NEWS RELEASE
October 24, 2022
NS Pharma Reports on Results of VILTEPSO® (viltolarsen) injection After Four Years of Treatment in Open-Label Extension Trial in Duchenne Muscular Dystrophy
Results were from a final analysis (up to Week 216) of the open-label extension trial of a VILTEPSO Phase 2 study.
In the study, the primary endpoint of Time to Stand and secondary endpoints, including Time to Run/Walk and Time to Climb 4 Stairs, were evaluated in comparison to a group-matched DMD historical control.
PARAMUS, NJ: October 24, 2022 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai), announced today that long-term efficacy and safety data (final analysis up to Week 216) from the open-label extension of a Phase 2 study of VILTEPSO® (viltolarsen) injection were presented at the 27th International Hybrid Annual Congress of the World Muscle Society (WMS 2022) in Halifax, Nova Scotia, Canada.
“These data represent the longest clinical experience of an exon 53 skipping therapy for the treatment of Duchenne muscular dystrophy. In this four-year study, Viltepso- treated patients maintained motor function over the first two years of treatment and experienced significant delay of disease progression over the following two years, compared with the CINRG DNHS control group which declined over this same time period,” said Leslie Magnus, MD, Vice President, Medical Affairs. “These data are encouraging as NS Pharma continues to evaluate the effect of Viltepso on improving or stabilizing motor function in a Phase 3 study.”
Data presented at WMS 2022 are from an open-label trial (N=16) that is the extension of a previous 24-week Phase 2 trial in North America. All 16 patients aged 4 to <10 years with DMD amenable to exon 53 skipping in the 24-week study elected to enroll in this long-term trial to continue evaluation of motor function and safety. Assessments of timed function tests (Time to Stand, Time to Run/Walk, Time to Climb 4 Stairs) were compared to a group-matched DMD historical control drawn from the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG DNHS). Both groups received a stable dose of glucocorticoid treatment. Efficacy results at Week 205 significantly favored viltolarsen compared to the CINRG DNHS control group for the primary endpoint of mean change from baseline for Time to Stand (viltolarsen 2.7 seconds vs. 8.3 seconds for CINRG DNHS, p=0.0040), and secondary endpoints including Time to Run/Walk 10 meters (viltolarsen 2.0 seconds vs. 6.0 seconds for CINRG DNHS, p=0.0002), and Time to Climb 4 Stairs (viltolarsen -0.01 m/s vs. -0.13 m/s for CINRG DNHS, p=0.0088).
The most frequently reported adverse events were mild to moderate in this 216-week open-label extension period and included cough, nasopharyngitis, rash, pyrexia, and vomiting. This safety profile was similar to that seen in the previous short-term study. There were no treatment-related serious adverse events and no treatment discontinuations.
“This long-term study gives physicians who treat patients with DMD important information about the long-term impact of VILTEPSO on ameliorating the disease course,” said Paula Clemens, MD, from the University of Pittsburgh School of Medicine. “Further study is ongoing, but these four-year data give confidence that VILTEPSO can be considered an important part of the treatment strategy for patients with DMD whose dystrophin mutations are amenable to exon 53 skipping therapy.”
In addition to this Phase 2 open-label extension study, NS Pharma continues to investigate the efficacy and safety of VILTEPSO in the confirmatory Phase 3 RACER53 trial. This study was initiated in October 2019 and is currently enrolling patients. The purpose of this Phase 3 randomized, double-blind, placebo-controlled trial is to evaluate the efficacy of viltolarsen on functional motor endpoints compared to placebo in patients with DMD amenable to exon 53 skipping.
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 DMD who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted with 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 DMD patients.
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 NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit https://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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NEWS RELEASE
September 17, 2021
VILTEPSO® (viltolarsen) injection Interim Long-Term Clinical Trial Data Scheduled for Presentation at the World Muscle Society 2021 Virtual Conference
PARAMUS, NJ: September 17, 2021 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai), announced today an electronic poster presentation on long-term efficacy and safety data (interim analysis at 109 weeks) from the open-label extension (up to 192 weeks) of a Phase 2 study of VILTEPSO® (viltolarsen) injection at the World Muscle Society 2021 Virtual Conference being held September 20–24.
The electronic poster presentation will be given by Paula Clemens, MD from the University of Pittsburgh School of Medicine on Thursday, September 23rd from 16:30 to 18:30 BST. For more information, please visit the World Muscle Society Virtual Conference website to view the full program: https://www.wms2021.com/page/programme.
About VILTEPSO® (viltolarsen) injection
Prior to its approval in the U.S., 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 DMD who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted with 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
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.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit https://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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NEWS RELEASE
April 1, 2021
NS Pharma’s VILTEPSO® (viltolarsen) Receives Permanent J-code from Centers for Medicare and Medicaid Services
PARAMUS, NJ: April 1, 2021 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Shigenobu Maekawa), announced that the J-code assigned by the Centers for Medicare and Medicaid Services (CMS) for VILTEPSO® (viltolarsen) injection is effective today.
