In rare disease research, collaboration is often the catalyst that transforms a promising idea into a life-changing treatment.
It’s this approach that allows us to develop life-changing medicines to treat rare diseases. As NS Pharma Vice President, Innovation Research Partnering Tatsuaki Morokata, Ph.D., puts it, “Collaborating with others acts as an accelerating system. A good collaboration enables us to do together what otherwise cannot be done.”
Tatsuaki Morokata, PhD, MBA
Vice President, Innovation Research Partnering, NS Pharma
Our expertise in pharmacology, clinical development, and regulatory affairs enables us to swiftly progress therapies from concept to clinic. Now, through our Innovation Research Partnering division, we’re forming strategic alliances with cutting-edge companies to turn pioneering science into real-world solutions.
One such partnership is with MiNA Therapeutics, the world leader in RNA activation (RNAa) drugs, revolutionary medicines that boost gene function. In April 2024, Nippon Shinyaku, Co., Ltd. (“Nippon Shinyaku”), the parent company of NS Pharma, entered a joint research agreement with MiNA Therapeutics to develop small activating RNAs (saRNAs) to treat rare and intractable diseases of the central nervous system (CNS) by RNAa.
The mission of NS Pharma is to push the boundaries of nucleic acid therapies1 with innovative drug discovery modalities. MiNA Therapeutics shares this goal, using pioneering RNAa medicines to target what was previously deemed “undruggable” by conventional approaches.
MiNA’s proprietary RNAa technology powers efficient saRNA synthesis and screening, while NS Pharma’s pharmacological expertise drives the drug development process. Co-creation combines these strengths, accelerating the delivery of high-quality candidates.
| The Challenge
Around 95% of rare diseases lack approved treatments.2 Conventional approaches, including gene therapy, often fail, particularly when targeting longer genes (>4.7 kb). |
The Strategy
RNAa therapeutics unlock possibilities for targets once deemed untreatable. We’re using this powerful technology to tackle severe neurological disorders. |
The Vision
Collaboration drives unique, effective solutions at unparalleled speed. By combining our strengths, we’re accelerating drug development to help patients sooner. |
Our alliance with MiNA focuses on enhancing gene expression in rare neurological disorders through cutting-edge RNAa therapeutics. The initial target of this collaboration is a severe neurodevelopmental disorder caused by a single defective gene. Although estimated to affect hundreds of thousands of people worldwide, there are currently no approved treatments for this condition.
This disorder is caused by mutations that result in the loss of function of a gene, leading to insufficient levels of the corresponding protein. saRNAs offer a novel way to address this by enhancing the affected gene’s expression, potentially restoring normal function.3 “MiNA’s agility and complementary expertise will enable us to bring this product to the clinic much faster, ultimately helping patients sooner,” Morokata emphasizes.
| 80%
Of rare diseases have a genetic cause2 Around 240 million people worldwide have a rare genetic disorder, with most lacking approved treatments. We’re expediting nucleic acid drug development to close this treatment gap. |
⬆2-10
Fold increase in gene expression4 RNAa therapeutics provide a game-changing opportunity to treat loss-of-function genetic disorders. These drugs restore physiological protein levels by substantially increasing endogenous gene expression. |
⬇50%
Decrease in candidate development timeline The complementary combination of MiNA’s systematic drug discovery approach and NS Pharma’s efficient clinical development process drastically reduces the time needed to produce a clinical candidate. |
With RNAa technology, we can upregulate genes underlying loss-of-function disorders that cause developmental, motor, and behavioral delays, offering solutions where other therapies have failed.
RNAa therapeutics directly tackle loss-of-function disorders by boosting natural gene expression, increasing it by 2 to 10 times.4 They uniquely activate challenging gene types, including large genes and those silenced epigenetically.
By activating endogenous gene expression, RNAa drugs achieve more physiologically relevant protein expression levels. This mitigates risks associated with overexpression or the permanent introduction of foreign genetic material. The highly specific nature of RNAa therapeutics also minimizes the potential for off-target effects.
Combining NS Pharma’s robust development pipeline with MiNA’s cutting-edge RNAa technology positions us to make significant strides in areas where traditional therapies have struggled.
