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

Preparation for
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.