DNDi 2025 R&D Programmes in Review
Drugs for Neglected Diseases initiative (DNDi)
International non-profit developing safe, effective, and affordable treatments for the most neglected patients.
Each stride forward we are making for neglected patients is propelled by a global coalition of partners united by a common vision: ensuring all people have access to the fruits of scientific progress.
We have marked exciting achievements over the past year across our portfolio of more than 40 research and development projects in nine disease areas – advancing medical innovations with the power to help eliminate neglected diseases, end terrible cycles of illness and poverty, and protect against future pandemics.
Explore our 2025 R&D Programmes in Review for the latest on the progress we’ve been making together – from the laboratory bench to patients’ bedsides. And scroll to learn more about the patients and partners pictured below!
SLEEPING SICKNESS
The European Medicines Agency issued a positive scientific opinion for acoziborole in February 2026, paving the way for the single-dose oral cure for T.b. gambiense sleeping sickness to be registered and distributed in endemic countries. Recruitment for the ACOZI-KIDS paediatric trial of the new treatment was completed in March 2025; results expected in 2026 could guide the extension of acoziborole’s indication to younger children who develop the disease.
Additional countries approved the extended indication for fexinidazole as first-line treatment for T.b. rhodesiense sleeping sickness, with new approvals granted in Zambia and Ethiopia. A key article on its use for the more acute form of the disease was published in The Lancet Global Health last May.
VISCERAL LEISHMANIASIS
Phase II trials of the promising potential new oral treatment LXE408 were completed in India and Ethiopia; results for both studies are expected in 2026. A Phase I trial of DNDI-6899 in healthy volunteers was initiated at Royal Liverpool University Hospital. The UK Medicines and Healthcare products Regulatory Agency provided positive feedback confirming that the non-clinical data package for DNDI-6174 adequately supports progression to a Phase I trial.
Efforts to improve existing treatments for both VL and PKDL continued with the WHO’s ongoing review of the evidence for new VL treatment combinations for Eastern Africa and new PKDL treatment combinations for Eastern Africa and South Asia; updated recommendations are expected in the first half of 2026.
CUTANEOUS LEISHMANIASIS
Our Phase III trial testing the combination of oral miltefosine and thermotherapy was completed. Although overall cure rates did not differ between miltefosine alone and the combination treatment, the combination therapy was significantly more effective than either miltefosine or thermotherapy alone in patients with lesions caused by L. braziliensis.
The protocol and supporting documents for the Phase II trial evaluating the safety, efficacy, and pharmacokinetic profile of two oral LXE408 regimens were submitted to regulatory authorities in Brazil and Panama, and approvals were obtained from both countries. Study initiation is expected in mid-2026.
CHAGAS DISEASE
A reliable test of cure to monitor treatment efficacy is critical to developing new treatments for Chagas disease. Additional evaluations of the MultiCruzi assay confirmed its potential as a marker of parasitological cure, and the project was recognized as DNDi’s 2025 Project of the Year in pre-clinical research.
Our screening project identified ten new T. cruzi active hit series and completed high-throughput screening of more than 160,000 compounds from four partner collections. We progressed toward nominating a pre-clinical candidate from the UW series and made significant advances in identifying the mechanism of action of Series-5824 (MT). The NuestroBen Phase III trial continued recruitment in Argentina and expanded to Bolivia to achieve regional scope.
PARASITIC WORMS
Recognized as DNDi’s project of the year in clinical research, emodepside showed a favourable safety profile and efficacy against both juvenile and adult O. volvulus worms that cause river blindness, meeting the criteria to proceed to part 2 of a Phase II trial.
As part of the eWHORM partnership, we continued work on Phase II studies evaluating the safety and efficacy of oxfendazole for the treatment of river blindness and several other helminthic diseases. We joined the WINGS-4-FGS consortium to advance therapeutics and access to diagnosis and treatment for female genital schistosomiasis and kicked off work with the IVM-KIDS consortium to develop a novel paediatric dispersible ivermectin formulation for young children affected by onchocerciasis, lymphatic filariasis, and soil-transmitted helminths.
MYCETOMA
An early access cohort protocol was initiated in Kassala, Sudan, providing fosravuconazole to patients with eumycetoma while generating real-world safety and efficacy data to inform policy and accelerate access. Partners submitted a regulatory dossier for the treatment to Sudan’s National Medicines and Poisons Board and initiated work to explore the feasibility of a multi-country Phase III trial of the treatment.
To inform risk mapping and planning for future interventions, we completed retrospective data collection on mycetoma cases in India and Senegal, household screening surveys in four regions of Ethiopia, and a socio-behavioural study of patient and community perspectives on treatment access, adherence, and stigma in Kenya.
DENGUE
With partners in the Dengue Alliance, we advanced pre-clinical studies of three host-directed therapy candidates, with two entering final in vivo and in vitro studies to confirm effects and inform dose selection. We continued work to identify biological targets for potential drugs and drug repurposing candidates that could be effective in preventing progression to severe dengue and concluded studies confirming sphingosine 1-phosphate receptor modulators as a valid approach to reducing vascular leakage in in vivo models of dengue infection.
DNDi continued work with partners to characterize the burden of dengue in Africa, with mathematical modelling and seroprevalence studies in Senegal, Ghana, and the DRC completed to inform updated global burden estimates.
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PANDEMIC PREPAREDNESS
The leading COVID Moonshot compound, ASAP-0017445, underwent extensive pre-clinical profiling, showing in vitro and in vivo efficacy against a panel of coronaviruses. Further testing to determine dosing is underway. The compound’s progress has been facilitated by DNDi’s participation in the AViDD ASAP project, which uses artificial intelligence to accelerate novel antiviral drug discovery and optimization.
