Facio’s parent publishes Annual Report 2017

Leiden, the Netherlands - April 24, 2018

Facio Therapies announced today that its parent company, FSHD Unlimited, published its Annual Report for the year 2017 that consists of a Board Report and the audited annual accounts. Facio ended 2017 with the selection of its first series of potential FSHD drug development candidates and with over €3M in cash, which will enable Facio to continue operations until at least the end of 2018. Facio also disclosed that it is engaging investors to raise additional financing in 2018.

Facio has been founded by FSHD-affected families for the single purpose of meeting the medical needs of all FSHD patients. This translates into a double mission:

  • product mission: to develop safe and effective therapies that stop the progression of FSHD.
  • socio-economic mission: to maximize access to therapy by ensuring affordable pricing, and to allocate 50% of future net profits to (non-medicinal) projects that give FSHD patients a better quality of life.

The Board Report explains the principles underlying Facio’s mission and details progress made in 2017 as well as plans for 2018. David Dasberg, managing director of Facio; Otto Postma, managing director of FSHD Unlimited; and Kees van der Graaf, chairman of the Board, discussed key success factors.

Product mission: Separating the wheat from the chaff

David: “We pursue our product mission in close collaboration with Evotec, a global leader in drug discovery and early drug development. Drug discovery works like a funnel. We tested 34,000 compounds for their ability to suppress DUX4, the muscle-toxic protein that causes FSHD, and came up with more than 300 compounds that did. This means we are able to separate positives (compounds that suppress DUX4) from negatives (compounds that don’t). But it doesn’t stop there. Importantly, we are also able to separate true positives (compounds that suppress DUX4 through pharmacological action) from false positives (compounds that only appear to suppress DUX4). That’s how we arrived at our first innovative series of potential FSHD drug development candidates. We are confident that this result is meaningful because our test system captures the human biology of FSHD and enables determining DUX4 protein levels. Technically, that is extremely complicated, and we remain the only entity in the FSHD field with that system.”

Socio-economic mission: Spending less, achieving more

Otto: “Drug pricing is hotly debated sometimes, but in our view it’s really a business issue that requires a business solution. The key to affordable pricing is the efficient use of capital, simply because that gives a bigger bang for the buck. We have two basic rules to manage our costs. First, we only spend money when that’s critical for developing a therapy, and we do that flexibly by outsourcing the associated work while keeping our fixed cost at a functional minimum (that’s the “buck” part). Second, we make sure that the money we do spend is productive by thoroughly managing the risks that are inherent to drug development (that’s the “bang” part). That way, it took us just two and a half years and less than €5M in total operational cost to get to where we are now. By pharmaceutical industry standards, that’s highly capital efficient.”

The bottom line: Patient power works

Kees: “We are proud of our roots, just as we are proud of so far having raised over €8M in equity from FSHD-affected families, their friends, FSHD foundations, and Evotec. But most important of all, Facio shows that it is possible for patients to empower themselves. Patients can truly come together and push for a safe, effective and affordable therapy by organizing a small team with the proven ability to smartly use capital for rigorous R&D of the highest quality. We fully intend to extend and expand that approach as we set our eyes on our first-in-human study, which we currently plan to start in 2021. Certainly, we are keen to move ahead quickly, but not at the expense of quality. We must – and will – continue to be rigorous in our work because we are very aware that our progress creates hope.”

The full Annual Report 2017 can 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).