2017 Annual Report

Responding to
Neglected Patients’ Needs
Through Innovation

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LEADERSHIP
MESSAGE
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Message

from the Chair of the Board and the Executive Director
  • Dr Marie-Paule Kieny

    Chair of the Board of Directors

  • Dr Bernard Pécoul

    Executive Director

DNDi will soon be marking the 15 years since its founding, an anniversary which comes at an exciting time in DNDi’s growth since 2003.


Our pipeline is maturing, and significant scientific milestones are on the cusp of being reached.


We have already delivered seven treatments for five diseases. This illustrates the wisdom of having invested early on, not only in long-term projects to discover and develop new chemical entities (NCEs), but also in short-term projects that would have a more immediate impact on improving clinical care for patients.


Our investments in drug discovery are beginning to bear fruit. DNDi’s portfolio currently includes more than 20 new chemical entities (NCEs) that are adapted to the needs of patients. Some will contribute to supporting the control or sustainable elimination of several neglected tropical diseases, including:

  • Sleeping sickness: Fexinidazole, DNDi’s first NCE, which is in the last stages of regulatory review, submitted by our industrial partner Sanofi. If successful, ‘fexi’ would be the first all-oral treatment for sleeping sickness.
  • Leishmaniasis: Novel drugs, based on four NCEs in early discovery research and six in pre-clinical development.

Engaging with partners

  • Contributed to Grupo Insud’s successful registration of the first treatment to be approved in the US for Chagas disease; the sale of Insud’s priority review voucher will go towards increasing access to treatment for neglected patients.
  • Worked with Insud’s non-profit partner Fundación Mundo Sano, to create a new Chagas access framework and put an end to the unacceptable situation where only 1% of people today have access to diagnosis and treatment.
  • Forged alliances with Pharco Pharmaceuticals, the Ministries of Health of Malaysia and Thailand, Doctors Without Borders (MSF), and the Foundation for Innovative New Diagnostics (FIND) to assess new approaches to hepatitis C diagnosis and treatment, including through clinical trials that showed ravidasvir is as safe and effective as the very best of existing treatments.

The need for political leadership

A comprehensive approach to meeting global health needs requires strong political leadership and national commitment. We saw strong examples of this in 2017, including:

  • The landmark decision of the Malaysian government to issue a “government use” licence enabling access to more affordable versions of an expensive and patented medicine to treat the more than 400,000 people living with hepatitis C in Malaysia – and beyond.
  • The ambitious programme on universal health coverage from the WHO Director-General, Dr Tedros, highly relevant for NTDs and antimicrobial resistance.
  • The pledge of the G20 Ministers of Health Summit under Germany’s presidency to invigorate research and development efforts to find new, urgently needed drugs.

Nearly 15 years ago, the founding partners of DNDi refused to accept as a given that neglected populations must continue to be forgotten by medical innovation.


On behalf of DNDi, we express our sincere gratitude to our many partners, our funders and donors, and our staff, who have made our collective successes possible.
Thank you!

2017 in numbers

 

666,917

PEOPLE

screened for sleeping sickness by 10 mobile teams in DR Congo and treated for a variety of diseases

OVER

2,500

PATIENTS

enrolled in the clinical studies completed in 2017

 

685

PEOPLE

trained to support clinical research, from Good Clinical Practice to the use of new diagnostic technologies and processes

44

R&D PROJECTS UNDERWAY

covering every stage of the R&D process

12

 

new R&D partnerships

869

PEOPLE

working on DNDi projects in partner organizations

170,407

COMPOUNDS SCREENED

to look for promising “hits” as potential drug candidates

3+2

 

3 COMPOUNDS advanced to pre- clinical stage, and 2 CANDIDATES advanced to clinical development

21

CLINICAL TRIALS

in progress in 7 disease areas at 52 sites in 15 countries

2

TECHNOLOGY TRANSFERS

in Malaysia and in Latin America – for the manufacture and supply of hepatitis C treatments

