PARAMUS, NJ: September 10, 2024 – NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced that the Food and Drug Administration (FDA) has granted rare pediatric disease designation to NS- 050/NCNP-03 which is being developed for the treatment of Duchenne muscular dystrophy (Duchenne). The FDA’s rare pediatric disease designation is granted for treatments of serious or life-threatening diseases that affect children under the age of 18 and fewer than 200,000 patients in the United States.
“The journey from first symptom to diagnosis and finally treatment can be long and challenging for patients with rare diseases and their caregivers,” said NS Pharma President Yukiteru Sugiyama, Ph.D. “We are grateful for this designation which can help us accelerate the development of this therapy for Duchenne.”
Duchenne is a progressive muscle wasting disease caused by a deficiency of the dystrophin protein. It leads to weakness of skeletal, cardiac and pulmonary muscles. There are many types of genetic mutations that can cause Duchenne, and NS- 050/NCNP-03 is being developed to treat patients with confirmed gene mutations amenable to exon 50 skipping therapy.
NS-050/NCNP-03 is an antisense oligonucleotide co-discovered by the National Center of Neurology and Psychiatry (NCNP) and Nippon Shinyaku. NS-050/NCNP- 03 skips part of the genetic information of the dystrophin gene and produces a functional dystrophin protein with a slightly shorter chain length, which is expected to have the effect of suppressing muscle function deterioration.
NS Pharma is working to develop products for patients with rare diseases. We are preparing a Phase 1 / 2 study to evaluate the safety and efficacy of NS-050/NCNP-03 in patients with Duchenne in Japan and the United States.
About Duchenne Muscular Dystrophy (Duchenne)
NEWS RELEASE
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 NEWS RELEASE Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com.
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July 10, 2024
NS Pharma Appoints New President to Oversee Next Phases of Orphan Drug Clinical Development and Commercialization
PARAMUS, NJ: July 10, 2024 – NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced that its Board of Directors has appointed a new President at its US headquarters. Yukiteru Sugiyama, Ph.D. replaces retiring president Tsugio Tanaka, MSc as of June 30, 2024.
Sugiyama received a doctorate degree in organic chemistry from Nagoya University in 1996. From 1996 until 2007, he worked in research and clinical development with Nippon Shinyaku in Japan, focusing on structural chemistry and blood cancer therapies. In 2007, he transitioned to the commercial division within the company.
In 2020, during the COVID-19 pandemic, Sugiyama transferred to NS Pharma in the US to work as assistant vice president overseeing commercial functions for VILTEPSO®(viltolarsen). In total, Sugiyama has worked for 28 years at Nippon Shinyaku with progressive levels of responsibility.
“Having served in both our science and patient focused fields, I have come to understand that having empathy for the patient is critical in the development of therapies to treat rare diseases,” Sugiyama explained. “As president, it is my mission to maintain heightened levels of empathy at NS Pharma, and to enhance transparency between all stakeholders.”
Sugiyama will lead the company through the next phases of clinical and commercial development for VILTEPSO and CAP-1002 (through a partnership with Capricor Therapeutics) – for the treatment of Duchenne muscular dystrophy (Duchenne). NS Pharma is also currently working on an exon 44 skipping therapy (Phase 2) and an exon 50 skipping therapy (Phase 1/2) for Duchenne, a selective JAK1 inhibition therapy for the treatment of eosinophilic granulomatosis with polyangiitis (EGPA), as well as several preclinical neurological therapies.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS NEWS RELEASE Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com.
US Media Contact:
media@nspharma.com
April 4, 2024
NS Pharma Announces Research Alliance with MiNA Therapeutics to Develop Therapies for Rare Diseases of the Central Nervous System
PARAMUS, NJ: April 4, 2024 – NS Pharma, Inc. (NS Pharma) announced that its parent company, Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) – headquartered in Kyoto, Japan, entered into a joint research agreement with MiNA Therapeutics – headquartered in London, United Kingdom, to develop nucleic acid drugs for the potential treatment of intractable and rare diseases of the central nervous system. The agreement was facilitated by the NS Pharma Innovation Research Partnering (IRP) team, located in Cambridge, MA.
“We are incredibly proud of our IRP division and the work it is doing with the great team at MiNA Therapeutics,” said NS Pharma President Tsugio Tanaka. “We are excited about the innovative possibilities that come from working with small activating RNA (RNAa) therapeutics.”
Through this agreement, MiNA Therapeutics will provide Nippon Shinyaku RNAa therapeutics, which are oligonucleotides that can increase the transcription of a target gene. Nippon Shinyaku will have the right to exercise an option to exclusively research and develop pharmaceutical candidates derived from the alliance with MiNA Therapeutics by paying lump sum payments and milestones associated with the progress of research and development. After exercising the option, the company will pay milestones and royalties commensurate with the progress of the development and the sales of the product(s) after launch.
“Through this research alliance with MiNA Therapeutics, we will continue our efforts to apply our nucleic acid drug technology in the central nervous system,” Tanaka stated. “NS Pharma is committed to extending our technology as far as it can go to help improve the lives of patients with rare diseases.”
