3SOF

WRAPUP 2-U.S. FDA staff backs CSL, GTC blood clot drugs
Wed Jan 7, 2009 12:25pm EST


WASHINGTON, Jan 7 (Reuters) - Two proposed therapies to treat rare blood disorders appear safe and effective for U.S. approval, including one made from genetically engineered goats, Food and Drug Administration staff reviewers said in documents released on Wednesday.

GTC Biotherapeutics Inc's (GTCB.O) anti-clotting drug Atryn, made from human proteins inserted into goat milk, and CSL Ltd's (CSL.AX) clot disorder treatment Riastap appeared to work in company studies, the FDA staff said in reports prepared for an advisory panel that meets on Friday.

Both products would need further study after they begin sales, the agency reviewers said.

On Friday, a panel of outside advisers will make a recommendation to the FDA on whether the two therapies are safe and effective. The agency usually takes their advice.

Shares of GTC soared 28 percent to 73 cents on the Nasdaq at midday on Wednesday.

The drug is GTC's first product to be submitted for approval in the United States and is part of a series of anti-clotting drugs that the Framingham, Massachusetts-based company is developing, officials said.

"This is an important tipping point for us," the company's chief executive, Geoff Cox, told Reuters on Tuesday. He added that Atryn, if approved, could bring in between $40 million and $50 million in the next four to five years.

Atryn treats hereditary antithrombin deficiency, in which irregular protein levels can cause excessive clotting. The treatment is made using a human protein produced in milk from genetically engineered goats.

While patients usually take traditional blood thinners such as warfarin, such drugs are not suitable for high-risk surgeries or giving birth, company officials have said.

In its review, the FDA said company data showed Atryn helped prevent clotting in patients and had an "acceptable" level of risks.

GTC is also developing further studies to test the drug in patients at risk for clots in non-hereditary conditions such as coronary bypass surgery, which if approved later by the FDA could also boost sales, Cox said.

To make Atryn, company scientists injected human DNA into goat cells to breed about 200 genetically modified goats at its Massachusetts facility. Females then express the human anti-clotting protein in their milk when they begin lactating after giving birth.

The drug is licensed to Ovation Pharmaceuticals Inc [OVAPH.UL] in the United States.

The other product under FDA review, Riastap, made by the company's CSL Behring unit, is intended for patients with congenital fibrinogen deficiency, which limits the blood's ability to clot.

Company research showed Riastap increased clot firmness, which was "sufficient" to support accelerated approval for drugs for serious diseases, FDA reviewers said. Accelerated approval requires further study after marketing to confirm the product provides meaningful benefits to patients.

The safety of Riastap, derived from human blood plasma, "appears to be acceptable," the FDA reviewers said. The drug is already sold in parts of Europe as Haemocomplettan P.

Australia-based CSL is the world's top maker of plasma products. CSL shares closed down nearly 3.3 percent in Australian trading before the FDA report was released.

Summaries for both drugs were released on the FDA's website at www.fda.gov/ohrms/dockets/ac/09/briefing/2009-4410B1-00-Index.html

Press Release Source: GTC Biotherapeutics, Inc.


FDA Advisory Committee Recommends GTC Biotherapeutics' ATryn* (antithrombin [Recombinant])
Friday January 9, 6:30 pm ET 
If approved, ATryn will be first recombinant human antithrombin available in the U.S.


ROCKVILLE, Md.--(BUSINESS WIRE)--GTC Biotherapeutics ("GTC", NASDAQ: GTCB) and OVATION Pharmaceuticals, Inc. announced today that the Blood Products Advisory Committee of the U.S. Food and Drug Administration (FDA) voted that ATryn is safe and efficacious for the prevention and treatment of venous thromboembolism in hereditary antithrombin deficient patients undergoing surgery or childbirth procedures. The FDA considers the recommendations of its Advisory Committees when making its determinations. If approved, ATryn will be the first recombinant human antithrombin available in the U.S.


"We are very pleased with the Advisory Committee's recommendation in support of the safety and efficacy of ATryn," said Geoffrey F. Cox, Ph.D., GTC's Chairman and Chief Executive Officer. "ATryn is the first transgenically produced therapeutic to achieve approval in Europe and undergo review by the FDA. ATryn is also a testament to our established strength in recombinant technology, and has the potential to provide an important new treatment option for patients with hereditary antithrombin deficiency."

In September 2008, the FDA assigned Priority Review to GTC's Biologic License Application, or BLA, for ATryn. Priority Review is granted to applications for products that, if approved, would provide significant advances in treatment or provide a treatment where no sufficient one already exists. Under Priority Review, the FDA's target date for action on the BLA is February 7, 2009. GTC has licensed ATryn to OVATION to develop and market it in the U.S.

