RegulationOrphan Drug (JP), Rare Pediatric Disease (US), Conditional marketing approval (JP), SAKIGAKE (JP), Fast Track (US), Priority Review (CN), Accelerated Approval (US)

Structure

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Gene Sequence

Sequence Code 29547660

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Clinical Trials associated with Viltolarsen

CTR20213302 / Active, not recruitingPhase 2

一项在能走动和不能走动的杜氏肌营养不良症（DMD）男童患者中评价Viltolarsen与自然史对照相比的安全性、耐受性和有效性的II 期、开放标签研究

[Translation] A Phase II, open-label study evaluating the safety, tolerability, and efficacy of viltolarsen compared with natural history controls in ambulatory and nonambulatory boys with Duchenne muscular dystrophy (DMD)

主要目的：评价viltolarsen以每周80 mg/kg的剂量静脉给药(IV)治疗能走动和卧床的≥8岁DMD男孩的安全性和耐受性。次要目的：与自然病程对照相比，以每周80 mg/kg的剂量静脉给药viltolarsen48周治疗能走动和卧床的≥8岁DMD男孩的疗效。探索性目的：评价viltolarsen治疗对患者DMD健康相关生活质量的影响，评价DMD患者的咳嗽强度，评价DMD患者走动能力的保留情况，评价日常活动和睡眠-觉醒模式，以探索viltolarsen治疗对患者DMD的影响。

[Translation]

Primary objective: To evaluate the safety and tolerability of viltolarsen at a dose of 80 mg/kg intravenously (IV) weekly in the treatment of ambulatory and bedridden boys aged ≥8 years with DMD. Secondary objective: To compare the efficacy of viltolarsen at a dose of 80 mg/kg IV weekly for 48 weeks in the treatment of ambulatory and bedridden boys aged ≥8 years with DMD compared with a natural history control. Exploratory objective: To evaluate the effect of viltolarsen treatment on the health-related quality of life of patients with DMD, evaluate the cough intensity of patients with DMD, evaluate the retention of ambulatory ability of patients with DMD, evaluate daily activities and sleep-wake patterns, in order to explore the effect of viltolarsen treatment on patients with DMD.

Start Date17 Mar 2022

Sponsor / Collaborator

Hangzhou Tigermed Consulting Co., Ltd.

[+1]

NCT04956289 / CompletedPhase 2

A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls

This is a phase II, open-label study where weekly doses of 80 mg/kg viltolarsen is administered intravenously over a 48-week treatment period to ambulant and non-ambulant DMD patients over the age of 8 years.

Start Date01 Jul 2021

Sponsor / Collaborator

NS Pharma, Inc.

[+1]

NCT04687020 / Active, not recruitingPhase 4

Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)

The VILT-502 study is Non-interventional Study(United States)/Low-intervention Clinical Trial (Canada) of Viltolarsen administered intravenously once weekly for 10 years to boys with DMD who complete the NS-065/NCNP-01-202 study.

Start Date10 Jun 2021

Sponsor / Collaborator

NS Pharma, Inc.

100 Clinical Results associated with Viltolarsen

100 Translational Medicine associated with Viltolarsen

100 Patents (Medical) associated with Viltolarsen

Literatures (Medical) associated with Viltolarsen

01 Sep 2024·Drug Metabolism and Disposition

The Usefulness of Determining Plasma and Tissue Concentrations of Phosphorodiamidate Morpholino Oligonucleotides to Estimate Their Efficacy in Duchenne Muscular Dystrophy Patients

Article

Author: Mitamura, Rei ; Imai, Shunji ; Kameyama, Tsubasa ; Tone, Yuichiro ; Watanabe, Naoki ; Mori, Jumpei ; Yamada, Tetsuhiro ; Kusano, Kazutomi

