PARAMUS, NJ: January 23, 2025 – NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced that Food and Drug Administration (FDA) has granted rare pediatric disease designation to NS-051/NCNP-04 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.
Duchenne is a progressive muscle wasting disease caused by a deficiency of the dystrophin protein. It leads to weakness of skeletal, cardiac, and respiratory muscles. There are many types of genetic mutations that can cause Duchenne, and NS-051/NCNP-04 is being developed to treat patients with confirmed gene mutations amenable to exon 51 skipping therapy.
NS-051/NCNP-04 is an antisense oligonucleotide co-discovered by the National Center of Neurology and Psychiatry (NCNP) and Nippon Shinyaku. NS-051/NCNP-04 promotes skipping of exon 51 of the dystrophin gene that produces a shortened dystrophin protein containing essential functional portions, which is expected to have the effect of stabilizing or improving muscle function.
NS Pharma is working to develop products for patients with rare diseases.
About Duchenne Muscular Dystrophy (Duchenne)
Duchenne is a form of muscular dystrophy that occurs primarily in males. It causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of Duchenne may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications. For more information about Duchenne, please visit wespeakduchenne.com.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com.
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media@nspharma.com
Selective JAK1 Inhibition is a 21st Century science. At NS Pharma, we are investigating this therapeutic approach for the treatment of rare diseases like eosinophilic granulomatosis with polyangiitis (EGPA).
With JAK1 inhibition, we consider two important variables: potency and target selectivity. These aspects are crucial when developing safe and effective therapies. Higher potency means a lower dose of the drug is needed for it to have its intended effect. High selectivity helps to ensure that the drug interacts only with its intended target, minimizing the risk of side effects.
The main challenge of working with JAK1 inhibitors to treat EPGA is that different therapies are needed to treat distinct stages of EGPA – no single therapy has been found to treat all stages of this condition.1 Scientists are still working to understand the causes of EGPA fully. This is complex, as they believe several factors are involved.
On the plus side, if JAK1 inhibition is effective, researchers hope it could reduce symptoms of EGPA regardless of disease stage. This would present a treatment opportunity for many patients with EGPA, rather than just a select few. Researchers are currently conducting a Phase 2 clinical trial to evaluate its safety and effectiveness in treating EGPA.
EGPA is extremely rare, affecting just about 0.0017% of the population.2 This makes accessing diagnosis and effective treatment challenging for patients. If JAK1 inhibition for EGPA is successful, this therapy could support healthier and happier futures for people with EGPA.
1 Eosinophilic Granulomatosis with Polyangiitis – Vasculitis Foundation. Accessed on July 19, 2024.https://www.vasculitisfoundation.org/education/vasculitis-types/eosinophilic-granulomatosis-with-polyangiitis/
1 Eosinophilic Granulomatosis with Polyangiitis (EGPA) – American Lung Association. Accessed on July 19, 2024. https://www.lung.org/lung-health-diseases/lung-disease-lookup/egpa
2 Vaglio A, Buzio C, Zwerina J. Eosinophilic granulomatosis with polyangiitis (Churg-Strauss): state of the art. Allergy. 2013 Mar;68(3):261-73. doi: 10.1111/all.12088
May 27, 2024
NS Pharma Shares Preliminary Results of Viltolarsen (NS-065 / NCNP-01)
Phase 3 Clinical Trial (RACER53 Study)
PARAMUS, NJ: May 27, 2024 – NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd., announced today that it has received preliminary analysis results from the global Phase 3 clinical trial (RACER53 study, NCT04060199) of NS-065/NCNP-01 (generic name: viltolarsen).
Viltolarsen was approved by the United States (US) Food and Drug Administration (FDA) in 2020 under the brand name VILTEPSO® – for the treatment of Duchenne muscular dystrophy (Duchenne) in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping – under the FDA accelerated approval pathway based on an increase in dystrophin production in skeletal muscle observed in treated patients. In the US, continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
The RACER53 Study is a randomized, double-blind, placebo-controlled, comparative study of 77 ambulatory boys with Duchenne. The study evaluated the efficacy and safety of an 80 mg/kg once weekly dosing of the treatment – versus placebo – for 48 weeks and was intended to serve as a confirmatory study.
