mmv · prof. michael ferguson, dr winston gutteridge, dr fatoumata nafo-traoré, dr robert newman,...
TRANSCRIPT
In 1999, MMV was a pioneering newcomer to the
world of antimalarial drug research. The World
Health Organization (WHO) was reporting more
than 300 million cases of malaria each year. The
malaria parasite had become resistant to widely-
used drugs. New medicines were desperately
needed as malaria continued to afflict and take
the lives of countless people across the world.
While the antimalarial market is huge in terms
of those in need, it is small in terms of profit.
By 1999, this imbalance had led to a virtually
empty pipeline of new antimalarial drugs.
Motivated by this glaring inequity and the need
to act in the face of a projected public health
disaster fuelled by escalating drug resistance, a
group of dedicated individuals and organizations
launched MMV. It started out modestly with only
USD 4 million in seed finance and three early-
stage projects in its portfolio, but was brimming
with ambition.
Today, 15 years on, this ambition has borne fruit.
By working in partnership with public and private
players, MMV has surpassed its original objective
to develop one new effective antimalarial before
the end of 2010. Since its foundation in 1999,
MMV and partners have developed and/
or registered four new medicines: Pyramax®,
(pyronaridine-artesunate) with Shin Poong;
Eurartesim® (dihydroartemisinin-piperaquine) with
Sigma-Tau; Artesun® (artesunate injection) with
Guilin; and Coartem® Dispersible (artemether-
lumefantrine) with Novartis.
Medicines for Malaria Venture and partners are working together to discover, develop and deliver new
effective and affordable antimalarials to give vulnerable populations a better chance of a healthy future,
and help ultimately eradicate this terrible disease.
Developing medicines, defeating malaria
Defeating Malaria Together
But we cannot stop there. To meet the ambitious
goal of malaria eradication, new medicines
with novel mechanisms of action are needed,
especially to fill the gap that might be left by
threats, such as resistance to artemisinin. We
also need new medicines to treat the most
vulnerable populations (children and pregnant
women), to cure relapsing malaria and to prevent
transmission.
As new medicines emerge from the pipeline,
MMV and partners collaborate to ensure that
they reach patients quickly and save lives.
With the largest-ever pipeline of antimalarial
molecules, MMV is set to develop and deliver
critical medicines for the ultimate eradication of
malaria.
AT AGLANCE
MMV
MMV shows how the market can work for the world’s poorest people.”Melinda Gates
Key achievements
Malaria Boxes 200dispatched to more than 30 countries since launch in December 2011, to catalyse malaria and neglected disease drug research.
Direct and in-kind support from our pharma partners more than trebling the value of each donor dollar for R&D.
=1 3.50USD USD
million treatments ofchild-friendlyCoartem®
Dispersible
250
( a r t e m e t h e r - l u m e f a n t r i n e ,
co-developed with Novart is) distributed to 50 countries since launch in 2009.
million
vials ofArtesun®
25(Guilin Pharmaceutical’s MMV-
supported injectable artesunate)
for severe malaria deliver-ed since 2010 – saving an est imated 165,000 additional lives compared to treatment with quinine.
enteredPhase IIItrialsin April 2014 – taking it closer to becoming the only new drug for relapsing malaria in over 60 years.
compounds 9targeting malaria eradi-cation, progressed from discovery to preclinical or clinical development.
new
MMV at a Glance 2014 I www.mmv.org
OZ439 together with piperaquine entered Phase IIB single-dose, combination studies in July 2014.
Tafe
noquin
e
MMV’s Product Development Partnership
(PDP) model has blossomed and born
fruit. By nurturing and strengthening
partnerships with clinicians and scientists
across academia and the pharmaceutical
industry, and combining this with rigorous
portfolio management, MMV has become
a highly cost-effective and productive
R&D organization. The model’s success
has created a virtuous circle that brings in
new donors and stakeholders.
