June 10, 2022
NS Pharma Receives Innovator Award at the BioNJ 29th Annual Dinner Meeting & Innovation Celebration in Recognition of the FDA’s Accelerated Approval of VILTEPSO® (viltolarsen) for the Treatment of Duchenne Muscular Dystrophy in the U.S.
PARAMUS, NJ: June 10, 2022 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai), was honored with a BioNJ Innovator Award at the BioNJ 29th Annual Dinner Meeting & Innovation Celebration taking place on Thursday, June 9, 2022. The Innovator Award recognizes NS Pharma for their commitment to patients and the launch of VILTEPSO® (viltolarsen) for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping therapy. VILTEPSO® was granted accelerated approval by the U.S. Food and Drug Administration (FDA) on August 12, 2020.
BioNJ is the life sciences trade association for New Jersey, representing nearly 400 research- based life sciences organizations and stakeholders across the healthcare ecosystem. The BioNJ Dinner Meeting & Innovation Celebration brought together hundreds of biotechnology and pharmaceutical professionals, academic leaders, patients, advocates, service providers and government officials to honor the groundbreaking medical innovation driven by companies with a footprint in New Jersey.
“On behalf of NS Pharma, it’s a tremendous honor to receive the BioNJ Innovator Award recognizing our efforts championing for patients with Duchenne muscular dystrophy,” said Tsugio Tanaka, President, NS Pharma, Inc. “We take pride in developing innovative, life- changing medicines for people affected by rare diseases, and this recognition reinforces our commitment to helping patients live healthier, happier lives.”
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 NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
About VILTEPSO® (viltolarsen) injection
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.
For more information about VILTEPSO, see full Prescribing Information.
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NEWS RELEASE
May 31, 2022
VILTEPSO® (viltolarsen) injection: Long-Term Efficacy and Safety Data Published in the Journal of Neuromuscular Diseases
Efficacy and safety results were based on analyses at week 109 from the open-label extension trial of a VILTEPSO Phase 2 study.
These data from the open-label extension study of VILTEPSO were previously presented at medical congresses and scientific meetings.
PARAMUS, NJ: May 31, 2022 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai), announced the publication of long-term efficacy and safety data based on analyses at 109 weeks from the 192-week open-label extension trial of a Phase 2 study of VILTEPSO® (viltolarsen) injection in the Journal of Neuromuscular Diseases. The article, “Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy,” is freely available under open access (click here).
“In this VILTEPSO open-label, long-term extension study, evaluation of functional clinical endpoints demonstrated maintenance of motor function versus functional decline in a historical control group over two years.” said Leslie Magnus, MD, Vice President, Medical Affairs. “These encouraging interim results with VILTEPSO support the continued need to research its clinical profile and its potential impact on maintaining mobility.”
Data published in the Journal of Neuromuscular Diseases 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, 6-Minute Walk Test) were compared to a matched DMD historical control group (Cooperative International Neuromuscular Research Group Duchenne Natural History Study).
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 DMD patients amenable to exon 53 skipping. Continued approval of VILTEPSO is dependent on verification of clinical benefit.
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 http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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NEWS RELEASE
June 21, 2021
Webcast of VILTEPSO® (viltolarsen) injection: Long-Term Data Scheduled for Presentation at the PPMD 2021 Virtual Annual Conference
PARAMUS, NJ: June 21, 2021 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Shigenobu Maekawa), announces a webcast during the PPMD 2021 Virtual Annual Conference held from June 23 to 26. The presentation will feature new, long-term efficacy and safety data (interim analyses at 109 weeks) from the open-label extension trial of a Phase 2 study of VILTEPSO® (viltolarsen) injection.
The data presentation will be given by Paula Clemens, MD from the University of Pittsburgh School of Medicine and will be webcast live at 8 PM EST on June 23. The webcast may be accessed at https://ppmd2021.sched.com/venue/Live+Stream. For more information including an archived version of the webcast, please visit the PPMD Virtual Annual Conference website (https://www.parentprojectmd.org/get- involved/attend-events/annual-conference-2021-virtual/agenda/).
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
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 http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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NEWS RELEASE
July 7, 2020
NS Pharma Announces Launch of Patient Support Hub
PARAMUS, NJ: July 7, 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 the launch of NS Support for patients with Duchenne muscular dystrophy (DMD) and their healthcare providers.