The primary endpoint of the study was Time to Stand from Supine evaluated as velocity (rise/sec). The viltolarsen group showed a trend of increased velocity from baseline after treatment for 48 weeks. However, the placebo group also showed a trend of increased velocity, and there was no statistically significant difference between the viltolarsen group and the placebo group.
Preliminary safety results indicated that all adverse events that occurred under viltolarsen treatment were mild or moderate. There were no treatment emergent adverse events that led to discontinuation of the drug during the study.
“We are currently conducting further detailed data analyses and identifying factors that may have influenced the results (e.g. age, treatment period, and effect of
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concomitant drugs including glucocorticoid therapy),” said NS Pharma President Tsugio Tanaka, MSc. “Considering the results of prior clinical studies, we have confidence that viltolarsen can be a beneficial treatment for amenable patients with Duchenne.”
Specifically, in addition to the increase in dystrophin production in skeletal muscle that formed the basis of the FDA approval, a previously reported Phase 2, open- label, long-term extension study evaluated viltolarsen in 16 subjects between the ages of four and 10 with Duchenne amenable to exon 53 skipping. The study found that subjects receiving viltolarsen showed statistically significant improvements in the study’s primary endpoint of mean change from baseline for Time to Stand at week 205 as compared to a historical control group that was matched for key factors. In this study, treatment emergent adverse events were primarily mild or moderate. No study participants discontinued the study drug due to adverse events.
NS Pharma is currently conducting further detailed data analyses, including post- hoc data analyses, and plans to work closely with regulatory authorities to determine how to proceed based on the results of this analysis and in the best interests of patients. The company will report on additional analyses and discussions with the regulatory authorities at a later date.
About VILTEPSO® (Viltolarsen) Injection
Prior to its approval in the U.S. in August 2020, VILTEPSO was granted Priority Review as well as Rare Pediatric Disease, Orphan Drug and Fast Track Designations. In March 2020, VILTEPSO was approved in Japan for the treatment of patients with Duchenne who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted the SAKIGAKE designation, orphan drug designation, and designation of Conditional Early Approval System.
Indication
VILTEPSO is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VILTEPSO. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Important Safety Information
Warnings and Precautions: Kidney toxicity was observed in animals who received viltolarsen. Although kidney toxicity was not observed in the clinical studies with
VILTEPSO, the clinical experience with VILTEPSO is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VILTEPSO.
Serum creatinine may not be a reliable measure of kidney function in patients with Duchenne. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VILTEPSO. Consider also measuring glomerular filtration rate before starting VILTEPSO. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio every three months.
Urine should be free of excreted VILTEPSO for monitoring of urine protein. Obtain urine either prior to VILTEPSO infusion, or at least 48 hours after the most recent infusion. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, which has the potential to generate a false positive result due to cross reaction with any VILTEPSO in the urine. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation.
Adverse Reactions: The most common adverse reactions include upper respiratory tract infection, injection site reaction, cough, and pyrexia.
To report an adverse event, or for general inquiries, please call NS Pharma Medical Information at 1-866-NSPHARM (1-866-677-4276)
For more information about VILTEPSO, see full Prescribing Information.
About Duchenne Muscular Dystrophy (Duchenne)
Duchenne is a progressive form of muscular dystrophy that occurs primarily in males. It causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of Duchenne may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications. For more information about Duchenne, please visit wespeakduchenne.com.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com.
U.S. Media Contact:
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.
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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:
March 6, 2024
NS Pharma, Inc. Shares New VILTEPSO® (Viltolarsen) Data at the MDA Clinical & Scientific Conference 2024
Evidence of meaningful benefit in pulmonary function for patients with Duchenne muscular dystrophy will also be presented at the AAN 2024 Annual Meeting
PARAMUS, NJ: March 6, 2024 – NS Pharma, Inc. (NS Pharma) is excited to announce participation in the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Orlando, Florida, March 3 – 6. The company presented a poster entitled “Pulmonary and motor function in ambulatory and non-ambulatory participants with Duchenne muscular dystrophy (Duchenne) treated with viltolarsen (VILTEPSO®)” which covers data from the Galactic53 trial demonstrating that the majority of participants receiving viltolarsen experienced meaningful benefit in pulmonary function, including percent predicted forced vital capacity (FVC%p).
“Galactic53 is the first trial with VILTEPSO to evaluate pulmonary function in participants with Duchenne,” explains NS Pharma Vice President Medical Affairs & Pharmacovigilance Leslie Magnus, MD, who also co-authored the poster. “Our team is encouraged by these results and will continue our research into treatments for rare disease.”
Galactic53 was a Phase 2, open-label, multicenter study of viltolarsen administered intravenously, 80mg/kg once weekly, in both ambulatory and non- ambulatory individuals with Duchenne who are amenable to exon 53 skipping therapy. The study also found that the upper limb motor function of participants was stabilized over 49 weeks in both ambulatory and non-ambulatory patients. Viltolarsen was well tolerated by participants, and the safety profile was consistent with previous reports.
View the poster online: https://www.nspharma.com/events. Additional data from
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this study will also be presented at the American Academy of Neurology (AAN) 2024 Annual Meeting, April 13 – 18 in Denver, Colorado and online.
About VILTEPSO® (Viltolarsen) Injection
Prior to its approval in the U.S. in August 2020, VILTEPSO was granted Priority Review as well as Rare Pediatric Disease, Orphan Drug and Fast Track Designations. In March 2020, VILTEPSO was approved in Japan for the treatment of patients with Duchenne who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted the SAKIGAKE designation, orphan drug designation, and designation of Conditional Early Approval System.
Indication
VILTEPSO is indicated for the treatment of Duchenne in patients who have a confirmed mutation of the Duchenne gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VILTEPSO. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Important Safety Information
Warnings and Precautions: Kidney toxicity was observed in animals who received viltolarsen. Although kidney toxicity was not observed in the clinical studies with VILTEPSO, the clinical experience with VILTEPSO is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VILTEPSO. Serum creatinine may not be a reliable measure of kidney function in patients with Duchenne.
Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VILTEPSO. Consider also measuring glomerular filtration rate before starting VILTEPSO. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio every three months.
Urine should be free of excreted VILTEPSO for monitoring of urine protein. Obtain urine either prior to VILTEPSO infusion, or at least 48 hours after the
most recent infusion. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, which has the potential to generate a false positive result due to cross reaction with any VILTEPSO in the urine. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation.
Adverse Reactions: The most common adverse reactions include upper respiratory tract infection, injection site reaction, cough, and pyrexia.
To report an adverse event, or for general inquiries, please call NS Pharma Medical Information at 1-866-NSPHARM (1-866-677-4276)
For more information about VILTEPSO, see full Prescribing Information.
About Duchenne Muscular Dystrophy (Duchenne)
Duchenne is a progressive form of muscular dystrophy that occurs primarily in males. It causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of Duchenne may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications. For more information about Duchenne, please visit wespeakduchenne.com.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com.
