Facio Therapies has selected a second series of potential candidates for FSHD drug development. This series encompasses a family of novel small-molecule compounds that repress DUX4 by engaging a molecular target that differs from the target of Facio\u2019s first series of potential drug development candidates.<\/p>\n
The key event in FSHD is the undue production of the DUX4 protein in skeletal muscle. DUX4 sets in motion a cascade of biochemical events that eventually result in the devastating effects of FSHD. In people without FSHD, the production of DUX4 in skeletal muscle is repressed by regulatory mechanisms. Facio\u2019s single goal is to develop therapies that restore this repression as much as possible.<\/p>\n
In January 2018, Facio announced the selection of a first series of potential FSHD drug development candidates. Facio pursues additional series because the severity and course of FSHD are highly variable. This makes it likely that more than one therapeutic drug will be needed to treat all people with FSHD. In order to address this issue head-on, Facio is building a portfolio of product candidates. As an additional advantage, the portfolio approach helps improve Facio\u2019s overall chance of success (or, as the saying goes, provides \u201cmultiple shots on goal\u201d).<\/p>\n
David Dasberg, Facio\u2019s Managing Director, pointed out that the compounds in both Facio\u2019s first and second series repress DUX4 expression in a concentration-dependent, drug-like fashion without impairing the mechanism underlying muscle repair and regeneration in FSHD. \u201cImportantly\u201d, he added, \u201cthe compounds in our second series do so by engaging a different target. This shows the power of our strategy to apply a unique drug screening platform to identify compounds that do repress DUX4, rather than compounds that may or may not repress DUX4 by fitting one of the many possible hypotheses about how DUX4 expression is regulated.\u201d<\/p>\n
Looking ahead, David said: \u201cWe plan to unravel the mode of action of our second compound series over the next months. Meantime, we anticipate announcing progress with our first compound series in the near future.\u201d<\/p>\n