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VERVE-102 Heart-2 Cohort 3 Data (HeFH)

VERV·VERVE-102·Familial Hypercholesterolemia··NCT05814822
current best date
Q4
'26
QTR6 months
Takeaway

Cohort 3 dose-escalation data for Verve’s in-vivo base-editing therapy targeting PCSK9 in heterozygous familial hypercholesterolemia (HeFH). Watch: PCSK9 protein knockdown %, LDL-C reduction, hepatic and immunogenicity safety events.

What’s at stake

market · comparables · base rate · prior moves
Market opportunity
company filings
Peer comparables
1 readout
ClinicalTrials.gov
Stage base rate
~14%
BIO Industry Analysis 2023 (2011–2020)
Avg prior-readout move
historical price reactions
Drug
gene therapy
VERVE-102
INN
MoALNP-delivered base editor targeting PCSK9

VERVE-102 is Verve Therapeutics' in vivo base-editing therapy designed to provide a one-time, permanent reduction in LDL cholesterol in patients with heterozygous familial hypercholesterolemia (HeFH) — a dominant genetic disorder affecting 1 in 250 people that causes severely elevated LDL from birth. A single IV infusion delivers an adenine base editor via lipid nanoparticles to liver cells, converting a single DNA letter in the PCSK9 gene to a permanent loss-of-function variant that prevents the liver from degrading LDL receptors. The goal is lasting LDL-C reduction from one infusion, as an alternative to lifelong statin therapy and bi-weekly PCSK9 inhibitor injections.

Indication
Cardio-Renal
Familial Hypercholesterolemia
MeSH · D006938

No primer in glossary yet.

Trial
active
NCT05814822
Heart-2 Phase 1b VERVE-102 in HeFH
Phase
Ph 2
N
70
Primary completion
Nov 15, 2026
Glossary · what this readout is measuring
2 terms detected in the takeaway
  • PCSK9target
    Proprotein Convertase Subtilisin/Kexin type 9

    Liver enzyme that degrades LDL receptors. Inhibiting it (mAbs, siRNA, base-editing) lowers LDL-C dramatically.

  • LDL-Cendpoint
    Low-density lipoprotein cholesterol

    "Bad" cholesterol. FcRn inhibitors are known to elevate LDL by 20-40%, which matters for chronic dosing in patients with cardiovascular risk.

Reference data · comparable readouts

How Ph2 readouts in Cardio-Renal have landed.

Reference, not prediction. We surface the historical record so you can read it yourself. We never extrapolate to the upcoming event.

Positive
0/0
0% in record
Primary endpoint hit
0%
across 0 readouts
Drug · sponsorPhase · yearOutcomeKey metricSource
omecamtiv mecarbil
CYTK · HF
Ph3 · Oct 2022mixednarrow CV-death/HF benefit; FDA declinedFDA
finerenone (FIDELIO-DKD)
BAYRY · CKD
Ph3 · Jul 2020positive18% reduction in primary compositePR
sorted by phase match, then recency · sources span PR wires, CT.gov, FDA notices, and conference presentations · we never editorialize the outcome label

Competitive landscape

4 peers in Cardio-Renal · ranked by indication + modality match
DrugCompanyModalityMechanismPhaseNext catalyst
AMVUTTRAvutrisiranALNYsiRNATTR-targeting RNAiPDUFA · Dec 26
plozasiranARWRsiRNAAPOC3-targeting RNAiCONFERENCE · May 26
olezarsenIONSoligonucleotideApoCIII antisense oligonucleotidePDUFA · Dec 26
XPHOZAHtenapanorARDXsmall moleculeNHE3 inhibitorCMC · Jul 26

Disclosure trail

1 observation · sorted by confidence
  1. Apr 15, 2026·15d agopinned · highest confidence
    HIGH confPR
    top claim
    Q4'26
    QTR
    VERVE-102 Heart-2 Cohort 3 dose-escalation data in patients with heterozygous familial hypercholesterolemia is expected in the fourth quarter of 2026.
    conf 85%via llm

MethodologyEvery catalyst date is anchored to a primary source. Disclosures with confidence ≥ 0.85 auto-publish; the rest are reviewed by a human within 24 hours. We never interpret data — we organize public information.

Disclaimer·BioSight aggregates publicly available data (SEC EDGAR, ClinicalTrials.gov, FDA, company press releases) and is provided as is for informational purposes only. Nothing here is investment, financial, legal, tax, or medical advice, or an offer to buy or sell any security. We make no warranty as to accuracy, completeness, or timeliness; dates and filings may contain errors or be superseded without notice; past events do not predict future outcomes. To the maximum extent permitted by law, BioSight, its operators, contributors, and affiliates disclaim all liability for any loss or damage arising from use of or reliance on this site. You are solely responsible for your own investment decisions — consult a licensed professional before acting on any information.

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