March 6, 2024

NS Pharma, Inc. Shares New VILTEPSO®  (Viltolarsen) Data at the MDA Clinical & Scientific Conference 2024

Evidence of meaningful benefit in pulmonary function for patients with Duchenne muscular dystrophy will also be presented at the AAN 2024 Annual Meeting

PARAMUS, NJ: March 6, 2024 – NS Pharma, Inc. (NS Pharma) is excited to announce participation in the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Orlando, Florida, March 3 – 6. The company presented a poster entitled “Pulmonary and motor function in ambulatory and non-ambulatory participants with Duchenne muscular dystrophy (Duchenne) treated with viltolarsen (VILTEPSO®)” which covers data from the Galactic53 trial demonstrating that the majority of participants receiving viltolarsen experienced meaningful benefit in pulmonary function, including percent predicted forced vital capacity (FVC%p).

“Galactic53 is the first trial with VILTEPSO to evaluate pulmonary function in participants with Duchenne,” explains NS Pharma Vice President Medical Affairs & Pharmacovigilance Leslie Magnus, MD, who also co-authored the poster. “Our team is encouraged by these results and will continue our research into treatments for rare disease.”

Galactic53 was a Phase 2, open-label, multicenter study of viltolarsen administered intravenously, 80mg/kg once weekly, in both ambulatory and non- ambulatory individuals with Duchenne who are amenable to exon 53 skipping therapy. The study also found that the upper limb motor function of participants was stabilized over 49 weeks in both ambulatory and non-ambulatory patients. Viltolarsen was well tolerated by participants, and the safety profile was consistent with previous reports.

View the poster online: https://www.nspharma.com/events. Additional data from

NEWS RELEASE

this study will also be presented at the American Academy of Neurology (AAN) 2024 Annual Meeting, April 13 – 18 in Denver, Colorado and online.

About VILTEPSO® (Viltolarsen) Injection

Prior to its approval in the U.S. in August 2020, VILTEPSO was granted Priority Review as well as Rare Pediatric Disease, Orphan Drug and Fast Track Designations. In March 2020, VILTEPSO was approved in Japan for the treatment of patients with Duchenne who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted the SAKIGAKE designation, orphan drug designation, and designation of Conditional Early Approval System.

Indication

VILTEPSO is indicated for the treatment of Duchenne in patients who have a confirmed mutation of the Duchenne 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

Warnings and Precautions: Kidney toxicity was observed in animals who received viltolarsen. Although kidney toxicity was not observed in the clinical studies with VILTEPSO, the clinical experience with VILTEPSO is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VILTEPSO. Serum creatinine may not be a reliable measure of kidney function in patients with Duchenne.

Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VILTEPSO. Consider also measuring glomerular filtration rate before starting VILTEPSO. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio every three months.

Urine should be free of excreted VILTEPSO for monitoring of urine protein. Obtain urine either prior to VILTEPSO infusion, or at least 48 hours after the

most recent infusion. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, which has the potential to generate a false positive result due to cross reaction with any VILTEPSO in the urine. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation.

Adverse Reactions: The most common adverse reactions include upper respiratory tract infection, injection site reaction, cough, and pyrexia.

To report an adverse event, or for general inquiries, please call NS Pharma Medical Information at 1-866-NSPHARM (1-866-677-4276)

For more information about VILTEPSO, see full Prescribing Information.

About Duchenne Muscular Dystrophy (Duchenne)

Duchenne is a progressive form of muscular dystrophy that occurs primarily in males. It causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of Duchenne may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications. For more information about Duchenne, please visit wespeakduchenne.com.

About NS Pharma, Inc.

NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com.

U.S. Media Contact:

media@nspharma.com

NEWS RELEASE

August 8, 2023

NS-018, an Investigational Treatment for Myelofibrosis, Receives Orphan Drug

Designation from the European Commission

NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai)

Paramus, NJ: August 8, 2023 – NS Pharma, Inc. announced today that the European Commission (EC) has granted Orphan Drug Designation to NS-018 (ilginatinib) an oral, selective JAK2 inhibitor which is being investigated for the treatment of myelofibrosis (MF).

The EC Orphan Drug Designation is issued to investigational treatments for diseases that affect fewer than 5 in 10,000 people in the European Union and are life-threatening or chronically debilitating. The designation provides for a ten-year marketing exclusivity period. In the US, NS-018 received Orphan Drug Designation by the U.S. Food and Drug Administration in December 2022.

