December 20, 2022
NS Pharma Announces Receipt of Orphan Drug Designation from the U.S. FDA for NS-018, an Investigational Treatment for Myelofibrosis
Paramus, NJ: December 20, 2022 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai), today announced that the U.S. FDA has granted Orphan Drug Designation to NS-018 (ilginatinib), an investigational treatment for myelofibrosis (MF), a rare and incurable blood cancer.
The FDA issues Orphan Drug Designations to support the development and evaluation of new treatments to prevent, diagnose, or treat a rare disease or condition.
MF is caused by buildup of excessive scar tissue in the bone marrow, which impairs the body’s ability to produce blood cells.1 In addition to impaired blood cell production, MF often leads to enlargement of the spleen (splenomegaly) which can lead to feelings of abdominal pain and pressure.1 Other common symptoms include fatigue, bone pain, fever, and weight loss.1 MF can be diagnosed at any age but is most common in men and women 65 years or older.1 The median survival of patients with MF is approximately six years.1
Several gene mutations are associated with MF, and the most common mutation is to the Janus kinase 2 (JAK2) gene.2 NS-018 is a highly selective and potent inhibitor of JAK2 developed by scientists from Nippon Shinyaku.
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.
Contact
U.S. Media Contact: media@nspharma.com
References:
https://www.mpnresearchfoundation.org/primary-myelofibrosis-pmf/
NEWS RELEASE
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 http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
Contact
U.S. Media Contact:
NEWS RELEASE
Oct 4, 2022
VILTEPSO® (viltolarsen) injection Four-Year Clinical Trial Data to be Presented at the World Muscle Society 2022 Conference
PARAMUS, NJ: Oct 4, 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 four-year efficacy and safety data from the open-label extension of a Phase 2 study of VILTEPSO® (viltolarsen), was accepted as a late-breaking e-poster and will be presented at the World Muscle Society 2022 Conference being held in Halifax, Canada from October 11-15.
“We are deeply thankful to the patients and their caregivers from the Phase 2 trial of VILTEPSO, all of whom chose to take part in the long-term extension study,” said Leslie Magnus, MD, Vice President, Medical Affairs. “These data represent the longest clinical experience with an exon 53 skipping therapy and we look forward to sharing the results with the Duchenne community.”
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.
Contact
U.S. Media Contact:
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.
Contact
U.S. Media Contact:
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.
Contact
U.S. Media Contact:
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 http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
Contact
U.S. Media Contact:
NEWS RELEASE
July 1, 2021
VILTEPSO® (viltolarsen) injection: Long-Term Efficacy and Safety Data Presented at the PPMD 2021 Virtual Annual Conference
Efficacy and safety results were based on interim analyses at week 109 from the open-label extension trial of a VILTEPSO Phase 2 study.
The primary endpoint of Time to Stand showed a statistically significant benefit for multiple time points over two years of treatment with VILTEPSO in comparison to DMD matched historical control group.
Additional secondary endpoints of motor function including Time to Run/Walk and 6- Minute Walk Test demonstrated consistent and statistically significant benefits in comparison to DMD historical controls.
There were no treatment related serious adverse events and no patients discontinued treatment.
PARAMUS, NJ: July 1, 2021 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai), announced that new long-term efficacy and safety data based on interim analyses at 109 weeks from the open-label extension trial of a Phase 2 study of VILTEPSO® (viltolarsen) injection were presented at the PPMD 2021 Annual Conference.
“These analyses showed that, after more than two years of treatment with VILTEPSO, patients maintained their motor function based on clinically relevant measurements while the DMD historic controls showed functional decline,” said Leslie Magnus, MD, Vice President, Medical Affairs.
This current open-label trial (N=16) is the extension of a previous 24-week trial in North America. All 16 patients in the short-term study elected to enroll in this long-term trial. Participants were assessed at weeks 37, 49, 73 and 109 and will continue to be assessed until study completion. These interim analyses of the timed function tests were conducted for all participants who had received at least 109 weeks of total treatment vs. the matched DMD historical control group (Cooperative International Neuromuscular Research Group
– Duchenne Natural History Study (CINRG-DNHS)).
The efficacy results were for Time to Stand from supine (mean change from baseline (seconds) at weeks 73 and 109 for viltolarsen was 0.21 and 0.43 vs CINRG-DNHS: 3.6 and 4.3, p<0.01), Time to Run/Walk 10 meters (mean change from baseline (seconds) at weeks 49, 73, and 109 for viltolarsen was -0.8, -0.9, and -0.4 vs CINRG-DNHS: 0.5, 1.3, 1.3, p<0.05), and 6-Minute Walk Test (mean change from baseline (meters) at week 109 for viltolarsen was 0.9 vs CINRG-DNHS: -65.6, p<0.05).
The most frequently reported adverse events were mild to moderate in this time-period and included cough, nasopharyngitis, rash, pyrexia, and vomiting. This safety profile was similar to that seen in the previous short-term study. To date, there were no treatment-related serious adverse events and no treatment discontinuations. These data provide important information on the efficacy and safety of the long-term use of viltolarsen for the treatment of DMD patients who are amenable to exon 53 skipping.
“Duchenne is a progressive disease of functional deterioration,” said study investigator Paula Clemens, MD, University of Pittsburgh Medical Center. “More research is needed, but a disease-modifying therapy that could stabilize and delay the loss of muscle function is needed for families with Duchenne and the healthcare professionals who specialize in its treatment.”
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.
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.
Contact
U.S. Media Contact:
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.
Contact
U.S. Media Contact:
NEWS RELEASE
May 12, 2021
VILTEPSO® (viltolarsen) injection Long-Term Clinical Trial Data to be Presented at the PPMD 2021 Virtual Annual Conference
PARAMUS, NJ: May 12, 2021 – 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 new, long-term efficacy and safety data (interim analysis at 109 weeks) from the open-label extension of a Phase 2 study of VILTEPSO® (viltolarsen) injection will be presented at the PPMD 2021 Annual Conference being held virtually from June 23-26.
“This VILTEPSO data we are presenting at the PPMD Annual Conference represents some of the longest treatment times with exon-skipping therapy reported to date,” said Leslie Magnus, MD, Vice President, Medical Affairs. “This data, compared to a matched historical control group, provides information on the potential long-term effectiveness and safety of VILTEPSO that is critical for healthcare professionals and Duchenne patients and their caregivers.”
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 trial is to confirm the clinical findings that were submitted under the Accelerated Approval pathway.
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 http://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 http://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
Contact
U.S. Media Contact: