The Wall Street Journal
By JONATHAN D. ROCKOFF
Pfizer Inc. is partnering with a San Diego biotechnology firm in an unusual early-stage drug-development deal that could be worth as much as $210 million and bolster the pharmaceutical giant’s new push into rare diseases, the companies said.
The collaboration with Zacharon Pharmaceuticals Inc. provides Pfizer access to technology for identifying drugs to treat genetic diseases called lysosomal storage disorders that cause mental retardation and lead to early death. Zacharon has identified molecules that have shown promise during testing in animals and test tubes. Pfizer plans to help further develop the compounds, and assist looking for others, the companies said.
The deal, which is expected to be announced Thursday, is the first by a new Pfizer division established to work on treatments for orphan and genetic diseases, said Ed Mascioli, the division’s chief.
“They clearly have expertise that we don’t have,” Dr. Mascioli said of Zacharon. He said the Pfizer unit, which has 20 researchers, is “actively” looking for more such collaborations.
Tie-ups between Big Pharma and small biotechs are not uncommon, but this deal is unusual in that larger drug makers have been loath to sign up at such an unproven, early stage. The number of so-called phase 1 early-stage compounds that haven’t found a Big Pharma patron rose to 904 in 2009, up from 233 in 2000, according to Defined Health, a biopharmaceutical industry business development and strategy firm.
But drug giants are also competing to gain a foothold in the treatment of rare diseases. Once a no-man’s land for the companies because the potential market was deemed too small, rare diseases have become increasingly attractive as big drug makers seek to replace aging blockbuster drugs.
The recent mapping of the human genome has identified potential targets for new drugs, Dr. Mascioli said. In addition, therapies for rare diseases can win quick regulatory approval, command high prices and require less marketing, industry experts added. The National Institutes of Health classifies a disease as rare, or orphan, if it affects less than 200,000 people in the U.S.
Since 2005, about a quarter of the new drugs approved each year aim to treat orphan diseases, according to the U.S. Food and Drug Administration.
Notably, French drug maker Sanofi Aventis SA recently agreed to pay at least $20.1 billion for Genzyme Corp., which sells treatments for lysosomal storage disorders including Gaucher’s and Fabry diseases. British pharmaceutical company GlaxoSmithKline PLC has been lining up partners for a rare diseases unit it launched last year.
Some in the industry caution that Big Pharma shouldn’t expect the moves to provide immediate relief for their bottom lines. “To accumulate the associated knowledge, like anything in pharma, takes a lot of years of experience and expertise,” said Angus Russell, chief executive of Shire PLC, a U.K. drug maker that sells treatments for lysosomal storage disorders and other rare diseases, the result of buying Transkaryotic Therapies in 2005.
The Pfizer-Zacharon collaboration will focus on six conditions resulting from a genetic defect that makes it difficult for the body’s cells to dispose of waste, which then dangerously accumulates.
Some of the diseases are currently treated by so-called enzyme-replacement therapies delivered intravenously. These infusions can relieve many symptoms, but not the neurological effects, because the therapies can’t cross the blood-brain barrier, according to the two companies.
Zacharon’s technology can identify compounds that can be made into pills with a therapeutic impact on the brain, said Robin Jackman, the firm’s chief executive. Zacharon is a closely held company whose sole institutional investor is venture capital fund Avalon Ventures, Mr. Jackman said.
Under terms of the deal, Pfizer will provide an unspecified upfront sum and drug-development funding, along with payments if development, regulatory and commercialization milestones are met. Zacharon could also receive additional royalty and sales milestones payments if any drugs go on sale.