Ionis Releases Data Update from Phase 2 Study in Infants with SMA

Ionis Pharmaceuticals provided an update on its ongoing open-label Phase 2 clinical study of nusinersen in infants with spinal muscular atrophy (SMA) at the American Academy of Neurology (AAN) meeting.

The data reported show that there have been no new events, as defined by progression to permanent ventilation or death, in the study since December 2014 with continued increases in event-free survival, muscle function scores as well as achievement of new developmental milestones. Data showing increases in neuromuscular electrophysiology measurements were also reported. The latest analysis also demonstrates that no nusinersen-related safety or tolerability concerns have been identified.

“We remain very encouraged with the performance of nusinersen. We and Biogen are committed to advancing nusinersen as rapidly as possible. Together we are actively preparing for potential filing and commercial launch of nusinersen. We have completed enrollment in CHERISH, the Phase 3 study in children with SMA and are nearing completion of enrollment in ENDEAR, the Phase 3 study in infants with SMA. This progress places us on track to have data from both of these controlled, Phase 3 studies in the first half of 2017,” said B. Lynne Parshall, chief operating officer at Ionis Pharmaceuticals.

On Thursday, April 21, 2016, Ionis will conduct a webcast to discuss the latest data presented at the AAN meeting, including the nusinersen Phase 2 study data.

A previous update on nusinersen (IONIS-SMNRx) released by Biogen and Ionis was posted in our news section in January 2016. This update provided general information on the clinical development of nusinersen.

Cure SMA Seed Funding

From 2003 to 2006, Cure SMA provided the seed funding needed to begin investigation into this therapeutic approach.

Several Cure SMA grants totaling over $500,000 were given to U Massachusetts, to help develop the intellectual property needed to investigate antisense oligonucleotides for SMA. The intellectual property generated with our funding was then licensed to Ionis Pharmaceuticals to create IONIS-SMNRx.

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