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DNDi_AR_2015
DNDi_AR_2015
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R&D MODEL<br />
& PORTFOLIO<br />
ADAPTED TREATMENTS FOR THE BENEFIT<br />
OF NEGLECTED PATIENTS<br />
The R&D strategies developed by DNDi since its inception aim to<br />
address the immediate needs of <strong>patients</strong> by improving existing<br />
therapeutic options in the short term, whilst undertaking longer<br />
term research to identify and develop entirely new compounds<br />
which will be valuable adapted tools, particularly for elimination<br />
targets set by the World Health Organization. Although not<br />
necessarily breakthrough medicines, six new treatments have<br />
been delivered to date as a result of the short-term strategy,<br />
which have brought significant benefits to <strong>patients</strong>.<br />
The year 2015 has been a turning<br />
point for DNDi, as long-term<br />
investments have now filled the<br />
drug development pipeline with<br />
thirteen new chemical entities<br />
(NCEs) included by the end of the<br />
year, the vast majority of which<br />
are orally available compounds for<br />
systemic use. The most clinically<br />
advanced of these are for sleeping<br />
sickness: fexinidazole, which was<br />
identified from compound mining<br />
and is a ten day oral treatment,<br />
and SCYX-7158, which is entering<br />
Phase II trials as a potential singledose<br />
oral treatment and is the first<br />
molecule to arise from DNDi’s lead<br />
optimization programme.<br />
Leishmaniasis is a complex family<br />
of diseases, and the identification<br />
of new compounds has proved<br />
challenging. Compound libraries<br />
from a variety of sources have been<br />
screened and, despite the inevitable<br />
loss of compounds to attrition, NCEs<br />
from the nitroimidazole, oxaborole,<br />
and aminopyrazole chemical<br />
families are undergoing lead<br />
optimization to combat Leishmania<br />
infections, with the nitroimidazole<br />
VL-0690 selected to go forward to<br />
14 › DNDi Annual Report 2015