6

MILLION

Value of in-kind contributions from partners

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DNDi R&D portfolio

December 2017

Key R&D achievements in 2017

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  • RESEARCH

  • TRANSLATION

  • DEVELOPMENT

  • IMPLEMENTATION

RESEARCH

TRANSLATION

DEVELOPMENT

IMPLEMENTATION

ASAQ ASMQ
New Chemical Entity (NCE)

2017 achievements
from DNDi regional offices

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India DNDI INDIA IN 2017 Japan DNDI JAPAN IN 2017 South-East Asia DNDI SOUTH-EAST ASIA IN 2017 Africa DNDI AFRICA IN 2017 DR Congo DNDI DR CONGO IN 2017 South Africa DNDI SOUTH AFRICA IN 2017 Latin America DNDI LATIN AMERICA IN 2017 North America DNDI NORTH AMERICA IN 2017 Switzerland DNDI HEADQUARTERS SWITZERLAND IN 2017
GARDP Rapid progress in first year

The WHO-DNDi joint initiative GARDP progressed its programmes in 2017, addressing antimicrobial resistance in neonatal sepsis, in sexually-transmitted infections, and through a ‘memory recovery and exploratory’ programme.

The Disease that Strikes Back

Could a form of leishmaniasis challenge elimination efforts in India?

READ THE STORY

7 new treatments delivered, recommended & implemented since 2007

Drug discovery and translational research

Open approaches and increased efficiency for new chemical entities

DNDi works through collaborations in all phases of drug research and development. In the very earliest stages of drug discovery, from molecule screening through to lead selection and optimization, this collaborative work takes place through key partnerships and drug discovery consortia that DNDi has gradually put in place over the last 15 years. Consortia are working on hit-to-lead and lead optimization activities for visceral leishmaniasis and Chagas disease with partners in the US, Canada, Europe, China, India, Japan, Korea, Australia, and finally Brazil where DNDi’s first early-stage research programme, Lead Optimization Latin America (LOLA), was launched in a neglected disease-endemic country.

NTD Drug Discovery Booster: speeding up drug discovery & cutting costs

  • 2 new partners in 2017 - 8 partners total
  • Millions of compounds screened for leishmaniasis and Chagas disease through a simultaneous search process across the pharmaceutical companies
  • 12 hit series identified, 4 of these investigated for proof of principle

Open Synthesis Network: crowdsourcing compound synthesis

  • 6 new partners in 2017 - 12 partner universities total
  • Master's and undergraduate students synthesizing compounds for leishmaniasis and Chagas disease
  • Partners in Brazil, India, Switzerland, UK, and USA

Unprecedented leishmaniasis portfolio: Towards new-generation treatments

  • New chemical classes with different mechanisms of action against Leishmania parasites
  • 6 compounds in pre-clinical development
  • 2 compounds ready to enter clinical development in 2018
  • 1 new combination for cutaneous leishmaniasis in Proof of concept study

Mycetoma Clinical
Trial Begins

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2017 clinical activities:
21 trials, 4,000+ participants

Clinical trial data transparency

DNDi recognizes the scientific importance and the ethical imperative of sharing data collected through its clinical trials for health research, and is a signatory to the WHO Joint Statement on Public Disclosure of Results from Clinical Trials.

Reset Map

DISEASES

  • Human African trypanosomiasis
  • Leishmaniasis
  • Chagas disease
  • Filarial disease
  • Mycetoma
  • Paediatric HIV
  • Hepatitis C

PHASES

  • PHASE I
  • PHASE II a/PoC
  • PHASE IIb/III
  • PHASE IV

STORIES

  • HIV: Introducing oral pellets for children living with HIV
  • Sleeping sickness: A study for the Congolese run by the Congolese
  • Hepatitis C: Enabling public health impact

DISEASES

  • Human African trypanosomiasis
  • Leishmaniasis
  • Chagas disease
  • Filarial disease
  • Mycetoma
  • Paediatric HIV
  • Hepatitis C

