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Last $61.88 USD
Change Today +0.84 / 1.38%
Volume 1.3M
ISIS On Other Exchanges
As of 5:20 PM 12/1/15 All times are local (Market data is delayed by at least 15 minutes).
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Company Description

Contact Info

2855 Gazelle Court

Carlsbad, CA 92010

United States

Phone: 760-931-9200


Isis Pharmaceuticals, Inc. engages in the discovery and development of a class of RNA (ribonucleic acid)-targeted drugs called antisense drugs using novel drug discovery platform. Drug Discovery and Development Development Projects KYNAMRO (mipomersen sodium) Injection The company’s novel lipid-lowering product, KYNAMRO (mipomersen sodium) injection, is on the market in the United States for patients with homozygous familial hypercholesterolemia (HoFH). In 2013, the U.S. Food and Drug Administration (FDA) approved the marketing application for KYNAMRO for patients with HoFH. Genzyme Corporation (Genzyme), a Sanofi Company, has also obtained marketing approval in other countries, including Mexico, Argentina, South Korea, and Peru; and is pursuing marketing approval in multiple additional markets. Genzyme has obtained marketing approval for KYNAMRO in the United States, South Korea, Argentina, Mexico, and Peru for use in patients with HoFH and is continuing to pursue approval in multiple additional markets. KYNAMRO is a novel, first-in-class, apo-B synthesis inhibitor for the reduction of low-density lipoprotein cholesterol. Severe and Rare Disease Franchise The company is discovering and developing antisense drugs to treat severe and rare and neurological diseases for which there is a need for new treatment options. The company’s strategic partnerships with Biogen Idec and Roche have supported advancing five drugs for the treatment of neuromuscular or neurological diseases in the company’s pipeline. Alicaforsen Alicaforsen is an antisense drug designed to target the intercellular adhesion molecule 1 (ICAM-1). ICAM-1 is over-expressed in various inflammatory disorders, including ulcerative colitis (UC) and pouchitis. The company licensed alicaforsen to Atlantic Pharmaceuticals Limited for pouchitis, UC, and other inflammatory diseases. The FDA and European Medicines Agency have since granted alicaforsen Orphan Drug Designation for the treatment of pouchitis in the United States and Europe, respectively. Atlantic Pharmaceuticals Limited is pursuing opportunities to fund further development of alicaforsen. ISIS-TTRRx ISIS-TTRRx is an antisense drug that the company designed to treat transthyretin amyloidosis (TTR) amyloidosis, a severe and rare genetic disease in which the patient inherits a mutant gene that produces a misfolded form of TTR, which progressively accumulates in tissues. The company is evaluating ISIS-TTRRx to treat two types of TTR amyloidosis, familial amyloid cardiomyopathy (FAC) and familial amyloid polyneuropathy (FAP). The company completed a Phase 1 study evaluating the safety and activity of ISIS-TTRRx in healthy volunteers. In 2013, the company initiated a randomized, double-blind, placebo-controlled 15-month Phase 3 study of ISIS-TTRRx in patients with FAP. The company plans to report data in 2017. The company’s partner, GSK, also plans to evaluate ISIS-TTRRx in patients with FAC and is in the planning stages of a Phase 3 study for this indication. ISIS-SMNRx ISIS-SMNRx is an antisense drug that the company designed to treat spinal muscular atrophy (SMA), a severe motor-neuron disease that is the leading genetic cause of infant mortality. The company designed ISIS-SMNRx to potentially treat all types of childhood SMA by altering the splicing of a closely related gene, SMN2, which leads to the increased production of fully functional SMN protein. The company developed a biomarker assay to measure levels of SMN protein in the cerebral spinal fluid of children and infants with SMA. The company is evaluating ISIS-SMNRx in a Phase 2 open-label, multiple-dose, dose-escalation study in children with SMA. It is also evaluating ISIS-SMNRx in a Phase 2 open-label, multiple-dose, dose-escalation study in 20 infants who have been diagnosed with SMA. The company is evaluating ISIS-SMNRx in two Phase 3 studies in infants and children with SMA. The company designed these studies to support marketing registration for ISIS-SMNRx in the United States and Europe. The Phase 3 study, ENDEAR, is a randomized, double-blind, sham-procedure controlled 13-month study in approximately


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Valuation ISIS Industry Range
Price/Earnings 100.0x
Price/Sales 23.6x
Price/Book 30.5x
Price/Cash Flow 528.1x
TEV/Sales 19.6x

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