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Ionis Pharmaceuticals Inc

IONS
Healthcare|Biotechnology|USA
$58.25
-6.02 (-9.37%)
Tangible Book
$2.96

About

Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and Biogen brought Spinraza to market in 2016 as a treatment for spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Ionis and AstraZeneca launched polyneuropathy drug Wainua in 2024. Ionis marked its first two independent launches in 2025 for Tryngolza (for FCS) and Dawnzera (for HAE).

Stock Price

+37.4%(1Y)

Pipeline Summary

156 total trials
44
Phase 1
75
Phase 2
33
Phase 3
0
Phase 4
Recruiting: 12Withdrawn: 4Completed: 117APPROVED_FOR_MARKETING: 2Terminated: 8Active: 10ENROLLING_BY_INVITATION: 1AVAILABLE: 2

Active Trials (22)

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
NCT06430385Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Phase 1Phase 2Recruiting
Enrollment: 48 participants
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
NCT05659901Pelizaeus-Merzbacher Disease
Recruiting
Enrollment: 32 participants
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
NCT07531745Dravet Syndrome
Phase 1Phase 2Recruiting
Enrollment: 32 participants
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
NCT05127226Angelman Syndrome
Phase 1Phase 2Recruiting
Enrollment: 70 participants
CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
NCT04136171Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Phase 3Active
Enrollment: 1,438 participants
View all 22 active trials →

BioScore

6.4/10
Watchlist

Ownership

Recent Insider Trades

Diaz Allene M.SELL
2026-07-0710,111 shares @ $85.42
Kordasiewicz Holly B.SELL
2026-07-07362 shares @ $84.3
BENNETT C FRANKSELL
2026-07-0720,536 shares @ $84.95
KLEIN JOSEPH IIISELL
2026-07-0111,518 shares @ $79.04
Baroldi JosephSELL
2026-06-265,635 shares @ $80.16

Recent News

2026-07-10 14:57:59
2026-07-10 14:46:12
2026-07-10 14:38:25
2026-07-10 14:00:03
2026-07-10 13:30:53
Data last updated: Jul 12, 2026, 07:27 PM