MMV at a Glance 2014 I www.mmv.org
The PDP model
Malaria...takes a child’s life every minute
kills ~627,000 people each year
– the vast majority are children
can kill within 24 hrs of symptom onset
can infect an African child up to
6 times a year
costs Africa USD 12-30 billion in lost
GDP every year
accounts for 40% of all public health
spending in Africa
is both a cause and consequence
of poverty
1. International Conference on Harmonisation of Technical
Requirements for Registration of Pharmaceuticals
for Human Use (ICH)
2. Good Laboratory Practice (GLP)
3. Good Clinical Practice (GCP)
4. Good Manufacturing Practice (GMP)
Over the years, MMV has worked in
partnership with more than 300 research
institutions and companies across the world.
Each partner brings expertise, enabling
technologies and research facilities, the value
of which often exceeds the funding received
from our donors.
In addition to innovative individual projects,
miniportfolios enable the efficient distribution of
resources that help to accelerate the discovery
of promising antimalarial compounds.
Meticulous selection processes are coupled
with support for the most promising candidate
drugs and quick termination of those that miss
milestones or do not meet MMV’s demanding
product profiles. This industry-style portfolio
management is not easy to execute but is
essential to maximise the use of every donor
dollar towards our highly-focused mission.
The innovative route to neglected disease drug development
Quality for allMMV strongly bel ieves that al l malaria
patients, rich or poor, deserve the best and
safest treatment possible. All new antimalarial
medicines co-developed by MMV must meet
high international standards, for example
those required by the European Medicines
Agency, the US FDA or WHO’s Prequalification
Programme. MMV and partners conduct
all clinical development projects to ICH1
guidelines every step of the way – from GLP2
standards for preclinical work, GCP3 standards
for clinical trials in malaria-endemic countries
that also adhere to national regulations, and
finally GMP4 standards during the manufacture
of the medicines.
Academic research and clinical trial sites
New medicines for malaria
Pharmaceutical research
Donors and in-kind contributions
MMV supported projects 4th quarter 2014Antimalarial drug discovery and development projects scientifically and/or financially supported by MMV.
MMV at a Glance 2014 I www.mmv.org
ESAC Expert Scientific Advisory Committee
GSB Global Safety Board
MMV Board of Directors/Executive Committee/Financial Audit Committee
APMACAccess and Product ManagementAdvisory Committee
APAC Authorization for Phase III/Advancement Committee
Access andProduct
Management
GO
VE
RN
AN
CE
Research Translational Development APMLead
optimizationPreclinical Human
volunteersPatient
exploratoryPatient
confirmatoryUnder review
Novartis Miniportfolio
Novartis 1 project
GSK Miniportfolio
AstraZeneca Miniportfolio
GSK 3 projects
Sanofi Orthologue Leads
AnacorOxaboroles
Liverpool School of Trop Med/Univ. Liverpool Tetraoxanes
Univ. Texas Southwestern/ Monash Univ./ Univ. WashingtonDHODH
CelgeneHeterocycles
Univ. CampinasHeterocycles
Daiichi-Sankyo Screening
TakedaScreening
Univ. SydneyOpen Source Drug Discovery
EisaiScreening
Merck SeronoAmino-alcohols
Other projects 15 projects
P218 DHFRBiotec/ (Monash Univ./ LondonSchool of Hygiene & Trop Med)
DSM265Takeda/ NIH
Artemether- lumefantrinedispersible Novartis
Artesunate for injection Guilin
Dihydro-artemisinin- piperaquine Sigma-Tau
Pyronaridine-artesunateShin Poong
Artesunate- amodiaquine
Sanofi/ DNDi
Univ. Cape TownAminopyridines
OZ439/PQPSanofi
Research Translational Development APMLead
optimizationPreclinical Human
volunteersPatient
exploratoryPatient
confirmatoryUnder review Post
approval
Rectal artesunate Cipla/ Strides/WHO-TDR
*
MMV048Univ. Cape Town/ Technology Innovation Agency
OZ439/FQSanofi
MMV Pathogen Box
Artesunate- mefloquine
Cipla/ DNDi *
Tafenoquine GSK
Dihydro-artemisinin-piperaquinepaediatric Sigma-Tau
Pyronaridine-artesunatepaediatricShin Poong/Univ. Iowa
KAE609 Novartis
KAF156Novartis
SP+AQ(sulfadoxine-pyrimethamine + amodiaquine)
Guilin
MMV121(Univ. Dundee)
PA92(Drexel Univ./ Univ. Washington/ Genomics Institute of the Novartis Research Foundation)
MMV253(AstraZeneca )
GSK030GSK
SJ733St Jude/ (National Institutes of Health [NIH]/Rutgers Univ.)
