Yeji

$IONS

IONIS PHARMACEUTICALS INC

Runway:7 mo
NasdaqMarket cap: —CIK 0000874015

Upcoming catalysts

5 surfaced
PDUFASep 22, 2026· in 108d
$IONSIONIS PHARMACEUTICALS INC
zilganersen · Alexander disease (AxD)
“Zilganersen is currently under Priority Review by the U.S. Food and Drug Administration with a Prescription Drug User Fee Act action date of September 22, 2026.”
PDUFADate TBD~ low confidence
$IONSIONIS PHARMACEUTICALS INC
olezarsen · Familial chylomicronemia syndrome (FCS)
“We are on track to submit a supplemental new drug application for both the 50 mg and 80 mg doses to the U.S. Food and Drug Administration ("FDA") by the end of the year and expect a Prescription Drug User Fee Act ("PDUFA") target action date in 2026.”
NDA/BLA filedDate TBD~ low confidence
$IONSIONIS PHARMACEUTICALS INC
“Bepirovirsen is currently under priority review by the U.S. Food and Drug Administration (FDA) with both Breakthrough and Fast Track Designation.”
NDA/BLA filedDate TBD
$IONSIONIS PHARMACEUTICALS INC
olezarsen · Familial chylomicronemia syndrome (FCS)
“In June 2024, the FDA accepted our NDA for patients with FCS for Priority Review with a Prescription Drug User Fee Act, or PDUFA, date of December nbsp;19, 2024.”
NDA/BLA filedDate TBD
$IONSIONIS PHARMACEUTICALS INC
olezarsen · Familial chylomicronemia syndrome (FCS)
“We are on track to submit a supplemental new drug application for both the 50 mg and 80 mg doses to the U.S. Food and Drug Administration ("FDA") by the end of the year and expect a Prescription Drug User Fee Act ("PDUFA") target action date in 2026.”

Drug pipeline

zilganersen
Alexander disease (AxD)
tryngolza
dawnzera
olezarsen
Familial chylomicronemia syndrome (FCS)
wainua
polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
ISIS EIF4E Rx
Non-small Cell Lung Cancer
ION356
Pelizaeus-Merzbacher Disease
Volanesorsen
Hypertriglyceridemia
ISIS 416858
End-stage Renal Disease (ESRD)
Donidalorsen
Hereditary Angioedema (HAE)
GHR-LRX
Acromegaly
ISIS-APO(a)Rx
Elevated Lipoprotein(a)
ION337
Dravet Syndrome
ISIS 388626
Type 2 Diabetes Mellitus
ISIS-GCGRRx- Dose Level 1
Type 2 Diabetes
ION537
Advanced Solid Tumors
IONIS-GHR-LRx
Acromegaly
ISIS 14803
Hepatitis C, Chronic
ISIS 353512
Inflammatory Diseases
ISIS-FXIRx Dose #2
Venous Thromboembolism
ISIS-FGFR4RX
Obesity
Scintigraphy scan
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
ISIS 113715
Type 2 Diabetes Mellitus
ISIS-GCCRRx
Type 2 Diabetes Mellitus
Eplontersen
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
ISIS 104838
Rheumatoid Arthritis
IONIS TMPRSS6-Lrx
Thalassemia
ISIS 3521
Lung Cancer
IONIS-STAT3Rx
Advanced Cancers
IONIS MAPTRx
Mild Alzheimer's Disease
ION775
Hypertriglyceridemia
sapablursen
Beta Thalassemia Intermedia
IONIS-ENaCRx
Healthy Subjects
ISIS 333611
Familial Amyotrophic Lateral Sclerosis
ION363
Amyotrophic Lateral Sclerosis
Alicaforsen
Crohn's Disease
Placebo
Hypertension
ION-827359
Chronic Bronchitis
IONIS-AGT-LRx
Chronic Heart Failure With Reduced Ejection Fraction
IONIS ANGPTL3-LRx
Hypertriglyceridemia
IONIS-FB-LRx
Macular Degeneration
Fomivirsen sodium
Cytomegalovirus Retinitis
ION464
Multiple System Atrophy
ION440
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
ISIS 325568
Type 2 Diabetes Mellitus
ISIS-GCGRRx - Dose Level 1
Type 2 Diabetes Mellitus
IONIS GHR-LRx
Acromegaly
ION717
Prion Disease
ISIS CRP Rx
Paroxysmal Atrial Fibrillation
IONIS FXI-LRx
Healthy Participants

Recent & active trials

NCTDrugPhaseStatusPrimary completion
NCT07531745ION337Phase 2Recruiting2030-12-01
NCT05071300EplontersenPhase 3Active Not Recruiting2029-08-01
NCT05667493EplontersenPhase 3Enrolling By Invitation2029-08-01
NCT07298447DonidalorsenPhase 3Recruiting2029-06-01
NCT05127226—Phase 2Recruiting2029-03-01
NCT05659901——Recruiting2029-03-01
NCT06150716ION356Phase 1Recruiting2028-06-01
NCT05130450olezarsenPhase 3Active Not Recruiting2028-02-01
NCT04165486ION464Phase 1Recruiting2027-09-01
NCT07604974ION775Phase 2Recruiting2027-09-01
NCT06430385ION440Phase 2Recruiting2027-09-01
NCT06914609—Phase 3Recruiting2027-08-01
NCT05185843olezarsenPhase 3Active Not Recruiting2027-06-01
NCT05681351olezarsenPhase 3Active Not Recruiting2027-03-01
NCT06153966ION717Phase 2Recruiting2027-02-01
NCT05392114DonidalorsenPhase 3Active Not Recruiting2026-12-01
NCT04768972ION363Phase 3Active Not Recruiting2026-06-01
NCT04136171EplontersenPhase 3Active Not Recruiting2026-04-01
NCT06073574——Active Not Recruiting2026-03-01
NCT06073587Scintigraphy scan—Active Not Recruiting2026-03-01

Recent catalysts

FDA approvalAug 21, 2025· 289d ago· success
$IONSIONIS PHARMACEUTICALS INC
dawnzera
“openFDA ORIG (TYPE 1) approval of DAWNZERA — application NDA219407”
PDUFADec 19, 2024· 534d ago· unknown
$IONSIONIS PHARMACEUTICALS INC
olezarsen · Familial chylomicronemia syndrome (FCS)
“In June 2024, the FDA accepted our NDA for patients with FCS for Priority Review with a Prescription Drug User Fee Act, or PDUFA, date of December nbsp;19, 2024.”
FDA approvalDec 19, 2024· 534d ago· success
$IONSIONIS PHARMACEUTICALS INC
tryngolza
“openFDA ORIG (TYPE 1) approval of TRYNGOLZA — application NDA218614”
FDA approvalDec 21, 2023· 898d ago
$IONSIONIS PHARMACEUTICALS INC
wainua · polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
“the U.S. Food and Drug Administration has approved Ionis' and AstraZeneca's WAINUA™ (eplontersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults”