PARAMUS, NJ: October 01, 2026 – NS Pharma, Inc. (Headquarters: Paramus, New Jersey; President: Yukiteru Sugiyama), a biopharmaceutical leader in rare disease and subsidiary of Nippon Shinyaku Co., Ltd. (Headquarters: Kyoto, Japan; President: Toru Nakai), announced today that the National Center of Neurology and Psychiatry (NCNP, Kodaira City; President, Kazuyuki Nakagome) presented 5-year efficacy and safety data from the open-label extension of an investigator-initiated clinical trial of brogidirsen (NS-089/NCNP-02) for the treatment of Duchenne muscular dystrophy (DMD) at the 31st annual International Congress of the World Muscle Society held in Hiroshima, Japan from September 29 to October 3, 2026.
Brogidirsen is an antisense oligonucleotide co-discovered by Nippon Shinyaku and NCNP as an investigational therapy for DMD patients with dystrophin gene mutations that are amenable to exon 44 skipping.
“We are excited by the longer-term data demonstrating the potential for brogidirsen to slow disease progression in DMD patients amenable to exon 44 skipping,” said NS Pharma President, Yukiteru Sugiyama, Ph.D. “It is important for us to continue investigating these therapeutic innovations in hopes of providing patients with additional treatment options and renewed hope.”
The presented data are based on the investigator-initiated clinical trial conducted by NCNP and its extension study conducted by Nippon Shinyaku. These studies evaluated the efficacy and safety of brogidirsen in 6 participants who received weekly IV dosing.
Findings include:
These results suggest the potential of brogidirsen to slow DMD disease progression in patients who are amenable to exon 44 skipping. The extension trial is ongoing to investigate the efficacy and safety of long-term administration. Further, a global Phase II study of brogidirsen is being conducted by Nippon Shinyaku and its subsidiary NS Pharma. Learn more at ClinicalTrials.gov.
About Duchenne Muscular Dystrophy (DMD)
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 DMD 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, please visit wespeakduchenne.com.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary and registered trademark of Nippon Shinyaku Co., Ltd. Backed by over one hundred years of development expertise, NS Pharma is dedicated to delivering life-changing care to people living with complex, rare diseases. For more information, please visit nspharma.com.
US Media Contact: [email protected]
PARAMUS, NJ: July 30, 2026 – NS Pharma, Inc. (Headquarters: Paramus, New Jersey; President: Yukiteru Sugiyama) announced today that its parent company, Nippon Shinyaku Co., Ltd. (Headquarters: Kyoto, Japan; President: Toru Nakai), has exercised its option rights under the option agreement entered into with AB2 BIO Ltd. (Headquarters: Lausanne, Switzerland, CEO: Djordje Filipovic) in January 2025 regarding Tadekinig alfa. Through the exercise of the option rights, Nippon Shinyaku has obtained exclusive commercialization rights for Tadekinig alfa in the United States for the treatment of monogenic IL-18 driven Hyperinflammatory Syndrome in patients with NLRC4 and XIAP mutations.
About NLRC4 mutation and XIAP deficiency
NLRC4 mutation and XIAP deficiency are rare and serious hereditary autoinflammatory diseases, that cause elevated levels of interleukin-18 (IL-18) leading to a variety of inflammatory symptoms. Most patients show symptoms in infancy, with inflammation persisting throughout their lives. There are currently no approved therapies specifically indicated for either disease.
“The exercise of this option agreement further exemplifies our long-term commitment to shepherding therapeutic advances into novel treatments that profoundly improve the lives of rare disease patients and address unmet needs,” said Dr. Yukiteru Sugiyama, NS Pharma President. “We are proud to partner with AB2 BIO to bring this much needed treatment to an underserved patient population.”
About Tadekinig alfa
Tadekinig alfa is a recombinant human IL-18 binding protein that suppresses pathological IL-18-related immune and inflammatory responses by specifically binding to excessively produced IL-18. Tadekinig alfa has received Orphan Drug Designation, Breakthrough Therapy Designation, and Rare Pediatric Disease Designation in the United States, as well as Orphan Drug Designation in Europe. Following regulatory approval in the United States, Tadekinig alfa is expected to be commercialized by NS Pharma, Inc. a wholly owned subsidiary of Nippon Shinyaku.