Click on the diseases to see related sites

Filarial diseases
  • Emodepside single ascending dose for onchocersiasis (UK)
  • Safety, tolerability, and PK of multiple ascending doses of emodepside (UK)
  • Relative bioavailability study of emodepside immediate release tablets and solution (UK)
HAT
  • Acoziborole pivotal study in adults with stage 1 and stage 2 HAT (DR Congo)
Mycetoma
  • Fosravuconazole proof-of-concept for eumycetoma patients (Sudan)
Cutaneous leishmaniasis
  • Thermotherapy & miltefosine combination proof-of-concept (Colombia, Peru)
PKDL
  • Short-course regimens for treatment of PKDL (Sudan)
  • Short-course regimens for treatment of PKDL (India, Bangladesh)
Chagas disease
  • Benznidazole new doses, improved treatment, and therapeutic associations (Bolivia)
  • Fexinidazole proof-of-concept (Spain)
HAT
  • Study of fexinidazole in special population groups, in in- and out-patients (DR Congo)
  • Fexinidazole pivotal study (DR Congo)
  • Fexinidazole study in adults with early-stage 1 + stage 2 HAT (DR Congo)
  • Fexinidazole study in children with both stage 1 + 2 HAT (DR Congo)
Hepatitis C
  • Ravidasvir/sofosbuvir combination therapy (Malaysia, Thailand)
Paediatric HIV
  • Lopinavir/ritonavir pellets with dual NRTIs implementation study in infants and young children (Kenya, Uganda, Tanzania)
Visceral leishmaniasis
  • Miltefosine/paromomycin Phase III trial for treatment of primary visceral leishmaniasis (VL) patients in Eastern Africa (Ethiopia, Kenya, Sudan, Uganda)
PKDL
  • Infectivity study of PKDL patients (Bangladesh)
HIV-VL
  • New treatments for HIV-VL co-infection (MSF study sponsored by DNDi) (India)
  • New treatments for HIV-VL co-infection (MSF study sponsored by DNDi) (Ethiopia)
PKDL
  • Follow-up study of PKDL patients (India)
Clinical trial data transparency

DNDi recognizes the importance of sharing data collected through its clinical trials for health research, and the ethical imperative to contribute to increasing scientific knowledge. As such, in May 2017, DNDi signed on to the WHO Joint Statement on Public Disclosure of Results from Clinical Trials and committed itself to WHO’s standards on clinical trial transparency, including registering all clinical trials in a publicly available register, promptly reporting trial results 12 months after completion of the trial, and publishing findings in open access journals.

HIV: Introducing oral pellets for children living with HIV

Since 2015, DNDi has been running the “LIVING” study in Kenya, Uganda, and Tanzania for an improved “2-in-1” oral pellet formulation, with 819 children enrolled by the end of 2017. Interim results show the 2-in-1 formulation is effective and well-tolerated by children, and highly acceptable to caregivers. By introducing the 2-in-1 oral pellets, which are an interim solution to replace bitter-tasting syrups, DNDi is paving the way for the more rapid introduction of a “4-in-1” fixed dose combination, on track to be delivered in 2019.

Sleeping sickness: A study for the Congolese run by the Congolese

More than 200 people in the Democratic Republic of the Congo (DR Congo) have been actively engaged in DNDi’s five-year clinical development effort for fexinidazole. This unprecedented effort generated data enabling Sanofi to submit a regulatory dossier to the European Medicines Agency. All data were collected by local health staff at nine clinical sites (DR Congo and Central African Republic), with 10 mobile teams from the Congolese National Sleeping Sickness Control Programme screening over 2 million people in remote villages – and more than 660,000 people in 2017 alone. All data (biological, quality assessment, follow-up) were collected and managed by Congolese partners.

Hepatitis C: Enabling public health impact

DNDi conducted the STORM-C-1 open label trial to assess the efficacy, safety, tolerance, and pharmacokinetics of the drug candidate ravidasvir combined with sofosbuvir for the treatment of hepatitis C virus (HCV) in Malaysia. Interim results showed that 12 weeks after completing treatment, 97% of the 301 patients enrolled were cured.

PHASES

  • PHASE I
  • PHASE II a/PoC
  • PHASE IIb/III
  • PHASE IV

STORIES

  • HIV: Introducing oral pellets for children living with HIV
  • Sleeping sickness: A study for the Congolese run by the Congolese
  • Hepatitis C: Enabling public health impact

Challenges of Conducting Clinical Trials in Remote Areas

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Strengthening capacities

Disease-specific research platforms and networks are central to DNDi’s commitment to strengthen existing research capacity in endemic countries and to support programme sustainability.

