Isis Begins Phase III Clinical Trial

On August 1, Isis Pharmaceuticals, Inc. announced today the initiation of a pivotal Phase III study evaluating ISIS-SMNRx in infants with spinal muscular atrophy (SMA). The Phase III 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.

“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 SMA community. As we continue development of ISIS-SMNRx and initiate the two pivotal studies in infants and children with SMA, we are in the planning stages for clinical studies in additional patient populations,” said 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.”

“Cure SMA is pleased that Isis is advancing to the next phase of clinical trials for ISIS-SMNRx. Controlled trials are the gold standard in proving the safety and efficacy of any drug,” said Kenneth Hobby, president of Cure SMA. “SMA is a devastating disease with no current therapeutic options. Cure SMA applauds Isis for progressing its development program in an expedient manner, and looks forward to additional trials in patients with SMA beginning later in 2014.”

This is the first ever Phase III trial for a new drug developed to treat the underlying cause of SMA in the US, with original funding from Cure SMA. ENDEAR, a Phase III 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. Additional efficacy endpoints are also included in the study.

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