Genzyme drug helps MS; serious side effect seen

By Julie Steenhuysen and Sitaraman Shankar

CHICAGO/FRANKFURT (Reuters) – A drug from Genzyme Corp. and
Schering helped reduce the risk of relapses in multiple
sclerosis patients, but caused a serious side effect in three
cases that led to a death, the companies said on Friday.

Genzyme and Schering have suspended dosing in the trial for
Campath, which is approved to treat a form of leukemia, while
they work closely with U.S. regulators and clinical
investigators “to ensure that a comprehensive approach is in
place to manage patient safety,” the companies said.

The side effect in the Phase II or mid-stage trial included
three cases of idiopathic thrombocytopenic purpura, or ITP, a
condition in which patients experience a low platelet count
that can result in abnormal bleeding.

One patient died from the condition. Two remaining cases
are being treated.

Genzyme and Schering said they have notified regulatory
authorities, trial sites and patients about risks. The
companies have consulted a panel of experts to advise the
companies on how to reduce risks of ITP.

The companies are in talks with the U.S. Food and Drug
Administration about what steps might be needed to protect
patient safety.

Analyst views differed widely on the results, with
Citigroup seeing the data as a strong positive for the stock,
and Deutsche Bank expressing doubt about Campath’s approval.

“This is a mixed result,” said Sal. Oppenheim analyst
Marcus Konstanti. “The drug seems very efficacious but with
severe side effects. The question is whether the companies can
bring a low dose to market or whether there are strong measures
to control side effects.”

He said the result could delay the launch of Campath in
treating multiple sclerosis beyond his earlier expectation of
2009 or 2010.

Treatments for multiple sclerosis already are under a
spotlight after the drug Tysabri, made by Ireland’s Elan Corp.
and Cambridge, Massachusetts-based Biogen Idec Inc., was pulled
from the market in February after it was linked to a rare and
potentially fatal brain disease.

“Given the recent heightened scrutiny related to Tysabri
safety concerns, we are skeptical that Campath will eventually
become a treatment for multiple sclerosis,” said Deutsche Bank
biotech analyst Jennifer Chao in a research note.

She said Campath’s safety and efficacy potential make it a
suitable cancer treatment, given the potential for saving
lives, but MS is a chronic autoimmune disease that may not
warrant the same risk of severe side effects.

Citigroup analyst Yaron Werber, however, said the results
could represent “a major advance in MS treatment.”

A Schering spokesman said the company was giving no launch
date or peak sales forecast for the drug in treating multiple
sclerosis.

Campath had sales of 63 million euros or $77 million last
year from its use in treating leukemia.

COMPARISON WITH REBIF

Schering shares reacted little to the announcement, trading
slightly higher at 51.1 euros, but underperforming a 1.3
percent rise in the DAX blue-chip index. Genzyme’s sales gained
57 cents, or .8 percent, to $72.23 in morning trade on Nasdaq.

While Campath met one of its key goals, reducing relapse,
it just missed a second, which was to show a statistically
significant reduction in the risk of progression of clinically
significant disability.

The companies said they will continue to collect both
efficacy and safety data from this Phase II trial — which
compared Campath (alemtuzumab) with Rebif, a drug from
Switzerland’s Serono — while preparing to initiate a Phase III
trial.

In Switzerland, Serono shares were also flat at 840 Swiss
francs.

Schering is keen to expand its multiple sclerosis franchise
because its flagship drug, Betaseron, is facing strong
competition from Rebif, Teva’s Copaxone and Biogen Idec’s
Avonex.