PARAMUS,
N.J., May 27, 2024 /PRNewswire/ -- NS Pharma,
Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd.,
announced today that it has received preliminary analysis results
from the global Phase 3 clinical trial (RACER53 study, NCT04060199)
of NS-065/NCNP-01 (generic name: viltolarsen).
"We have confidence in viltolarsen and
continue our commitment to the Duchenne community," said the NS
Pharma President.
Viltolarsen was approved by the United
States (US) Food and Drug Administration (FDA) in 2020 under
the brand name VILTEPSO® – for the treatment of Duchenne
muscular dystrophy (Duchenne) in patients who have a confirmed
mutation of the dystrophin gene that is amenable to exon 53
skipping – under the FDA accelerated approval pathway based on an
increase in dystrophin production in skeletal muscle observed in
treated patients. In the US, continued approval for this indication
may be contingent upon verification and description of clinical
benefit in a confirmatory trial.
The RACER53 Study is a randomized, double-blind,
placebo-controlled, comparative study of 77 ambulatory boys with
Duchenne. The study evaluated the efficacy and safety of an 80
mg/kg once weekly dosing of the treatment – versus placebo – for 48
weeks and was intended to serve as a confirmatory study.
The primary endpoint of the study was Time to Stand from Supine
evaluated as velocity (rise/sec). The viltolarsen group showed a
trend of increased velocity from baseline after treatment for 48
weeks. However, the placebo group also showed a trend of increased
velocity, and there was no statistically significant difference
between the viltolarsen group and the placebo group.
Preliminary safety results indicated that all adverse events
that occurred under viltolarsen treatment were mild or moderate.
There were no treatment emergent adverse events that led to
discontinuation of the drug during the study.
"We are currently conducting further detailed data analyses and
identifying factors that may have influenced the results (e.g. age,
treatment period, and effect of concomitant drugs including
glucocorticoid therapy)," said NS Pharma President Tsugio Tanaka, MSc. "Considering the results of
prior clinical studies, we have confidence that viltolarsen can be
a beneficial treatment for amenable patients with Duchenne."
Specifically, in addition to the increase in dystrophin
production in skeletal muscle that formed the basis of the FDA
approval, a previously reported Phase 2, open-label, long-term
extension study evaluated viltolarsen in 16 subjects between the
ages of four and 10 with Duchenne amenable to exon 53 skipping. The
study found that subjects receiving viltolarsen showed
statistically significant improvements in the study's primary
endpoint of mean change from baseline for Time to Stand at week 205
as compared to a historical control group that was matched for key
factors. In this study, treatment emergent adverse events were
primarily mild or moderate. No study participants discontinued the
study drug due to adverse events.
NS Pharma is currently conducting further detailed data
analyses, including post-hoc data analyses, and plans to work
closely with regulatory authorities to determine how to proceed
based on the results of this analysis and in the best interests of
patients. The company will report on additional analyses and
discussions with the regulatory authorities at a later date.
About VILTEPSO® (Viltolarsen)
Injection
Prior to its approval in the U.S. in August 2020, VILTEPSO was granted Priority Review
as well as Rare Pediatric Disease, Orphan Drug and Fast Track
Designations. In March 2020, VILTEPSO
was approved in Japan for the
treatment of patients with Duchenne who are amenable to exon 53
skipping therapy. Prior to its approval in Japan, VILTEPSO was granted the SAKIGAKE
designation, orphan drug designation, and designation of
Conditional Early Approval System.
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
Warnings and
Precautions: Kidney toxicity was observed in animals who received
viltolarsen. Although kidney toxicity was not observed in the
clinical studies with VILTEPSO, the clinical experience with
VILTEPSO is limited, and kidney toxicity, including potentially
fatal glomerulonephritis, has been observed after administration of
some antisense oligonucleotides. Kidney function should be
monitored in patients taking VILTEPSO.
Serum creatinine may not be a reliable measure of kidney
function in patients with Duchenne. Serum cystatin C, urine
dipstick, and urine protein-to-creatinine ratio should be measured
before starting VILTEPSO. Consider also measuring glomerular
filtration rate before starting VILTEPSO. During treatment, monitor
urine dipstick every month, and serum cystatin C and urine
protein-to-creatinine ratio every three months.
Urine should be free of excreted VILTEPSO for monitoring of
urine protein. Obtain urine either prior to VILTEPSO infusion, or
at least 48 hours after the most recent infusion. Alternatively,
use a laboratory test that does not use the reagent pyrogallol red,
which has the potential to generate a false positive result due to
cross reaction with any VILTEPSO in the urine. If a persistent
increase in serum cystatin C or proteinuria is detected, refer to a
pediatric nephrologist for further evaluation.
Adverse Reactions: The most common adverse reactions include
upper respiratory tract infection, injection site reaction, cough,
and pyrexia.
To report an adverse event, 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.
About Duchenne Muscular Dystrophy (Duchenne)
Duchenne
is a progressive form of muscular dystrophy that occurs primarily
in males. It causes progressive weakness and loss of skeletal,
cardiac, and respiratory muscles. Early signs of Duchenne may
include delayed ability to sit, stand or walk. There is a
progressive loss of mobility, and by adolescence, patients with
Duchenne may require the use of a wheelchair. Cardiac and
respiratory muscle problems begin in the teenage years and lead to
serious, life-threatening complications. For more information about
Duchenne, please visit wespeakduchenne.com.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly
owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a
registered trademark of the Nippon Shinyaku Co., Ltd. For more
information, please visit nspharma.com.
U.S. Media Contact:
media@nspharma.com
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SOURCE NS Pharma, Inc.