About Nippon Shinyaku
Based on Nippon Shinyaku’s business philosophy, “Helping people lead healthier, happier lives,” we aim to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness. Please visit our website (https://www.nippon-shinyaku.co.jp/english/) for products or detailed information.
About AB2 BIO Ltd.
AB2 Bio is a biotech company developing innovative therapies for the treatment of severe systemic autoinflammatory diseases and conditions driven by IL-18. The company is advancing Tadekinig alfa in a wide range of IL-18 mediated hyperinflammatory diseases and conditions, including rare orphan diseases with high unmet medical needs, at clinical and pre-clinical phase.
AB2 Bio was founded in 2010 and is headquartered in the Innovation Park at the Ecole Polytechnique Fédérale de Lausanne (EPFL), Switzerland. More information can be found on http://www.ab2bio.com
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary and registered trademark of Nippon Shinyaku Co., Ltd. NS Pharma is dedicated to delivering life-changing care to people living with complex, rare diseases backed by over one hundred years of development expertise. For more information, please visit nspharma.com
US Media Contact: [email protected]
PARAMUS, NJ: June 26, 2026 – NS Pharma, Inc. (NS Pharma; Headquarters: Paramus, NJ; President: Yukiteru Sugiyama) announced today that its parent company, Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters: Kyoto, Japan; President: Toru Nakai) has entered into an option agreement with Elixirgen Therapeutics, Inc. (Elixirgen; Headquarters, Baltimore, MD; CEO: Aki Ko) under which Nippon Shinyaku may obtain the exclusive worldwide rights to develop and commercialize EXG-7001 for the treatment of Duchenne muscular dystrophy (DMD). The agreement was facilitated by the NS Pharma Innovation Research Partnering (IRP) team, located in Cambridge, MA.
“Collaboration is essential in furthering scientific innovation, and we are proud to partner with Elixirgen to expand treatment options for the Duchenne community,” said NS Pharma President Yukiteru Sugiyama. “This partnership represents our long-term commitment to supporting our community and the necessary scientific exploration needed to combat rare disease.”
EXG-7001 is an investigational, locally administered, mRNA based therapeutic drug that expresses full-length human dystrophin protein. Regardless of genetic mutation, it is intended to suppress local muscle dysfunction by expressing the full-length dystrophin protein, which is deficient within the muscle cells of patients with DMD. EXG-7001 is in preclinical development for the treatment of DMD. Elixirgen is currently preparing for clinical trials in the United States. Following the exercise of the option rights by Nippon Shinyaku and regulatory approval in the United States, NS Pharma intends to commercialize EXG-7001.
About Duchenne Muscular Dystrophy (Duchenne/DMD)
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 Nippon Shinyaku
Based on Nippon Shinyaku’s business philosophy, “Helping people lead healthier, happier lives,” we aim to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness. For more information, please visit nippon-shinyaku.co.jp/english.
About Elixirgen Therapeutics, Inc.
Elixirgen is a clinical stage biotechnology company focused on the treatment of rare and aging-associated diseases using its cutting-edge technologies. For more information, visit ElixirgenTx.com and follow on LinkedIn.
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.
US Media Contact: [email protected]
PARAMUS, NJ: June 23, 2026 – NS Pharma, Inc. (NS Pharma; Headquarters: Paramus, New Jersey, USA; President: Yukiteru Sugiyama) announced today that its parent company, Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters: Kyoto, Japan; President: Toru Nakai), has entered into a Sponsored Research Agreement (SRA) with Boston Children’s Hospital (Headquarters: Boston, Massachusetts, USA; Chief Executive Officer: Kevin B. Churchwell) for the purpose of developing innovative therapeutic drugs in the field of neurology. The agreement was facilitated by the NS Pharma Innovation Research Partnering (IRP) team, located in Cambridge, MA.
