National Center of Neurology and Psychiatry News

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|||||||||| Review, Journal: Clinical applications of exon skipping antisense oligonucleotides in neuromuscular diseases. (Pubmed Central) - Jun 5, 2025
Many of these newly developed exon skipping ASOs have been studied in clinical trials in DMD patients, and early findings suggest clear improvements in molecular efficacy compared to the earlier version of ASOs, although the safety track record may not be the same as the first generation compounds. Here, we summarise the recent preclinical and clinical developments of ASOs and discuss the future challenges of exon skipping therapies for DMD and other neuromuscular diseases.

||||||||||  brogidirsen (NS-089/NCNP-02) / Nippon Shinyaku, National Center of Neurology and Psychiatry
Trial completion date, Trial primary completion date:  NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) -  Jun 5, 2025
P2, N=20, Recruiting,  Sponsor: NS Pharma, Inc.
Here, we summarise the recent preclinical and clinical developments of ASOs and discuss the future challenges of exon skipping therapies for DMD and other neuromuscular diseases. Trial completion date: Nov 2025 --> Sep 2026 | Trial primary completion date: Nov 2025 --> Sep 2026

|||||||||| brogidirsen (NS-089/NCNP-02) / Nippon Shinyaku, National Center of Neurology and Psychiatry
Brogidirsen -The First Dual-Targeting Exon 44 Skipping Drug for Duchenne Muscular Dystrophy Safety, Tolerability, and High Dystrophin Restoration in Long-Term Clinical Studies (Poster Hall) - Apr 10, 2025 - Abstract #ASGCT2025ASGCT_1638;
P1/2, P2
The extension study is ongoing, and a Phase 2 trial in the USA (NCT05996003), sponsored by NS Pharma, Inc., is also in progress to further evaluate its safety and efficacy. Disease Focus of Abstract:Muscular Dystrophy (all forms)

|||||||||| Viltepso (viltolarsen) / Nippon Shinyaku, National Center of Neurology and Psychiatry
Evaluation of the efficacy of Viltolarsen to Duchenne muscular dystrophy using muscular imaging (Poster venue (Osaka International Convention Center 3F Event Hall)) - Mar 23, 2025 - Abstract #JSNE2025JSNE_1298;
This is the first report of muscle mass evaluation using CT after viltolarsen treatment. In both cases 1 and 2, the %MVI was maintained compared to the control, suggesting the effectiveness of viltolarsen.

|||||||||| brogidirsen (NS-089/NCNP-02) / Nippon Shinyaku, National Center of Neurology and Psychiatry
Dystrophin restoration via exon-skipping modulates muscle lipidome in Duchenne muscular dystrophy (Venue 12 (Osaka International Convention Center 7F Conference Rooms 701-702)) - Mar 23, 2025 - Abstract #JSNE2025JSNE_349;
[Methods] We utilized biopsied skeletal muscles collected from 6 DMD patients enrolled in the Phase 1/2 trial of Brogidirsen, an exon 44 skipping drug...[Conclusions] We demonstrate that specific alterations of lipidome are seen in DMD, which could be regulated by dystrophin expression. Further research studying the relationship between the permeability of myofiber plasma membranes and PE alterations might pave the way for the development of membrane reinforcement therapy for the disease.

|||||||||| Viltepso (viltolarsen) / Nippon Shinyaku, National Center of Neurology and Psychiatry
Retrospective data, Journal: Safety and efficacy of viltolarsen treatment in patients with Duchenne muscular dystrophy: A retrospective study with 3-year follow-up. (Pubmed Central) - Feb 23, 2025
Viltolarsen was a safe and effective treatment for patients with DMD, and the findings highlighted the importance of once-weekly and uninterrupted viltolarsen treatment. The distinct safety and efficacy of viltolarsen require further investigation using a large number of cases and a long follow-up period.

