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Sunday, 06/18/2006 4:05:31 AM

Sunday, June 18, 2006 4:05:31 AM

Got goat milk?

[GTCB is featured in today’s Framingham, MA Daily News.]

http://www.metrowestdailynews.com/businessNews/view.bg?articleid=133110

>>
By Andrew J. Manuse
Daily News Staff
Sunday, June 18, 2006

GTC Biotherapeutics Inc. has about 1,350 goats on its Charlton farm, but the Framingham-based biotech company isn’t making feta cheese.

About 20 percent of the goats are "transgenic," meaning their genetic structure has been altered with foreign DNA, and in their milk, they produce biologic drugs designed to treat an anticlotting deficiency in humans.

Since the transgenic goats’ milk is not for consumption -- at least not by mouth -- GTC needs the other goats, which include 75 to 80 bucks, or male goats, to help produce and nurture the kids.

GTC has been working on producing drugs inside mammalian milk since 1991, right after the company spun-off from Genzyme Corp. of Cambridge, and set up its central Massachusetts farm in 1994. Dr. Harry Meade, the founding scientist of the technology, who is now GTC’s chief scientific officer, came up with the idea even earlier, and the whole process has been ongoing for 20 years.

Now, the company is within three months of validating its scientific process with European approval of its first drug from transgenic animals. The scientific committee of the European Medicines Agency on June 2 recommended approval of ATryn, GTC’s drug for patients with a genetic deficiency for an anticlotting blood protein. In the United States, GTC is involved in Phase III clinical trials with ATryn and expects to file for approval of the drug with the Food and Drug Administration by mid-2007.

"The people of GTC are pioneers in the application of transgenic technology to the production of therapeutic proteins," said Dr. Geoffrey Cox, the company’s chief executive and chairman, in an e-mail. "The successful completion of the (European) review process is much more than a validation of the technology, it is an affirmation of the vision and persistence of our people."

In the United States, about 60,000 people who lack the anticlotting protein antithrombin in their blood because of a genetic defect could benefit from ATryn if it’s approved by the FDA, said Thomas Newberry, a spokesman for GTC. The defect affects between one in 3,000 to 5,000 people.

Because of the hereditary problem, these patients develop blood clots that often build up in their legs, a condition called thrombosis which is very painful, Newberry said. The clots can break off and flow to another part of the body, a condition called embolism, and can be fatal if the clots get lodged in the heart or brain, he said.

To prevent thrombosis and embolism, patients take a drug called Warfarin to prevent the blood from clotting at all. This becomes a problem during surgery or pregnancy, because patients can bleed to death, and that’s where ATryn comes in.

Currently, patients are given the antithrombin protein from blood donors’ plasma, but there is an insufficient and unreliable supply of the protein, for various reasons, Newberry said. Once GTC’s drug is approved, patients will have a steady, safe and plentiful supply of the protein, he said.

But if GTC gets approval of ATryn for use in human treatment, the approval will not just affect the one drug. The acceptance of ATryn would also validate the use of transgenic animals to produce biologic pharmaceutical products, Newberry said.

Other companies already produce biologic products using extremely expensive devices, called bioreactors, he said. Abbott Laboratories’ uses bioreactors to produce its psoriatic arthritis drug, Humira.

GTC’s method is less expensive "by a factor of 10," Newberry said.

In the bioreactor method, companies use a standard Chinese hamster ovary cell, inject it with a drug’s DNA, and "grow" the drug in a giant "bath" of nutrients and oxygen. As the cells divide in the bath, they also produce the drug.

To produce 10 kilograms of biologic drugs using a bioreactor requires about $500 million of machinery, Newberry said. "We can build to the same capacity using goats for $50 million," he said. "Our technology, if large scale, has the ability to change the way (biologic) drugs are produced."

GTC’s method is less expensive because it doesn’t require new bioreactors to produce more of the same drugs. All the company has to do is breed more transgenic goats. The company can produce 2 to 10 grams of the drug per liter of milk, and each goat typically produces slightly more than 2 liters of milk per day, he said.

The transgenic goat technology can also be used to produce larger proteins [they meant to say “difficult-to-express proteins”], which means that GTC’s method may unlock the potential to produce drugs to treat a larger array of human diseases and disorders.

