Drugs / ISIS 333611
last change Mar 2013 re-read 3 minutes ago

ISIS 333611

Antisense oligonucleotide targets SOD1 via inhibition

Developed for
familial amyotrophic lateral sclerosis
Investigated by
ALS Association · Ionis Pharmaceuticals, Inc. · Muscular Dystrophy Association

Trials 1

201020112012201320142015201620172018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1 NCT01041222 Jan 2010 → Dec 2011 familial amyotrophic lateral sclerosis Ionis Pharmaceuticals, Inc. Completed Met primary

News releases announcing trial results or a regulatory action · 1

DateIssuerRelease
2007-12-13 Ionis Pharmaceuticals Regulatory Isis Announces ISIS 333611 Granted Orphan Drug Status for Treatment of ALS ionis.com ↗
Isis Pharmaceuticals, Inc. (Nasdaq: ISIS) announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISIS 333611 for the treatment of an inherited form of amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig's disease.

All press releases naming this drug 1 release

DateIssuerRelease

Evidence & citations 5 cited values

Every value below carries the sentence it was read from. 2 sources stand behind the page.

FieldValueCited text
Known as ISIS 333611 ClinicalTrials.gov intervention name — accepted as the source's own label NCT01041222 ↗
Action Inhibit “the antisense oligonucleotide ISIS 333611 delivered to CSF decreased SOD1 mRNA and protein concentrations” PMID 23541756 ↗ Mar 2013
Modality Antisense oligonucleotide “An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis” PMID 23541756 ↗ Mar 2013
Route Intrathecal “we delivered ISIS 333611 by intrathecal infusion” PMID 23541756 ↗ Mar 2013
Target SOD1 “An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis” PMID 23541756 ↗ Mar 2013