NS Support can be reached by telephone at 833-677-8778, Monday through Friday from 8 am to 8 pm ET. Caregivers and healthcare providers can call to learn more information about NS Support and request notifications about product availability and program enrollment.
“Based on our conversations with the DMD community, there is a high demand for assistance with navigating the complicated insurance landscape for DMD patients amenable to exon 53 skipping therapy,” said Tsugio Tanaka, President, NS Pharma, Inc. “We want to ensure the smoothest patient experience possible and a rapid journey towards access and treatment. That is why we are launching NS Support now, so we can begin the process of engaging with families who want to learn more about what NS Support can offer.”
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 compl tions.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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NEWS RELEASE
May 26, 2020
NS Pharma Announces Publication of Clinical Trial Data for Viltolarsen in DMD Patients in JAMA Neurology
Viltolarsen is an exon skipping therapy being reviewed under the FDA Accelerated Approval pathway for the treatment of Duchenne muscular dystrophy in patients amenable to exon 53 skipping therapy
PARAMUS, NJ: May 26, 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 JAMA Neurology has published results from a clinical trial of viltolarsen, an investigational agent being evaluated in Duchenne muscular dystrophy (DMD) patients who are amenable to exon 53 skipping therapy.
“In this study, 100% of patients were shown to have more dystrophin after treatment with viltolarsen and 88% achieved dystrophin levels of greater than 3%,” said lead study author and investigator Paula Clemens, MD, University of Pittsburgh School of Medicine. “These increases were seen in patients as young as four years of age and after six months or less of treatment, which underscores the impressive results seen in this study.”
This Phase 2, two-period, dose-finding study enrolled 16 DMD patients, from four to less than 10 years of age, who were amenable to exon 53 skipping therapy. Study participants were randomized to two doses of viltolarsen (40 mg/kg/wk and 80 mg/kg/wk) for 20 to
24 weeks. At the end of the study, treatment with viltolarsen was associated with statistically significant increases in mean dystrophin expression (40 mg/kg/wk: p<0.001; 80 mg/kg/wk p=0.012). Mean dystrophin levels of 5.7% and 5.9% were observed in comparison to mean baseline levels of 0.3% and 0.6% in the 40 mg/kg/wk and 80 mg/kg/wk groups, respectively. Fourteen out of 16 patients (88%) reached dystrophin levels greater than 3%.
The most common treatment emergent adverse events occurring in greater than one patient were: cold, cough, nasal congestion, bruising, joint pain, diarrhea and vomiting. No serious adverse events were observed in the study.
“Lack of functional dystrophin is recognized as the singular underlying cause of the devastating impact of DMD,” said study author and investigator Vamshi Rao, MD, Ann & Robert H. Lurie Children’s Hospital of Chicago. “As a pediatric neurologist who specializes in the treatment of DMD, I am encouraged by the dystrophin increases observed in this study and the potential of viltolarsen to address the underlying cause of DMD.”
“This is one of the first studies of exon skipping therapies in DMD to generate rigorous data on the primary biomarker of dystrophin protein,” said study author Eric Hoffman, PhD, Associate Dean for Research and Professor of Pharmaceutical Sciences at Binghamton University. “Having assisted in the development of viltolarsen for many years, and decades more researching the genetics and genomics of DMD, I am pleased by the results of this study and the implications for families facing DMD.”
Viltolarsen has not yet been approved in the U.S. and its New Drug Application was recently granted Priority Review by the FDA with an anticipated action date in the third quarter of 2020. In March 2020, viltolarsen was approved in Japan for the treatment of DMD patients amenable to exon 53 skipping therapy.
“We are deeply committed to the development of viltolarsen and offering healthier futures to DMD patients and families,” said Tsugio Tanaka, President, NS Pharma, Inc. “Based on these results we are optimistic that, if it is approved, viltolarsen will become an important new treatment option for DMD patients and healthcare providers.”
NS Pharma continues to study the safety and efficacy of viltolarsen 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 Viltolarsen
Viltolarsen has been granted Rare Pediatric Disease, Orphan Drug and Fast Track Designations in the U.S. The viltolarsen New Drug Application was granted Priority Review by the FDA with an anticipated action date in the third quarter of 2020. In March 2020, viltolarsen 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, viltolarsen was granted with the SAKIGAKE designation, Orphan drug designation, and designation of Conditional Early Approval System.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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