Currently, four kinds of phosphorodiamidate morpholino oligomers (PMOs), such as viltolarsen, have been approved for the treatment of Duchenne muscular dystrophy (DMD); however, it is unclear whether human efficacy can be estimated using plasma concentrations. This study summarizes the tissue distribution of viltolarsen in mice and cynomolgus monkeys and evaluates the relationship between exposure and efficacy based on exon skipping. In the tissue distribution studies, all muscles in DMD-model mice showed higher concentrations of viltolarsen than those in wild-type mice and cynomolgus monkeys, and the concentrations in skeletal muscle were correlated with the exon-skipping efficiency in mice and cynomolgus monkeys. In addition, a highly sensitive bioanalytical method using liquid chromatography with tandem mass spectrometry shows promise for determining plasma concentrations up to a later time point, and the tissue (muscle)/plasma concentration ratio (Kp) in DMD-model mice was shown to be useful for predicting changes in pharmacodynamic (PD) markers in humans. Our results suggest that pharmacokinetic (PK)/PD analysis can be conducted by using the human PK profile or Kp values and skipping efficiency in DMD-model mice. This information will be useful for the efficient and effective development of PMOs as therapeutic agents. SIGNIFICANCE STATEMENT: We evaluated the relationship between the plasma or tissue concentrations and the efficiency of exon skipping for viltolarsen as an example phosphorodiamidate morpholino oligomers in the skeletal and cardiac muscle of mice and cynomolgus monkeys for pharmacokinetic/pharmacodynamic (PK/PD) analysis. The results suggest that PK/PD analysis can be conducted by using the human PK profile or tissue (muscle)/plasma concentration ratios and skipping efficiency in DMD-model mice.

01 Sep 2024·Advances in Therapy

Correction to: Evolving Role of Viltolarsen for Treatment of Duchenne Muscular Dystrophy

Author: Vincik, LeighAnn Y ; Kaye, Alan D ; Armstrong, Catherine J ; Lauck, Lillian V ; Howard, Jeffery T ; Staples, Abigail A ; Ahmadzadeh, Shahab ; Shekoohi, Sahar ; Dautel, Alexandra D ; McGregor, David

01 Jul 2024·BioDrugs

Mechanisms of Action of the US Food and Drug Administration-Approved Antisense Oligonucleotide Drugs

Review

Author: Rasmussen, Theodore P ; Bahal, Raman ; Bochanis, Adara ; Zhuo, Selena ; Manautou, José E ; Zhong, Xiao-Bo ; Sang, Angela

Antisense oligonucleotides (ASOs) are single stranded nucleic acids that target RNA. The US Food and Drug Administration has approved ASOs for several diseases. ASOs utilize three principal modes of action (MOA). The first MOA is initiated by base-pairing between the ASO and its target mRNA, followed by RNase H-dependent mRNA degradation. The second MOA is triggered by ASOs that occlude splice acceptor sites in pre-mRNAs leading to skipping of a mutation-bearing exon. The third MOA involves ASOs that sterically hinder mRNA function, often inhibiting translation. ASOs contain a variety of modifications to the sugar-phosphate backbone and bases that stabilize the ASO or render them resistant to RNase activity. RNase H-dependent ASOs include inotersen and eplontersen (for hereditary transthyretin amyloidosis), fomiversen (for opportunistic cytomegalovirus infection), mipomersen (for familial hypercholesterolemia), and tofersen [for amyotrophic lateral sclerosis (ALS)]. Splice modulating ASOs include nursinersen (for spinal muscular atrophy) and eteplirsen, golodirsen, viltolarsen, and casimersen (all for the treatment of Duchenne muscular dystrophy). In addition, a designer ASO, milasen, was used to treat a single individual afflicted with Batten disease. Since ASO design relies principally upon knowledge of mRNA sequence, the bench to bedside pipeline for ASOs is expedient compared with protein-directed drugs. [Graphical abstract available.].

