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VILTEPSO significantly increased dystrophin production

88% (7 out of 8 people) showed increases of ~3% or higher, measured at week 25 of treatment.

These significant increases in dystrophin production with VILTEPSO were identified by a method called western blot and verified by a highly sensitive measuring technique known as mass spectrometry.

VILTEPSO increased dystrophin levels to
nearly 6% of normal

VILTEPSO was studied in 16 ambulatory (walking) boys ages 4 to less than 10 years who were receiving a stable dose of corticosteroids for at least 3 months.

In this graph, their average dystrophin levels at week 25 of VILTEPSO treatment are compared with their average dystrophin levels before treatment.

In the same clinical study, additional results
included evidence of dystrophin production and
timed motor function tests

Dystrophin Production Evidence

*Exon 53 skipping efficiency assessed by RT-PCR. Mean dystrophin levels assessed by mass spectrometry. Dystrophin localization assessed by immunofluorescence staining. RT-PCR=reverse transcriptase-polymerase chain reaction.

Motor Function Tests

Functional tests were compared to Duchenne natural history (DNHS) data as the control group rather than to placebo. Definitive conclusions should not be drawn. Functional data are not in the US Prescribing Information.

^†Control subjects were matched for age and corticosteroids. Negative time means less time; positive time means more time. Negative distance means less distance traveled; positive distance means greater distance traveled. Negative NSAA means lower score compared to baseline; positive NSAA means higher score compared to baseline.

Safety profile evaluated in two 24-week clinical studies

Adverse reactions reported in ≥10% of people with DMD treated with VILTEPSO 80 mg/kg once weekly

^*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.

No patients in the clinical trial discontinued treatment as a result of treatment-related Serious Adverse Events (SAEs)

MUSCLE FUNCTION DATA

An extended view of patients on VILTEPSO
over 4 years

Functional tests were compared to Duchenne natural history data as the control group rather than to placebo.
Definitive conclusions should not be drawn. Functional data are not in the US Prescribing Information.

Time to stand over 4 years*

With VILTEPSO, the mean change from baseline at week 205 was 2.7 seconds and in the CINRG group the mean change from baseline at week 205 was 8.3 seconds.

Time to stand measures the amount of time it takes for a DMD patient to go from lying on their back to standing.

Seconds^†

Time to run/walk 10 meters over 4 years*

With VILTEPSO, the mean change from baseline at week 205 was 2.0 seconds and in the CINRG group the mean change from baseline at week 205 was 6.0 seconds.

Time to run/walk 10 meters measures the amount of time it takes for a DMD patient to run or walk 10 meters.

Seconds^†

Time to climb four stairs over 4 years*

With VILTEPSO, the mean change from baseline at week 205 was 3.1 seconds and in the CINRG group the mean change from baseline at week 205 was 6.1 seconds.

Time to climb four stairs measures the amount of time it takes for a DMD patient to climb four stairs.

Seconds^†

^*The control subjects for this trial were matched for age, ambulatory status, corticosteroid use, and geographic location from the CINRG DNHS registry.
^†Negative time means less time; positive time means more time.
CINRG=Cooperative International Neuromuscular Research Group; DNHS=Duchenne Natural History Study.

Safety assessment for open-label,
four-year extension study data

AE=adverse event; TEAE=treatment-emergent AE; wk=week.

No patients discontinued the study as a result of
treatment-related Serious Adverse Events (SAEs)

EXPAND

Indication

VILTEPSO is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VILTEPSO. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Important Safety Information

In clinical studies, no patients experienced kidney toxicity during treatment with VILTEPSO. However, kidney toxicity from drugs like VILTEPSO may be possible. Your doctor may monitor the health of your kidneys before starting and during treatment with VILTEPSO.
Common side effects include upper respiratory tract infection, injection site reaction, cough, and fever.
You are encouraged to report adverse events related to VILTEPSO. To do so, or for general inquiries, please call NS Pharma Medical Information at 1-866-NSPHARM (1-866-677-4276).

For more information about VILTEPSO, see full Prescribing Information.

Efficacy & Safety

In a clinical study, 100% of patients taking VILTEPSO showed an increase in dystrophin levels

VILTEPSO significantly increased dystrophin production

VILTEPSO increased dystrophin levels to
nearly 6% of normal