Pipex Pharmaceuticals Receives FDA Orphan Drug Designation for Oral TTM for Idiopathic Pulmonary Fibrosis
One-Year Phase I/II Clinical Trial in Refractory IPF Patients Already Completed by Leading IPF Clinical Research Group
May 12, 2008: 09:23 AM EST
Pipex Pharmaceuticals, a specialty pharmaceutical company developing innovative late-stage drug candidates for the treatment of neurologic and autoimmune diseases, announced today that it has received an Orphan Drug designation for oral tetrathiomolybdate (oral TTM) from the FDA for the treatment of Idiopathic Pulmonary Fibrosis (IPF), a deadly pulmonary disease with no FDA-approved therapy.
IPF is a fatal respiratory disease characterized by progressive loss of lung function due to extensive fibrosis of lung tissues that are essential for respiration and life. According to the Coalition for Pulmonary Fibrosis, IPF affects an estimated 128,000 patients in the U.S., including 48,000 new patients per year, and results in approximately 30,000 deaths in the U.S. annually, more than the number of U.S. deaths annually from either breast or prostate cancer. Currently, there are no FDA-approved therapies for IPF. The standard of care for IPF patients is high-dose corticosteroids and immunosuppressants, which have numerous side effects that increase patient morbidity.
Steve H. Kanzer, CPA, JD, Chairman and Chief Executive Officer of Pipex stated, "Oral TTM has completed a 12 month open label phase I/II clinical trial in refractory IPF patients the results of which were reported at the May 2007 Annual Meeting of the American Thoracic Society. Provided that we elect to file an IND for oral TTM and pursue the further clinical development of oral TTM for IPF, this orphan drug designation provides additional exclusivity that supports our exclusively licensed issued U.S. Patent 6,855,340 and pending international patents covering the use of oral TTM for IPF and other high value fibrotic indications. The highly successful Orphan Drug Act of 1983 (as amended) would now provide our potential pharmaceutical partners or acquirors with a 50% tax credit for Phase III oral TTM IPF clinical trial expenses, for example, thereby reducing the after-tax cost of any such additional clinical trials to as little as 27%."
The United States Orphan Drug Act of 1983 was created to provide incentives for companies to develop and market treatments for diseases or conditions affecting fewer than 200,000 people in the United States. The Orphan Drug designation provides eligibility for a seven-year period of market exclusivity in the United States after product approval, an accelerated review process, grant funding, tax benefits, and an exemption from user fees.
About Oral TTM in Fibrotic Diseases
Oral TTM is a small-molecule, anti-copper agent that is highly specific for the reduction of free copper in serum, the most toxic form of copper in the body, and is thus suited for the treatment of central nervous system (CNS) diseases in which abnormal serum and CNS copper homeostasis are implicated. Pipex is developing oral TTM for fibrotic disorders based upon the rationale that the fibrotic disease process is dependent upon the availability of free copper in the body. Oral TTM has demonstrated the ability to inhibit fibrosis in a number of well established animal models through the sequestration of available copper and inhibition of key fibrotric cytokines, including secreted protein acid rich in cysteine (SPARC), NFkB, TGF-B, FGF-2, IL-1, IL-6, IL-8, and connective tissue growth factor (CTGF).
About Pipex Pharmaceuticals, Inc.
Pipex Pharmaceuticals, Inc. ("Pipex") is a specialty pharmaceutical company that is developing four proprietary, late-stage drug candidates for the treatment of neurologic and fibrotic diseases. Pipex's strategy is to exclusively in-license proprietary, clinical-stage drug candidates and to complete the further clinical testing, manufacturing and regulatory requirements sufficient to seek marketing authorizations via the filing of New Drug Applications (NDAs). Pipex's drug candidates address the following market opportunities, Multiple Sclerosis (MS), Dry Age-Related Macular Degeneration (AMD), Fibromyalgia, Huntington's Disease, Alzheimer's Disease, neurologic Wilson's disease and Idiopathic Pulmonary Fibrosis. For further information please visit www.pipexinc.com.
This release contains forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, including statements regarding Pipex's plans for its product candidates. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "targets," "likely," "will," "would," "should," and "could," and similar expressions or words identify forward-looking statements. Such forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Pipex is at an early stage of development and may not ever have any products that generate significant revenue. Important factors that could cause actual results to differ materially from those reflected in Pipex's forward-looking statements include, among others, a failure of Pipex's product candidates to be demonstrably safe and effective, a failure to obtain regulatory approval for the company's products or to comply with ongoing regulatory requirements, a lack of acceptance of Pipex's product candidates in the marketplace, a failure of the company to become or remain profitable, Pipex's inability to obtain the capital necessary to fund its research and development activities, a loss of any of the company's key scientists or management personnel, and other factors described in Pipex's report on Form 10-K for the year ended December 31, 2007. No forward-looking statements can be guaranteed and actual results may differ materially from such statements. The information in this release is provided only as of the date of this release, and Pipex undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.