As we look to the future, our collaboration with MiNA Therapeutics represents more than just a strategic partnership—it embodies our commitment to advancing the treatment of rare diseases through co-creation and innovation.
We are committed to finding solutions for the unmet medical needs of patients worldwide. “By combining our strengths and resources, we can compete with very large pharmaceutical companies, particularly in the rare disease space,” Morokata explains. With our deep expertise and patient-centric focus, we are eager to partner with like-minded organizations to bring groundbreaking treatments to those in need.
Together, we can make a difference.
>Partner with us to advance rare disease treatment<
References
At NS Pharma, leadership is more than just a role—it’s a commitment to empowering teams, fostering innovation, and building strong partnerships that push the boundaries of rare disease treatments.
The newest member of our executive team, Don Foy, embodies this approach. With over 24 years of pharma experience—including 17 in leadership roles—Don brings a wealth of expertise in sales, marketing, market access, and commercial strategy to his role as head of Commercial. This expertise is grounded in a leadership style that prioritizes trust, collaboration, and professional development.
Don Foy, Head of Commercial, NS Pharma
Trust as the Cornerstone of Leadership
Shinrai is the Japanese word for trust, a core value that shapes every aspect of our work at NS Pharma. For Don, building trust through integrity is not just a professional value but a personal one, rooted in lessons he learned from his father.
He remembers how his father, an association executive, led with empathy, care, and a deep commitment to his team’s well-being: “I observed how he treated and led people, and when he retired, the accolades that he received from his staff were quite overwhelming,” Don recalls.
This early lesson has shaped Don’s belief that great leadership comes from genuinely caring for those you lead: “Trust is the foundation for everything, be it internal or external relationships,” he explains. “As a leader, you have to demonstrate care and concern for the people you serve. They also need to see your vulnerabilities. The more you can do that, the more you connect and build trust with your people.”
For Don and the rest of NS Pharma’s executive team, trust built on transparency and integrity is the critical element that fosters collaboration, enhances performance, and creates lasting partnerships. Shinrai isn’t just an internal value at NS Pharma—it shapes our relationships with healthcare professionals, industry partners, and the patients we serve.
[Don with his father]
Collaboration accelerates drug development. At NS Pharma, the Commercial team is pivotal in maintaining strategic partnerships that allow us to bring life-changing therapies to patients with rare diseases.
A prime example is our ongoing collaboration with Capricor Therapeutics to launch deramiocel (CAP-1002), a new cell therapy product for Duchenne muscular dystrophy. Don’s team ensures seamless execution at every step of the partnership—from planning to launch. This starts with aligning internal and external stakeholders through open communication and mutual accountability.
According to Don, successful partnerships hinge on three fundamental principles:
This hands-on approach enables NS Pharma’s Commercial team to be proactive, adapt swiftly to meet challenges, and ensure complete alignment—keeping the focus on delivering safe and effective therapies to patients as quickly as possible.
Our extensive commercial infrastructure is one of our greatest strengths, providing an invaluable advantage to our partners. With cross-continental reach and a finely tuned distribution network, we deliver an efficient, scalable platform to drive sales and maximize market reach.
The Commercial team supports this infrastructure, with senior directors overseeing patient services, market access, sales, marketing, and operations. “Our senior directors coordinate with consultants, healthcare professionals, patients, payers, advocacy organizations, and other groups, keeping us aligned to ensure we successfully accomplish our commercial objectives,” Don explains.
Through NS Pharma’s strategic leadership, cross-departmental coordination, and robust international sales infrastructure, our partners can confidently focus on innovation, knowing they have a dependable partner for commercialization success.
>Partner with us to advance rare disease treatment<
Shaping the Future Together
Under Don’s leadership, the Commercial team continues to build strong partnerships, empower future leaders, and drive the development of groundbreaking therapies that make a real difference in the lives of patients with rare diseases. “To be able to manage Viltepso, a four-year-old product in the marketplace, and the opportunity to launch an exciting new product in cell therapy,” remarks Don. “What an exciting time to be at NS Pharma.”
