Ionis Pharmaceuticals, Inc., a commercial-stage biotechnology company, provides RNA-targeted medicines in the United States. The company offers TRYNGOLZA reduces triglyceride levels in adults with familial chylomicronemia syndrome (FCS) and acute pancreatitis; WAINUA for the treatment of the polyneuropathy of hereditary transthyretin-medicated amyloidosis (ATTRv-PN) in adults; and SPINRAZA for pediatric and adult patients with spinal muscular atrophy (SMA). It also provides QALSODY for the treatment of Amyotrophic Lateral Sclerosis (ALS); TEGSEDI for the treatment of ATTRv-PN in adults; and WAYLIVRA for treatment for FCS and familial partial lipodystrophy. It also develops products under Phase 3 clinical trials, such as Olezarsen for patients with hypertriglyceridemia (SHTG) and cardiovascular disease (CVD); Donidalorsen for patients with hereditary angioedema; and Zilganerse, a potential treatment for people with genetically confirmed Alexander disease, as well as ION582 that has completed Phase 2 clinical trial for the potential treatment of AS, a rare genetic neurological disease. In addition, the company develops Eplontersen to degrade mutant and wild-type TTR mRNA through binding to the TTR mRNA; Pelacarsen to inhibit the production of apolipoprotein(a) in the liver to offer a direct approach for reducing lipoprotein(a); Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus; Sefaxersen to reduce the production of complement factor B and lower activation of the alternative complement pathway; and Ulefnersen to reduce the production of the fused in sarcoma, as well as other mid-stage pipeline investigational medicines. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with GSK, AstraZeneca, Novartis, and Roche, as well as with Metagenomi. The company was incorporated in 1989 and is headquartered in Carlsbad, California. Show more
Location: 2855 Gazelle Court, Carlsbad, CA, 92010, United States | Website: https://www.ionis.com | Industry: Biotechnology | Sector: Healthcare
Market Cap
6.335B
52 Wk Range
$23.95 - $52.34
Previous Close
$39.78
Open
$39.77
Volume
2,481,663
Day Range
$39.55 - $40.10
Enterprise Value
6.155B
Cash
2.148B
Avg Qtr Burn
-125.4M
Insider Ownership
0.84%
Institutional Own.
-
Qtr Updated
03/31/25
Drug Pipeline
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Drug & Indication | Stage & Event | Catalyst Date |
---|---|---|
QALSODY™ (Tofersen) Details Amyotrophic lateral sclerosis | Approved Update | |
TRYNGOLZA™ (olezarsen) Details Familial chylomicronemia syndrome | Approved Update | |
WAINUA™ Eplontersen (AKCEA-TTR-LRx) Details Transthyretin amyloidosis | Approved Quarterly sales | |
SPINRAZA Details Spinal muscular atrophy | Approved Quarterly sales | |
PDUFA Approval decision | ||
ION582 Details Angelman Syndrome | Phase 3 Data readout | |
Bepirovirsen (IONIS-HBVRx) Details Hepatitis B | Phase 3 Data readout | |
Ulefnersen (ION363 ) Details Amyotrophic lateral sclerosis | Phase 3 Data readout | |
Eplontersen (AKCEA-TTR-LRx) Details Diabetic cardiomyopathy, Transthyretin amyloidosis, Transthyretin Amyloid Cardiomyopathy | Phase 3 Data readout | |
Pelacarsen Details Heart disease, Cardiovascular disease | Phase 3 Data readout | |
Zilganersen (ION373) Details Alexander Disease | Phase 3 Data readout | |
Phase 3 Data readout | ||
ION839 / AZD2693 Details Non-alcoholic steatohepatitis | Phase 2b Data readout | |
ION224 Details Non-alcoholic steatohepatitis | Phase 2b Update | |
IONIS-MAPTRx (BIIB080) Details Alzheimer's disease | Phase 2 Data readout | |
IONIS-FB-LRx Details IgA nephropathy | Phase 2 Update | |
Tominersen (IONIS-HTT Rx) Details Huntington's disease | Phase 2 Update | |
IONIS-AGT-LRx Details Heart failure, Pulmonary hypertension | Phase 2 Update | |
IONS356 (PLP1) Details Pelizaeus-Merzbacher disease | Phase 1/2 Data readout | |
Cimdelirsen (IONIS-GHR-LRx) Details Acromegaly | Failed Discontinued | |
Fesomersen (IONIS-FXI-LRx) Details Hepatitis B | Failed Discontinued | |
IONIS-ENaC-2.5Rx (antisense medicine) Details Cystic fibrosis | Failed Discontinued | |
IONIS-FB-LRx Details Geographic atrophy | Failed Discontinued |