Facio partners with Evotec and initiates FSHD drug discovery programLeiden, the Netherlands - April 29, 2015
Facio Therapies and Evotec have entered into an agreement aimed at the identification of compounds showing activity as a potential treatment to stop the progression of FSHD. This agreement marks the start of finding a drug to overcome FSHD.
Facio’s drug discovery program aims to identify compounds that increase the activity of SMCHD1 and restore the repression of DUX4. These compounds are so-called small molecules, relatively simple compounds that are manufactured by chemical synthesis.
The project with Evotec entails the set-up and execution of an automated high-throughput screen to identify small molecules having a positive effect on SMCHD1 and DUX4 activity in human FSHD-affected muscle cell lines. The compounds that show promising activity in this screen are expected to be available in the first half of 2016. These compounds will require extensive further testing to produce compounds that are suitable for the development of a therapeutic for the treatment of FSHD. All in all, this is a challenging process that may take several years before clinical testing in humans can begin.
“The start of our drug discovery program is a significant step forward in our pursuit to overcome FSHD”, said Facio’s Managing Director David Dasberg. “Evotec is a leader in the field of drug discovery and their expertise with muscle tissue will allow us to move forward as fast as possible.”
Dr Mario Polywka, Chief Operating Officer of Evotec, commented: “We are very excited about the opportunity to collaborate with Facio on this important disease. With our world-leading expertise in tissue analysis and high-throughput screening we are confident to add real value to the progression of this program.”
Evotec is a worldwide leading drug discovery and development company focused on rapidly progressing innovative product approaches with leading pharmaceutical and biotechnology companies. Evotec has long-term alliances with partners including Bayer, Boehringer Ingelheim, CHDI, Genentech, Janssen Pharmaceuticals, MedImmune/AstraZeneca, Roche and UCB. For more information, please visit www.evotec.com.
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).