“Nusinersen already exceeds what is necessary for approval”

In June 2015, after the release of phase 2 data for Nusinersen, Neeham & Company senior analyst for Biotechnology, Dr. Chad Messer Ph.D, boosted his price target on shares for Ionis Pharmaceuticals (NASDAQ: Ionis) saying that shares did not fully reflect the recent SMA Data. Hesser commented, “Ionis recently presented updates from two ongoing Phase II studies with Nusinersen. We remain impressed with the efficacy of these studies and believe investors do not fully appreciate their significance. Specifically, investors may be confused about how sick Spinal Muscular Atrophy (SMA) patients are. The sickest Type I SMA infants have a life expectancy of 10.5 mo vs 16.7 mo (and counting) in an ongoing Phase II trial. Importantly, Nusinersen has corroborated these survival improvements with meaningful functional improvements in CHOP INTEND in infants and HFMSE in children. Phase III studies are well underway but we reiterate our belief that the efficacy demonstrated so far by Nusinersen already exceeds what is necessary for approval in a deadly pediatric orphan indication.”

Nusinersen has shown efficacy in Phase II trials, continuing placebo trials in a terminal disease like SMA is ignoring the desperate need for a treatment for these patients.


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