National Center of Neurology & Psychiatry

PARAMUS, N.J., Sept. 10, 2024 /PRNewswire/ -- 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. Continue Reading We are grateful for this designation which can help us accelerate the development of this therapy for Duchenne. Post this Yukiteru Sugiyama, PhD, NS Pharma President "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) 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: [email protected] SOURCE NS Pharma, Inc.

Pictured: Sign at Pfizer’s world headquarters in New York iStock , JHVEPhoto Pfizer’s investigational Duchenne muscular dystrophy gene therapy, fordadistrogene movaparvovec, failed in a late-stage study to significantly improve motor function in patients versus placebo. Pfizer on Wednesday announced that its investigational Duchenne muscular dystrophy gene therapy, fordadistrogene movaparvovec, fell short of its primary efficacy endpoint in the Phase III CIFFREO study. The company did not provide specific data in its announcement, only revealing that the gene therapy candidate was unable to significantly improve motor function in boys aged four to seven years, compared to placebo. Fordadistrogene movaparvovec also failed key secondary endpoints, including 10-meter run/walk velocity and time to rise from floor velocity. Despite showing no significant signals of efficacy, fordadistrogene movaparvovec had a manageable safety profile, with most adverse events being mild to moderate in severity. Treatment-related serious toxicities generally responded to management. Dan Levy, development head for Duchenne muscular dystrophy (DMD) at Pfizer, said in a statement that the company is “extremely disappointed” by the results. “We plan to share more detailed results from the study at upcoming medical and patient advocacy meetings” to help improve future research on DMD and develop effective treatment options for the disease, Levy said. Pfizer will continue to monitor study participants while it evaluates the next best steps for fordadistrogene movaparvovec, according to the announcement. Mainly afflicting young boys between two and three years of age, DMD is a severe genetic disorder that involves progressive muscular degeneration. The disease typically manifests initially as weakness and difficulties moving, before eventually affecting vital organs and leading to various complications, such as respiratory failure. DMD is caused by mutations to the gene encoding for the dystrophin protein, which is important for the stability and function of muscles. Fordadistrogene movaparvovec addresses the underlying cause of DMD by delivering a shortened but functional copy of the dystrophin gene. The candidate uses an AAV9 capsid delivery mechanism to specifically target muscle tissue. In addition to CIFFREO, Pfizer is assessing fordadistrogene movaparvovec in a Phase II DMD study. In May 2024, the pharma announced that a patient in the study “passed away suddenly” but did not provide the cause of death. In December 2021, Pfizer reported another patient death associated with fordadistrogene movaparvovec, which led the pharma to pause screening and dosing in its Phase Ib study. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com .

2024.03.11 News Release March 8th, 2024 TAGCyx biotechnologies Inc. TAGCyx biotechnologies Inc. (President and CEO: Chizuko Koseki), and Shinji Oki, PhD, at National Center of Neurology and Psychiatry (President: Kazuyuki Nakagome), Japan, announced entering into a research collaboration for the treatment for systemic lupus erythematosus (SLE) using a novel nucleic acid aptamer. TAGCyx has invented its proprietary artificial nucleic acid base-pair technology, Xenoligo® that enables to produce high avidity and selective functional DNA aptamers and is engaged in its own drug discovery business and drug discovery partnerships. Shinji Oki, PhD, Chief of the Demyelinating Disease Section at Department of Immunology, National Institute of Neuroscience, National Center of Neurology and Psychiatry (NCNP), is an expert in the research area of the mechanisms of immune cell function regulation, leading to the development of treatments for autoimmune diseases. Dr. Oki aims to develop novel therapies through the search for new target molecules that will lead to new drug discovery. Both parties intend to collaborative research to evaluate the efficacy of the DNA novel aptamers created by the artificial base-containing DNA aptamer discovery platform, Xenoligo®, for the treatment of SLE with in vitro and in vivo studies using SLE disease mouse model at NCNP. SLE is an autoimmune disease that is more prevalent in women of childbearing age, and SLE with neuropsychiatric symptoms are known to be intractable and show poor prognosis and pregnancies complicated with SLE are also known to be particularly high-risk for both mother and child. Nucleic acid aptamers have extremely low immunogenicity and placental permeability, and it is hoped that this research collaboration will lead to the development of safer treatments for SLE. For further information about this press release, please contact: TAGCyx biotechnologies Inc. Website E-Mail: info@tagcyx.com For further information about this research, please contact: National Center of Neurology and Psychiatry (NCNP) Shinji Oki, PhD Website E-Mail: soki@ncnp.go.jp