FSHD Unlimited publishes 2016 Annual Report

Leiden, the Netherlands - May 5, 2017

Facio provides updates on operational progress and financial results

Facio Therapies announced today that its parent company, FSHD Unlimited, published its audited Annual Report for the year 2016. Having raised €875,000 in equity, the Facio group of companies ended the report period with €1.1M in cash and cash equivalents. Expenses were mainly directed at Facio’s drug discovery program, resulting in 300+ validated hits early 2017 with the potential to tackle the cause of FSHD. These results enable Facio to start building a portfolio of DUX4-repressing compounds representing multiple shots on goal.

During 2016, Facio achieved two breakthroughs in FSHD drug discovery. In August, Facio announced the development of the first-ever validated platform to screen large numbers of compounds for their ability to repress the production of the natural DUX4 protein in cultured muscle cells derived from FSHD patient biopsies. Undue production of DUX4 in skeletal muscle is the cause of FSHD. In December, Facio announced the identification of a variety of compounds with the desired effect (known as “hits”) in its proprietary platform after screening 34,000 compounds. In April 2017, validation of 300+ hits was announced. Currently, Facio and its drug discovery partner, Evotec, are further characterizing these hits, especially with respect to how they work on DUX4. On that basis, selected hits will undergo chemical modification to optimize their safety and efficacy.

“In 2016, we beat the odds by doing something that many believed impossible: establish the very first natural DUX4 drug discovery platform,”noted Kees van der Graaf, Chairman of FSHD Unlimited. “Even more importantly, our platform generated the largest pool of thoroughly validated hits in the FSHD field. We now look forward to developing a portfolio of truly viable disease-modifying drug candidates.”

Facio arose from, works for, and is owned by the FSHD community. In November 2016, Facio raised €875,000 in equity from existing shareholders as well as the Canadian and the Dutch FSHD Foundation. Since September 2014, Facio has received a total of over €3.3M in equity funding.

We are very proud of the strong and growing support from the FSHD community,” commented Kees van der Graaf. “We expect to raise additional capital from the community in 2017, further strengthening our patient-centric business approach towards the development of safe, effective and affordable FSHD drugs.”

The full 2016 Annual Report may be downloaded here.

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).