renadirsen (DS-5141) / Daiichi Sankyo, Orphan Disease Treatment Institute - LARVOL DELTA

Tissue distribution of renadirsen sodium, a dystrophin exon-skipping antisense oligonucleotide, in heart and diaphragm after subcutaneous administration to cynomolgus monkeys. (PubMed, Nucleosides Nucleotides Nucleic Acids) - "The findings on exon 45-skipped dystrophin mRNA in these muscle tissues were consistent with the concentration of renadirsen in these tissues. Because it is not feasible to directly evaluate drug concentration and exon skipping in the heart and diaphragm in humans, the pharmacokinetics and pharmacodynamics of renadirsen in these tissues in monkeys are crucial for the design and interpretation of clinical settings."

Journal

Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=8 | Active, not recruiting | Sponsor: Daiichi Sankyo Co., Ltd. | Trial completion date: Mar 2024 ➔ Jan 2027 | Trial primary completion date: Mar 2024 ➔ Jan 2027

Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy. (PubMed, Pharmaceutics) - "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."

Journal • Review • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=8 | Active, not recruiting | Sponsor: Daiichi Sankyo Co., Ltd. | Trial completion date: Mar 2023 ➔ Mar 2024 | Trial primary completion date: Mar 2023 ➔ Mar 2024

Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=8 | Active, not recruiting | Sponsor: Daiichi Sankyo Co., Ltd. | Trial completion date: Mar 2022 ➔ Mar 2023 | Trial primary completion date: Mar 2022 ➔ Mar 2023

Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Renadirsen, a Novel 2'OMeRNA/ENA Chimera Antisense Oligonucleotide, Induces Robust Exon 45 Skipping for Dystrophin In Vivo. (PubMed, Curr Issues Mol Biol) - "Notably, systemic delivery of renadirsen sodium promoted dystrophin exon skipping in cardiac muscle, skeletal muscle, and diaphragm, compared with AOs with the same sequence as renadirsen but conventionally modified by PMO and 2'OMePS. These findings suggest the promise of renadirsen sodium as a therapeutic agent that improves not only skeletal muscle symptoms but also other symptoms in DMD patients, such as cardiac dysfunction."

Journal • Preclinical • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2; N=8; Active, not recruiting; Sponsor: Daiichi Sankyo Co., Ltd.; Enrolling by invitation ➔ Active, not recruiting

Clinical • Enrollment closed • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Daiichi Sankyo Announces the Results Summary of Phase 1/2 Clinical Trial in Japan for DS-5141 (PipelineReview) - P1/2, N=7; NCT02667483; Sponsor: Daiichi Sankyo Co., Ltd.; "Daiichi Sankyo Company...announced the results summary of the Phase 1/2 clinical trial in Japan (hereafter, the study) of DS-5141...No safety concerns, such as discontinuation or clinically significant adverse events, were observed in the study. Efficacy in terms of the production of messenger RNA with exon 45 skipping of the dystrophin gene (the trial’s secondary endpoint) was found in all patients, and the expression of dystrophin protein (the trial’s primary endpoint) showed a clear increase in several patients. Analysis of the trial result is currently ongoing."

P1/2 data • Duchenne Muscular Dystrophy

Study of DS-5141b in Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P1/2; N=7; Completed; Sponsor: Daiichi Sankyo Co., Ltd.; Active, not recruiting ➔ Completed

Clinical • Trial completion • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2; N=8; Enrolling by invitation; Sponsor: Daiichi Sankyo Co., Ltd.; Not yet recruiting ➔ Enrolling by invitation

Clinical • Enrollment open • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Study of DS-5141b in Participants With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2; N=8; Not yet recruiting; Sponsor: Daiichi Sankyo Co., Ltd.

Clinical • New P2 trial • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Study of DS-5141b in Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P1/2; N=7; Active, not recruiting; Sponsor: Daiichi Sankyo Co., Ltd.; Trial completion date: Dec 2019 ➔ Dec 2020; Trial primary completion date: Dec 2019 ➔ Dec 2020

Clinical • Trial completion date • Trial primary completion date