MF is caused by buildup of excessive scar tissue in the bone marrow, which impairs the body’s ability to produce blood cells.1 In addition to impaired blood cell production, MF often leads to enlargement of the spleen (splenomegaly) which can lead to feelings of abdominal pain and pressure.1 Other common symptoms include fatigue, bone pain, fever, and weight loss.1 MF can be diagnosed at any age but is most common in men and women 65 years or older.1 The median survival of patients with MF is approximately six years.1

Several gene mutations are associated with MF, and the most common mutation is to the Janus kinase 2 (JAK2) gene.2 NS-018 is a highly selective and potent inhibitor of JAK2 developed by scientists from Nippon Shinyaku.

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 registered trademark of the Nippon Shinyaku group of companies.

Contact

U.S. Media Contact:

media@nspharma.com

References:

  1. What is primary myleofibrosis? MPN Research Foundation. Accessed at:

https://www.mpnresearchfoundation.org/primary-myelofibrosis-pmf/

  1. Myelofibrosis. Mayo Clinic. Accessed at: https://www.mayoclinic.org/diseases-

conditions/myelofibrosis/symptoms-causes/syc-20355057

NEWS RELEASE

July 28, 2023

FDA Grants Breakthrough Therapy Designation to NS-089/NCNP-02 for the Treatment of Duchenne Muscular Dystrophy

NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka) is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai)

PARAMUS, NJ: July 28, 2023 – NS Pharma, Inc. announced today the U.S. Food & Drug Administration (FDA) has granted Breakthrough Therapy Designation to NS- 089/NCNP-02, an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.

The Breakthrough Therapy Designation for NS-089/NCNP-02 is based on results from a first-in-human, investigator-initiated clinical trial conducted in Japan.1 The FDA issues Breakthrough Therapy Designation to expedite the development and review of medicines which are intended to treat serious or life-threatening diseases. The criteria require preliminary clinical evidence that indicates the drug may demonstrate substantial improvement over available therapies on a clinically meaningful endpoint(s). In June 2023, NS-089/NCNP-02 was granted Rare Pediatric Disease Designation by the FDA.

NS-089/NCNP-02 is an antisense nucleotide discovered through joint research between NS Pharma’s parent company, Nippon Shinyaku, and the National Center of Neurology and Psychiatry (Kodaira City, President: Kazuyuki Nakagome).

Clinical development of NS-089/NCNP-02 includes a planned Phase 2 study in the United States conducted by NS Pharma and a Phase 2 study conducted in Japan by

Nippon Shinyaku. Additional details will be provided once the trials are ready to begin enrolling participants.

About Duchenne Muscular Dystrophy (Duchenne)

Duchenne is a progressive form of muscular dystrophy that occurs primarily in males. Duchenne causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of Duchenne may include delayed ability to sit, stand or walk.

There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications.

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 registered trademark of the Nippon Shinyaku group of companies.

Contact

U.S. Media Contact: media@nspharma.com

Reference

1. National Center of Neurology and Psychiatry Press release on March 17, 2022.

https://www.ncnp.go.jp/topics/2022/20220317e.html

NEWS RELEASE

June 15, 2023

NS Pharma Announces FDA Clearance to Initiate a Phase I/II Study for NS-050/NCNP-03, an Exon 50 Skipping Candidate for the Treatment of Duchenne Muscular Dystrophy

NS-050/NCNP-03 is NS Pharma’s second exon skipping investigational therapy to receive FDA clinical study initiation clearance in 2023. Their exon 44 skipping treatment, NS-089/NCNP-02 was cleared on March 31, 2023.

NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka) is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai)

PARAMUS, NJ: June 15, 2023 – NS Pharma, Inc. announced today the U.S. Food & Drug Administration (FDA) has agreed to the planned Phase I/II study of

NS-050/NCNP-03, an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 50 skipping therapy. Study assessments will include dystrophin production, muscle strength, mobility and functional exercise capacity.

“This is the second trial clearance from the FDA that NS Pharma has received this year and marks the third candidate from our R&D pipeline to begin clinical trials in Duchenne,” said Tsugio Tanaka, President, NS Pharma, Inc. “Our rapid development plans reflect the urgent needs of the Duchenne community and our commitment to extending the impact of our exon skipping technology.”