News (Medical) associated with Viltolarsen

15 Oct 2024

·www.prnewswire.com

NS Pharma's Galactic53 Trial Data Is Published in Scientific Reports

PARAMUS, N.J., Oct. 15, 2024 /PRNewswire/ -- 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. Continue Reading 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. Both ambulatory and nonambulatory participants receiving viltolarsen experienced improved pulmonary function. Post this "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 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 Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com. US Media Contact: [email protected] SOURCE NS Pharma, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultOrphan DrugDrug ApprovalClinical Study

24 Sep 2024

·www.fiercebiotech.com

Wave surfs DMD success to regulators' doors, sending stock up

Shares in Wave Life Sciences opened up 39% at $7.40. Wave Life Sciences has met its goal in a Duchenne muscular dystrophy (DMD) study, positioning it to talk to regulators about accelerated approval while continuing to track patients through to the completion of the trial.The biotech reported 5.5% mean absolute unadjusted dystrophin in its analysis of nine ambulatory boys with exon 53 DMD after 24 weeks of treatment. Wave was targeting expression levels greater than 5% going into the readout. The 5.5% figure is derived from an analysis that excluded one non-ambulatory patient who had much lower levels of dystrophin after receiving Wave’s oligonucleotide WVE-N531.Wave’s result is in line with the data that supported accelerated approval of NS Pharma’s exon 53 DMD drug Viltepso. NS Pharma reported (PDF) mean dystrophin levels of 5.9% of normal by Week 25, which represented a 5.3% increase over baseline. However, the mean result in NS Pharma’s eight-patient study was driven by two outliers.Two patients had dystrophin levels above 10% of normal. One other patient topped 5%. Levels were below 4.1% in the five other patients, with three people expressing less than 3.1%. Wave saw levels of dystrophin exceed 5% of normal in six patients. Levels in three of the other patients ranged from 3.3% to 4.8%. Expression in the other, non-ambulatory Wave patient was 1% of normal. Wave also shared absolute muscle content adjusted dystrophin. WVE-N531 contains chemistry that is intended to enable high drug exposure in muscle, the tissue that is at the center of the health challenges faced by people with DMD. Wave reported mean muscle content-adjusted dystrophin expression of 9%.Again, the result reflected consistent performance across patients. Expression was 1.2% in the outlier at the bottom end of the range. Levels ranged from 6% to 13.9% in eight of the remaining patients. Only one ambulatory patient had an expression level—4.6%—below the 5% threshold targeted by Wave.The biotech generated the results in a trial that administered WVE-N531 every two weeks, the same schedule as Viltepso. Wave believes its data support monthly dosing, though. Patients had “a tremendous amount of drug in the muscle with a 61-day half life,” Wave CEO Paul Bolno, M.D., said on a call with analysts to discuss the data. If the oligonucleotide is there, the muscle should produce the protein. “Not only will we not lose efficacy, but we will continue to preserve that dynamic range of production of dystrophin,” Bolno said. “We know [monthly dosing] is a necessity for families and boys. We recognize the burden of having to go in for a weekly IV infusion.” Wave is switching patients in the trial to monthly dosing. The biotech expects to report 48-week data in the first quarter of 2025. Feedback from regulators on a pathway to accelerated approval is due around the same time.Shares in Wave opened up 39% at $7.40 on Tuesday morning.

Clinical ResultOligonucleotideClinical Study

10 Jul 2024

·www.prnewswire.com

NS Pharma Appoints New President to Oversee Next Phases of Orphan Drug Clinical Development and Commercialization

PARAMUS, N.J., July 10, 2024 /PRNewswire/ -- 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. Continue Reading "As president, it is my mission to maintain heightened levels of empathy at NS Pharma." Post this Yukiteru Sugiyama, PhD, NS Pharma President 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 Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com. US Media Contact: [email protected] SOURCE NS Pharma, Inc.