In GTC’s method, described by Newberry, the company combines the genetic "instructions" for making the desired drug with the genes inside a goat that make proteins in the animal’s milk. The company microinjects the so-called "transgene" into a goat egg, then grows the egg into a "founder" animal inside a mother goat.

When the founder doe has its own kids, not only will they become living bioreactors, but also the mother will produce milk that contains the desired protein. [After some front-end processing,] the milk is frozen and sent off to Cambrex Bio Science, Hopkinton Inc., where it is processed. The end product is a white powder that is combined with purified water in a hospital and injected into patients.

"First-time transgenic approval is a big deal," said Anthony Rotunno, vice president and general manager for Cambrex in Hopkinton. "It validates the technology."

Cambrex is in the business of purifying biologic drugs, and traditionally works with companies using bioreactors. Rotunno said it was the company’s intent to stay with GTC for the life of its product.

As far as the goats are concerned, "we have an ethical obligation to the animals," Newberry said. "We are here to make sure the goats have a good life."

The animals are based on 180 acres of developed and certified pharmaceutical farmland on a 300-acre parcel. They have veterinarians watching over them 100 percent of the time, Newberry said. They are given food and water that is checked and double-checked for purity.

Mike Schofield, a veterinarian specializing in goats, makes sure that the animals are healthy and monitors their pregnancies and live births.
"They’re normal animals in every other respect other than carrying that other piece of DNA," Schofield said. "We don’t see any effects other than the fact that they produce the proteins that we want."

The animals also have good lives, Newberry said. They have climbing rocks and salt licks and plenty of toys, ranging from balls hanging from the ceiling and rolling toys on the floor. They butt both toys with their heads when they’re tired of butting one another, Newberry said. "A lot of goats play king of the hill like 10-year-old boys," he said.

The goats, which come from cold mountain climates, actually prefer the winter, and have the opportunity to roam inside and outside of their barns. But they cannot roam off the farm. They’re effectively quarantined from the rest of the world by two sets of fences monitored at all times. The farm meets stringent requirements to ensure the goats are both clean and disease free.

They are tended by farm hands, who had traditionally worked with farm animals on agricultural farms. GTC has provided jobs for these people, who have lost work in the otherwise declining agricultural industry in central Massachusetts. Overall the farm employs 75 people, and the rest of GTC’s 132 employees are based in Framingham.

As part of its clinical research, GTC is looking into other uses of ATryn, such as the treatment of patients involved in trauma [i.e. sepsis]. The company also has several other projects in the works, including a human serum albumin, a malaria vaccine and an antibody to solid tumors [CD137].

In addition, GTC is producing two drugs for Merrimack Pharmaceuticals Inc. of Cambridge for use in its clinical program. Merrimack is involved in two studies, one for a product to treat rheumatoid arthritis [#msg-8163705] and another to treat psoriasis [#msg-10727170]. [Data will be available on the RA study any day.]

GTC owns several patents for its transgenic goat method, including a broad patent for the production of therapeutic proteins in the milk of any mammal in the United States [#msg-9174865].

"We’ve actually used this position not so much to keep people out, but to work with others and cross-license it," said Newberry. "Our position is to have more people use the technology," which, of course, will drive future revenue for the company.

The company has directly spent about $220 million to $230 million on the technology so far, which is actually a small amount compared to other drug development processes which can cost up to $800 million, Newberry said.

"Part of it, I’d like to think, is intelligent management," he said. "We’ve (also) defrayed the development cost by working with partners over the years."

But since the company has been "consuming cash" thus far, it saw a $30.1 million loss in the fiscal year ended in January 2006. It had a $29.5 million loss the prior year.

"We salute what GTC has done," said Stephen Mulloney, director of public policy for the Cambridge-based Massachusetts Biotechnology Council. "Time will tell whether transgenic goats have a future in the industry, but I believe they do, and this is exciting news.

"The intent of GTC’s production is to make manufacturing easier, and perhaps cheaper," Mulloney continued. "It certainly is not a short cut. It’s another way the biotech industry is addressing the need to meet unmet medical needs."

Mulloney said GTC is one of 300 companies in Massachusetts directly engaged in biotech research, development and manufacturing. The company is also a longstanding member of the council, he said.
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