Ilginatinib

NS Pharma’s NS-089/NCNP-02 (brogidirsen) is intended for patients with Duchenne muscular dystrophy. Credit: Jose Luis Calvo via Shutterstock.com. The US Food and Drug Administration (FDA) has awarded rare paediatric disease (RPD) designation to NS Pharma’s investigational therapy, NS-089/NCNP-02 (brogidirsen), to treat Duchenne muscular dystrophy. The condition is responsive to exon 44-skipping therapy. Recommended Reports Reports LOA and PTSR Model - KVD-001 in Diabetic Macular Edema GlobalData Reports LOA and PTSR Model - Triamcinolone Acetonide in Diabetic Macular Edema GlobalData View all Companies Intelligence Nippon Shinyaku Co Ltd NS Pharma Inc View all NS-089/NCNP-02 is an antisense nucleotide discovered in collaborative research between the National Center for Psychiatry and Neurological Medicine and Nippon Shinyaku , the parent firm of NS Pharma. The drug candidate’s clinical development comprises a Phase II clinical trial to be carried out in the US by NS Pharma and a Phase II study to be conducted by Nippon Shinyaku in Japan. NS Pharma will provide further trial data once subject enrolment has commenced. The regulatory agency granted RPD status for therapies that could potentially treat serious or life-threatening ailments affecting 200,000 US patients who are 18 years or younger. Duchenne is a progressive type of muscular dystrophy seen primarily in men. It leads to gradual weakness and skeletal, cardiac and respiratory muscle loss. In December 2022, NS Pharma received orphan drug status from the US FDA for an investigational candidate, NS-018 (ilginatinib), to treat myelofibrosis, a rare type of blood cancer.

NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka) is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai) PARAMUS, N.J., Feb. 17, 2023 /PRNewswire/ -- NS Pharma, Inc. announced today the first patient enrollment in a Phase 2 clinical study of NS-018 (ilginatinib), an investigational treatment for myelofibrosis (MF), a rare and incurable blood cancer. The Phase 2b study is an open-label, multicenter, randomized, controlled, 2-arm study to assess the efficacy and safety of orally administered NS-018 versus best available therapy in subjects with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis with severe thrombocytopenia (Platelet Count <50,000/μL). ClinicalTrials.gov identifier: NCT04854096 ( ) About Myelofibrosis (MF) 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 About NS-018 NS-018 is a highly selective and potent inhibitor of JAK2 developed by scientists from Nippon Shinyaku and was recently granted Orphan Drug Designation from the U.S. Food and Drug Administration (FDA). About NS Pharma, Inc. NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit . NS Pharma is a registered trademark of the Nippon Shinyaku group of companies. U.S. Media Contact: media@nspharma.com References: What is pimary myleofibrosis? MPN Research Foundation. Accessed at: Myelofibrosis. Mayo Clinic. Accessed at: View original content: SOURCE NS Pharma