Facio broadens portfolio of potential drug development candidates

Leiden, the Netherlands - August 30, 2018

Facio Therapies announced today that it 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’s first series of potential drug development candidates.

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’s single goal is to develop therapies that restore this repression as much as possible.

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’s overall chance of success (or, as the saying goes, provides “multiple shots on goal”).

David Dasberg, Facio’s Managing Director, pointed out that the compounds in both Facio’s 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. “Importantly”, he added, “the 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.”

Looking ahead, David said: “We 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.”

About FSHD

FSHD (facioscapulohumeral dystrophy) is a skeletal muscle wasting disease that devastates the lives of over 700,000 people worldwide and those close to them. The loss of muscle strength has a huge impact on daily life. Living with FSHD means living with pain, fatigue, and social isolation. Above all, the future becomes uncertain because the course of the disease is unpredictable. About 20% of people with FSHD end up in a wheelchair. Currently, no therapy for FSHD is available other than forms of temporary symptomatic relief.

About Facio Therapies BV

Facio Therapies, established in 2014, is a Netherlands-based company with a single focus: to overcome FSHD by developing a causal therapy that restores the natural repression of the muscle-toxic protein, DUX4. When unduly produced in skeletal muscle, DUX4 sets in motion a cascade of cellular events that eventually result in the muscle wasting seen in FSHD. Facio is the only one in the FSHD field with a fully automated, high-throughput screening platform based on quantifying the DUX4 protein in unadulterated (“primary”) FSHD-affected muscle cells. Facio delivered the first-ever therapeutically relevant proof of principle in FSHD by showing that one of its lead candidates, an orally active small molecule, represses DUX4 levels produced by human FSHD-affected muscle cells in a unique animal model (“in vivo”).

Rooted in, and dedicated to working for the FSHD community, Facio’s business approach is to have a positive impact on lives rather than to maximize financial gain. Since inception, Facio has raised over €16M in equity funding from FSHD-affected families, their friends, FSHD foundations, and Facio’s drug discovery partner, Evotec. Facio’s Board consists of business leaders from the FSHD community – Kees van der Graaf (Chairman; Netherlands), Neil Camarta (Canada), Dave Mackay (USA), Bill Moss (Australia), and Chip Wilson (Canada) – and Evotec’s CSO, Cord Dohrmann (Germany).