Our results to date show that it is, in fact, possible to get a bigger bang for the buck. In little more than one year and at operational costs of only €1.8M (cumulated since inception), we developed the first and only FSHD drug discovery platform that enables reliable quantification of natural DUX4 protein levels in unadulterated (or “primary”) human FSHD-affected muscle cells. In three more months and for an additional €0.5M, we screened 34,000 compounds for their ability to repress DUX4. In four more months and for an additional €0.7M, we validated over 300 hits. In nine more months and for an additional €1.7M, we generated our first series of FSHD drug development candidates. After an additional 8 months and €2.1M of extensive experimental work, we selected lead candidates from this first series, while also selecting a second series as a back-up. In the meantime, we had already invested heavily in our animal model, so just one month and €0.4M later, we were able to prove that our lead series works in an FSHD-relevant animal model.
We are proud of having achieved this level of progress in a little over three years and at a total operational cost of slightly over €7M. We remain, by (bio)pharmaceutical industry standards, extremely capital efficient.