TPP1 minimal essential: 3-day cure/artemisinin-based combination therapies
› Artemether-lumefantrine dispersible (Coartem® Dispersible), generic by Ajanta
› Dihydroartemisinin-piperaquine (Eurartesim®)
› Dihydroartemisinin-piperaquine paediatric (Eurartesim®)
› Pyronaridine-artesunate (Pyramax®)
› Pyronaridine-artesunate paediatric (Pyramax®)
› Artesunate-amodiaquine (CoarsucamTM, ASAQ/Winthrop®) FDC generics by Ajanta,
Ipca, Guilin and co-blistered generics by Strides & Cipla
› Artesunate-mefloquine, co-blistered generic by Acino/Mepha
Potential single-dose agents that could be combined into SERCaP
› OZ439/FQ
› OZ439/PQP
› KAE609
› KAF156
› Tafenoquine
Intermittent chemoprevention
› Sulfadoxine-pyrimethamine + amodiaquine (SP+AQ)
Severe malaria
› Rectal artesunate
› Artesunate for injection (Artesun®)
Fast clearance (TCP1)
Long duration (TCP2)
Relapse prevention (TCP3a)
Transmission blocking (TCP3b)
Chemoprevention (TCP4)
To develop the individual compounds for combination into the TPPs,
MMV has defined five Target Candidate Profiles (TCPs):
Non-artemisinin-based therapy
Artemisinin-based therapy
Bioequivalence studies planned in preparation for WHO prequalification
Included in MMV portfolio post registration
First review or approval by WHO Prequalification, or by regulatory bodies who are
ICH members or observers
The malaria community has defined two Target Product Profiles (TPPs) for medicines to make
eradication achievable: TPP1: a treatment combination that is ideally a Single Exposure Radical
cure and Prophylaxis (SERCaP) and TPP2: Single Exposure Chemoprotection (SEC).
Target Product Profiles and Target Candidate Profiles
Postapproval
1. To support new drug introduction, MMV
provides product briefings to policy decision
makers, and creates simple, understandable
packaging and training materials. For exam-
ple, in 2013 and 2014, MMV and partners,
Clinton Health Access Initiative and Malaria
Consortium, signed agreements with five of
six malaria-endemic countries, in which we
will scale-up the use of injectable artesunate
to treat severe malaria, with funding from UNI-
TAID. This programme also allows MMV to
support pharmaceutical partners to develop a
WHO-prequalified artesunate suppository for
pre-referral treatment of severe malaria.
2. MMV has pioneered new initiatives to
enhance the reach of new medicines via
the public and private sector. Our Consortium
for ACT private sector subsidy (CAPSS) ini-
tiative in Uganda first established key private-
sector subsidy principles, including that a
subsidy leads to a significant increase in the
use of ACTs. The principles were later em-
ulated in the Affordable Medicines Facility
for Malaria (AMFm) and are now a standard
option provided by the Global Fund via the Pri-
vate Sector Co-payment Mechanism.
3. Monitoring the uptake of quality medi-
cines is critical for MMV. We gather market
MMV at a Glance 2014 I www.mmv.org
16 countries85 partnerships
8 countries94 partnerships
11 countries86 partnerships
21 countries98 partnerships
Working in partnershipSince its inception, MMV has successfully worked in over 50 countries with more than 300 partners from the public and private sectors,
from NGOs and non-profit organizations, as well as from clinical trial sites.
Access for health impactMMV’s Access and Product Management (APM) team focuses on four key areas to ensure new antimalarials reach people in need:
intelligence on how new medicines are pro-
cured and ultimately reach patients. Key work
includes SMS for Life in Tanzania, where mobile
technology was used to track drug availabili-
ty, and our collaboration with IMS to routinely
gather data on antimalarial flow into Zambia.
