney damage, and even bladder cancer among other symptoms.
The disease may have long-term irreversible consequences, including
infertility. Schistosomiasis control focuses on reducing disease through
periodic, large-scale population pharmaceutical treatments, adequate
sanitation, and snail control.
KE Y FAC TS 43
• The disease causes an estimated 20,000 deaths globally each year.
• In 2014, at least 258 million people required preventive treatment for schistosomiasis with 90% of people
living in Africa.
• More than 61.6 million people were reported to have been treated in 2014.
• In 2014, transmission of the disease has been reported from 78 countries.
42 http://www.who.int/mediacentre/factsheets/fs115/en/
43 http://www.who.int/mediacentre/factsheets/fs115/en/
38
CURRENT R&D PROJEC TS
COMPA N Y
PA RTNER S
PROJEC T
PH A SE
T Y PE
Astellas & Merck
Swiss TPH, Lygature, Fiocruz
(Farmanguinhos), Simcyp, SCI
Pediatric praziquantel formulation
Phase II
Medicine
Swiss TPH, helminGuard,
McGill Uni
Concept generation: target to hit optimization
Basic research
Medicine
BRI
Pharmacology: praziquantel mode of action
Basic research
Medicine
Company
Praziquantel optimization and prequalification
Clinical trials (Phase
I, bioequivalence)
Medicine
UCSD
Targeted screening
Lead identification
Medicine
Merck
MSD
Total R&D projects for schistosomiasis: 5
39
TRACHOMA
DISE A SE IMPAC T44
Trachoma is a disease of the eye caused by infection with the bacterium
Chlamydia trachomatis. The disease causes irreversible blindness or visual
impairment in people. Infection spreads through personal contact (via hands,
clothes, or bedding) and by flies that have been in contact with discharge
from the eyes or nose of an infected person. With repeated episodes of
infection over many years, the eyelashes may be drawn in so that they rub
on the surface of the eye, with pain and discomfort and permanent damage
to the cornea.
KE Y FAC TS 45
•T
he disease is considered a public health problem in 42 countries.
•A
n estimated 1.9 million people are left blind or visually impaired as a result of the disease.
•O
ver 200 million people live in trachoma endemic areas and are at risk of trachoma blindness.
• I n 2015, more than 185,000 people received surgical treatment for the advanced stage of the disease,
and 56 million people were treated with antibiotics.
CURRENT R&D PROJEC TS
COMPA N Y
PA RTNER S
PROJEC T
PH A SE
T Y PE
Pfizer
EMCF, ITI
International Trachoma Initiative: Azithromycin,
Macrolide antibiotic
Development (manufacturing)
Medicine
Total R&D projects for trachoma: 1
44 http://www.who.int/rabies/human/en/
45 http://www.who.int/rabies/human/en/
40
CURRENT R&D PROJEC TS
COMPA N Y
PA RTNER S
PROJEC T
PH A SE
T Y PE
AbbVie
DNDi
Compound screening, preclinical support,
technical consulting
Lead identification
Medicine
Astellas
AIST
Discovery of anti-protozoan parasite drugs for
Trypanosoma cruzi
Discovery
Medicine
AstraZeneca
DNDi
Focused compound library screening at Swiss
TPH, Inst. Pasteur K
Lead identification
Medicine
Bayer
company
Lampit, Nifurtimox pediatric (dosing in children)
Phase III
Medicine
Development of treatments
Lead optimization
Medicine
GHIT, DNDi
Screening program
Hit identification
Medicine
DNDi
Screening program (Natural Products Library)
Hit identification
Medicine
DNDi, GHIT
E1224
Phase II
Medicine
DNDi, GHIT, (Shionogi,
Takeda, AstraZeneca, GHIT)
NTD Drug Discovery Booster (DDB-Chagas)
Lead generation
Medicine
Broad Inst., GHIT
Focused compound library screening
Drug discovery
(optimization)
Medicine
SVI, Baylor College, Aeras,
GHIT
Adjuvant to support vaccine development:
Chagas vaccine with SVI
Preclinical
Vaccine
Fiocruz, GHIT
Adjuvant to support vaccine development:
Chagas vaccine with Fiocruz
Preclinical
Vaccine
Dundee Uni
Lead optimization project
Lead optimization
Medicine
Wellcome
Lead optimization III for Chagas disease
Lead optimization
Medicine
Wellcome/Dundee Uni
(Drug Discovery Unit)
Hit to Lead identification Wellcome & Dundee
Uni (Drug Discovery Unit)
Lead identification
