Healthcare Industry News:  hemophilia A 

Biopharmaceuticals Licensing

 News Release - March 22, 2007

Pain Therapeutics Licenses Biotechnology To Treat Rare Disorder

Technology Developed at Stanford University Expected To Reach Clinic in 2008

SOUTH SAN FRANCISCO, Calif., March 22 (HSMN NewsFeed) -- Pain Therapeutics, Inc. (Nasdaq: PTIE ) announced today it has licensed new technology to treat hemophilia, a rare blood disorder. The technology, originally developed by researchers at Stanford University School of Medicine, is expected to enter the clinic in 2008.

"This licensing is part of our strategy to develop novel drug candidates in hematology/oncology, while maintaining a business model that requires a modest outlay of cash," said Remi Barbier, president and chief executive officer of Pain Therapeutics.

"Over the next 12 months, we plan to develop a novel drug candidate that specifically targets a key mechanism underlying hemophilia," added Nadav Friedmann, PhD, MD, chief operating and medical officer of Pain Therapeutics. "Basic research will continue to be conducted at Stanford, while we focus solely on the initiation of a clinical program."

Pain Therapeutics continues to expect its net cash requirements for 2007 to be approximately $10 million, inclusive of program expenses related to hemophilia. At December 31, 2006 the Company's cash, cash requirements and marketable securities were $204.4 million.

About Hemophilia

Hemophilia is a rare, inherited blood clotting disorder. It affects approximately 400,000 people worldwide. People with hemophilia Are deficient in the key proteins-Factor VIII (hemophilia A) or Factor IX (hemophilia B)- that are vital to prevent uncontrolled bleeding. Both forms of hemophilia Are characterized by spontaneous or prolonged bleeding, typically into patients' joints and soft tissue. Patients with hemophilia A or B are dependent on protein replacement therapy with Factor VIII or Factor IX, respectively, for life. In the U.S., the cost of protein replacement therapy can easily exceed $100,000 per patient per year. Outside of Western countries, many patients with hemophilia lack access to protein replacement therapy; left untreated, they have a short life expectancy.

About The New Technology

As an alternative to replacement therapy, Michele P. Calos, PhD, associate professor of genetics at Stanford University School of Medicine, has developed a novel gene integration technology. Her patented technology proposes to replace daily or weekly protein replacement therapy with a single, lasting procedure that promotes normal blood coagulation. The key is to insert a healthy copy of the human gene for Factor IX, a protein responsible for blood clotting, into the cells of a hemophilia patient. If this method promotes robust, persistent and normal levels of Factor IX, patients may be cured of hemophilia. Importantly, this method of gene integration does not rely on viral vectors. Instead, Dr. Calos has demonstrated that a bacteriophage integration system provides a precise method for directing and inserting a gene of interest into chromosomes.

In a pre-clinical experiment published in Nature Biotechnology, this method of gene integration was shown to be highly site-specific. It also resulted in robust, persistent and normal levels of Factor IX in mice. Furthermore, protein expression levels remained stable throughout the eight months of the experiment. Full details of the experiment are available in a publication titled, "Site-specific Genomic Integration Produces Therapeutic Factor IX Levels in Mice" (Nature Biotechnology 20, 1124-1128 (2002)).

In 2002, Dr. Calos co-founded Poetic Genetics, Inc. to broadly commercialize her technology. Under the terms of a license agreement between Pain Therapeutics and Poetic Genetics, Poetic was paid an undisclosed upfront fee and is eligible to receive milestone payments totaling $4 million in the aggregate, based on clinical and regulatory progress, and a 4% royalty on net sales (or 6% if the first U.S. sale of a licensed product occurs before January 1, 2011). In exchange, Pain Therapeutics was granted exclusive, worldwide commercial rights to the technology in all indications in hemophilia And pain management.

About Pain Therapeutics, Inc.

Pain Therapeutics is a biopharmaceutical company that develops novel drugs for pain management and hematology/oncology. We have three investigational drug candidates in clinical programs. Remoxy(TM) and PTI-202 are proprietary, abuse-resistant forms of opioid drugs. Oxytrex(TM) is a novel, next-generation painkiller that potentially offers less physical dependence than currently marketed opioid painkillers. We are also developing a novel radio-labeled monoclonal antibody to treat metastatic melanoma, a rare but deadly form of skin cancer. The FDA has not yet evaluated the merits, safety or efficacy of our drug candidates. For more information, please visit our website at

Note Regarding Forward-Looking Statements: This press release contains forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995 (the "Act"). Pain Therapeutics disclaims any intent or obligation to update these forward-looking statements, and claims the protection of the Safe Harbor for forward-looking statements contained in the Act. Examples of such statements include, but are not limited to, any statements relating to the timing, scope or expected outcome of the Company's clinical development of its drug candidates, including its novel technology for the treatment of hemophilia, the potential benefits of the Company's drug candidates, including hemophilia, the Company's plan to initiate a clinical program in hemophilia in 2008, the Company's expected net cash requirements in 2007 and the size of the potential market for the Company's products. Such statements are based on management's current expectations, but actual results may differ materially due to various factors. Such statements involve risks and uncertainties, including, but not limited to, those risks and uncertainties relating to difficulties or delays in development and testing of the Company's drug candidates, unexpected adverse side effects or inadequate therapeutic efficacy of the Company's drug candidates (including the risk that current and past results of clinical trials are not necessarily indicative of future results of clinical trials), the uncertainty of patent protection for the Company's intellectual property or trade secrets and unanticipated research and development and other costs. For further information regarding these and other risks related to the Company's business, investors should consult the Company's filings with the Securities and Exchange Commission.

Source: Pain Therapeutics

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