NS Pharma’s parent company, Nippon Shinyaku, plans to begin clinical trial enrollment for NS-050/NCNP-03 in the US during the second half of 2023. Additional details will be provided once the trial is ready to begin enrolling participants.

“Exon skipping therapies have the potential to treat a wide range of patients with Duchenne, but more than half of Duchenne patients potentially amenable to exon skipping therapy have no approved treatment options that target their specific mutation,” said Vamshi Rao, MD, Ann & Robert H. Lurie Children’s Hospital of Chicago. “That is why it is exciting that this year we are beginning studies of two treatments that have the potential to reach even more Duchenne patients with targeted exon skipping therapies.”

In addition to NS-050/NCNP-03, NS Pharma’s parent company, Nippon Shinyaku, has three investigational exon skipping candidates in various stages of preclinical development.

About Duchenne Muscular Dystrophy (Duchenne)

Duchenne is a progressive form of muscular dystrophy that occurs primarily in males. Duchenne causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of Duchenne may include delayed ability to sit, stand or walk.

There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications.

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 registered trademark of the Nippon Shinyaku group of companies.

Contact

U.S. Media Contact:

media@nspharma.com

January 6, 2023

NS Pharma opens new U.S drug discovery center in Cambridge, MA

NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka) is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai)

Paramus, NJ: January 6, 2023 – NS Pharma announced today the opening of a new office in Cambridge, Massachusetts, to establish the NS Pharma Innovation Research Partnering division.

The new Boston area location is the company’s center for research and development (R&D) in the United States and will diversify Nippon Shinyaku’s R&D portfolio through open innovation. Cambridge, MA, is home to many notable universities, hospitals, research institutions, and bio- venture firms. Establishing a presence in this thriving drug discovery ecosystem enables the company to increase its access to the world’s most advanced seeds of drug discovery technology. The new division will pursue meaningful research collaboration opportunities and promote acceleration and diversification of in-house R&D activities on behalf of Nippon Shinyaku.

“The creation of NS Pharma Innovation Research Partnering division underscores our continued commitment to finding solutions for the unmet needs of patients around the world,” said Tsugio Tanaka, President, NS Pharma, Inc. “As a leader in scientific innovation, Cambridge, MA, will be a natural fit as a hub for our future research collaborations, and we are thrilled to be joining this vibrant community.”

[Office Profile]

Name: NS Pharma, Innovation Research Partnering

Address: One Broadway, Floor 14, Cambridge, Massachusetts, 02142, USA

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 registered trademark of the Nippon Shinyaku group of companies.

Contact

U.S. Media Contact: media@nspharma.com

NEWS RELEASE

NEWS RELEASE

Oct 4, 2022

VILTEPSO® (viltolarsen) injection Four-Year Clinical Trial Data to be Presented at the World Muscle Society 2022 Conference

PARAMUS, NJ: Oct 4, 2022 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai), announced today that four-year efficacy and safety data from the open-label extension of a Phase 2 study of VILTEPSO® (viltolarsen), was accepted as a late-breaking e-poster and will be presented at the World Muscle Society 2022 Conference being held in Halifax, Canada from October 11-15.

“We are deeply thankful to the patients and their caregivers from the Phase 2 trial of VILTEPSO, all of whom chose to take part in the long-term extension study,” said Leslie Magnus, MD, Vice President, Medical Affairs. “These data represent the longest clinical experience with an exon 53 skipping therapy and we look forward to sharing the results with the Duchenne community.”

About VILTEPSO® (viltolarsen) injection

Prior to its approval in the U.S. in August 2020, VILTEPSO was granted Priority Review as well as Rare Pediatric Disease, Orphan Drug and Fast Track Designations. In March 2020, VILTEPSO was approved in Japan for the treatment of patients with DMD who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted with the SAKIGAKE designation, Orphan Drug designation, and designation of Conditional Early Approval System.

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

Warnings and Precautions: Kidney toxicity was observed in animals who received viltolarsen. Although kidney toxicity was not observed in the clinical studies with VILTEPSO, the clinical experience with VILTEPSO is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VILTEPSO. Serum creatinine may not be a reliable measure of kidney function in DMD patients.

Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VILTEPSO. Consider also measuring glomerular filtration rate before starting VILTEPSO. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio every three months.

