Isis Pharmaceuticals has initiated a pivotal Phase III study evaluating ISIS-SMNRx in infants with spinal muscular atrophy (SMA), the most common genetic cause of infant mortality. The Phase III study entitled ENDEAR, is the first of several planned studies in a broad and comprehensive late-stage clinical development program for ISIS-SMNRx. ENDEAR 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. Additional efficacy endpoints are also included in the study. Isis plans to initiate a second pivotal study in children with SMA later this year. The company is continuing the development of ISIS-SMNRx and is in the planning stages for clinical studies in additional patient populations. The clinical and preclinical data 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.
