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VERVE-101

Phase 1

Heterozygous Familial Hypercholesterolemia | Small molecule | Metabolic |Verve Therapeutics, Inc.|Last Updated: May 8, 2025

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
CONTROLLEDDMC
Total Trials1
Total Enrollment13
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05398029A Study of VERVE-101 in Patients With Familial Hypercholesterolemia and Cardiovascular DiseasePHASE1 COMPLETED 13Jul 5, 2022Feb 14, 2025May 8, 20253 New Zealand, United Kingdom
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Study Endpoints
Primary Endpoints
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).
up to Day 365
Secondary Endpoints
Evaluation of maximum observed concentration (Cmax)
up to Day 365
Evaluation of time to maximum observed concentration (tmax)
up to Day 365
Evaluation of terminal elimination half-life (t1/2)
up to Day 365
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part A: Single Ascending Dose Escalation/Adaptive DesignEXPERIMENTALParticipants will receive a single dose of VERVE-101 in multiple dose-escalation cohorts.
Part B: Single Dose ExpansionEXPERIMENTALParticipants will receive a single dose of VERVE-101 selected based on the doses studied in Part A.
Interventions
NameTypeDescription
VERVE-101DRUGIntravenous (IV) infusion.
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: * Male and/or female participants 18 up to 75 years at time of signing of informed consent * Female participants not of child-bearing potential * Diagnosis of HeFH * Established ASCVD Exclusion Criteria: * Active or history of chronic liver disease * Current treatment with PCS...

Countries:New ZealandUnited Kingdom
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