We couldn’t agree more. Whether you’re looking to partner with NS Pharma or join our growing team, we invite you to be part of the exciting breakthroughs shaping the future of rare disease treatment. Together, guided by the principles of Shinrai and a shared commitment to improving patient outcomes, we’re building a future that brings hope and healing to those who need it most.
Transform the future of rare disease treatment with us.
KYOTO, Japan, January 27, 2025 – Nippon Shinyaku Co., Ltd. (Headquarters: Kyoto, Japan, President: Toru Nakai) announced that Nippon Shinyaku and AB2 BIO Ltd. (Headquarters: Lausanne, Switzerland, CEO: Djordje Filipovic) have entered into an option agreement for the rights of commercialization of Tadekinig alfa for the treatment of NLRC4 mutation and XIAP deficiency in the U.S. by Nippon Shinyaku. AB2 BIO Ltd. is developing Tadekinig alfa for the treatment of NLRC4 mutation and XIAP deficiency in the U.S. and Europe.
NLRC4 mutation and XIAP deficiency are types of rare and serious hereditary autoinflammatory diseases, and elevated levels of interleukin 18 (IL-18) cause a variety of inflammatory symptoms. Most patients show symptoms in infancy and the inflammation persists throughout their lives. The number of patients with both NLRC4 mutation and XIAP deficiency is small, and the long-term prognosis is not clear, but these are serious diseases that make it difficult for many patients to reach adulthood. There are no drugs on the market for the treatment of either of these diseases, and new effective treatments are needed.
Tadekinig alfa is a recombinant human IL-18 binding protein, and it suppresses IL-18 related immune and inflammatory responses by specifically binding to excessively produced IL-18. Tadekinig alfa has received Orphan Drug Designation, Breakthrough Therapy Designation and Rare Pediatric Disease Designation in the U.S., and Orphan Drug Designation in Europe. In addition, AB2 BIO Ltd. has completed Phase III trials in North America and Europe, and is currently preparing for submission of a Biologics License Application (BLA) in the U.S.
After Nippon Shinyaku exercises its option rights and AB2 BIO Ltd. obtains the BLA approval in the U.S., NS Pharma, Inc. (New Jersey, USA, President: Yukiteru Sugiyama), a wholly owned subsidiary of Nippon Shinyaku, will market Tadekinig alfa.
Nippon Shinyaku is working to develop new treatments for intractable and rare diseases and aims to be a company that is trusted by society through the creation of unique drugs.
About Nippon Shinyaku
Based on Nippon Shinyaku’s business philosophy, “Helping people lead healthier, happier lives,” we aim to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness. Please visit our website (https://www.nippon-shinyaku.co.jp/english/) for products or detailed information.
About AB2 BIO Ltd.
AB2 Bio is a biotech company developing innovative therapies for the treatment of severe systemic autoinflammatory diseases and conditions driven by IL-18. The company is advancing Tadekinig alfa in a wide range of IL-18 mediated hyperinflammatory diseases and conditions, including rare orphan diseases with high unmet medical needs, at clinical and pre-clinical phase.
AB2 Bio was founded in 2010 and is headquartered in the Innovation Park at the Ecole Polytechnique Fédérale de Lausanne (EPFL), Switzerland. More information can be found on http://www.ab2bio.com.
Contact
Corporate Communications Dept., Nippon Shinyaku Co., Ltd.
e_mail_kouhou@po.nippon-shinyaku.co.jp
KYOTO, Japan, January 14, 2025 – Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters: Kyoto; President, Toru Nakai) announced that Nippon Shinyaku and REGENXBIO Inc. (REGENXBIO; Headquarters: Rockville, Maryland, USA; CEO: Curran M. Simpson, NASDAQ: RGNX) have entered into an exclusive license agreement for RGX-121 and RGX-111 for the treatment of Mucopolysaccharidosis II and I (MPS II and I), respectively. Under the terms of the licensing agreement, Nippon Shinyaku will receive exclusive commercialization rights in the United States (U.S.) and exclusive development and commercialization rights in Asia including Japan, and REGENXBIO will retain commercial rights in the rest of the world. After approval of the Biologics License Application in the U.S., RGX-121 and RGX-111 will be marketed by NS Pharma, Inc. (New Jersey, USA; President: Yukiteru Sugiyama), a wholly owned subsidiary of Nippon Shinyaku, in the U.S.
