Recent Updates
Recently added Catalysts

WVE-N531

Phase 2

Duchenne Muscular Dystrophy | Small molecule | Neurology |Wave Life Sciences Ltd.|Last Updated: Mar 6, 2026

Target and mechanism

Molecular targetDMD exon 53
ModalitySmall molecule

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

UNCONTROLLEDDMC
Total Trials2
Total Enrollment201

FDA Designations

RARE_PEDIATRIC_DISEASE

Clinical trial landscape

WVE-N531 · 2 trials · 1 indication

Phase 2 1Phase 1 1
NCT07209332Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular DystrophyDuchenne Muscular Dystrophy
ENROLLING BY_INVITATION175 Analytics
PHASE2ENROLLING BY_INVITATION
Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
NCT07209332175
Unlock trial analytics

Study Endpoints

Primary Endpoints

Number of participants with treatment related adverse events as assessed by the following parameters:
Time Frame: Week 0 through Week 96

* Complete Physical examination, including height and weight * Vital Signs (via blood pressure, temperature and pulse) * Safety Laboratory Tests (including complete blood cell count, urinalysis and clinical chemistry) * ECG (12- Lead single tracing) * ECHO (including left ventricular ejection fraction) * Pulmonary Function Tests (including Peak Flow Rate, Cough Peak Flow, Forced Vital Capacity and Maximum Inspiratory Pressure)

Part A: Safety: Proportion of patients with adverse events (AEs)
Day 1 (initial dose) up to 24 weeks after the last dose of Part A
Part B: Pharmacodynamics: Dystrophin level (% normal dystrophin) as assessed by Western blot of muscle tissue following multiple doses of WVE-N531
At Week 26 and at Week 50 of Part B
Part C: Pharmacodynamics: Change from baseline dystrophin level (% normal dystrophin) as assessed by a validated assay analysis in muscle tissue following multiple doses of WVE-N531
At Baseline and following 24 weeks of treatment in Part C

Secondary Endpoints

North Star Ambulatory Assessment (NSAA) (Version 2.0) composite score, each item assessed using a 3 point scale, including:
At baseline Week 0 through Week 96
Performance of the Upper Limb (PUL) (Version 2.0) (measured by total score and score of high level shoulder dimension, Mid-level Elbow Dimension, Distal Wrist and Hand Dimension)
At baseline Week 0 through Week 96
Stride Velocity 95th Centile (SV95C) (collected in 3 consecutive weeks)
At baseline Week 0 through Week 96
Unlock Study Endpoints

Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
WVE-N531EXPERIMENTAL -

Interventions

NameTypeDescription
WVE-N531DRUGWVE-N531 is an antisense oligonucleotide (ASO)
Unlock Study Design Details

Eligibility Criteria

Age Range4 Years to 18 Years
SexMALE
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: 1. Participated in a prior study of WVE-N531 and has not experienced any significant toxicities due to WVE-N531 or significant clinical deterioration of general health since the last dose or early discontinuation visit. 2. Of note: if there will be a greater than 31-day gap betw...

Countries:JordanUnited KingdomUnited States
Unlock Eligibility Criteria

Frequently asked questions about WVE-N531

What is WVE-N531 used for?

WVE-N531 is an investigational therapy being developed for Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration. It is currently in clinical development and has not been approved by the FDA. The drug is designed to target the DMD gene, specifically exon 53, which is mutated in a subset of DMD patients.

What does WVE-N531 target?

WVE-N531 targets exon 53 of the DMD gene, which encodes the dystrophin protein. By targeting this specific exon, the therapy aims to restore the reading frame of the gene, potentially allowing for the production of a functional dystrophin protein in patients with Duchenne Muscular Dystrophy who have mutations amenable to exon 53 skipping.

Who makes WVE-N531?

WVE-N531 is being developed by Wave Life Sciences Ltd., a biopharmaceutical company traded on the NASDAQ under the ticker symbol WVE. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational drug for the treatment of Duchenne Muscular Dystrophy.

What phase is WVE-N531 in?

WVE-N531 is currently in Phase 2 clinical development. It has received Rare Pediatric Disease designation from the FDA, which is granted for serious or life-threatening conditions affecting fewer than 200,000 people in the U.S. The drug is investigational and has not yet been approved for commercial use.

What clinical trials is WVE-N531 in?

WVE-N531 is being studied in two clinical trials. The first is NCT07209332, an open-label extension study in patients with Duchenne Muscular Dystrophy, enrolling 175 participants in Jordan and the United Kingdom. The second is NCT04906460, known as FORWARD-53, an open-label study with 26 participants in the U.S., Jordan, and the U.K.

Is WVE-N531 the same as FORWARD-53?

No, WVE-N531 is the drug being investigated, while FORWARD-53 is the name of one of its clinical trials. The FORWARD-53 trial, identified as NCT04906460, is an open-label study evaluating WVE-N531 in patients with Duchenne Muscular Dystrophy. The drug itself is not synonymous with the trial name.