Isis Pharmaceuticals, Inc. (ISIS) announced positive results from phase II studies on ISIS-SMNRx in infants and children suffering from spinal muscular atrophy (SMA). Results were presented at the 19th International World Muscle Society (WMS) Congress.
Results from the study in infants suffering from SMA revealed an increase in muscle function scores after being treated with ISIS-SMNRx in both the dosing cohorts (6 mg and 12 mg). Data did not reveal serious adverse events including death in most of the infants treated with the candidate in the study.
Results also revealed greater levels of full lengths of SMN2 mRNA and SMN protein in tissues in ISIS-SMNRx treated infants compared to those not treated with ISIS-SMNRx. ISIS-SMN is currently being developed for the treatment of infants suffering from SMA in the phase III ENDEAR study.
Results from an open label study on ISIS-SMNRx in children suffering from SMA were also announced. It revealed that mean increases in muscle function scores from baseline continued for an extended period. Moreover, increase in the six-minute walk and the upper limb mobility test were superior to previously reported data. Isis Pharma intends to initiate a second phase III (CHERISH) study on ISIS-SMNRx in children suffering from SMA later in the year.
We are encouraged by Isis Pharma’s progress with ISIS-SMNRx. The candidate is being developed in collaboration with Biogen Idec (BIIB).
Isis Pharma carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Medivation, Inc. (MDVN) and Ligand Pharmaceuticals Incorporated (LGND). Both hold a Zacks Rank #1 (Strong Buy).
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