* GALNS FDA filing expected by end of March
* Biggest seller could be cancer drug
By Deena Beasley
SAN FRANCISCO Jan 8 BioMarin Pharmaceutical Inc
, which makes high-priced drugs used to treat rare
diseases, would not consider a buy-out offer for even a 25-30
percent premium, according to the company's chief executive
"The best way to prevent that (a takeover) is to keep the
stock up, and we are doing that," CEO Jacques Bienaime said
during an interview at the JP Morgan Healthcare Conference in
He said any decision to sell would come from large
shareholders, not him nor the board.
Big pharmaceutical companies are seeing patents expire on
many of their older, best-selling products, which will spur
competition from lower-priced generic drugs. Many of these
companies are interested in drugs for rare diseases, which can
command prices well over $100,000 a year. Sanofi's 2011
acquisition of Genzyme is the largest such takeover deal so far.
But Bienaime questioned whether it makes sense for
traditional large pharmaceutical companies to enter the market
for so-called "orphan drugs."
"They don't understand this," he said, adding that much of
the talk at big pharma surrounding orphan drugs is on the
scientific side - not the business side.
And, given the increased focus on rising healthcare costs,
he said it is less politically feasible for a big pharma company
to launch a drug with an annual price tag of $400,000 than it is
for smaller company such as BioMarin.
The company, which generates annual revenue near $500
million, expects to file by the end of March for U.S. approval
of GALNS, an enzyme replacement therapy designed to treat
Morquio A syndrome, a rare, inherited disorder that causes
skeletal dysplasia, short stature and joint abnormalities. There
is currently no approved treatment for the disease.
The launch of GALNS would double sales at BioMarin to $1
billion a year, said Bienaime.
More than a quarter of the 39 new medicines approved in the
United States last year were designated for such conditions.
BioMarin's market capitalization is near $5 billion. It now
has four drugs on the market, two of which treat other metabolic
disorders caused by the absence of certain enzymes.
But the company's biggest product could be a cancer drug
currently known as BMN673. It belongs to a class of drugs called
PARP inhibitors that help kill cancer cells by blocking an
enzyme important in repairing DNA damage.
"It is by far the most potent PARP inhibitor out there,"
Bienaime said during an interview here at the JP Morgan
Healthcare Conference, noting that around 8 percent of breast
cancer patients diagnosed each year have the genetic mutation
targeted by PARP inhibitors.
The company expects to present data from Phase 2 trials of
the drug, including breast and ovarian cancer, in late May or
early June at the annual meeting of the American Society for
Bienaime said BioMarin has the financial ability to develop
the cancer drug on its own.
"We have no plans to change our strategy. If you have the
only therapy, you have pricing leverage," he said.
Bienaime also said that the cost to the healthcare system of
orphan drugs is less than it might seem. "These patients are not
going away," he said. "They require very expensive care."
The CEO estimated that the total market for the "ultra
orphan drugs" coveted by BioMarin - meaning treatments aimed at
patient populations of under 10,000 - now totals around $5