GTCB news..America's First Transgenic Drug - Just A Step Away From Approval
2/3/2009 6:13 AM ET
The first drug to be produced from genetically engineered animals appears very close to becoming real in the United States.
On February 7, the FDA is slated to decide, whether to approve GTC Biotherapeutics Inc.'s (GTC), anti-clotting medicine ATryn to treat patients with hereditary antithrombin deficiency, a rare clotting disorder, when they undergo surgery or face childbirth. The drug was granted orphan drug status by the FDA in 2007. If approved in the U.S., the drug is expected to generate sales of $40 million - $50 million over the next four to five years.
ATryn, which is made from the milk of genetically engineered goats, has already won the backing of the regulatory agency's Blood Products Advisory Committee. The panel has also voted ATryn as "safe and efficacious to prevent and treat venous thromboembolism in hereditary antithrombin deficient patients facing surgery or childbirth". ATryn was approved in Europe in 2006 and the drug is marketed there by LEO Pharma.
According to statistics, a total of 60,000 people in the U.S. and about 80,000 people in Europe have hereditary antithrombin deficiency, or HD. People with hereditary antithrombin deficiency are at an increased risk for venous blood clots, including pulmonary embolism and deep vein thrombosis.
ATryn given in intravenous formulation is for use only during high-risk procedures such as surgery and childbirth, and it is not a replacement for blood thinners prescribed to counter the anti-thrombin deficiency under normal treatment plan.
Company Overview
GTC Biotherapeutics, as it is now known, was spun-off from Genzyme Corp. (GENZ) in 1993 under the name Genzyme Transgenics Corp. The company changed its name to GTC Biotherapeutics in 2002.
ATryn, the first recombinant antithrombin product, is extracted from the milk of goats, which have a human DNA (gene) inserted into them to make them able to produce the human antithrombin in their milk. Antithrombin is a protein that helps keep blood from clotting.
ATryn contains the active substance antithrombin alfa, a copy of the natural blood protein, and it works by blocking thrombin, one of the substances involved in blood coagulation. GTC has licensed ATryn to Ovation Pharmaceuticals Inc. to develop and market the drug in the U.S.
In addition to ATryn, GTC is developing additional recombinant forms of therapeutic proteins normally found in human blood plasma as well as monoclonal antibodies. According to GTC, these products have potential applications in hematology, oncology, and autoimmune diseases.
Pharming - A Quick Glance
In biological context, Pharming refers to the production of therapeutic human proteins in transgenic farm animals. Commonly used transgenic animals are pigs, cows, sheep, and goats. Transgenic animals refer to those animals whose genetic make-up is altered to include selected genes from other animals or species.
Synthetic human insulin produced in 1982 by Eli Lilly & Co. (LLY) is the first successful pharmaceutical product produced by employing transgenic technology. The synthetic human insulin was produced by genetically modified bacteria in bioreactors.
Goats As Bioreactors - Why?
Patients with blood clotting disorders who are facing childbirth or who have to undergo surgery have to stop taking blood thinners like Heparin or Warfarin several days before, as it will lead to excessive bleeding if the drugs are not discontinued. Such patients are given replacement anti-thrombin. Up till now, human blood plasma is the only source of replacement anti-thrombin.
According to Geoffrey Cox, CEO of GTC, it takes just 18 months to produce a lactating animal and in a single year one goat produces the equivalent of 90,000 blood collections. Goats have a gestation period of about 5 months and reach maturity in 1 year.
Moreover, production of recombinant proteins using transgenic livestock costs less than when it is produced in bacterial or mammalian cell culture bioreactor facilities or through cell lines.
Financial Metrics
Since its inception in 1993, GTC has been operating at a loss. For the third-quarter ended September 28, 2008 the company's net loss narrowed to $6.06 million or $0.06 per share from $8.39 million or $0.11 per share in the year-ago period. Revenue for the quarter rose to $2.93 million from $2.58 million in the comparable quarter a year before.
On December 23, 2008 the company completed a $15 million convertible debt financing from LFB Biotechnologies. The net proceeds at closing, after transaction costs and establishment of a restricted cash account, were approximately $10 million. Under the agreement with LFB, the convertible debt will mature on June 30, 2012, and will bear interest at an annual rate of 8%. LFB also has a warrant to purchase GTC common stock at $0.31 per share at any time after June 1, 2009.
January 21, 2009 - GTC, which received a Nasdaq warning about a possible delisting of its stock, regained compliance with the minimum market capitalization listing requirement. The company also has until at least July 20, 2009, to regain compliance with the minimum $1 bid price requirement for stay listed on the Nasdaq market. Since early February 2008, the stock has been languishing below $1. The company expects to build on its strengths with expected near term news regarding ATryn's regulatory review and its commercial launch in the U.S.
The stock closed Monday's trade at $0.85 on an above average volume of 2.23 million shares.
Closing Thoughts
The much-awaited FDA approval of ATryn for the rare hereditary antithrombin deficiency could facilitate FDA approval of the drug for more prevalent disorders. The drug, if approved could also open the door to several 'pharmed' drugs. The Netherlands based Pharming Group N.V. is another biotech company, which is developing medicines using milk from genetically engineered cows and rabbits.
The FDA usually follows the advice of its expert committees, though not mandatory. However, given the FDA's recent stringent drug approval standards, one has to wait and see what the future holds for the drug.
.... So moving Atryn from bench to bedside depends only on the decision of the FDA, which is expected to be announced on February 7. Stay tuned.
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