FSHD Unlimited publishes its first Annual Report

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 .