Urine should be free of excreted VILTEPSO for monitoring of urine protein. Obtain urine either prior to VILTEPSO infusion, or at least 48 hours after the most recent infusion. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, which has the potential to generate a false positive result due to cross reaction with any VILTEPSO in the urine. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation.

Adverse Reactions: The most common adverse reactions include upper respiratory tract infection, injection site reaction, cough, and pyrexia.

To report an adverse event, or for general inquiries, please call NS Pharma Medical Information at 1-866-NSPHARM (1-866-677-4276)

For more information about VILTEPSO, see full Prescribing Information.

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 registered trademark of the Nippon Shinyaku group of companies.

Contact

U.S. Media Contact:

media@nspharma.com

NEWS RELEASE

July 1, 2021

VILTEPSO® (viltolarsen) injection: Long-Term Efficacy and Safety Data Presented at the PPMD 2021 Virtual Annual Conference

Efficacy and safety results were based on interim analyses at week 109 from the open-label extension trial of a VILTEPSO Phase 2 study.

The primary endpoint of Time to Stand showed a statistically significant benefit for multiple time points over two years of treatment with VILTEPSO in comparison to DMD matched historical control group.

Additional secondary endpoints of motor function including Time to Run/Walk and 6- Minute Walk Test demonstrated consistent and statistically significant benefits in comparison to DMD historical controls.

There were no treatment related serious adverse events and no patients discontinued treatment.

PARAMUS, NJ: July 1, 2021 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai), announced that new long-term efficacy and safety data based on interim analyses at 109 weeks from the open-label extension trial of a Phase 2 study of VILTEPSO® (viltolarsen) injection were presented at the PPMD 2021 Annual Conference.

“These analyses showed that, after more than two years of treatment with VILTEPSO, patients maintained their motor function based on clinically relevant measurements while the DMD historic controls showed functional decline,” said Leslie Magnus, MD, Vice President, Medical Affairs.

This current open-label trial (N=16) is the extension of a previous 24-week trial in North America. All 16 patients in the short-term study elected to enroll in this long-term trial. Participants were assessed at weeks 37, 49, 73 and 109 and will continue to be assessed until study completion. These interim analyses of the timed function tests were conducted for all participants who had received at least 109 weeks of total treatment vs. the matched DMD historical control group (Cooperative International Neuromuscular Research Group

– Duchenne Natural History Study (CINRG-DNHS)).

The efficacy results were for Time to Stand from supine (mean change from baseline (seconds) at weeks 73 and 109 for viltolarsen was 0.21 and 0.43 vs CINRG-DNHS: 3.6 and 4.3, p<0.01), Time to Run/Walk 10 meters (mean change from baseline (seconds) at weeks 49, 73, and 109 for viltolarsen was -0.8, -0.9, and -0.4 vs CINRG-DNHS: 0.5, 1.3, 1.3, p<0.05), and 6-Minute Walk Test (mean change from baseline (meters) at week 109 for viltolarsen was 0.9 vs CINRG-DNHS: -65.6, p<0.05).

The most frequently reported adverse events were mild to moderate in this time-period and included cough, nasopharyngitis, rash, pyrexia, and vomiting. This safety profile was similar to that seen in the previous short-term study. To date, there were no treatment-related serious adverse events and no treatment discontinuations. These data provide important information on the efficacy and safety of the long-term use of viltolarsen for the treatment of DMD patients who are amenable to exon 53 skipping.

“Duchenne is a progressive disease of functional deterioration,” said study investigator Paula Clemens, MD, University of Pittsburgh Medical Center. “More research is needed, but a disease-modifying therapy that could stabilize and delay the loss of muscle function is needed for families with Duchenne and the healthcare professionals who specialize in its treatment.”

In addition to this Phase 2 open-label extension  study, NS  Pharma continues to investigate the efficacy and safety of VILTEPSO in the confirmatory Phase 3 RACER53 trial. This study was initiated in October 2019 and is currently enrolling patients. The purpose of this Phase 3 randomized, double-blind, placebo-controlled trial is to evaluate the efficacy of viltolarsen on functional motor endpoints compared to placebo in DMD patients amenable to exon 53 skipping.

