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.
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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
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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
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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.
US Media Contact:
Selective JAK1 Inhibition is a 21st Century science. At NS Pharma, we are investigating this therapeutic approach for the treatment of rare diseases like eosinophilic granulomatosis with polyangiitis (EGPA).
With JAK1 inhibition, we consider two important variables: potency and target selectivity. These aspects are crucial when developing safe and effective therapies. Higher potency means a lower dose of the drug is needed for it to have its intended effect. High selectivity helps to ensure that the drug interacts only with its intended target, minimizing the risk of side effects.
The main challenge of working with JAK1 inhibitors to treat EPGA is that different therapies are needed to treat distinct stages of EGPA – no single therapy has been found to treat all stages of this condition.1 Scientists are still working to understand the causes of EGPA fully. This is complex, as they believe several factors are involved.
On the plus side, if JAK1 inhibition is effective, researchers hope it could reduce symptoms of EGPA regardless of disease stage. This would present a treatment opportunity for many patients with EGPA, rather than just a select few. Researchers are currently conducting a Phase 2 clinical trial to evaluate its safety and effectiveness in treating EGPA.
EGPA is extremely rare, affecting just about 0.0017% of the population.2 This makes accessing diagnosis and effective treatment challenging for patients. If JAK1 inhibition for EGPA is successful, this therapy could support healthier and happier futures for people with EGPA.
1 Eosinophilic Granulomatosis with Polyangiitis – Vasculitis Foundation. Accessed on July 19, 2024.https://www.vasculitisfoundation.org/education/vasculitis-types/eosinophilic-granulomatosis-with-polyangiitis/
1 Eosinophilic Granulomatosis with Polyangiitis (EGPA) – American Lung Association. Accessed on July 19, 2024. https://www.lung.org/lung-health-diseases/lung-disease-lookup/egpa
2 Vaglio A, Buzio C, Zwerina J. Eosinophilic granulomatosis with polyangiitis (Churg-Strauss): state of the art. Allergy. 2013 Mar;68(3):261-73. doi: 10.1111/all.12088
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)
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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:
July 10, 2024
NS Pharma Appoints New President to Oversee Next Phases of Orphan Drug Clinical Development and Commercialization
PARAMUS, NJ: July 10, 2024 – NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced that its Board of Directors has appointed a new President at its US headquarters. Yukiteru Sugiyama, Ph.D. replaces retiring president Tsugio Tanaka, MSc as of June 30, 2024.
Sugiyama received a doctorate degree in organic chemistry from Nagoya University in 1996. From 1996 until 2007, he worked in research and clinical development with Nippon Shinyaku in Japan, focusing on structural chemistry and blood cancer therapies. In 2007, he transitioned to the commercial division within the company.
In 2020, during the COVID-19 pandemic, Sugiyama transferred to NS Pharma in the US to work as assistant vice president overseeing commercial functions for VILTEPSO®(viltolarsen). In total, Sugiyama has worked for 28 years at Nippon Shinyaku with progressive levels of responsibility.
“Having served in both our science and patient focused fields, I have come to understand that having empathy for the patient is critical in the development of therapies to treat rare diseases,” Sugiyama explained. “As president, it is my mission to maintain heightened levels of empathy at NS Pharma, and to enhance transparency between all stakeholders.”
Sugiyama will lead the company through the next phases of clinical and commercial development for VILTEPSO and CAP-1002 (through a partnership with Capricor Therapeutics) – for the treatment of Duchenne muscular dystrophy (Duchenne). NS Pharma is also currently working on an exon 44 skipping therapy (Phase 2) and an exon 50 skipping therapy (Phase 1/2) for Duchenne, a selective JAK1 inhibition therapy for the treatment of eosinophilic granulomatosis with polyangiitis (EGPA), as well as several preclinical neurological therapies.
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:
media@nspharma.com
May 27, 2024
NS Pharma Shares Preliminary Results of Viltolarsen (NS-065 / NCNP-01)
Phase 3 Clinical Trial (RACER53 Study)
PARAMUS, NJ: May 27, 2024 – NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd., announced today that it has received preliminary analysis results from the global Phase 3 clinical trial (RACER53 study, NCT04060199) of NS-065/NCNP-01 (generic name: viltolarsen).
Viltolarsen was approved by the United States (US) Food and Drug Administration (FDA) in 2020 under the brand name VILTEPSO® – for the treatment of Duchenne muscular dystrophy (Duchenne) in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping – under the FDA accelerated approval pathway based on an increase in dystrophin production in skeletal muscle observed in treated patients. In the US, continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
The RACER53 Study is a randomized, double-blind, placebo-controlled, comparative study of 77 ambulatory boys with Duchenne. The study evaluated the efficacy and safety of an 80 mg/kg once weekly dosing of the treatment – versus placebo – for 48 weeks and was intended to serve as a confirmatory study.
The primary endpoint of the study was Time to Stand from Supine evaluated as velocity (rise/sec). The viltolarsen group showed a trend of increased velocity from baseline after treatment for 48 weeks. However, the placebo group also showed a trend of increased velocity, and there was no statistically significant difference between the viltolarsen group and the placebo group.
Preliminary safety results indicated that all adverse events that occurred under viltolarsen treatment were mild or moderate. There were no treatment emergent adverse events that led to discontinuation of the drug during the study.
“We are currently conducting further detailed data analyses and identifying factors that may have influenced the results (e.g. age, treatment period, and effect of
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concomitant drugs including glucocorticoid therapy),” said NS Pharma President Tsugio Tanaka, MSc. “Considering the results of prior clinical studies, we have confidence that viltolarsen can be a beneficial treatment for amenable patients with Duchenne.”
