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
NEWS RELEASE
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:
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
NEWS RELEASE
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.
NEWS RELEASE
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:
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.
NEWS RELEASE
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:
NEWS RELEASE
November 21, 2023
NS Pharma Unveils Duchenne Heroes, a Program to Inspire and Amplify the Voices of People Living with Duchenne Muscular Dystrophy
Paramus, NJ: November 21, 2023 – NS Pharma, Inc. (“NS Pharma”) today announced the launch of Duchenne Heroes a program created to raise awareness and champion the voices of people affected by Duchenne muscular dystrophy (Duchenne). Duchenne Heroes are individuals, caregivers and family members who hope to inspire others by sharing their personal experiences in navigating diagnosis and daily life with Duchenne.
Duchenne Heroes share their authentic insights on a new educational website, WeSpeakDuchenne.com, which provides straightforward information to help families navigate the complexities of the Duchenne journey, and a platform to hear from others who have been down the same path.
“Our goal is to make a positive impact in the lives of people living with Duchenne by providing helpful information and uplifting voices from the community,” said Gilberto Gil, Director of Patient and Caregiver Marketing at NS Pharma. “The Duchenne Heroes’ insights and authentic experiences can inspire families who may be navigating a Duchenne diagnosis. It is my hope that these unique perspectives combined with easy-to-understand information about Duchenne Muscular Dystrophy, will make WeSpeakDuchenne.com a valuable online resource for the community.”
WeSpeakDuchenne.com includes educational resources such as a “Duchenne-opedia,” a glossary of useful terms to know, as well as a primer on the importance of increasing dystrophin for people with Duchenne, and a section where caregivers share their responses to commonly asked questions around diagnosis and treatment. The Duchenne Heroes Stories page shines a light on different families’ path to diagnosis and the learnings that helped as they navigate life with Duchenne.
“It means a lot to our family to participate in the Duchenne Heroes program as we know how life-changing a Duchenne diagnosis can be,” said Elizabeth Cojeen, member of the Duchenne Heroes program. “Our son, Emmett, inspires us every day with his bright smile and big personality to stay positive, and we hope sharing our story helps other families do the same.”
Additional Duchenne Heroes stories will be included over time to showcase diverse perspectives around topics relevant to the broader community.
“The biggest lesson I’ve learned since my son Brantley’s Duchenne diagnosis is that there is no clear-cut path or single solution for a lot of life’s transitions,” said Dianna Marlow, member of the Duchenne Heroes program. “Our family’s journey, like everyone else’s, is unique when it comes to things like schooling, independence and accessibility. As Duchenne Heroes, we’re proud to share our experiences if it helps to bring ideas or encouragement to other families.”
In addition to being featured on the website, Duchenne Heroes will share their stories across NS Pharma’s social media platforms (Facebook: @NS Pharma, Inc.; X: @NSPharmaInc.) and make appearances at advocacy events in the coming year.
To learn more about the Duchenne Heroes program and explore educational resources, please visit WeSpeakDuchenne.com.
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 pulmonary 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 use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and can 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 https://www.nspharma.com. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.
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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.
Contact
U.S. Media Contact:
NEWS RELEASE
August 8, 2023
NS-018, an Investigational Treatment for Myelofibrosis, Receives Orphan Drug
Designation from the European Commission
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 8, 2023 – NS Pharma, Inc. announced today that the European Commission (EC) has granted Orphan Drug Designation to NS-018 (ilginatinib) an oral, selective JAK2 inhibitor which is being investigated for the treatment of myelofibrosis (MF).
The EC Orphan Drug Designation is issued to investigational treatments for diseases that affect fewer than 5 in 10,000 people in the European Union and are life-threatening or chronically debilitating. The designation provides for a ten-year marketing exclusivity period. In the US, NS-018 received Orphan Drug Designation by the U.S. Food and Drug Administration in December 2022.
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.
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References:
https://www.mpnresearchfoundation.org/primary-myelofibrosis-pmf/