|||||||||| Journal: Assessment of Phosphorodiamidate Morpholino Oligomer Treatment Patterns for Patients with Duchenne Muscular Dystrophy: A MarketScan Claims Analysis. (Pubmed Central) - Jan 30, 2025
Nonetheless, the observed persistence may have been underestimated given shortcomings of claims data and payer coverage considerations. Caution should be exercised when inferring treatment effectiveness or tolerability based on observed treatment patterns from claims data alone for weight-based, infused PMO treatments.

|||||||||| brogidirsen (NS-089/NCNP-02) / Nippon Shinyaku, National Center of Neurology and Psychiatry
P1/2 data, Journal: Phase 1/2 trial of brogidirsen: Dual-targeting antisense oligonucleotides for exon 44 skipping in Duchenne muscular dystrophy. (Pubmed Central) - Jan 12, 2025
P1/2
These promising results warrant a subsequent trial for DMD. This study was registered at ClinicalTrials.gov (NCT04129294).

|||||||||| Review, Journal: Antisense oligonucleotide as novel therapies for neurogenetic disorders (Pubmed Central) - Jan 12, 2025
This review has elaborated the mechanism of ASO therapies, including basic rationales, modifications, side effects and delivery routes. It also systemically summarized the FDA-approved ASO therapeutics and their applications for various neurological disorders, and discussed the limitations and challenges the real-world market may face and issues genetic counselor should take into consideration in the near future.

|||||||||| Viltepso (viltolarsen) / Nippon Shinyaku, National Center of Neurology and Psychiatry
Journal: The usefulness of determining plasma and tissue concentrations of phosphorodiamidate morpholino oligonucleotides to estimate their efficacy in Duchenne muscular dystrophy patients. (Pubmed Central) - Aug 14, 2024
Significance Statement We compare that plasma and tissue concentrations with the efficiency of exon skipping for viltolarsen as an example phosphorodiamidate morpholino oligomers in skeletal and cardiac muscle of mice and cynomolgus monkeys for pharmacokinetics/pharmacodynamics (PK/PD) analysis. Our results suggest that PK/PD analysis can be conducted by using the human PK profile or Kp values and skipping efficiency in DMD model mice.

|||||||||| FDA event, Review, Journal: Mechanisms of Action of the US Food and Drug Administration-Approved Antisense Oligonucleotide Drugs. (Pubmed Central) - Jul 14, 2024
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.

|||||||||| FDA event, Review, Journal: Comprehensive review of adverse reactions and toxicology in ASO-based therapies for Duchenne Muscular Dystrophy: From FDA-approved drugs to peptide-conjugated ASO. (Pubmed Central) - Jul 10, 2024
This article provides a comprehensive review and discussion of the available data pertaining to adverse reactions and toxicology associated with FDA-approved ASO drugs for DMD. Furthermore, it offers insights into the emerging category of peptide-conjugated ASO drugs those are clinical and preclinical trials, shedding light on their potential benefits and challenges.

|||||||||| OCH-NCNP1 / Eisai
Phase ? Clinical Trial of NKT Cell-targeting Glycolipid OCH-NCNP1 for Patients with Relapsing Multiple Sclerosis (Colorado Convention Center | Exhibit Hall B-E) - Mar 8, 2024 - Abstract #AAN2024AAN_3229;
Furthermore, it offers insights into the emerging category of peptide-conjugated ASO drugs those are clinical and preclinical trials, shedding light on their potential benefits and challenges. OCH is a promising treatment for multiple sclerosis, especially for SPMS.

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku
Enrollment closed, Trial completion date, Trial primary completion date:  Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X) (clinicaltrials.gov) -  Feb 13, 2024
P3, N=74, Active, not recruiting,  Sponsor: NS Pharma, Inc.
OCH is a promising treatment for multiple sclerosis, especially for SPMS. Enrolling by invitation --> Active, not recruiting | Trial completion date: Jun 2026 --> Nov 2025 | Trial primary completion date: Jun 2026 --> Oct 2025

