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Ionis Pharmaceuticals Completes Target Enrollment for Nusinersen Phase 3 Study, CHERISH, in Children with Spinal Muscular Atrophy

CARLSBAD, Calif., Jan. 12, 2016 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (IONS) announced today that it has earned a milestone payment of $2.15 million from Biogen for completing the target enrollment of the Phase 3 CHERISH study. The study is designed to support marketing approval of nusinersen in children with spinal muscular atrophy (SMA).

"Achieving our target enrollment number in CHERISH brings us one step closer to a marketing application for children with SMA. We also plan to complete enrollment in ENDEAR, our Phase 3 study evaluating nusinersen in infants with SMA, in the first half of this year. This progress sets the stage for Phase 3 data from both of these programs in 2017," said B. Lynne Parshall, chief operating officer at Ionis Pharmaceuticals. "We would like to thank the parents and families for their participation in CHERISH as well as the investigators and sites for the achievement of this milestone. We also appreciate the support from the broader SMA community. We, along with Biogen, are committed to advancing nusinersen toward the market as rapidly as possible."

CHERISH, a Phase 3 study of nusinersen, is a randomized, double-blind, sham-procedure controlled fifteen month study in approximately 117 children who are non-ambulatory with SMA between the ages of 2 to 12. The study will evaluate the efficacy and safety of 12 mg doses of nusinersen with a primary endpoint of a change in the Hammersmith Functional Motor Scale-Expanded (HFMSE), a validated method to measure changes in muscle function in patients with SMA. Additional efficacy endpoints are also included in the study. For further study information, please visit and search for nusinersen or the identifier number NCT02193074 or visit the nusinersen study site at

SMA is a severe genetic disease that affects approximately 30,000 to 35,000 patients in the United States, Europe and Japan. There are no approved treatments for SMA. The disease is caused by a loss of, or defect in, the survival motor neuron 1 (SMN1) gene, leading to a decrease in the survival motor neuron (SMN) protein. SMN is critical to the health and survival of nerve cells in the spinal cord that are responsible for neuromuscular growth and function. One in 50 people, the equivalent of about six million people in the United States, are carriers of a defective SMN1 gene, which is unable to produce fully functional SMN protein. Carriers experience no symptoms and do not develop the disease. However, when both parents are carriers, there is a one in four chance that their child will have SMA.

Natural history studies have been conducted in patients with SMA. Type I is the most severe form of SMA and most infants with Type I SMA die in infancy. In a 2009 paper by Rudnik-Schoneborni, the median age for event-free survival in infants with Type I SMA was 6.1 months. In a contemporaneous study published in 2014 by the Pediatric Neuromuscular Clinical Research group (PNCR)ii, the median age for event-free survival in infants with two copies of SMN2 was 10.5 months. The severity of SMA correlates with the amount of SMN protein. Infants with Type I SMA produce very little SMN protein and have a life expectancy of less than two years. Children with Type II have greater amounts of SMN protein but still have a shortened lifespan and are never able to walk. Children with Type III have a normal lifespan but accumulate life-long physical disabilities as they grow.