Healthcare professionals
VILTEPSO is an exon-skipping therapy studied in children as young as 4 years
VILTEPSO is designed to bind to and induce skipping of exon 53 of the dystrophin pre-mRNA, resulting in the production of a shortened dystrophin protein that contains essential functional portions
An estimated 10% of patients with DMD would be amenable to exon 53 skipping*
VILTEPSO was discovered through an innovative triple-screening approach designed to identify antisense oligonucleotides with the highest possible exon-skipping efficiency
*Common dystrophin gene mutations eligible for exon 53 skipping include deletions of exons 45-52, 47-52, 48-52, 49-52, 50-52, and 52.
mRNA=messenger RNA.
VILTEPSO provided significant improvements in dystrophin expression
In Study 1 the efficacy of 20-24 weeks of VILTEPSO was evaluated in ambulant males aged 4 to <10 years
Study 1: Mean increase in dystrophin expression to nearly 6% of normal with VILTEPSO (80 mg/kg/wk) vs 0.6% at baseline
*P-value for change from baseline at week 25 was statistically significant.
Study design: Phase 2 study with an initial 4-week randomized placebo-controlled period for safety followed by a 20-week open-label treatment period with 2 dose cohorts (N=16) that included patients aged 4 to <10 years with DMD amenable to exon 53 skipping.
DYSTROPHIN WHERE IT MATTERS: Immunofluorescence staining showed VILTEPSO-induced increases in dystrophin levels were correctly localized to the muscle cell membrane.
100% of patients showed an increase in dystrophin levels with VILTEPSO
Study 1: Dystrophin levels at 25 weeks vs baseline
Exon 53 skipping was observed, on average, in 43.9% of dystrophin mRNA molecules in patients treated with VILTEPSO (80 mg/kg/wk), as measured by RT-PCR.
Safety profile evaluated in two 24-week clinical studies
Adverse reactions reported in ≥10% of DMD patients treated with VILTEPSO 80 mg/kg once weekly (pooled Studies 1 and 2)
*Upper respiratory tract infection includes the following terms: upper respiratory tract infection, nasopharyngitis, and rhinorrhea.
†Injection site reaction includes the following terms: injection site bruising, injection site erythema, injection site reaction, and injection site swelling.
VILTEPSO offers a choice of treatment location—at home or at a treatment center
VILTEPSO is given as an 80-mg/kg weekly intravenous infusion
The appropriate dose of VILTEPSO is calculated based upon patient weight, at a recommended weekly dose of 80 mg/kg
VILTEPSO is infused for 60 minutes by a healthcare professional, at home or at a treatment center
