August 06,2026
| Stage | Code No. (Generic name) |
Therapeutic field | Indications | Origin | Development | Target area for application |
|---|---|---|---|---|---|---|
| Launch Phase III | NS-065/NCNP-01 (viltolarsen) | intractable and rare diseases | Duchenne muscular dystrophy | Co-development with National Center of Neurology and Psychiatry | Nippon Shinyaku Co., Ltd. | Japan/U.S. |
| Filed | CAP-1002 (deramiocel) | intractable and rare diseases | Duchenne muscular dystrophy cardiomyopathy | Partnered with Capricor Therapeutics, Inc. | Capricor Therapeutics, Inc. | U.S. |
| Filed | RGX-121 (clemidsogene lanparvovec) | intractable and rare diseases | Mucopolysaccharidosis Type II | Partnered with REGENXBIO Inc. | REGENXBIO Inc. | U.S. |
| Filed | GA101 (obinutuzumab) | intractable and rare diseases | Idiopathic nephrotic syndrome | In-licensed from Chugai Pharmaceutical Co., Ltd. | Co-development with Chugai Pharmaceutical Co., Ltd. | Japan |
| Preparation for BLA | Tadekinig alfa | intractable and rare diseases | NLRC4 and XIAP mutations | AB2 Bio Ltd. | AB2 Bio Ltd. | U.S. |
| Phase III | ZX008 (fenfluramine hydrochloride) | intractable and rare diseases | CDKL5 deficiency disorder | Distribution partnership with UCB S.A. (formerly known as Zogenix, Inc.) | UCB S.A. (formerly known as Zogenix, Inc.) | Japan |
| Phase III | GA101 (obinutuzumab) | intractable and rare diseases | Lupus nephritis | In-licensed from Chugai Pharmaceutical Co., Ltd. | Co-development with Chugai Pharmaceutical Co., Ltd. | Japan |
| Extra renal lupus | ||||||
| Phase III | CAP-1002 (deramiocel) | intractable and rare diseases | Duchenne muscular dystrophy | Partnered with Capricor Therapeutics, Inc. | Capricor Therapeutics, Inc. | U.S. |
| Phase III | LY3527727 (pirtobrutinib) | hematologic malignancies | Mantle cell lymphoma | Alliance agreement in Japan with Eli Lilly Japan K.K. | Eli Lilly Japan K.K. | Japan |
| Chronic lymphocytic leukemia | ||||||
| Phase III | NS-304 (selexipag) | cardiovascular | Arteriosclerosis obliterans | Nippon Shinyaku Co., Ltd. | Nippon Shinyaku Co., Ltd. | Japan |
| Preparation for Phase III | ZX008 (fenfluramine hydrochloride) | intractable and rare diseases | Rett syndrome | Distribution partnership with UCB S.A. (formerly known as Zogenix, Inc.) | UCB S.A. (formerly known as Zogenix, Inc.) | Japan |
| Phase II | NS-580 | gynecology | Endometriosis | Nippon Shinyaku Co., Ltd. | Nippon Shinyaku Co., Ltd. | Japan |
| urological diseases | Chronic prostatitis / Chronic pelvic pain syndrome | |||||
| Phase II | NS-089/NCNP-02 (brogidirsen) | intractable and rare diseases | Duchenne muscular dystrophy | Co-development with National Center of Neurology and Psychiatry | Nippon Shinyaku Co., Ltd. | Japan/U.S. |
| Phase II | NS-229 | intractable and rare diseases | Eosinophilic granulomatosis with polyangiitis | Nippon Shinyaku Co., Ltd. | Nippon Shinyaku Co., Ltd. | Japan/U.S. |
| Preparation for Phase II | NS-035 | intractable and rare diseases | Fukuyama congenital muscular dystrophy | Co-development with Kobe University | Nippon Shinyaku Co., Ltd. | Japan |
| Preparation for Phase II | NS-863 | cardiovascular | Pulmonary arterial hypertension | Nippon Shinyaku Co., Ltd. | Nippon Shinyaku Co., Ltd. | Japan/U.S. |
| Pulmonary hypertension associated with interstitial lung disease | ||||||
| Preparation for Phase II | NS-025 | urological diseases | Urological diseases | Nippon Shinyaku Co., Ltd. | Nippon Shinyaku Co., Ltd. | Japan |
| autoimmune disease | Xerostomia (dry mouth) associated with Sjögren’s disease | Aquelys Therapeutics, Inc. | U.S. | |||
| Phase I/II | NS-050/NCNP-03 | intractable and rare diseases | Duchenne muscular dystrophy | Co-development with National Center of Neurology and Psychiatry | Nippon Shinyaku Co., Ltd. | Japan/U.S. |
| Phase I/II | ATSN-101 | intractable and rare diseases | GUCY2D-associated Leber congenital amaurosis | In-licensed from Atsena Therapeutics, Inc. | Atsena Therapeutics, Inc. | U.S. |
| Phase I/II | RGX-111 | intractable and rare diseases | Mucopolysaccharidosis Type I | Partnered with REGENXBIO Inc. | REGENXBIO Inc. | U.S. |
| Phase I | NS-917 (radgocitabine) | hematologic malignancies | Relapsed/refractory acute myeloid leukemia | In-licensed from Delta-Fly Pharma, Inc. | Nippon Shinyaku Co., Ltd. | Japan |
| Phase I | NS-245 | inflammatory diseases | Inflammatory diseases | Nippon Shinyaku Co., Ltd. | Nippon Shinyaku Co., Ltd. | Japan |
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