Following the initiation of the strategic collaboration announced in July 2025, Nippon Shinyaku has selected key research themes as proposed by Boston Children’s Hospital as joint research programs. Through co-creation, Nippon Shinyaku will integrate Boston Children’s Hospital’s world class research capabilities with the company’s drug discovery expertise to accelerate the creation of new neurologic therapeutic options for patients with unmet medical needs.
“This new agreement demonstrates meaningful collaborative progress towards furthering rare disease research and the benefits of our combined expertise,” said Dr. Yukiteru Sugiyama, NS Pharma President. “We look forward to continuing to deepen our alliance with Boston Children’s Hospital and setting a positive example of impactful partnership for our rare disease communities.”
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.
US Media Contact:
[email protected]
PARAMUS, NJ: March 9, 2026 – NS Pharma, Inc. (NS Pharma, New Jersey, USA; President, Yukiteru Sugiyama ), a biopharmaceutical leader in rare diseases and subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that the National Center of Neurology and Psychiatry (NCNP, Kodaira City; President, Kazuyuki Nakagome) presented 4.5-year safety and efficacy data based on the open-label extension study, including an investigator-initiated clinical trial of brogidirsen (NS-089/NCNP-02) for the treatment of Duchenne muscular dystrophy (DMD) at the 2026 MDA Clinical & Scientific Conference from March 8-11, 2026.
Brogidirsen is an antisense oligonucleotide co-discovered by Nippon Shinyaku and NCNP as an investigational therapy for DMD patients with dystrophin gene mutations that are amenable to exon 44 skipping.
“We are thrilled to see the data continue to demonstrate long-term efficacy in DMD patients amenable to exon 44 skipping and while maintaining patient safety,” said NS Pharma President, Yukiteru Sugiyama, Ph.D. “We are committed to expanding available treatment options to the DMD community and we look forward to realizing that ambition.”
The presented data are based on the first-in-human Phase 1/2 open-label investigator-initiated clinical trial conducted by NCNP and the subsequent Phase 2 open-label extension trial conducted by Nippon Shinyaku. These studies evaluate the safety and efficacy of brogidirsen in six participants who received weekly IV dosing of brogidirsen.
Findings include:
These findings support the potential of brogidirsen to modify the progression of DMD. This extension trial is ongoing to investigate the safety and efficacy of longer-term administration.
A second, ongoing global Phase II study of brogidirsen is being conducted by Nippon Shinyaku and NS Pharma to further evaluate the safety and efficacy of brogidirsen. Learn more at ClinicalTrials.gov.
About Duchenne Muscular Dystrophy (DMD)
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 DMD 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, 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.
US Media Contact:
[email protected]
PARAMUS, NJ: October 14, 2025 – NS Pharma, Inc. (NS Pharma), a biopharmaceutical leader in rare disease and subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that the National Center of Neurology and Psychiatry (NCNP, Kodaira City; President, Kazuyuki Nakagome) presented 3.5-year efficacy and safety data from the open-label extension of an investigator-initiated clinical trial of brogidirsen (NS-089/NCNP-02) for the treatment of Duchenne muscular dystrophy (DMD)at the 30th annual International Congress of the World Muscle Society from October 7 to 11, 2025.
Brogidirsen is an antisense oligonucleotide co-discovered by Nippon Shinyaku and NCNP as an investigational therapy for DMD patients with dystrophin gene mutations that are amenable to exon 44 skipping.
“These long-term motor function data suggest the potential for brogidirsen to slow disease progression in DMD patients amenable to exon 44 skipping and support the robust increases in dystrophin production seen earlier in the trial,” said NS Pharma President, Yukiteru Sugiyama, Ph.D. “We are proud to offer hope to families and continue to demonstrate our commitment to the DMD community
The presented data are based on the investigator-initiated clinical trial conducted by NCNP and its extension study conducted by Nippon Shinyaku. These studies evaluated the efficacy and safety of brogidirsen in 6 participants who received weekly IV dosing of brogidirsen.
Findings include:
• Consistent functional benefits – High exon 44 skipping efficiency and dystrophin expression levels were observed in biopsied muscles at week 25/26 and week 99/100.