As we work with endemic-country partners to conduct clinical trials, central to DNDi’s mission and vision is the idea of strengthening existing clinical research capacities to ensure the sustainability of our work, and increasing endemic countries’ ability to respond to their own research needs.


The disease-specific research platforms and networks created by DNDi and partners form a central part of this objective. To date, platforms or networks have been established for leishmaniasis in both East Africa and Latin America, for Chagas disease, and for human African trypanosomiasis. A filarial clinical research network is also beign set up, and efforts are underway to develop a network of researchers working in paediatric HIV.

685 PEOPLE TRAINED IN 2017

People trained 1,200 1,000 800 600 400 200 0 2010 95 59 2012 85 131 100 2013 132 106 134 2014 226 235 275 2015 482 99 517 2016 262 270 96 2017 31 120 373 161 2011 62 70 108 +9%

Platforms & Networks

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Access - Transitioning from registration to implementation

Ending the Neglect of Chagas in Colombia

SEE STORY

Hepatitis C: A public health approach for access

Despite availability of effective and safe treatment options for hepatitis C virus (HCV), access to treatment is out of reach for millions due to high prices. DNDi has partnered with a range of stakeholders who share the common goal of increasing access to affordable HCV treatment, including: Pharco (Egypt) in the development of ravidasvir; the Malaysian Ministry of Health, which is taking a public health approach to treating HCV; and industrial partners Pharmaniaga (Malaysia) and Insud/Elea (Argentina), to secure a much more affordable price of USD 300-500 per treatment course.
Results suggest that the ravidasvir/sofosbuvir combination is comparable to the best treatments available today, with a radical difference: price. But to use this drug combination, countries would also need access to affordable sofosbuvir. In September 2017, Malaysia issued a “government use” licence to source generic sofosbuvir, a move which has allowed it to accelerate access to affordable HCV treatment in its public hospitals.


Fexinidazole: Using the regulatory process to pave the way for access

In December 2017, a few months after the conclusion of large-scale DNDi clinical trials, our industrial partner Sanofi submitted the registration dossier for fexinidazole as the first all-oral treatment for sleeping sickness. In a promising move for access, a submission was made to the European Medicines Agency under what is known as Article 58.

If fexinidazole is registered in late 2018, regulatory approval will only be the first step. Translation into national policy and treatment guidelines will be needed as well as community awareness efforts and treatment knowledge for health providers.

HIV: Preparing for the 4-in-1 by encouraging uptake of the 2-in-1

DNDi is working with Indian manufacturer Cipla to develop a “4-in-1”, a solid first-line fixed-dose combination combining four antiretrovirals (abacavir, lamivudine, lopinavir, and ritonavir), as granules contained in a capsule. Caregivers will be able to open the capsules and give the granules to children with soft food, breast milk, or milk and water. These granules will not require refrigeration, and they will be taste-masked and easy to dose across various weight bands. Submission for US FDA registration will be pursued in late 2018.

Policy, fundraising & communications

 

6

NATIONAL TREATMENT POLICIES

or guidelines revised to reflect the use of DNDi-delivered treatment, and 4 policy changes in progress

EUR

83.4

MILLION

Multi-year funds secured for DNDi and GARDP in 2017

22

PEER-REVIEWED SCIENTIFIC PUBLICATIONS

on DNDi’s research, of which 87% were open access publications

Policy

WHO Director General Dr Tedros has declared universal health coverage (UHC) to be the priority for his mandate. This has important implications for NTDs, which are diseases of poverty affecting most countries. Delivering quality UHC depends on the availability of and access to safe, effective, and affordable medicines, underscoring the continued need for R&D for NTDs as a key component of the UHC agenda. NTD programmes provide entry points to some of the world’s hardest-to-reach communities, and many policymakers have identified the ability to address NTDs as a “litmus test” for UHC.

