Robin Jackman, Zacharons president and CEO, is the first to
admit that there arent too many people outside the biotech world
who understand the science at the core of his small company.
Glycobiology, to a lot of people, is a new word, Jackman said,
referring to the study of how sugars or, more specifically, glycan
molecules that are complex sugar polymers in the body influence
biological events such as diseases. But its an area that has
enormous therapeutic potential.
Fortunately for Zacharon, the folks at New York-based
pharmaceutical giant Pfizer Inc. not only understand this science,
but see clear market potential for the platform Zacharon has
developed for targeting glycans to treat a range of rare and
life-ending genetic disorders.
In a deal announced April 7, Pfizer agreed to pay Zacharon $210
million through a combination of upfront payments and
research-and-development funding. Zacharon also is eligible for
additional payments for meeting development milestones, plus
royalties and sales milestones after the drug reaches the
commercial market.
Zacharon was actively seeking a partner that would be able to
take its small-molecule therapies through clinical testing and into
medical settings, Jackman said. Pfizer, which started up a business
unit last summer to focus on orphan (or rare) diseases, happens to
specialize in small-molecule medicines and was also looking for a
partner with commercial promise.
A Significant Need
Were going to have the Zacharon scientists continue to do what
they do best, and Pfizer will be doing all of the other things that
must be done to develop a drug, said Ed Mascioli, head of Pfizers
new Orphan & Genetic Diseases Unit. That includes making sure
the drug is safe, is being absorbed into the bloodstream as
intended, and has all of the other properties a drug must have
before its given to patients, Mascioli said. He estimated that
clinical trials could begin in three years, with commercialization
happening two years after that.
Clearly, both of us are interested in moving this forward as
fast as possible, Jackman said. Zacharon will hire two new
employees to help speed the development, bringing its full-time
staff to 14. The clinical need is pretty significant.
The diseases that Zacharon targets lysosomal storage diseases
are very rare, but their diagnoses are bleak. People affected are
infants and small children, and the disorders can cause mental
retardation and death at a young age, Jackman said.
Current treatments involve enzyme replacement, which is done
through an IV. But enzymes are too large to enter the brain, and
thus cannot help neurological symptoms. Also, the
enzyme-replacement approach doesnt work for all lysosomal storage
diseases.
Small-molecule drugs, on the other hand, can be taken in pill
form and break the so-called blood-brain barrier, Jackman noted.
And Zacharons platform shows promise for helping a whole range of
diseases, including rare cancers.
Years of Hard Work Paying Off
The Pfizer deal is very validating to the company, said Jay
Lichter, Zacharon chairman and a member of Avalon Ventures, the
venture capital firm that has provided most of the funding for
Zacharon. It confirms everything weve been trying to do for the
last three or four years.
Lichter, who served as chief executive officer of Zacharon in
2008 and early 2009, said its encouraging that existing
enzyme-replacement therapies for lysosomal storage diseases are
showing sales of $500 million to $600 million annually. Its clear
that the market is there, he said. Insurance companies are willing
to pay a premium if youre able to improve the quality of life for
these kids.
He noted that theres been greater interest recently among big
pharmaceutical companies to pursue drugs for orphan diseases, in
part because there are fewer regulatory hurdles and less
competition. With a drug that treats a chronic disease such as
diabetes, the U.S. Food and Drug Administration requires two
well-controlled clinical trials. But with diseases affecting fewer
than 200,000 people in the U.S., only one trial is needed. When the
alternative is death, you can put up with more side effects, he
said.
Jeffrey Esko, a company co-founder who is now a professor in the
Department of Cellular and Molecular Medicine at UC San Diego,
called the Pfizer deal a huge step for Zacharon.
To have a partner thats one of the largest pharmaceutical
companies in the world, thats something we dreamed about, said
Esko, who serves as a scientific adviser to Zacharon. It shows that
Zacharons approach is a recognized strategy.
Kelly Quigley is a freelance writer for the San Diego Business
Journal.