As of April 1, 2021, healthcare providers may use the Level II HCPCS code: J1427 (Injection, Viltolarsen, 10 mg) when submitting claims for VILTEPSO. J-codes are unique identifiers used by commercial insurance plans, Medicare, Medicaid and other government insurance programs. The assignment of a permanent J-code for VILTEPSO will help to streamline billing and reimbursement for home infusion providers, hospitals, outpatient treatment centers and physicians’ offices.
“The designation of a permanent J-code for VILTEPSO is good news for families because simplifying billing and payment enables improved patient access,” said Tsugio Tanaka, President, NS Pharma, Inc. “We encourage families to contact our NS Support team who can help guide you through the access process and advocate on your behalf.”
VILTEPSO is commercially available through a network of specialty distributors and specialty pharmacy providers. The distribution partners in this limited network were selected for their experience providing responsive, reliable service to healthcare providers and families.
About NS Support
NS Support provides comprehensive, personalized access services and care coordination for providers and patients. By enrolling in NS Support, patients and providers receive customized resources and ongoing, highly-responsive assistance every step of the way.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit https://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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NEWS RELEASE
August 12, 2020
NS Pharma’s VILTEPSO™ (viltolarsen) injection Now FDA-Approved in the U.S. for the Treatment of Duchenne Muscular Dystrophy in Patients Amenable to Exon 53 Skipping Therapy
Patients taking VILTEPSO showed an increase in dystrophin expression to an average of 5.9% of normal after 20-24 weeks of treatment.
Overall, in a pivotal study of VILTEPSO 100% of patients showed an increase in dystrophin levels after treatment and 88% of patients showed dystrophin levels of 3% of normal or greater.
PARAMUS, NJ: August 12, 2020 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President Shigenobu Maekawa), announced today that the U.S. Food & Drug Administration (FDA) has approved VILTEPSO™ (viltolarsen) injection for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping therapy. VILTEPSO received an Accelerated Approval by the FDA based on an increase in dystrophin, a key protein for supporting muscle health. Since a lack of dystrophin is the underlying cause of DMD, increasing dystrophin as much and as early as possible is a key goal in the treatment of DMD. VILTEPSO is the first and only exon 53 skipping therapy to demonstrate an increase in dystrophin in children as young as four years old. The continued approval of VILTEPSO may be contingent on confirmation of a clinical benefit in a Phase 3 confirmatory trial.
DMD is caused by genetic mutations that prevent dystrophin production. Patients with DMD experience progressive and irreversible muscle loss with symptoms appearing as early as two years of age. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications.
The VILTEPSO New Drug Application (NDA) submission included results from a Phase 2, two-period study in patients aged four to less than 10 years of age conducted in North America (Study 1, N=16) and a multicenter, open-label study in boys five to less than 18 years of age conducted in Japan (Study 2, N=16).
In Study 1, of those patients who received the recommended dose of 80 mg/kg/wk (N=8), 100% of patients (8/8) showed an increase in dystrophin levels after treatment with VILTEPSO and 88% of patients (7/8) showed dystrophin levels of 3% or greater than normal. Overall, after 20-24 weeks of treatment a mean increase in dystrophin expression to nearly 6% of normal was observed with VILTEPSO (80 mg/kg/wk) versus 0.6% at baseline.
The most common side effects of VILTEPSO included upper respiratory tract infection, injection site reaction, cough and fever.
“For decades, neurologists who treat DMD have hoped for the discovery of therapies capable of significantly improving dystrophin production, and the magnitude of dystrophin increases observed with VILTEPSO are impressive,” said study investigator Vamshi Rao, MD, Ann & Robert H. Lurie Children’s Hospital of Chicago. “The approval of VILTEPSO is an exciting development for DMD patients amenable to exon 53 skipping therapy and may rapidly become a foundational treatment for these patients.”
Patients receiving treatment with VILTEPSO have the option and flexibility to receive infusions at their home or at a hospital or treatment center. VILTEPSO is administered by a trained healthcare professional as an 80 mg per kg of body weight 60-minute weekly intravenous infusion.
NS Pharma will provide families, physicians and healthcare professionals dedicated and individualized resources every step of the way through the NS Support program. NS Pharma will be hosting a series of webinars on the comprehensive care coordination available through NS Support. Follow us on LinkedIn and Twitter for information and registration for upcoming webinars.
“On behalf of NS Pharma and Nippon Shinyaku, I would like to express our deepest gratitude to the families and physicians who participated in our clinical trials and made today’s approval possible,” said Tsugio Tanaka, President, NS Pharma, Inc. “We are proud to now offer an important new treatment option to help address the significant unmet needs caused by this devastating disease.”
NS Pharma continues to study the safety and efficacy of VILTEPSO in the confirmatory Phase 3 RACER53 trial. This study was initiated in October 2019 and is currently enrolling. The purpose of this Phase 3 trial is to confirm the clinical findings that were submitted under the Accelerated Approval pathway.
About Duchenne Muscular Dystrophy (DMD)
DMD is a progressive form of muscular dystrophy that occurs primarily in males. DMD causes progressive weakness and loss of skeletal, cardiac, and pulmonary muscles. Early signs of DMD may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with DMD may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications.
About VILTEPSO™ (viltolarsen) injection
Prior to its approval in the U.S., 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 DMD who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted with 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
For additional safety information, please see the full Prescribing Information.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit https://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
Contact
U.S. Media Contact: