Monitoring board: treatment 'futile'
By Terri Somers
Union-Tribune Staff Writer
2:00 a.m. February 13, 2009
Lupus patients across the nation were distraught by the news yesterday
that La Jolla Pharmaceutical stopped the clinical trial of its drug
Riquent.
An independent monitoring board looked at preliminary trial data and
determined that using the drug was "futile" in stopping deadly
symptoms of lupus, the company said in a one-paragraph news release.
"I'm sitting here at the computer reading the press release and I'm
absolutely devastated," said Kathleen Arntsen, a Vernon, N.Y., woman
with the auto-immune disease.
It is the fourth lupus drug failure in recent years, said Arntsen, a
national advocate for lupus patients. Drugs by Genelabs Technologies
and Cell Cept disappointed in late stage clinical trials for lupus, as
did Biogen Idec's cancer drug Rituxan.
There has not been a new drug approval for lupus in 50 years, leaving
doctors to fend off the disease with chemotherapy drugs and steroids.
"Every time a drug comes up, it just doesn't make it through," said
Arntsen, who runs the nonprofit Lupus Foundation of Mid and Northern
New York.
La Jolla Pharmaceutical executives have not seen the clinical trial
data, which had been blinded to them but not the monitoring panel, so
company officials had no comment yesterday.
After its own experts pore over the data, the company will have a
better idea of whether to scrap its Riquent program, or perhaps argue
that the monitoring board wasn't reading the data correctly.
The failure of the only drug in the 100-person company's pipeline sent
its shares plummeting $2.11, or 91 percent, to close at 20 cents
yesterday.
Five weeks ago, La Jolla Pharmaceutical announced that it licensed
some of the development rights for the drug to BioMarin Pharmaceutical
in a deal worth up to $289 million. Novato-based BioMarin, which has
$586 million in cash reserves, paid $15 million upfront, split evenly
into cash and an equity investment.
That company's shares were less devastated by the news yesterday,
dropping 3 percent to $18.90.
La Jolla Pharmaceutical has struggled for years to bring Riquent to
market as a treatment for episodes of kidney inflammation that result
from lupus, a disease that causes the body's immune system to run amok.
About half of the 1 million lupus patients in the United States and
Europe have these so-called renal flares. The body can handle only so
many flares before developing kidney disease, which can cause death.
This trial may have been Riquent's third strike.
The drug failed to prove effectiveness in a 1999 test, and in 2003 it
again failed to prove effective in delaying episodes of kidney
inflammation.
The company then asked the Food and Drug Administration to consider
approving Riquent for its ability to reduce certain antibodies that
play a role in the flares. That prompted the trial that came to an
abrupt halt yesterday.
In San Diego's patient advocacy group, as well as in New York, people
with lupus were excited that the tiny local company was tackling the
perplexing and complicated disease, said Hollaine Hopkins, direction
of the Lupus Foundation of Southern California.
"This is a real blow," Hopkins said.
Many patients referred to La Jolla Pharmaceutical as "the little
engine that could," because it survived the previous failures, only to
try again, Arntsen said.
Meanwhile, she and other patient advocates threw themselves into
lobbying and educational campaigns to explain that lupus is a
heterogenous disease: no two patients are alike, therefore no drug can
be expected to be a one-size-fits-
Until a new drug emerges, Arntsen, like many other lupus patients,
will continue to take drugs that shut down her immune system – drugs
that were approved for cancer or organ transplant patients – and make
all types of common infections a deadly threat.
"I'm supposed to be giving hope and encouragement to other people, but
my heart's been broken," Arntsen said.
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