PIPELINE

Nippon Shinyaku Co., Ltd. has been developing compounds in several therapeutic fields. NS Pharma, Inc. has been developing NS-229 (for Eosinophilic granulomatosis with polyangiitis), NS-065/NCP-01 (for Duchenne muscular dystrophy), and NS-089/NCNP-02 (for Duchenne muscular dystrophy) on behalf of Nippon Shinyaku Co., Ltd.

MECHANISM

STAGE

FIELD

INDICATION

CODE

Exon 53
skipping

Launch/
Phase 3

Neurology

Duchenne muscular
dystrophy

NS-065/NCNP-01
(viltolarsen)*

MECHANISM

STAGE

FIELD

INDICATION

CODE

Cell therapy

Phase 3

Neurology

Duchenne muscular
dystrophy

CAP-1002†

MECHANISM

STAGE

FIELD

INDICATION

CODE

Exon 44
skipping

Phase 2

Neurology

Duchenne muscular
dystrophy

NS-089/NCNP-02
(brogidirsen)

MECHANISM

STAGE

FIELD

INDICATION

CODE

Exon 50
skipping

Phase 1/2

Neurology

Duchenne muscular
dystrophy

NS-050/NCNP-03

MECHANISM

STAGE

FIELD

INDICATION

CODE

Selective JAK1
inhibition

Phase 1/2

Inflammatory Diseases

Eosinophilic
granulomatosis
with polyangiitis (EGPA)

NS-229

MECHANISM

STAGE

FIELD

INDICATION

CODE

Exon 51
skipping

Preclinical

Neurology

Duchenne muscular
dystrophy

NS-051/
NCNP-04

MECHANISM

STAGE

FIELD

INDICATION

CODE

Exon 45
skipping

Preclinical

Neurology

Duchenne muscular
dystrophy

-

MECHANISM

STAGE

FIELD

INDICATION

CODE

Exon 55
skipping

Preclinical

Neurology

Duchenne muscular
dystrophy

-


EXPAND

EXPANDED ACCESS POLICY
NS Pharma, Inc. (NS Pharma) supports Expanded Access programs when we have substantial scientific evidence to support both the safety and the efficacy of investigational therapies based on the results from clinical studies. At this time, NS Pharma does not have unapproved, investigational therapies available for Expanded Access. This policy is not applied to NS Pharma’s approved drug, Viltepso®.

NS Pharma is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families and patient communities through education, empathy, and awareness.

NS Pharma’s mission is to bring life-changing therapies to patients with rare diseases and serious or life-threatening illnesses or conditions. Expanded Access refers to the use of an investigational therapy outside of a clinical trial for potential treatment of a serious condition in a patient. The US Food and Drug Administration (FDA) has established guidelines1) for Expanded Access taking into consideration the following points:

  • The patient has a serious or life-threatening illness or condition with no comparable or satisfactory alternative therapies;
  • The patient is unable to participate in a clinical trial;
  • The investigational drug is in active clinical development with sufficient data available;
  • A benefit-risk analysis, based on both the available clinical data, supports making the investigational drug available;
  • Making the investigational drug available will not negatively impact or delay the conduct of clinical trials; and
  • Adequate supply of the investigational drug is available.
NS Pharma is focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals to make our therapies available broadly to patients as quickly as possible. We are privileged to collaborate with clinical investigators and with patients who participate in our studies to develop life-changing therapies. We believe this approach will serve patients who could be helped by the therapies we are developing.

For a list of NS Pharma sponsored clinical trials currently recruiting patients, please visit www.clinicaltrials.gov. With regard to inquiries related to NS Pharma’s Expanded Access Policy, please contact us at expandedaccess@nspharma.com. We regularly monitor this mailbox and will use our best efforts to acknowledge each inquiry within 7 business days after receipt.
  1. Compassionate use refers to requests for access under the Federal Food, Drug, and Cosmetic Act Section 561A (FDA’s Expanded Access Program) and Section 561B (“Investigational Drugs For Use By Eligible Patients” as added by the Right To Try Act, P.L. 115-176).

*

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.