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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 107
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