Isis Pharmaceuticals, Inc. ( ISIS ) recently
presented encouraging data on its candidate, ISIS-SMN, from a phase
I study (n = 28). The data was presented at the 65th annual meeting
of the American Academy of Neurology (AAN).CYTOKINETCS INC (CYTK): Free Stock Analysis
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The open-label, single-dose, dose-escalation (1, 3, 6 and 9 mg)
study evaluated the safety, tolerability and pharmacokinetic
profile of ISIS-SMN in children with spinal muscular atrophy (SMA).
It was found that ISIS-SMN was well tolerated at all dosages.
Additionally, encouraging improvement in muscle function was also
Isis Pharma is also evaluating ISIS-SMN in a multiple-dose phase
Ib/2a study. The company expects to report data from this study by
late 2013 or early 2014. Isis Pharma intends to initiate phase
II/III studies on ISIS-SMN in infants in 2013 and children in the
first half of 2014.
Last month, Isis Pharma received a $7.5 million milestone payment
from GlaxoSmithKline ( GSK ). The payment
primarily relates to the initiation of a phase II/III clinical
study of ISIS-TTRRx. The study will span 15 months enrolling 200
patients. Isis is developing ISIS-TTRRx in collaboration with Glaxo
for the potential treatment of transthyretin (TTR)
We note that Isis Pharma's lead product, Kynamro (mipomersen
sodium), received FDA approval in Jan 2013 for the treatment of
familial hypercholesterolemia (FH). Isis Pharma's partner
Sanofi ( SNY ) is marketing the
product in the US. Sanofi is also looking to get the drug approved
in the rest of the world including Europe.
Isis Pharma currently carries a Zacks Rank #3 (Hold).
Cytokinetics, Inc. ( CYTK ) looks more
attractive in the pharma sector with a Zacks Rank #1 (Strong