(Adds data from infant trial, updates share prices)
Feb 21 Shares of Isis Pharmaceuticals
reached a new high on Friday after the company said early trials
of its experimental treatment for a rare spinal disorder showed
that it increased muscle function in children and kept infants
with the most severe form of the disease alive and off
Isis is developing the "antisense" drug, known as
ISIS-SMNRx, in partnership with Biogen Idec to treat
spinal muscular atrophy. Isis is a leader in the field of
antisense, which aims to interfere at the genetic level to
regulate formation of disease-causing proteins.
Shares of Isis rose 16 percent to close at $59.00 on the
Nasdaq exchange. Shares of Biogen gained 3.6 percent to close at
Spinal muscular atrophy (SMA), 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 children with SMA 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.
It also reported interim results from a trial of infants
with type 1 SMA, showing that all four babies in the low-dose
arm of the study were alive without needing respiratory
assistance after being in the study for over six months. The
four have an average age near 12.5 months.
Typically, about half of infants born with the most severe
form of SMA will not make it to their first birthday and most
will not make it to their second birthday, Dr. Tom Crawford,
professor of neurology and pediatrics at Johns Hopkins
Children's Center and a study investigator, said in a statement.
Isis said one infant in the high-dose arm of the trial died
due to pneumonia early in the treatment portion of the study.
In the trial of older children, two 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..., the bar is low."
He said ISIS-SMNRx 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 the
antisense drug in infants with spinal muscular atrophy in the
middle of this year and a similar study in children later this
The company said it will present additional details from the
earlier-stage SMA trials at upcoming medical conferences.
(Reporting By Deena Beasley; Editing by Jonathan Oatis and