U.S. Media Contact:
Brogidirsen, an investigational exon 44 skipping agent for the treatment of Duchenne muscular dystrophy: Clinical trial design (Phase 2)
Paula R. Clemens1,2, Michelle L. Previtera3 , Robert A. Crozier3 , Leslie Magnus 3 , Eric P. Hoffman4 , Hirofumi Komaki 5,6 , Yoshitsugu Aoki 7 , and Vamshi K. Rao8 1
Department of Neurology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; 2Department of Veterans Affairs Medical Center, Pittsburgh, PA, USA; 3NS Pharma, Inc., Paramus, NJ, USA; 4School of Pharmacy and Pharmaceutical Sciences, Binghamton University – SUNY, Binghamton, NY, USA; 5Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan; 6Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan; 7Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan; 8Division of Neurology, Ann and Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, USA
A Phase 1/2 study of NS-050/NCNP-03, an investigational exon 50 skipping therapy, in boys with Duchenne muscular dystrophy (Meteor50): Trial design
Pulmonary and motor function in ambulatory and nonambulatory participants with Duchenne muscular dystrophy treated with viltolarsen
January 22, 2024
The European Commission Grants Orphan Drug Designation to NS-229 for the Treatment of Eosinophilic Granulomatosis with Polyangiitis
PARAMUS, NJ: January 22, 2024 – NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd, announced today that the European Commission (EC) has granted orphan drug designation to NS-229, which is being developed for the treatment of the rare disease eosinophilic granulomatosis with polyangiitis (EGPA).
The orphan drug designation by the EC is issued for drugs which are intended to treat diseases that affect fewer than five in 10,000 people in the European Union and are life- threatening or chronically debilitating. The Orphan Drug Designation provides NS Pharma with a ten-year marketing exclusivity period, supporting the company’s continued development and evaluation of this therapy.
EGPA is an autoimmune disease that is generally preceded by symptoms of bronchial asthma and allergic rhinitis. This inflammation in the small blood vessels can cause tissue and organ damage to the lungs, sinuses, peripheral nerves, skin, and kidneys. The cause of EGPA is unknown.
“EGPA is a serious, life-threatening disease with unmet medical need,” explained NS Pharma Vice President, Research & Development, Takeshi Seita. “We are encouraged that our innovative therapy will proceed in development for the patients who need
treatment.”
NS-229 is a potent and selective Janus kinase (JAK) 1 inhibitor, developed in-house, which suppresses excessive activation of T cells, B cells and certain white blood cells. As a result, it is anticipated that NS-229 could reduce tissue damage and curb various symptoms of EGPA. A Phase II global study of NS-229 is scheduled to be conducted by NS Pharma.
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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 group of companies. For more information, please visit nspharma.com.
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December 21, 2023
NS-089/NCNP-02 Receives Orphan Drug Designation from the European Commission for the Treatment of Duchenne Muscular Dystrophy
PARAMUS, NJ: December 21, 2023 – NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd., announced that, on December 13, 2023, the European Commission (EC) has granted orphan drug designation for NS-089/NCNP-02, which is being developed for the treatment of Duchenne muscular dystrophy (Duchenne), a rare and deadly genetic disorder that occurs primarily in males. There are various genetic mutations that cause Duchenne, and NS-089/NCNP-02 targets a gene mutation that can be treated by exon 44 skipping.
The orphan drug designation by the EC is issued to drugs which are intended for diseases that affect fewer than five in 10,000 people in the European Union (EU) and are life- threatening or chronically debilitating. The designation provides NS Pharma with a ten- year marketing exclusivity period, supporting the company’s continued development and evaluation of this therapy.
NS-089/NCNP-2 was previously granted rare pediatric disease designation in June 2023, designated as a breakthrough therapy in July 2023, and designated as an orphan drug in July 2023 by the U.S. Food and Drug Administration (FDA).
“We are one step closer to helping patients with Duchenne who are amenable to exon 44 skipping access life-changing treatment,” said NS Pharma Vice President, Research & Development, Takeshi Seita. “Our team is ready and excited to continue our development of this innovative science.”
NS-089/NCNP-02 is an antisense nucleic acid discovered through joint research between Nippon Shinyaku and the National Center of Neurology and Psychiatry (NCNP). It 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 has been actively working to develop agents for the treatment of intractable and rare diseases, with a goal of launching treatments for patients with Duchenne as soon as possible.
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About Duchenne Muscular Dystrophy (Duchenne)
Duchenne is a progressive form of muscular dystrophy that occurs primarily in males. It causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of Duchenne may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications. For more information about Duchenne, please visit wespeakduchenne.com.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies. For more information, please visit nspharma.com.
U.S. Media Contact: