IONS - Ionis Pharmaceuticals

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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

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52 Wk Range

$ - $

Previous Close

$N/A

Open

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Day Range

N/A - N/A

Volume

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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 & IndicationStage & EventCatalyst Date
TRYNGOLZA™ (olezarsen) Details
Familial chylomicronemia syndrome

Approved

Update

QALSODY™ (Tofersen) Details
Amyotrophic lateral sclerosis

Approved

Update

Approved

Quarterly sales

SPINRAZA Details
Spinal muscular atrophy

Approved

Quarterly sales

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

Phase 3

Data readout

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

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