the wayforward needs-drivenpublic healthresearch · 2018. 9. 13. · cpg-d35 (cl) amino pyrazoles...
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The way forward
needs-driven public
health research
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Hepatiti
s C
DNDi’s PRIORITY:
Neglected
Patients
DNDi’s PRIORITY:
Neglected
Patients
Paediatri
c HIV
Filarial
disease
s
Sleepin
g
sicknes
s
Chagas
disease
Leishmaniasis
MalariaMycetoma
… from Bench to Bedside
Not for Profit R&D
Responding to the Needs of Patients Suffering from Neglected
Diseases…
© S
co
tt N
els
on
fo
r T
he
Ne
w Y
ork
Tim
es
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7 new treatments available and 15 new chemical entities in the pipeline
Screen Hit to Lead Pre-clinical Phase I Phase IIa/PoCLead Opt. RegistrationPhase IIb/III Access
Fosravuconazole
DNDi R&D Portfolio
Research DevelopmentTranslation Implementation
MycetomaMycetoma
Ravidasvir/
SofosbuvirHepatitis CHepatitis C
Two ‘4-in-1’
LPV/r FDC
granules
LPV/r pellets
with dual NRTI
Superbooster
Therapy
Paediatric HIV/TB
Superbooster
Therapy
Paediatric HIV/TB
PaediatricHIVPaediatricHIV
Screening EmodepsideAbbV4083
TylaMacMacro
Filaricide 3Filaria
New Benz
Regimens +/-
fosravuconazole
Screening
Biomarkers
Chagas
H2L
Fexinidazole
Benznidazole
Paediatric Dosage
Form
Benznidazole
Paediatric Dosage
Form
Chagas
Lead OptChagasChagas
SSG&PM
Africa
SSG&PM
Africa
New Treatments
for HIV/VL
Anfoleish
(CL)
ScreeningDNDI-5421
DNDI-5610
oxaboroles
Leish
H2L
DNDI-0690
nitroimidazole
Fexi/MF
Combination
New VL Treatments
Asia
New VL Treatments
Asia
New Treatments
for PKDL
New VL
Treatments
Latin America
New CL
Combination
CpG-D35
(CL)
Amino
pyrazoles
DNDI-6148
oxaborole
CGH VL
Series 1
MF/Paromomycin
Combo for Africa
Leishmaniasis
FexinidazoleSCYX-7158
oxaboroleSCYX-1330682
SCYX-1608210
oxaboroles
NECT
Nifurtimox-Eflornithine
Combination Therapy
NECT
Nifurtimox-Eflornithine
Combination Therapy
Human African Trypano-somiasis
Human African Trypano-somiasis
Malaria
FDC ASAQ
Malaria
FDC ASAQ
Malaria
FDC ASMQ
Malaria
FDC ASMQ June 2016
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nathalie strub wourgaft – CT conference Genevai September 2016 slide 4
Characteristics of our model
• Not for Profit
• Patients-need driven
• Independence – public/private funding
• Governance (Public Research Founding Institutions)
• Policies
– IP / public good
– Delinkage / affordable
– Sharing data / open source
– Transparency
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nathalie strub wourgaft – CT conference Genevai September 2016 slide 5
Characteristics of our CTs
• Pipeline focused on unmet medical needs
• Product development has to meet a disease-specific «Target
Product Profile» developped with public health partners
• Our CTs are conducted in endemic countries with often:
– Poor, voiceless, non literate, i.e vulnerable patients
– Investigators with little if no experience in CTs
– Poorly resourced Ethics Committees and / or National Regulatory
Authorities
5
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A Key Role for Regional A Key Role for Regional A Key Role for Regional A Key Role for Regional Disease Platforms Disease Platforms Disease Platforms Disease Platforms
Defining patient needs and Target Product Profile (TPP)
Strengthening local capacities
Conducting clinical trials (Phase II/III studies)
Facilitating Registration of new therapies
Accelerating implementation of new therapies, ensure therapies
reach patients
LEISHMANIASIS
HAT
CHAGAS
LEISHMANIASIS
How do we work:
Using & strenghtening
research capacities in
endemic regions
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nathalie strub wourgaft – CT conference Genevai September 2016 slide 7
Sleeping sickness• Lethal disease
• Major endemicity in Democratic Republic of Congo
• Need for better treatments (oral, no lumbar puncture)
• No treatment recently developped for that indication
� Fexinidazole identified through compound mining
� Contract with industrial partner Sanofi
� Tested in Phase I subjects of sub-saharan origin in France
� Art58 EMA scientific advice to share & obtain advice on clinical devpt
strategy
� Infrastructure and HR (GCP/clinical) capacity strenghthening +++
� Joint «North/South/South» ethics review
� Phase 1 results published
� Working according international standards
> 500 patients recruited in 10 clinical sites
� Regular updates made to the WHO-NTD working group on HAT elimination
� Aiming at Regulatory collaborative review for regulatory approvals
� Dossier includes Risk management Plan that can address needs for future
follow-up
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nathalie strub wourgaft – CT conference Genevai September 2016 slide 8
Challenges
– Ethical concerns around vulnerability
– EC/NRAs capacity to reveiw CTs
– Transparency: technical, cost, timing of access to data
– Regulatory marketing autorisation: do these disease deserve a «priority
process?»
– Post approval safety detection / Pharmacovigilance
• Future
– Defining public health priorities and expected value of new drugs
– Promote inclusive collaborative procedures with public health actors
– R&D Observatory and Fund …
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Thank you