Facio screens 34,000 compoundsLeiden, the Netherlands - December 28, 2016
Facio Therapies announced today that it has screened approximately 34,000 compounds for their ability to repress the production of the muscle-toxic DUX4 protein in cultured muscle cells derived from FSHD patient biopsies. As planned, a variety of compounds with the desired effect (known as “hits”) have been identified.
In September 2016, Facio announced that, together with its drug discovery partner, German-based Evotec, it had established the first-ever screening platform that enables reliable quantification of naturally occurring DUX4 protein in cultured FSHD-affected muscle cells. Undue production of DUX4 is the cause of FSHD. When produced in muscle tissue, DUX4 is highly toxic due to a cascade of events eventually resulting in the devastating effects of FSHD. In people without FSHD, the production of DUX4 is repressed by regulatory mechanisms in the muscle cell. Facio’s single goal is to pursue a therapy that restores this repression as much as possible.
“This is the very first large scale FSHD screening campaign on record that quantifies the naturally occurring DUX4 in patient derived skeletal muscle cells”, noted David Dasberg, Facio’s Managing Director. He added: “Hits are the foundation for the development of our FSHD drug candidates, so we have to be very thorough. That’s why we are now subjecting all our hits to a battery of additional tests to weed out false positives. We look forward to completing this process in the months ahead.”
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).