Product Development

<Japan>

February 07,2025

Stage Code No.
(Generic name)
Therapeutic field Indications Origin Development
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.
Preparing for launch NS-304
(selexipag)
intractable and rare diseases pediatric pulmonary arterial hypertension Nippon Shinyaku Co., Ltd. Nippon Shinyaku Co., Ltd.
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.)
Phase III GA101
(obinutuzumab)
intractable and rare diseases lupus nephritis In-licensed from
Chugai Pharmaceutical Co., Ltd.
Co-development with
Chugai Pharmaceutical Co., Ltd.
Phase III GA101
(obinutuzumab)
intractable and rare diseases pediatric nephrotic syndrome In-licensed from
Chugai Pharmaceutical Co., Ltd.
Co-development with
Chugai Pharmaceutical Co., Ltd.
Phase III GA101
(obinutuzumab)
intractable and rare diseases extra renal lupus In-licensed from
Chugai Pharmaceutical Co., Ltd.
Co-development with
Chugai Pharmaceutical Co., Ltd.
Phase III LY3527727
(pirtobrutinib)
hematologic malignancies mantle cell lymphoma Alliance agreement in Japan with
Eli Lilly Japan K.K.
Eli Lilly Japan K.K.
Phase III LY3527727
(pirtobrutinib)
hematologic malignancies chronic lymphocytic leukemia Alliance agreement in Japan with
Eli Lilly Japan K.K.
Eli Lilly Japan K.K.
Phase II NS-304
(selexipag)
cardiovascular arteriosclerosis obliterans Nippon Shinyaku Co., Ltd. Nippon Shinyaku Co., Ltd.
Phase II NS-580 gynecology endometriosis Nippon Shinyaku Co., Ltd. Nippon Shinyaku Co., Ltd.
Phase II NS-580 urological diseases chronic prostatitis / chronic pelvic pain syndrome Nippon Shinyaku Co., Ltd. Nippon Shinyaku Co., Ltd.
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.
Phase II NS-229 intractable and rare diseases eosinophilic granulomatosis with polyangiitis Nippon Shinyaku Co., Ltd. Nippon Shinyaku Co., Ltd.
Phase I/II NS-401
(tagraxofusp)
hematologic malignancies blastic plasmacytoid dendritic cell neoplasm In-licensed from
The Menarini Group
Nippon Shinyaku Co., Ltd.
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.
Phase I NS-917
(radgocitabine)
hematologic malignancies relapsed/refractory acute myeloid leukemia In-licensed from
Delta-Fly Pharma, Inc.
Nippon Shinyaku Co., Ltd.
Phase I NS-025 urological diseases urological diseases Nippon Shinyaku Co., Ltd. Nippon Shinyaku Co., Ltd.
Phase I NS-863 cardiovascular cardiovascular diseases Nippon Shinyaku Co., Ltd. Nippon Shinyaku Co., Ltd.

<Overseas>

Stage Code No.
(Generic name)
Therapeutic field Indications Origin Development
U.S. 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.
BLA filing CAP-1002
(deramiocel)
intractable and rare diseases Duchenne muscular dystrophy cardiomyopathy Partnered with
Capricor Therapeutics, Inc.
Capricor Therapeutics, Inc.
Rolling Submission RGX-121
(clemidsogene lanparvovec)
intractable and rare diseases Mucopolysaccharidosis Type Ⅱ Partnered with
REGENXBIO Inc.
REGENXBIO Inc.
Phase III CAP-1002
(deramiocel)
intractable and rare diseases Duchenne muscular dystrophy Partnered with
Capricor Therapeutics, Inc.
Capricor Therapeutics, Inc.
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.
Phase II NS-229 intractable and rare diseases eosinophilic granulomatosis with polyangiitis Nippon Shinyaku Co., Ltd. Nippon Shinyaku Co., Ltd.
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.
Phase I/II ATSN-101 intractable and rare diseases GUCY2D-associated Leber congenital amaurosis In-licensed from
Atsena Therapeutics, Inc.
Atsena Therapeutics, Inc.
Phase I/II RGX-111 intractable and rare diseases Mucopolysaccharidosis Type Ⅰ Partnered with
REGENXBIO Inc.
REGENXBIO Inc.