“It just went completely white.” This is the how fisherman Akoyo Osumaka describes going blind in 2011, in the remote village of Babagulu in the Democratic Republic of Congo (DRC). It was a slow, creeping blindness that began a year earlier. It robbed him of his livelihood and threw his family life into disarray.
The Japanese pharmaceutical company Astellas Pharma Inc. is the eighth pharmaceutical company to join the Neglected Tropical Diseases Drug Discovery Booster, a global consortium launched in 2015 by DNDi to speed up the process and cut the cost of finding new treatments for leishmaniasis and Chagas disease.
Phase II/III study results confirmed that fexinidazole, DNDi’s first new chemical entity is safe and effective, and presents significant advantages over current treatment as it removes the need for lumbar puncture and systematic patient hospitalization. A regulatory dossier has been submitted to the European Medicines Agency for fexinidazole as the first all-oral treatment for sleeping sickness.
Three new chemical entities entered the pre-clinical pipeline, bringing to six the number of new compounds progressing in the leishmaniasis portfolio, with two already nominated as clinical candidates to progress to Phase I. The first patient was recruited in India in Phase II studies to test new treatments for PKDL, while clinical sites were being prepared in Bangladesh and Sudan. A site was initiated in Sudan for a Phase III study to test a new combination treatment for leishmaniasis in the African region. In Latin America, a Phase II study on new combination treatments for cutaneous leishmaniasis progressed well.
Two Phase II trials are ongoing, with the study in Bolivia on benznidazole for improved treatment of chronic indeterminate Chagas disease having completed recruitment at the end of 2017, and a study in Spain in the early stages of patient recruitment to evaluate fexinidazole as Chagas treatment. A pilot project to increase access to diagnosis and treatment in five endemic communities in Colombia showed excellent interim results and will report one-year results in mid-2018. In 2018, DNDi and Fundación Mundo Sano will launch a new Chagas Disease Regional Access Framework.
A first-line “4-in-1” fixed-dose combination (abacavir/lamivudine/lopinavir/ritonavir) is on track to be submitted for registration in late 2018. Preliminary results from the LIVING study for an interim optimized paediatric formulation were presented at the end of 2017, showing that these “oral pellets” are effective, well tolerated and well accepted by caregivers and children.
Dr Robert Don, Discovery and Pre-Clinical Director at DNDi (2005-2017), reflects on challenges and achievements in drug discovery at DNDi over the last 12 years.
The journey to the sleeping sickness trial site in Isangi from the DNDi office in Kinshasa begins in the domestic airport of DRC’s capital city and ends more than a day later halfway across the country in a barge crossing the Congo river. In between: hours spent navigating potholed dirt roads, collapsed bridges, checkpoints, and multiple river crossings. Once at Isangi, canoes must be used to reach many of the patients as there are no roads.
Yet for DNDi’s clinical team in DRC, Isangi is one of the easier-to-reach sites. The DRC and the Central African Republic (CAR) both pose daunting challenges that must be overcome to develop better treatments for patients suffering from sleeping sickness.
A l’occasion de l’Evénement économique 2017 du 9 novembre dernier, la Chambre de commerce, d’industrie et des services de Genève (CCIG), le Département de la sécurité et de l’économie (DSE) et l’Office de Promotion des Industries et des Technologies (OPI) ont décerné le Prix 2017 de l’innovation à l’initiative Médicaments contre les Maladies Négligées (DNDi).
As part of the ‘Evénement économique 2017’ that took place on November 9, the Geneva Chamber of Commerce, Industry and Services (CCIG), the Department of Security and Economy (DSE) and the Office for the Promotion of Industries and Technologies (OPI) awarded the Innovation Prize 2017 to the Drugs for Neglected Diseases initiative (DNDi).
GARDP has set up its team, scientific advisory committee, and R&D strategy. Three programmes are underway: neonatal sepsis, gonorrhoea, and an initiative to recover and connect the vital knowledge of scientists in antibiotic drug development. In recent weeks, both the G20 Health Ministers and outgoing WHO Director-General Margaret Chan have expressed support for GARDP and called for more R&D into drug-resistant infections.
Bernard Pécoul, Executive Director, DNDi
Ambitious targets serve the obvious purpose of galvanising the NTD community around a shared goal, and the London Declaration succeeded in bringing new actors to the table and casting a much-needed spotlight on the neglect of these diseases. But the job is far from done, and we should not be lulled into a sense of security or complacency by too much celebrating of good news.