Mucopolysaccharidosis (MPS) is a congenital metabolic disorder in which a specific enzyme is defective or inactive due to genetic factors, resulting in the accumulation of specific glycosaminoglycans (“GAGs”), a type of mucopolysaccharide, and is classified into several forms according to the gene responsible for the disease. The accumulation of GAGs causes systemic organ damage, including the central nervous system, in severe cases, and the prognosis is 10 to 15 years of age. Currently, there is no curative treatment for the disease, and the mainstay of treatment is the suppression of progression through enzyme replacement therapy.
RGX-121 and RGX-111 are first-in-class, investigational gene therapies for the treatment of MPS II and MPS I, respectively. For RGX-121, REGENXBIO has received Fast Track Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy Designation, and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA). Submission of a rolling Biologics Licensing Application (BLA) for RGX-121 is ongoing. For RGX-111, REGENXBIO has received Fast Track Designation, Rare Pediatric Disease Designation, and Orphan Drug Designation from the FDA and has been conducting a Phase I/II trial in the U.S., Brazil, and Israel.
Nippon Shinyaku is focusing on the field of intractable, rare disorders. We expect that RGX-121 and RGX-111 will contribute to the treatment of patients suffering from MPS.
Entry into force of this agreement is subject to completion of review under the Hart-Scott-Rodino (HSR) Antitrust Reform Act in the U.S.
About MPS II
MPS II, also called Hunter syndrome, is a rare, congenital metabolic disorder caused by a deficiency in the iduronate-2-sulfatase, one of the enzymes that degrades glycosaminoglycans (GAGs). The lack of this enzyme causes heparan sulfate and dermatan sulfate to accumulate in all body tissues. When it develops, it causes systemic symptoms such as growth retardation, osteoarticular symptoms, valvular heart disease, and central nervous system disorders. The current treatment for this disorder is palliative care, enzyme replacement therapy (ERT) and bone marrow and stem cell transplantation.
About MPS I
MPS I is a congenital dysmetabolic disease caused by congenital deficiency or reduced activity of alpha-L-isuronidase, one of the enzymes that degrades glycosaminoglycans (GAGs) in steps, resulting in intracellular accumulation of dermatan sulfate and heparan sulfate and damage to multiple organs. MPS I is classified into two types: severe, with intellectual disability, which is also called Hurler’s syndrome and mild.
About HSR Antitrust Reform Act of 1976
The HSR Act provides that before certain size of mergers, tender offers or other acquisition transactions (including certain grants of executive compensation) may be completed, both parties must file notification forms with the Antitrust Division of the Department of Justice and the Federal Trade Commission, and a statutory waiting period expires or is terminated. The HSR Act has been expanded for pharmaceutical licensing agreements in 2013.
About Nippon Shinyaku
Based on Nippon Shinyaku’s business philosophy, “Helping people lead healthier, happier lives,” we aim to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness.
Please visit our website (https://www.nippon-shinyaku.co.jp/english/) for products or detailed information.
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. For more information, please visit www.regenxbio.com.
Contact
Corporate Communications Dept., Nippon Shinyaku Co., Ltd.
e_mail_kouhou@po.nippon-shinyaku.co.jp
PARAMUS, NJ: January 13, 2025 – NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced that the National Center of Neurology and Psychiatry (NCNP) has published in the journal Cell Reports Medicine the results of an investigator-initiated clinical trial (first in human trial) of NS-089/NCNP-02 (brogidirsen), which is being developed by Nippon Shinyaku for the treatment of Duchenne muscular dystrophy (Duchenne). A global Phase II study of NS-089/NCNP-02 is being conducted by Nippon Shinyaku and NS Pharma. Please check the press release from NCNP for a summary of the paper. Additionally, the paper is available under open access here.
NS-089/NCNP-02 is an antisense oligonucleotide co-discovered by Nippon Shinyaku and NCNP and is expected to be a therapeutic drug for Duchenne patients who have dystrophin gene mutations amenable to exon 44 skipping.
