FSHD Unlimited publishes its first Annual ReportLeiden, the Netherlands - June 27, 2016
Facio provides updates on operational progress and financial results
FSHD Unlimited, the parent company of both Facio Therapies and Facio Intellectual Property, has published its first audited Annual Report, which covers the period of September 2014 (inception) through December 2015. Having raised €2,475,000 in equity, the Facio group of companies ended the report period with €1,428,593 in cash and cash equivalents. Expenses were mainly directed at Facio’s drug discovery program towards a therapy for FSHD. The first result of that program – an initial series of candidate therapeutic compounds – is expected to become available before the end of 2016.
Together with German-based Evotec, and with the support of Leiden University Medical Center (Netherlands) and University of Rochester Medical Center (USA), Facio started its drug discovery program in June 2015. The aim is to identify small-molecule compounds able to take the DUX4 gene, which in FSHD is responsible for production of the toxic DUX4 protein, back to the repressed state seen in people without FSHD. Importantly, measuring DUX4 protein levels in patient-derived muscle cells captures the natural biological complexity of FSHD. In the second half of 2015, Facio fully focused on thoroughly characterizing FSHD-affected muscle cell lines and on defining the conditions that enable testing thousands of compounds. Facio expects to obtain an initial series of compounds with the desired effect before the end of 2016. Immediately thereafter, Facio will investigate their mode of action in FSHD and conduct extensive further testing towards so-called lead compounds suitable for development into a human therapeutic.
“Research has shown that DUX4 expression in FSHD is highly sporadic and limited to a small fraction of muscle cell nuclei”, noted Kees van der Graaf, Chairman of FSHD Unlimited. “Therefore, reliably measuring DUX4 protein in FSHD-affected muscle cells to enable testing thousands of compounds in an automated process is a tremendous technical challenge. We believe that meeting this challenge represents a major advancement beyond the state of the art. Moreover, generating the initial series of candidate therapeutics would mean a major step towards a disease-modifying therapy in less than 18 months.”
Facio actively pursues alignment with the FSHD community. The €2,475,000 raised in equity during the report period includes €2M from leading community members, Kees van der Graaf and Bill Moss AO, as well as €475,000 from the FSHD Global Research Foundation (Australia) and private investors. In early 2016, the FSHD Stichting (Netherlands) provided a €100,000 convertible loan.
“We are very grateful for the support from the FSHD community”, commented Kees van der Graaf. “While our cash position at year-end 2015 is sufficient to fund our operations for at least the year 2016, we are pleased to note growing interest from parties both within and outside the community in financially supporting our program.”
The full 2014-2015 Annual Report may be downloaded here.
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).