||||||||||  brogidirsen (NS-089/NCNP-02) / Nippon Shinyaku, National Center of Neurology and Psychiatry
Enrollment open, Trial completion date, Trial initiation date, Trial primary completion date:  NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) -  Jan 5, 2024
P2, N=20, Recruiting,  Sponsor: NS Pharma, Inc.
Enrolling by invitation --> Active, not recruiting | Trial completion date: Jun 2026 --> Nov 2025 | Trial primary completion date: Jun 2026 --> Oct 2025 Not yet recruiting --> Recruiting | Trial completion date: May 2025 --> Sep 2025 | Initiation date: Aug 2023 --> Jan 2024 | Trial primary completion date: May 2025 --> Sep 2025

||||||||||  OCH-NCNP1 / Eisai
Trial completion:  Phase II Clinical Trial of OCH-NCNP1 (clinicaltrials.gov) -  Nov 9, 2023
P2, N=30, Completed,  Sponsor: National Center of Neurology and Psychiatry, Japan
Not yet recruiting --> Recruiting | Trial completion date: May 2025 --> Sep 2025 | Initiation date: Aug 2023 --> Jan 2024 | Trial primary completion date: May 2025 --> Sep 2025 Active, not recruiting --> Completed

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku
Trial completion, Trial completion date, Trial primary completion date:  Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) (clinicaltrials.gov) -  Oct 27, 2023
P3, N=77, Completed,  Sponsor: NS Pharma, Inc.
Active, not recruiting --> Completed Recruiting --> Completed | Trial completion date: Dec 2024 --> Oct 2023 | Trial primary completion date: Nov 2024 --> Oct 2023

|||||||||| NS-089/NCNP-02 / Nippon Shinyaku, National Center of Neurology and Psychiatry
Journal: Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide. (Pubmed Central) - Oct 19, 2023
NS-089/NCNP-02 induced exon 44 skipping in skeletal and cardiac muscle of cynomolgus monkeys. In conclusion, NS-089/NCNP-02, an antisense oligonucleotide with a novel connected-sequence design, showed highly efficient exon skipping both in

||||||||||  brogidirsen (NS-089/NCNP-02) / Nippon Shinyaku, National Center of Neurology and Psychiatry
New P2 trial:  NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) -  Aug 16, 2023
P2, N=20, Not yet recruiting,  Sponsor: NS Pharma, Inc.

|||||||||| OCH-NCNP1 / Eisai
Phase ? Clinical Trial of NKT Cell-Targeting Glycolipid OCH-NCNP1 for Patients with Relapsing Multiple Sclerosis (Brown 3) - Jul 21, 2023 - Abstract #MSMilan2023MSMilan_1585;
In conclusion, NS-089/NCNP-02, an antisense oligonucleotide with a novel connected-sequence design, showed highly efficient exon skipping both in OCH is a promising treatment for multiple sclerosis, especially for SPMS.

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku, National Center of Neurology and Psychiatry
Trial completion:  Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53) (clinicaltrials.gov) -  Jul 20, 2023
P2, N=20, Completed,  Sponsor: NS Pharma, Inc.
OCH is a promising treatment for multiple sclerosis, especially for SPMS. Active, not recruiting --> Completed

|||||||||| NS-089/NCNP-02 / Nippon Shinyaku, National Center of Neurology and Psychiatry
Clinical protocol, P1/2 data, Journal: Systemic administration of the antisense oligonucleotide NS-089/NCNP-02 for skipping of exon 44 in patients with Duchenne muscular dystrophy: Study protocol for a phase I/II clinical trial. (Pubmed Central) - Jun 20, 2023
Active, not recruiting --> Completed Exon-skipping therapy using ASOs shows promise in selected patients, and this first-in-human study is expected to provide critical information for subsequent clinical development of NS-089/NCNP-02.

|||||||||| Review, Journal: Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy. (Pubmed Central) - Mar 30, 2023
These upcoming therapies often utilize novel drug chemistries to address limitations of existing therapies, and their development could herald the next generation of antisense therapy. This review article aims to summarize the current state of development for antisense-based therapies for the treatment of Duchenne muscular dystrophy, exploring candidates designed for both exon skipping and gene knockdown.

