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What: Shares of Isis Pharmaceuticals (NASDAQ:IONS), a developer of in-house and partnered therapies utilizing its antisense drug-technology platform, spiked higher by as much as 20% after reporting interim results from its multiple dose study of ISIS-SMN Rx in children with spinal muscular atrophy (SMA).
So what: According to the press release from Isis, ISIS-SMN Rx was well-tolerated at all dose levels, and "consistent with single-dose observations, time and dose-dependent increases in muscle function were observed in children treated with multiple doses of ISIS-SMN Rx." Isis also notes an assay test designed to measure SMN protein in the cerebral spinal fluid showed time and dose-dependent increase in both single- and multiple-dose studies. SMN protein levels were observed to be 120% and 160% above baseline in the two highest doses nine to 14 months after first dosing. Utilizing the Hammersmith Functional Motor Scale-Expanded point scale to measure changes in muscle function for patients with SMA, the treated children in the 3 mg, 6 mg, and 9 mg cohorts achieved mean increases in HFMSE scores of 1.5 points, 2.3 points, and 3.7 points, respectively, nine months following their first dose of ISIS-SMN Rx. Isis plans to give children rolling over into an extension study a 12 mg dose every six months.
Now what: Isis is truly one of a handful of remarkable biotech companies in that it's set up with a number of development partners and also has a number of in-house drugs in its pipeline. ISIS-SMN Rx is a drug being co-developed with Biogen Idec (NASDAQ:BIIB), with Biogen holding the option to license the compound until completion of the first successful phase 2/3 study or the completion of two phase 2/3 studies. There are few therapies on the market to treat SMA, so ISIS-SMN Rx is providing genuine hope for improvement to current sufferers. Safety doesn't appear to be an issue at the moment, and its impressive half-life -- Isis mentions muscle function scores were improved up to 14 months after the first dose -- certainly gives the impression that it could make for a fantastic long-term maintenance therapy. Because of its extensive pipeline, I'd postulate that Isis could have additional upside even following today's large move higher.
Isis Pharmaceuticals may be off to the races today, but even it may have a hard time keeping up with this top stock in 2014
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Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.
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