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 is for DMD patients with a confirmed mutation that is amenable to exon 53 skipping
Exon skipping is a genetic technique that, in essence, “skips over” an exon next to the deleted exon(s) in patients with DMD. 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 treatment with an exon 53-skipping therapy such as VILTEPSO.
Common deletions that can be treated with VILTEPSO include 45-52, 47-52, 48-52, 49-52, 50-52, and 52 of the dystrophin gene. In the United States, about 2% of patients with DMD have an exon 52 deletion which is amenable to an exon 53-skipping therapy such as VILTEPSO.
Deletions amenable to exon 53 skipping
See how exon skipping worksVILTEPSO 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.
- Efficacy was assessed based on the change from baseline in dystrophin protein level, measured as percentage of the dystrophin level in healthy subjects at Week 25
- In patients who received VILTEPSO 80 mg/kg once weekly, mean dystrophin levels increased from 0.6% (standard deviation [SD] 0.8) of normal at baseline to 5.9% (SD 4.5) of normal by Week 25, with a mean change in dystrophin of 5.3% (SD 4.5) of normal levels (P=0.01) as assessed by validated Western blot (normalized to myosin heavy chain)
- > Median change from baseline was 3.8%
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
