By Matthew Willett

Genzyme Transgenics Corp., the company that's logged many firsts in transgenic compound production techniques, became the first company to achieve successful results of Phase III trials of a transgenically produced product sufficient to support FDA filing.

The news firmly positions the Framingham, Mass., company to file a biologics license application early in 2001 for its recombinant human antithrombin III (rhATIII), which is produced in the milk of goats, the company said.

The company said Thursday it met the primary endpoints in a second Phase III trial evaluating the ability of rhATIII to restore heparin sensitivity and assist in managing coagulation during cardiopulmonary bypass surgery (p<0.001).

The second Phase III trial endpoints were consistent with the results of an identically structured Phase III trial announced in January. (See BioWorld Today, Jan. 11, 2000, p. 1.)

Genzyme's stock (NASDAQ:GZTC) rose $2.375 Thursday, closing at $35.50.

GTC President and CEO Sandra Nusinoff Lehrman said the news cements the company's leadership position in the transgenic production market and opens avenues for manufacturing collaborations in the genomics market.

The Genzyme technology puts transgenically modified goat mammary glands to work producing the rhATIII, and could be used for large-scale production of any number of proteins and monoclonal antibodies, Genzyme said.

"We believe that we will see coming from genomics corporations like [Millennium Pharmaceuticals Inc. and Human Genome Sciences Inc.] a number of proteins or protein receptors that could be very important for the next generation of therapeutics," Lehrman said. "There's not a lot of capacity available to manufacture these proteins for the number of patients that need them, and we believe that Genzyme can be the bridge between the technology and the companies in bringing these compounds forward to patients."

The market for genomics therapeutics, including proteins and monoclonal antibodies, could approach a production bottleneck in the future, with up to 500 possible targets awaiting development and little facility beyond Genzyme's transgenic goat production technique to fill the demand, she said.

"We can say there is a lot of enthusiasm about antibodies," she said. "The top six of nine antibodies that have been approved sold $1.3 billion to $1.5 billion in 1999, and we consider that the tip of the iceberg, since there are between 200 and 500 antibodies out there and more than 20 in the clinic right now."

Genzyme director of investor relations Tom Newberry said genomics therapeutic production is an issue that has not yet been addressed sufficiently.

"There are a number of monoclonal antibodies and specialized proteins that will be new candidates or in testing, and eventually some will make it forward as drugs, and we see it's a problem to get those drugs in a commercial-size volume," he said. "It's an issue for investors and particularly for patients to have the capacity to produce those out of what's essentially the best production facility in the world, a mammary gland."

Partners for the technology will fit into the company's strategy for the future, he said.

Meanwhile, he said, the company is focused on the recently received data. In the upcoming months it plans to present the trial data for peer review and communicate with the FDA concerning the structure of the BLA Genzyme plans to submit in early 2001.

"We've just got the data and we'll discuss with the FDA about what the package will look like, but we're currently anticipating an early 2001 filing," Lehrman said.

Genzyme Transgenics has a joint venture with Genzyme General called ATIII LLC for development, marketing and distribution of the product.