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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

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