About VILTEPSO® (viltolarsen) injection

Prior to its approval in the U.S. in August 2020, VILTEPSO was granted Priority Review as well as Rare Pediatric Disease, Orphan Drug and Fast Track Designations. In March 2020, VILTEPSO was approved in Japan for the treatment of patients with DMD who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted with the SAKIGAKE designation, Orphan Drug designation, and designation of Conditional Early Approval System.

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.

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 registered trademark of the Nippon Shinyaku group of companies.

Contact

U.S. Media Contact:

media@nspharma.com

NEWS RELEASE

May 12, 2021

VILTEPSO® (viltolarsen) injection Long-Term Clinical Trial Data to be Presented at the PPMD 2021 Virtual Annual Conference

PARAMUS, NJ: May 12, 2021 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Shigenobu Maekawa), announced today that new, long-term efficacy and safety data (interim analysis at 109 weeks) from the open-label extension of a Phase 2 study of VILTEPSO® (viltolarsen) injection will be presented at the PPMD 2021 Annual Conference being held virtually from June 23-26.

“This VILTEPSO data we are presenting at the PPMD Annual Conference represents some of the longest treatment times with exon-skipping therapy reported to date,” said Leslie Magnus, MD, Vice President, Medical Affairs. “This data, compared to a matched historical control group, provides information on the potential long-term effectiveness and safety of VILTEPSO that is critical for healthcare professionals and Duchenne patients and their caregivers.”

In addition to this Phase 2 open-label extension study, NS Pharma continues to investigate the efficacy and safety of VILTEPSO in the confirmatory Phase 3 RACER53 trial. This study was initiated in October 2019 and is currently enrolling patients. The purpose of this Phase 3 trial is to confirm the clinical findings that were submitted under the Accelerated Approval pathway.

About VILTEPSO® (viltolarsen) injection

Prior to its approval in the U.S., VILTEPSO was granted Priority Review as well as Rare Pediatric Disease, Orphan Drug and Fast Track Designations. In March 2020, VILTEPSO was approved in Japan for the treatment of patients with DMD who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted with the SAKIGAKE designation, Orphan Drug designation, and designation of Conditional Early Approval System.

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.

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 registered trademark of the Nippon Shinyaku group of companies.

Contact

U.S. Media Contact:

media@nspharma.com

NEWS RELEASE

August 19, 2020

VILTEPSO™ (viltolarsen) injection Now Commercially Available in the U.S.\

PARAMUS, NJ: August 19, 2020 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Shigenobu Maekawa), announced today that VILTEPSO™ (viltolarsen) injection is now commercially available in the U.S. On August 12, 2020, the U.S. Food & Drug Administration (FDA) approved VILTEPSO for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping therapy.

To help navigate access and reimbursement with VILTEPSO, NS Pharma is offering services and resources to patients, caregivers and their healthcare providers through the NS Support™ program.

“The response by the DMD community to the FDA approval of VILTEPSO has been incredibly uplifting, and underscores NS Pharma’s responsibility that goes beyond the development of new and exciting treatment options,” said Tsugio Tanaka, President, NS Pharma, Inc. “Through our NS Support program, we will assist each patient and family obtain access to VILTEPSO, and help navigate obstacles related to location in the U.S. or financial circumstances.”

VILTEPSO is commercially available through a network of specialty distributors and specialty pharmacy providers. The distribution partners in this limited network were selected for their experience providing responsive, reliable service to healthcare providers and families with DMD.

Patients receiving treatment with VILTEPSO have the option and flexibility to receive

infusions at their home or at a hospital or treatment center. VILTEPSO is administered by a trained healthcare professional as an 80 mg per kg of body weight 60-minute weekly intravenous infusion.

VILTEPSO received an Accelerated Approval by the FDA based on an increase in dystrophin, a key protein for supporting muscle health. The continued approval of VILTEPSO may be  contingent on confirmation of a clinical benefit in a Phase 3 confirmatory trial.

About VILTEPSO™ (viltolarsen) injection

Prior to its approval in the U.S., VILTEPSO was granted Priority Review as well as Rare Pediatric Disease, Orphan Drug and Fast Track Designations. In March 2020, VILTEPSO was approved in Japan for the treatment of patients with DMD who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, VILTEPSO was granted with the SAKIGAKE designation, Orphan Drug designation, and designation of Conditional Early Approval System.

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

For additional safety information, please see the full Prescribing Information.

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 registered trademark of the Nippon Shinyaku group of companies.

Contact

U.S. Media Contact:

media@nspharma.com