Ionis Pharmaceuticals Inc
IONSHealthcare|Biotechnology|USA
$58.25
-6.02 (-9.37%)
Tangible Book
$2.96
Upcoming Data Readouts (17)
Dec 2026
Est. completion
P3Hereditary Angioedema
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
NCT05392114n=154
Feb 2027
Est. completion
P1P2Prion Disease
PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
NCT06153966n=76
Mar 2027
Est. completion
P3Severe Hypertriglyceridemia
CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)
NCT05681351n=885
Jun 2027
Est. completion
P3Familial Chylomicronemia Syndrome
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen
NCT05185843n=24
Aug 2027
Est. completion
Sep 2027
Est. completion
P1P2Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
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)
NCT06430385n=48
Sep 2027
Est. completion
P2Hypertriglyceridemia
A Study to Assess the Safety, Tolerability and Efficacy of ION775
NCT07604974n=72
Sep 2027
Est. completion
P1Multiple System Atrophy
Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)
NCT04165486n=40
Feb 2028
Est. completion
P3Familial Chylomicronemia Syndrome
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)
NCT05130450n=60
Jun 2028
Est. completion
P1Pelizaeus-Merzbacher Disease
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
NCT06150716n=24
Mar 2029
Est. completion
Pelizaeus-Merzbacher Disease
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
NCT05659901n=32
Mar 2029
Est. completion
P1P2Angelman Syndrome
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
NCT05127226n=70
Jun 2029
Est. completion
P3Hereditary Angioedema (HAE)
Donidalorsen Treatment in Children With Hereditary Angioedema
NCT07298447n=20
Aug 2029
Est. completion
P3Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
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
NCT05071300n=151
Dec 2030
Est. completion
P1P2Dravet Syndrome
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
NCT07531745n=32
Dec 2030
Est. completion
Dec 2030
Est. completion
Awaiting Results (5)
Trials past primary completion date but still active — data readout may be imminent.
Jun 2026
P3
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)
NCT04768972Apr 2026
P3
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)
NCT04136171Aug 2025
P3
A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)
NCT04849741