• Maintenance of motor function – Participants who remain ambulant after long-term brogidirsen administration maintained their motor function in evaluations including North Star Ambulatory Assessment.
• Acceptable safety profile – After 3.5-year of receiving brogidirsen, no serious or severe adverse events, or anaphylaxis related to long-term brogidirsen administration, were reported, and there were no discontinuations.
These findings support the potential of brogidirsen to modify the progression of DMD. This long-term extension trial is ongoing to investigate the efficacy and safety of longer-term administration.
Further, a global Phase II study of brogidirsen is being conducted by Nippon Shinyaku and NS Pharma, Inc. (Headquarters: New Jersey, USA; President: Yukiteru Sugiyama). Learn more at ClinicalTrials.gov.
About Duchenne Muscular Dystrophy (DMD)
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 DMD 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, 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.
US Media Contact:
[email protected]
PARAMUS, NJ: September 19, 2025 – NS Pharma, Inc. announced today that the U.S. Food & Drug Administration (FDA) has granted Orphan Drug Designation to NS-051/NCNP-04 which is being developed for the treatment of Duchenne muscular dystrophy (Duchenne) in patients amenable to exon 51 skipping. 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-051/NCNP-04 previously received Rare Pediatric Disease Designation from the FDA in January 2025. For more information, see the press release here.
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 the skipping of exon 51 within the dystrophin gene, causing the production of a shortened dystrophin protein containing essential functional portions. This is expected to have the effect of stabilizing or improving muscle function.
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.
US Media Contact:
[email protected]
PARAMUS, NJ: September 9, 2025 – NS Pharma, Inc. (NS Pharma) a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that the U.S Food & Drug Administration (FDA) has granted Fast Track designation to NS-229, which is being developed for the treatment of the rare disease eosinophilic granulomatosis with polyangiitis (EGPA). NS-229 is being investigated as a selective Janus kinase 1 (JAK1) inhibitor to help regulate immune cell function and prevent the immune system from causing tissue damage.
FDA Fast Track designation status is granted to treatments of serious medical conditions that fulfil an unmet medical need. This designation allows for expedited FDA review, including more frequent collaboration with the FDA throughout the application process, to facilitate the delivery of important new therapies more quickly. NS-229 was granted Orphan Drug Designation by the FDA in April 2025.
About EGPA
EGPA, previously known as Churg-Strauss syndrome, is a rare autoimmune disease that causes inflammation in the small-to-medium-sized blood vessels which can cause tissue and organ damage to the lungs, sinuses, peripheral nerves, skin, and kidneys. EGPA is generally preceded by symptoms of bronchial asthma and allergic rhinitis. The cause is unknown. It is estimated that EGPA affects between 5,600 and 14,500 people in the U.S.*
A Phase 2, double-blind, randomized placebo-controlled global study of NS-229 is being conducted by Nippon Shinyaku and NS Pharma to assess the efficacy and safety of the investigational JAK1 inhibitor NS-229, in treating EGPA patients. By inhibiting JAK1, NS-229 may help regulate the overactive immune response characteristic of EGPA, potentially limiting the damage to heathy tissues.
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 www.nspharma.com.
US Media Contact:
[email protected]
*Estimated prevalences 1.7 / 100,0001) and 4.4 / 100,0002) were multiplied by a 2023 U.S. population estimate of around 330 million and rounded to nearest hundred.
1) Bell, CF., Lau, M., Shen, Q. Clinical and Economic Characteristics of Patients Diagnosed with Eosinophilic Granulomatosis with Polyangiitis (EGPA, formerly Churg-Strauss Syndrome) in the United States [abstract]. Arthritis Rheumatol. 2018; 70 (suppl 9).
2) Berti A, Cornec D, Crowson CS, Specks U, Matteson EL. The Epidemiology of Antineutrophil Cytoplasmic Autoantibody-Associated Vasculitis in Olmsted County, Minnesota: A Twenty-Year US Population-Based Study. Arthritis Rheumatol. 2017;69:2338-2350.