Medicine
Wellcome
Hit to Lead identification Wellcome
Lead identification
Medicine
TCOLF, Calibr
Develop novel therapies to prevent and treat
Chagas & leishmaniasis
Discovery
Medicine
Merck
DNDi, Swiss TPH
Concept generation hit finding
Basic research
Medicine
MSD
DNDi
Targeted screening and hit structure–activity
relationship (SAR) development
Lead identification
Medicine
company
Chagas disease project 1: developing safe
treatment for Chagas disease
Drug discovery
Medicine
company
Chagas disease project 2: developing safe
treatment for Chagas disease
Drug discovery
Medicine
CDIPD (UCSD), WIPO
Re:Search
PDE5 inhibitors
Drug discovery
Medicine
CONICET
Compound screening
Lead optimization
Medicine
DNDi
Focused compound library screening
Lead generation
Medicine
GHIT, DNDi
NTD Drug Discovery Booster
Drug discovery
Medicine
DNDi, GHIT
Compound screening
Drug discovery
(lead generation)
Medicine
DNDi, GHIT
NTD Drug Discovery Booster
Drug discovery
(lead generation)
Medicine
Celgene
Daiichi Sankyo
Eisai
GlaxoSmithKline
DNDi, Antwerp Uni, Epichem,
Monash Uni
Novartis
Pfizer
Sanofi
Shionogi
Takeda
Total R&D projects for American trypanosomiasis (Chagas disease): 27
41
Abbreviations
A BBRE VIATION
PA RTNER’S FULL N A ME
Aeras
Aeras Global TB Vaccine Foundation
AIST
National Institute of Advanced Industrial Science and Technology
Antwerp Uni
University of Antwerp
A-WOL
Anti Wolbachia (A-WOL) Consortium based at the LSTM
Baylor College
Baylor College of Medicine
BMGF
Bill & Melinda Gates Foundation
Bonn Uni
Bonn University
BRI
Biomedical Research Institute
Broad Inst.
Broad Institute
Buea Uni
University of Buea
Calibr
California Institute for Biomedical Research
CBMSO
Centro de Biologia Molecular Severo Ochoa
CSIC
Spanish National Council for Research
DMPK
Drug Metabolism and Pharmacokinetics
DNDi
Drugs for Neglected Diseases initiative
Dundee Uni
Dundee University
EMCF
The Edna McConnell Clark Foundation
Epichem
Epichem Pty Ltd
Farmanguinhos (Fiocruz)
Instituto de Technologia em Fármacos (Fundação Oswaldo Cruz)
Fiocruz
Fundação Oswaldo Cruz
Georgia Uni
University of Georgia
GHIT
Global Health Innovative Technology Fund
ITI
International Trachoma Initiative
Inst. Pasteur K
Institut Pasteur Korea
KU Leuven
Rega Institute KU Leuven
Liv Uni
Liverpool University
LSHTM
London School of Hygiene and Tropical Medicine
A BBRE VIATION
PA RTNER’S FULL N A ME
LSTM
Liverpool School of Tropical Medicine
Lygature
Lygature, Netherlands
MacDA
Macrofilaricide Drug Accelerator
McGill Uni
McGill University
Monash Uni
Monash University
Monclair State Uni
Monclair State University
NEU
Northeastern University
NIAID
National Institute of Allergy and Infectious Diseases
NEB
New England BioLabs
NPIMR
Northwick Park Institute for Medical Research
SCI
Schistosomiasis Control Initiative (Imperial College London)
Simcyp
Simcyp Ltd
SVI
Sabin Vaccine Institute
Swiss TPH
Swiss Tropical and Public Health Institute
TCOLF
Tres Cantos Open Lab Foundation
UCSD
University of California, San Diego
UCSF
University of California, San Francisco
Uni Leon
Universidad de León
USAMRIID
United States Army Medical Research Institute of Infectious Diseases
Vanderbilt Uni
Vanderbilt University
Wellcome
Wellcome Trust
WHO
World Health Organization
WIPO
World Intellectual Property Organization
WRAIR
Walter Reed Army Institute of Research
Photos on cover and on pages 2, 4, 5, 7, 9, 13, 14, 17, 19 are reproduced with the permission of GlaxoSmithKline.
43
ABOUT IFPMA
IFPMA represents the research-based pharmaceutical companies and associations
across the globe. The research-based pharmaceutical industry’s over 2 million employees
research, develop and provide medicines and vaccines that improve the life of patients
worldwide. Based in Geneva, IFPMA has official relations with the United Nations and
contributes industry expertise to help the global health community find solutions that
improve global health.
International Federation
of Pharmaceutical
Manufacturers & Associations
www.ifpma.org
Chemin des Mines 9
P.O. Box 195
1211 Geneva 20
Switzerland