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

TARGET
INDICATION

ORIGIN

DEVELOPMENT

MECHANISM
OF ACTION

Phase I/II

NS-018

Hematologic
malignancies

Myelofibrosis

Nippon
Shinyaku

NS Pharma

Selective JAK2
inhibition

STAGE

CODE

FIELD

TARGET
INDICATION

ORIGIN

DEVELOPMENT

MECHANISM
OF ACTION

Phase III

NS 065/
NCNP-01
(viltolarsen)*

Neurology

Duchenne
muscular
dystrophy

Nippon
Shinyaku,
NCNP

NS Pharma

Exon 53
skipping

*Viltepso (viltolarsen) injection was approved in the United States on August 12, 2020 (accelerated approval); approved in Japan (conditional approval)
NCNP=National Center of Neurology and Psychiatry.

For more information, go to www.VILTEPSO.com
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.