The Berlin Declaration that followed the first-ever G20 Health Ministers meeting in May 2017 cautioned that success in the fight against AMR cannot be achieved with current treatments. It recognized the importance of ‘reactivating the R&D pipeline through incentive mechanisms that avoid the reliance on high price/volume combinations’ to ensure sustainable access. It called for ‘broadening the voluntary financial support’ for initiatives, including GARDP, which ‘reinvigorate R&D in science and industry for antimicrobials’.

2017 was marked by the Malaysian government’s decision to reaffirm its strong commitment to providing access to treatments for hepatitis C by issuing a “government use” licence enabling access to more affordable versions of sofosbuvir, an expensive and patented treatment for hepatitis C. This is a landmark decision for the more than 400,000 people living with hepatitis C in Malaysia.

Fundraising

Funds raised in 2017 for the neglected disease portfolio include increased support for the sleeping sickness programme from the Bill & Melinda Gates Foundation, as well as from private donors primarily from the US, through the HAT campaign launched last year, but also in Europe. Other noteworthy contributions include the first grant from the GHIT Fund secured for mycetoma, and from the European and Developing Countries Clinical Trials Partnership for a visceral leishmaniasis clinical trial in four African countries. In addition, the first grant entirely dedicated to DNDi’s HCV programme was received from the Médecins Sans Frontières/Doctors Without Borders (MSF) Transformational Investment Capacity.

In May, the G20 Health Ministers cautioned that success in the fight against antimicrobial resistance (AMR) cannot be achieved with the current treatments. The Declaration welcomed and sought to build on initiatives such as GARDP, to ‘reinvigorate research and development in science and industry for antimicrobials.’ The German Federal Ministry of Health and Ministry of Education and Research then hosted a pledging conference for GARDP in September 2017. A total of EUR 56 M was pledged by Germany, Luxembourg, Monaco, the Netherlands, South Africa, Switzerland, the United Kingdom, and the Wellcome Trust for the development of new and improved treatments to fight antibiotic resistance and contribute to ensuring healthy lives and well-being for all. Of this total, EUR 5.5 M was received in 2017.

Since 2003, DNDi’s cumulative income reached EUR 491 M, against a target of EUR 650 M by 2023. By the end of 2017, GARDP had EUR 64 M in commitments and pledges, almost 25% of the EUR 270 M total funding required to deliver GARDP’s objectives by 2023.

More on DNDi donors

Communications

In 2017, DNDi joined forces with the WHO Collaborating Centre for Leishmaniasis at the Instituto de Salud Carlos III, Madrid, to co-organize the 6th World Congress on leishmaniasis. The event attracted 1,500 scientific experts, health decision-makers, WHO, government representatives of endemic countries, and all major organizations involved in the fight against leishmaniasis. Among the outputs from the conference were a PLOS special collection dedicated to leishmaniasis; the ‘100 students’ initiative programme allowing students and young scientists from esourcelimited settings to attend, and supporting professional development of a new generation of scientists; and the launch of a manifesto to raise awareness of the urgent need for more research on mucocutaneous leishmaniasis, a very neglected form of leishmaniasis.

In 2017, DNDi staff members authored or co-authored 22 peer-reviewed publications. Of these, nine had a lead author or co-author from an endemic country, and 18 were published in an open access journal, in keeping with DNDi's commitment to open access.

Here is the full list of DNDi 2017 scientific publications Here is the list of GARDP 2017 publications

In recognition of its innovative and collaborative model, DNDi was awarded the Innovation Prize 2017 by the Geneva Chamber of Commerce, Industry and Services, the Department of Security and Economy, and the Office for the Promotion of Industries and Technologies.

A word of thanks

DNDi would like to thank all its donors worldwide for their loyal commitment and collaboration since 2003. To date, DNDi has delivered seven new treatments and aims to bring 16-18 treatments to patients living with neglected diseases by 2023. DNDi is grateful for the support received from all donors who contributed toward the advancement of its mission and goals. Listed are supporters who have given a cumulative contribution of more than USD or EUR 10,000, as well as collaborative funding partners.

Photo credits: Benoit Marquet-DNDi; Kishore Pandit-DNDi; Felipe Abondano-DNDi; Neil Brandvold-DNDi; Bobby Tan-DNDi; Scholars and Gentlemen/DNDi.

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