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NCT05071300Phase 3ACTIVE NOT RECRUITING

A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Study Summary

The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN).

Conditions
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Interventions / Drugs
Eplontersen
View on ClinicalTrials.gov ↗
Sponsor
IONS
Ionis Pharmaceuticals
Antisense Therapeutics
D50/100
Study Details
Enrollment151 participants
Primary Completion—
Lead SponsorIonis Pharmaceuticals, Inc.
Data Retrieved2026-04-11 04:17:53.139212+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: 151 participants
Sponsor Catalyst Profile

Ionis Pharmaceuticals (IONS) is the sponsoring company for this trial. BiotechSign currently grades this company D (50/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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