GTC Biotherapeutics Drug Passes Clinical Milestone, Helping Blaze the Trail for U.S. “Pharming”

2/5/08

GTC Biotherapeutics (NASDAQ: GTCB), which bioengineers goats to secrete drugs in their milk, announced late yesterday that its groundbreaking medicine for a rare blood-clotting disorder cleared the main hurdle in a pivotal U.S. trial. The advance paves the way for the Framingham, MA-based company to seek FDA approval for the drug around mid-year, potentially making it the first medicine from “pharming”—manufacturing drugs in bioengineered animals and plants—to reach the U.S. market.

In 2006, the drug ATryn was approved in Europe to treat the same clotting disorder, making it the first pharmed medicine to garner regulatory endorsement. GTC’s European partner, France’s LEO Pharma A/S, launched ATryn last year in the U.K. to treat the rare condition, hereditary antithrombin deficiency, or HD, in patients undergoing high-risk surgical procedures.

GTC’s clinical-trial news was expected and had little impact on its stock price—GTC was trading at $1.06, up 4 cents a share, soon after the market opened this morning. The company’s shares have languished under a dollar in recent months, prompting the Nasdaq last month to notify GTC that its stock might be delisted. It hasn’t helped GTC’s stock price that the market for the rare clotting disorder is very small. In fact, the U.S. trial required only 31 patients.

But if GTC succeeds in getting FDA approval for ATryn, which may occur near the end of 2008, the regulatory path for pharmed drugs will have been blazed, potentially facilitating FDA approval of ATryn for more prevalent disorders and opening the door for other pharmed drugs. GTC and other pharming startups have long dreamed of revolutionizing biotech manufacturing by mass-producing protein drugs in goats and other animals whose DNA is implanted with human genes.

The clinical advance is also expected to clear the way for GTC to form a partnership to commercialize ATryn in the U.S. The company earlier said it plans to conclude a partnership agreement during this year’s first quarter—stay tuned.

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