4. The APM team’s engagement with the
“voice of countries” also helps us shape
our R&D work. For example, market research
has evaluated the acceptability of different
product presentations and is guiding critical
formulation decisions for single-dose cures
currently in development. Separately, APM is
supporting the development of a new complete
cure for relapsing malaria by documenting
current practices and attitudes regarding such
treatment.
By working hand-in-hand with MMV’s science
team and partners, APM ensures that MMV
will develop highly effective medicines that
respond to the evolving needs of malaria
patients and the marketplace.
GATHERING MARKETINTELLIGENCE
SUPPORTING INTRODUCTION
ENHANCINGREACH
INFORMINGR&D
GOAL:
Minimise time-to-market
for newly registered MMV
medicines in priority
countries.
GOAL:
Ensure timely availability
of key market data to inform
product development and
launch, and measure impact.
GOAL:
Expand access to
medicines, through existing
and innovative channels to
improve availability.
GOAL:
Help align product
development criteria with
unmet medical needs.
Board of DirectorsMr Ray Chambers (Chairman), Dr Pedro Alonso,
Dr David Brandling-Bennett, Dr Ernest Darkoh,
Prof. Michael Ferguson, Dr Winston Gutteridge,
Dr Fatoumata Nafo-Traoré, Dr Robert Newman,
Dr David Reddy, Dr Dennis Schmatz and Mr Per
Wold-Olsen.
MMV North America Inc. Board MembersDr Dennis Schmatz (President of the Board of
North America Inc.), Dr David Bowen, Mr Ray
Chambers, Dr David Reddy and Ms Wendy Taylor.
Expert Scientific Advisory Committee (ESAC)Dr David McGibney (Co-chairman, Development),
Dr Dennis Schmatz (Co-chairman, Discovery), Dr
Aileen Allsop, Dr Salim Abdullah, Prof. Kelly Chibale,
Dr Robert Clay, Prof. Simon Croft, Prof. Umberto
D’Alessandro, Prof. Ogobara Doumbo, Dr Michael
Dunne, Prof Paul Fish, Dr Kasturi Haldar, Prof Dennis
Kyle, Dr Trevor Laird, Prof John Lambert, Dr Mary
Mader, Dr Michael Makanga, Dr Christine Manyando,
Dr George K Mooney, Prof. François Nosten, Dr
Bernhards Ogutu, Dr Paul Reider, Dr David Roberts,
Dr Peter Siegl, Dr Per Sjoberg, Prof. Dennis Smith,
Prof. Terrie Taylor, Dr Neena Valecha, Dr Thomas
Wellems and Dr Matthew Wyvratt.
Access & Product Management Advisory Committee (APMAC)Prof. Christian Lengeler (Chairman), Dr Neena
Valecha (Vice Chairman), Ms Valentina Buj,
Dr Elizabeth Chizema, Dr Graciela Diap,
Dr Alexander Dodoo, Dr Gunther Faber,
Dr Douglas Lungu, Dr David McGibney,
Prof. Ric Price, Ms Melanie Renshaw,
Dr Claude Emile Rwagacondo, Dr Richard
Steketee, Prof. Andy Stergachis and
Dr Brenda Waning.