||||||||||  brogidirsen (NS-089/NCNP-02) / Nippon Shinyaku, National Center of Neurology and Psychiatry
Trial completion date, Trial primary completion date:  Extension Study of NS-089/NCNP-02 in DMD (clinicaltrials.gov) -  Mar 3, 2023
P2, N=6, Active, not recruiting,  Sponsor: Nippon Shinyaku Co., Ltd.
This review article aims to summarize the current state of development for antisense-based therapies for the treatment of Duchenne muscular dystrophy, exploring candidates designed for both exon skipping and gene knockdown. Trial completion date: Jul 2023 --> Jul 2026 | Trial primary completion date: Jan 2023 --> Jan 2026

|||||||||| Vyondys 53 (golodirsen) / Sarepta Therap, NS-089/NCNP-02 / Nippon Shinyaku, National Center of Neurology and Psychiatry, Viltepso (viltolarsen) / Nippon Shinyaku
Journal: Restoring Dystrophin Expression with Exon 44 and 53 Skipping in the DMD Gene in Immortalized Myotubes. (Pubmed Central) - Nov 20, 2022
Two exon 53 skipping PMOs, golodirsen and viltolarsen, have received conditional approval for treating patients due to their ability to restore dystrophin protein expression...We introduce how to quantify exon-skipping efficiencies and dystrophin rescue levels represented by RT-PCR and western blotting, respectively. The screening methods using immortalized patient myotubes can serve to find exon-skipping PMO drug candidates.

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku
Enrollment closed, Enrollment change, Trial completion date, Trial primary completion date:  Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) (clinicaltrials.gov) -  Oct 19, 2022
P4, N=9, Active, not recruiting,  Sponsor: NS Pharma, Inc.
The screening methods using immortalized patient myotubes can serve to find exon-skipping PMO drug candidates. Enrolling by invitation --> Active, not recruiting | N=16 --> 9 | Trial completion date: Nov 2031 --> Oct 2032 | Trial primary completion date: Nov 2031 --> Sep 2032

||||||||||  OCH-NCNP1 / Eisai
Enrollment closed, Trial completion date, Trial primary completion date:  Phase II Clinical Trial of OCH-NCNP1 (clinicaltrials.gov) -  Sep 29, 2022
P2, N=30, Active, not recruiting,  Sponsor: National Center of Neurology and Psychiatry, Japan
Enrolling by invitation --> Active, not recruiting | N=16 --> 9 | Trial completion date: Nov 2031 --> Oct 2032 | Trial primary completion date: Nov 2031 --> Sep 2032 Enrolling by invitation --> Active, not recruiting | Trial completion date: Aug 2022 --> May 2023 | Trial primary completion date: Aug 2022 --> May 2023

||||||||||  brogidirsen (NS-089/NCNP-02) / Nippon Shinyaku, National Center of Neurology and Psychiatry
Trial completion:  Exploratory Study of NS-089/NCNP-02 in DMD (clinicaltrials.gov) -  Sep 29, 2022
P1/2, N=6, Completed,  Sponsor: National Center of Neurology and Psychiatry, Japan
Enrolling by invitation --> Active, not recruiting | Trial completion date: Aug 2022 --> May 2023 | Trial primary completion date: Aug 2022 --> May 2023 Enrolling by invitation --> Completed

|||||||||| NS-089/NCNP-02 / Nippon Shinyaku, National Center of Neurology and Psychiatry
A Phase I/II study of NS-089/NCNP-02, Exon 44 skipping drug, in patients with Duchenne muscular dystrophy (Poster area - Ballroom B1-B2) - Aug 20, 2022 - Abstract #WMS2022WMS_298;
All motor function scales including NSAA consistently tend to decrease in part 1 stage, but their functions tend to improve in part 2 stage. Our first-in-human studies shall provide critical data of safety, potential efficacy and pharmacokinetics of NS-089/NCNP-02 for subsequent clinical development of NS-089/NCNP-02, with the ultimate aim of expanding treatment capacity for DMD and other neuromuscular conditions.