Specifically, in addition to the increase in dystrophin production in skeletal muscle that formed the basis of the FDA approval, a previously reported Phase 2, open- label, long-term extension study evaluated viltolarsen in 16 subjects between the ages of four and 10 with Duchenne amenable to exon 53 skipping. The study found that subjects receiving viltolarsen showed statistically significant improvements in the study’s primary endpoint of mean change from baseline for Time to Stand at week 205 as compared to a historical control group that was matched for key factors. In this study, treatment emergent adverse events were primarily mild or moderate. No study participants discontinued the study drug due to adverse events.
NS Pharma is currently conducting further detailed data analyses, including post- hoc data analyses, and plans to work closely with regulatory authorities to determine how to proceed based on the results of this analysis and in the best interests of patients. The company will report on additional analyses and discussions with the regulatory authorities at a later date.
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)
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:
April 4, 2024
NS Pharma Announces Research Alliance with MiNA Therapeutics to Develop Therapies for Rare Diseases of the Central Nervous System
PARAMUS, NJ: April 4, 2024 – NS Pharma, Inc. (NS Pharma) announced that its parent company, Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) – headquartered in Kyoto, Japan, entered into a joint research agreement with MiNA Therapeutics – headquartered in London, United Kingdom, to develop nucleic acid drugs for the potential treatment of intractable and rare diseases of the central nervous system. The agreement was facilitated by the NS Pharma Innovation Research Partnering (IRP) team, located in Cambridge, MA.
“We are incredibly proud of our IRP division and the work it is doing with the great team at MiNA Therapeutics,” said NS Pharma President Tsugio Tanaka. “We are excited about the innovative possibilities that come from working with small activating RNA (RNAa) therapeutics.”
Through this agreement, MiNA Therapeutics will provide Nippon Shinyaku RNAa therapeutics, which are oligonucleotides that can increase the transcription of a target gene. Nippon Shinyaku will have the right to exercise an option to exclusively research and develop pharmaceutical candidates derived from the alliance with MiNA Therapeutics by paying lump sum payments and milestones associated with the progress of research and development. After exercising the option, the company will pay milestones and royalties commensurate with the progress of the development and the sales of the product(s) after launch.
“Through this research alliance with MiNA Therapeutics, we will continue our efforts to apply our nucleic acid drug technology in the central nervous system,” Tanaka stated. “NS Pharma is committed to extending our technology as far as it can go to help improve the lives of patients with rare diseases.”
Nippon Shinyaku has been developing compounds in several therapeutic fields, including hematologic malignancies and intractable/orphan diseases. The company’s U.S. subsidiary is NS Pharma, headquartered in Paramus, NJ with additional offices in Cambridge.
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About MiNA Therapeutics
MiNA Therapeutics is the global leader in small activating RNA (RNAa) therapeutics. Harnessing innate mechanisms of gene activation, RNAa therapeutics are a revolutionary new class of medicines that can restore or boost normal function of genes and thereby protein-modulated pathways in cells. The company is advancing a proprietary pipeline of new medicines with an initial focus on genetic medicine, while collaborating with leading pharmaceutical companies to apply its technology platform across a broad range of other therapeutic areas.
Based on its unique know-how in RNA activation, MiNA Therapeutics is expanding the possibilities of RNA-based medicine. www.minatx.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.
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April 4, 2024
NS Pharma Announces Research Alliance with MiNA Therapeutics to Develop Therapies for Rare Diseases of the Central Nervous System
PARAMUS, NJ: April 4, 2024 – NS Pharma, Inc. (NS Pharma) announced that its parent company, Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) – headquartered in Kyoto, Japan, entered into a joint research agreement with MiNA Therapeutics – headquartered in London, United Kingdom, to develop nucleic acid drugs for the potential treatment of intractable and rare diseases of the central nervous system. The agreement was facilitated by the NS Pharma Innovation Research Partnering (IRP) team, located in Cambridge, MA.
“We are incredibly proud of our IRP division and the work it is doing with the great team at MiNA Therapeutics,” said NS Pharma President Tsugio Tanaka. “We are excited about the innovative possibilities that come from working with small activating RNA (RNAa) therapeutics.”
Through this agreement, MiNA Therapeutics will provide Nippon Shinyaku RNAa therapeutics, which are oligonucleotides that can increase the transcription of a target gene. Nippon Shinyaku will have the right to exercise an option to exclusively research and develop pharmaceutical candidates derived from the alliance with MiNA Therapeutics by paying lump sum payments and milestones associated with the progress of research and development. After exercising the option, the company will pay milestones and royalties commensurate with the progress of the development and the sales of the product(s) after launch.
“Through this research alliance with MiNA Therapeutics, we will continue our efforts to apply our nucleic acid drug technology in the central nervous system,” Tanaka stated. “NS Pharma is committed to extending our technology as far as it can go to help improve the lives of patients with rare diseases.”
Nippon Shinyaku has been developing compounds in several therapeutic fields, including hematologic malignancies and intractable/orphan diseases. The company’s U.S. subsidiary is NS Pharma, headquartered in Paramus, NJ with additional offices in Cambridge.
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About MiNA Therapeutics
MiNA Therapeutics is the global leader in small activating RNA (RNAa) therapeutics. Harnessing innate mechanisms of gene activation, RNAa therapeutics are a revolutionary new class of medicines that can restore or boost normal function of genes and thereby protein-modulated pathways in cells. The company is advancing a proprietary pipeline of new medicines with an initial focus on genetic medicine, while collaborating with leading pharmaceutical companies to apply its technology platform across a broad range of other therapeutic areas.
Based on its unique know-how in RNA activation, MiNA Therapeutics is expanding the possibilities of RNA-based medicine. www.minatx.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: