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WVE-N531

Phase 2

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

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment201
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07209332Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular DystrophyPHASE2 ENROLLING BY_INVITATION 175 — — Dec 28, 2025Mar 1, 2029Mar 6, 20263 Jordan, United Kingdom
NCT04906460Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)PHASE1 RECRUITING 26 — — Sep 28, 2021Apr 24, 2027Dec 15, 20255 United States, Jordan +1
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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
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
WVE-N531EXPERIMENTAL -
Interventions
NameTypeDescription
WVE-N531DRUGWVE-N531 is an antisense oligonucleotide (ASO)
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Eligibility Criteria
Age Range4 Years — 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
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04906460primaryCompletionDate: changed
LOWMay 26, 2026NCT07209332primaryCompletionDate: changed
LOWMay 24, 2026NCT04906460studyFirstPostDate: changed
LOWMay 24, 2026NCT07209332studyFirstPostDate: changed