Vaderis: A case study in navigating the orphan drug development maze

In the six years it took to progress from an idea to clinical proof of concept, Vaderis had to navigate a variety of challenges in its attempt to develop the first AKT inhibitor for a non-cancer orphan disease where there were no clinical trial endpoints, let alone approved treatments.

The genesis of Vaderis Therapeutics AG was in 2018, when CEO Nicholas Benedict and his fellow co-founders and former Novartis AG (SIX:NOVN; NYSE:NVS) colleagues CSO Pierre Saint-Mezard and CMO Damien Picard met to discuss an opportunity raised in a Nature Communications paper...