vesleteplirsen (SRP-5051) / Sarepta Therap - LARVOL DELTA

MOMENTUM: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - P2 | N=62 | Terminated | Sponsor: Sarepta Therapeutics, Inc. | Trial completion date: Jan 2029 ➔ Feb 2025 | Active, not recruiting ➔ Terminated; The study was terminated following review of safety data.

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

Comprehensive review of adverse reactions and toxicology in ASO-based therapies for Duchenne Muscular Dystrophy: From FDA-approved drugs to peptide-conjugated ASO. (PubMed, Curr Res Toxicol) - "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."

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

MOMENTUM: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - P2 | N=62 | Active, not recruiting | Sponsor: Sarepta Therapeutics, Inc. | Trial completion date: Mar 2025 ➔ Jan 2029

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

A Study for Participants With Duchenne Muscular Dystrophy (DMD) Who Participated in Studies of SRP-5051 (clinicaltrialsregister.eu) - P1/2 | N=15 | Sponsor: Sarepta Therapeutics, Inc.

New P1/2 trial • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Musculoskeletal Diseases

MOMENTUM: Two-Part Study for Dose Determination of SRP-5051 (Vesleteplirsen) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - P2 | N=62 | Active, not recruiting | Sponsor: Sarepta Therapeutics, Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Aug 2024 ➔ Mar 2025

Enrollment closed • Trial 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

MOMENTUM: Two-Part Study for Dose Determination of SRP-5051 (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - P2; N=60; Recruiting; Sponsor: Sarepta Therapeutics, Inc.; Active, not recruiting ➔ Recruiting

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

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (clinicaltrials.gov) - P1/2; N=15; Terminated; Sponsor: Sarepta Therapeutics, Inc.; N=60 ➔ 15; Trial completion date: Jul 2024 ➔ Aug 2021; Active, not recruiting ➔ Terminated; Trial primary completion date: Jul 2024 ➔ Aug 2021; The sponsor has decided to integrate 5051-102 into 5051-201. Participants from 5051-102 will be eligible to enroll in 5051-201 Part B.

Clinical • Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

MOMENTUM: Two-Part Study for Dose Determination of SRP-5051 (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - P2; N=60; Active, not recruiting; Sponsor: Sarepta Therapeutics, Inc.; Trial completion date: May 2022 ➔ Aug 2024; Trial primary completion date: May 2022 ➔ Sep 2023

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

Pharmacology and toxicology of eteplirsen and SRP-5051 for DMD exon 51 skipping: an update. (PubMed, Arch Toxicol) - "Issues faced by eteplirsen and SRP-5051, including efficacy and safety, are identified. Lastly, the current state of eteplirsen and exon-skipping therapy in general as a strategy for the treatment of DMD are discussed."

Journal • Review • Cardiovascular • Congestive Heart Failure • Dermatology • Duchenne Muscular Dystrophy • Genetic Disorders • Heart Failure • Muscular Dystrophy • Otorhinolaryngology • Respiratory Diseases

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (clinicaltrials.gov) - P1/2; N=60; Active, not recruiting; Sponsor: Sarepta Therapeutics, Inc.; Enrolling by invitation ➔ Active, not recruiting

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

MOMENTUM: Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - P2; N=70; Active, not recruiting; Sponsor: Sarepta Therapeutics, Inc.; Recruiting ➔ Active, not recruiting

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

[VIRTUAL] Biological Efficacy of the Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer SRP-5051 in Preclinical Models of Duchenne Muscular Dystrophy (MDA 2021) - "In vivo systems included a disease-relevant, humanized DMD mouse model, hDMD del52/mdx, that contains a DMD transgene with exon 52 deleted on an mdx mouse background (lacking endogenous dystrophin), and a primate model using healthy male cynomolgus monkeys. SRP-5051 showed dose-dependent efficacy in preclinical models. These data justify the monthly SRP-5051 dosing regimen used in ongoing clinical studies, and support further clinical investigation of this PPMO."

Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • HSP90AA1

MOMENTUM: Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - P2; N=70; Recruiting; Sponsor: Sarepta Therapeutics, Inc.; N=45 ➔ 70; Trial completion date: Aug 2021 ➔ May 2022; Trial primary completion date: Aug 2021 ➔ May 2022

Clinical • Enrollment change • Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

SRP-5051: “Safe and well-tolerated with no clinical or laboratory-based safety signals”; Duchenne muscular dystrophy (Sarepta Therapeutics) - MOMENTUM Multiple-Ascending Dose Study of SRP-5051 for Duchenne Muscular Dystrophy: “Consistently higher tissue exposure, exon-skipping and dystrophin observed with 20 mg/kg SRP-5051 at 12 weeks compared to 30 mg/kg eteplirsen at 24 weeks”

P2 data • Duchenne Muscular Dystrophy

Sarepta Therapeutics Announces Positive Clinical Results from MOMENTUM, a Phase 2 Clinical Trial of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51 (GlobeNewswire) - P2, N=45; MOMENTUM (NCT04004065); Sponsor: Sarepta Therapeutics, Inc.; "Sarepta...today announced results from the ongoing MOMENTUM study (Study 5051-201), a global Phase 2 clinical trial, of SRP-5051, its next-generation treatment for patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping...Results from Part A of the multi-ascending dose MOMENTUM study found consistently higher tissue exposure, exon-skipping and dystrophin production in patients taking a monthly dose of SRP-5051 compared to baseline..."

P2 data • Duchenne Muscular Dystrophy

Sarepta Therapeutics to Share Clinical Update for SRP-5051, its Investigational PPMO for the Treatment of Duchenne Muscular Dystrophy (GlobeNewswire) - "Sarepta Therapeutics...today announced that on Monday, Dec. 7, 2020 at 8:30 am Eastern Time (ET), it will host a webcast and conference call to present interim data from the MOMENTUM study, a multiple-ascending dose clinical trial of SRP-5051 for the treatment of Duchenne muscular dystrophy."

P2 data • Duchenne Muscular Dystrophy

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (clinicaltrials.gov) - P1/2; N=60; Enrolling by invitation; Sponsor: Sarepta Therapeutics, Inc.; Recruiting ➔ Enrolling by invitation

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

MOMENTUM: Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - P2; N=45; Recruiting; Sponsor: Sarepta Therapeutics, Inc.; N=24 ➔ 45; Trial completion date: Mar 2021 ➔ Aug 2021; Trial primary completion date: Mar 2021 ➔ Aug 2021

Clinical • Enrollment change • Trial completion date • Trial primary completion date

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P1; N=15; Completed; Sponsor: Sarepta Therapeutics, Inc.; Recruiting ➔ Completed; N=30 ➔ 15

Clinical • Enrollment change • Trial completion

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (clinicaltrials.gov) - P1/2; N=54; Recruiting; Sponsor: Sarepta Therapeutics, Inc.; N=30 ➔ 54; Trial completion date: Nov 2020 ➔ Jul 2024; Trial primary completion date: Nov 2020 ➔ Jul 2024

Clinical • Enrollment change • Trial completion date • Trial primary completion date

MOMENTUM: A Phase 2 Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - P2; N=24; Recruiting; Sponsor: Sarepta Therapeutics

Clinical • New P2 trial

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (clinicaltrials.gov) - P1/2; N=30; Recruiting; Sponsor: Sarepta Therapeutics; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open