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 & IndicationStage & EventCatalyst Date
QALSODY™ (Tofersen) Details
Amyotrophic lateral sclerosis

Approved

Update

TRYNGOLZA™ (olezarsen) Details
Familial chylomicronemia syndrome

Approved

Update

Approved

Quarterly sales

SPINRAZA Details
Spinal muscular atrophy

Approved

Quarterly sales

Donidalorsen (IONIS-PKK-LRx) Details
Hereditary angioedema

Susp. Mover™

PDUFA

Approval decision

ION582 Details
Angelman Syndrome

Phase 3

Data readout

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

Phase 3

Data readout

IONIS-APOCIII-LRx (Olezarsen) Details
Severe hypertriglyceridemia

Susp. Mover™

Phase 3

Data readout

ION839 / AZD2693 Details
Non-alcoholic steatohepatitis

Phase 2b

Data readout

ION224 Details
Non-alcoholic steatohepatitis

Phase 2b

Update

Phase 2

Data readout

IONIS-FB-LRx Details
IgA nephropathy

Phase 2

Update

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

Failed

Discontinued

Failed

Discontinued

Failed

Discontinued

IONIS-FB-LRx Details
Geographic atrophy

Failed

Discontinued