Executive ChangePhase 2Orphan Drug

100 Deals associated with Viltolarsen

External Link

KEGG	Wiki	ATC	Drug Bank
D11528	Viltolarsen	J05AB	DB15005

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Duchenne Muscular Dystrophy Exon 53 Skipping Mutation	JP	Nippon Shinyaku Co., Ltd.	25 Mar 2020

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Muscular Dystrophy, Duchenne	Phase 2	CN	Fuji Yakuhin Co., Ltd.	10 Aug 2021
Muscular Dystrophy, Duchenne	Phase 2	CN	Nippon Shinyaku Co., Ltd.	10 Aug 2021

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04956289 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	20	Viltolarsen	csbmfdfmgm(yzwsjjuhof) = ummsdlqztd nizurypkup (vwdkstkxht, txenlrbniz - hvsbblaaqz) View more	-	20 Aug 2024
NCT04060199 (RACER53, Corporate Publications) Manual	Phase 3	Muscular Dystrophy, Duchenne	77	viltolarsen	(xydbxpricu) = there was no statistically significant difference between the viltolarsen group and the placebo group. mxfhawnpyj (ofmrhnyopc ) View more	Negative	27 May 2024
				Placebo
NCT04956289 (MDA2024) Manual	Phase 2	Muscular Dystrophy, Duchenne dystrophin mutations	20	Viltolarsen 80 mg/kg/week	(tmukcpmbqq) = ccqzcemhoa ncgzrlwycm (ojthsjjiwc ) View more	Positive	03 Mar 2024
NCT03167255 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	16	NS-065/NCNP-01 (NS-065/NCNP-01 40mg/kg)	catuwqlkdc(tfxatbiuwg) = jmcahevebg iojxwyhezw (osojpjpnwi, sxlasdtvey - cjkktriogx) View more	-	18 Nov 2022
				NS-065/NCNP-01 (NS-065/NCNP-01 80mg/kg)	catuwqlkdc(tfxatbiuwg) = wykijphdgu iojxwyhezw (osojpjpnwi, gyaqytjfql - qbhtwibzhl) View more
NCT03167255 (Corporate Publications) Manual	Phase 2	Muscular Dystrophy, Duchenne Exon 53 Skipping	16	glucocorticoid treatment+Viltolarsen	(qptplkmtgd) = ycygxqazzy wevgommdsl (ifcqatyskq ) View more	Positive	24 Oct 2022
				glucocorticoid treatment	(qptplkmtgd) = pdaynuyfry wevgommdsl (ifcqatyskq ) View more
NCT02740972 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	16	NS-065/NCNP-01 (NS-065/NCNP-01 40mg/kg)	kbduqiwgqq(jvycodigbp) = yhgpvcifsl saoqmfrshd (yvbuvnewrt, xnkbopstlt - fnxrfifvvg) View more	-	12 Jul 2021
				NS-065/NCNP-01 (NS-065/NCNP-01 80mg/kg)	kbduqiwgqq(jvycodigbp) = cxnblxqcve saoqmfrshd (yvbuvnewrt, opkozjfbfo - jkbvwoduvz) View more
Corporate Publications Manual	Phase 2	Muscular Dystrophy, Duchenne Exon 53 Skipping	16	viltolarsen	(mzqsykjuld) = sfvwlojxda aksqtsqxbw (dnepgedptl ) View more	Positive	01 Jul 2021
				historical control	(mzqsykjuld) = npmtthaodu aksqtsqxbw (dnepgedptl ) View more
NCT02740972 (Pubmed \| JAMA Neurol)	Phase 2	Muscular Dystrophies	16	Viltolarsen 40 mg/kg	jmruxqrbnf(cxdwcrlyal) = ilmefozucn elckqcvtji (txfjdoypaz )	-	26 May 2020
				Viltolarsen 80 mg/kg	jmruxqrbnf(cxdwcrlyal) = vzlytjgjzm elckqcvtji (txfjdoypaz )
NCT02081625 (pubmed \| Sci Transl Med) Manual	Phase 1	Muscular Dystrophy, Duchenne Exon 53 Skipping	10	NS-065/NCNP-01	(rxbhjekfgx) = ansqxbwgvw uwrlmpgzoi (tmxylhhqlj ) View more	Positive	18 Apr 2018

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