Facio reinforces teamLeiden, the Netherlands - January 16, 2015
Facio Therapies has retained Joris De Maeyer and Lars Bastiaanse to support its drug discovery and development program aimed at stopping the progression of FSHD. Joris is responsible for drug discovery and early development, while Lars is responsible for Facio’s overall drug development strategy.
Joris De Maeyer is seconded to Facio from DM&S consulting (Mechelen, Belgium) as Director R&D. He has over 13 years’ experience leading outsourced and collaborative programs in drug discovery and early drug development. Joris had various roles of increasing responsibility at Johnson & Johnson, Movetis and Shire. In his most recent position as Director Discovery, he was responsible for part of Shire’s GI and CNS discovery portfolios. Joris holds a PhD (medical sciences) from Ghent University and an MSc (bioscience engineering in cell and gene biotechnology) from the Catholic University of Leuven.
Lars Bastiaanse is seconded to Facio from PMD Consulting (Riel, Netherlands) as Director Development. He has 20 years’ experience with drug development, project management, and change management. Among his clients are both established and start-up biopharmaceutical companies from Europe, the US, and Japan. Lars was general manager of Kinesis Pharma and of 3D-PharmXchange, two independent drug development consultancy firms. Prior, he held positions of increasing seniority at various Johnson & Johnson companies.
The key event in FSHD is the production of a protein called DUX4. This protein plays a normal role in early fetal development, but is highly toxic when produced in muscle tissue. In people without FSHD, the DUX4 gene is repressed in muscle tissue. Facio’s goal is to overcome FSHD by developing a therapeutic drug that takes the DUX4 gene back to its repressed state by boosting the activity of a natural repressor of DUX4, a protein called SMCHD1. In FSHD the activity of SMCHD1 is too low. The first step towards this goal will be the discovery of small molecules that boost SMCHD1 activity. Small molecules are relatively simple molecules that are manufactured by chemical synthesis. Most therapeutic drugs are small molecules.
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).