NEWS RELEASE
May 12, 2020

First Patient Enrolled in Phase 3 Trial of Viltolarsen(NS-065/NCNP-01), an Investigational Antisense Oligonucleotide
KYOTO, Japan: May 12, 2020 - Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President,Shigenobu Maekawa) announced today that NS Pharma (Paramus NJ, USA; President,Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku, has enrolled the first
patient in a Phase 3 trial of viltolarsen in Japan.
This Phase 3 trial is a randomized, double-blind, placebo-controlled, global study
including Japan and the U.S., assessing the safety and efficacy of viltolarsen inambulant boys with Duchenne muscular dystrophy (DMD) who are amenable to exon
53 skipping therapy. In this trial, patients will receive once weekly intravenous (IV)infusions of viltolarsen 80 mg/kg or placebo for 48 weeks. The study has a planned
enrollment of 74 boys with DMD who are 4 to less than 8 years old. Clinical efficacy will
be assessed at regularly scheduled study visits, and will include functional tests* suchas Time to Stand Test (TTSTAND), the primary endpoint. Additional functional tests will
also be assessed as the secondary endpoints. Safety will be assessed through thecollection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), vital
signs, and physical examinations throughout the study. Additional information about
this Phase 3 trial (NCT04060199) can be found on www.clinicaltrials.gov.

The overall clinical development of viltolarsen is characterized by our unwaveringcommitment to conduct robust trials that will help demonstrate the potential impact ofviltolarsen on patients with this devastating disease.
In March 2020, viltolarsen was approved in Japan for the treatment of DMD patientsamenable to exon 53 skipping therapy. Viltolarsen has not yet been approved in the U.S.
and its New Drug Application was recently granted Priority Review by the FDA with an
anticipated action date in the third quarter of 2020.

1/2

Functional Tests*Time to Stand Test (TTSTAND)Time to Run/Walk 10 Meters Test (TTRW)Six-minute Walk Test (6MWT)North Star Ambulatory Assessment (NSAA)

Time to Climb 4 Steps Test (TTCLIMB)

Hand-held dynamometer (elbow extension, elbow flexion, knee extension andknee flexion on the dominant side only).

About Duchenne Muscular Dystrophy (DMD)
DMD is a progressive form of muscular dystrophy that occurs primarily in males. DMD
causes progressive weakness and loss (atrophy) of skeletal, cardiac, and pulmonary
muscles. Early signs of DMD may include delayed ability to sit, stand, or walk anddifficulties learning to speak. DMD may also affect learning and memory, as well as
communication and certain social emotional skills. By adolescence, patients with DMD
may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in
the teen years and lead to serious, life- threatening complications.
About Viltolarsen
Viltolarsen has been granted a Rare Pediatric Disease Designation, Orphan Drug
Designation, and a Fast Track Designation in the U.S. Viltolarsen has received marketing
authorization under an accelerated approval pathway in Japan in March 2020.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more
information, please visit http://www.nspharma.com. NS Pharma is a registeredtrademark of the Nippon Shinyaku group of companies.

Contact
Corporate Communications Dept., Nippon Shinyaku
FAX: +81-75-321-9128

2/2