Biotech and FSHD
by Manuel Cabral

UP TO DATE : FSHD and biotech, the next step

(Jan 18, 2017) - FDA Removes Resolaris' Partial Clinical Hold: Muscular Dystrophy Trials Back on Schedule RDR Staff 
In mid-December, aTyr released data from their Phase 1b/2 trial of resolaris for the treatment
of multiple rare genetically distinct myopathies such as limb-girdle muscular dystrophy 2B
(LGMD2B), and facioscapulohumeral muscular dystrophy (FSHD). While the data looked
promising, the FDA put the trial on partial clinical hold based on a dosing ceiling.
That was then, this is now. Today, the company announced that not only has the FDA has
removed the hold, allowing aTyr to continue trial for resolaris, but the FDA has fast tracked
the orphan drug as well.
The Phase 1b/2 trial was a 12-week, international, open-label, intra-patient, placebo, dose
escalation study administering intravenous infusions of resolaris with dose escalation in
LGMD2B and FSHD adult patients in 2 groups, twice weekly.
• Group A: 4 patients with FSHD received infusions of Resolaris with the highest dosing up
to 1.0 mg/kg biweekly for a period of 4 weeks.
• Group B: 10 patients with LGMD and 4 patients with FSHD received infusions of Resolaris
with the highest dosing up to 3.0 mg/kg biweekly for a period of 4 weeks.
Both groups showed statistically significant results in treatment.
With the Fast Track Designation, the FDA will facilitate and expedite development and
review of resolaris. Through the Fast Track program, a product may be eligible for priority
review at the time of BLA and may be eligible to submit sections of the BLA on a rolling
basis as data become available.
? aTyr Pharma Inc (NASDAQ) Jumps Higher on Fast Track Decision- January 21, 2017

2) Resverlogix Corp (Canada)
Resverlogix Highlights Unique Properties of Apabetalone in Both a Rare Muscular
Dystrophy (FSHD) and Neurodegenerative Eye Disease
Emergence of new potential indications for apabetalone (RVX-208) via third party research
CALGARY , Feb. 13, 2017 /CNW/ - Resverlogix Corp. ("Resverlogix" or the "Company")
(RVX.TO) today highlighted two potentially new indications recently identified by third
party academic research involving its lead drug, apabetalone. The first indication was
published in the Journal of Neuroinflammation which described the inhibition of
bromodomain and extra-terminal (BET) epigenetic readers, including apabetalone (RVX-
208), as having therapeutic potential in degenerative diseases of the eye (retinal). The second
indication was highlighted by research conducted at Saint Louis University , demonstrating
apabetalone mediated modulation of important targets in Facioscapulohumeral Muscular
Dystrophy (FSHD). Both indications are areas of interest and licensing potential for
Findings on Facioscapulohumeral Muscular Dystrophy: FSHD is a debilitating disease that
arises from a genetic defect and is one of the most common types of muscular dystrophy
affecting approximately 12 in 100,000 people. This muscle disorder initially affects the
muscles of the face, shoulder blades and upper arms with eventual spread to other muscles of
the body. In research conducted at Saint Louis University and subsequently used in a patent
application, BET inhibitors were shown to inhibit expression of the DUX4 gene, which is
expected to reduce the severity of symptoms in this disease. Apabetalone treatment, when
compared to other BET inhibitors, demonstrated a similar repression of DUX4, however no
transient suppression of MYH2 (a differentiation marker for myotubes) was observed. This
differential effect on the marker of muscle cell differentiation was unique to apabetalone and
demonstrated the distinct properties of this BET inhibitor.
Treatment of FSHD via BET protein inhibition offers a novel therapeutic indication for
apabetalone and this class of compounds.

04/11 Resolaris is currently in a Phase 1b/2 clinical trial in adult patients with facioscapulohumeral muscular dystrophy (FSHD);
05/11 Life Technologies Corporation The Company is conducting approximately three open label trials in patients with early onset FSHD, in adult patients with FSHD or LGMD2B 
03/12 Life Technologies Corporation. The Company is conducting approximately three open label trials in patients with early onset FSHD, in adult patients with FSHD or LGMD2B
06/12 aTyr Pharma: Another Phase 1b/2 trial is testing patients with an early-onset form of FSHD. In addition, Resolaris is currently being evaluated in two Phase 1b/2
23/12 Acceleron Pharma (XLRN) Says First Patient Treated in Phase 2 Trial of ACE-083 in FSHD
Our understanding of its novel mechanism of action suggests that ACE-083 may become an important new treatment for FSHD patients with muscle
April 24, 2017 
aTyr Pharma Announces Promising Top-Line Results from Resolaris™ Phase 1b/2 Clinical Trial in Patients with Early Onset Facioscapulohumeral Muscular Dystrophy
1.  63% of Patients Observed Increased Muscle Strength -
2. Resolaris Demonstrated a Generally Well-Tolerated Safety Profile in Younger Patient Population

June 20, 2017 
Evotec Announces Strategic Investment in Facio Therapies to Support Development of Novel FSHD Therapy. Evotec to participate in Facio Therapies' 2017 funding round alongside members of the FSHD community (total volume: EUR 4.8 m) Evotec expands its efforts in using patient-derived disease models to identify and develop disease-modifying therapies in areas of high unmet medical need