Responding to Neglected Patients’ Needs Through Innovation Spring Gombe-Götz Policy Advocacy Manager 8 November, 2018 Federal Parliament Belgium Best Science for the Most Neglected
15 years ago: the creation of DND i 1999 Nobel Peace Prize awarded to MSF, who commits the prize money to the Drugs for Neglected Diseases Working Group Initial meeting in Nairobi 2003 DNDi creation with founding partners: • Kenya Medical Research Institute, Kenya Institut Pasteur, France • • Indian Council of Medical Research, India • Médecins Sans Frontières • Ministry of Health, Malaysia • Oswaldo Cruz Foundation (Fiocruz), Brazil WHO –TDR (Special Programme for • Research and Training in Tropical Diseases) as a permanent observer
Dynamic portfolio: New disease areas, new models… Neglected diseases Neglected patients Neglected models Antimicrobial resistance Mycetoma Hepatitis C Treated patients Excluded patients Public health approach Testing ravuconazole Incubation of GARD
Short-, medium- and long-term approaches to address immediate patient needs and deliver innovative medicines > New chemical entities > New formulations (NCEs) > Completing registration dossier > New indications for existing > Geographical extension drugs Long-term projects Medium-term projects Short-term projects Translation Research Development Implementation Development > 5 3-5 1-2 YEARS YEARS YEARS 4
DND i portfolio: Improve existing treatments & develop NCEs RESEARCH TRANSLATION DEVELOPMENT IMPLEMENTATION Screen Hit to Lead Lead Opt. Pre-clinical Phase I Phase IIa/PoC Phase IIb/III Registration Access SCYX-1330682 NECT NECT Acoziborole Fexinidazole SCYX-1608210 HAT Nifurtimox-Eflornithine Nifurtimox-Eflornithine Combination Therapy Combination Therapy New Treatments DNDI-6148 DNDI-5421 SSG&PM SSG&PM Leish H2L Screening for HIV/VL DNDI-5610 Africa Africa DNDI-0690 Booster New Treatments LEISH. Amino H2L New VL Treatments New VL Treatments DNDI-5561 for PKDL pyrazoles Asia Asia Daiichi- GSK3186899 CGH VL MF/Paromomycin Sankyo LH2L DDD853651 Series 1 Combo for Africa GSK3494245 New VL DDD1305143 Leish New CL Treatments L205 Series CpG-D35 (CL) Combination Latin America New Benz Chagas H2L Benznidazole Benznidazole Biomarkers Screening Regimens +/- CHAGAS Paediatric Dosage Paediatric Dosage Booster H2L fosravuconazole Chagas Form Form Daiichi- C205 series Fexinidazole Sankyo CH2L Macro Oxfendazole Emodepside FILARIA Screening Filaricide 3 ABBV-4083 Fosravuconazole MYCETOMA Superbooster LPV/r pellets Two ‘4-in-1’ PAEDIATRIC with dual NRTI Therapy LPV/r/ABC/3TC HIV Paediatric HIV/TB Ravidasvir/ HEPATITIS C Ravidasvir Sofosbuvir Malaria Malaria FDC ASMQ FDC ASAQ New Chemical Entity (NCE); Fexinidazole (for HAT and Chagas disease) = 1 NCE; Fosravuconazole = 1NCE
DND i ’s success is only possible through innovative partnerships CRITERIA FOR SUCCESS � Share the same vision � Mutual understanding Over 170 � Involvement throughout the whole process Partnerships Worldwide Biotechs Pharmaceutical companies PDPs International organisations NGOs CROs Universities Research Institutes
Open innovation to speed up drug discovery. NTD Drug Discovery Booster, Est. 2015 Open Synthesis Network, Est. 2016 The Mycetoma Open Source project (MycetOS), Est. 2017 Anybody & Everybody!
