BioMarin Halts Trials of Neutralase Heart Drug

BioMarin Pharmaceutical Inc., an unprofitable biotechnology company with one drug on the market, said it stopped testing of its experimental heart medicine Neutralase because of safety concerns.

Neutralase, given to bypass surgery patients to reduce bleeding, was in the final stage of human trials when an independent drug safety group concluded there was a “high probability” that BioMarin’s medicine wouldn’t prove to be safe or effective, the company said.

BioMarin had predicted the drug might have as much as $500 million in sales after a 2007 introduction, said Joshua Grass, a spokesman for Novato, Calif.-based BioMarin. The decision removes an advanced product from the company’s pipeline, said Richard Jarvis, a Pictet & Cie analyst, who downgraded his rating on the shares to “reduce” from “neutral” Monday.

“They saw this as the largest top-line potential product in their portfolio,” said Jarvis, who doesn’t own BioMarin shares. “This is a big surprise for investors and for the company.”

Shares of BioMarin fell 66 cents, or 7.7%, to $7.93 on Nasdaq, after touching $7. They have risen 12% this year.

BioMarin, which in April won approval of its first drug, Aldurazyme, narrowed estimates for its 2003 net loss by $5 million and shaved $13 million from its expected 2004 net loss because of the halt in spending on Neutralase development.

“We’re clearly disappointed by the results, but we think that with the products we have in the portfolio, our forecast of attaining profitability in 2006 remains unchanged,” Fredric Price, chief executive of BioMarin, said.

Researchers planned to enroll 600 heart-bypass surgery patients in the Neutralase trial, and stopped at 150. The safety board found that adverse events associated with bypass surgery, such as another hospitalization, occurred more frequently in patients taking Neutralase than in those on standard therapy, said Emil Kakkis, the company’s senior vice president for scientific affairs.

In April, BioMarin won U.S. approval for Aldurazyme, a treatment for a rare genetic disorder called MPS 1, which analysts estimate could generate $500 million in peak annual sales. People with the disorder lack an enzyme needed to prevent a kind of sugar from building up in cells.

BioMarin has “other promising product opportunities, including Aryplase,” which is in final-stage testing, Price said. Aryplase treats Maroteaux-Lamy Syndrome, a disease that stems from the lack of an enzyme that normally helps cells clean out debris.

The company estimates the market for Aryplase will be $100 million annually. BioMarin plans to file for U.S. approval of the drug a year from now and expects a ruling within 12 months because the drug has been granted fast-track status by the Food and Drug Administration.