NS Pharma has been actively working to develop agents for the treatment of intractable and rare diseases, with a goal of launching treatments for patients with Duchenne as soon as possible.
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 group of companies. For more information, please visit nspharma.com.
U.S. Media Contact:
media@nspharma.com
PARAMUS, NJ: November 26, 2024 – NS Pharma, Inc. (NS Pharma) announced a change of leadership within its Commercial division. Effective September 9, 2024, Donald Foy – who had previously served as national sales director – was appointed to the role of vice president, Commercial. Jennifer Tamberino – who had been Regional Business Director, East, National Sales – was promoted to backfill Foy’s former position as national sales director.
“It is with incredible excitement that we announce Don’s promotion to lead our sales, marketing, market access, operations and patient services departments as our new head of Commercial at NS Pharma,” said NS Pharma President Yukiteru Sugiyama, Ph.D. “With Don and Jennifer at the helm, we are well- positioned to execute our plans for growth in the rare disease space in the United States. Our new leadership structure is designed to foster industry collaboration and company innovation from the top down.”
Foy replaces outgoing executive Gardner Gendron, who held the position for five years and oversaw the launch and commercialization of viltolarsen (VILTEPSO) for the treatment of Duchenne muscular dystrophy (Duchenne), a rare muscle-wasting neurological disorder.
Prior to joining NS Pharma, Foy served in leadership roles for 17 years in the pharmaceutical industry, with more than 24 years of experience both in sales and in cross-functional positions supporting a diverse set of stakeholders.
Tamberino has a four-year tenure with NS Pharma demonstrating effective leadership and business acumen.
NEWS RELEASE
NS Pharma currently has one commercial product, VILTEPSO, with several others in its pipeline for the treatment of Duchenne and, separately, the treatment of eosinophilic granulomatosis with polyangiitis (EGPA), a form of vasculitis. It is also slated to market deramiocel (CAP-1002), which is being developed by Capricor Therapeutics for the treatment of Duchenne cardiomyopathy.
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 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 group of companies. For more information, please visit nspharma.com.
U.S. Media Contact:
KYOTO, Japan and Durham, North Carolina, USA, November 13, 2024 – Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters: Kyoto; President, Toru Nakai) and Atsena Therapeutics, Inc. (Atsena; Headquarters: Durham, North Carolina, USA, Chief Executive Officer (CEO) : Patrick Ritschel) have entered into an exclusive license agreement for the commercialization of ATSN-101 in the territory of the U.S. and for the development and commercialization of ATSN-101 in the territory of Japan for advancing Atsena’s first-in-class, investigational gene therapy ATSN-101 for Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1).
Under the terms of the licensing agreement, Nippon Shinyaku will receive exclusive commercial rights in the U.S. and Japan, and Atsena will retain commercial rights in the rest of the world. ATSN-101 will be marketed by NS Pharma, Inc. (New Jersey, USA, President: Yukiteru Sugiyama), a wholly owned subsidiary of Nippon Shinyaku in the U.S.
Atsena will receive an upfront payment, additional milestone payments, downstream royalties based on sales and will be reimbursed as it continues development work on ATSN-101, including an anticipated global pivotal trial.
ATSN-101 is a first-in-class, investigational gene therapy for the treatment of LCA1. Atsena has received Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy Designation and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for ATSN-101. In the event Atsena receives a Rare Pediatric Disease Priority Review Voucher (PRV) in connection with the approval of the Biologic
NEWS RELEASE
License Application for ATSN-101, Atsena shall own and retain all rights, title and interest in such PRV.
“ATSN-101 provides a potential, innovative treatment in an area where no approved solutions currently exist,” said Nippon Shinyaku President Toru Nakai. “We are excited by the opportunity of this novel ocular gene therapy and our collaboration with Atsena and its groundbreaking science.”
