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WVE-006

Phase 1

Alpha-1 Antitrypsin Deficiency (AATD) | Small molecule | Other |Wave Life Sciences Ltd.|Last Updated: Mar 23, 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
RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment47
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06186492A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)PHASE1 COMPLETED 47 — — Nov 14, 2023Feb 13, 2025Mar 23, 20261 United Kingdom
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Study Endpoints
Primary Endpoints
The Proportion of Participants With Adverse Events
Adverse events are collected from the date of consent until up to 85 days after the dose in Period 1 and up to 113 days after the first dose in Period 2.

The number of participants who reported an adverse event (AE) will be summarised.

Secondary Endpoints
Single Ascending Dose (Period 1) - Area Under the Plasma Concentration Time Curve for WVE-006 From Time of Dosing to the Last Measurable Concentration (AUClast)
Samples collected at the following timepoints: Day 1 pre-dose, 30 mins, 1 hr, 2 hr, 4 hr, 6 hr, 8 hr & 12 hr post-Day 1 dose, Day 2: 24 hr & 36 hr post-Day 1 dose, Day 3: 48 hr post-Day 1 dose, Days 8, 15, 22, 29, 36, 43, 50, 57, 64, 71, 78 & 85.
Single Ascending Dose - Maximum Concentration of WVE-006 in Plasma (Cmax)
Samples collected at the following timepoints: Day 1 pre-dose, 30 mins, 1 hr, 2 hr, 4 hr, 6 hr, 8 hr & 12 hr post-Day 1 dose, Day 2: 24 hr & 36 hr post-Day 1 dose, Day 3: 48 hr post-Day 1 dose, Days 8, 15, 22, 29, 36, 43, 50, 57, 64, 71, 78 & 85.
Multiple Ascending Doses - Area Under the Plasma Concentration Time Curve for WVE-006 From Time of Dosing to the Last Measurable Concentration (AUClast) - Day 1
Samples collected at the following timepoints for Day 1 measurement: Day 1 pre-dose, 30 mins, 1 hr, 2 hr, 4 hr, 6 hr, 8 hr, 12 hr & 24 hr post-dose.
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Single Ascending Dose (SAD): WVE-006 30 milligram (mg) or placeboEXPERIMENTAL -
SAD: WVE-006 100 mg or placeboEXPERIMENTAL -
SAD WVE-006 200 mg or placeboEXPERIMENTAL -
SAD: WVE-006 400 mg or placeboEXPERIMENTAL -
SAD: WVE-006 600 mg or placeboEXPERIMENTAL -
Multiple Dose: WVE-006 600 mg Every 2 weeks (Q2W) or placeboEXPERIMENTAL -
Interventions
NameTypeDescription
WVE-006DRUGRNA editing oligonucleotide
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Healthy as determined by the Investigator, based on a medical evaluation. * Genetic testing confirming PI\*MM. * Participant has been a non-smoker for at least 1 year prior to screening. Exclusion Criteria: * Participant has a history of multiple drug allergies or of allergi...

Countries:United Kingdom
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