) 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
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
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
). 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.
). While Biogen and Alexion are Zacks Rank #1 (Strong Buy)
stocks, Alkermes is a Zacks Rank #2 (Buy) stock.
ALKERMES INC (ALKS): Free Stock Analysis
ALEXION PHARMA (ALXN): Free Stock Analysis
BIOGEN IDEC INC (BIIB): Free Stock Analysis
ISIS PHARMACEUT (ISIS): Free Stock Analysis
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