"The committee's recommendation takes us a step closer to making ATryn available to people in the U.S. with hereditary antithrombin deficiency, a rare clotting disorder associated with severe complications for which there are few treatment options," said Jeffrey S. Aronin, OVATION President and Chief Executive Officer. "Consistent with our overall focus on addressing unmet medical needs of small patient populations, bringing ATryn to market would give us the opportunity to make a meaningful difference in the lives of people suffering from this rare disorder."

Antithrombin works as a natural anticoagulant in the human body by regulating thrombin, which plays an important role in controlling the formation of blood clots. ATryn was developed with the key objective to provide the purity, safety and consistency of an unlimited supply of recombinant antithrombin. Purified recombinant antithrombin has the same amino acid sequence as antithrombin derived from human plasma.

People with hereditary antithrombin deficiency are at increased risk for venous blood clots, including pulmonary embolism and deep vein thrombosis. The prevalence of hereditary antithrombin deficiency in the general population is approximately one in 2,000 to one in 3,000. Half these patients may experience a thrombosis before 25 years of age and based on one study, up to 85 percent may suffer a thromboembolic event by age 50.

About ATryn* (antithrombin [Recombinant])

ATryn is the first recombinant antithrombin product approved in the world and the first antithrombin product that has been approved through the centralized European Medicines Agency (EMEA) procedure in the European Union.

ATryn is under review by the FDA for the prevention and treatment of peripartum and perioperative thromboembolic events in patients with hereditary antithrombin deficiency.

The most common adverse events listed in the approved European labeling that may occur during ATryn treatment include dizziness, headache, bleeding, nausea, bleeding at injection site and increased bleeding during treatment. As with any intravenous protein product, allergic type hypersensitivity reactions are possible.

About GTC Biotherapeutics

GTC Biotherapeutics develops, supplies, and commercializes therapeutic proteins produced through transgenic animal technology. In addition to ATryn, GTC is developing a portfolio of recombinant human plasma proteins with known therapeutic properties. These proteins include recombinant forms of human coagulation factors VIIa, VIII, and IX, which are used for the treatment of hemophilia, and alpha-1 antitrypsin. GTC also has a monoclonal antibody portfolio that includes a monoclonal antibody to CD20 and a monoclonal antibody to CD137. GTC's intellectual property includes a patent in the United States through 2021 for the production of any therapeutic protein in the milk of any transgenic mammal. GTC's transgenic production platform is particularly well suited to enabling cost effective development of proteins that are difficult to express in traditional recombinant production systems as well as proteins that are required in large volumes. Additional information is available on the GTC web site, http://www.gtc-bio.com.

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including without limitation statements regarding the timing of the FDA's review of the BLA for ATryn. Such forward-looking statements are subject to a number of risks, uncertainties and other factors that could cause actual results to differ materially from future results expressed or implied by such statements. Factors that may cause such differences include, but are not limited to, the risks and uncertainties discussed in GTC's most recent Annual Report on Form 10-K and its other periodic reports filed with the Securities and Exchange Commission, including the uncertainties associated with dependence upon the actions of regulatory agencies.

GTC cautions investors not to place undue reliance on the forward-looking statements contained in this release. These statements speak only as of the date of this document, and GTC undertakes no obligation to update or revise the statements, except as may be required by law.

About OVATION Pharmaceuticals

OVATION is a fast growing biopharmaceutical company that develops and commercializes medically necessary therapies to satisfy unmet medical needs for patients with severe illnesses. Headquartered in Deerfield, Ill., with products available in more than 85 countries, OVATION is committed to having a significant impact on patients' lives through its focus on central nervous system (CNS), hematology/oncology, and hospital-based therapies. The three new launches the company expects over the next three years will be fueled largely by its late-stage CNS pipeline, which is one of the most robust in the industry. OVATION has been recognized for excellence in the global pharmaceutical and biotechnology industries with the 2006 and 2007 "Pharma Company of the Year" award from Scrip magazine for small to mid-sized enterprises. More information about the company, its products and full prescribing information may be found at www.ovationpharma.com.

* ATryn is a registered trademark of GTC Biotherapeutics




Contact:
GTC Biotherapeutics, Inc.
Thomas E. Newberry, 508-370-5374
Vice President, Corporate Communications and Government Relations
[email protected]
or
OVATION Pharmaceuticals, Inc.
Sally Benjamin Young, 847-727-3265
Vice President of Communications
[email protected]

--------------------------------------------------------------------------------
Source: GTC Biotherapeutics, Inc.

Things are shaping up for the better in this Small biotech.
This one hovers around a buck and dips below occasionally but not permanently. Once good news comes out, watch out above! 