“This collaboration creates a path to accelerate the development of ATSN-101 and validates Atsena’s pioneering technology and development capabilities. We anticipate this will be the first of many ocular gene therapy treatments from our clinical portfolio to come,” said Patrick Ritschel, CEO of Atsena Therapeutics. “We look forward to working with Nippon Shinyaku as we advance ATSN-101 into a pivotal trial and potential approval to provide an innovative solution to patients and families affected by LCA1 around the world.”
About GUCY2D-associated Leber congenital amaurosis (LCA1)
LCA1 is a monogenic eye disease that disrupts the function of the retina. It is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. LCA1 is one of the most common forms of LCA and there are no approved treatments for it.
About ATSN-101
ATSN-101 is an investigational, subretinal AAV5 gene therapy being evaluated in an ongoing Phase I/II clinical trial for LCA1. At 12 months post-treatment, ATSN-101 has demonstrated durable, clinically meaningful improvements in vision at the high dose and is well-tolerated. Results from this trial were recently published in The Lancet.
About Rare Pediatric Disease Designation
The Rare Pediatric Disease Designation is granted by the FDA for promoting the development of new drugs for rare diseases that occur in the U.S. before the age of 18 and affect fewer than 200,000 people. With this designation, the product will be eligible to receive a Priority Review Voucher upon approval that could be used to advance another program.
About Regenerative Medicine Advanced Therapy Designation
The Regenerative Medicine Advanced Therapy (RMAT) Designation is an FDA- designated system for advanced regenerative medicine therapies targeted at developing serious diseases that have shown certain effect in clinical trials based on the 21st Century Cures Act in the U.S. Companies with RMAT designations are given the opportunity for priority review and accelerated approval for the product.
About Orphan Drug Designation
The Orphan Drug Designation is provided for orphan drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. With orphan designation, the FDA grants a seven-year market exclusivity for the product and provides certain incentives such as tax credits towards the cost of development, upon approval.
About Nippon Shinyaku
Based on Nippon Shinyaku’s business philosophy, “Helping people lead healthier, happier lives,” we aim to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness.
Please visit our website (https://www.nippon-shinyaku.co.jp/english/) for products or detailed information.
About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. In an ongoing Phase I/II clinical trial, the company is evaluating ATSN-101 for LCA1, one of the most common causes of blindness in children. In another ongoing clinical trial, the company is evaluating ATSN-201 for X-linked retinoschisis (XLRS), a genetic condition affecting boys and men that is typically diagnosed in childhood. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit https://atsenatx.com/.
Nippon Shinyaku contact:
e_mail_kouhou@po.nippon-shinyaku.co.jp
Atsena contacts:
Media: tplohoros@6degreespr.com
Business: info@atsenatx.com
Exon-skipping is a therapeutic approach to helping people with genetic disorders produce shorter, but still usable, proteins by “skipping over” the defective parts of genes. While this type of science has been investigated since the late 1970s, much is still unknown about its potential efficacy.*
NS Pharma is investigating how far we can take this innovative therapy when it comes to helping people with Duchenne muscular dystrophy (Duchenne), a genetic muscle wasting disease. Specifically, we are investigating exon-skipping in patients with mutations amenable to skipping of exons 44, 45, 50, 51, 53 and 55.
Here are some commonly asked questions about exon-skipping for the treatment of Duchenne:
Genetic testing can help you find out if you have Duchenne and, if so, which genetic mutation is the cause. Performed by a healthcare provider, this simple test can identify the specific mutation that is inhibiting dystrophin production in the body. Here’s a discussion guide you can use to help you through these important conversations with your doctor.
Not all genetic mutations causing Duchenne can be treated with exon-skipping therapy at this time. This exon-finder tool can tell you if exon-skipping therapy is an option for your specific mutation. This tool is not intended to replace discussions with your doctor or genetic counselor.
Various treatments are used to manage Duchenne symptoms, including steroids and physical therapy. However, these treatments do not increase dystrophin. Exon-skipping is the only option that can increase dystrophin in eligible people with Duchenne. Gene therapy is another option to increase micro-dystrophin in people with Duchenne.
No, exon-skipping is not a cure for Duchenne, but it may halt or delay symptom progression for many people with Duchenne.
