Feb 21 Shares of Isis Pharmaceuticals
reached a new high on Friday after the company said a mid-stage
trial of its experimental treatment for a rare spinal disorder
showed that it increased muscle function in children.
Isis is developing the drug, known as ISIS-SMN, in
partnership with Biogen Idec. Isis is a leader in the
development of "antisense" drugs, which aim to interfere at the
genetic level to regulate formation of proteins.
Shares of Isis were up 15 percent at $58.82, while shares of
Biogen were up 4 percent at $348.09 in afternoon trading on
Isis said patients treated with the highest dose of the drug
had an average 3.7-point increase in muscle function after nine
months. In addition, a recently developed biomarker test showed
increases in levels of a protein critical to health and survival
of nerve cells in the spinal cord.
Spinal muscular atrophy, which affects around 35,000
patients in the United States, Europe and Japan, is caused by a
loss of, or defect in, a gene associated with the survival motor
Isis said two trial patients experienced serious side
effects, but they were not considered to be related to the drug.
"We do think these data support the bull case on the program
although there are still questions on magnitude and safety at
higher doses," UBS Securities analyst Matthew Roden said in a
research note. "However, with no effective treatment for SMA
(spinal muscular atrophy), the bar is low."
He said ISIS-SMN is not included in his earnings estimates
for Isis, but noted that bullish analysts have estimated sales
at $1 billion a year.
Isis said it plans to start a pivotal-stage trial of
ISIS-SMN in children with spinal muscular atrophy later this