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New therapeutic targets: moving form pre-clinical data to phase 2 studies

Presented By
Prof. Martin Wilkins, Imperial College London, United Kingdom
Conference
ERS 2018

In the pathogenesis of PAH, i.e. the development of vasoconstriction and excessive proliferation, 3 pathways have a central role: endothelin, nitric oxide, and prostacyclin. These pathways provide validated targets for PAH drugs [9].

“Despite the success of these drugs in improving the wellbeing of our patients, we feel that we can do better”, said Prof. Martin Wilkins (Imperial College London, UK) at the beginning of his lecture. “The ultimate goal is to stop disease progression and, if possible, reverse the remodelling that characterises PAH. The problem is that we lack knowledge about the interplay between these pathways in the remodelling process. The good news is that we have a number of druggable targets in each of these pathogenic mechanisms (see Table). The real challenge is to prioritise and identify which of these candidate drugs might offer the most rapid progress.”

Many of these drugs have entered clinical trials. However, only prostacyclin analogu...



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