Isis Pharmaceuticals initiates Phase 3 Study of ISIS-SMN Rx in infants with spinal muscular atrophy


Isis Pharmaceuticals has announced the initiation of a pivotal Phase 3 study evaluating ISIS-SMNRx in infants with spinal muscular atrophy. The company 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 spinal muscular atrophy later this year.

ENDEAR, a Phase 3 study of ISIS-SMNRx, is a randomised, double-blind, sham-procedure controlled thirteen month study in approximately 110 infants diagnosed with spinal muscular atrophy. The study will evaluate the efficacy and safety of a 12mg dose of ISIS-SMNRx with a primary endpoint of survival or permanent ventilation. Additional efficacy endpoints are also included in the study.

“The successful advancement of ISIS-SMNRx from a preclinical drug candidate to late-stage studies within just a few years reflects the effectiveness of our strategic alliance with Biogen Idec and the benefit of working closely together with combined expertise, and the support from the spinal muscular atrophy community. As we continue development of ISIS-SMNRx and initiate the two pivotal studies in infants and children with spinal muscular atrophy, we are in the planning stages for clinical studies in additional patient populations,” says B Lynne Parshall, chief operating officer at Isis. “The clinical and preclinical data we have generated to date, including data in multiple open-label clinical studies, across multiple measures with ISIS-SMNRx, support the initiation of these studies, which fully assess the safety and efficacy benefits of this experimental treatment.”