MMV TeamNada Abla Geiser, Marc Adamy, Nada Araeipour,
Adam Aspinall, Jaya Banerji, Chris Barker, Lidiya
Bebrevska, Soazig Bertrand, Benjamin Blasco,
Grégory Bonnaud, Isabelle Borghini-Fuhrer, Emilie
Burlot, Jeremy Burrows, Andrea Buscaglia, Brice
Campo, Stephan Chalon, Stéphanie Cherbuin, Marion
Colombani, Maud Couturier, Gelavizh Daghigh, Helen
Demarest, Heidi Divecha, Christina do Paço, Matthew
Doherty, Cristina Donini, Celia Doudou, Stephan
Duparc, Mélanie Dupuy, Myriam El Gaaloul, Isabelle
Fontaine, Sylvie Fonteilles-Drabek, Penny Grewal
Daumerie, Joan Herbert, Pierre Hugo, George Jagoe,
Alexis Kamdjou, Sonia Kanobana, Franziska
Karyabwite, Wiweka Kaszubska, Coline Legrand,
Didier Leroy, Andrea Lucard, Antonia Lundquist,
Adrienne MacDonald, Fiona Macintyre, Maud Majeres
Lugand, Neil McCarthy, Aleksandra Misiorowska,
Jörg Möhrle, Alicja Poczatenko, Elizabeth Poll, Anya
Ramalho, Elena Ramos, David Reddy (CEO), Emilie
Rossignol, Mélanie Rouillier, Marie-Ange Roustan,
Thomas Rückle, Binetou Sané, Sonia Schnieper
Samec, Thomas Spangenberg, Tareq Sunderji,
Yuko Takase, Simona Valigova, Wesley van Voorhis,
Helen Weir, Tim Wells and Paul Willis.
Editors: Elizabeth Poll and
Jaya Banerji
Photos: MMV (cover a & p 4c),
Merck Serono (cover b),
BMC St Jude (p 1) and Séverine Pillet (p 4a)
Jennifer Jackson (p 4b)
Designer: ComStone-Pierre Chassany
Medicines for Malaria Venture (MMV) International Centre Cointrin
Route de Pré-Bois 20 - PO Box 1826
1215 Geneva 15 - Switzerland
T + 41 22 555 03 00 - F + 41 22 555 03 69
www.mmv.org | [email protected]
Focus on finances Medicines for Malaria Venture receives
s u s t a i n e d f u n d i n g a n d s u p p o r t
f rom government agencies, pr ivate
foundations, international organizations,
corporations, corporate foundations
and private individuals. These funds are
used to finance the MMV portfolio of R&D
projects as well as specific, targeted
access and delivery interventions that
aim to make it easier for vulnerable
populations to access MMV products.
In 2013, MMV secured significant funding
commitments of USD 238 from four major
donors. This success, founded on our
donors’ firm belief in MMV’s vision and ability
to realise it, continued into 2014. In the
first 10 months of 2014, the Bill & Melinda
Gates Foundation and the United Kingdom’s
Department for International Development
(DFID) pledged an additional USD 40 million
to support MMV’s work.
Nevertheless, this still leaves a shortfall over
the next 5 years to sustain the portfolio and
advance towards the goal of elimination and
eradication. Progress is dependent on the
availability of long-term funding.
Figure 1 Sources of funding from 1999 to 2018
Project-Related R&D 77.0%
Access & Product Management 9.4%
External Relations & Advocacy 4.1%
Governance & Stakeholders 0.2%
Management & Administration 9.3%
Figure 2 MMV expenditure 2013: USD 65.2 million
Public and private investments need to
increase to support MMV’s discovery,
development and delivery of next-generation
antimalarials to help eradicate malaria.
As a result, MMV is striving to expand
and develop current and new donors and
negotiate the best terms with our partners.
Bill & Melinda Gates Foundation 62.87%
United Kingdom’s Department for International Development (DFID) 15.99%
Wellcome Trust 3.66%
United States Agency for International Development (USAID) 2.93%
Netherlands Minister for Development Cooperation 2.21%
Irish Aid 2.14%
Swiss Government SDC 2.13%
National Institutes of Health (NIH) 1.48%
Spanish Agency for International Development 1.36%
World Bank 0.96%
Rockefeller Foundation 0.72%
ExxonMobil Foundation 0.72%
World Health Organization/Roll Back Malaria (WHO/RBM) 0.67%
Global Health Innovative Technology Fund (GHIT) 0.46%
UNITAID 0.43%
Newcrest Mining Limited 0.41%
Norwegian Agency for Development Cooperation (Norad) 0.35%
Australian Government Department of Foreign Affairs and Trade (DFAT) 0.30%
BHP Billiton 0.09%
MerckSerono 0.06%
Individual donors 0.05%
EU CRIMALDDI 0.01%
© November 2014 Medicines for Malaria Venture - All rights reserved