JP Paccaud

7/31/2019 JP Paccaud

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Jean-Pierre Paccaud, PhD

European Parliament, May 30th 2012

New Drugs for

Neglected Diseases

New Hope forForgotten Patients

Breaking the disease-poverty cycle: Dopharmaceutical companies deliver on R&D for

Neglected Diseases in developing countries?


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NTDs, the PDP Model and DNDi


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Burden of Neglected Tropical Diseases

This map displays countries endemic for each of these diseasesbased on 2009-2010 data and international borders.(from: www.unitingtocombatntds.org)

Buruli UlcerChagas disease (Americantrypanosomiasis)CysticercosisDengue/Severe dengueDracunculiasis (guinea-worm disease)

EchinococcosisFascioliasisHuman African trypanosomiasisLeishmaniasisLeprosyLymphatic filariasisOnchocerciasisRabiesSchistosomiasis

Soil transmitted helminthiasisTrachomaYaws


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HIV/AIDS TB Malaria NTD Diarrhea Respiratory

Diagnostics

Microbicides &preventatives

Therapeuticproduct

Vaccine Human hookwormvaccine initiative

VaccineDevelopment

Program

PDPs work across different diseases and modalities

&Source:

Product Development Partnerships (PDPs)
http://www.sabin.org/http://www.dndi.org/http://www.sabin.org/http://www.dndi.org/http://www.oneworldhealth.org/http://www.iavi.org/http://www.meningvax.org/index.htmhttp://www.finddiagnostics.org/


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Malaria Leishmaniasis

Sleeping Sickness (HAT) Chagas Disease

Paediatric HIV

Helminth infections

Responding to the Needs of PatientsSuffering from Neglected Diseases


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Vision:

A collaborative, patients needs-driven, virtual,non-profit drug R&D organisation to developnew treatments against the most neglected

communicable diseases

DNDisVision & Objectives

Objectives:

Deliver 11 to 13 new treatments by 2018 for sleepingsickness, Chagas disease, leishmaniasis, malaria,

paediatric HIV and specific helminth infections Establish a robust pipeline for future needs

Use and strengthen existing capacity in disease-endemiccountries


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Easy to Use Affordable Field-Adapted Non-Patented

6 New Treatments Developed Since 2007


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DNDiPortfolio: A Mix of Existing Drugs & NCEs

December 2011

Discovery Pre-clinical Clinical Implementation

Nitroimidazole backup(HAT)Screening

ConsolidatedLead

Optimization

VL-2098 (VL)

Alternative formulationsof Amphotericin B (VL)

Nitroimidazole backup(VL)

Fenarimol series(Chagas)

K777 (Chagas)

Flubendazole -Macrofilaricide

(Helminth)

Improved PI for 1st-line Prodrugs of LPV/RTV New formulations of

LPV/r(Paed. HIV)

Fexinidazole (HAT)

Oxaborole SCYX-7158(HAT)

New VL treatmentsBangladesh

New VL treatments

Africa AmBisome Miltefosine

New VL treatments(Latin America)

HIV / VL

Azoles E1224& Biomarker (Chagas)

Benznidazole Paed.dosage form (Chagas)

NECT (Stage 2 HAT)Nifurtimox - Eflornithine

Co-administration

SSG/PMco-administration

VL in Africa

New VL treatmentsin Asia

(SD AmBisome,3 drug combinations)

ASMQFixed-DoseArtesunate/ Mefloquine

ASAQFixed-DoseArtesunate/ Amodiaquine

Oxaborole backup (HAT)

Major CollaboratorsSources for hit and leadcompounds:GSK, Anacor, Merck, Pfizer,Novartis (GNF, NITD), TBAlliance, ...Screening Resources:Eskitis, Institut Pasteur Korea,

SCYNEXIS, Univ. Dundee, ...Reference screeningcentres:LSHTM, Swiss Tropical &Public Health Institute,University of Antwerp


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Utilizing and Strengthening ResearchCapacities in Disease-Endemic Countries

Major Role of RegionalDisease Platforms:

Defining patients needs andtarget product profile (TPP)

Strengthening local capacities

Conducting clinical trials(Phase II/III studies)

Facilitating registration

Accelerating implementationof new treatments (Phase IV& pharmacovigilance studies)

HAT

VL

CHAGAS


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FACT Project budget: EUR 15.3 million, Public financing has been critical Project-specific funding provided through FP5

(EUR 1 Mio)

+ +

Industrial Partners:

Sanofi-AventisEllipse, Catalent

Cipla

Funding:

EUs INCODEV,France, Netherlands,

Spain, UK, MSF

Scientific coordination

& project management

DNDi/TDR

DNDis FACT Project

Development of Fixed-dose Artemisinin-based Combination Therapies


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Initial Objectives:

Responseto public health need Easyto use

Affordable

Available as public good

Today: support implementation ofASAQ (artesunate-amodiaquine) andASMQ (artesunate-mefloquine)

ASMQ (Farmanguinhos)

ASAQ (sanofi)

DNDis FACT Project

2 new treatments delivered


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ASAQ:

Transfer oftechnology

toZenufa

Tanzania

Implemented in Partnership with sanofi

Registered in 2007, prequalified by WHOin 2008

Registered in 30 sub-Saharan Africancountries, in India, and in Colombia

More than 120M Treatments Distributed

Only FDC with a 3 years shelf life

Ambitious risk management plan(Pharmacovigilance)

Available at 0.5$ for children, 1$ foradults

DNDis FACT Project


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PRND and Horizon 2020

M i Ch ll f S t i bl R&D


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Main Challenges for Sustainable R&D

for Neglected Patients

IP & OpenInnovation

RegulatoryBarriers

SustainableFinancing

R&DCoordination

Private SectorInvolvment


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How can EU help fill the gap for PRNDs?

Horizon 2020 Framework

Strengthen European Competitiveness

and EUs position in science

Promote industrial leadership in innovation

and support SMEs

IMI

Pre-competitive mechanism to

support outstanding science

between academia & industry

EDCTPPartnership to support Phase

IIb-III clinical trials for TB, HIV

and malaria

Strengthen capacities in

endemic countries


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Feature Global Health R&D into Horizon2020, and in particular poverty-relatedand Neglected Tropical Diseases

Increase funding for NDs

Improved mechanisms to support entiredevelopment process, from translationalresearch to implementation

Better coordination of research prioritiesat European level

More flexibility on public/privatecollaborations schemes

EDCTP 2 with wider scope (NTDs) andmore resources

How can EU help fill the gap for PRNDs?


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Thank You to All Our Partners & Donors

www.dndi.org

via the 4th Sector HealthProject implemented by AbtAssociates, Inc.

by
http://www.ge.ch/

JP Paccaud

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