PIPELINE
Nippon Shinyaku Co., Ltd. has been developing compounds in several therapeutic fields, including hematologic malignancies and intractable/orphan diseases. NS Pharma, Inc. has been developing NS-018 (for myelofibrosis) and NS-065/NCNP-01 (for Duchenne muscular dystrophy) on behalf of Nippon Shinyaku Co., Ltd.
STAGE
CODE
FIELD
INDICATION
MECHANISM
Phase 3
NS-065/NCNP-01
(viltolarsen)*
Neurology
Duchenne muscular
dystrophy
Exon 53
skipping
STAGE
CODE
FIELD
INDICATION
MECHANISM
Phase 3
CAP-1002†
Neurology
Duchenne muscular
dystrophy
Cell therapy
STAGE
CODE
FIELD
INDICATION
MECHANISM
Phase 1/2
NS-089/
NCNP-02
Neurology
Duchenne muscular
dystrophy
Exon 44
skipping
STAGE
CODE
FIELD
INDICATION
MECHANISM
Phase 2
NS-018
Hematologic
malignancies
Myelofibrosis
Selective JAK2
inhibition
STAGE
CODE
FIELD
INDICATION
MECHANISM
Preclinical
NS-050
Neurology
Duchenne muscular
dystrophy
Exon 50
skipping
STAGE
CODE
FIELD
INDICATION
MECHANISM
Preclinical
NS-051
Neurology
Duchenne muscular
dystrophy
Exon 51
skipping
STAGE
CODE
FIELD
INDICATION
MECHANISM
Preclinical
NS-045
Neurology
Duchenne muscular
dystrophy
Exon 45
skipping
STAGE
CODE
FIELD
INDICATION
MECHANISM
Preclinical
NS-055
Neurology
Duchenne muscular
dystrophy
Exon 55
skipping
*
Viltepso (viltolarsen) injection was approved in the United States on August 12, 2020 (accelerated approval); approved in Japan (conditional approval).
†
Nippon Shinyaku Co., Ltd. is partnering with Capricor Therapeutics, which will be responsible for the progress and development of this program.
NCNP=National Center of Neurology and Psychiatry.
Indication
VILTEPSO is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VILTEPSO. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Important Safety Information
In clinical studies, no patients experienced kidney toxicity during treatment with VILTEPSO. However, kidney toxicity from drugs like VILTEPSO may be possible. Your doctor may monitor the health of your kidneys before starting and during treatment with VILTEPSO.
Common side effects include upper respiratory tract infection, injection site reaction, cough, and fever.
For more information about VILTEPSO, see full Prescribing Information.