The open-label, multiple-dose, dose-escalation pilot phase II study will be performed in eight infants for the treatment of SMA.
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. (BIIB).
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 observed.
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 SMA.
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.
Repligen Corp. (RGEN) 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). Right now, Cleveland BioLabs, Inc. (CBLI) looks more attractive with a Zacks Rank #1 (Strong Buy).
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