The drug has successfully passed two phases of clinical trial and the results of phase 2
will be published in the second quarter of 2021.
Facioscapulohumeral muscular dystrophy, or FSHD, is a rare, progressive,
and debilitating disease for which there is no valid treatment.
The disease is characterized by a gradual decrease in skeletal muscle,
which initially causes weakness of the muscles of the face, shoulders, arms and trunk.
Skeletal muscle weakness leads to significant physical limitations, including the inability to smile and the
difficulty of using the arms for activities. Eventually, many patients become
dependent on the use of a wheelchair for daily activities.
This study is a multi-stage study for phase 2 that is randomized, controlled (with placebo) and in a parallel
and multi-purpose group designed to evaluate the efficacy and safety of losemipod in the treatment of patients
with FSHD muscular dystrophy within 48 weeks. Is.
Patients will participate in this study for approximately 53 weeks. There will be a 4-week screening period,
a 48-week placebo-controlled treatment period, and a 7-day safety follow-up period.
Patients must confirm the diagnosis of FSHD1, and genetic confirmation must be obtained
prior to MRI screening and basal muscle biopsy.
Patients receive medication at random.
That is, take 15 mg of losemipod or placebo twice a day as two 7.5 mg tablets per dose orally.
A total of 4 tablets or 30 mg per day for 48 weeks.
The primary endpoint of this study is to evaluate the effect of leucomipod in inhibiting or reducing
DUX4 (by skeletal muscle biopsies in FSHD patients).
Secondary endpoints include assessment of leucomipod immunity and tolerance,
leucomipod plasma concentrations, leucomipod levels in skeletal muscle,
and leucomipod target involvement in blood and skeletal muscle in FSHD patients.