Facio enters into agreement with scientific leaders to advance its drug discovery programLeiden, the Netherlands - June 23, 2015
Leading academic institutes active in the field of FSHD have agreed to transfer materials to Facio for its drug discovery program aimed at stopping the progression of FSHD.
Facio’s drug discovery program is designed to identify compounds showing activity as a potential treatment to stop the progression of FSHD. The Dutch based Leiden University Medical Center, department of Human Genetics led by Prof Dr Silvère van der Maarel (“LUMC”), Dr Stephen Tapscott, a full member of Fred Hutchinson Cancer Research Center’s Human Biology Division (“Fred Hutch”) and Prof Dr Rabi Tawil from the Neuromuscular Disease Center at the University of Rochester (“URMC”) will support this program by providing human FSHD-affected muscle cell lines and know-how.
Together with Evotec, Facio recently started its drug discovery program to identify small molecules that increase the activity of SMCHD1 and restore the repression of DUX4. The results from this program are expected in the first half of 2016.
Professor van der Maarel: “Our providing material to Facio supports the translation of the combined research on FSHD, performed by Dr Tapscott, Dr Tawil and myself, towards an FSHD therapy. I am really pleased working with the Facio team and look forward to the breakthrough towards a causal therapy for people with FSHD.”
David Dasberg, managing director of Facio, commented: “We are very pleased to work together with these leading scientist in the field of FSHD, who have revealed the major role of SMCHD1 in the progression of this epigenetic disease. Together with our partner Evotec and with the support from Drs van der Maarel, Tapscott and Tawil we believe we can overcome FSHD together.”
For more information on the Leiden University Medical Center and the department of Human Genetics, please visit LUMC
For more information on Fred Hutch and the Human Biology Division, please visit Fred Hutch
For more information on the University of Rochester and the MDA Neuromuscular Disease Center, please visit URMC
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).