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NCT04768972Phase 3ACTIVE NOT RECRUITING

FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)

Study Summary

The primary purpose of this study is to evaluate the efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS).

Conditions
Amyotrophic Lateral Sclerosis
Interventions / Drugs
ION363Placebo
View on ClinicalTrials.gov ↗
Sponsor
IONS
Ionis Pharmaceuticals
Antisense Therapeutics
D50/100
Study Details
Enrollment89 participants
Primary Completion—
Lead SponsorIonis Pharmaceuticals, Inc.
Data Retrieved2026-04-11 04:17:53.139337+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: 89 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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