*https://pubmed.ncbi.nlm.nih.gov/30171532/
US-NSPCO-0069
PARAMUS, NJ: October 15, 2024 – NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced that the Galactic53 study of VILTEPSO® (generic name: viltolarsen) – a treatment for Duchenne muscular dystrophy (Duchenne) – has been published in the journal Scientific Reports. The paper, “Safety and efficacy of viltolarsen in ambulatory and nonambulatory males with Duchenne muscular dystrophy”, contains data from an open-label multicenter study, which was the first to evaluate the effects of viltolarsen on pulmonary function in participants with Duchenne.
Ten ambulatory and ten nonambulatory participants ages eight years and older – with a confirmed deletion of the dystrophin gene that could be treated by exon 53 skipping
– received 80 mg/kg of the drug intravenously once weekly for 48 weeks. Safety was evaluated as the primary endpoint, and pulmonary and motor function were evaluated as secondary efficacy endpoints. The pulmonary endpoints were compared to natural history data with matched patient backgrounds as a control group.
“We’re encouraged by these results for patients with exon 53 skip-amenable mutations,” explains NS Pharma Vice President Medical Affairs & Pharmacovigilance Leslie Magnus, MD, who co-authored the study. “We’re especially glad to report positive data was seen in both nonambulatory and ambulatory patients.”
All treatment-emergent adverse events were mild or moderate. Four were considered treatment-related, and no participants discontinued. The side effect profile seen in this study is consistent with that reported in previous studies, and the treatment was well tolerated.
Both ambulatory and nonambulatory participants receiving viltolarsen experienced improved pulmonary function with higher percent predicted forced vital capacity (FVC%p) and higher peak cough flow (PCF) at Week 49 compared with controls. Ninety (90)% (nine / ten) of nonambulatory participants receiving viltolarsen had an increase or stabilization in FVC%p from baseline, and 60% (six / ten) of participants maintained FVC%p values >50% at Week 49, which is the recommended threshold
NEWS RELEASE
for needing cough assist and nighttime ventilation interventions. For ambulatory participants treated with viltolarsen, 90% (nine / ten) of viltolarsen treated participants had an increase or stabilization in FVC%p from baseline, and all treated participants maintained FVC%p values >50% at Week 49.
Viltolarsen treated participants, including those who were nonambulant, also showed stabilized arm strength and mobility as assessed by performance of upper limb (PUL) scores over the 48-week treatment period.
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 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 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 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 NEWS RELEASE Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com.
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PARAMUS, NJ: September 10, 2024 – NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced that the Food and Drug Administration (FDA) has granted rare pediatric disease designation to NS- 050/NCNP-03 which is being developed for the treatment of Duchenne muscular dystrophy (Duchenne). The FDA’s rare pediatric disease designation is granted for treatments of serious or life-threatening diseases that affect children under the age of 18 and fewer than 200,000 patients in the United States.
“The journey from first symptom to diagnosis and finally treatment can be long and challenging for patients with rare diseases and their caregivers,” said NS Pharma President Yukiteru Sugiyama, Ph.D. “We are grateful for this designation which can help us accelerate the development of this therapy for Duchenne.”
Duchenne is a progressive muscle wasting disease caused by a deficiency of the dystrophin protein. It leads to weakness of skeletal, cardiac and pulmonary muscles. There are many types of genetic mutations that can cause Duchenne, and NS- 050/NCNP-03 is being developed to treat patients with confirmed gene mutations amenable to exon 50 skipping therapy.
NS-050/NCNP-03 is an antisense oligonucleotide co-discovered by the National Center of Neurology and Psychiatry (NCNP) and Nippon Shinyaku. NS-050/NCNP- 03 skips part of the genetic information of the dystrophin gene and produces a functional dystrophin protein with a slightly shorter chain length, which is expected to have the effect of suppressing muscle function deterioration.
NS Pharma is working to develop products for patients with rare diseases. We are preparing a Phase 1 / 2 study to evaluate the safety and efficacy of NS-050/NCNP-03 in patients with Duchenne in Japan and the United States.
About Duchenne Muscular Dystrophy (Duchenne)
NEWS RELEASE
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 NEWS RELEASE Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com.
US Media Contact: