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Ionis Pharmaceuticals NasdaqGS:IONS 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.
Location: 2855 Gazelle Court, Carlsbad, CA, 92010, United States | Website: https://www.ionis.com | Industry: Biotechnology | Sector: Healthcare
Market Cap
NaN
52 Wk Range
$ - $
Previous Close
$N/A
Open
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Day Range
N/A - N/A
Volume
N/A
Avg Volume
N/A
Enterprise Value
5.162B
Cash
2.148B
Avg Qtr Burn
-125.4M
Short % of Float
9.27%
Insider Ownership
0.84%
Institutional Own.
-
Qtr Updated
03/31/25
Drug Pipeline
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Drug & Indication | Stage & Event | Catalyst Date |
---|---|---|
TRYNGOLZA™ (olezarsen) Details Familial chylomicronemia syndrome | Approved Update | |
QALSODY™ (Tofersen) Details Amyotrophic lateral sclerosis | Approved Update | |
WAINUA™ Eplontersen (AKCEA-TTR-LRx) Details Transthyretin amyloidosis | Approved Quarterly sales | |
SPINRAZA Details Spinal muscular atrophy | Approved Quarterly sales | |
Donidalorsen (IONIS-PKK-LRx) Details Hereditary angioedema | PDUFA Approval decision | |
Eplontersen (AKCEA-TTR-LRx) Details Diabetic cardiomyopathy, Transthyretin amyloidosis, Transthyretin Amyloid Cardiomyopathy | Phase 3 Data readout | |
Ulefnersen (ION363 ) Details Amyotrophic lateral sclerosis | Phase 3 Data readout | |
Bepirovirsen (IONIS-HBVRx) Details Hepatitis B | Phase 3 Data readout | |
Zilganersen (ION373) Details Alexander Disease | Phase 3 Data readout | |
IONIS-APOCIII-LRx (Olezarsen) Details Severe hypertriglyceridemia | Phase 3 Data readout | |
Pelacarsen Details Heart disease, Cardiovascular disease | Phase 3 Data readout | |
ION582 Details Angelman Syndrome | Phase 3 Initiation | |
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 | |
IONIS-ENaC-2.5Rx (antisense medicine) Details Cystic fibrosis | Failed Discontinued | |
Fesomersen (IONIS-FXI-LRx) Details Hepatitis B | Failed Discontinued | |
IONIS-FB-LRx Details Geographic atrophy | Failed Discontinued |