52wk Range: 0.11 - 1.19
Volume: 2,782,484
Avg Vol (3m): 855,097

Technicals
Percentage Gainer

Last Price Quote is:
38.99%above 13-day MA
104.39%above 50-day MA
RS Rating: 97 

Fundamentals
Key Data:
Market Cap (M): $69.83 
P/E Ratio: NA 
PEG Ratio: N/A 
Next Earnings: 03/05/2009
Last Analyst Rating: Neutral

FRAMINGHAM, Mass., Jan 21, 2009 (BUSINESS WIRE) -- GTC Biotherapeutics, Inc. (GTCB, Trade) received notice from the Listing Qualifications Staff ("Staff") of The NASDAQ Stock Market indicating that GTC has regained compliance with the minimum market value of listed securities requirement for continued listing on The NASDAQ Capital Market, as set forth in Marketplace Rule 4310 (c)(3), by exceeding $35 million of market capitalization for 10 consecutive trading days. Based on NASDAQ's recent extension of the temporary suspension of Marketplace Rule 4450(a)(5), GTC has until at least July 20, 2009 to regain compliance with the minimum $1.00 bid price requirement for continued listing.

"We are pleased to have regained compliance with the minimum market capitalization listing requirement," stated Geoffrey F. Cox, GTC's Chairman of the Board and CEO. "We look forward to continuing to build on our strengths with expected near term news regarding ATryn's regulatory review and its commercial launch in the USA."

ATryn(R), GTC's recombinant form of human antithrombin, was recently recommended by the Blood Products Advisory Committee, or BPAC, to be safe and efficacious for the prevention and treatment of venous thromboembolism in hereditary antithrombin deficient patients undergoing surgery or childbirth procedures. The Food and Drug Administration, or FDA, instituted guidance last week regarding the review and approval of a New Animal Drug Application, or NADA, for animals that incorporate additional genetic information. GTC worked closely with FDA staff to present all relevant information and participated in all field inspections necessary to support the review of an NADA for the animals that produce recombinant human antithrombin in their milk. The FDA's preliminary review of this information was presented to the BPAC. Under the Priority Review process, the target action date for a decision by the FDA on the Biologics License Application for ATryn(R) is February 7, 2009. OVATION Pharmaceuticals, GTC's partner for development and commercialization of ATryn(R) in the United States, is planning for commercial launch of the product following FDA approval.

About GTC Biotherapeutics

GTC Biotherapeutics develops, supplies, and commercializes therapeutic proteins produced through transgenic animal technology. In addition to ATryn(R), GTC is developing a portfolio of recombinant human plasma proteins with known therapeutic properties. These proteins include recombinant forms of human coagulation factors VIIa, VIII, and IX, which are used for the treatment of hemophilia, and alpha-1 antitrypsin. GTC also has a monoclonal antibody portfolio that includes a monoclonal antibody to CD20 and a monoclonal antibody to CD137. GTC's intellectual property includes a patent in the United States through 2021 for the production of any therapeutic protein in the milk of any transgenic mammal. GTC's transgenic production platform is particularly well suited to enabling cost effective development of proteins that are difficult to express in traditional recombinant production systems as well as proteins that are required in large volumes. Additional information is available on the GTC web site, http://www.gtc-bio.com.

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including without limitation statements regarding the prospects for and timing of determinations by NASDAQ and the FDA, and the prospects for the commercial launch of ATryn(R). Such forward-looking statements are subject to a number of risks, uncertainties and other factors that could cause actual results to differ materially from future results expressed or implied by such statements. Factors that may cause such differences include, but are not limited to, the risks and uncertainties discussed in GTC's most recent Annual Report on Form 10-K and its other periodic reports filed with the Securities and Exchange Commission, including the risks and uncertainties associated with dependence upon the actions of NASDAQ and the FDA, as well as the actions of partners. GTC cautions investors not to place undue reliance on the forward-looking statements contained in this release. These statements speak only as of the date of this document, and GTC undertakes no obligation to update or revise the statements, except as may be required by law.

SOURCE: GTC Biotherapeutics, Inc.


 
GTC Biotherapeutics, Inc.   
Thomas E. Newberry, 508-370-5374   
Vice President, Corporate Communications & Government Relations   
[email protected] 

Copyright Business Wire 2009



52wk Range: 0.11 - 1.13
Volume: 3,005,262
Avg Vol (3m): 1,022,200

Technicals
Percentage Gainer

Last Price Quote is:
24.58%above 13-day MA
79.84%above 50-day MA
RS Rating: N/A 

Fundamentals
Key Data:
Market Cap (M): $84.38 
P/E Ratio: NA 
PEG Ratio: N/A 
Next Earnings: 03/05/2009
Last Analyst Rating: Neutral

Nice day...Milestones are within reach and positive PR's will bolster the PPS.
Buy GTCB for a nice percentage gain.
Things are shaping up for the better in this Small biotech.
Healthy consolidation after the dip...Back above the 200-Day MA

52wk Range: 0.11 - 0.95
Volume: 2,956,591
Avg Vol (3m): 648,284

Technicals
Percentage Gainer

Last Price Quote is:
46.17%above 13-day MA
47.90%above 50-day MA
RS Rating: 7 

Fundamentals
Earnings Expected on May 7

Key Data:
Market Cap (M): $44.86 
P/E Ratio: NA 
PEG Ratio: N/A 
Next Earnings: 05/07/2009
Last Analyst Rating: Neutral