March 21, 2000
Collateral Therapeutics Trial
Produces Promising Results
By RON WINSLOW
Staff Reporter of THE WALL STREET JOURNAL
Collateral Therapeutics Inc. said its experimental gene therapy for chest pain
caused by heart disease proved to have beneficial effects in a small clinical
trial, sending its shares soaring 56%.
If the preliminary evidence of what the company sketchily described as
"very positive biological effects" is borne out when all the data are fully
analyzed, the study will be among the first so-called placebo-controlled trials
of any type of gene therapy to show benefit to patients.
Shares of Collateral Therapeutics, based in San Diego, rose $14.4375 to
$40.1875 in 4 p.m. trading on the Nasdaq Stock Market Monday. The
company is developing the treatment with Schering AG of Germany.
Exercise Improvement
In the trial, involving a total of 67 patients, the company said 13 patients
given a certain dose of the treatment averaged a 30% improvement in the
amount of time they were able to walk on an exercise treadmill 12 weeks
later -- the principal measure of effectiveness.The company didn't disclose
the relative benefit compared with 16 patients on placebo, or dummy
treatment -- declining even to specify precisely which one of five different
doses was effective -- but it said the difference was considered statistically
significant.
Another 38 patients were given one of the four other doses of the treatment,
which the company calls Generx. Their exercise treadmill tests were, on
average, better than those given a placebo, but the differences weren't
statistically significant, the company said. The treatment, intended to spur the
heart to grow its own vessels around blocked arteries -- a process called
angiogenesis -- appeared to be safe at all doses, the company said.
Tim Henry, a cardiologist and angiogenesis researcher at Hennepin County
Medical Center, Minneapolis, Minn., who wasn't involved in the Collateral
Therapeutics study, called the result "encouraging." But in such a small
study, "it's really hard to tell" whether the therapy is working or not, he said.
"It needs to be confirmed in a larger trial."
Plan for Large Trial
The company plans to meet with U.S. regulators to plan a large-scale trial
needed to win marketing approval for the therapy, said Robert L. Engler, a
co-founder of Collateral Therapeutics and a researcher at University of
California at San Diego. It expects to apply for such a trial in the fourth
quarter, he added.
The company issued sketchy details Monday, Dr. Engler said, because the
findings that were available were considered material to the company's
financial position.
The company's treatment involves the gene for a substance called fibroblast
growth factor-4, or FGF-4. It is encased in an inactive virus called an
adenovirus and delivered to the coronary arteries via a catheter that is
threaded into the heart from the groin. Researchers believe that the virus
delivers the gene to cells, which use it to become minifactories for FGF-4.
The growth factor then stimulates the body's own natural tendency to grow
new blood vessels to circumvent blockages in the coronary arteries that
cause chest pain called angina.
As previously reported, one of the 67 patients in the trial died about five
months after the one-time administration of the treatment. The company
reiterated Monday that evaluations by the company and by outside
investigators determined that it is unlikely the death resulted from the
treatment.
Write to Ron Winslow at ron.winslow@wsj.com |
