Isis gains on SMA data
Isis Pharmaceuticals Inc. (NASDAQ:ISIS) was up $4.47 (14%) to $36.27 on Thursday after reporting additional data from a Phase I trial evaluating single intrathecal injections of ISIS-SMNRx to treat spinal muscular atrophy (SMA). At 9-14 months post-treatment, Isis said "most" patients who received the two highest doses of ISIS-SMNRx showed improvement in muscle function, with no patient who received the highest dose experiencing a decline in muscle function. The trial enrolled 28 medically stable SMA patients aged 2-14 years.
Data from a Phase II trial in infants and a Phase Ib/IIa trial in patients age 2-15 are expected in late 2013 or early 2014, with Phase III testing slated to begin early next year. The product is an antisense oligonucleotide that modulates splicing of survival of motor neuron 2 centromeric (SMN2) mRNA. Biogen Idec Inc. (NASDAQ:BIIB) has an option to ISIS-SMNRx from Isis under a 2012 deal (see BioCentury, July 2, 2012). ...