Drugs / brogidirsen
brogidirsen
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT04129294 | Dec 2019 → May 2022 | Duchenne muscular dystrophy | National Center of Neurology and Psychiatry, Japan | Completed | No outcome recorded |
| Phase 22 trials | ||||||
| Phase 2 | NCT05996003 | Feb 2024 → Sep 2026 overdue | Duchenne muscular dystrophy | NS Pharma, Inc. | Recruiting | No outcome recorded |
| Phase 2 | NCT05135663 | Jun 2021 → Apr 2027 expected | Duchenne muscular dystrophy | Nippon Shinyaku Co., Ltd. | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 6
| Date | Issuer | Release |
|---|---|---|
| 2026-03-09 | NS Pharma, Inc. | Results Brogidirsen (NS-089/NCNP-02) 4.5-Year Clinical Trial Data for the Treatment of Duchenne Muscular Dystrophy Presented at 2026 MDA Clinical & Scientific Conference nspharma.com ↗
From the initiation of weekly administration, participants maintained motor function in assessments such as North Star Ambulatory Assessment (NSAA). |
| 2025-10-14 | NS Pharma, Inc. | Results Brogidirsen (NS-089/NCNP-02) 3.5 Year Clinical Trial Data for the Treatment of Duchenne Muscular Dystrophy Presented at 2025 World Muscle Society Congress nspharma.com ↗
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. |
| 2025-10-14 | Nippon Shinyaku Co., Ltd. | Results デュシェンヌ型筋ジストロフィー治療剤「ブロギジルセン（NS-089/NCNP-02）」の世界筋学会における投与3.5年までの結果発表について nippon-shinyaku.co.jp ↗
有効性評価の結果、本剤を投与した被験者の生検筋では、投与25/26週および99/100週時において、エクソン44スキッピング効率およびジストロフィンの発現量が高い傾向を示しました。 |
| 2023-12-21 | NS Pharma, Inc. | Regulatory NS-089/NCNP-02 Receives Orphan Drug Designation from the European Commission for the Treatment of Duchenne Muscular Dystrophy nspharma.com ↗ |
| 2023-07-07 | NS Pharma, Inc. | Regulatory FDA Grants Rare Pediatric Disease Designation to NS-089/NCNP-02 for the Treatment of Duchenne Muscular Dystrophy nspharma.com ↗
NS Pharma, Inc. announced today the U.S. Food & Drug Administration (FDA) has granted Rare Pediatric Disease Designation to NS- 089/NCNP-02 (brogidirsen) an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy. |
| 2023-04-14 | NS Pharma, Inc. | Regulatory NS Pharma Announces FDA Clearance to Initiate Phase II Study for NS-089/NCNP-02, an Exon 44 Skipping Candidate for the Treatment of Duchenne Muscular Dystrophy nspharma.com ↗ |
All press releases naming this drug 19 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | brogidirsen | “we developed brogidirsen, a dual-targeting ASO composed of two directly connected 12-mer sequences targeting exon 44 using phosphorodiamidate morpholino oligomers.” PMID 39793573 ↗ Jan 2025 |
| Known as | NCNP-02 | ChEMBL registry synonym — accepted as the source's own label CHEMBL5095368 ↗ |
| Known as | NS-089 | ChEMBL registry synonym — accepted as the source's own label CHEMBL5095368 ↗ |
| Known as | NS-089/NCNP-02 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05996003 ↗ |
| Route | Intravenous | “administered by weekly IV infusion” NCT05996003 ↗ |