Friday, June 16, 2023
NS Pharma, Inc. has announced that the U.S. Food & Drug Administration (FDA) has granted approval for the planned Phase I/II study of NS-050/NCNP-03. This investigational therapy targets exon 50 skipping and is intended for patients with Duchenne muscular dystrophy. The study will evaluate key factors such as dystrophin production, muscle strength, mobility, and functional exercise capacity.
According to Tsugio Tanaka, President of NS Pharma, Inc., this FDA clearance marks the second trial approval obtained by the company in the current year. It also signifies the initiation of clinical trials for the third candidate in their Duchenne research and development pipeline. NS Pharma's rapid development plans reflect their commitment to addressing the pressing needs of the Duchenne community and expanding the impact of their exon skipping technology.
Enrollment for the clinical trial of NS-050/NCNP-03 in the United States is planned to commence in the second half of 2023, as stated by NS Pharma's parent company, Nippon Shinyaku. Further details regarding the trial and participant enrollment will be provided in due course.
Dr. Vamshi Rao from Ann & Robert H. Lurie Children's Hospital of Chicago emphasized the potential of exon skipping therapies to benefit a wide range of Duchenne patients. Currently, over half of the eligible patients for exon skipping therapy have no approved treatment options that address their specific mutation. Dr. Rao expressed excitement about the initiation of studies for two treatments this year, including NS-050/NCNP-03, which have the potential to reach more Duchenne patients through targeted exon skipping therapies.
In addition to NS-050/NCNP-03, Nippon Shinyaku, the parent company of NS Pharma, is actively pursuing the development of three other investigational exon skipping candidates at various stages of preclinical development.
Duchenne muscular dystrophy is a progressive condition primarily affecting males, characterized by the weakening and loss of skeletal, cardiac, and respiratory muscles. Early signs may include delays in sitting, standing, or walking, with a progressive loss of mobility and the potential need for a wheelchair during adolescence. Cardiac and respiratory muscle problems often emerge in the teenage years, leading to severe and life-threatening complications.