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku, National Center of Neurology and Psychiatry
Enrollment closed, Trial completion date, Trial primary completion date:  Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53) (clinicaltrials.gov) -  Jul 5, 2022
P2, N=20, Active, not recruiting,  Sponsor: NS Pharma, Inc.
Our first-in-human studies shall provide critical data of safety, potential efficacy and pharmacokinetics of NS-089/NCNP-02 for subsequent clinical development of NS-089/NCNP-02, with the ultimate aim of expanding treatment capacity for DMD and other neuromuscular conditions. Recruiting --> Active, not recruiting | Trial completion date: May 2024 --> Sep 2023 | Trial primary completion date: May 2024 --> Aug 2023

|||||||||| NS-089/NCNP-02 / Nippon Shinyaku, National Center of Neurology and Psychiatry
A Phase I/II Study of NS-089/NCNP-02, Exon 44 Skipping Drug, in Patients with Duchenne Muscular Dystrophy (Poster Board Number: Tu-106; Hall D) - May 6, 2022 - Abstract #ASGCT2022ASGCT_1711;
We then compared the efficiency of exon 44 skipping induced by NS-089/NCNP-02 in differentiated MYOD1-UDCs (UDC-myotubes) and muscle biopsy samples from all the subjects. Our first-in-human studies shall provide critical data of safety, efficacy and pharmacokinetics of NS-089/NCNP-02 for subsequent clinical development of NS-089/NCNP-02, with the ultimate aim of expanding treatment capacity for DMD and other neuromuscular conditions.

||||||||||  OCH-NCNP1 / Eisai
Trial completion date, Trial primary completion date:  Phase II Clinical Trial of OCH-NCNP1 (clinicaltrials.gov) -  Apr 11, 2022
P2, N=30, Enrolling by invitation,  Sponsor: National Center of Neurology and Psychiatry, Japan
Our first-in-human studies shall provide critical data of safety, efficacy and pharmacokinetics of NS-089/NCNP-02 for subsequent clinical development of NS-089/NCNP-02, with the ultimate aim of expanding treatment capacity for DMD and other neuromuscular conditions. Trial completion date: Nov 2021 --> Aug 2022 | Trial primary completion date: Oct 2021 --> Aug 2022

||||||||||  brogidirsen (NS-089/NCNP-02) / Nippon Shinyaku, National Center of Neurology and Psychiatry
Trial completion date, Trial primary completion date:  Exploratory Study of NS-089/NCNP-02 in DMD (clinicaltrials.gov) -  Apr 11, 2022
P1/2, N=6, Enrolling by invitation,  Sponsor: National Center of Neurology and Psychiatry, Japan
Trial completion date: Nov 2021 --> Aug 2022 | Trial primary completion date: Oct 2021 --> Aug 2022 Trial completion date: Dec 2021 --> May 2022 | Trial primary completion date: Sep 2021 --> May 2022

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku, National Center of Neurology and Psychiatry
Phase classification:  Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) -  Dec 7, 2021
P2, N=16, Completed,  Sponsor: NS Pharma, Inc.
Trial completion date: Dec 2021 --> May 2022 | Trial primary completion date: Sep 2021 --> May 2022 Phase classification: P2b --> P2

||||||||||  brogidirsen (NS-089/NCNP-02) / Nippon Shinyaku, National Center of Neurology and Psychiatry
New P2 trial:  Extension Study of NS-089/NCNP-02 in DMD (clinicaltrials.gov) -  Nov 25, 2021
P2, N=6, Active, not recruiting,  Sponsor: Nippon Shinyaku Co., Ltd.