DNDi and Nucleoside Booster project partners screened 23 nucleosides with broad-spectrum antiviral activity, identifying four promising candidates after cytotoxicity and dose-response testing. The TMEM-16 series progressed with the design and synthesis of new salicylamide derivatives with improved physiochemical profiles.
HIV
A Phase II clinical trial of sustained-release flucytosine (5FC) for cryptococcal meningitis was initiated, with recruitment underway at sites in Malawi and Tanzania. An early safety review found no concerns and confirmed adequate drug exposure. If successful, this twice-daily formulation would replace the current four-times-daily regimen — a significant simplification for overburdened health facilities.
DNDi and IMPAACT4HIV project partners began training trainers and healthcare workers to facilitate the integration of a package of care for people living with advanced HIV disease (AHD) in a hub-and-spoke model in Kinshasa. In the DRC, work began to integrate mpox screening, diagnosis, infection prevention and control, treatment linkage, and surveillance activities into existing AHD platforms.
HEPATITIS C
Results from the Malaysia Ministry of Health-led EASE study confirmed that an 8-week regimen of ravidasvir + sofosbuvir is non-inferior to the standard 12-week course in non-cirrhotic patients, reducing the cost of treatment by 40%. The shorter regimen was approved by the National Pharmaceutical Regulatory Agency (NPRA), Ministry of Health Malaysia, with safety evaluation to monitor any development of resistance to continue for five years.
In Thailand, where HCV remains a significant public health concern, DNDi and partners supported the development of the regulatory submission for ravidasvir, which was submitted to authorities in June.
R&D portfolio - Delivering impact for neglected patients
Together with our partners, we are working on over 40 projects, including more than 20 focused on identifying or developing new chemical entities.
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Thank you to the patients and partners pictured above
SLEEPING SICKNESS: Francis and his wife, Lusitiya, live in Gome village, Ntchisi, Malawi. Francis was the first patient in the country to receive fexinidazole, the newest treatment for rhodesiense sleeping sickness proven effective through research conducted by DNDi and its partners. @ Thoko Chikondi-DNDi
VISCERAL LEISHMANIASIS: Madhuri works in the fields of Bihar while managing the responsibilities of caring for her large family. Diagnosed with visceral leishmaniasis 2017, her illness and the costs of private care forced her to leave school. She was never able to return to her studies. @ JDot-DNDi
CUTANEOUS LEISHMANIASIS: Jorge, a farmer near Santa Fe de Antioquia, Colombia, spent months misdiagnosed before receiving proper treatment for cutaneous leishmaniasis, which caused him to develop painful skin lesions. He faces long, difficult journeys to access the medical care he needs. @ Sydelle Willow Smith-DNDi
CHAGAS DISEASE: Sisters Alejandrina and Maria Delfina have completed their treatment for Chagas disease. They are part of the remote Wiwa indigenous community in Colombia’s Sierra Nevada de Santa Marta, where DNDi collaborates with local indigenous health authorities to improve Chagas testing and treatment. @ Neil Branvold-DNDi
PARASITIC WORMS: Bipin, from Mirzapur village in Bihar, India, first noticed swelling in his leg at the age of 15 but was only diagnosed with lymphatic filariasis in 2021. Years of fever and painful episodes affected his ability to work. Despite ongoing swelling, he continues to work as a security guard and cycles nearly 17 kilometres each day. @ JDot-DNDi
MYCETOMA: Ali, a student from Ethiopia’s Afar Region, is seeking treatment for mycetoma at Boru Meda Hospital near Dessie. Mycetoma is a chronic, progressively destructive infection that affects the skin and deep tissues, and sometimes the bone. Ali has lived with the disease for six years. @ Kumerra Gemechu-DNDi
DENGUE: Ploypilin owns a small shop in Khlong Toey, Bangkok, Thailand. Her two daughters were hospitalized with dengue, a common illness in children that requires close monitoring. They have recovered, but Ploypilin fears a second infection, which can be far more dangerous than the first. @ Luke Duggleby-DNDi
PANDEMIC PREPAREDNESS: Carolina Alvadia is a researcher at the Centre for Medicines Discovery at the University of Oxford, a partner in the COVID Moonshot and ASAP Drug Discovery Consortium that enabled the discovery and optimization of the broad-spectrum coronavirus inhibitor ASAP-0017445. @ Stuart March-DNDi
HIV: Elube, a mother of two from Lilongwe, Malawi, survived cryptococcal meningitis after collapsing at home and being rushed to Kamuzu Central Hospital. For more than a year, she endured worsening headaches, neck pain, vomiting, and repeated episodes of losing consciousness. @ Thoko Chikondi-DNDi
HEPATITIS C: Ng Song Ping is a farmer in rural Pulau Pinang, Malaysia. After being diagnosed with hepatitis C, he had to wait over a decade before receiving treatment. Song Ping was cured after receiving treatment that included ravidasvir, the first hepatitis C drug developed through South-South collaboration. @ Abang Amirrul Hadi-DNDi
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So much fantastic progress!! Wow! Thank you to all involved- you're changing the world!
While DNDi focuses on life-saving R&D, we are solving the 'delivery crisis' in remote areas. At RSMIG, our $75,000 Smart Diagnostic Kiosk is not a luxury—it is a lifeline for the high-altitude villages of Kanungu, Uganda. Our broader $602,000 'Hope for Children' framework is a complete survival ecosystem. It covers nutrition, two age-specific medical tracks, emergency transportation, and professional staffing. In places where clinical access is zero, an investment of $75k in AI-driven diagnostics means the difference between life and death. We are not just implementing technology; we are ensuring child survival where it’s hardest to reach.