Shares of Isis Pharmaceuticals (ISIS) shot up 15.5% with the company reporting positive top-line results from an ongoing mid-stage study being conducted in children with spinal muscular atrophy (SMA).

Interim results from the open-label, multiple dose study conducted on ISIS-SMN Rx showed that children on the 9mg dosage experienced an average increase of 3.7 points in muscle function score. An increase in SMN protein was also observed.

Three doses of ISIS-SMNRx - 3 mg, 6 mg or 9 mg – were administered. Children moving on to the extension study will be administered a maintenance dose of 12 mg every six months.

ISIS-SMNRx was also found to be well tolerated at all dose levels. Two serious adverse events, which not considered to be related to the drug, were reported.

The results were consistent with observations in earlier single-dose studies. Additional data will be provided at an upcoming medical meeting. Isis Pharma said that it remains on track to initiate a phase III study in children with SMA later this year.

Isis Pharma also reported interim results on ISIS-SMNRx from a phase II study being conducted in infants with SMA. Interim data from the ongoing open label, multiple dose study showed that all four infants in the 6 mg cohort remained in the study for more than six months and are now about nine and a half to 16 months old. All four are alive with none requiring permanent respiratory assistance.

Even though the data is on only four infants, the results are encouraging and support the advancement of ISIS-SMNRx into phase III studies for infants. Detailed results from the study should be out in April at the American Academy of Neurology meeting.

Isis Pharma said that it remains on track to start a phase III study in infants with SMA in mid-14.

We are encouraged with Isis Pharma’s progress with ISIS-SMNRx. The candidate is being developed in collaboration with Biogen Idec (BIIB). Isis Pharma and Biogen have four collaboration agreements – one for ISIS-SMNRx, the second for the development of antisense drugs to treat myotonic dystrophy type 1, a third for the discovery and development of antisense drugs against three novel targets for neurological disorders and the final agreement for the use of Isis Pharma's antisense technology for the development of treatments for neurological diseases.

Isis Pharma is a Zacks Rank #3 (Hold) stock. Better-ranked stocks in the biotech sector include Biogen, Alexion Pharmaceuticals, Inc. (ALXN) and Alkermes plc (ALKS). While Biogen and Alexion are Zacks Rank #1 (Strong Buy) stocks, Alkermes is a Zacks Rank #2 (Buy) stock.

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