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NCT06672237Phase 3ACTIVE NOT RECRUITING

A Phase 3 Study of NTLA-2001 in ATTRv-PN

Study Summary

This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.

Conditions
Neuromuscular DiseaseNeuromuscular Diseases (NMD)Neurodegenerative DiseaseNeurodegenerative Disease, HereditaryNeurodegenerative DiseasesNeuromuscular DiseasesNerve DisordersNervous System DiseaseNervous System DiseasesGenetic Disease, InbornAmyloidosis, FamilialAmyloidosis, HereditaryAmyloidosisPolyneuropathiesAmyloid NeuropathiesAmyloid Neuropathies, FamilialPeripheral Nervous System DiseasePeripheral Nervous System DiseasesMetabolism, Inborn ErrorsMetabolic Diseases
Interventions / Drugs
nexiguran ziclumeranNormal Saline as Placebo
View on ClinicalTrials.gov ↗
Sponsor
NTLA
Intellia Therapeutics
Gene Editing
C55/100
Study Details
Enrollment50 participants
Primary Completion—
Lead SponsorIntellia Therapeutics
Data Retrieved2026-04-11 04:23:01.073848+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 50 participants
Sponsor Catalyst Profile

Intellia Therapeutics (NTLA) is the sponsoring company for this trial. BiotechSign currently grades this company C (55/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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