Pharma Update
Anti-latent myostatin mAb in the neuromuscular disorder FSHD
Ph II (MANOEUVRE) in FSHD, a disorder with high unmet need, ongoing
FSHD: Rare genetic disease with
high unmet need
Ph II (MANOEUVRE) trial design in FSHD
Progressive asymmetric
skeletal muscle weakness
and wasting predominantly
in the face, shoulder and
upper arm muscles
The lower limb and
abdominal muscles
are also involved
Enrollment
N=48
GYM329
Pre-
ய
E
R
Treatment
Placebo
(1:1)
4 weeks
GYM329
52-week double-blind
treatment
52-week active treatment
extension
Other symptoms can include retinal vascular pathology,
hearing loss and respiratory impairment
• Genetic muscle disorder driven by ectopic
•
•
expression of the DUX4 transcription factor
Estimated global prevalence of 3.2-4.6 per
100,000, affecting adults and children¹
Currently no DMTs against muscle wasting and
weakness in FSHD
Primary endpoints:
Percent change in contractile muscle volume (CMV) of quadriceps femoris as assessed by MRI bilaterally
Percentage of participants with adverse events (AEs)
• Preclinical studies showed that GYM329 has superior muscle strength-improvement effects in mice
compared with other anti-myostatin therapies²
• Best-in-Disease potential: Ph II (MANOEUVRE) of GYM329 in FSHD ongoing
• GYM329 is further explored in additional diseases and combinations
1. Faux-Nightingale A, et al. Arch Rehabil Res Clin Transl. 2021; 2. Igawa T, et al. Immunol Rev. 2016; DMT-disease modifying therapy; E-enrollment; R=randomization; FSHD-facioscapulohumeral muscular dystrophy;
MRI-magnetic resonance imaging; DUX4-double homeobox 4; DMT-Disease modifying therapy; GYM329 in collaboration with Chugai
Roche
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