For each disease, a Target Product Profile to guide all decisions: HCV TPP 8 Characteristics Ideal Acceptable >95% SVR >= 95% SVR Efficacy Safety No side effects Minimal side effects Pangenotypic Yes Yes Treatment duration Same regimen F0-F4 12 weeks F0-F3 10 weeks (8 wks ?) F0-F3 12 weeks with RBV F4 12 weeks F4 24 weeks without RBV F4 Populations Mono & HIV co-infected Mono & HIV co-infected ; polytransfused & people who polytransfused & people who inject drugs inject drugs Dosing FDC once a day Two tablets once a day (+/-RBV) None in HIV/HCV Manageable in HIV/HCV Drug-Drug Interactions POC diagnosis, Triage (F?), POC diagnosis, Triage (F?), Monitoring SVR12 SVR12, Minimal safety monitoring ~ US$ 300 by 2017 ~ US$ 300 by 2020 Cost
DNDi HCV strategic objectives: Severe Disease • Develop new, affordable, pan- genotypic TT for HCV • Simplify HCV test & treat strategies and develop innovative models of care to support scale up • Improve access (IP, regulatory, pricing, etc.) and affordability of HCV TT in countries Minimal Disease
DNDi Hepatitis C Strategy: 3 pillars 3 1 2 Simplify Catalyse Accelerate TREATMENT ACCESS R&D STRATEGIES Working with health Accelerating the development Supporting affordable providers to scale – of promising drug access to all DAAs up treatment candidates with with with Primary Pharma Pharma healthcare companies companies doctors Civil Society Non Governments organisations Governmental Governments Organizations
A mature and dynamic portfolio, with strong partnerships and donor support GARDP: new initiative 39 R&D projects 20 entirely created by WHO and DND i new chemical entities in 8 disease areas and incubated by DND i (NCEs) in the pipeline with 7 treatments delivered 210 staff 4 disease-specific clinical EUR 532 million raised Half in endemic countries trial platforms equally from public Close to 1,000 people Several technology and private sources working on DNDi projects transfers
7 new treatments delivered since 2007 � Easy to use 2010 SSG&PM � Affordable Visceral leishmaniasis in E Africa � Field-adapted Now 1st line in all countries � Non-patented 2011 PAEDIATRIC BENZNIDAZOLE 2007 ASAQ Chagas disease Malaria Two sources developed >500 million patients reached 2011 NEW VL TREATMENT ASIA 2008 ASMQ Visceral leishmaniasis in Asia Malaria Support to elimination Used in Africa and Asia programme 2016 SUPERBOOSTER THERAPY 2009 NECT Paediatric HIV Sleeping sickness Recommended by WHO 100% of stage-2 patients
Sleeping sickness, two new oral treatments to change the history of the disease Since 2009 2018 2021 15 years ago NECT Fexinidazole Acoziborole Melarsoprol Improved therapy Single-dose, oral Oral (10 days) Toxic, resistant Eflornithine Not available
The Global Antibiotic R&D Partnership ( GARDP) Created by WHO and DND i , incubated by DND i Antimicrobial resistance is a major and rapidly growing global public health challenge, with estimates of up to 700,000 deaths per year. Pathogens DRUG Focus: RESISTANCE & UNMET Key Diseases & Drug-resistant bacterial infections • NEED syndromes populations for which adequate treatment is not available. Address global health priorities that • reflect the realities of clinical practice. EXTERNAL CONSULTATION AND Scope: GOVERNANCE Global including low- and middle- • income countries. TARGET PRODUCT PROFILES
GARDP’s priorities and programmes Developing and delivering new or improved antibiotic treatments for which , while endeavouring to ensure sustainable access. 2023 objectives Neonatal sepsis: developing treatments for highly drug-resistant infections in babies. Develop 4 new treatments through: • Improving existing antibiotics Paediatric antibiotics: exploring ways to • Developing new chemical entities. optimize current and develop new antibiotics for children. Build a robust pipeline of pre-clinical Sexually-transmitted infections: develop a and clinical candidates end to end. new treatment for drug-resistant gonorrhoea and other STIs. Actively support appropriate use of Memory recovery and exploratory: revive and access to new antibiotic and evaluate old knowledge and abandoned treatments. projects, support early research.
The support of our donors: €532M received out of €730M needed by 2023 57% 43% UNRESTRICTED FUNDING (48%) - € 255M • United Kingdom – DFID (€136.4M) – (£113M) Public Private • Médecins Sans Frontières (€83.5M) EUR EUR • Switzerland – SDC (€17.4M) – (CHF 19.2M) 303M • 229M Spain – AECID (€12M) • Other Private Foundations - Rockefeller, Slim, Starr, FINEP, Moreau, BBVA (€5.6M) STRICTLY RESTRICTED FUNDING (23%) – € 121M PORTFOLIO FUNDING (29%)- € 156M • Bill & Melinda Gates Foundation (€53M) – ($63.3M) • Bill & Melinda Gates Foundation (€50.6M) – Japan GHIT Fund (€20.2M) • ($60.4M) • Wellcome Trust (€16.2M) • Netherlands – DGIS (€33M) • European Union – FP5,6,7& EDCTP (€13.6M) • France – AFD & MAEE (€16.3M) • Médecins Sans Frontières (€10.2M) • Unitaid (€12.5M) – ($14.4M) • USA – NIH/NIAID (€1.6M) – ($1.8M) • Germany – KfW & GTZ (€20.1M) United Kingdom – DFID (€1.6M) – (£1.2M) • USAID (€9.4M) – ($10M) • • Switzerland – Canton de Genève (€1.9M) • Fundación Mundo Sano (PRV) (€6.2M) • UBS Optimus Foundation (€1.5M) – (CHF 2M) • Medicor Foundation (€3.8M) • Switzerland – SDC (€0.7M) – (CHF 0.9M) • WHO/TDR (€2.6M) • The Global Fund – AMFm (€0.5M) • Norway – NORAD (€2.8M) Ruta’N Medellin (€0.3M) • Brazil - BNDES & MoH (€0.4M) • • Kalacore (€0.3M) Note: Does not include GARDP funding
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