Isis Pharmaceuticals, Inc.
) recently commenced a phase II study with its spinal muscular
atrophy (SMA) candidate, ISIS-SMNRx.
The open-label, multiple-dose, dose-escalation pilot phase II
study will be performed in eight infants for the treatment of
With the enrollment of the first patient which is expected in
May 2013, Isis Pharma will become eligible to receive a $3.5
million milestone payment from partner,
Biogen Idec Inc.
In Mar 2013, data from an open-label phase I study evaluating
ISIS-SMNRx in children with SMA was presented. Results showed
that ISIS-SMNRx was well tolerated at all dose levels tested.
Additionally, improvements in Hammersmith scores were also
Moreover, in Nov 2012, Isis Pharma started phase a Ib/IIa
study to evaluate ISIS-SMNRx in children with SMA. The
information from this study will aid in finding the dose for the
phase II/III registration-directed study in children with
According to information provided by Isis Pharma, around
30,000-35,000 patients in the US, Europe and Japan suffer from
SMA. According to The SMA Trust, one child out of every 6,000
births is affected by SMA.
) is developing RG3039 for the treatment of SMA. It has also
entered into a licensing agreement for the advancement of its SMA
program. RG3039 is in phase I trial - the first two arms of this
trial will be completed by Repligen.
Isis Pharma and Biogen Idec carry a Zacks Rank #3 (Hold).
Cleveland BioLabs, Inc.
) looks more attractive with a Zacks Rank #1 (Strong Buy).
BIOGEN IDEC INC (BIIB): Free Stock Analysis
CLEVELAND BIOLB (CBLI): Free Stock Analysis
ISIS PHARMACEUT (ISIS): Free Stock Analysis
REPLIGEN (RGEN): Free Stock Analysis Report
To read this article on Zacks.com click here.