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku
Trial completion:  MASTERKEY: Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) -  Nov 24, 2021
P2, N=16, Completed,  Sponsor: NS Pharma, Inc.
Phase classification: P2b --> P2 Active, not recruiting --> Completed

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku
Enrollment status:  Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) (clinicaltrials.gov) -  Nov 22, 2021
P4, N=16, Enrolling by invitation,  Sponsor: NS Pharma, Inc.
Active, not recruiting --> Completed Recruiting --> Enrolling by invitation

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku
Enrollment status:  Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X) (clinicaltrials.gov) -  Nov 15, 2021
P3, N=74, Enrolling by invitation,  Sponsor: NS Pharma, Inc.
Recruiting --> Enrolling by invitation Recruiting --> Enrolling by invitation

||||||||||  brogidirsen (NS-089/NCNP-02) / Nippon Shinyaku, National Center of Neurology and Psychiatry
Trial primary completion date:  Exploratory Study of NS-089/NCNP-02 in DMD (clinicaltrials.gov) -  Sep 1, 2021
P1/2, N=6, Enrolling by invitation,  Sponsor: National Center of Neurology and Psychiatry, Japan
Recruiting --> Enrolling by invitation Trial primary completion date: Mar 2021 --> Sep 2021

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku, National Center of Neurology and Psychiatry
Phase classification:  Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) -  Jul 12, 2021
P2b, N=16, Completed,  Sponsor: NS Pharma, Inc.
Trial primary completion date: Mar 2021 --> Sep 2021 Phase classification: P2 --> P2b

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku, National Center of Neurology and Psychiatry
New P2 trial:  Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53) (clinicaltrials.gov) -  Jul 8, 2021
P2, N=20, Recruiting,  Sponsor: NS Pharma, Inc.

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku
Enrollment open:  Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X) (clinicaltrials.gov) -  Jul 1, 2021
P3, N=74, Recruiting,  Sponsor: NS Pharma, Inc.
Phase classification: P2 --> P2b Not yet recruiting --> Recruiting

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku
Enrollment open:  Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) (clinicaltrials.gov) -  Jun 30, 2021
P4, N=16, Recruiting,  Sponsor: NS Pharma, Inc.
Not yet recruiting --> Recruiting Not yet recruiting --> Recruiting

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku
Trial completion date, Trial primary completion date:  MASTERKEY: Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) -  Apr 27, 2021
P2, N=16, Active, not recruiting,  Sponsor: NS Pharma, Inc.
Not yet recruiting --> Recruiting Trial completion date: Aug 2021 --> Jan 2022 | Trial primary completion date: May 2021 --> Oct 2021

||||||||||  OCH-NCNP1 / Eisai
Trial completion date, Trial primary completion date:  Phase II Clinical Trial of OCH-NCNP1 (clinicaltrials.gov) -  Mar 3, 2021
P2, N=30, Enrolling by invitation,  Sponsor: National Center of Neurology and Psychiatry, Japan
Trial completion date: Aug 2021 --> Jan 2022 | Trial primary completion date: May 2021 --> Oct 2021 Trial completion date: Oct 2020 --> Nov 2021 | Trial primary completion date: Sep 2020 --> Oct 2021

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku
New P3 trial:  Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X) (clinicaltrials.gov) -  Feb 23, 2021
P3, N=74, Not yet recruiting,  Sponsor: NS Pharma, Inc.

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku
New P4 trial:  Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) (clinicaltrials.gov) -  Dec 28, 2020
P4, N=16, Not yet recruiting,  Sponsor: NS Pharma, Inc.

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku
Trial completion date, Trial primary completion date:  MASTERKEY: Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) -  Apr 22, 2020
P2, N=16, Active, not recruiting,  Sponsor: NS Pharma, Inc.
Trial completion date: Oct 2020 --> Nov 2021 | Trial primary completion date: Sep 2020 --> Oct 2021 Trial completion date: Dec 2020 --> Aug 2021 | Trial primary completion date: Dec 2020 --> May 2021

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku
Trial initiation date:  Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) (clinicaltrials.gov) -  Apr 20, 2020
P3, N=74, Recruiting,  Sponsor: NS Pharma, Inc.
Trial completion date: Dec 2020 --> Aug 2021 | Trial primary completion date: Dec 2020 --> May 2021 Initiation date: Oct 2019 --> Apr 2020

||||||||||  Viltepso (viltolarsen) / Nippon Shinyaku
New trial:  The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation (clinicaltrials.gov) -  Apr 6, 2020
P, N=0, Available,  Sponsor: NS Pharma, Inc.



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