Date: 2014-08-01
Type of information: Initiation of the trial
phase: 3
Announcement: initiation of the trial
Company: Isis Pharmaceuticals (USA - CA), now Ionis Pharmaceuticals (USA - CA)
Product: ISIS-SMNRx
Action
mechanism: antisense oligonucleotide. ISIS-SMNRx is designed to alter the splicing of a closely related gene (SMN2) to increase production of fully functional SMN protein. TheFDA granted orphan drug status and fast track designation to ISIS-SMNRx for the treatment of patients with SMA. Isis is currently in collaboration with Biogen Idec to develop and potentially commercialize the investigational compound, ISIS-SMNRx, to treat all types of SMA. Under the terms of the January 2012 agreement, Isis is responsible for global development and Biogen Idec has the option to license the compound until completion of the first successful Phase 2/3 study or the completion of two Phase 2/3 studies.
Disease: spinal muscular atrophy (SMA)
Therapeutic area: Neuromuscular diseases - Rare diseases - Genetic diseases
Country: Canada, USA
Trial
details: ENDEAR, a Phase 3 study of ISIS-SMNRx, is a randomized, double-blind, sham-procedure controlled thirteen month study in approximately 110 infants diagnosed with SMA. The study will evaluate the efficacy and safety of a 12 mg dose of ISIS-SMNRx with a primary endpoint of survival or permanent ventilation. Subjects will be randomized 2:1 to receive ISIS-SMN Rx or a sham procedure control, respectively. (NCT02193074)
Latest
news: * On August 1, 2014, Isis Pharmaceuticals announced the initiation of a pivotal Phase 3 study evaluating ISIS-SMNRx in infants with spinal muscular atrophy. Isis plans to dose the first infant in this study within the next few weeks, at which time Isis will earn an $18 million milestone payment from its development partner, Biogen Idec. The Phase 3 study, ENDEAR, is the first of several planned studies in a broad and comprehensive late-stage clinical development program for ISIS-SMNRx. Isis plans to initiate a second pivotal study in children with SMA later this year.