Nippon Shinyaku has been developing compounds in several therapeutic fields, including hematologic malignancies and intractable/orphan diseases. The company’s U.S. subsidiary is NS Pharma, headquartered in Paramus, NJ with additional offices in Cambridge.
NEWS RELEASE
About MiNA Therapeutics
MiNA Therapeutics is the global leader in small activating RNA (RNAa) therapeutics. Harnessing innate mechanisms of gene activation, RNAa therapeutics are a revolutionary new class of medicines that can restore or boost normal function of genes and thereby protein-modulated pathways in cells. The company is advancing a proprietary pipeline of new medicines with an initial focus on genetic medicine, while collaborating with leading pharmaceutical companies to apply its technology platform across a broad range of other therapeutic areas.
Based on its unique know-how in RNA activation, MiNA Therapeutics is expanding the possibilities of RNA-based medicine. www.minatx.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:
NEWS RELEASE
October 17, 2023
NS-089/NCNP-02 Preclinical Data Published in Molecular Therapy Nucleic Acids
NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka) is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai)
PARAMUS, NJ: October 17, 2023 – NS Pharma, Inc. announced today the publication of preclinical data on NS-089/NCNP-02 (brogidirsen), an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy, in the journal Molecular Therapy Nucleic Acids. The article, “Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual targeting antisense oligonucleotide,” is available under open access here.
NS-089/NCNP-02 is an antisense nucleotide discovered through joint research between NS Pharma’s parent company, Nippon Shinyaku, and the National Center of Neurology and Psychiatry (Kodaira City; President, Kazuyuki Nakagome). The article, co-authored with the National Center of Neurology and Psychiatry, describes the process that led to the discovery of NS-089/NCNP-02 and its molecular properties, including nucleic acid sequences that target two separate sites within exon 44 of the dystrophin pre-mRNA sequence. The article also presents in vitro and in vivo preclinical efficacy data regarding dystrophin protein expression.
Clinical development of NS-089/NCNP-02 includes a planned Phase 2 study in the United States conducted by NS Pharma and a Phase 2 study conducted in Japan by Nippon Shinyaku. NS-089/NCNP-02 has previously received Rare Pediatric Disease, Breakthrough Therapy and Orphan Drug Designations from the U.S. Food and Drug
Administration.
About Duchenne Muscular Dystrophy (Duchenne)
Duchenne is a progressive form of muscular dystrophy that occurs primarily in males. Duchenne 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.
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
August 7, 2023
FDA Grants Orphan Drug Designation to NS-089/NCNP-02 for the
Treatment of Duchenne Muscular Dystrophy
NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka) is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai)
PARAMUS, NJ: August 7, 2023 – NS Pharma, Inc. announced today the U.S. Food & Drug Administration (FDA) has granted Orphan Drug Designation to NS-089/NCNP-02, an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.
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.
NS-089/NCNP-02 previously received Rare Pediatric Disease Designation from the FDA in June 2023 and Breakthrough Therapy Designation in July 2023.
NS-089/NCNP-02 is an antisense nucleotide discovered through joint research between NS Pharma’s parent company, Nippon Shinyaku, and the National Center of Neurology and Psychiatry (Kodaira City; President, Kazuyuki Nakagome).
Clinical development of NS-089/NCNP-02 includes a planned Phase 2 study in the United States conducted by NS Pharma and a Phase 2 study conducted in Japan by Nippon Shinyaku. Additional details will be provided once the trials are ready to begin enrolling participants.
About Duchenne Muscular Dystrophy (Duchenne)
Duchenne is a progressive form of muscular dystrophy that occurs primarily in males. Duchenne 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.
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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February 17, 2023
NS Pharma Announces First Patient Enrolled in
Phase 2 Study to Assess Efficacy and Safety of NS-018 Compared to Best Available Therapy (BAT) in Patients With Myelofibrosis
NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka) is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai)
Paramus, NJ: February 17, 2023 – NS Pharma, Inc. announced today the first patient enrollment in a Phase 2 clinical study of NS-018 (ilginatinib), an investigational treatment for myelofibrosis (MF), a rare and incurable blood cancer.
The Phase 2b study is an open-label, multicenter, randomized, controlled, 2-arm study to assess the efficacy and safety of orally administered NS-018 versus best available therapy in subjects with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis with severe thrombocytopenia (Platelet Count <50,000/μL).
ClinicalTrials.gov identifier: NCT04854096
(https://clinicaltrials.gov/ct2/show/NCT04854096)
About Myelofibrosis (MF)
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
About NS-018
NS-018 is a highly selective and potent inhibitor of JAK2 developed by scientists from Nippon
Shinyaku and was recently granted Orphan Drug Designation from the U.S. Food and Drug Administration (FDA).
NEWS RELEASE
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.
U.S. Media Contact:
References:
Accessed at: https://www.mpnresearchfoundation.org/primary-myelofibrosis-pmf/
Accessed at: https://www.mayoclinic.org/diseases-conditions/myelofibrosis/symptoms-
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
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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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.
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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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U.S. Media Contact: